The US Food and Drug Administration (FDA) approved first gene therapy, a treatment that uses patient’s own immune cells to fight acute lymphoblastic leukaemia.
The approval will usher new approach to treatment of cancer and other serious and life-threatening diseases. Studies have shown that 83% of patients responded to this treatment, achieving remission within three months.
The gene therapy treatment was originally developed by researchers at University of Pennsylvania and licensed to pharma giant Novartis. It was previously identified as CAR-T cell therapy (CTL019) or tisagenlecleucel and now is called Kymriah.
The treatment is completely different compared to present popular immunotherapy drugs called “checkpoint inhibitors” used to harness immune system that treat variety of cancers by helping the body’s natural T cells better spot tumors.
The CAR-T treatment is not a pill or form of chemotherapy. It uses gene therapy techniques not to fix disease-causing genes (cells) but to turbocharge T cells, immune system cells that cancer can evade. These cells removed from patient’s blood along with white blood cells are encoded with viral vector, reprogrammed in lab. They are reprogrammed to harbor chimeric antigen receptor (CAR) that specifically targets cancer cells. The revived and reprogrammed cells after returned to the patient continue multiplying to fight disease for months or years.