Gene treatment approved for second cancer
The FDA approves a second version of a groundbreaking treatment that genetically alters patients’ cells to attack cancer — this time, to fight aggressive non-Hodgkin lymphoma.
The Food and Drug Administration approved a second version of a groundbreaking treatment Wednesday that genetically alters patients’ cells to attack cancer — this time, to fight aggressive nonHodgkin lymphoma.
The treatment is for adults with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment, such as chemotherapy and bone-marrow transplants. The group numbers about 7,500 patients a year in the United States.
The one-time infusion, known as CAR T-cell therapy, is made by Kite Pharma, which is based in Santa Monica, Calif., and recently was bought by Gilead Sciences for $11.9 billion. Kite announced Wednesday that the treatment’s brand name will be Yescarta and its price will be $373,000.
In late August, the FDA cleared the first CAR T-cell therapy, which is designed for children and young adults whose leukemia doesn’t respond to standard treatments. About 600 patients in the United States fall into that category every year. Kymriah, which costs $475,000, is manufactured by Novartis.
Biotech analysts had expected the Kite price to be lower than Kymriah’s, in part because the number of eligible patients is larger and the response rates are lower. Even so, Yescarta’s cost is likely to stoke the ongoing debate about high drug prices.
The FDA approval is the latest step forward for the fast-moving field of immunotherapy, which aims to bolster the immune system to attack malignancies. CAR T-cell therapies are among several approaches, along with treatments called checkpoint inhibitors and cancer vaccines, but they have recently grabbed much of the attention. Dozens of other companies also are working on them.
“Today, marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases,” FDA Commissioner Scott Gottlieb said in a statement, adding that the approval demonstrates “the continued momentum of this promising new area of medicine.”
A CAR T-cell therapy involves a complicated and customized procedure in which T cells — sometimes called the foot soldiers of the immune system — are removed from the patient. They are sent to a special lab and genetically modified to target a protein on the surface of the patient’s cancer cells. Once the modified cells are returned to the patient, their numbers expand exponentially as they become an army of cancer fighters.
The FDA said the safety and efficacy of Yescarta were established in a multi-center trial of more than 100 adults with large B-cell lymphoma.
“This is not just an incremental benefit,” said David Chang, Kite’s chief medical officer. “It raises the potential that a cure can be possible.” Some of the first patients who underwent treatment now have been in remission for three to five years, he said, although he cautioned that it’s still too early to know whether those patients are cured.