FDA Warns of Cancer Risk Linked to CAR-T Therapies
The Food and Drug Administration requires companies that market specialized cancer therapies known as CAR-T to add a boxed warning that the treatments themselves can cause cancer.
The agency noted that the benefits still outweighed the risks of the therapy, which involves removing a type of white blood cell – T cells – and then genetically engineering it to create proteins called chimeric antigen receptors (CAR). The engineered cells are returned to a patient's blood and allow the T cells to attach to and kill cancer cells.
But the therapies, which mainly treat blood cancers including multiple myeloma, had already come with a warning of dangerous immune reactions and neurological risks. And the new warning follows reports of about two dozen cases of secondary cancers that federal health officials and others suspected were caused by CAR-T treatments, although more research may be needed to establish a definitive link. The therapy has been used by an estimated 25,000 to 30,000 patients since its initial FDA approval in 2017.
Cancer patients receiving CAR-T treatments typically have few options left and are unlikely to change course even with the new warning, said Dr. John DiPersio, an oncologist at Washington University in St. Louis.
“The risk of not using this therapy for most patients who receive it is rapid progression of their disease or certain death,” he said.
The FDA raised concerns about the adverse effects of the treatments late last year.
In letters dated Jan. 19, the agency outlined warnings to be included by some of the companies that make CAR-T therapies, which had also been ordered to monitor patients for secondary cancers and report them to the FDA . The secondary cancers can lead to hospitalizations or death, the agency noted, requiring drug companies to warn on drug labels that secondary cancers “can occur weeks after the infusion and can have fatal consequences. ”
The FDA has sent letters to these companies: Bristol Myers Squibb, maker of Abecma; Juno Therapeuticsa Bristol-Myers Squibb Company, maker of Breyanzi; Janssen Biotech from Johnson & Johnson, maker of Carvykti; Novartis, from Kymriah; And Kite Pharmafrom Yescarta.
Given the bleak prognosis of the patients considering CAR-T therapies, Dr. DiPersio, the new warning amounted to “much ado about nothing.” He said he hoped the news would not deter further investment or research into treatments for other serious medical conditions. Some drugmakers are studying the use of CAR-T therapy to treat lupus, an autoimmune disease.
“We can't create such a fearful environment where companies and researchers shy away from this approach because people think it's too dangerous – because it's not,” he said.
Financial analysts also predicted little impact, Brian Abrahams, an analyst at RBC Capital Markets, said in an email.
“It's still a very rare side effect,” he said. “These are sick patients receiving these therapies, and most CAR-T treatments have shown very clear benefits, including extended survival – so we do not expect these label changes to materially reduce use of the agents.”