US Food and Drug Administration Approves First Gene Therapy

August 30, 2017

Today the U.S. Food and Drug Administration (FDA) approved Kymriah, the first-ever gene therapy for cancer. It will be used to treat children and young adults with leukemia.

Kymriah, better known as CAR T-cell treatment, entails modifying a patient’s own T-cells genetically, and then using them to target and kill B-cell acute lymphoblastic leukemia cells. The treatment has been called revolutionary and signals a new era in cancer treatment.

Disease-fighting T-cells are removed from a patient and then genetically modified so that they can better recognize and attack cancer cells. Once put back into the body, the T-cells can circulate for years attacking the disease.

For children with B-cell acute lymphoblastic leukemia, blood is drawn and then the T-cells are shipped to a lab where they are genetically engineered. Patients are then infused with the modified blood, which allows the T-cells to find and kill the leukemia.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp.
The initial treatment will cost $475,000 and additional treatments will be free.

To date, the FDA has granted 76 applications for trials involving experimental CAR-T therapies. The new treatment has the potential to work in other cancer patients whose T-cells can be collected and modified, and then used to target tumors.