2017: The year gene therapy became a ‘clinical reality’

| | December 15, 2017
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This year, gene therapy finally became a clinical reality. The U.S. Food and Drug Administration approved two personalized treatments that engineer a patient’s own immune system to hunt down and kill cancer cells. The treatments, the first gene therapies ever approved by the FDA, work in people with certain blood cancers, even patients whose cancers haven’t responded to other treatments.

Called CAR-T cell immunotherapy (for chimeric antigen receptor T cell), one is for kids and young adults with B cell acute lymphoblastic leukemia, or ALL, approved in August (SN Online: 8/30/17). The other is for adults with non-Hodgkin lymphoma, approved in October. Other CAR-T cell therapies are in testing, including a treatment for multiple myeloma.

“It’s a completely different way of treating cancer,” says pediatric oncologist Stephan Grupp, who directs the Cancer Immunotherapy Program at the Children’s Hospital of Philadelphia. Grupp spearheaded the clinical trials of the newly approved ALL therapy, called Kymriah.

Of the 63 kids and young adults treated in a clinical trial of Kymriah, 83 percent had their cancers go into remission within three months.

One drawback is the price. Kymriah costs $475,000 for a onetime treatment, according to Novartis, which makes Kymriah. The non-Hodgkin lymphoma treatment made by Gilead Sciences, called Yescarta, is listed at $373,000.

Read full, original post: Approval of gene therapies for two blood cancers led to an ‘explosion of interest’ in 2017

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