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FDA announces first U.S. gene therapy approval for cancer treatment

FDA announces first US gene therapy approval for cancer treatment (Photo: CNN Newsource)

LYNCHBURG, Va. (WSET) -- The Food and Drug Administration announced the approval of a leukemia treatment that it considers to be the first gene therapy cleared for the U.S. Market.

The treatment, called Kymriah, is an additional option for patients who haven't had success with first-line drugs.

It contains a patient's own, genetically modified immune cells, reprogrammed to recognize and kill the cancer.

"This is a brand new way of treating cancer," said Dr. Stephan Grupp of Children's Hospital of Philadelphia to ABC News, who treated the first child with CAR-T cell therapy — a girl who'd been near death but now is cancer-free for five years and counting. "That's enormously exciting."

Kymriah is aimed at treating relapses in B-cell acute lymphoblastic leukemia, which is the most common type of cancer among children.

The treatment costs $475,000, but patients who don't respond after a month won't be charged.

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