FDA Investigates Safety of CAR T-Cell Therapy
After identifying T-cell malignancies in some patients treated with CAR T-cell therapy, the FDA is launching an investigation into the safety of the treatment.
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- In a safety notice published on November 28, 2022, the United States Food and Drug Administration (FDA) announced an investigation into the safety of chimeric antigen receptor (CAR) T-cell therapy.
The investigation was launched after the FDA received reports of T-cell malignancies in patients receiving BCMA- or CD19-directed autologous CAR T-cell immunotherapies. Through clinical trial data and post-marketing adverse event reporting, the FDA noted that T-cell malignancies, including CAR-positive lymphoma, were a risk for any patient treated with these immunotherapies.
The safety statement identifies the following FDA-approved CAR-T cell treatments that have been linked to malignancies:
According to the FDA, secondary malignancies are always considered a risk when gene therapy products integrate lentiviral or retroviral vectors. To acknowledge these risks, the aforementioned FDA-approved gene therapies have secondary malignancies listed as a class warning in the prescribing information.
Despite this risk, the FDA maintains that the benefits of CAR-T cell therapy and the approved drugs outweigh the potential risks based on currently available information. However, the administration is launching an investigation to gain a better understanding of potentially adverse severe outcomes and guide future regulatory action.
As the administration explores and analyzes the safety information, it has issued recommendations to patients and providers regarding CAR-T cell therapies. First, patients treated with BCMA-directed or CD19-directed autologous CAR T-cell immunotherapies should be monitored for new malignancies indefinitely.
Should a new malignancy be identified after a patient is treated with one of the affected therapeutic interventions, the event should be reported to the manufacturer and the FDA. Upon receiving the report, manufacturers should provide instructions for collecting patient samples and testing for the CAR transgene.
The outcomes of this safety investigation could have critical implications in the oncology landscape. While CAR-T cell therapy and other targeted therapeutics have advanced treatment options and enhanced precision medicine, the potential risks could impact future development and uptake. As the FDA investigates the outcomes and makes the information publicly available, patients, providers, and pharmaceutical companies should monitor changes and conduct risk assessments to evaluate the benefits of the treatment on a case-by-case basis.
