FDA Approves First Gene Therapy for Leukemia

August 31, 2017

(NPR) – The Food and Drug Administration on Wednesday announced what the agency calls a “historic action” — the first approval of a cell-based gene therapy in the United States. The FDA approved Kymriah, which scientists refer to as a “living drug” because it involves using genetically modified immune cells from patients to attack their cancer. The drug was approved to treat children and young adults up to age 25 suffering from a form of acute lymphoblastic leukemia who do not respond to standard treatment or have suffered relapses.