Heidi Ledford in Nature:
External advisers to the US Food and Drug Administration (FDA) have thrown their support behind a therapy that genetically engineers a patient’s own immune cells to target and destroy cancer cells. In a unanimous vote on 12 July, the panel determined that the benefits of the treatment, called CAR-T therapy, outweigh its risks. The vote comes as the agency considers whether to issue its first approval of a CAR-T therapy — a drug called tisagenlecleucel that's manufactured by Novartis of Basel, Switzerland. The FDA is not obligated to follow the recommendations of its advisers, but it often does.
Novartis is seeking approval to use tisagenlecleucel to treat children and young adults that have a form of acute leukaemia, and who have not sufficiently responded to previous treatment or have relapsed since that treatment. In the United States, about 15% of children and young adults with acute leukaemia relapse following treatment. Studies have shown that CAR-T therapies can produce lasting remissions in such cases. In one key trial of tisagenlecleucel, which started in 2015, 82.5% of 63 patients experienced overall remissions. The trial lacked a control group, so investigators cannot yet say with certainty how much of an effect the treatment had. But many of those participants have remained cancer free for months or years. Many of the FDA's advisers were effusive in their praise. "This is a major advance, and is ushering in a new era," said Malcolm Smith, a paediatric oncologist at the National Institutes of Health in Bethesda, Maryland. Timothy Cripe, an oncologist at Nationwide Children's Hospital in Columbus, Ohio, called it one of the most exciting things he has seen in his lifetime.