FDA Approves First Gene Therapy

FDA Approves First Gene Therapy
August 30, 2017

The U.S. Food and Drug Administration made a historic announcement on August 30, 2017 making the first gene therapy available in the United States. The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia.

Scientists refer to Kymriah as a “living drug” because it involves using genetically modified immune cells from patients to attack their cancer. “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said in a written statement.

The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It’s also known as CAR-T cell therapy.

The treatment, which is also called CTL019, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses.

Watch the news coverage in CNN video. Read the full press release by the U.S. Food and Drug Administration here.