The United States approved the first gene therapy in the nation — a treatment that uses a patient’s own immune cells to fight childhood leukaemia.
The treatment is made by Novartis and is called Kymriah (tisagenlecleucel).
This type of immunotherapy, known as a CAR-T cell therapy, was known by the term CTL019 until now.
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.
Studies have shown that 83% of patients responded to the treatment, achieving remission within three months.
The treatment is not a pill or a form of chemotherapy. It uses a patient’s own immune cells, called T-cells, along with white blood cells.
These cells are removed from a patient, sent to a lab, and encoded with a viral vector, reprogrammed, and returned to the patient.