Novartis leukemia treatment could be first US gene therapy
A treatment for a common childhood blood cancer could become the first gene therapy available in the U.S.
A Food and Drug Administration advisory panel voted 10-0 on Wednesday in favor of the leukemia treatment developed by the University of Pennsylvania and Novartis Corp. The FDA usually follows recommendations from its expert panels, but isn't obligated to do so.
The therapy could be the first of a wave of treatments custom-made to target a patient's cancer. Called CAR-T, this type of therapy involves removing immune cells from a patients' blood, reprogramming them to create an army of cells that can zero in on and destroy cancer cells 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.