This month, Novartis’ Kymriah became the first gene-therapy drug to receive US FDA approval. The $475,000 drug was approved for use to treat B-cell acute lymphoblastic leukemia in individuals under 25 years old who have not responded to other therapies. This is about 600 people per year. The CAR T-cell therapy removes T-cells from the body and inserts a gene designed to attack leukemia cells into them. The T-cells are then introduced back into the patient. Because of the possibility of serious side effects, Novartis is limiting distribution to hospitals and personnel that have been appropriately trained. Other types of cancer have also been shown to respond to such a treatment, and Novartis will seek approval this year of a similar drug to treat non-Hodgkin lymphoma in adults.

Source: Washington Post