A new cancer treatment endorsed Wednesday by the Federal Drug Administration's Oncologic Drugs Advisory Committee is being described by one panel member as "the most exciting thing I've seen in my lifetime."
CAR T-cell therapy, made by Novartis, retrains a patient's immune cells so they target and attack cancer; the cells are removed from the body, sent to Novartis' plant so they can be genetically modified, and then shipped back to the patient for infusion. Novartis is seeking approval for use in children and young adults with leukemia who have relapsed despite undergoing chemotherapy. Stephen Grupp, the oncologist who led Novartis' trial, said most of the patients experienced cytokine release syndrome, which results in fever and flulike symptoms, and some had seizures and delirium.
If the FDA approves CAR T-cell therapy, which could happen by the end of September, it would be the first gene therapy cleared for use in the United States. Thomas Whitehead's daughter, Emily, was the first child to go through the treatment, at age six. She had leukemia and was close to death, he said, and while the CAR T-cell therapy nearly killed her, she survived and no longer has cancer, The Washington Post reports. He spoke in front of the Oncologic Drugs Advisory Committee on Wednesday and told them, "If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she's standing right beside me."