- U.S. regulators approved a second gene therapy for a blood cancer, a one-time, custom-made treatment for aggressive lymphoma in adults.
- The Food and Drug Administration allowed sales of the treatment from Kite Pharma.
- It uses the same technology, called CAR-T, as the first gene therapy approved in the U.S. in August, a treatment for childhood leukemia from Novartis Pharmaceuticals.
- 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.
- The treatment, called Yescarta, will cost $373,000 per patient, according to drugmaker Gilead Sciences. Kite became a subsidiary of Foster City, California-based Gilead this month.
- CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer can often evade.
- The T cells are filtered from a patient’s blood, reprogrammed to target and kill cancer cells, and then hundreds of millions of copies are grown.
- Returned to the patient, all the revved-up cells can continue multiplying to fight disease for months or years. That’s why these immunotherapy treatments are called “living drugs.”
- “Today’s approval of Yescarta is a very significant advance for lymphoma patients and for the cancer community as a whole.
- Kite’s therapy is for patients with three types of aggressive, or fast-growing, large B-cell lymphoma.
- The most common one accounts for about a third of the estimated 72,000 new cases of non-Hodgkin lymphoma diagnosed each year.
- Yescarta, also known as axicabtagene ciloleucel, was approved for patients who have already been treated with at least two cancer drugs that either didn’t work for them or eventually stopped working.
- Yescarta is not a benign treatment, though — three people died after getting the treatment, which can cause serious side effects.
- The FDA is requiring Kite to do a long-term safety study and train hospitals to quickly spot and handle those reactions.
- In the key test, Yescarta was given to 101 patients. About 72% saw their cancer shrink and about half showed no sign of disease eight months later.