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The FDA Just Approved the First 'Living' Therapy to Treat Childhood Leukemia

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On Wednesday, the Food and Drug Administration approved a treatment that genetically modifies a patient’s own blood cells in order to attack childhood leukemia, a landmark decision opens the door for an era of FDA-sanctioned human gene modification.

The treatment, called CAR-T, is made by the drug company Novartis. Doctors first harvest a patient’s white blood cells and then, in the lab, reprogram them into tumor assassins. The cells are then placed back into the body where they zero in on and destroy cancerous cells. Novartis’ drug, Kymriah, is approved to treat children and young adults who relapse after initial treatment for a rare and deadly form of blood cancer, acute lymphoblastic leukemia. (The FDA called it the first-approved “gene therapy,” though experts quibble over whether that term applies.)


The drug was one of several CAR-T therapies rushing to be the first to market. In a key clinical trial of 63 people, 83 percent of participants were cancer-free after three months. (Juno, another company developing a CAR-T therapy, was forced to halt its trial earlier this year after patient deaths.)


Some of those patients, though, did eventually relapse after several months. And the drug comes with serious side-effects, including a reaction known as cytokine release syndrome, that can prove fatal if not controlled with immunosuppressant drugs.

Novartis did not disclose how much it intends to charge for the game-changing drug, but analysts have projected it will likely cost hundreds of thousands of dollars for a course of treatment.

On Twitter, FDA commissioner Scott Gottlieb called the approval historic.

“Today is an important milestone in a long journey to transform clinical medicine using modern advances in genomics,” he said.