The U.S. Food and Drug Administration has issued a briefing document that accepts the efficacy of a type of CAR T-cell therapy in treating advanced cases of leukemia in children and young adults. The documents and other material were posted two days before the July 12 meeting of the agency’s Oncologic Drugs Advisory Committee, which will vote whether to recommend that the treatment, made by Novartis, should be approved for use, and if so, what requirements will accompany its marketing and distribution.
The staff’s review of the evidence said that questions remain about whether the Swiss pharmaceutical company can reliably produce and deliver the CAR T-cell therapy on a larger scale. It also raised questions about how long the treatment will benefit children and young adults whose cancer goes into remission after receiving it.
In response to the report, Dr. David Maloney, medical director of cellular immunotherapy at Fred Hutchinson Cancer Research Center and medical director of the Bezos Family Immunotherapy Clinic at Seattle Cancer Care Alliance, said:
“Fred Hutch welcomes the report from the U.S. Food and Drug Administration about moving a CAR T-cell therapy forward as a treatment for advanced cases of leukemia in children and young adults. Its recommendations could lead to more patients with leukemia receiving this lifesaving therapy in the coming months, and it affirms the promise of cellular immunotherapy as a treatment for other cancers. While the FDA briefing document highlights how CAR T-cell therapy needs further research, we at Fred Hutch are working every day in labs and in clinics to advance our understanding of these therapies and to make them even more effective and safe. The Hutch’s efforts are moving us closer to the day when these therapies are available to more patients around the world.”