The treatment known as CTL019 was developed by lead researcher Carl June at the University of Pennsylvania and licensed to Novartis as a treatment for pediatric acute lymphoblastic leukemia.

It works by taking a patient’s own white blood cells, called T-cells, then altering them to allow them to recognize and kill cancer cells.

The treatment is best known for saving the life of a young girl named Emily Whitehead, who is now 12 and has been cancer-free since she underwent the experimental therapy at age six. She attended Wednesday’s hearing with her parents.

In 2014, US regulators designated CTL019 as a “breakthrough therapy” and put the experimental immunotherapy agent on the fast track to market approval, after 89 percent of leukemia patients in early trials saw their cancers disappear.

It was the first cancer immunotherapy to receive the breakthrough designation. More are expected to follow in the coming years as the field of immunotherapy grows. 

The approach works by extracting a patient’s T-cells, then genetically programming them in the lab to target cancer cells that produce a protein called CD19.

The altered T-cells are then re-injected into the patient’s body, where they multiply and attack cancer.

Side effects, however, can be severe, and include spiking fever and crashing blood pressure.

“Although this therapy is technologically somewhat complicated and is associated with certain serious side effects, it, indeed, has been proven to be amazingly effective,” said Kanti Rai, chief of the chronic lymphocytic leukemia research and treatment program at Northwell Health Cancer Institute in New York.

Rai described the panel’s recommendation as “the first important forward step in our road to conquer cancer.”

The FDA does not have to accept the recommendations of its advisory panel, but usually does. DM