Cancer Treatment Caution: FDA Alerts on Potential Increased Risks with CAR-T Therapy

The FDA has mandated drug manufacturers to add a boxed warning, its strictest safety label, to the prescribing information for CAR-T therapy, a type of cancer treatment.

The FDA decision is a response to reports linking CAR-T therapy to a heightened risk of rare blood cancers. While acknowledging this risk, Carly Kempler, an FDA spokesperson, emphasized that the overall benefits of CAR-T therapy outweigh the potential risks.

Following the identification of rare blood cancers in individuals undergoing CAR-T therapy, the FDA has registered 25 reported cases. This accumulation of cases has led to the determination by the FDA to initiate the process of revising the labeling associated with CAR-T treatments.

Bruce Levine, a professor at the University of Pennsylvania, noted additional abstracts last year in the journal Blood that highlighted a potential cancer risk associated with CAR-T therapy, influencing the FDA’s action.

CAR-T therapy, or chimeric antigen receptor T cell therapy, utilizes a patient’s immune cells to treat certain blood cancers. Despite the recent warnings, CAR-T therapy has proven highly effective in challenging cases, with reports of curing patients of diseases like leukemia.

FDA’s Guidelines on CAR-T Cancer Risk

The Food and Drug Administration (FDA) issued directives to the manufacturers of five CAR-T drugs, namely Bristol Myers Squibb, Gilead Sciences’ Kite Pharma, Johnson & Johnson, and Novartis, instructing them to present proposed modifications to their product labels within a stipulated period of 30 days.

The changes should reflect the rare cases where CAR-T therapy might increase the risk of blood cancers. The FDA’s letters give the option for drugmakers to submit rebuttals if they disagree with the requirement.

Novartis, in response, expressed a lack of sufficient evidence supporting a link between its treatment, Kymriah, and cancer. Despite this, the company committed to collaborating with the FDA to update its label appropriately. 

Johnson & Johnson and Gilead Sciences also stated their intention to work with the FDA on label updates, while Bristol Myers Squibb is evaluating its next steps, noting no observed cancer cases associated with its treatment.