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Medical News & Perspectives
December 12, 2017

The Promise and Challenges of CAR-T Gene Therapy

JAMA. 2017;318(22):2167-2169. doi:10.1001/jama.2017.15605

The recent approvals of 2 one-time gene therapies for refractory pediatric acute lymphoblastic leukemia (ALL) and adult relapsed or refractory diffuse large B-cell lymphoma mark a milestone for gene therapy and may open up a new treatment option for thousands of patients for whom traditional cancer therapies have failed.

In August, the US Food and Drug Administration (FDA) approved tisagenlecleucel, a chimeric antigen receptor T-cell (CAR T-cell) therapy for the treatment of patients with refractory B-cell precursor ALL who are up to 25 years old. About 3100 patients a year develop B-cell precursor ALL, and 15% to 20% do not respond to or experience a relapse after traditional therapies, according to the FDA. This treatment is indicated for the subset of this group who have been unsuccessfully treated with other therapies more than once. In the therapy, a patient’s own T cells are removed and genetically engineered to recognize and destroy leukemia cells that have the CD19 antigen on their cell surface. Then, the cells are infused back into the patient. The treatment will be offered at about 30 centers.