Novel leukemia treatment could be 1st U.S. 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 CART, 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 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.”

After decades of setbacks and disappointments in efforts to fix, replace or change genes to cure diseases, several companies are near the finish line in a race to bring CAR-T and other types of gene therapy to patients. Kite Pharma also has a CAR-T therapy under FDA review and Juno Therapeutics and others are in late stages of testing.

The FDA is expected to decide whether to approve the Novartis treatment in the next few months. The drugmaker 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 suffered serious side effects. Eleven patients died, four from side effects and seven from their leukemia.

Novartis said in a statement after the vote that it has long believed CAR-T therapy could “change the cancer treatment paradigm.”