Breakthrough gene therapy for cancer approved in US

Novartis drug Kymriah, costing $475,000, alters patient’s own cells to fight cancer

A US authority has approved the first-ever treatment that genetically alters a patient’s own cells to fight cancer, a milestone that is expected to transform treatment in the coming years.

The new therapy, which would cost just under €400,000, turns a patient’s cells into a “living drug” and trains them to recognise and attack the disease. It is part of the rapidly growing field of immunotherapy that bolsters the immune system through drugs and other therapies and has, in some cases, led to long remissions and possibly even cures.

The Food and Drug Administration has given the therapy, marketed as Kymriah and made by Novartis, the greenlight to treat children and young adults with an aggressive type of leukaemia - B-cell acute lymphoblastic leukaemia - that has resisted standard treatment or relapsed.

Novartis and other companies have been racing to develop gene therapies for other types of cancers, and experts expect more approvals in the near future.


Dr Scott Gottlieb, the FDA commissioner, said that more than 550 types of experimental gene therapy were being studied.


There are drawbacks to the therapy such as life-threatening side effects, including dangerous drops in blood pressure and the FDA has said hospitals and doctors should be trained and certified to administer it, and that they stock a certain drug needed to quell severe reactions.

Kymriah, which will be given to patients just once and must be made individually for each patient, will cost $475,000.

Novartis said that if a patient does not respond within a month of the treatment beginning, there will be no charge. The company also said it would provide financial help to families who were uninsured or underinsured.

About 600 children and young adults a year in the United States would be candidates for the treatment. The approval was based largely on a trial in 63 severely ill children and young adults who had a remission rate of 83 per cent within three months - a high rate, given that relapsed or treatment-resistant disease is often quickly fatal.

The treatment was originally developed by researchers at the University of Pennsylvania and licensed to Novartis.

Dr. Kevin J. Curran, a paediatric oncologist at Memorial Sloan Kettering Cancer Centre in Manhattan, said he expected that eventually this type of treatment would work for other types of cancer.

“This is a big paradigm shift, using this living drug,” Curran said. “It will provide a lot of hope. This is the beginning.”

New York Times