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Ground-Breaking Immunotherapy for Childhood Leukemia Gets FDA OK


A new treatment designed to help children and young adults battling recurrence of the most common type of childhood cancer received approval from the U.S. Food and Drug Administration (FDA) this week.

The therapy is called CAR-T cell therapy. It involves taking cells from a patient’s blood, re-engineering them in a lab to attack a specific protein on the cancerous cells, and returning them to the patient’s bloodstream, where they seek out and destroy cancer cells, says oncologist Mikkael Sekeres, MD, MS, Director of Cleveland Clinic Cancer Center’s Leukemia Program.

“We take that immune system, treat it and expose it to the leukemia cells itself, so that what grows out is an immune system that is specific for the leukemia — that knows how to attack the leukemia,” Dr. Sekeres says. “That immune system is then re-infused back into the patient so that, hopefully, the leukemia will disappear.”

Anti-rejection drugs, which usually are needed after transplants, are unnecessary because the cancer-fighting cells are from the patient’s own body, Dr. Sekeres says.

First leukemia therapy of its kind