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.