Advanced Skin Cancer Was Once a Death Sentence. Immunotherapy Is Changing That
Nobel Prize for 'Landmark' Cancer Research Conducted at UC Berkeley
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New Cancer Care Dilemma: Patients Want Immunotherapy Even When Evidence Is Lacking
Medicare to Require Hospitals to Post Prices Online
Gene Therapy Was Boy's Last Chance to Stop Leukemia. And It Worked.
CRISPR-Cas9 Used to Uncover Immunotherapy-Resistant Genes
Hype Exceeds Evidence on Cancer Immunotherapy (Commentary)
Cuba Has a Lung Cancer Vaccine; Now U.S. Patients Will Test It
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Though the country's infrastructure and cars are stuck in the 1950s, its healthcare system is remarkably innovative.","credit":"\u003ca href=\"http://blog.muchoviaje.com/semana-santa-para-todos-los-gustos-y-bolsillos/\" target=\"_blank\">MuchoViaje\u003c/a>","description":null,"imgSizes":{"thumbnail":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-160x109.jpg","width":160,"height":109,"mimeType":"image/jpeg"},"medium":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-800x547.jpg","width":800,"height":547,"mimeType":"image/jpeg"},"medium_large":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-768x525.jpg","width":768,"height":525,"mimeType":"image/jpeg"},"large":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-1020x697.jpg","width":1020,"height":697,"mimeType":"image/jpeg"},"fd-sm":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-960x656.jpg","width":960,"height":656,"mimeType":"image/jpeg"},"post-thumbnail":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-672x372.jpg","width":672,"height":372,"mimeType":"image/jpeg"},"twentyfourteen-full-width":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-1024x576.jpg","width":1024,"height":576,"mimeType":"image/jpeg"},"xxsmall":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-240x164.jpg","width":240,"height":164,"mimeType":"image/jpeg"},"xsmall":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-375x256.jpg","width":375,"height":256,"mimeType":"image/jpeg"},"small":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-520x355.jpg","width":520,"height":355,"mimeType":"image/jpeg"},"guest-author-32":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-32x32.jpg","width":32,"height":32,"mimeType":"image/jpeg"},"guest-author-50":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-50x50.jpg","width":50,"height":50,"mimeType":"image/jpeg"},"guest-author-64":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-64x64.jpg","width":64,"height":64,"mimeType":"image/jpeg"},"guest-author-96":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-96x96.jpg","width":96,"height":96,"mimeType":"image/jpeg"},"guest-author-128":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-128x128.jpg","width":128,"height":128,"mimeType":"image/jpeg"},"detail":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res-150x150.jpg","width":150,"height":150,"mimeType":"image/jpeg"},"kqedFullSize":{"file":"https://ww2.kqed.org/app/uploads/sites/13/2016/11/Cuba_high-res.jpg","width":1024,"height":700}},"fetchFailed":false,"isLoading":false}},"audioPlayerReducer":{"postId":"stream_live"},"authorsReducer":{"byline_futureofyou_444705":{"type":"authors","id":"byline_futureofyou_444705","meta":{"override":true},"slug":"byline_futureofyou_444705","name":"David Keyton\u003cbr />Jim Heintz \u003cbr />The Associated Press","isLoading":false},"byline_futureofyou_443435":{"type":"authors","id":"byline_futureofyou_443435","meta":{"override":true},"slug":"byline_futureofyou_443435","name":"Michelle Andrews, NPR","isLoading":false},"byline_futureofyou_442280":{"type":"authors","id":"byline_futureofyou_442280","meta":{"override":true},"slug":"byline_futureofyou_442280","name":"Casey Ross\u003cbr />STAT","isLoading":false},"byline_futureofyou_440994":{"type":"authors","id":"byline_futureofyou_440994","meta":{"override":true},"slug":"byline_futureofyou_440994","name":"Ricardo Alonso-Zaldivar\u003cbr />The Associated Press","isLoading":false},"byline_futureofyou_434938":{"type":"authors","id":"byline_futureofyou_434938","meta":{"override":true},"slug":"byline_futureofyou_434938","name":"Barry Starr","isLoading":false},"byline_futureofyou_351698":{"type":"authors","id":"byline_futureofyou_351698","meta":{"override":true},"slug":"byline_futureofyou_351698","name":"Dr. Nathan Gay and Dr. Vinay Prasad\u003cbr />\u003ca href=\"https://www.statnews.com/\">STAT\u003c/a>","isLoading":false},"lindseyhoshaw":{"type":"authors","id":"5432","meta":{"index":"authors_1591205172","id":"5432","found":true},"name":"Lindsey Hoshaw","firstName":"Lindsey","lastName":"Hoshaw","slug":"lindseyhoshaw","email":"lhoshaw@kqed.org","display_author_email":false,"staff_mastheads":[],"title":"KQED Contributor","bio":"Lindsey Hoshaw is a former interactive producer for KQED Science. 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Her work is regularly rebroadcast on numerous NPR and PBS shows. She has won several regional Emmy awards, a regional and a national Edward R. Murrow award. The Association for Health Journalists awarded Lesley best beat coverage. The Society of Professional Journalists has recognized her reporting several times. The Society of Environmental Journalists spotlighted her ongoing coverage of California's historic drought. Before joining KQED in 2016, she covered food and sustainability for Capital Public Radio, the environment for Colorado Public Radio, and reported for both KUOW and KCTS9 in Seattle. When not hunched over her laptop Lesley enjoys skiing with her daughter, cycling with her partner or scheming their next globetrotting adventure. Before motherhood she relished dancing tango till sunrise. 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Immunotherapy Is Changing That","publishDate":1540882861,"format":"audio","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp>Most cancer patients are haunted by the same two questions:\u003c/p>\n\u003cp>\"Why me?\"\u003c/p>\n\u003caside class=\"pullquote alignright\">'If I had been diagnosed five years prior, who knows if I would be here.'\u003c/aside>\n\u003cp>\"Why now?\"\u003c/p>\n\u003cp>Not Ashley Walton.\u003c/p>\n\u003cp>Walton, 34, actually feels lucky advanced melanoma struck when it did.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>“If I had been diagnosed five years prior, who knows if I would be here,\" she says.\u003c/p>\n\u003cp>Stage 4 melanoma used to be a death sentence. The disease doesn’t respond to radiation or chemotherapy, and patients survived, on average, less than a year.\u003c/p>\n\u003cp>But over the last decade, doctors are successfully using a new approach, one significantly different than the treatment options available for the last 150 years.\u003c/p>\n\u003cp>Instead of burning or poisoning cancer cells, new medicines unleash the body's natural defenses to fight them.\u003c/p>\n\u003cp>This treatment is called immunotherapy.\u003c/p>\n\u003cfigure id=\"attachment_444546\" class=\"wp-caption aligncenter\" style=\"max-width: 1857px\">\u003cimg class=\"size-full wp-image-444546\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/09/IMG_6200-e1537470859372.jpg\" alt=\"\" width=\"1857\" height=\"1209\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372.jpg 1857w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-160x104.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-800x521.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-768x500.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-1020x664.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-1200x781.jpg 1200w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-1180x768.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-960x625.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-240x156.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-375x244.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-520x339.jpg 520w\" sizes=\"(max-width: 1857px) 100vw, 1857px\">\u003cfigcaption class=\"wp-caption-text\">Ashley Walton drinks fluid to prepare for magnetic resonance imaging (MRI) scan to check on the progress of her melanoma. \u003ccite>(Lesley McClurg/ KQED)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>\u003cstrong>Beating the Odds\u003c/strong>\u003c/p>\n\u003cp>When Walton was 26, she found a mole on the back of her hip.\u003c/p>\n\u003cp>\"It started morphing into this ugly, dark, bleeding thing,\" she says, grimacing. \"I just knew something was wrong.\"\u003c/p>\n\u003cp>Doctors surgically removed her tumor. But a couple of years later, she discovered a tiny lump in her abdomen. It felt like a popcorn kernel, and within a few weeks grew to the size of a walnut.\u003c/p>\n\u003cp>A biopsy revealed she had stage 4 melanoma.\u003c/p>\n\u003cp>Walton searched online for information about the disease. She recalls the moment she discovered the average survival rate: six to nine months.\u003c/p>\n\u003cp>\"I remember sort of losing my hearing, almost losing my vision to where I felt like I was in a tunnel,\" she says.\u003c/p>\n\u003cp>But when she consulted with her oncologist, Dr. Adil Daud of UC San Francisco, he had consoling news.\u003c/p>\n\u003cp>“He told me if there's any time to have a melanoma -- right now is a pretty good time to have it -- because there's a lot of stuff opening up to you,\" she says.\u003c/p>\n\u003cp>Dr. Daud was referring to immune checkpoint inhibitors, which he says are increasing survival rates by at least threefold. These cancer drugs help the immune system do what it’s supposed to -- fight pathogens.\u003c/p>\n\u003cp>Normally the immune system recognizes disease-causing organisms. But cancer cells are unusual because they go undetected as harmful. Immune checkpoint inhibitors make them visible for attack.\u003c/p>\n\u003cfigure id=\"attachment_444547\" class=\"wp-caption aligncenter\" style=\"max-width: 1728px\">\u003cimg class=\"size-full wp-image-444547\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/09/IMG_6291-e1537471184446.jpg\" alt=\"\" width=\"1728\" height=\"1166\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446.jpg 1728w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-160x108.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-800x540.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-768x518.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-1020x688.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-1200x810.jpg 1200w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-1180x796.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-960x648.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-240x162.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-375x253.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-520x351.jpg 520w\" sizes=\"(max-width: 1728px) 100vw, 1728px\">\u003cfigcaption class=\"wp-caption-text\">Ashley Walton and her mother in a quarterly doctor appointment at UCSF Medical Center. \u003ccite>(Lesley McClurg/ KQED)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>\u003cb>Treatment Earns Nobel Prize\u003c/b>\u003c/p>\n\u003cp>The Food and Drug Administration’s approval of the first immune checkpoint inhibitor in 2011 marked a breakthrough in the field.\u003c/p>\n\u003cp>The technology behind Ipilimumab, which is branded as Yervoy, was developed in a UC Berkeley lab in the 1990s, by a student named Matthew Krummel, now an immunotherapy researcher at \u003ca href=\"http://krummellab.com/\" target=\"_blank\" rel=\"noopener\">UCSF\u003c/a>.\u003c/p>\n\u003cp>“I was a very frustrated graduate student for a few years, trying to develop an antibody that would do something,\" Krummel says.\u003c/p>\n\u003cp>He wanted to influence how cells behave. After many long nights, Krummel noticed one antibody successfully manipulating the movement of immune cells.\u003c/p>\n\u003cp>“You can drive them like a car; you can accelerate them; or you can brake them,\" Krummel says. \"And then it was really like playtime.”\u003c/p>\n\u003cp>He injected the antibodies into mice with cancer. In the very first set of experiments, their tumors shrunk.\u003c/p>\n\u003cp>Earlier this month, Krummel’s thesis advisor, James Allison, won the Nobel Prize for Medicine for their lab's work on immune checkpoint inhibitors.\u003c/p>\n\u003cp>\u003cstrong>Years of Harrowing Treatments\u003c/strong>\u003c/p>\n\u003caside class=\"pullquote alignright\">'Across different tumor types, only about 20 percent of patients with cancer respond to today's immunotherapy.'\u003ccite>Dr. Adil Daud, UCSF\u003c/cite>\u003c/aside>\n\u003cp>When Walton started immunotherapy treatment, including Ipilimumab, the 90-minute drips were followed by side effects like fever, diarrhea, rash, vomiting and gastritis. This is not uncommon in immunotherapy, as the drugs can put a patient’s immune system into overdrive provoking an attack on healthy cells, tissues and organs.\u003c/p>\n\u003cp>Walton’s tumors initially shrunk, but within six months, new tumors cropped up, in her abdomen. Her oncologist then ran through the list of available drugs, moving on as each one, sometimes tried in combination, in turn failed to deliver the knockout blow.\u003c/p>\n\u003cp>“It's absolutely exhausting emotionally and physically to know that there's no option for you,” says Walton. “Those were the days when I felt like I will probably die from this, and I’ll die young.”\u003c/p>\n\u003cp>Finally, eight years later, she heard the magic word: remission. Dr. Daud isn't sure if it was the buildup of multiple immunotherapy drugs or some new combination that did the trick.\u003c/p>\n\u003cp>“There are so many advancements being made in the field of immunotherapy that even if [one] doesn't cure you, it gets you to the next big thing,” says Walton.\u003c/p>\n\u003cp>Now that she has been off immunotherapy for 10 months, Walton tentatively asked Dr. Daud something that had been her mind for a long time.\u003c/p>\n\u003cp>\"So, what do you think about pregnancy or trying to start a family?\" asked Walton.\u003c/p>\n\u003cp>\"I think this is a good time to get pregnant, actually,\" responded Dr. Daud.\u003c/p>\n\u003cp>Both doctor and patient could barely contain their glee, punctuating the question-and-answer session with happy giggles. Walton \u003cspan style=\"font-weight: 400\">crosses her fingers and smiles. \u003c/span>Throughout her treatment, doctors warned that getting pregnant would be too dangerous. But Daud now trusts her body's ability to support a child.\u003c/p>\n\u003cp>\u003cstrong>A Hopeful Future\u003c/strong>\u003c/p>\n\u003cp>Stories like Walton's are sparking a lot of excitement among oncologists.\u003c/p>\n\u003cp>\"So imagine when we’ve gone from a time when we had nothing to offer, to today, and they are talking about a cure for some patients with advanced melanoma,\" says Dr. Leonard Lichtenfeld, deputy chief medical officer for the American Cancer Society.\u003c/p>\n\u003cp>Scientists and major pharmaceutical companies are waging huge bets on immunotherapy. There are currently about 1,000 active trials to develop the next miracle drug. Lichtenfeld is optimistic, but he is also cautious about adding to the hype.\u003c/p>\n\u003cp>“We don’t know in how many cancers they are going to be effective,” he says. “And we still don’t know how to harness the maximum benefit from these drugs by, say, using them in combination with other drugs.”\u003c/p>\n\u003cp>Currently, 40 percent of advanced melanoma patients still do not respond to immunotherapy. The statistics are even worse for other cancers.\u003c/p>\n\u003cp>“It is still very common to not have immune treatment work at all,” says Dr. Daud. “Across different tumor types, only about 20 percent of patients with cancer respond to today's immunotherapy, so the numbers are much more sobering.\"\u003c/p>\n\u003cp>He hypothesizes that age, gender, concurring autoimmune diseases, or even gut bacteria may influence patient responsiveness, but he says we know very little about these factors.\u003c/p>\n\u003cp>“I think there's a lot of fundamental questions about the immune system that we just simply don't know the answer to,” says Daud.\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>“And yet I do foresee a day when we use other types of treatment infrequently to treat cancer, and most cancers will be treated with immunotherapy. But we still have a long ways to go.”\u003c/p>\n\n","blocks":[],"excerpt":"'If I had been diagnosed five years prior, who knows if I would be here,' says Ashley Walton, who after eight draining years of treatment has finally heard the magic word: remission.","status":"publish","parent":0,"modified":1540998273,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":51,"wordCount":1224},"headData":{"title":"Advanced Skin Cancer Was Once a Death Sentence. Immunotherapy Is Changing That | KQED","description":"'If I had been diagnosed five years prior, who knows if I would be here,' says Ashley Walton, who after eight draining years of treatment has finally heard the magic word: remission.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Advanced Skin Cancer Was Once a Death Sentence. Immunotherapy Is Changing That","datePublished":"2018-10-30T07:01:01.000Z","dateModified":"2018-10-31T15:04:33.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"444527 https://ww2.kqed.org/futureofyou/?p=444527","disqusUrl":"https://ww2.kqed.org/futureofyou/2018/10/30/advanced-skin-cancer-was-once-a-death-sentence-immunotherapy-is-changing-that/","disqusTitle":"Advanced Skin Cancer Was Once a Death Sentence. Immunotherapy Is Changing That","audioUrl":"https://www.kqed.org/.stream/anon/radio/science/2018/10/McClurgImmuneTherapy.mp3","audioTrackLength":383,"path":"/futureofyou/444527/advanced-skin-cancer-was-once-a-death-sentence-immunotherapy-is-changing-that","parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Most cancer patients are haunted by the same two questions:\u003c/p>\n\u003cp>\"Why me?\"\u003c/p>\n\u003caside class=\"pullquote alignright\">'If I had been diagnosed five years prior, who knows if I would be here.'\u003c/aside>\n\u003cp>\"Why now?\"\u003c/p>\n\u003cp>Not Ashley Walton.\u003c/p>\n\u003cp>Walton, 34, actually feels lucky advanced melanoma struck when it did.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>“If I had been diagnosed five years prior, who knows if I would be here,\" she says.\u003c/p>\n\u003cp>Stage 4 melanoma used to be a death sentence. The disease doesn’t respond to radiation or chemotherapy, and patients survived, on average, less than a year.\u003c/p>\n\u003cp>But over the last decade, doctors are successfully using a new approach, one significantly different than the treatment options available for the last 150 years.\u003c/p>\n\u003cp>Instead of burning or poisoning cancer cells, new medicines unleash the body's natural defenses to fight them.\u003c/p>\n\u003cp>This treatment is called immunotherapy.\u003c/p>\n\u003cfigure id=\"attachment_444546\" class=\"wp-caption aligncenter\" style=\"max-width: 1857px\">\u003cimg class=\"size-full wp-image-444546\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/09/IMG_6200-e1537470859372.jpg\" alt=\"\" width=\"1857\" height=\"1209\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372.jpg 1857w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-160x104.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-800x521.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-768x500.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-1020x664.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-1200x781.jpg 1200w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-1180x768.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-960x625.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-240x156.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-375x244.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6200-e1537470859372-520x339.jpg 520w\" sizes=\"(max-width: 1857px) 100vw, 1857px\">\u003cfigcaption class=\"wp-caption-text\">Ashley Walton drinks fluid to prepare for magnetic resonance imaging (MRI) scan to check on the progress of her melanoma. \u003ccite>(Lesley McClurg/ KQED)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>\u003cstrong>Beating the Odds\u003c/strong>\u003c/p>\n\u003cp>When Walton was 26, she found a mole on the back of her hip.\u003c/p>\n\u003cp>\"It started morphing into this ugly, dark, bleeding thing,\" she says, grimacing. \"I just knew something was wrong.\"\u003c/p>\n\u003cp>Doctors surgically removed her tumor. But a couple of years later, she discovered a tiny lump in her abdomen. It felt like a popcorn kernel, and within a few weeks grew to the size of a walnut.\u003c/p>\n\u003cp>A biopsy revealed she had stage 4 melanoma.\u003c/p>\n\u003cp>Walton searched online for information about the disease. She recalls the moment she discovered the average survival rate: six to nine months.\u003c/p>\n\u003cp>\"I remember sort of losing my hearing, almost losing my vision to where I felt like I was in a tunnel,\" she says.\u003c/p>\n\u003cp>But when she consulted with her oncologist, Dr. Adil Daud of UC San Francisco, he had consoling news.\u003c/p>\n\u003cp>“He told me if there's any time to have a melanoma -- right now is a pretty good time to have it -- because there's a lot of stuff opening up to you,\" she says.\u003c/p>\n\u003cp>Dr. Daud was referring to immune checkpoint inhibitors, which he says are increasing survival rates by at least threefold. These cancer drugs help the immune system do what it’s supposed to -- fight pathogens.\u003c/p>\n\u003cp>Normally the immune system recognizes disease-causing organisms. But cancer cells are unusual because they go undetected as harmful. Immune checkpoint inhibitors make them visible for attack.\u003c/p>\n\u003cfigure id=\"attachment_444547\" class=\"wp-caption aligncenter\" style=\"max-width: 1728px\">\u003cimg class=\"size-full wp-image-444547\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/09/IMG_6291-e1537471184446.jpg\" alt=\"\" width=\"1728\" height=\"1166\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446.jpg 1728w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-160x108.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-800x540.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-768x518.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-1020x688.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-1200x810.jpg 1200w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-1180x796.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-960x648.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-240x162.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-375x253.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2018/09/IMG_6291-e1537471184446-520x351.jpg 520w\" sizes=\"(max-width: 1728px) 100vw, 1728px\">\u003cfigcaption class=\"wp-caption-text\">Ashley Walton and her mother in a quarterly doctor appointment at UCSF Medical Center. \u003ccite>(Lesley McClurg/ KQED)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>\u003cb>Treatment Earns Nobel Prize\u003c/b>\u003c/p>\n\u003cp>The Food and Drug Administration’s approval of the first immune checkpoint inhibitor in 2011 marked a breakthrough in the field.\u003c/p>\n\u003cp>The technology behind Ipilimumab, which is branded as Yervoy, was developed in a UC Berkeley lab in the 1990s, by a student named Matthew Krummel, now an immunotherapy researcher at \u003ca href=\"http://krummellab.com/\" target=\"_blank\" rel=\"noopener\">UCSF\u003c/a>.\u003c/p>\n\u003cp>“I was a very frustrated graduate student for a few years, trying to develop an antibody that would do something,\" Krummel says.\u003c/p>\n\u003cp>He wanted to influence how cells behave. After many long nights, Krummel noticed one antibody successfully manipulating the movement of immune cells.\u003c/p>\n\u003cp>“You can drive them like a car; you can accelerate them; or you can brake them,\" Krummel says. \"And then it was really like playtime.”\u003c/p>\n\u003cp>He injected the antibodies into mice with cancer. In the very first set of experiments, their tumors shrunk.\u003c/p>\n\u003cp>Earlier this month, Krummel’s thesis advisor, James Allison, won the Nobel Prize for Medicine for their lab's work on immune checkpoint inhibitors.\u003c/p>\n\u003cp>\u003cstrong>Years of Harrowing Treatments\u003c/strong>\u003c/p>\n\u003caside class=\"pullquote alignright\">'Across different tumor types, only about 20 percent of patients with cancer respond to today's immunotherapy.'\u003ccite>Dr. Adil Daud, UCSF\u003c/cite>\u003c/aside>\n\u003cp>When Walton started immunotherapy treatment, including Ipilimumab, the 90-minute drips were followed by side effects like fever, diarrhea, rash, vomiting and gastritis. This is not uncommon in immunotherapy, as the drugs can put a patient’s immune system into overdrive provoking an attack on healthy cells, tissues and organs.\u003c/p>\n\u003cp>Walton’s tumors initially shrunk, but within six months, new tumors cropped up, in her abdomen. Her oncologist then ran through the list of available drugs, moving on as each one, sometimes tried in combination, in turn failed to deliver the knockout blow.\u003c/p>\n\u003cp>“It's absolutely exhausting emotionally and physically to know that there's no option for you,” says Walton. “Those were the days when I felt like I will probably die from this, and I’ll die young.”\u003c/p>\n\u003cp>Finally, eight years later, she heard the magic word: remission. Dr. Daud isn't sure if it was the buildup of multiple immunotherapy drugs or some new combination that did the trick.\u003c/p>\n\u003cp>“There are so many advancements being made in the field of immunotherapy that even if [one] doesn't cure you, it gets you to the next big thing,” says Walton.\u003c/p>\n\u003cp>Now that she has been off immunotherapy for 10 months, Walton tentatively asked Dr. Daud something that had been her mind for a long time.\u003c/p>\n\u003cp>\"So, what do you think about pregnancy or trying to start a family?\" asked Walton.\u003c/p>\n\u003cp>\"I think this is a good time to get pregnant, actually,\" responded Dr. Daud.\u003c/p>\n\u003cp>Both doctor and patient could barely contain their glee, punctuating the question-and-answer session with happy giggles. Walton \u003cspan style=\"font-weight: 400\">crosses her fingers and smiles. \u003c/span>Throughout her treatment, doctors warned that getting pregnant would be too dangerous. But Daud now trusts her body's ability to support a child.\u003c/p>\n\u003cp>\u003cstrong>A Hopeful Future\u003c/strong>\u003c/p>\n\u003cp>Stories like Walton's are sparking a lot of excitement among oncologists.\u003c/p>\n\u003cp>\"So imagine when we’ve gone from a time when we had nothing to offer, to today, and they are talking about a cure for some patients with advanced melanoma,\" says Dr. Leonard Lichtenfeld, deputy chief medical officer for the American Cancer Society.\u003c/p>\n\u003cp>Scientists and major pharmaceutical companies are waging huge bets on immunotherapy. There are currently about 1,000 active trials to develop the next miracle drug. Lichtenfeld is optimistic, but he is also cautious about adding to the hype.\u003c/p>\n\u003cp>“We don’t know in how many cancers they are going to be effective,” he says. “And we still don’t know how to harness the maximum benefit from these drugs by, say, using them in combination with other drugs.”\u003c/p>\n\u003cp>Currently, 40 percent of advanced melanoma patients still do not respond to immunotherapy. The statistics are even worse for other cancers.\u003c/p>\n\u003cp>“It is still very common to not have immune treatment work at all,” says Dr. Daud. “Across different tumor types, only about 20 percent of patients with cancer respond to today's immunotherapy, so the numbers are much more sobering.\"\u003c/p>\n\u003cp>He hypothesizes that age, gender, concurring autoimmune diseases, or even gut bacteria may influence patient responsiveness, but he says we know very little about these factors.\u003c/p>\n\u003cp>“I think there's a lot of fundamental questions about the immune system that we just simply don't know the answer to,” says Daud.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"floatright"},"numeric":["floatright"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>“And yet I do foresee a day when we use other types of treatment infrequently to treat cancer, and most cancers will be treated with immunotherapy. But we still have a long ways to go.”\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/444527/advanced-skin-cancer-was-once-a-death-sentence-immunotherapy-is-changing-that","authors":["11229"],"categories":["futureofyou_1062","futureofyou_1","futureofyou_73"],"tags":["futureofyou_103","futureofyou_686","futureofyou_80"],"featImg":"futureofyou_444544","label":"futureofyou"},"futureofyou_444705":{"type":"posts","id":"futureofyou_444705","meta":{"index":"posts_1591205157","site":"futureofyou","id":"444705","score":null,"sort":[1538417110000]},"guestAuthors":[],"slug":"nobel-prize-for-landmark-cancer-research-conducted-at-berkeley","title":"Nobel Prize for 'Landmark' Cancer Research Conducted at UC Berkeley","publishDate":1538417110,"format":"aside","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>https://www.youtube.com/watch?time_continue=899&v=ScuTG1bzSHY\u003c/p>\n\u003cp>The Nobel Prize in medicine was awarded Monday to two researchers from the United States and Japan for advances in discovering how the body’s immune system can fight off the scourge of cancer.[contextly_sidebar id=\"CWuF65jUQjKNMEH73SFwE7Eh0o965day\"]\u003c/p>\n\u003cp>The $1.01 million prize will be shared by James Allison and Tasuku Honjo.\u003c/p>\n\u003cp>Allison conducted his research at the University of California-Berkeley and is now at the M.D. Anderson cancer center in Houston. Honju works at Japan’s Kyoto University.\u003c/p>\n\u003cp>Their parallel work concerned proteins that act as brakes on the body’s immune system and it constitutes “a landmark in our fight against cancer,” said a statement from the Nobel Assembly of Sweden’s Karolinska Institute, which selects winners of the annual prestigious award.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>Allison studied a known protein and developed the concept into a new treatment approach, while Honjo discovered a new protein that also operated as a brake on immune cells.\u003c/p>\n\u003cp>“I’m honored and humbled to receive this prestigious recognition,” Allison said in a statement released by the university’s MD Anderson Cancer Center in Houston, where he is a professor.\u003c/p>\n\u003cp>“A driving motivation for scientists is simply to push the frontiers of knowledge. I didn’t set out to study cancer, but to understand the biology of T cells, these incredible cells that travel our bodies and work to protect us,” he said.[contextly_sidebar id=\"TrEVYlEaRWGIwxl3wZCxQN7b50EP4tx2\"]\u003c/p>\n\u003cp>Allison takes care in his statement to give credit to “a succession of graduate students, postdoctoral fellows and colleagues at MD Anderson, the University of California, Berkeley, and Memorial Sloan Kettering Cancer Center” who joined in the research.\u003c/p>\n\u003cp>Allison’s and Honjo’s prize-winning work started in the 1990s and was part of significant advances in cancer immunotherapy.\u003c/p>\n\u003cp>“In some patients, this therapy is remarkably effective,” Jeremy Berg, editor-in-chief of the \u003cem>Science\u003c/em> family of journals, told The Associated Press. “The number of different types of cancers for which this approach to immunotherapy is being found to be effective in at least some patients continues to grow.”\u003c/p>\n\u003cp>Therapy developed from Honjo’s work led to long-term remission in patients with metastatic cancer that had been considered essentially untreatable, the Nobel Assembly said.[contextly_sidebar id=\"JxHmQ4y9gmGqrPe0YiiMCtrrGiOV1Oid\"]\u003c/p>\n\u003cp>Berg said that former President Jimmy Carter’s cancer, which had spread to his brain, was treated with one of the drugs developed from Honjo’s work.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>The physics prize is to be announced Tuesday, followed by chemistry. The winner of the Nobel Peace Prize will be named Friday and the economics laureate will be announced next Monday. No literature prize is being given this year.\u003c/p>\n\n","blocks":[],"excerpt":"The research, which has led to drugs that release the brakes on the immune system, constitutes “a landmark\" in the fight against cancer.","status":"publish","parent":0,"modified":1538445547,"stats":{"hasAudio":false,"hasVideo":true,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":15,"wordCount":471},"headData":{"title":"Nobel Prize for 'Landmark' Cancer Research Conducted at UC Berkeley | KQED","description":"The research, which has led to drugs that release the brakes on the immune system, constitutes “a landmark" in the fight against cancer.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Nobel Prize for 'Landmark' Cancer Research Conducted at UC Berkeley","datePublished":"2018-10-01T18:05:10.000Z","dateModified":"2018-10-02T01:59:07.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"444705 https://ww2.kqed.org/futureofyou/?p=444705","disqusUrl":"https://ww2.kqed.org/futureofyou/2018/10/01/nobel-prize-for-landmark-cancer-research-conducted-at-berkeley/","disqusTitle":"Nobel Prize for 'Landmark' Cancer Research Conducted at UC Berkeley","source":"Events","nprByline":"David Keyton\u003cbr />Jim Heintz \u003cbr />The Associated Press","path":"/futureofyou/444705/nobel-prize-for-landmark-cancer-research-conducted-at-berkeley","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\u003cp>\u003cspan class='utils-parseShortcode-shortcodes-__youtubeShortcode__embedYoutube'>\n \u003cspan class='utils-parseShortcode-shortcodes-__youtubeShortcode__embedYoutubeInside'>\n \u003ciframe\n loading='lazy'\n class='utils-parseShortcode-shortcodes-__youtubeShortcode__youtubePlayer'\n type='text/html'\n src='//www.youtube.com/embed/ScuTG1bzSHY'\n title='//www.youtube.com/embed/ScuTG1bzSHY'\n allowfullscreen='true'\n style='border:0;'>\u003c/iframe>\n \u003c/span>\n \u003c/span>\u003c/p>\u003cp>\u003cp>The Nobel Prize in medicine was awarded Monday to two researchers from the United States and Japan for advances in discovering how the body’s immune system can fight off the scourge of cancer.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>The $1.01 million prize will be shared by James Allison and Tasuku Honjo.\u003c/p>\n\u003cp>Allison conducted his research at the University of California-Berkeley and is now at the M.D. Anderson cancer center in Houston. Honju works at Japan’s Kyoto University.\u003c/p>\n\u003cp>Their parallel work concerned proteins that act as brakes on the body’s immune system and it constitutes “a landmark in our fight against cancer,” said a statement from the Nobel Assembly of Sweden’s Karolinska Institute, which selects winners of the annual prestigious award.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>Allison studied a known protein and developed the concept into a new treatment approach, while Honjo discovered a new protein that also operated as a brake on immune cells.\u003c/p>\n\u003cp>“I’m honored and humbled to receive this prestigious recognition,” Allison said in a statement released by the university’s MD Anderson Cancer Center in Houston, where he is a professor.\u003c/p>\n\u003cp>“A driving motivation for scientists is simply to push the frontiers of knowledge. I didn’t set out to study cancer, but to understand the biology of T cells, these incredible cells that travel our bodies and work to protect us,” he said.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>Allison takes care in his statement to give credit to “a succession of graduate students, postdoctoral fellows and colleagues at MD Anderson, the University of California, Berkeley, and Memorial Sloan Kettering Cancer Center” who joined in the research.\u003c/p>\n\u003cp>Allison’s and Honjo’s prize-winning work started in the 1990s and was part of significant advances in cancer immunotherapy.\u003c/p>\n\u003cp>“In some patients, this therapy is remarkably effective,” Jeremy Berg, editor-in-chief of the \u003cem>Science\u003c/em> family of journals, told The Associated Press. “The number of different types of cancers for which this approach to immunotherapy is being found to be effective in at least some patients continues to grow.”\u003c/p>\n\u003cp>Therapy developed from Honjo’s work led to long-term remission in patients with metastatic cancer that had been considered essentially untreatable, the Nobel Assembly said.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>Berg said that former President Jimmy Carter’s cancer, which had spread to his brain, was treated with one of the drugs developed from Honjo’s work.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>The physics prize is to be announced Tuesday, followed by chemistry. The winner of the Nobel Peace Prize will be named Friday and the economics laureate will be announced next Monday. No literature prize is being given this year.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/444705/nobel-prize-for-landmark-cancer-research-conducted-at-berkeley","authors":["byline_futureofyou_444705"],"categories":["futureofyou_1","futureofyou_73"],"tags":["futureofyou_103","futureofyou_686","futureofyou_80","futureofyou_607"],"collections":["futureofyou_1097"],"featImg":"futureofyou_444717","label":"source_futureofyou_444705"},"futureofyou_443435":{"type":"posts","id":"futureofyou_443435","meta":{"index":"posts_1591205157","site":"futureofyou","id":"443435","score":null,"sort":[1531861237000]},"guestAuthors":[],"slug":"insurers-and-government-are-slow-to-cover-expensive-car-t-cancer-therapy","title":"Insurers And Government Are Slow To Cover Expensive CAR-T Cancer Therapy","publishDate":1531861237,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>Patients whose blood cancers have failed to respond to repeated rounds of chemotherapy may be candidates for a new type of gene therapy that could send their cancers into remission for years. But the two approved therapies, with price tags of hundreds of thousands of dollars, have roiled the insurance approval process, leading to delays and, in some cases, denials of coverage, clinicians and analysts say.[contextly_sidebar id=\"ReV9Gv0J6j7ADmv2dqbEuegAVdiLN5dE\"]\u003c/p>\n\u003cp>The therapy involves collecting patients' own T cells, a type of white blood cell, genetically modifying them, and then infusing them back into patients, where they hunt down and kill cancer cells. Known as \u003ca href=\"https://www.cancer.gov/about-cancer/treatment/research/car-t-cells\" target=\"_blank\" rel=\"noopener\">CAR-T cell therapy\u003c/a>, it's been characterized as a \"living drug\" by some researchers.\u003c/p>\n\u003cp>Two different CAR-T drugs — \u003ca href=\"https://www.us.kymriah.com/diffuse-large-b-cell-lymphoma-adults/?site=KYDDAY0DTCBR0040&source=01030&gclid=CNDIg4PMpNwCFRj1swod-MwKyw&gclsrc=ds\" target=\"_blank\" rel=\"noopener\">Kymriah\u003c/a> and \u003ca href=\"https://www.yescarta.com/therapy#how-yescarta-is-different\" target=\"_blank\" rel=\"noopener\">Yescarta\u003c/a> — were approved by the FDA last year to treat patients whose blood cancers haven't responded to at least two other rounds of treatment.\u003c/p>\n\u003cp>Kymriah is \u003ca href=\"https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm574154.htm\">approved\u003c/a> for people up to age 25 with a form of acute lymphoblastic leukemia, the most common cancer in children. Kymriah and Yescarta are \u003ca href=\"https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm606540.htm\" target=\"_blank\" rel=\"noopener\">both\u003c/a> \u003ca href=\"https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm581296.htm\" target=\"_blank\" rel=\"noopener\">approved\u003c/a> for adults with advanced lymphomas.\u003c/p>\n\u003cp>Researchers report that some critically ill patients who received the therapy have remained cancer-free for as long as five years.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>\"This is what patients need,\" says \u003ca href=\"https://www.mayo.edu/research/faculty/lin-yi-m-d-ph-d/bio-00092684\" target=\"_blank\" rel=\"noopener\">Dr. Yi Lin\u003c/a>, a hematologist who oversees the CAR-T cell practice and research for the Mayo Clinic. \"With the likelihood of getting patients into durable survival, we don't want to deny them the therapy.\" She says she receives no personal financial support from the drugs' makers.[contextly_sidebar id=\"nsB5Luqi8Mrdsb9vLceD3T77zYYY4sKK\"]\u003c/p>\n\u003cp>But the treatment comes at a cost — the drug treatments are hugely expensive. Kymriah and Yescarta cost $373,000 for a one-time infusion to treat adults with advanced lymphomas, while Kymriah costs $475,000 to treat acute lymphoblastic leukemia in children and young adults. That's the cost of the drug itself; in addition, many patients experience serious side effects that can land them in a hospital intensive care unit for weeks, \u003ca href=\"https://khn.org/news/cascade-of-costs-could-push-new-gene-therapy-above-1-million-per-patient/\" target=\"_blank\" rel=\"noopener\">pushing treatment costs to more than $1 million\u003c/a>.\u003c/p>\n\u003cp>All of this gives government and private insurers pause.\u003c/p>\n\u003cp>Most commercial insurers are covering CAR-T cell therapies now, but they do so on an individual basis, writing single-patient agreements each time, say cancer specialists. Large insurers that are already familiar with complicated therapies like stem-cell transplants are getting speedier at handling requests for CAR-T cell treatment, they say. But that's not always the case at smaller or regional plans, where delays can add weeks to the approval process.\u003c/p>\n\u003cp>\"A request for CAR-T may end up with somebody on the payer authorization team who doesn't understand the technology or the urgency of the request, when somebody has only weeks or months to live,\" says \u003ca href=\"https://www.asbmt.org/about/contact-us\" target=\"_blank\" rel=\"noopener\">Stephanie Farnia\u003c/a>, director of health policy and strategic relations at the American Society for Blood and Marrow Transplantation.\u003c/p>\n\u003cp>Farnia is in contact with many of the more than 50 medical centers that are authorized to provide treatment. The process of getting to a treatment center and evaluated for therapy is involved, she says. \"To then be substantially delayed due to paperwork is incredibly frustrating\" for patients.[contextly_sidebar id=\"mCjXSziNW7nkG4M52QmA48lgOAVmv3eg\"]\u003c/p>\n\u003cp>Medicare and Medicaid often pose greater coverage challenges than do private insurers, according to insurance experts.\u003c/p>\n\u003cp>Some Medicaid programs don't cover the treatment, says \u003ca href=\"AndrewsCAR-TandInsuranceDFedit.docx\" target=\"_blank\" rel=\"noopener\">Dr. Michael Bishop\u003c/a>, director of the cellular therapy program in the hematology/oncology section at the University of Chicago. Medicaid, the state-federal health program, covers children in low-income households and some adults.\u003c/p>\n\u003cp>\"Medicaid has been very tough,\" he says. \"Certain states just deny coverage — even states with balanced budgets.\"\u003c/p>\n\u003cp>States \u003ca href=\"https://icer-review.org/wp-content/uploads/2017/07/ICER_CAR_T_Final_Evidence_Report_032318.pdf\" target=\"_blank\" rel=\"noopener\">have to evaluate the cost as well as the drugs' effectiveness\u003c/a>, says \u003ca href=\"http://medicaiddirectors.org/about/staff/\" target=\"_blank\" rel=\"noopener\">Matt Salo\u003c/a>, executive director of the National Association of Medicaid Directors.\u003c/p>\n\u003cp>\"Medicaid is a finite pot of money, and it's stretched threadbare even on a good day,\" he says.\u003c/p>\n\u003cp>People who are on Medicare, the health insurance program for people age 65 and older and some people with disabilities, typically haven't faced coverage denials to date, clinicians say. But the government's reimbursement rates are raising concerns for providers.\u003c/p>\n\u003cp>Last spring, Medicare announced payment rates for providers who administer Yescarta and Kymriah on an outpatient basis. The payments would more than cover the costs of the drugs. Medicare beneficiaries' out-of-pocket costs would be capped at $1,340 plus the beneficiaries' Part B deductible (if that hasn't already been met), the agency says.[contextly_sidebar id=\"zRKGtFwAO4CBGEIycv3lNE5oWVwoZuVf\"]\u003c/p>\n\u003cp>The problem with this plan? Facilities typically provide treatment on an inpatient basis, not outpatient, because of the potential for severe, systemic side effects.\u003c/p>\n\u003cp>\"There's a lot of toxicity and questions about whether it can even be provided in an outpatient setting,\" says Gary Goldstein, the business manager at the blood and marrow transplant program at Stanford Health Care in Stanford, Calif.\u003c/p>\n\u003cp>For inpatient care, \"CAR-T cell therapy ... would be paid at a much lower amount compared to outpatient hospital use,\" according to officials at the Centers for Medicare & Medicaid Services.\u003c/p>\n\u003cp>The agency is considering how to handle payment for inpatient CAR-T care for the fiscal year that starts in October. For now, some medical centers are absorbing whatever Medicare doesn't pay.\u003c/p>\n\u003cp>\"How can you tell a patient who's 66, 'If only you'd gotten lymphoma when you were 64'?\" Goldstein asks.\u003c/p>\n\u003cp>But the current approach can't continue indefinitely, he says.\u003c/p>\n\u003cp>\"Even if there aren't any centers that are making that decision today, if coverage doesn't change for Medicare, it absolutely is going to be a problem tomorrow,\" says Goldstein.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>\u003ca href=\"http://khn.org/\" target=\"_blank\" rel=\"noopener\">\u003cem>Kaiser Health News\u003c/em>\u003c/a>\u003cem>, a nonprofit news service, is an editorially independent program of the Kaiser Family Foundation, and is not affiliated with Kaiser Permanente. Michelle Andrews is on Twitter \u003c/em>\u003ca href=\"https://twitter.com/mandrews110\" target=\"_blank\" rel=\"noopener\">\u003cem>@mandrews110\u003c/em>\u003c/a>\u003cem>.\u003c/em>\u003c/p>\n\u003cdiv class=\"fullattribution\">Copyright 2018 Kaiser Health News. To see more, visit \u003ca href=\"http://www.kaiserhealthnews.org/\" target=\"_blank\" rel=\"noopener\">Kaiser Health News\u003c/a>.\u003cimg src=\"https://www.google-analytics.com/__utm.gif?utmac=UA-5828686-4&utmdt=Insurers+And+Government+Are+Slow+To+Cover+Expensive+CAR-T+Cancer+Therapy&utme=8(APIKey)9(MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004)\">\u003c/div>\n\n","blocks":[],"excerpt":"Treatment costs for the immunotherapy can run to more than $1 million. Some state Medicaid programs aren't paying for the treatment, and Medicare's complicated payment rates have hospitals worried.","status":"publish","parent":0,"modified":1531864937,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":28,"wordCount":987},"headData":{"title":"Insurers And Government Are Slow To Cover Expensive CAR-T Cancer Therapy | KQED","description":"Treatment costs for the immunotherapy can run to more than $1 million. Some state Medicaid programs aren't paying for the treatment, and Medicare's complicated payment rates have hospitals worried.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Insurers And Government Are Slow To Cover Expensive CAR-T Cancer Therapy","datePublished":"2018-07-17T21:00:37.000Z","dateModified":"2018-07-17T22:02:17.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"443435 https://ww2.kqed.org/futureofyou/?p=443435","disqusUrl":"https://ww2.kqed.org/futureofyou/2018/07/17/insurers-and-government-are-slow-to-cover-expensive-car-t-cancer-therapy/","disqusTitle":"Insurers And Government Are Slow To Cover Expensive CAR-T Cancer Therapy","source":"Health","nprImageCredit":"Fanatic Studio","nprByline":"Michelle Andrews, NPR","nprImageAgency":"Collection Mix: Subjects RF/Getty Images","nprStoryId":"629543151","nprApiLink":"http://api.npr.org/query?id=629543151&apiKey=MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004","nprHtmlLink":"https://www.npr.org/sections/health-shots/2018/07/17/629543151/insurers-and-government-are-slow-to-cover-expensive-car-t-cancer-therapy?ft=nprml&f=629543151","nprRetrievedStory":"1","nprPubDate":"Tue, 17 Jul 2018 15:36:00 -0400","nprStoryDate":"Tue, 17 Jul 2018 08:02:49 -0400","nprLastModifiedDate":"Tue, 17 Jul 2018 15:36:53 -0400","path":"/futureofyou/443435/insurers-and-government-are-slow-to-cover-expensive-car-t-cancer-therapy","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Patients whose blood cancers have failed to respond to repeated rounds of chemotherapy may be candidates for a new type of gene therapy that could send their cancers into remission for years. But the two approved therapies, with price tags of hundreds of thousands of dollars, have roiled the insurance approval process, leading to delays and, in some cases, denials of coverage, clinicians and analysts say.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>The therapy involves collecting patients' own T cells, a type of white blood cell, genetically modifying them, and then infusing them back into patients, where they hunt down and kill cancer cells. Known as \u003ca href=\"https://www.cancer.gov/about-cancer/treatment/research/car-t-cells\" target=\"_blank\" rel=\"noopener\">CAR-T cell therapy\u003c/a>, it's been characterized as a \"living drug\" by some researchers.\u003c/p>\n\u003cp>Two different CAR-T drugs — \u003ca href=\"https://www.us.kymriah.com/diffuse-large-b-cell-lymphoma-adults/?site=KYDDAY0DTCBR0040&source=01030&gclid=CNDIg4PMpNwCFRj1swod-MwKyw&gclsrc=ds\" target=\"_blank\" rel=\"noopener\">Kymriah\u003c/a> and \u003ca href=\"https://www.yescarta.com/therapy#how-yescarta-is-different\" target=\"_blank\" rel=\"noopener\">Yescarta\u003c/a> — were approved by the FDA last year to treat patients whose blood cancers haven't responded to at least two other rounds of treatment.\u003c/p>\n\u003cp>Kymriah is \u003ca href=\"https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm574154.htm\">approved\u003c/a> for people up to age 25 with a form of acute lymphoblastic leukemia, the most common cancer in children. Kymriah and Yescarta are \u003ca href=\"https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm606540.htm\" target=\"_blank\" rel=\"noopener\">both\u003c/a> \u003ca href=\"https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm581296.htm\" target=\"_blank\" rel=\"noopener\">approved\u003c/a> for adults with advanced lymphomas.\u003c/p>\n\u003cp>Researchers report that some critically ill patients who received the therapy have remained cancer-free for as long as five years.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>\"This is what patients need,\" says \u003ca href=\"https://www.mayo.edu/research/faculty/lin-yi-m-d-ph-d/bio-00092684\" target=\"_blank\" rel=\"noopener\">Dr. Yi Lin\u003c/a>, a hematologist who oversees the CAR-T cell practice and research for the Mayo Clinic. \"With the likelihood of getting patients into durable survival, we don't want to deny them the therapy.\" She says she receives no personal financial support from the drugs' makers.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>But the treatment comes at a cost — the drug treatments are hugely expensive. Kymriah and Yescarta cost $373,000 for a one-time infusion to treat adults with advanced lymphomas, while Kymriah costs $475,000 to treat acute lymphoblastic leukemia in children and young adults. That's the cost of the drug itself; in addition, many patients experience serious side effects that can land them in a hospital intensive care unit for weeks, \u003ca href=\"https://khn.org/news/cascade-of-costs-could-push-new-gene-therapy-above-1-million-per-patient/\" target=\"_blank\" rel=\"noopener\">pushing treatment costs to more than $1 million\u003c/a>.\u003c/p>\n\u003cp>All of this gives government and private insurers pause.\u003c/p>\n\u003cp>Most commercial insurers are covering CAR-T cell therapies now, but they do so on an individual basis, writing single-patient agreements each time, say cancer specialists. Large insurers that are already familiar with complicated therapies like stem-cell transplants are getting speedier at handling requests for CAR-T cell treatment, they say. But that's not always the case at smaller or regional plans, where delays can add weeks to the approval process.\u003c/p>\n\u003cp>\"A request for CAR-T may end up with somebody on the payer authorization team who doesn't understand the technology or the urgency of the request, when somebody has only weeks or months to live,\" says \u003ca href=\"https://www.asbmt.org/about/contact-us\" target=\"_blank\" rel=\"noopener\">Stephanie Farnia\u003c/a>, director of health policy and strategic relations at the American Society for Blood and Marrow Transplantation.\u003c/p>\n\u003cp>Farnia is in contact with many of the more than 50 medical centers that are authorized to provide treatment. The process of getting to a treatment center and evaluated for therapy is involved, she says. \"To then be substantially delayed due to paperwork is incredibly frustrating\" for patients.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>Medicare and Medicaid often pose greater coverage challenges than do private insurers, according to insurance experts.\u003c/p>\n\u003cp>Some Medicaid programs don't cover the treatment, says \u003ca href=\"AndrewsCAR-TandInsuranceDFedit.docx\" target=\"_blank\" rel=\"noopener\">Dr. Michael Bishop\u003c/a>, director of the cellular therapy program in the hematology/oncology section at the University of Chicago. Medicaid, the state-federal health program, covers children in low-income households and some adults.\u003c/p>\n\u003cp>\"Medicaid has been very tough,\" he says. \"Certain states just deny coverage — even states with balanced budgets.\"\u003c/p>\n\u003cp>States \u003ca href=\"https://icer-review.org/wp-content/uploads/2017/07/ICER_CAR_T_Final_Evidence_Report_032318.pdf\" target=\"_blank\" rel=\"noopener\">have to evaluate the cost as well as the drugs' effectiveness\u003c/a>, says \u003ca href=\"http://medicaiddirectors.org/about/staff/\" target=\"_blank\" rel=\"noopener\">Matt Salo\u003c/a>, executive director of the National Association of Medicaid Directors.\u003c/p>\n\u003cp>\"Medicaid is a finite pot of money, and it's stretched threadbare even on a good day,\" he says.\u003c/p>\n\u003cp>People who are on Medicare, the health insurance program for people age 65 and older and some people with disabilities, typically haven't faced coverage denials to date, clinicians say. But the government's reimbursement rates are raising concerns for providers.\u003c/p>\n\u003cp>Last spring, Medicare announced payment rates for providers who administer Yescarta and Kymriah on an outpatient basis. The payments would more than cover the costs of the drugs. Medicare beneficiaries' out-of-pocket costs would be capped at $1,340 plus the beneficiaries' Part B deductible (if that hasn't already been met), the agency says.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>The problem with this plan? Facilities typically provide treatment on an inpatient basis, not outpatient, because of the potential for severe, systemic side effects.\u003c/p>\n\u003cp>\"There's a lot of toxicity and questions about whether it can even be provided in an outpatient setting,\" says Gary Goldstein, the business manager at the blood and marrow transplant program at Stanford Health Care in Stanford, Calif.\u003c/p>\n\u003cp>For inpatient care, \"CAR-T cell therapy ... would be paid at a much lower amount compared to outpatient hospital use,\" according to officials at the Centers for Medicare & Medicaid Services.\u003c/p>\n\u003cp>The agency is considering how to handle payment for inpatient CAR-T care for the fiscal year that starts in October. For now, some medical centers are absorbing whatever Medicare doesn't pay.\u003c/p>\n\u003cp>\"How can you tell a patient who's 66, 'If only you'd gotten lymphoma when you were 64'?\" Goldstein asks.\u003c/p>\n\u003cp>But the current approach can't continue indefinitely, he says.\u003c/p>\n\u003cp>\"Even if there aren't any centers that are making that decision today, if coverage doesn't change for Medicare, it absolutely is going to be a problem tomorrow,\" says Goldstein.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>\u003ca href=\"http://khn.org/\" target=\"_blank\" rel=\"noopener\">\u003cem>Kaiser Health News\u003c/em>\u003c/a>\u003cem>, a nonprofit news service, is an editorially independent program of the Kaiser Family Foundation, and is not affiliated with Kaiser Permanente. Michelle Andrews is on Twitter \u003c/em>\u003ca href=\"https://twitter.com/mandrews110\" target=\"_blank\" rel=\"noopener\">\u003cem>@mandrews110\u003c/em>\u003c/a>\u003cem>.\u003c/em>\u003c/p>\n\u003cdiv class=\"fullattribution\">Copyright 2018 Kaiser Health News. To see more, visit \u003ca href=\"http://www.kaiserhealthnews.org/\" target=\"_blank\" rel=\"noopener\">Kaiser Health News\u003c/a>.\u003cimg src=\"https://www.google-analytics.com/__utm.gif?utmac=UA-5828686-4&utmdt=Insurers+And+Government+Are+Slow+To+Cover+Expensive+CAR-T+Cancer+Therapy&utme=8(APIKey)9(MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004)\">\u003c/div>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/443435/insurers-and-government-are-slow-to-cover-expensive-car-t-cancer-therapy","authors":["byline_futureofyou_443435"],"categories":["futureofyou_1062","futureofyou_1","futureofyou_73"],"tags":["futureofyou_103","futureofyou_1470","futureofyou_952","futureofyou_686","futureofyou_1029","futureofyou_35"],"collections":["futureofyou_1097"],"featImg":"futureofyou_443437","label":"source_futureofyou_443435"},"futureofyou_442280":{"type":"posts","id":"futureofyou_442280","meta":{"index":"posts_1591205157","site":"futureofyou","id":"442280","score":null,"sort":[1528139030000]},"guestAuthors":[],"slug":"new-cancer-care-dilemma-patients-want-immunotherapy-even-when-evidence-is-lacking","title":"New Cancer Care Dilemma: Patients Want Immunotherapy Even When Evidence Is Lacking","publishDate":1528139030,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp class=\"danger-zone\">Immunotherapy is a source of great hope in cancer care. It has \u003ca href=\"https://www.statnews.com/2017/09/12/cancer-immunotherapy-improvement/\">rescued\u003c/a>some patients from the brink, while giving others a reason to believe that they, too, could beat the long odds.[contextly_sidebar id=\"0iktmFOQroWtY1hQ3e3h8lY9q5AbDMyB\"]\u003c/p>\n\u003cp class=\"danger-zone\">But these therapies are also creating a vexing dilemma for doctors: Their patients, citing \u003ca href=\"https://www.ispot.tv/ad/w_y8/keytruda-its-tru-donnas-story-living-longer-is-possible\" target=\"_blank\" rel=\"noopener\">television ads\u003c/a> and media accounts of miraculous recoveries, are pushing hard to try them, even when there is little to no evidence the drugs will work for their particular cancer.\u003c/p>\n\u003cp class=\"danger-zone\">Doctors want to give their patients every shot at survival, but can they justify prescribing a drug when it hasn’t been tested for that patient’s type of cancer? Many of these treatments bring risks of painful — even life-threatening— side effects and carry total price tags pushing $1 million. In some cases, insurers won’t pay.\u003c/p>\n\u003cp class=\"\">“Whether it works or not, the burden both financially and emotionally on families and patients is massive,” said Dr. Vicki Jackson, chief of Massachusetts General Hospital’s palliative care unit, which helps patients with their decision-making process. “If you try it and it doesn’t work, then you’ve used up all your life savings.”\u003c/p>\n\u003cp class=\"\">There are few objective guideposts to determine which patients should get which treatments, or even undergo genetic testing to determine whether they are among the minority of patients who might benefit substantially from immuno-oncology medicines. The dividing line is often drawn by variations in age, tumor stage, and underlying health status — and the weight given to those measures still relies on physician discretion.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>“Is it ethically and morally appropriate to not offer these potentially curative options on the basis of a very slight difference in kidney or liver function?” asked Dr. Ephraim Hochberg, a Mass. General oncologist who specializes in lymphoma.\u003c/p>\n\u003cp>Precision medicine, and the use of genetic markers to predict the success of treatments, promises to help answer these questions. Scientists at Stanford, for example, \u003ca href=\"https://www.statnews.com/2018/05/14/cancer-immunotherapy-pet-predictor/\">engineered\u003c/a> a radioactive molecule, detectable by PET imaging, to track whether a patient’s T cells are activated and thus capable of fighting cancer if the patient is given immunotherapy.[contextly_sidebar id=\"FOtikbkxZiNfNj2OB2DLb5zd76Z6QV0k\"]\u003c/p>\n\u003cp>The technique worked in lab mice but has not yet been tried in human patients, and other such approaches are further off. Meanwhile, patients and doctors are left to make life and death treatment decisions on uncertain terrain.\u003c/p>\n\u003cp>This struggle plays out on a near-daily basis at Mass. General, a Harvard-affiliated academic medical center with a deep well of resources and expertise. STAT interviews with specialists at the hospital reveal both the complexity of the variables at play, and the difficulty of establishing clear treatment protocols when data on real-world outcomes is still lacking.\u003c/p>\n\u003cp>The extent of these difficulties is only magnified for oncologists working with fewer resources in smaller community hospitals, where the vast majority of the nation’s cancer patients receive their care.\u003c/p>\n\u003cp>The uncertainty over treatment eligibility primarily pertains to a new class of treatments that harness the power of a patient’s immune system to attack cancer cells. These drugs have reached the market fairly recently, and have been tested in patients with a limited number of cancer types. And even in those types, the \u003ca href=\"https://www.statnews.com/2017/03/08/immunotherapy-cancer-breakthrough/\">drugs work only some of the time\u003c/a>.\u003c/p>\n\u003cp>These treatments include the checkpoint inhibitors Keytruda and Opdivo, CAR-T drugs such as Yescarta and \u003ca href=\"https://www.statnews.com/2017/08/30/novartis-car-t-cancer-approved/\">Kymriah\u003c/a>, and neo-antigen vaccines.\u003c/p>\n\u003cp>Hochberg often deals with patients who want to try \u003ca href=\"https://www.statnews.com/2017/10/27/car-t-kite-cell-journey/\">CAR-T therapy\u003c/a>, which re-engineers a patient’s T cells to recognize molecules on cancer cells and attack them. CAR-T drugs cost between $375,000 and $475,000, and the administration of treatment and follow-up care adds hundreds of thousands of thousands of dollars to the total bill.\u003c/p>\n\u003cp>In theory, CAR-T therapies can be tried on older patients whose bodies cannot withstand chemotherapy and radiation, but the National Comprehensive Cancer Network, which publishes treatment guidelines, has not established detailed eligibility criteria.\u003c/p>\n\u003cp>A similar struggle is playing out over the use of checkpoint inhibitors. It is not always clear which patients should get tested for a rare genetic condition, known as mismatch repair syndrome, that indicates receptivity to these drugs. While the testing is routinely conducted in treatment for patients with uterine and colon cancers, it is not always done for patients with other types of cancer.\u003c/p>\n\u003cp>“The fundamental problem is that it’s a costly test, so people are reluctant to make it automatic,” said Dr. David Ryan, clinical director of Mass. General’s Cancer Center. “Right now we’re relying on the oncologist to pick up those patients where there’s a reasonable chance of having mismatch repair deficiency, and ordering that test from pathology. Everybody’s having a hard time figuring out how to do this.”[contextly_sidebar id=\"blUI6ansejyqkxbdlWCDpHI5OeCoOQ3E\"]\u003c/p>\n\u003cp>Insurers play a key role in making sure that sky-high spending for these drugs is based on evidence of efficacy. But their involvement cuts both ways. In some cases, they deny coverage for treatments even when they are recommended by oncologists who believe there is a strong scientific basis to back up their decisions.\u003c/p>\n\u003cp>That scenario arose recently for Dr. Zosia Piotrowska, a medical oncologist at Mass. General who specializes in treating lung cancer. She recommended a combination of targeted therapies for a 50-year-old mother of four who has struggled with metastatic lung cancer for five years.\u003c/p>\n\u003cp>The patient’s cancer is exceedingly rare and marked by a gene mutation that indicates greater receptivity to targeted therapies. Piotrowska said various combinations of treatments had allowed the patient to live a normal life for several years, but her insurer recently denied coverage for the new round of treatment, which comes with a price of $16,000 a month.\u003c/p>\n\u003cp>Piotrowska appealed the decision, but the insurer still denied coverage, citing a lack of support for the treatment option in National Comprehensive Cancer Network guidelines. Though such guidelines set forth detailed protocols, they do not address the myriad nuances of each type of cancer and rapid changes in treatment options.\u003c/p>\n\u003cp>“The guidelines can’t keep up with the pace of research,” Piotrowska said. “As we learn more and more and develop better treatments, certain patients fall into these loopholes where we understand the biology of what’s going on, we have a treatment, but you can’t explain it to the insurer.”\u003c/p>\n\u003cp>That denial of coverage means the patient must decide whether to pay out of pocket and drain family savings for an uncertain benefit. Piotrowska said this patient has enough resources to continue with treatment, but for the vast majority of patients, the price would mean certain financial ruin.\u003c/p>\n\u003cp>The advent of immune-based therapies is only making the decisions harder. For a \u003ca href=\"https://www.statnews.com/2017/03/08/immunotherapy-cancer-breakthrough/\">small percentage\u003c/a> of patients, the treatments can be a savior, but without comprehensive insurance coverage, they are guaranteed to lead to financial distress. And because there is not yet much data on how the patients who initially benefit are faring in the long run, it is nearly impossible for new patients and their families to assess costs and benefits.\u003c/p>\n\u003cp>Doctors said the confusion over who should get immune-based treatments is bound to be exacerbated by the passage last month of the controversial \u003ca href=\"https://www.statnews.com/2018/05/30/trump-signs-right-to-try/\">“right-to-try” legislation\u003c/a>, which aims to give patients with terminal illness a different pathway to try experimental medicines that haven’t yet been approved by the Food and Drug Administration. It will not be clear how the law will affect patients until regulations are clarified in the coming months.[contextly_sidebar id=\"uFgg9fWxQTxW5cFzzQ7kroOuopy28usR\"]\u003c/p>\n\u003caside class=\"read-more standard\">\n\u003cdiv class=\"read-more-text\">\u003c/div>\n\u003c/aside>\n\u003cp>In some ways, the pressures and uncertainties posed by new cancer treatments are good problems to have. Julie Guillot said she wishes she’d had the luxury of such challenges when her son was sick.\u003c/p>\n\u003cp>Zach was diagnosed with acute myeloid leukemia when he was 5. For a boy of that age, concerns about unclear clinical benefits of treatments and sky high costs do not apply in the same way they might for an older patient. All the calculations point to the same answer: Try everything you can.\u003c/p>\n\u003cp>Zach received heavy doses of chemotherapy and three bone marrow transplants during years of expensive treatment that left him suffering from uncontrollable fevers, nausea, and infections. He died in 2014.\u003c/p>\n\u003cp>Guillot now works as an \u003ca href=\"https://www.statnews.com/2017/02/20/car-t-therapy-critics-just-dont-get/\">advocate\u003c/a> to help other patients get access to the best treatments and to ensure that experimentation in cancer care can continue to proceed on its inevitably hard and messy course toward cures.\u003c/p>\n\u003cp>“If we are avoidant of new therapies because of the risk, the lack of data, or the cost, breakthrough therapies like CAR-T and bone marrow transplant would never be developed,” she said. “When you are faced with this, people are willing to take risks for a chance to live.”\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>\u003cem>This story was originally published by \u003ca href=\"https://www.statnews.com/2018/06/04/cancer-care-dilemma-immunotherapy/\" target=\"_blank\" rel=\"noopener\">STAT\u003c/a>, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery.\u003c/em>\u003c/p>\n\n","blocks":[],"excerpt":"Doctors want to give their patients every shot at survival, but can they justify prescribing a drug when it hasn’t been tested for that patient’s type of cancer? ","status":"publish","parent":0,"modified":1528133486,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":27,"wordCount":1545},"headData":{"title":"New Cancer Care Dilemma: Patients Want Immunotherapy Even When Evidence Is Lacking | KQED","description":"Doctors want to give their patients every shot at survival, but can they justify prescribing a drug when it hasn’t been tested for that patient’s type of cancer? ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"New Cancer Care Dilemma: Patients Want Immunotherapy Even When Evidence Is Lacking","datePublished":"2018-06-04T19:03:50.000Z","dateModified":"2018-06-04T17:31:26.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"442280 https://ww2.kqed.org/futureofyou/?p=442280","disqusUrl":"https://ww2.kqed.org/futureofyou/2018/06/04/new-cancer-care-dilemma-patients-want-immunotherapy-even-when-evidence-is-lacking/","disqusTitle":"New Cancer Care Dilemma: Patients Want Immunotherapy Even When Evidence Is Lacking","source":"Health","nprByline":"Casey Ross\u003cbr />STAT","path":"/futureofyou/442280/new-cancer-care-dilemma-patients-want-immunotherapy-even-when-evidence-is-lacking","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp class=\"danger-zone\">Immunotherapy is a source of great hope in cancer care. It has \u003ca href=\"https://www.statnews.com/2017/09/12/cancer-immunotherapy-improvement/\">rescued\u003c/a>some patients from the brink, while giving others a reason to believe that they, too, could beat the long odds.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp class=\"danger-zone\">But these therapies are also creating a vexing dilemma for doctors: Their patients, citing \u003ca href=\"https://www.ispot.tv/ad/w_y8/keytruda-its-tru-donnas-story-living-longer-is-possible\" target=\"_blank\" rel=\"noopener\">television ads\u003c/a> and media accounts of miraculous recoveries, are pushing hard to try them, even when there is little to no evidence the drugs will work for their particular cancer.\u003c/p>\n\u003cp class=\"danger-zone\">Doctors want to give their patients every shot at survival, but can they justify prescribing a drug when it hasn’t been tested for that patient’s type of cancer? Many of these treatments bring risks of painful — even life-threatening— side effects and carry total price tags pushing $1 million. In some cases, insurers won’t pay.\u003c/p>\n\u003cp class=\"\">“Whether it works or not, the burden both financially and emotionally on families and patients is massive,” said Dr. Vicki Jackson, chief of Massachusetts General Hospital’s palliative care unit, which helps patients with their decision-making process. “If you try it and it doesn’t work, then you’ve used up all your life savings.”\u003c/p>\n\u003cp class=\"\">There are few objective guideposts to determine which patients should get which treatments, or even undergo genetic testing to determine whether they are among the minority of patients who might benefit substantially from immuno-oncology medicines. The dividing line is often drawn by variations in age, tumor stage, and underlying health status — and the weight given to those measures still relies on physician discretion.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>“Is it ethically and morally appropriate to not offer these potentially curative options on the basis of a very slight difference in kidney or liver function?” asked Dr. Ephraim Hochberg, a Mass. General oncologist who specializes in lymphoma.\u003c/p>\n\u003cp>Precision medicine, and the use of genetic markers to predict the success of treatments, promises to help answer these questions. Scientists at Stanford, for example, \u003ca href=\"https://www.statnews.com/2018/05/14/cancer-immunotherapy-pet-predictor/\">engineered\u003c/a> a radioactive molecule, detectable by PET imaging, to track whether a patient’s T cells are activated and thus capable of fighting cancer if the patient is given immunotherapy.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>The technique worked in lab mice but has not yet been tried in human patients, and other such approaches are further off. Meanwhile, patients and doctors are left to make life and death treatment decisions on uncertain terrain.\u003c/p>\n\u003cp>This struggle plays out on a near-daily basis at Mass. General, a Harvard-affiliated academic medical center with a deep well of resources and expertise. STAT interviews with specialists at the hospital reveal both the complexity of the variables at play, and the difficulty of establishing clear treatment protocols when data on real-world outcomes is still lacking.\u003c/p>\n\u003cp>The extent of these difficulties is only magnified for oncologists working with fewer resources in smaller community hospitals, where the vast majority of the nation’s cancer patients receive their care.\u003c/p>\n\u003cp>The uncertainty over treatment eligibility primarily pertains to a new class of treatments that harness the power of a patient’s immune system to attack cancer cells. These drugs have reached the market fairly recently, and have been tested in patients with a limited number of cancer types. And even in those types, the \u003ca href=\"https://www.statnews.com/2017/03/08/immunotherapy-cancer-breakthrough/\">drugs work only some of the time\u003c/a>.\u003c/p>\n\u003cp>These treatments include the checkpoint inhibitors Keytruda and Opdivo, CAR-T drugs such as Yescarta and \u003ca href=\"https://www.statnews.com/2017/08/30/novartis-car-t-cancer-approved/\">Kymriah\u003c/a>, and neo-antigen vaccines.\u003c/p>\n\u003cp>Hochberg often deals with patients who want to try \u003ca href=\"https://www.statnews.com/2017/10/27/car-t-kite-cell-journey/\">CAR-T therapy\u003c/a>, which re-engineers a patient’s T cells to recognize molecules on cancer cells and attack them. CAR-T drugs cost between $375,000 and $475,000, and the administration of treatment and follow-up care adds hundreds of thousands of thousands of dollars to the total bill.\u003c/p>\n\u003cp>In theory, CAR-T therapies can be tried on older patients whose bodies cannot withstand chemotherapy and radiation, but the National Comprehensive Cancer Network, which publishes treatment guidelines, has not established detailed eligibility criteria.\u003c/p>\n\u003cp>A similar struggle is playing out over the use of checkpoint inhibitors. It is not always clear which patients should get tested for a rare genetic condition, known as mismatch repair syndrome, that indicates receptivity to these drugs. While the testing is routinely conducted in treatment for patients with uterine and colon cancers, it is not always done for patients with other types of cancer.\u003c/p>\n\u003cp>“The fundamental problem is that it’s a costly test, so people are reluctant to make it automatic,” said Dr. David Ryan, clinical director of Mass. General’s Cancer Center. “Right now we’re relying on the oncologist to pick up those patients where there’s a reasonable chance of having mismatch repair deficiency, and ordering that test from pathology. Everybody’s having a hard time figuring out how to do this.”\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>Insurers play a key role in making sure that sky-high spending for these drugs is based on evidence of efficacy. But their involvement cuts both ways. In some cases, they deny coverage for treatments even when they are recommended by oncologists who believe there is a strong scientific basis to back up their decisions.\u003c/p>\n\u003cp>That scenario arose recently for Dr. Zosia Piotrowska, a medical oncologist at Mass. General who specializes in treating lung cancer. She recommended a combination of targeted therapies for a 50-year-old mother of four who has struggled with metastatic lung cancer for five years.\u003c/p>\n\u003cp>The patient’s cancer is exceedingly rare and marked by a gene mutation that indicates greater receptivity to targeted therapies. Piotrowska said various combinations of treatments had allowed the patient to live a normal life for several years, but her insurer recently denied coverage for the new round of treatment, which comes with a price of $16,000 a month.\u003c/p>\n\u003cp>Piotrowska appealed the decision, but the insurer still denied coverage, citing a lack of support for the treatment option in National Comprehensive Cancer Network guidelines. Though such guidelines set forth detailed protocols, they do not address the myriad nuances of each type of cancer and rapid changes in treatment options.\u003c/p>\n\u003cp>“The guidelines can’t keep up with the pace of research,” Piotrowska said. “As we learn more and more and develop better treatments, certain patients fall into these loopholes where we understand the biology of what’s going on, we have a treatment, but you can’t explain it to the insurer.”\u003c/p>\n\u003cp>That denial of coverage means the patient must decide whether to pay out of pocket and drain family savings for an uncertain benefit. Piotrowska said this patient has enough resources to continue with treatment, but for the vast majority of patients, the price would mean certain financial ruin.\u003c/p>\n\u003cp>The advent of immune-based therapies is only making the decisions harder. For a \u003ca href=\"https://www.statnews.com/2017/03/08/immunotherapy-cancer-breakthrough/\">small percentage\u003c/a> of patients, the treatments can be a savior, but without comprehensive insurance coverage, they are guaranteed to lead to financial distress. And because there is not yet much data on how the patients who initially benefit are faring in the long run, it is nearly impossible for new patients and their families to assess costs and benefits.\u003c/p>\n\u003cp>Doctors said the confusion over who should get immune-based treatments is bound to be exacerbated by the passage last month of the controversial \u003ca href=\"https://www.statnews.com/2018/05/30/trump-signs-right-to-try/\">“right-to-try” legislation\u003c/a>, which aims to give patients with terminal illness a different pathway to try experimental medicines that haven’t yet been approved by the Food and Drug Administration. It will not be clear how the law will affect patients until regulations are clarified in the coming months.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003caside class=\"read-more standard\">\n\u003cdiv class=\"read-more-text\">\u003c/div>\n\u003c/aside>\n\u003cp>In some ways, the pressures and uncertainties posed by new cancer treatments are good problems to have. Julie Guillot said she wishes she’d had the luxury of such challenges when her son was sick.\u003c/p>\n\u003cp>Zach was diagnosed with acute myeloid leukemia when he was 5. For a boy of that age, concerns about unclear clinical benefits of treatments and sky high costs do not apply in the same way they might for an older patient. All the calculations point to the same answer: Try everything you can.\u003c/p>\n\u003cp>Zach received heavy doses of chemotherapy and three bone marrow transplants during years of expensive treatment that left him suffering from uncontrollable fevers, nausea, and infections. He died in 2014.\u003c/p>\n\u003cp>Guillot now works as an \u003ca href=\"https://www.statnews.com/2017/02/20/car-t-therapy-critics-just-dont-get/\">advocate\u003c/a> to help other patients get access to the best treatments and to ensure that experimentation in cancer care can continue to proceed on its inevitably hard and messy course toward cures.\u003c/p>\n\u003cp>“If we are avoidant of new therapies because of the risk, the lack of data, or the cost, breakthrough therapies like CAR-T and bone marrow transplant would never be developed,” she said. “When you are faced with this, people are willing to take risks for a chance to live.”\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"floatright"},"numeric":["floatright"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>\u003cem>This story was originally published by \u003ca href=\"https://www.statnews.com/2018/06/04/cancer-care-dilemma-immunotherapy/\" target=\"_blank\" rel=\"noopener\">STAT\u003c/a>, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery.\u003c/em>\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/442280/new-cancer-care-dilemma-patients-want-immunotherapy-even-when-evidence-is-lacking","authors":["byline_futureofyou_442280"],"categories":["futureofyou_1"],"tags":["futureofyou_103","futureofyou_1077","futureofyou_141","futureofyou_61","futureofyou_686"],"collections":["futureofyou_1093","futureofyou_1097"],"featImg":"futureofyou_442283","label":"source_futureofyou_442280"},"futureofyou_440994":{"type":"posts","id":"futureofyou_440994","meta":{"index":"posts_1591205157","site":"futureofyou","id":"440994","score":null,"sort":[1524769220000]},"guestAuthors":[],"slug":"medicare-to-require-hospitals-to-post-prices-online","title":"Medicare to Require Hospitals to Post Prices Online","publishDate":1524769220,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>Medicare will require hospitals to post their standard prices online and make electronic medical records more readily available to patients, officials said Tuesday.\u003c/p>\n\u003cp>The program is also starting a comprehensive review of how it will pay for costly new forms of immunotherapy to battle cancer.[contextly_sidebar id=\"HODGqRLEtV1B3RpHUo0k0t9wyrSPA5lr\"]\u003c/p>\n\u003cp>Seema Verma, head of the Centers for Medicare and Medicaid Services, said the new requirement for online prices reflects the Trump administration’s ongoing efforts to encourage patients to become better-educated decision makers in their own care.\u003c/p>\n\u003cp>“We are just beginning on price transparency,” said Verma. “We know that hospitals have this information and we’re asking them to post what they have online.”\u003c/p>\n\u003cp>Hospitals are required to disclose prices publicly, but the latest change would put that information online in machine-readable format that can be easily processed by computers. It may still prove to be confusing to consumers, since standard rates are like list prices and don’t reflect what insurers and government programs pay.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>Patients concerned about their potential out-of-pocket costs from a hospitalization would still be advised to consult with their insurer. Most insurance plans nowadays have an annual limit on how much patients must pay in copays and deductibles — although traditional Medicare does not.[contextly_sidebar id=\"2h9g5iEXdqdOugweYZDg5bWHhS2W2vNa\"]\u003c/p>\n\u003cp>Likewise, many health care providers already make computerized records available to patients, but starting in 2021 Medicare would base part of a hospital’s payments on how good a job they do.\u003c/p>\n\u003cp>Using electronic medical records remains a cumbersome task, and the Trump administration has invited technology companies to design secure apps that would let patients access their records from all their providers instead of having to go to different portals.\u003c/p>\n\u003cp>Verma also announced Medicare is starting a comprehensive review of how it will pay for a costly new form of immunotherapy called CAR-T. It’s gene therapy that turbocharges a patient’s own immune system cells to attack cancer.\u003c/p>\n\u003cp>Immune system T cells are filtered from the patient’s own blood and reprogrammed to target and kill cancer cells that had managed to evade them. Hundreds of millions of copies of the revved-up cells are then returned to the patient’s blood to take on the cancer.\u003c/p>\n\u003cp>Though only a couple of such treatments have been approved for blood cancers, the cost can exceed $370,000 per patient.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>“It’s a new area for the agency,” said Verma. “We haven’t seen drugs priced at this level and we’re having to think about our strategy.”\u003c/p>\n\n","blocks":[],"excerpt":"Medicare is also starting a comprehensive review of how it will pay for a costly new form of immunotherapy called CAR-T. Though only a couple of such treatments have been approved for blood cancers, the cost can exceed $370,000 per patient.","status":"publish","parent":0,"modified":1524764184,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":14,"wordCount":444},"headData":{"title":"Medicare to Require Hospitals to Post Prices Online | KQED","description":"Medicare is also starting a comprehensive review of how it will pay for a costly new form of immunotherapy called CAR-T. Though only a couple of such treatments have been approved for blood cancers, the cost can exceed $370,000 per patient.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Medicare to Require Hospitals to Post Prices Online","datePublished":"2018-04-26T19:00:20.000Z","dateModified":"2018-04-26T17:36:24.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"440994 https://ww2.kqed.org/futureofyou/?p=440994","disqusUrl":"https://ww2.kqed.org/futureofyou/2018/04/26/medicare-to-require-hospitals-to-post-prices-online/","disqusTitle":"Medicare to Require Hospitals to Post Prices Online","source":"Health","nprByline":"Ricardo Alonso-Zaldivar\u003cbr />The Associated Press","path":"/futureofyou/440994/medicare-to-require-hospitals-to-post-prices-online","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Medicare will require hospitals to post their standard prices online and make electronic medical records more readily available to patients, officials said Tuesday.\u003c/p>\n\u003cp>The program is also starting a comprehensive review of how it will pay for costly new forms of immunotherapy to battle cancer.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>Seema Verma, head of the Centers for Medicare and Medicaid Services, said the new requirement for online prices reflects the Trump administration’s ongoing efforts to encourage patients to become better-educated decision makers in their own care.\u003c/p>\n\u003cp>“We are just beginning on price transparency,” said Verma. “We know that hospitals have this information and we’re asking them to post what they have online.”\u003c/p>\n\u003cp>Hospitals are required to disclose prices publicly, but the latest change would put that information online in machine-readable format that can be easily processed by computers. It may still prove to be confusing to consumers, since standard rates are like list prices and don’t reflect what insurers and government programs pay.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>Patients concerned about their potential out-of-pocket costs from a hospitalization would still be advised to consult with their insurer. Most insurance plans nowadays have an annual limit on how much patients must pay in copays and deductibles — although traditional Medicare does not.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>Likewise, many health care providers already make computerized records available to patients, but starting in 2021 Medicare would base part of a hospital’s payments on how good a job they do.\u003c/p>\n\u003cp>Using electronic medical records remains a cumbersome task, and the Trump administration has invited technology companies to design secure apps that would let patients access their records from all their providers instead of having to go to different portals.\u003c/p>\n\u003cp>Verma also announced Medicare is starting a comprehensive review of how it will pay for a costly new form of immunotherapy called CAR-T. It’s gene therapy that turbocharges a patient’s own immune system cells to attack cancer.\u003c/p>\n\u003cp>Immune system T cells are filtered from the patient’s own blood and reprogrammed to target and kill cancer cells that had managed to evade them. Hundreds of millions of copies of the revved-up cells are then returned to the patient’s blood to take on the cancer.\u003c/p>\n\u003cp>Though only a couple of such treatments have been approved for blood cancers, the cost can exceed $370,000 per patient.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>“It’s a new area for the agency,” said Verma. “We haven’t seen drugs priced at this level and we’re having to think about our strategy.”\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/440994/medicare-to-require-hospitals-to-post-prices-online","authors":["byline_futureofyou_440994"],"categories":["futureofyou_1","futureofyou_73"],"tags":["futureofyou_1077","futureofyou_952","futureofyou_1008","futureofyou_61","futureofyou_419","futureofyou_686"],"featImg":"futureofyou_441002","label":"source_futureofyou_440994"},"futureofyou_439584":{"type":"posts","id":"futureofyou_439584","meta":{"index":"posts_1591205157","site":"futureofyou","id":"439584","score":null,"sort":[1519659048000]},"guestAuthors":[],"slug":"new-gene-therapy-gives-teen-a-second-chance-after-cancer","title":"Gene Therapy Was Boy's Last Chance to Stop Leukemia. And It Worked.","publishDate":1519659048,"format":"audio","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>When Shaun Banagan hops up on the exam table in a doctor's office at UCSF Benioff Children’s Hospital his small frame barely crumples the wax paper. The shy 13-year-old has very thin arms and slight wrists.\u003c/p>\n\u003caside class=\"pullquote alignright\">'If this had been a year or two ago we wouldn’t have had this treatment to offer him.'\u003ccite>Dr. Jennifer Willert, UCSF\u003c/cite>\u003c/aside>\n\u003cp>It's October, 2017, and Shaun and his family are waiting anxiously for his oncologist, Dr. Jennifer Willert, to read his latest lab results.\u003c/p>\n\u003cp>“He’s in full remission right now without any detectable disease,\" Willert says. \"So, he’s as negative as you can possibly get!\"\u003c/p>\n\u003cp>The small teen pulls aside his surgical face mask and smiles.\u003c/p>\n\u003cp>\u003cstrong>A Grueling Battle \u003c/strong>\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>Shaun is one of at least 50\u003cstrong> \u003c/strong>children who are in remission because of a breakthrough treatment that engineers the body's own immune system to fight cancer. Last August, the U.S. Food and Drug Administration (FDA) \u003ca href=\"https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm\" target=\"_blank\" rel=\"noopener\">approved\u003c/a> the treatment -- the first accepted gene therapy in the nation -- based on the success of clinical trials like Shaun's.\u003c/p>\n\u003cp>But Shaun's family didn’t know if this day would ever come. Six years ago, he was diagnosed with a form of leukemia called Acute Lymphoblastic Leukemia (ALL), which is the most common cancer in children. The National Cancer Institute estimates that about 3,100 patients under age 20 are diagnosed with ALL each year in America.\u003c/p>\n\u003cfigure id=\"attachment_439703\" class=\"wp-caption alignright\" style=\"max-width: 800px\">\u003cimg class=\"size-medium wp-image-439703\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/02/IMG_4083-e1519260166462-800x511.jpg\" alt=\"\" width=\"800\" height=\"511\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-800x511.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-160x102.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-768x491.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-1020x652.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-1180x754.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-960x613.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-240x153.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-375x240.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-520x332.jpg 520w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462.jpg 1809w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003cfigcaption class=\"wp-caption-text\">Shaun Banagan with his mom, Sheila, and his dad, Clifford, at a doctor appointment in San Francisco. \u003ccite>(Lesley McClurg/ KQED)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Shaun started chemotherapy for the blood cancer a few weeks after his diagnosis. It was his eighth birthday.\u003c/p>\n\u003cp>Suddenly, playing outside was off limits. Shaun's condition made him prone to bleeding. Shaun's mom, Sheila Banagan, started home schooling him -- that is, when he didn’t have fever or nausea. He spent hours day after day at the hospital, and before long he lost his hair.\u003c/p>\n\u003cp>\u003cstrong>A Flicker of Hope \u003c/strong>\u003c/p>\n\u003cp>Then, after three-and-a-half years, Shaun’s blood tests were normal. His nurses filled the clinic with banners and balloons. They were just about to cut the cake when a doctor interrupted the party.\u003c/p>\n\u003cp>The cancer had returned.\u003c/p>\n\u003cp>“It just crushes your world,\" Sheila Banagan says. \"The littlest tiniest bit of hope was taken away from me.\"\u003c/p>\n\u003cp>Shaun endured a second round of intensified chemotherapy to prepare his system for a bone marrow transplant. The procedure went well, but six months later he relapsed.\u003c/p>\n\u003cdiv class=\"sharedaddy show-for-medium-up\">\u003ca href=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223.jpg\">\u003cimg class=\"aligncenter size-full wp-image-439753\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223.jpg\" alt=\"\" width=\"975\" height=\"802\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223.jpg 975w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-160x132.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-800x658.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-768x632.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-960x790.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-240x197.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-375x308.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-520x428.jpg 520w\" sizes=\"(max-width: 975px) 100vw, 975px\">\u003c/a>\u003c/div>\n\u003cdiv class=\"show-for-small-only\">\u003c/div>\n\u003cp> \u003c/p>\n\u003caside class=\"pullquote alignright\">'The science is exciting. The results have been spectacular for some people.'\u003ccite>Dr. Len Lichtenfeld,\u003cbr>\nAmerican Cancer Society\u003c/cite>\u003c/aside>\n\u003cp>\u003cstrong>Last-Ditch Option\u003c/strong>\u003c/p>\n\u003cp>Dr. Willert knew there was only one possibility left. UCSF was just about to launch its first clinical trial using a breakthrough therapy.\u003c/p>\n\u003cp>“If this had been a year or two ago we wouldn’t have had this treatment to offer him,\" says Willert.\u003c/p>\n\u003cp>The therapy is called \u003ca href=\"https://www.cancer.org/treatment/treatments-and-side-effects/treatment-types/immunotherapy/car-t-cell1.html\" target=\"_blank\" rel=\"noopener\">CAR-T\u003c/a>-- which stands for chimeric antigen receptor (CAR) T-cell therapy -- and it's intended to super-boost the immune system. Doctors try CAR-T when everything else fails.\u003c/p>\n\u003cp>Shaun’s medical team shipped a sample of his blood to a lab. There, scientists genetically engineered his immune cells to detect his cancer cells and destroy them. In other words, they hacked the cells. Technicians multiplied massive numbers of new cells and then sent the reprogrammed blood back to San Francisco where Shaun received an infusion. His blood was now full of virulent cancer killers.\u003c/p>\n\u003cp>\u003cb>'Severe' Side Effects\u003c/b>\u003c/p>\n\u003cp>The key to CAR-T therapy is that it solves a central problem with cancer. Usually the body's immune system doesn't recognize cancer cells as foreign, and that's why they can proliferate without the body's usual defenses. CAR-T therapy is like giving the immune system glasses.\u003c/p>\n\u003cp>\"CAR-T can wake the immune system up so it recognizes cancer cells as abnormal,\" says Dr. Len Lichtenfeld, Deputy Chief Medical Officer for the American Cancer Society. \"Then it goes after the cancer in a very real way, but that attack can sometimes be very vicious. It can cause very severe side effects.\"\u003c/p>\n\u003cp>Shaun fought off high fevers and flu-like symptoms in the Intensive Care Unit for a few weeks.\u003c/p>\n\u003cp>Lennie Sender, an oncologist at Children’s Hospital of Orange County, has dubbed the reprogrammed cells 'Frankenstein molecules' because they don't just go after cancer cells; CAR-T cells kill good immune cells too -- white blood cells called B-cells that destroy viruses and infections in the body.\u003c/p>\n\u003cp>\"You lose your B-cells,\" says Sender. \"So, now you have to give patients monthly transfusions of replacement, and it could be for life.\"\u003c/p>\n\u003cp>\u003cstrong>Thumbs Up from the Federal Government\u003c/strong>\u003c/p>\n\u003cp>But the FDA is convinced the risks are worth it for some patients. A little over a month after the FDA approved CAR-t to treat Shaun's condition, the agency \u003ca href=\"https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm581216.htm\" target=\"_blank\" rel=\"noopener\">approved\u003c/a> the therapy for large B-cell lymphoma. This type of lymphoma is one of several types of \u003ca href=\"https://www.cancer.org/cancer/non-hodgkin-lymphoma.html\">non-Hodgkin's lymphoma\u003c/a>.\u003c/p>\n\u003cp>[contextly_sidebar id=\"JGmX60chbAt46mWyiOqoudvsmxPMm9J5\"]The procedure runs about a half million dollars per patient because each treatment has to be individually engineered, but scientists hope technological advancements can change that. Some experts say CAR-T marks a watershed moment in learning how to harness the immune system to fight cancer. Thousands of clinical trials are going on right now to push the science of immunotherapy forward.\u003c/p>\n\u003cp>\u003cstrong>A New Frontier\u003c/strong>\u003c/p>\n\u003cp>\"The science is exciting. The results have been spectacular for some people,\" says Lichtenfeld. \"There's no question.\"\u003c/p>\n\u003cp>He cautions, though, the science is still in early stages. \"Unfortunately, for some people it has not worked. We can never forget that.\"\u003c/p>\n\u003cp>A year after his CAR-T treatment, Shaun’s mom, Sheila, is grateful he's still in remission.\u003c/p>\n\u003cp>“This is really the greatest gift that our family received,\" Sheila says, tears pooling in her eyes. \"It’s a miracle.”\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>Shaun’s life isn’t quite back to normal yet; his immune system is still too fragile for him to play outside with his brother and sister, or attend school. Shaun hopes he'll be back in class next fall.\u003c/p>\n\n","blocks":[],"excerpt":"A last-ditch effort to genetically engineer a child's immune system marks a watershed moment in cancer treatment.","status":"publish","parent":0,"modified":1569282806,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":36,"wordCount":1069},"headData":{"title":"Gene Therapy Was Boy's Last Chance to Stop Leukemia. And It Worked. | KQED","description":"A last-ditch effort to genetically engineer a child's immune system marks a watershed moment in cancer treatment.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Gene Therapy Was Boy's Last Chance to Stop Leukemia. And It Worked.","datePublished":"2018-02-26T15:30:48.000Z","dateModified":"2019-09-23T23:53:26.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"439584 https://ww2.kqed.org/futureofyou/?p=439584","disqusUrl":"https://ww2.kqed.org/futureofyou/2018/02/26/new-gene-therapy-gives-teen-a-second-chance-after-cancer/","disqusTitle":"Gene Therapy Was Boy's Last Chance to Stop Leukemia. And It Worked.","source":"Your Genes","audioTrackLength":323,"path":"/futureofyou/439584/new-gene-therapy-gives-teen-a-second-chance-after-cancer","audioUrl":"https://www.kqed.org/.stream/anon/radio/science/2018/02/CARTtherapy.mp3","audioDuration":325000,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>When Shaun Banagan hops up on the exam table in a doctor's office at UCSF Benioff Children’s Hospital his small frame barely crumples the wax paper. The shy 13-year-old has very thin arms and slight wrists.\u003c/p>\n\u003caside class=\"pullquote alignright\">'If this had been a year or two ago we wouldn’t have had this treatment to offer him.'\u003ccite>Dr. Jennifer Willert, UCSF\u003c/cite>\u003c/aside>\n\u003cp>It's October, 2017, and Shaun and his family are waiting anxiously for his oncologist, Dr. Jennifer Willert, to read his latest lab results.\u003c/p>\n\u003cp>“He’s in full remission right now without any detectable disease,\" Willert says. \"So, he’s as negative as you can possibly get!\"\u003c/p>\n\u003cp>The small teen pulls aside his surgical face mask and smiles.\u003c/p>\n\u003cp>\u003cstrong>A Grueling Battle \u003c/strong>\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>Shaun is one of at least 50\u003cstrong> \u003c/strong>children who are in remission because of a breakthrough treatment that engineers the body's own immune system to fight cancer. Last August, the U.S. Food and Drug Administration (FDA) \u003ca href=\"https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm\" target=\"_blank\" rel=\"noopener\">approved\u003c/a> the treatment -- the first accepted gene therapy in the nation -- based on the success of clinical trials like Shaun's.\u003c/p>\n\u003cp>But Shaun's family didn’t know if this day would ever come. Six years ago, he was diagnosed with a form of leukemia called Acute Lymphoblastic Leukemia (ALL), which is the most common cancer in children. The National Cancer Institute estimates that about 3,100 patients under age 20 are diagnosed with ALL each year in America.\u003c/p>\n\u003cfigure id=\"attachment_439703\" class=\"wp-caption alignright\" style=\"max-width: 800px\">\u003cimg class=\"size-medium wp-image-439703\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/02/IMG_4083-e1519260166462-800x511.jpg\" alt=\"\" width=\"800\" height=\"511\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-800x511.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-160x102.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-768x491.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-1020x652.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-1180x754.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-960x613.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-240x153.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-375x240.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462-520x332.jpg 520w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/IMG_4083-e1519260166462.jpg 1809w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003cfigcaption class=\"wp-caption-text\">Shaun Banagan with his mom, Sheila, and his dad, Clifford, at a doctor appointment in San Francisco. \u003ccite>(Lesley McClurg/ KQED)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Shaun started chemotherapy for the blood cancer a few weeks after his diagnosis. It was his eighth birthday.\u003c/p>\n\u003cp>Suddenly, playing outside was off limits. Shaun's condition made him prone to bleeding. Shaun's mom, Sheila Banagan, started home schooling him -- that is, when he didn’t have fever or nausea. He spent hours day after day at the hospital, and before long he lost his hair.\u003c/p>\n\u003cp>\u003cstrong>A Flicker of Hope \u003c/strong>\u003c/p>\n\u003cp>Then, after three-and-a-half years, Shaun’s blood tests were normal. His nurses filled the clinic with banners and balloons. They were just about to cut the cake when a doctor interrupted the party.\u003c/p>\n\u003cp>The cancer had returned.\u003c/p>\n\u003cp>“It just crushes your world,\" Sheila Banagan says. \"The littlest tiniest bit of hope was taken away from me.\"\u003c/p>\n\u003cp>Shaun endured a second round of intensified chemotherapy to prepare his system for a bone marrow transplant. The procedure went well, but six months later he relapsed.\u003c/p>\n\u003cdiv class=\"sharedaddy show-for-medium-up\">\u003ca href=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223.jpg\">\u003cimg class=\"aligncenter size-full wp-image-439753\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223.jpg\" alt=\"\" width=\"975\" height=\"802\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223.jpg 975w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-160x132.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-800x658.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-768x632.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-960x790.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-240x197.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-375x308.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2018/02/CART_DESKTOP_V04_180223-520x428.jpg 520w\" sizes=\"(max-width: 975px) 100vw, 975px\">\u003c/a>\u003c/div>\n\u003cdiv class=\"show-for-small-only\">\u003c/div>\n\u003cp> \u003c/p>\n\u003caside class=\"pullquote alignright\">'The science is exciting. The results have been spectacular for some people.'\u003ccite>Dr. Len Lichtenfeld,\u003cbr>\nAmerican Cancer Society\u003c/cite>\u003c/aside>\n\u003cp>\u003cstrong>Last-Ditch Option\u003c/strong>\u003c/p>\n\u003cp>Dr. Willert knew there was only one possibility left. UCSF was just about to launch its first clinical trial using a breakthrough therapy.\u003c/p>\n\u003cp>“If this had been a year or two ago we wouldn’t have had this treatment to offer him,\" says Willert.\u003c/p>\n\u003cp>The therapy is called \u003ca href=\"https://www.cancer.org/treatment/treatments-and-side-effects/treatment-types/immunotherapy/car-t-cell1.html\" target=\"_blank\" rel=\"noopener\">CAR-T\u003c/a>-- which stands for chimeric antigen receptor (CAR) T-cell therapy -- and it's intended to super-boost the immune system. Doctors try CAR-T when everything else fails.\u003c/p>\n\u003cp>Shaun’s medical team shipped a sample of his blood to a lab. There, scientists genetically engineered his immune cells to detect his cancer cells and destroy them. In other words, they hacked the cells. Technicians multiplied massive numbers of new cells and then sent the reprogrammed blood back to San Francisco where Shaun received an infusion. His blood was now full of virulent cancer killers.\u003c/p>\n\u003cp>\u003cb>'Severe' Side Effects\u003c/b>\u003c/p>\n\u003cp>The key to CAR-T therapy is that it solves a central problem with cancer. Usually the body's immune system doesn't recognize cancer cells as foreign, and that's why they can proliferate without the body's usual defenses. CAR-T therapy is like giving the immune system glasses.\u003c/p>\n\u003cp>\"CAR-T can wake the immune system up so it recognizes cancer cells as abnormal,\" says Dr. Len Lichtenfeld, Deputy Chief Medical Officer for the American Cancer Society. \"Then it goes after the cancer in a very real way, but that attack can sometimes be very vicious. It can cause very severe side effects.\"\u003c/p>\n\u003cp>Shaun fought off high fevers and flu-like symptoms in the Intensive Care Unit for a few weeks.\u003c/p>\n\u003cp>Lennie Sender, an oncologist at Children’s Hospital of Orange County, has dubbed the reprogrammed cells 'Frankenstein molecules' because they don't just go after cancer cells; CAR-T cells kill good immune cells too -- white blood cells called B-cells that destroy viruses and infections in the body.\u003c/p>\n\u003cp>\"You lose your B-cells,\" says Sender. \"So, now you have to give patients monthly transfusions of replacement, and it could be for life.\"\u003c/p>\n\u003cp>\u003cstrong>Thumbs Up from the Federal Government\u003c/strong>\u003c/p>\n\u003cp>But the FDA is convinced the risks are worth it for some patients. A little over a month after the FDA approved CAR-t to treat Shaun's condition, the agency \u003ca href=\"https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm581216.htm\" target=\"_blank\" rel=\"noopener\">approved\u003c/a> the therapy for large B-cell lymphoma. This type of lymphoma is one of several types of \u003ca href=\"https://www.cancer.org/cancer/non-hodgkin-lymphoma.html\">non-Hodgkin's lymphoma\u003c/a>.\u003c/p>\n\u003cp>\u003c/p>\u003cp>\u003c/p>\u003cp>The procedure runs about a half million dollars per patient because each treatment has to be individually engineered, but scientists hope technological advancements can change that. Some experts say CAR-T marks a watershed moment in learning how to harness the immune system to fight cancer. Thousands of clinical trials are going on right now to push the science of immunotherapy forward.\u003c/p>\n\u003cp>\u003cstrong>A New Frontier\u003c/strong>\u003c/p>\n\u003cp>\"The science is exciting. The results have been spectacular for some people,\" says Lichtenfeld. \"There's no question.\"\u003c/p>\n\u003cp>He cautions, though, the science is still in early stages. \"Unfortunately, for some people it has not worked. We can never forget that.\"\u003c/p>\n\u003cp>A year after his CAR-T treatment, Shaun’s mom, Sheila, is grateful he's still in remission.\u003c/p>\n\u003cp>“This is really the greatest gift that our family received,\" Sheila says, tears pooling in her eyes. \"It’s a miracle.”\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>Shaun’s life isn’t quite back to normal yet; his immune system is still too fragile for him to play outside with his brother and sister, or attend school. Shaun hopes he'll be back in class next fall.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/439584/new-gene-therapy-gives-teen-a-second-chance-after-cancer","authors":["11229"],"categories":["futureofyou_1062","futureofyou_1","futureofyou_1064"],"tags":["futureofyou_103","futureofyou_1470","futureofyou_686","futureofyou_80","futureofyou_1471"],"collections":["futureofyou_1097"],"featImg":"futureofyou_439705","label":"source_futureofyou_439584"},"futureofyou_434938":{"type":"posts","id":"futureofyou_434938","meta":{"index":"posts_1591205157","site":"futureofyou","id":"434938","score":null,"sort":[1503514774000]},"guestAuthors":[],"slug":"crispr-cas9-used-to-uncover-immunotherapy-resistant-genes","title":"CRISPR-Cas9 Used to Uncover Immunotherapy-Resistant Genes","publishDate":1503514774,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp>\u003cspan style=\"font-weight: 400\">Immunotherapy -- using a patient’s immune system to recognize and destroy cancer cells -- has been one of the more exciting developments in cancer treatment. Where once there was little hope for some patients, now they can be treated, and with a lot fewer side effects than conventional treatments like chemotherapy and radiation. \u003c/span>\u003c/p>\n\u003caside class=\"pullquote alignright\">Trying to find the genetic culprits that make so many patients resistant to immunotherapy treatment.\u003c/aside>\n\u003cp>But \u003ca href=\"https://ww2.kqed.org/futureofyou/2017/03/17/hype-exceeds-evidence-on-cancer-immunotherapy-commentary/\" target=\"_blank\" rel=\"noopener noreferrer\">not every patient responds\u003c/a> to immunotherapy. For example, the drug Keytruda, used to treat advanced melanoma, does not work in around 60 percent of patients.\u003c/p>\n\u003cp>In a new \u003ca href=\"https://www.nature.com/articles/nature23477.epdf?referrer_access_token=mKoxGWPHyj4t-ukC4sL_U9RgN0jAjWel9jnR3ZoTv0Okbs8jg0lEwUt3XKoawrVg16iZejXcB6lspWAJGFczH8lpUo6hOfGtyRCcmSjWq4g33HqoXWUscKhNXztdf2eBKUYufopzHZZqaSaWT7n6CI8U7FW--zdqjX720jFKI3KCxYG6HzNyDxbEWPBSela-j6r8_wLXlDaPHtPIwCNWC_g6yJkizH863SYaFBbHedzKz_aqbEUuzpFHVih_KU0EpIDA7Ek7FvQ62RvtpKXRpCqNSVhV60SGzlJRca9pndg%3D&tracking_referrer=www.the-scientist.com%3Chttps://protect-us.mimecast.com/s/6ROrB0CgO9YuJ?domain=nature.com\" target=\"_blank\" rel=\"noopener noreferrer\">study\u003c/a>, published this month in \u003ci>\u003cspan style=\"font-weight: 400\">Nature\u003c/span>\u003c/i>\u003cspan style=\"font-weight: 400\">, a group of \u003c/span>scientists from the National Cancer Institute\u003cb> \u003c/b>\u003cspan style=\"font-weight: 400\">set out to find why immunotherapy fails in so many people. The researchers used the gene‐editing tool CRISPR‐Cas9 to uncover \u003c/span>554 \u003cspan style=\"font-weight: 400\">genes that may, when mutated, cause advanced melanoma tumors to be resistant to the treatment\u003c/span>\u003cspan style=\"font-weight: 400\">. If scientists can find which of these genes are the culprits, drugs could potentially be created to correct them, making the cancers responsive to immunotherapy.\u003c/span>\u003c/p>\n\u003cp>\u003cstrong>A Gene Implicated\u003c/strong>\u003c/p>\n\u003cp>Finding these 554 genes was like finding a needle in a haystack. The researchers started out with a cancer cell that responds to immunotherapy. They then disabled, one at a time in the cell, almost all of the known 19,050 human genes, cutting each in different spots. After incapacitating an additional 3,000 parts of the DNA that don't fit the classical definition of a gene, the scientists ended up generating 100,000 different cancer cells, each varying by only a single DNA change. Of these 100,000 cells, 554 showed resistance to immunotherapy.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>The researchers then showed mutation of one those genes, called APLNR, was most likely responsible for immunotherapy resistance in some cancer patients.\u003c/p>\n\u003cp>When the scientists disabled APLNR in a cancer cell, the cell became resistant. The scientists then added a working copy of APLNR, and the cell's resistance disappeared.\u003c/p>\n\u003cp>Next the researchers searched through databases with the DNA sequences of patients' immunotherapy-resistant tumors. The scientists discovered that some did have mutations in the APLNR gene.\u003c/p>\n\u003cp>Doing the experiment in petri dishes is only the first step. Unlike in this process, real-world cancers can’t be made sensitive to immunotherapy by adding back a working gene, because too few cells would adopt it. Instead, a drug would have to be developed. Researchers will also need to experiment with the other 553 genes to see which behave like APLNR.\u003c/p>\n\u003cp>\u003cstrong>CRISPR-Cas9 -- A Killer App\u003c/strong>\u003c/p>\n\u003cp>This gargantuan effort would have been much more difficult even a few years ago. It's only with the advent of the CRISPR‐Cas9 gene‐editing system that it could be done so efficiently.\u003c/p>\n\u003cp>The big advantage of the enzyme Cas9, the workhorse of the CRISPR‐Cas9 system, is how easily it can be programmed to precisely cut the right spot in the over six feet of DNA each of us has packed into every cell. It's that ease of use that allowed these scientists to program Cas9 to specifically travel to more than 100,000 different spots in the cancer cell’s DNA.\u003c/p>\n\u003cp>This is a really exciting use for CRISPR‐Cas9 that a lot of people have not heard of. While most news stories focus on using the system to directly cure a disease or even to make designer babies, this gene- editing tool is revolutionizing the kind of fundamental research that could help us discover new drugs.\u003c/p>\n\u003cp>To say nothing of what it is teaching us about basic biology.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>Dr. Barry Starr is a scientist in Stanford University's \u003ca href=\"https://med.stanford.edu/genetics.html\" target=\"_blank\" rel=\"noopener noreferrer\">Department of Genetics\u003c/a>. He runs both the \u003ca href=\"https://med.stanford.edu/genetics/tech.html\" target=\"_blank\" rel=\"noopener noreferrer\">Stanford at The Tech\u003c/a> program and the \u003ca href=\"http://genetics.thetech.org/\" target=\"_blank\" rel=\"noopener noreferrer\">Understanding Genetics\u003c/a> website with \u003ca href=\"https://www.thetech.org/\" target=\"_blank\" rel=\"noopener noreferrer\">The Tech Museum of Innovation\u003c/a> in San Jose, California. He earlier worked as a research scientist in the biotechnology field.\u003c/p>\n\n","blocks":[],"excerpt":"Scientists used the CRISPR‐Cas9 gene-editing tool to uncover 554 genes that may have mutated in ways that make advanced melanoma tumors resistant to immunotherapy.","status":"publish","parent":0,"modified":1503941266,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":17,"wordCount":666},"headData":{"title":"CRISPR-Cas9 Used to Uncover Immunotherapy-Resistant Genes | KQED","description":"Scientists used the CRISPR‐Cas9 gene-editing tool to uncover 554 genes that may have mutated in ways that make advanced melanoma tumors resistant to immunotherapy.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"CRISPR-Cas9 Used to Uncover Immunotherapy-Resistant Genes","datePublished":"2017-08-23T18:59:34.000Z","dateModified":"2017-08-28T17:27:46.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"434938 https://ww2.kqed.org/futureofyou/?p=434938","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/08/23/crispr-cas9-used-to-uncover-immunotherapy-resistant-genes/","disqusTitle":"CRISPR-Cas9 Used to Uncover Immunotherapy-Resistant Genes","nprByline":"Barry Starr","path":"/futureofyou/434938/crispr-cas9-used-to-uncover-immunotherapy-resistant-genes","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>\u003cspan style=\"font-weight: 400\">Immunotherapy -- using a patient’s immune system to recognize and destroy cancer cells -- has been one of the more exciting developments in cancer treatment. Where once there was little hope for some patients, now they can be treated, and with a lot fewer side effects than conventional treatments like chemotherapy and radiation. \u003c/span>\u003c/p>\n\u003caside class=\"pullquote alignright\">Trying to find the genetic culprits that make so many patients resistant to immunotherapy treatment.\u003c/aside>\n\u003cp>But \u003ca href=\"https://ww2.kqed.org/futureofyou/2017/03/17/hype-exceeds-evidence-on-cancer-immunotherapy-commentary/\" target=\"_blank\" rel=\"noopener noreferrer\">not every patient responds\u003c/a> to immunotherapy. For example, the drug Keytruda, used to treat advanced melanoma, does not work in around 60 percent of patients.\u003c/p>\n\u003cp>In a new \u003ca href=\"https://www.nature.com/articles/nature23477.epdf?referrer_access_token=mKoxGWPHyj4t-ukC4sL_U9RgN0jAjWel9jnR3ZoTv0Okbs8jg0lEwUt3XKoawrVg16iZejXcB6lspWAJGFczH8lpUo6hOfGtyRCcmSjWq4g33HqoXWUscKhNXztdf2eBKUYufopzHZZqaSaWT7n6CI8U7FW--zdqjX720jFKI3KCxYG6HzNyDxbEWPBSela-j6r8_wLXlDaPHtPIwCNWC_g6yJkizH863SYaFBbHedzKz_aqbEUuzpFHVih_KU0EpIDA7Ek7FvQ62RvtpKXRpCqNSVhV60SGzlJRca9pndg%3D&tracking_referrer=www.the-scientist.com%3Chttps://protect-us.mimecast.com/s/6ROrB0CgO9YuJ?domain=nature.com\" target=\"_blank\" rel=\"noopener noreferrer\">study\u003c/a>, published this month in \u003ci>\u003cspan style=\"font-weight: 400\">Nature\u003c/span>\u003c/i>\u003cspan style=\"font-weight: 400\">, a group of \u003c/span>scientists from the National Cancer Institute\u003cb> \u003c/b>\u003cspan style=\"font-weight: 400\">set out to find why immunotherapy fails in so many people. The researchers used the gene‐editing tool CRISPR‐Cas9 to uncover \u003c/span>554 \u003cspan style=\"font-weight: 400\">genes that may, when mutated, cause advanced melanoma tumors to be resistant to the treatment\u003c/span>\u003cspan style=\"font-weight: 400\">. If scientists can find which of these genes are the culprits, drugs could potentially be created to correct them, making the cancers responsive to immunotherapy.\u003c/span>\u003c/p>\n\u003cp>\u003cstrong>A Gene Implicated\u003c/strong>\u003c/p>\n\u003cp>Finding these 554 genes was like finding a needle in a haystack. The researchers started out with a cancer cell that responds to immunotherapy. They then disabled, one at a time in the cell, almost all of the known 19,050 human genes, cutting each in different spots. After incapacitating an additional 3,000 parts of the DNA that don't fit the classical definition of a gene, the scientists ended up generating 100,000 different cancer cells, each varying by only a single DNA change. Of these 100,000 cells, 554 showed resistance to immunotherapy.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>The researchers then showed mutation of one those genes, called APLNR, was most likely responsible for immunotherapy resistance in some cancer patients.\u003c/p>\n\u003cp>When the scientists disabled APLNR in a cancer cell, the cell became resistant. The scientists then added a working copy of APLNR, and the cell's resistance disappeared.\u003c/p>\n\u003cp>Next the researchers searched through databases with the DNA sequences of patients' immunotherapy-resistant tumors. The scientists discovered that some did have mutations in the APLNR gene.\u003c/p>\n\u003cp>Doing the experiment in petri dishes is only the first step. Unlike in this process, real-world cancers can’t be made sensitive to immunotherapy by adding back a working gene, because too few cells would adopt it. Instead, a drug would have to be developed. Researchers will also need to experiment with the other 553 genes to see which behave like APLNR.\u003c/p>\n\u003cp>\u003cstrong>CRISPR-Cas9 -- A Killer App\u003c/strong>\u003c/p>\n\u003cp>This gargantuan effort would have been much more difficult even a few years ago. It's only with the advent of the CRISPR‐Cas9 gene‐editing system that it could be done so efficiently.\u003c/p>\n\u003cp>The big advantage of the enzyme Cas9, the workhorse of the CRISPR‐Cas9 system, is how easily it can be programmed to precisely cut the right spot in the over six feet of DNA each of us has packed into every cell. It's that ease of use that allowed these scientists to program Cas9 to specifically travel to more than 100,000 different spots in the cancer cell’s DNA.\u003c/p>\n\u003cp>This is a really exciting use for CRISPR‐Cas9 that a lot of people have not heard of. While most news stories focus on using the system to directly cure a disease or even to make designer babies, this gene- editing tool is revolutionizing the kind of fundamental research that could help us discover new drugs.\u003c/p>\n\u003cp>To say nothing of what it is teaching us about basic biology.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>Dr. Barry Starr is a scientist in Stanford University's \u003ca href=\"https://med.stanford.edu/genetics.html\" target=\"_blank\" rel=\"noopener noreferrer\">Department of Genetics\u003c/a>. He runs both the \u003ca href=\"https://med.stanford.edu/genetics/tech.html\" target=\"_blank\" rel=\"noopener noreferrer\">Stanford at The Tech\u003c/a> program and the \u003ca href=\"http://genetics.thetech.org/\" target=\"_blank\" rel=\"noopener noreferrer\">Understanding Genetics\u003c/a> website with \u003ca href=\"https://www.thetech.org/\" target=\"_blank\" rel=\"noopener noreferrer\">The Tech Museum of Innovation\u003c/a> in San Jose, California. He earlier worked as a research scientist in the biotechnology field.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/434938/crispr-cas9-used-to-uncover-immunotherapy-resistant-genes","authors":["byline_futureofyou_434938"],"categories":["futureofyou_1062","futureofyou_1","futureofyou_1064"],"tags":["futureofyou_94","futureofyou_1275","futureofyou_686"],"featImg":"futureofyou_434994","label":"futureofyou"},"futureofyou_351698":{"type":"posts","id":"futureofyou_351698","meta":{"index":"posts_1591205157","site":"futureofyou","id":"351698","score":null,"sort":[1489766850000]},"guestAuthors":[],"slug":"hype-exceeds-evidence-on-cancer-immunotherapy-commentary","title":"Hype Exceeds Evidence on Cancer Immunotherapy (Commentary)","publishDate":1489766850,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp>People with cancer face many challenges, including the symptoms of the disease, the toxicity of the treatment, financial costs, and social expectations. Here’s a new threat: navigating their care in an \u003ca href=\"https://www.statnews.com/2016/09/25/cancer-immunotherapy-caution/\" target=\"_blank\">ocean of hype\u003c/a>.\u003c/p>\n\u003caside class=\"pullquote alignright\">Using cancer statistics and FDA approvals, an estimate of the percentage of cancer patients who might benefit from immunotherapy produces a surprising result, given the way the drugs are described.\u003c/aside>\n\u003cp>Cancer drugs are all too often hailed as miracles, breakthroughs, game-changers, or even cures, even when they are no such thing. We recently reported \u003ca href=\"http://jamanetwork.com/journals/jamaoncology/fullarticle/2464965\" target=\"_blank\">in JAMA Oncology\u003c/a> that these words were used 50 percent of the time to describe drugs not approved by the FDA, and 14 percent of the time to describe drugs that had only worked in mice. The leap from helping a mouse to saving a human is uncertain, long, and overwhelmingly unsuccessful.\u003c/p>\n\u003cp>Even when we do have drugs that work, hype may mislead us about how well they work and how many people they will benefit.\u003c/p>\n\u003cp>Consider \u003ca href=\"https://www.statnews.com/2016/08/23/cancer-car-t-side-effects/\" target=\"_blank\">immunotherapy\u003c/a>. This new form of cancer therapy, which uses the body’s own immune system to fight cancer, has captivated the public imagination, is a topic of the nightly news, and has been featured in at least one \u003ca href=\"https://www.ispot.tv/ad/AL_Z/opdivo-longer-life\" target=\"_blank\">Super Bowl ad\u003c/a>.\u003c/p>\n\u003cp>When immunotherapy works, the result is terrific, even life-changing. Today, though, only a tiny minority of patients expected to die from cancer will benefit from immunotherapy. As is often the case, hype sadly exceeds evidence, creating misunderstandings between patients and their doctors.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>Although immunotherapies have been used \u003ca href=\"https://www.amazon.com/Commotion-Blood-Immune-System-Technology/dp/0805037969\" target=\"_blank\">for a hundred years\u003c/a>, such as the deliberate injection of bacteria into the body to stimulate the immune system, 2011 marked the approval of the first immunotherapy for cancer, a so-called checkpoint inhibitor named ipilimumab (Yervoy). This class of drugs unleashes the body’s immune system against cancer, and is the subject of much enthusiasm.\u003c/p>\n\u003cp>Using U.S. national cancer statistics and FDA approvals, we estimated the percent of cancer patients who might actually benefit from immunotherapy. The result was surprising, given the way these drugs are described.\u003c/p>\n\u003cp>To do this, we first calculated the percent of cancers for which immunotherapy has been approved as of February 2017. From that number we determined that two-thirds (68.8 percent) of Americans predicted to die of cancer will die of one that currently has no FDA-approved immunotherapy options\u003cstrong>.\u003c/strong> These include prostate cancer, colon cancer, and ovarian cancer, among others.\u003c/p>\n\u003cp>We next determined the percentage of cancer patients that could expect to see their tumor shrink from immunotherapy. Tumor shrinkage is widely considered to be a prerequisite to benefiting from these drugs. Only 26 percent of patients had this happen.\u003c/p>\n\u003cp>Finally, we combined those two calculations and asked, of all patients dying of cancer in America this year, how many might benefit from a checkpoint inhibitor drug? We assumed the best-case scenario: that every patient with one of these cancers could afford the drug and get access to it.\u003c/p>\n\u003caside class=\"pullquote alignright\">The leap from helping a mouse to saving a human is uncertain, long, and overwhelmingly unsuccessful.\u003c/aside>\n\u003cp>The answer was just 8 percent. We also ran the numbers another way by setting a lower bar for success, and credited these drugs for any patient whose cancer did not grow substantially during follow-up. Even with that adjustment, the estimate was less than 10 percent.\u003c/p>\n\u003cp>What do these results mean? When immunotherapy works, there is no argument — the results are terrific. Patients with otherwise life-threatening cancers live far longer than expected and some may even be cured. But at least today, few patients can expect to be among the lucky ones.\u003c/p>\n\u003cp>Some argue that these drugs will be approved for more cancers in the years to come, or that they may work better in combination with other drugs or one another. While we hope that comes true, it is not the reality today. And for several common cancers, like colon and breast cancer, we already know that these drugs work poorly — there is a reason why the first approvals were in cancers like melanoma — and we fear the percentage of people benefiting from cancer immunotherapy will not change greatly.\u003c/p>\n\u003cp>Who is to blame for the disconnect between reality and hype? All of us. Doctors, researchers, the pharmaceutical industry, reporters, patient advocates — all use \u003ca href=\"http://jamanetwork.com/journals/jamaoncology/fullarticle/2464965\" target=\"_blank\">sensational language\u003c/a> to describe these drugs. To make matters worse, the United States is one of the only countries to permit direct-to-consumer advertising, resulting in an astonishing \u003ca href=\"http://www.vox.com/2016/8/29/12685026/american-drug-ads-tv\" target=\"_blank\">80 drug ads\u003c/a> airing every hour — some of which \u003ca href=\"https://www.nytimes.com/2016/08/09/opinion/cancer-drug-ads-vs-cancer-drug-reality.html\" target=\"_blank\">are misleading\u003c/a>.\u003c/p>\n\u003cp>We owe it to people with cancer to do better. Navigating the waters of accurate information and reasonable hope is a big challenge for oncology. Deciding when and how to treat cancer is a sacred journey that patients and their doctors make together. Distorting the effectiveness of treatments in the public eye can tear the very fabric that unites patients and doctors. Misunderstanding ensues. Expectations become disappointments. A good death becomes a bad one.\u003c/p>\n\u003cp>The intrusive nature of hype — without context, without nuance, and without limit — can be a huge challenge faced by cancer patients in America. For that reason, it should come as no surprise that many cancer patients have \u003ca href=\"http://jamanetwork.com/journals/jamaoncology/article-abstract/2533530\" target=\"_blank\">an inflated understanding\u003c/a> of their prognosis compared to what their doctors understand. Too many patients and their families are inevitably let down when they find themselves among the 90 percent who don’t benefit from immunotherapy.\u003c/p>\n\u003cp>We are not pessimists in our quest to improve survival and quality of life for cancer patients. Instead, we are optimists that we can all do better in communicating the reality of cancer care to patients, to the public, and even to physicians. That way, we may all make more honest choices if and when we must cope with cancer.\u003c/p>\n\u003cp>\u003cem>Nathan Gay, MD, is an oncology fellow at Oregon Health and Science University. Vinay Prasad, MD, is assistant professor in the Division of Hematology Oncology at Oregon Health and Science University and the author of “Ending Medical Reversal.” The views expressed in this article are the authors’ personal opinions and do not represent those of OHSU.\u003c/em>\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>\u003cem>\u003cspan style=\"font-weight: 400\">This \u003ca href=\"https://www.statnews.com/2017/03/08/immunotherapy-cancer-breakthrough/\">story\u003c/a> was originally published by STAT, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery.\u003c/span>\u003c/em>\u003c/p>\n\n","blocks":[],"excerpt":"Despite advances in immunotherapy less than 10 percent of cancer patients will benefit, estimate two researchers.","status":"publish","parent":0,"modified":1489767971,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":21,"wordCount":1063},"headData":{"title":"Hype Exceeds Evidence on Cancer Immunotherapy (Commentary) | KQED","description":"Despite advances in immunotherapy less than 10 percent of cancer patients will benefit, estimate two researchers.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Hype Exceeds Evidence on Cancer Immunotherapy (Commentary)","datePublished":"2017-03-17T16:07:30.000Z","dateModified":"2017-03-17T16:26:11.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"351698 https://ww2.kqed.org/futureofyou/?p=351698","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/03/17/hype-exceeds-evidence-on-cancer-immunotherapy-commentary/","disqusTitle":"Hype Exceeds Evidence on Cancer Immunotherapy (Commentary)","nprByline":"Dr. Nathan Gay and Dr. Vinay Prasad\u003cbr />\u003ca href=\"https://www.statnews.com/\">STAT\u003c/a>","path":"/futureofyou/351698/hype-exceeds-evidence-on-cancer-immunotherapy-commentary","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>People with cancer face many challenges, including the symptoms of the disease, the toxicity of the treatment, financial costs, and social expectations. Here’s a new threat: navigating their care in an \u003ca href=\"https://www.statnews.com/2016/09/25/cancer-immunotherapy-caution/\" target=\"_blank\">ocean of hype\u003c/a>.\u003c/p>\n\u003caside class=\"pullquote alignright\">Using cancer statistics and FDA approvals, an estimate of the percentage of cancer patients who might benefit from immunotherapy produces a surprising result, given the way the drugs are described.\u003c/aside>\n\u003cp>Cancer drugs are all too often hailed as miracles, breakthroughs, game-changers, or even cures, even when they are no such thing. We recently reported \u003ca href=\"http://jamanetwork.com/journals/jamaoncology/fullarticle/2464965\" target=\"_blank\">in JAMA Oncology\u003c/a> that these words were used 50 percent of the time to describe drugs not approved by the FDA, and 14 percent of the time to describe drugs that had only worked in mice. The leap from helping a mouse to saving a human is uncertain, long, and overwhelmingly unsuccessful.\u003c/p>\n\u003cp>Even when we do have drugs that work, hype may mislead us about how well they work and how many people they will benefit.\u003c/p>\n\u003cp>Consider \u003ca href=\"https://www.statnews.com/2016/08/23/cancer-car-t-side-effects/\" target=\"_blank\">immunotherapy\u003c/a>. This new form of cancer therapy, which uses the body’s own immune system to fight cancer, has captivated the public imagination, is a topic of the nightly news, and has been featured in at least one \u003ca href=\"https://www.ispot.tv/ad/AL_Z/opdivo-longer-life\" target=\"_blank\">Super Bowl ad\u003c/a>.\u003c/p>\n\u003cp>When immunotherapy works, the result is terrific, even life-changing. Today, though, only a tiny minority of patients expected to die from cancer will benefit from immunotherapy. As is often the case, hype sadly exceeds evidence, creating misunderstandings between patients and their doctors.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>Although immunotherapies have been used \u003ca href=\"https://www.amazon.com/Commotion-Blood-Immune-System-Technology/dp/0805037969\" target=\"_blank\">for a hundred years\u003c/a>, such as the deliberate injection of bacteria into the body to stimulate the immune system, 2011 marked the approval of the first immunotherapy for cancer, a so-called checkpoint inhibitor named ipilimumab (Yervoy). This class of drugs unleashes the body’s immune system against cancer, and is the subject of much enthusiasm.\u003c/p>\n\u003cp>Using U.S. national cancer statistics and FDA approvals, we estimated the percent of cancer patients who might actually benefit from immunotherapy. The result was surprising, given the way these drugs are described.\u003c/p>\n\u003cp>To do this, we first calculated the percent of cancers for which immunotherapy has been approved as of February 2017. From that number we determined that two-thirds (68.8 percent) of Americans predicted to die of cancer will die of one that currently has no FDA-approved immunotherapy options\u003cstrong>.\u003c/strong> These include prostate cancer, colon cancer, and ovarian cancer, among others.\u003c/p>\n\u003cp>We next determined the percentage of cancer patients that could expect to see their tumor shrink from immunotherapy. Tumor shrinkage is widely considered to be a prerequisite to benefiting from these drugs. Only 26 percent of patients had this happen.\u003c/p>\n\u003cp>Finally, we combined those two calculations and asked, of all patients dying of cancer in America this year, how many might benefit from a checkpoint inhibitor drug? We assumed the best-case scenario: that every patient with one of these cancers could afford the drug and get access to it.\u003c/p>\n\u003caside class=\"pullquote alignright\">The leap from helping a mouse to saving a human is uncertain, long, and overwhelmingly unsuccessful.\u003c/aside>\n\u003cp>The answer was just 8 percent. We also ran the numbers another way by setting a lower bar for success, and credited these drugs for any patient whose cancer did not grow substantially during follow-up. Even with that adjustment, the estimate was less than 10 percent.\u003c/p>\n\u003cp>What do these results mean? When immunotherapy works, there is no argument — the results are terrific. Patients with otherwise life-threatening cancers live far longer than expected and some may even be cured. But at least today, few patients can expect to be among the lucky ones.\u003c/p>\n\u003cp>Some argue that these drugs will be approved for more cancers in the years to come, or that they may work better in combination with other drugs or one another. While we hope that comes true, it is not the reality today. And for several common cancers, like colon and breast cancer, we already know that these drugs work poorly — there is a reason why the first approvals were in cancers like melanoma — and we fear the percentage of people benefiting from cancer immunotherapy will not change greatly.\u003c/p>\n\u003cp>Who is to blame for the disconnect between reality and hype? All of us. Doctors, researchers, the pharmaceutical industry, reporters, patient advocates — all use \u003ca href=\"http://jamanetwork.com/journals/jamaoncology/fullarticle/2464965\" target=\"_blank\">sensational language\u003c/a> to describe these drugs. To make matters worse, the United States is one of the only countries to permit direct-to-consumer advertising, resulting in an astonishing \u003ca href=\"http://www.vox.com/2016/8/29/12685026/american-drug-ads-tv\" target=\"_blank\">80 drug ads\u003c/a> airing every hour — some of which \u003ca href=\"https://www.nytimes.com/2016/08/09/opinion/cancer-drug-ads-vs-cancer-drug-reality.html\" target=\"_blank\">are misleading\u003c/a>.\u003c/p>\n\u003cp>We owe it to people with cancer to do better. Navigating the waters of accurate information and reasonable hope is a big challenge for oncology. Deciding when and how to treat cancer is a sacred journey that patients and their doctors make together. Distorting the effectiveness of treatments in the public eye can tear the very fabric that unites patients and doctors. Misunderstanding ensues. Expectations become disappointments. A good death becomes a bad one.\u003c/p>\n\u003cp>The intrusive nature of hype — without context, without nuance, and without limit — can be a huge challenge faced by cancer patients in America. For that reason, it should come as no surprise that many cancer patients have \u003ca href=\"http://jamanetwork.com/journals/jamaoncology/article-abstract/2533530\" target=\"_blank\">an inflated understanding\u003c/a> of their prognosis compared to what their doctors understand. Too many patients and their families are inevitably let down when they find themselves among the 90 percent who don’t benefit from immunotherapy.\u003c/p>\n\u003cp>We are not pessimists in our quest to improve survival and quality of life for cancer patients. Instead, we are optimists that we can all do better in communicating the reality of cancer care to patients, to the public, and even to physicians. That way, we may all make more honest choices if and when we must cope with cancer.\u003c/p>\n\u003cp>\u003cem>Nathan Gay, MD, is an oncology fellow at Oregon Health and Science University. Vinay Prasad, MD, is assistant professor in the Division of Hematology Oncology at Oregon Health and Science University and the author of “Ending Medical Reversal.” The views expressed in this article are the authors’ personal opinions and do not represent those of OHSU.\u003c/em>\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"floatright"},"numeric":["floatright"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>\u003cem>\u003cspan style=\"font-weight: 400\">This \u003ca href=\"https://www.statnews.com/2017/03/08/immunotherapy-cancer-breakthrough/\">story\u003c/a> was originally published by STAT, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery.\u003c/span>\u003c/em>\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/351698/hype-exceeds-evidence-on-cancer-immunotherapy-commentary","authors":["byline_futureofyou_351698"],"categories":["futureofyou_1062","futureofyou_1"],"tags":["futureofyou_103","futureofyou_1205","futureofyou_686","futureofyou_80"],"featImg":"futureofyou_352337","label":"futureofyou"},"futureofyou_277993":{"type":"posts","id":"futureofyou_277993","meta":{"index":"posts_1591205157","site":"futureofyou","id":"277993","score":null,"sort":[1480696267000]},"guestAuthors":[],"slug":"cuba-has-a-lung-cancer-vaccine-now-u-s-patients-will-test-it","title":"Cuba Has a Lung Cancer Vaccine; Now U.S. Patients Will Test It","publishDate":1480696267,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>Mick Phillips' doctor is surprised he's still alive.\u003c/p>\n\u003cp>The 69-year-old Wisconsin man was diagnosed with late-stage lung cancer in 2010 and his doctor said he had less than a year to live.\u003c/p>\n\u003cp>That was six years ago.\u003c/p>\n\u003cp>\"For all intents and purposes he shouldn’t be here,\" says Phillips' oncologist, \u003ca href=\"http://www.fvho.org/providers/timothy-goggins-md/\" target=\"_blank\">Timothy Goggins\u003c/a>.\u003c/p>\n\u003cp>Dr. Goggins and Phillips both credit his survival to a lung cancer drug that isn't available in the U.S. and that Phillips has had to purchase illegally in Cuba.\u003c/p>\n\u003caside class=\"pullquote alignright\">'Cancer won’t be cured, it will be turned into a chronic condition.’\u003ccite>Agustin Lage,Cuba’s Center of Molecular Immunology\u003c/cite>\u003c/aside>\n\u003cp>U.S. researchers think if the treatment works it could be used to prevent lung cancer in patients that are high risk. \u003ca href=\"https://www.roswellpark.org/\" target=\"_blank\">Roswell Park Cancer Institute\u003c/a> in Buffalo, New York has begun testing CIMAvax, the Cuban-developed immunotherapy for lung cancer. If successful, the trial will pave the way for potential FDA approval, after which the drug could become commercially available in the U.S.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>The medicine has relatively minor side effects and appears to have \u003ca href=\"http://clincancerres.aacrjournals.org/content/early/2016/02/27/1078-0432.CCR-15-0855\" target=\"_blank\">prolonged the lives of patients\u003c/a> who have stage four lung cancer.\u003c/p>\n\u003cp>CIMAvax stimulates the body's immune system to fight the lung cancer. In this way it's a vaccine, but one that you take to stop the spread of late-stage cancer rather than prevent it in the first place.\u003c/p>\n\u003cp>“It’s like a flu vaccine,\" says \u003ca href=\"https://www.roswellpark.org/grace-dy\" target=\"_blank\">Dr. Grace Dy\u003c/a>, principal investigator for the trial. \"It’s the same concept. You give a drug so the body develops an antibody to the drug;\u003cb> \u003c/b>you’re using your body’s own natural systems to develop a therapy.\"\u003c/p>\n\u003cp>\u003cspan style=\"font-weight: 400\">Lung cancer kills \u003ca href=\"http://www.lung.org/lung-health-and-diseases/lung-disease-lookup/lung-cancer/learn-about-lung-cancer/lung-cancer-fact-sheet.html?referrer=https://www.google.com/\" target=\"_blank\">nearly 160,000 Americans\u003c/a> annually. \u003c/span>Most people are diagnosed at a late stage and more than half die within a year.\u003c/p>\n\u003cp>This is likely the first time the \u003ca href=\"https://clinicaltrials.gov/ct2/show/NCT02955290\" target=\"_blank\">FDA has allowed\u003c/a> a clinical trial of a Cuban-made therapy in a U.S. health care facility. It's not uncommon for the\u003cstrong> \u003c/strong>FDA to allow U.S. clinical trials in order to study therapies from other countries. It is extremely rare, however, for that therapy to come from Cuba.\u003c/p>\n\u003cp>And it's popular\u003cstrong>.\u003c/strong>\u003c/p>\n\u003cp>\u003cspan style=\"font-weight: 400\">As of mid-November, nearly 200 people had \u003ca href=\"https://www.roswellpark.org/cimavax-form/?utm_source=page&utm_medium=button&utm_term=form1&utm_content=cimavax\" target=\"_blank\">applied online\u003c/a> to participate in the 60-90 person Phase II and Phase III studies.\u003c/span>\u003c/p>\n\u003cp>\"I’m not surprised that there’s interest,” says Dr. Dy. \u003cspan style=\"font-weight: 400\">“When you have cancer and it’s potentially life-threatening you want to explore all the options.\"\u003c/span>\u003c/p>\n\u003cfigure id=\"attachment_281617\" class=\"wp-caption aligncenter\" style=\"max-width: 960px\">\u003cimg class=\"size-full wp-image-281617\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/11/DrAgustinLage.jpg\" alt=\"Dr. Augustin Lage (left) in Havana with Mick Phillips, researcher Gisela Gonzalez, and Maya Phillips.\" width=\"960\" height=\"720\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-160x120.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-800x600.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-768x576.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-240x180.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-375x281.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-520x390.jpg 520w\" sizes=\"(max-width: 960px) 100vw, 960px\">\u003cfigcaption class=\"wp-caption-text\">Dr. Augustin Lage (left) in Havana with Mick Phillips, researcher Gisela Gonzalez, and Maya Phillips. \u003ccite>(Courtesy Maya Phillips)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Mick Phillips didn't think he had much longer to live when he brought back CIMAvax from Cuba in 2011 and asked a nurse friend to give him the injections.\u003c/p>\n\u003cp>\"He had a 5 percent chance of living five years,\" says Dr. Goggins who practices at \u003ca href=\"http://www.fvho.org/\" target=\"_blank\">Fox Valley Hemotology & Oncology\u003c/a> in Appleton, Wisconsin.\u003c/p>\n\u003cp>Today, Phillips is in remission from stage 4 lung cancer.\u003c/p>\n\u003cp>\"I keep telling people, if you believe in God, it’s God,\" says Dr. Goggins. \"If you believe in science, it’s CIMAvax.”\u003c/p>\n\u003cp>\u003cstrong>How\u003c/strong> \u003cstrong>the Cancer Vaccine Works\u003c/strong>\u003c/p>\n\u003cp>Lung cancer cells feed off a protein called epidermal growth factor or EGF. The protein supports normal cell growth but also fuels cancer cells.\u003c/p>\n\u003cp>The vaccine triggers the immune system to create antibodies that bind to and remove\u003cb> \u003c/b>EGF, thereby starving the cancer of its \"food.\"\u003c/p>\n\u003cp>\u003c!-- iframe plugin v.4.3 wordpress.org/plugins/iframe/ -->\u003cbr>\n\u003ciframe width=\"100%\" src=\"https://www.youtube.com/embed/RaP4TsslJGw?rel=0\" frameborder=\"0\" height=\"500\" scrolling=\"yes\" class=\"iframe-class\">\u003c/iframe>\u003c/p>\n\u003cp>Normal cells can live without EGF and use other growth-promoting proteins instead. But the cancer cells can't, and without EGF they're unable to multiply and spread.\u003c/p>\n\u003cp>CIMAvax has relatively few side effects when compared to chemotherapy, the traditional standard of care for late stage lung cancer. Chemo is hard on the body because it kills healthy cells (in addition to cancer cells) and causes side effects like hair loss, vomiting, loss of appetite and exhaustion.\u003c/p>\n\u003cp>Because CIMAvax doesn't kill cells, its side effects are relatively minor, such as chills or a fever that can be treated with over-the-counter medications.\u003c/p>\n\u003cp>Cuban patients that responded to CIMAvax had high levels of EGF, and t\u003cspan style=\"font-weight: 400\">heir bodies produced high levels of antibodies to combat the protein after they received the vaccine.\u003cstrong> \u003c/strong>\u003c/span>\u003c/p>\n\u003cp>Roswell Park will be looking to see if the same response emerges in its clinical trial— whether the most significant improvement is in patients with high EGF and antibody levels.\u003c/p>\n\u003cp>If the study shows promise it could be good news for other types of cancer that also rely on EGF, such as prostate, breast and colon cancer.\u003c/p>\n\u003cp>The most recent \u003ca href=\"http://clincancerres.aacrjournals.org/content/early/2016/06/24/1078-0432.CCR-15-0855\" target=\"_blank\">Cuban clinical trial \u003c/a>of CIMAvax is comparatively small at about 400 patients, which raises questions about how reliable the findings are.\u003c/p>\n\u003cp>UCSF oncologist \u003ca href=\"http://www.bivonalab.net/people/\" target=\"_blank\">Trever Bivona\u003c/a> says the study isn't large enough to determine whether the findings are statistically significant.\u003c/p>\n\u003cfigure id=\"attachment_278549\" class=\"wp-caption alignright\" style=\"max-width: 1920px\">\u003cimg class=\"size-full wp-image-278549\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/11/CIMAvax-EGF-3.jpeg\" alt=\"The lung cancer vaccine is currently available in Cuba, Peru, Venezuela, Paraguay, Colombia and Bosnia and Herzegovina. U.S. researchers start clinical trials this month.\" width=\"1920\" height=\"1240\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3.jpeg 1920w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-160x103.jpeg 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-800x517.jpeg 800w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-768x496.jpeg 768w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-1020x659.jpeg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-1180x762.jpeg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-960x620.jpeg 960w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-240x155.jpeg 240w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-375x242.jpeg 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-520x336.jpeg 520w\" sizes=\"(max-width: 1920px) 100vw, 1920px\">\u003cfigcaption class=\"wp-caption-text\">The lung cancer vaccine is currently available in Cuba, Peru, Venezuela, Paraguay, Colombia and Bosnia and Herzegovina. U.S. researchers start clinical trials this month. \u003ccite>(Centro de Inmunología Molecular)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>\"I think we need a bigger more controlled study before making any conclusions,\" Bivona says.\u003c/p>\n\u003cp>The other problem with the Cuba study is that it had no control group. It compared patients getting CIMAvax to patients getting palliative care.\u003c/p>\n\u003cp>\"There may be preliminary findings here,\" Bivona says, \"but it’s certainly not a slam dunk.\"\u003c/p>\n\u003cp>\u003cstrong>Will Cuban Drugs Be Approved Under Trump?\u003c/strong>\u003c/p>\n\u003cp>If you're contemplating a flight to Havana, it's important to note that most U.S. travel to Cuba is still restricted.\u003c/p>\n\u003cp>The \u003cspan style=\"font-weight: 400\">U.S. Treasury Office of Foreign Assets Control says people and companies can \u003ca href=\"https://www.treasury.gov/resource-center/sanctions/Pages/licensing.aspx\" target=\"_blank\">apply for a specific license\u003c/a> to visit Cuba and purchase pharmaceuticals like CIMAvax. \u003c/span>\u003c/p>\n\u003cp>\u003cspan style=\"font-weight: 400\">Because the relationship between\u003c/span>\u003cspan style=\"font-weight: 400\"> Cuba and America has warmed, federal officials could potentially approve an application due to this change in \"foreign relations.\"\u003c/span>\u003c/p>\n\u003cp>However, President-elect Donald Trump has vowed to overturn all of President Obama's executive orders. Though it's unclear what that means, Obama's \u003ca href=\"https://www.whitehouse.gov/the-press-office/2014/12/17/fact-sheet-charting-new-course-cuba\" target=\"_blank\">2014 executive order\u003c/a> on Cuba expanded opportunities for Americans to legally visit Cuba.\u003c/p>\n\u003cp>And Vice President-elect Mike Pence has specifically said he will \"\u003ca href=\"http://www.politico.com/story/2016/10/mike-pence-cuba-executive-orders-229827\" target=\"_blank\">reverse Barack Obama’s executive orders on Cuba\u003c/a>.\"\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>Regardless, Dr. Dy says CIMAvax won't be available in pharmacies anytime soon. Roswell Park will start phase I of the clinical trial this month and it'll be months before it can start phase II and then phase III. She says an ambitious goal would be to have the medicine available by 2021.\u003c/p>\n\n","blocks":[],"excerpt":"A New York hospital is the nation's first to test a Cuban-developed lung cancer vaccine.","status":"publish","parent":0,"modified":1480705549,"stats":{"hasAudio":false,"hasVideo":true,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":42,"wordCount":1167},"headData":{"title":"Cuba Has a Lung Cancer Vaccine; Now U.S. Patients Will Test It | KQED","description":"A New York hospital is the nation's first to test a Cuban-developed lung cancer vaccine.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Cuba Has a Lung Cancer Vaccine; Now U.S. Patients Will Test It","datePublished":"2016-12-02T16:31:07.000Z","dateModified":"2016-12-02T19:05:49.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"277993 http://ww2.kqed.org/futureofyou/?p=277993","disqusUrl":"https://ww2.kqed.org/futureofyou/2016/12/02/cuba-has-a-lung-cancer-vaccine-now-u-s-patients-will-test-it/","disqusTitle":"Cuba Has a Lung Cancer Vaccine; Now U.S. Patients Will Test It","source":"Future of You","customPermalink":"2016/11/16/cuba-has-a-lung-cancer-vaccine-now-u-s-patients-will-test-it/","path":"/futureofyou/277993/cuba-has-a-lung-cancer-vaccine-now-u-s-patients-will-test-it","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Mick Phillips' doctor is surprised he's still alive.\u003c/p>\n\u003cp>The 69-year-old Wisconsin man was diagnosed with late-stage lung cancer in 2010 and his doctor said he had less than a year to live.\u003c/p>\n\u003cp>That was six years ago.\u003c/p>\n\u003cp>\"For all intents and purposes he shouldn’t be here,\" says Phillips' oncologist, \u003ca href=\"http://www.fvho.org/providers/timothy-goggins-md/\" target=\"_blank\">Timothy Goggins\u003c/a>.\u003c/p>\n\u003cp>Dr. Goggins and Phillips both credit his survival to a lung cancer drug that isn't available in the U.S. and that Phillips has had to purchase illegally in Cuba.\u003c/p>\n\u003caside class=\"pullquote alignright\">'Cancer won’t be cured, it will be turned into a chronic condition.’\u003ccite>Agustin Lage,Cuba’s Center of Molecular Immunology\u003c/cite>\u003c/aside>\n\u003cp>U.S. researchers think if the treatment works it could be used to prevent lung cancer in patients that are high risk. \u003ca href=\"https://www.roswellpark.org/\" target=\"_blank\">Roswell Park Cancer Institute\u003c/a> in Buffalo, New York has begun testing CIMAvax, the Cuban-developed immunotherapy for lung cancer. If successful, the trial will pave the way for potential FDA approval, after which the drug could become commercially available in the U.S.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>The medicine has relatively minor side effects and appears to have \u003ca href=\"http://clincancerres.aacrjournals.org/content/early/2016/02/27/1078-0432.CCR-15-0855\" target=\"_blank\">prolonged the lives of patients\u003c/a> who have stage four lung cancer.\u003c/p>\n\u003cp>CIMAvax stimulates the body's immune system to fight the lung cancer. In this way it's a vaccine, but one that you take to stop the spread of late-stage cancer rather than prevent it in the first place.\u003c/p>\n\u003cp>“It’s like a flu vaccine,\" says \u003ca href=\"https://www.roswellpark.org/grace-dy\" target=\"_blank\">Dr. Grace Dy\u003c/a>, principal investigator for the trial. \"It’s the same concept. You give a drug so the body develops an antibody to the drug;\u003cb> \u003c/b>you’re using your body’s own natural systems to develop a therapy.\"\u003c/p>\n\u003cp>\u003cspan style=\"font-weight: 400\">Lung cancer kills \u003ca href=\"http://www.lung.org/lung-health-and-diseases/lung-disease-lookup/lung-cancer/learn-about-lung-cancer/lung-cancer-fact-sheet.html?referrer=https://www.google.com/\" target=\"_blank\">nearly 160,000 Americans\u003c/a> annually. \u003c/span>Most people are diagnosed at a late stage and more than half die within a year.\u003c/p>\n\u003cp>This is likely the first time the \u003ca href=\"https://clinicaltrials.gov/ct2/show/NCT02955290\" target=\"_blank\">FDA has allowed\u003c/a> a clinical trial of a Cuban-made therapy in a U.S. health care facility. It's not uncommon for the\u003cstrong> \u003c/strong>FDA to allow U.S. clinical trials in order to study therapies from other countries. It is extremely rare, however, for that therapy to come from Cuba.\u003c/p>\n\u003cp>And it's popular\u003cstrong>.\u003c/strong>\u003c/p>\n\u003cp>\u003cspan style=\"font-weight: 400\">As of mid-November, nearly 200 people had \u003ca href=\"https://www.roswellpark.org/cimavax-form/?utm_source=page&utm_medium=button&utm_term=form1&utm_content=cimavax\" target=\"_blank\">applied online\u003c/a> to participate in the 60-90 person Phase II and Phase III studies.\u003c/span>\u003c/p>\n\u003cp>\"I’m not surprised that there’s interest,” says Dr. Dy. \u003cspan style=\"font-weight: 400\">“When you have cancer and it’s potentially life-threatening you want to explore all the options.\"\u003c/span>\u003c/p>\n\u003cfigure id=\"attachment_281617\" class=\"wp-caption aligncenter\" style=\"max-width: 960px\">\u003cimg class=\"size-full wp-image-281617\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/11/DrAgustinLage.jpg\" alt=\"Dr. Augustin Lage (left) in Havana with Mick Phillips, researcher Gisela Gonzalez, and Maya Phillips.\" width=\"960\" height=\"720\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-160x120.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-800x600.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-768x576.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-240x180.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-375x281.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/DrAgustinLage-520x390.jpg 520w\" sizes=\"(max-width: 960px) 100vw, 960px\">\u003cfigcaption class=\"wp-caption-text\">Dr. Augustin Lage (left) in Havana with Mick Phillips, researcher Gisela Gonzalez, and Maya Phillips. \u003ccite>(Courtesy Maya Phillips)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Mick Phillips didn't think he had much longer to live when he brought back CIMAvax from Cuba in 2011 and asked a nurse friend to give him the injections.\u003c/p>\n\u003cp>\"He had a 5 percent chance of living five years,\" says Dr. Goggins who practices at \u003ca href=\"http://www.fvho.org/\" target=\"_blank\">Fox Valley Hemotology & Oncology\u003c/a> in Appleton, Wisconsin.\u003c/p>\n\u003cp>Today, Phillips is in remission from stage 4 lung cancer.\u003c/p>\n\u003cp>\"I keep telling people, if you believe in God, it’s God,\" says Dr. Goggins. \"If you believe in science, it’s CIMAvax.”\u003c/p>\n\u003cp>\u003cstrong>How\u003c/strong> \u003cstrong>the Cancer Vaccine Works\u003c/strong>\u003c/p>\n\u003cp>Lung cancer cells feed off a protein called epidermal growth factor or EGF. The protein supports normal cell growth but also fuels cancer cells.\u003c/p>\n\u003cp>The vaccine triggers the immune system to create antibodies that bind to and remove\u003cb> \u003c/b>EGF, thereby starving the cancer of its \"food.\"\u003c/p>\n\u003cp>\u003c!-- iframe plugin v.4.3 wordpress.org/plugins/iframe/ -->\u003cbr>\n\u003ciframe width=\"100%\" src=\"https://www.youtube.com/embed/RaP4TsslJGw?rel=0\" frameborder=\"0\" height=\"500\" scrolling=\"yes\" class=\"iframe-class\">\u003c/iframe>\u003c/p>\n\u003cp>Normal cells can live without EGF and use other growth-promoting proteins instead. But the cancer cells can't, and without EGF they're unable to multiply and spread.\u003c/p>\n\u003cp>CIMAvax has relatively few side effects when compared to chemotherapy, the traditional standard of care for late stage lung cancer. Chemo is hard on the body because it kills healthy cells (in addition to cancer cells) and causes side effects like hair loss, vomiting, loss of appetite and exhaustion.\u003c/p>\n\u003cp>Because CIMAvax doesn't kill cells, its side effects are relatively minor, such as chills or a fever that can be treated with over-the-counter medications.\u003c/p>\n\u003cp>Cuban patients that responded to CIMAvax had high levels of EGF, and t\u003cspan style=\"font-weight: 400\">heir bodies produced high levels of antibodies to combat the protein after they received the vaccine.\u003cstrong> \u003c/strong>\u003c/span>\u003c/p>\n\u003cp>Roswell Park will be looking to see if the same response emerges in its clinical trial— whether the most significant improvement is in patients with high EGF and antibody levels.\u003c/p>\n\u003cp>If the study shows promise it could be good news for other types of cancer that also rely on EGF, such as prostate, breast and colon cancer.\u003c/p>\n\u003cp>The most recent \u003ca href=\"http://clincancerres.aacrjournals.org/content/early/2016/06/24/1078-0432.CCR-15-0855\" target=\"_blank\">Cuban clinical trial \u003c/a>of CIMAvax is comparatively small at about 400 patients, which raises questions about how reliable the findings are.\u003c/p>\n\u003cp>UCSF oncologist \u003ca href=\"http://www.bivonalab.net/people/\" target=\"_blank\">Trever Bivona\u003c/a> says the study isn't large enough to determine whether the findings are statistically significant.\u003c/p>\n\u003cfigure id=\"attachment_278549\" class=\"wp-caption alignright\" style=\"max-width: 1920px\">\u003cimg class=\"size-full wp-image-278549\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/11/CIMAvax-EGF-3.jpeg\" alt=\"The lung cancer vaccine is currently available in Cuba, Peru, Venezuela, Paraguay, Colombia and Bosnia and Herzegovina. U.S. researchers start clinical trials this month.\" width=\"1920\" height=\"1240\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3.jpeg 1920w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-160x103.jpeg 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-800x517.jpeg 800w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-768x496.jpeg 768w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-1020x659.jpeg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-1180x762.jpeg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-960x620.jpeg 960w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-240x155.jpeg 240w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-375x242.jpeg 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/11/CIMAvax-EGF-3-520x336.jpeg 520w\" sizes=\"(max-width: 1920px) 100vw, 1920px\">\u003cfigcaption class=\"wp-caption-text\">The lung cancer vaccine is currently available in Cuba, Peru, Venezuela, Paraguay, Colombia and Bosnia and Herzegovina. U.S. researchers start clinical trials this month. \u003ccite>(Centro de Inmunología Molecular)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>\"I think we need a bigger more controlled study before making any conclusions,\" Bivona says.\u003c/p>\n\u003cp>The other problem with the Cuba study is that it had no control group. It compared patients getting CIMAvax to patients getting palliative care.\u003c/p>\n\u003cp>\"There may be preliminary findings here,\" Bivona says, \"but it’s certainly not a slam dunk.\"\u003c/p>\n\u003cp>\u003cstrong>Will Cuban Drugs Be Approved Under Trump?\u003c/strong>\u003c/p>\n\u003cp>If you're contemplating a flight to Havana, it's important to note that most U.S. travel to Cuba is still restricted.\u003c/p>\n\u003cp>The \u003cspan style=\"font-weight: 400\">U.S. Treasury Office of Foreign Assets Control says people and companies can \u003ca href=\"https://www.treasury.gov/resource-center/sanctions/Pages/licensing.aspx\" target=\"_blank\">apply for a specific license\u003c/a> to visit Cuba and purchase pharmaceuticals like CIMAvax. \u003c/span>\u003c/p>\n\u003cp>\u003cspan style=\"font-weight: 400\">Because the relationship between\u003c/span>\u003cspan style=\"font-weight: 400\"> Cuba and America has warmed, federal officials could potentially approve an application due to this change in \"foreign relations.\"\u003c/span>\u003c/p>\n\u003cp>However, President-elect Donald Trump has vowed to overturn all of President Obama's executive orders. Though it's unclear what that means, Obama's \u003ca href=\"https://www.whitehouse.gov/the-press-office/2014/12/17/fact-sheet-charting-new-course-cuba\" target=\"_blank\">2014 executive order\u003c/a> on Cuba expanded opportunities for Americans to legally visit Cuba.\u003c/p>\n\u003cp>And Vice President-elect Mike Pence has specifically said he will \"\u003ca href=\"http://www.politico.com/story/2016/10/mike-pence-cuba-executive-orders-229827\" target=\"_blank\">reverse Barack Obama’s executive orders on Cuba\u003c/a>.\"\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"floatright"},"numeric":["floatright"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>Regardless, Dr. Dy says CIMAvax won't be available in pharmacies anytime soon. Roswell Park will start phase I of the clinical trial this month and it'll be months before it can start phase II and then phase III. She says an ambitious goal would be to have the medicine available by 2021.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/277993/cuba-has-a-lung-cancer-vaccine-now-u-s-patients-will-test-it","authors":["5432"],"categories":["futureofyou_452","futureofyou_1062","futureofyou_1","futureofyou_73"],"tags":["futureofyou_686"],"featImg":"futureofyou_281486","label":"source_futureofyou_277993"}},"programsReducer":{"possible":{"id":"possible","title":"Possible","info":"Possible is hosted by entrepreneur Reid Hoffman and writer Aria Finger. Together in Possible, Hoffman and Finger lead enlightening discussions about building a brighter collective future. The show features interviews with visionary guests like Trevor Noah, Sam Altman and Janette Sadik-Khan. Possible paints an optimistic portrait of the world we can create through science, policy, business, art and our shared humanity. It asks: What if everything goes right for once? How can we get there? 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But is this once sleepy suburb ready for them?","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/American-Suburb-Podcast-Tile-703x703-1.jpg","officialWebsiteLink":"/news/series/american-suburb-podcast","meta":{"site":"news","source":"kqed","order":"13"},"link":"/news/series/american-suburb-podcast/","subscribe":{"npr":"https://rpb3r.app.goo.gl/RBrW","apple":"https://itunes.apple.com/WebObjects/MZStore.woa/wa/viewPodcast?mt=2&id=1287748328","tuneIn":"https://tunein.com/radio/American-Suburb-p1086805/","rss":"https://ww2.kqed.org/news/series/american-suburb-podcast/feed/podcast","google":"https://podcasts.google.com/feed/aHR0cHM6Ly9mZWVkcy5tZWdhcGhvbmUuZm0vS1FJTkMzMDExODgxNjA5"}},"baycurious":{"id":"baycurious","title":"Bay Curious","tagline":"Exploring the Bay Area, one question at a time","info":"KQED’s new podcast, Bay Curious, gets to the bottom of the mysteries — both profound and peculiar — that give the Bay Area its unique identity. And we’ll do it with your help! You ask the questions. You decide what Bay Curious investigates. And you join us on the journey to find the answers.","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Bay-Curious-Podcast-Tile-703x703-1.jpg","imageAlt":"\"KQED Bay Curious","officialWebsiteLink":"/news/series/baycurious","meta":{"site":"news","source":"kqed","order":"4"},"link":"/podcasts/baycurious","subscribe":{"apple":"https://podcasts.apple.com/us/podcast/bay-curious/id1172473406","npr":"https://www.npr.org/podcasts/500557090/bay-curious","rss":"https://ww2.kqed.org/news/category/bay-curious-podcast/feed/podcast","google":"https://podcasts.google.com/feed/aHR0cHM6Ly93dzIua3FlZC5vcmcvbmV3cy9jYXRlZ29yeS9iYXktY3VyaW91cy1wb2RjYXN0L2ZlZWQvcG9kY2FzdA","stitcher":"https://www.stitcher.com/podcast/kqed/bay-curious","spotify":"https://open.spotify.com/show/6O76IdmhixfijmhTZLIJ8k"}},"bbc-world-service":{"id":"bbc-world-service","title":"BBC World Service","info":"The day's top stories from BBC News compiled twice daily in the week, once at weekends.","airtime":"MON-FRI 9pm-10pm, TUE-FRI 1am-2am","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/BBC-World-Service-Podcast-Tile-360x360-1.jpg","officialWebsiteLink":"https://www.bbc.co.uk/sounds/play/live:bbc_world_service","meta":{"site":"news","source":"BBC World Service"},"link":"/radio/program/bbc-world-service","subscribe":{"apple":"https://itunes.apple.com/us/podcast/global-news-podcast/id135067274?mt=2","tuneIn":"https://tunein.com/radio/BBC-World-Service-p455581/","rss":"https://podcasts.files.bbci.co.uk/p02nq0gn.rss"}},"code-switch-life-kit":{"id":"code-switch-life-kit","title":"Code Switch / Life Kit","info":"\u003cem>Code Switch\u003c/em>, which listeners will hear in the first part of the hour, has fearless and much-needed conversations about race. Hosted by journalists of color, the show tackles the subject of race head-on, exploring how it impacts every part of society — from politics and pop culture to history, sports and more.\u003cbr />\u003cbr />\u003cem>Life Kit\u003c/em>, which will be in the second part of the hour, guides you through spaces and feelings no one prepares you for — from finances to mental health, from workplace microaggressions to imposter syndrome, from relationships to parenting. The show features experts with real world experience and shares their knowledge. Because everyone needs a little help being human.\u003cbr />\u003cbr />\u003ca href=\"https://www.npr.org/podcasts/510312/codeswitch\">\u003cem>Code Switch\u003c/em> offical site and podcast\u003c/a>\u003cbr />\u003ca href=\"https://www.npr.org/lifekit\">\u003cem>Life Kit\u003c/em> offical site and podcast\u003c/a>\u003cbr />","airtime":"SUN 9pm-10pm","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Code-Switch-Life-Kit-Podcast-Tile-360x360-1.jpg","meta":{"site":"radio","source":"npr"},"link":"/radio/program/code-switch-life-kit","subscribe":{"apple":"https://podcasts.apple.com/podcast/1112190608?mt=2&at=11l79Y&ct=nprdirectory","google":"https://podcasts.google.com/feed/aHR0cHM6Ly93d3cubnByLm9yZy9yc3MvcG9kY2FzdC5waHA_aWQ9NTEwMzEy","spotify":"https://open.spotify.com/show/3bExJ9JQpkwNhoHvaIIuyV","rss":"https://feeds.npr.org/510312/podcast.xml"}},"commonwealth-club":{"id":"commonwealth-club","title":"Commonwealth Club of California Podcast","info":"The Commonwealth Club of California is the nation's oldest and largest public affairs forum. 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