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• Posted January 24, 2024

FDA Warns of Rare Secondary Cancer Risk With CAR-T Therapies

WEDNESDAY, Jan. 24, 2024 (Healthday News) -- The U.S. Food and Drug Administration has told drugmakers to add a boxed warning to a type of cancer treatment called CAR-T therapy, saying the treatment itself may sometimes cause a secondary cancer.

Still, FDA spokesperson Carly Kempler told NBC News that, despite the new warning, "the overall benefits of these products continue to outweigh their potential risks."

Twenty-five reports of rare blood cancers in patients who had gotten CAR-T therapy prompted the agency to add the boxed warning, Kempler said.

CAR-T therapy uses a patient's own immune cells to fight blood cancers such as leukemia, multiple myeloma and lymphoma. Immune cells are harvested from the patient and then genetically altered in a lab to make them target cancer cells. Once tweaked, the immune cells are infused back into the patient.

It's a powerful therapy: In 2022, doctors who had treated two leukemia patients with CAR-T a decade ago reported that the treatment had essentially cured the patients.

“This has been a game changer when we think about treating lymphoma and other diseases,” Dr. Matthew Frigault, clinical director of the Massachusetts General Hospital Cellular Immunotherapy Program, told NBC News.

In 2017, the first CAR-T therapy, Novartis' drug Kymriah, was approved by the FDA. Another five therapies have since been approved.

The makers of five of these drugs -- Bristol Myers Squibb, for Abecma and Breyanzi; Gilead Sciences' Kite Pharma, for Yescarta; Johnson & Johnson's Carvykti; and Novartis, for Kymriah -- must submit proposed label changes in the next 30 days to note that CAR-T therapy can raise the risk of rare blood cancers, the FDA said.

If the drugmakers disagree, they can submit a rebuttal explaining why a change isn't needed, NBC News reported.

In a statement, a spokesperson for Novartis said the company has not found “sufficient evidence” to support a link between cancer and its treatment. However, the company will work with the FDA to update its label “appropriately,” the spokesman said.

Spokespersons for Johnson & Johnson and Gilead Sciences also told NBC News that they would work with the agency to update their labels.

A spokesperson for Bristol Myers Squibb said the company is evaluating “next steps” following the FDA's notice, although it has not seen any cancer cases associated with its treatment.

CAR-T treatments are still relatively new, Frigault noted, so the FDA has required the makers of these therapies to conduct 15-year follow-up studies to measure the potential risk of secondary cancers.

The FDA “is not saying that every single one of the cases they've reported has clearly shown CAR-T has led to this, but more that there may be an association," he said. "This is what the FDA does. They look for a signal."

If CAR-T does cause cancer, the risk is likely very small, Dr. Hemant Murthy, a hematology-oncology physician at the Mayo Clinic in Florida, told NBC News.

“I don't really see this affecting too much of practice,” Murthy said.

Dr. Saad Usmani, a myeloma physician and cell therapist at Memorial Sloan Kettering, noted that other cancer treatments, such as radiation and chemotherapy, also carry a risk of secondary cancers.

“The [boxed warning] change is expected given the recent reports, albeit very low incidence in such cases,” he told NBC News.

More information

The National Cancer Institute has more on CAR-T therapy.

SOURCE: U.S. Food and Drug Administration, news release, Jan. 23, 2024; NBC News