First genetic engineering therapy approved by the FDA for leukemia

Enlarge / Scanning electron micrograph of a human T cell. (credit: NIAID/NIH)

For the first time, the Food and Drug Administration has approved a therapy that involves genetically engineering a patient's own cells, the agency announced Wednesday.

The therapy, called Kymriah (tisagenlecleucel) by Novartis, will be used to reprogram the immune cells of pediatric and young adult patients with a certain type of leukemia, called B-cell acute lymphoblastic leukemia. During a 22-day out-of-body retraining, patients' immune cells-specifically T cells that patrol the body and destroy enemies-get a new gene that allows them to identify and attack the leukemia cells.

Such therapies, called CAR-T therapies, have shown potential for effectively knocking back cancers in several trials, raising hopes of researchers and patients alike. But they come with severe safety concerns-plus potentially hefty price tags.

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