US approval for breakthrough cancer gene therapy

Updated / Thursday, 31 Aug 2017 14:23

The US Food and Drug Administration described the approval as 'a historic action'

A treatment that uses a patient's own immune cells to fight leukemia has received approval in the United States, the first time any gene therapy has done so.

The move is seen as opening a new era in the fight against cancer.

The breakthrough treatment is made by Novartis and is called Kymriah, or tisagenlecleucel.

This type of immunotherapy is known as Chimeric Antigen Receptor T Cell or CAR-T cell therapy.

It was approved by the US Food and Drug Administration for children and young adult patients up to 25 years old with a form of acute lymphoblastic leukemia.

The FDA described the approval as "a historic action" that would usher "in a new approach to the treatment of cancer and other serious and life-threatening diseases".

Studies have shown that 83% of patients responded to the one-time only 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, reprogrammed, and then returned to the patient.