US 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.
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."
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.
At that point, patients are generally out of options and only have about a 10% chance of even temporary remission of their cancer.
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.
A different type of gene therapy is waiting in the wings at the FDA.
Spark Therapeutics’ treatment for a rare form of blindness could be approved within months. It aims to improve vision by replacing a defective gene needed to process light.
Other gene therapies for blood cancers are being tested and scientists think they may work for solid tumours within several years.
