FDA Approves First U.S. Gene Therapy Cancer Treatment

A cancer research scientist examining cells in a 96-well plate / Getty Images
A cancer research scientist examining cells in a 96-well plate / Getty Images

The U.S. Food and Drug Administration has approved, for the first time in the United States, a gene therapy aimed at treating cancer.

The new therapy, Kymriah, is believed to treat leukemia by allowing the body to better target cancer cells. The therapy takes a sample of the patient's immune cells and modifies them with a virus. The patient's modified cells can turn around and better target the cancer cells, CNN reported Wednesday.

Kymriah allows patients, for whom first line drugs failed, another chance.

FDA Commissioner Dr. Scott Gottlieb released a statement on Kymriah's potential for patients, saying it will revolutionize cancer treatment.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," Gottlieb said.

It is predicted, from the data available to date, that 89 percent of patients who use the therapy will survive at least six months while 79 percent will make it at least a year. At the one year mark, most patients would be relapse-free.

At this point, the revolutionary treatment developed by Swiss multinational pharmaceutical company Novartis is not a first option. The therapy comes with potentially serious side effects, and a $475,000 price tag. The company says, however, that patients who do not respond within a month of treatment will not be charged.

"Novartis is collaborating with (Centers for Medicaid Services) to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month. Future potential indications would be reviewed for the most relevant outcomes-based approach," the company said in a statement.

Gene therapy has increasingly become a potential treatment explored by scientists, and many are excited about the development, in part, because of where it may lead in the fight against cancer and other diseases.