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Saturday
Sep022017

First gene therapy that targets leukemia approved in the US 

iStock/Thinkstock(NEW YORK) -- The Food and Drug Administration has approved a landmark treatment that genetically modifies a patient's blood cells to target and destroy childhood leukemia.

The treatment bolsters T cells, which researchers siphon from a patient's blood and then program to hold a chimeric antigen receptor that targets cancer. It also causes them to multiply by the hundreds of thousands, which allows T cells to continue fighting diseases.

Called "CAR-T cell therapy," it is the first gene therapy to enter the U.S. market. Novartis Pharmaceuticals priced the one-time infusion at $475,000. However, the company said it would waive costs for patients who didn’t respond to the treatment within a month. 

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