FDA approves gene therapy to treat a rare cancer

On August 30, the U.S. Food and Drug Administration approved a novel gene therapy for patients with a rare type of leukemia. This is the first time the agency has greenlighted a gene therapy approach for use in the United States.

The treatment, called CAR-T immunotherapy, uses genetically engineered T cells, immune system fighters usually tasked with identifying invaders in the body, such as bacteria, viruses or foreign cells. The engineered cells, called CAR-T cells, are customized for each patient. An individual’s T cells are collected and altered in the lab to carry a new gene that directs the T cells to attack certain cancer cells. Those modified T cells are injected back into the patient to seek out and destroy the cancer.

CAR-T cell therapy has shown promise in fighting a type of cancer called B-cell acute lymphoblastic leukemia, or ALL, which can strike children and adults. Several CAR-T approaches are in the works, including an experimental therapy that made headlines in 2015 when it was used in England to treat a 1-year-old girl with leukemia.

The FDA approved an approach to CAR-T therapy manufactured by Novartis Pharmaceuticals, called Kymriah. The approval applies to children and young adults (up to age 25) with B-cell ALL that doesn’t respond to treatment or has relapsed.