The treatment, called Yescarta, treats a type of blood cancer called aggressive B-cell non-Hodgkin lymphoma.
The highly personalized cancer treatment called CAR T-cell therapy (CAR is short for chimeric antigen receptor) is made by Kite Pharma, a company that in August was acquired by biotech giant Gilead Sciences.
It's a type of cancer immunotherapy, which harnesses the body's immune system to take on cancer cells. In this case, the therapy removes a person's cells, reengineers them, then puts them back in their body to attack cancer cells.
In August, the FDA approved another CAR-T therapy, called Kymriah, to treat treat pediatric acute lymphoblastic leukemia in people up to age 25. The treatment was heralded as the first gene therapy, an area that's gaining traction as other treatments get closer to approval.
"Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer," FDA Commissioner Scott Gottlieb said in a news release.
How CAR-T therapies work
Yescarta isn't your run-of-the-mill pill - or even a biologic drug, like insulin - that can be mass produced. Since the therapy is made from a person's own immune system, the process can take about three weeks.
- To start, a doctor removes some white blood cells, the part of our body's immune system responsible for combatting infections and foreign substances, from a patient. In a healthy body, the immune system can recognize abnormal, cancerous cells, but for people with cancer, it doesn't recognize that the cells are spreading.
- Then the cells are taken to a manufacturing facility at which point the cells are reengineered to recognize cancer cells and wipe them out.
- Those reprogrammed cells are sent back and administered to the patient.
Ana Pelisson/Business Insider; Novartis
The one-time treatments won't come cheap. While Gilead might price the drug based on how well it works or opt for a refund if there hasn't been a response, as is the case with Kymriah, Yescarta's price tag could still be $325,000. Analysts expect the therapy to generate $1.7 billion in sales by 2022.
'A big new field of medicine'
Kymriah, and Yescarta are the first two CAR-T cell therapies to get approved, and several more are in the works. Juno Therapeutics, and Bluebird Bio are among a growing group of biotech companies working with CAR-T.
"We're at the very beginning of what's going to be a big new field of medicine," said David Epstein, who helped license Kymriah from the University of Pennsylvania while at Novartis.
Epstein left Novartis in 2016 as CEO of its pharmaceuticals divisions. He's now the executive chairman of Rubius Therapeutics, a biotech firm that's also working with cell therapy to develop treatments like Kymriah that don't have to be as personalized. The hope is that one day doctors will be able to prescribe a cell therapy and use it that same day instead of waiting weeks to get it back, and possibly extend beyond the blood cancers treatments like Kymriah and axi-cel are able to treat.