FDA votes to recommend gene-therapy leukemia treatment

FDA votes to recommend gene-therapy leukemia treatment

FDA votes to recommend gene-therapy leukemia treatment

The panel of cancer experts voted 10-0 Wednesday, recommending that the treatment be approved.

Despite unknowns surrounding the therapy, "it is hard to argue with the unprecedented clinical success we have seen in this population of patients who do not have other viable treatment options", added Grzegorz S. Nowakowski, MD, of the Mayo Clinic in Rochester, Minn.

The new drug class, known as chimeric antigen receptor T-cell or CAR-T, involves a complicated process of extracting immune system T-cells from an individual patient, altering the cell DNA to sharpen their ability to spot and kill cancer cells, and infusing them back into the patient.

The FDA is not required to follow the panel's recommendation but often does.

If cleared by the FDA, it would be the first gene therapy approved in the United States.

Yesterday, the FDA's Oncologic Drugs Advisory Committee (ODAC) held a hearing to evaluate Novartis CTL019. Novartis is seeking approval for use in children and young adults with leukemia who have relapsed despite undergoing chemotherapy. The drug was tried on patients whose cancer had relapsed or didn't respond to standard treatments.

When a patient is treated under the Novartis process, T cells are extracted from a patient's blood, frozen and sent to the company's plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.

"While CAR-T is a promising new type of immunotherapy, it is not commercially available and we have yet to complete our evaluation", said T.J. Crawford, a spokesman for Aetna Inc.

Novartis' groundbreaking cancer T-cell therapy CTL019 has won unanimous backing from advisers to the FDA, paving the way for approval in the coming months. Novartis's therapy commandeers the body's immune system to destroy the cells where the blood cancer begins.

He said in a blog: "With every paper I read, it becomes more and more clear that CAR-T is not a biotech race to be won, but rather a revolutionary therapy to be explored and applied to other cancers".

The FDA is expected to decide whether to approve the Novartis treatment in the next few months.

The last three years of development work at Novartis has been marked by organizational restructuring along with the advance and retreat of rival therapies - all while sorting through a threatening list of toxicity factors. He said the treatment is "ushering in a new era". Her father said his family was more afraid of the side effects of the full-body radiation that's used ahead of a transplant than of the experimental immunotherapy.

In this pivotal study informing the committee's decision, roughly half of 68 patients receiving the drug experienced this complication, though none died from it. She was the first child to receive the treatment.

Part of Novartis' education effort will likely address safety concerns as several companies have reported patient deaths in clinical trials for CAR-T therapies. Researchers also are exploring auto T-cell therapy's use for multiple myeloma and chronic lymphocytic leukemia, the disease that afflicted Ludwig.

In addition to understanding that process, the staffers wrote, Novartis will need to understand critical product quality attributes unique to autologous vehicle T-cell products-as well as to understand and address sources of variability seen in the products.

Related news