Novel cancer treatment wins endorsement of FDA advisers

Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for desperately ill children far outweigh its potentially dangerous side effects.

The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a new path in the immunotherapy frontier.

Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most exciting thing I’ve seen in my lifetime.”

Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and young adults whose leukemia doesn’t respond to traditional treatments — a group that numbers 600 or so patients a year in this country. But the approach also is being tested for a range of diseases from nonHodgkin lymphoma and multiple myeloma to solid tumors.

If cleared by the FDA, it would be the first gene therapy approved in the United States. But unlike traditional gene therapy, the new treatment doesn’t replace disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.

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.

Once inside the body, the cells multiply exponentially and go hunting for the CD19 protein, which appears on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated 22 days, Novartis officials told the committee Wednesday.

From the start of Wednesday’s meeting, committee members made clear that they were not concerned about the treatment’s efficacy, which has been well established — 83 percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to handle possible shot-term toxicities, as well as longterm safety risks and manufacturing quality.