FDA Investigating Secondary Cancer Risks From CAR-T Cell Treatments

Officials are investigating a potential link between certain blood cancer treatments and the development of new, more severe cancers that may result in adverse health outcomes.

Federal regulators are looking into the safety of certain immunotherapy cancer treatments, which may increase the risk of certain blood cancer patients developing new or more severe types of secondary cancers, after the treatment ends.

The Food and Drug Administration (FDA) issued a press release on November 28, indicating that individuals receiving chimeric antigen receptor T cell (CAR-T) therapy treatments, including BCMA-directed and CD19-directed immunotherapies, can develop serious secondary T cell malignancies afterwards, which may result in hospitalization or even death.

Officials initiated an investigation into the potential side effects of CAR-T treatments, after becoming aware of several reports involving T cell malignancies that developed among cancer patients who underwent the immunotherapies. Secondary cancers were identified in both drug trial participants and patients who had completed treatment for aggressive blood cancers, such as lymphoma, certain types of leukemia, and multiple myeloma.

CAR T Cell Therapy

Chimeric antigen receptor (CAR) T cell therapy is a type of immunotherapy treatment that uses genetically modified immune cells, known as T-cells, to locate and destroy cancer cells. The altered gene therapy is approved for use in patients with advanced, difficult-to-treat, or reoccurring lymphoma, leukemia, and multiple myeloma blood cancers.

There are currently six FDA-approved treatments among the drug class, which include Abecma (idecabtagene vicleucel), Breyanzi (lisocabtagene maraleucel), Carvykti (ciltacabtagene autoleucel), Kymriah (tisagenlecleucel), Tecartus (brexucabtagene autoleucel), and Yescarta (axicabtagene ciloleucel). These treatments were approved under the requirement that the FDA conduct a 15-year observational study to assess the potential for long-term adverse health outcomes and secondary malignancies that could occur after treatment.

While officials acknowledged that the secondary cancer risks are disclosed in the prescribing information and the products are labeled with a class warning, the FDA is currently evaluating whether further regulatory action may be needed to address the risks.

The FDA recommends that patients or clinical trial participants receiving these treatments be monitored for the rest of their life by a health care professional for new cancers that may develop.

Patients and trial participants are urged to report any new malignancies they may develop following CAR-T treatment to the manufacturer, and obtain instructions on how to collect patient samples for testing purposes.

Individuals are also encouraged to report any adverse events experienced after receiving treatment, including T cell malignancies, to the FDA MedWatch Adverse Event Reporting Program.

For more information, medical professionals, investigators, patients, and caregivers may contact the FDA Center for Biologics Evaluation and Research (CBER) by email at ocod@fda.hhs.gov.