KUWAIT CITY, Jan 24: The Food and Drug Administration (FDA) has issued a directive to several pharmaceutical companies, urging them to incorporate a boxed warning—the agency's most robust safety label—into the prescribing information for CAR-T therapy. This decision comes in response to concerns that the therapy itself may heighten the risk of cancer in individuals undergoing treatment. Despite the cautionary move, Carly Kempler, an FDA spokesperson, emphasized that the overall benefits of CAR-T products continue to outweigh potential risks.

The FDA's decision to update labels stems from reported cases of rare blood cancers in patients who had previously undergone CAR-T therapy. As of Monday, the agency had received 25 such reports, prompting a thorough evaluation and subsequent action. Bruce Levine, a cancer gene therapy professor at the University of Pennsylvania, highlighted that two abstracts published late last year in the journal Blood also suggested a potential cancer risk linked to CAR-T therapy, likely prompting the FDA's intervention.

CAR-T therapy, short for chimeric antigen receptor T cell therapy, employs a patient's immune cells to treat specific blood cancers, including leukemia, multiple myeloma, and lymphoma. The treatment involves harvesting T cells, genetically altering them in a lab to target cancer cells, and reintroducing them into the patient—an approach deemed highly effective, especially in challenging cases. Despite concerns, experts acknowledge the groundbreaking impact of CAR-T therapy in treating lymphoma and other diseases.

Since the approval of the first CAR-T therapy, Novartis' Kymriah, in 2017, the FDA has granted approval to five additional therapies. Following the recent FDA directive, pharmaceutical companies, including Bristol Myers Squibb (Abecma and Breyanzi), Gilead Sciences' Kite Pharma (Yescarta), Johnson & Johnson (Carvykti), and Novartis (Kymriah), have received letters mandating proposed label changes within the next 30 days. The updates are expected to acknowledge the rare occurrence of CAR-T therapy potentially increasing the risk of rare blood cancers. If drugmakers disagree, they have the option to submit a rebuttal.

In response, Novartis, Johnson & Johnson, and Gilead Sciences have expressed their commitment to working with the FDA to update their labels appropriately. However, Novartis maintained that it has not found sufficient evidence to establish a clear link between cancer and its treatment. Despite these reassurances, patient safety remains a top priority for pharmaceutical companies.

While the question of how CAR-T therapy could cause cancer remains uncertain, healthcare professionals underscore the need for ongoing evaluation and monitoring. Dr. Hemant Murthy, a hematology-oncology physician at the Mayo Clinic, believes the risk, if present, is likely very small, given the administration of over 27,000 doses of CAR-T therapy in the U.S. Thus, the impact on medical practice is expected to be minimal, with the label change supporting physicians' existing discussions with patients about the potential risk of secondary cancers following cancer treatment. Overall, the medical community acknowledges the evolving nature of CAR-T therapy and the importance of managing current cancer cases while remaining vigilant to potential risks.