The US Food and Drug Administration approved a new leukemia treatment, which the agency considers the first gene therapy. This treatment uses a patient’s own immune cells to fight childhood leukaemia.
The treatment is made by Novartis and is called Kymriah (tisagenlecleucel).
Each dose of Kymriah contains a patient's own immune cells, which are sent to a lab to be genetically modified using a virus. This therapy -- known as chimeric antigen receptor T-cell therapy, or CAR-T, was known by the term CTL019 until now -- gives the cells the ability to recognize and kill the source of cancer.
It was approved by the U.S. Food and Drug Administration for children and young adult patients up to age 25 with a form of acute lymphoblastic leukaemia.