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`F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000 - The New York Times
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`https://www.nytimes.com/2017/08/30/health/gene-therapy-cancer.html
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`F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000
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`By Denise Grady
`Aug. 30, 2017
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`The Food and Drug Administration on Wednesday approved the first-ever treatment that genetically alters a patient’s own cells to fight
`cancer, a milestone that is expected to transform treatment in the coming years.
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`The new therapy turns a patient’s cells into a “living drug,” and trains them to recognize and attack the disease. It is part of the rapidly
`growing field of immunotherapy that bolsters the immune system through drugs and other therapies and has, in some cases, led to long
`remissions and possibly even cures.
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`The therapy, marketed as Kymriah and made by Novartis, was approved for children and young adults for an aggressive type of leukemia
`— B-cell acute lymphoblastic leukemia — that has resisted standard treatment or relapsed. The F.D.A. called the disease “devastating and
`deadly” and said the new treatment fills an “unmet need.”
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`Novartis and other companies have been racing to develop gene therapies for other types of cancers, and experts expect more approvals
`in the near future. Dr. Scott Gottlieb, the F.D.A. commissioner, said that more than 550 types of experimental gene therapy were being
`studied.
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`There are drawbacks to the approach. Because Kymriah can have life-threatening side effects, including dangerous drops in blood
`pressure, the F.D.A. is requiring that hospitals and doctors be specially trained and certified to administer it, and that they stock a certain
`drug needed to quell severe reactions.
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`Kymriah, which will be given to patients just once and must be made individually for each, will cost $475,000. Novartis said that if a patient
`does not respond within the first month after treatment, there will be no charge. The company also said it would provide financial help to
`families who were uninsured or underinsured.
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`Emily Whitehead, shown here in May, was near death at age 6 from leukemia and became
`the first pediatric patient to receive the experimental gene therapy. She is now 12 and has
`been in remission for more than five years. Children’s Hospital of Philadelphia, via Associated
`Press
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`Discussing the high price during a telephone news conference, a Novartis official noted that bone-marrow transplants, which can cure
`some cases of leukemia, cost even more, from $540,000 to $800,000.
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`About 600 children and young adults a year in the United States would be candidates for the new treatment.
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`The approval was based largely on a trial in 63 severely ill children and young adults who had a remission rate of 83 percent within three
`months — a high rate, given that relapsed or treatment-resistant disease is often quickly fatal.
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`The treatment was originally developed by researchers at the University of Pennsylvania and licensed to Novartis. It was identified in
`previous reports as CAR-T cell therapy, CTL019 or tisagenlecleucel.
`https://www.nytimes.com/2017/08/30/health/gene-therapy-cancer.html
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`F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000 - The New York Times
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`The first child to receive the therapy was Emily Whitehead, who was 6 and near death from leukemia in 2012 when she was treated, at the
`Children’s Hospital of Philadelphia. Now 12, she has been free of leukemia for more than five years.
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`To customize Kymriah for individual patients, white blood cells called T cells will be removed from a patient’s bloodstream at an approved
`medical center, frozen, shipped to Novartis in Morris Plains, N.J., for genetic engineering and multiplying, frozen again and shipped back
`to the medical center to be dripped into the patient. That processing is expected to take 22 days.
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`Novartis said the treatment would be available at an initial network of 20 approved medical centers to be certified within a month, a
`number that would be expanded to 32 by the end of the year. Five centers will be ready to start extracting T cells from patients within
`three to five days, the company said.
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`An intravenous bag of Kymriah, which must be customized for individual patients. It is
`expected to cost $475,000 and can have potentially fatal side effects. Novartis, via
`Associated Press
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`Certification is being required because the revved-up T cells can touch off an intense reaction, sometimes called a cytokine storm, that can
`cause high fever, low blood pressure, lung congestion, neurological problems and other life-threatening complications. Medical staff
`members need training to manage these reactions, and hospitals are being told that before giving Kymriah to patients, they must be sure
`that they have the drug needed to treat the problems, tocilizumab, also called Actemra.
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`Dr. Kevin J. Curran, a pediatric oncologist at Memorial Sloan Kettering Cancer Center in Manhattan, said his hospital was “99 percent” of
`the way through the certification process, and would soon be offering Kymriah.
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`“This is a big paradigm shift, using this living drug,” Dr. Curran said. “It will provide a lot of hope. This is the beginning.”
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`He said he expected that eventually this type of treatment would work for other, more common types of cancer, not just for leukemia.
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`The F.D.A.’s approval of Kymriah ushers in “a new approach to the treatment of cancer and other serious and life-threatening diseases,”
`the agency said in a statement, noting that the new therapy is “the first gene therapy available in the United States.”
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`Dr. Carl June, a leader in developing the treatment at the University of Pennsylvania, recalled that in 2010, when tests showed that the
`first patient was leukemia-free a month after being treated, he and his colleagues did not believe it. They ordered another biopsy to be
`sure.
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`“Now, I have to keep pinching myself to see that this happened,” Dr. June said, his voice breaking with emotion. “It was so improbable that
`this would ever be a commercially approved therapy, and now it’s the first gene therapy approved in the United States. It’s so different
`from all the pharmaceutical models. I think the cancer world is forever changed.”
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`A version of this article appears in print on , Section B, Page 1 of the New York edition with the headline: F.D.A. Backs Gene Therapy For Leukemia
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`https://www.nytimes.com/2017/08/30/health/gene-therapy-cancer.html
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