FDA approves first gene therapy for certain leukemia patients


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. Read 11 remaining paragraphs | Comments

FDA approves first gene therapy for certain leukemia patients


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