A panel of cancer experts Wednesday unanimously endorsed a leukemia treatment that could be the first gene therapy available in the U.S.
The Food and Drug Administration (FDA) advisory panel voted 10-0 to recommend approval of the treatment, which was devised by the University of Pennsylvania and Novartis Corp. The FDA is not required to follow the panel’s recommendation, but often does.
The one-time treatment would be used on children and young adults with advanced leukemia.
The therapy could be the first of a wave of treatments custom-made to target a patient’s cancer. Called CAR-T, it involves removing immune cells from a patients’ blood, reprogramming them to create an army of cells that can recognize and destroy cancer and injecting them back into the patient.
“This is a major advance,” said panel member Dr. Malcolm A. Smith of the National Cancer Institute. He said the treatment is “ushering in a new era.”
The vote came after lengthy discussion and impassioned pleas from the fathers of two young patients whose lives were saved by the therapy. The one-time leukemia treatment would be for children and young adults with the most common form of childhood cancer, known as ALL.
“Our daughter was going to die and now she leads a normal life,” said Tom Whitehead, of Philipsburg, Pa. His daughter Emily, now 12, was the first child to receive the experimental therapy, five years ago. “We believe when this treatment is approved, it will save thousands of children’s lives around the world.”
Novartis is seeking approval to use the treatment for patients aged 3 to 25 with a blood cancer called acute lymphoblastic leukemia whose disease has spread or failed to respond to standard treatment. That happens to more than 600 patients in the U.S. each year. At that point, they have limited options — all more toxic than the CAR-T therapy — and survival chances are slim. ALL accounts for a quarter of all cancers in children under age 15.
In a key test, results were far better than chemotherapy and even newer types of cancer drugs. Of the 52 patients whose results were analyzed, 83 percent had complete remission, meaning their cancer vanished. Most patients…