The Food and Drug Administration on 06 September 2017 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. The disease is a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About 3,100 patients who are 20 and younger are diagnosed with ALL each year.
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. Based on those results, an FDA advisory panel recommended the approval in July.