A Food and Drug Administration (FDA) panel unanimously said “yes” to a gene therapy to fight off leukemia, a type of cancer in the blood or bone marrow. The gene therapy was developed by Novartis, a global healthcare company. The treatment could become the first gene therapy that is available in the United States.
The gene therapy is called CAR-T. How does it work? It would require taking out immune cells from the blood. The cell will then be reprogrammed to create a new group of cells to destroy cancer. It is then injected back into the patient. A clinical trial for 63 people with acute lymphoblastic leukemia took place, and 52 patients went into remission after the treatment. The cost of the therapy would be hundreds of thousands of dollars, but the treatment happens only once.
Parents of children living with leukemia say if the treatment is approved, it will save thousands of lives around the world. They are begging the FDA to approve the treatment. Those who receive the treatment will be put in a registry and will be monitored for 15 years. The FDA is expected to make a decision to approve the treatment by October, 2017.