FDA Investigating Cancer Risks in Gene Therapy Treatments

The U.S. Food and Drug Administration (FDA) is investigating the cancer treatment CAR-T after it was notified of new blood cancers among individuals who received the treatment.

The FDA “has received reports of T-cell malignancies, including chimeric antigen receptor CAR-positive lymphoma, in patients who received treatment with BCMA- or CD19-directed autologous CAR T cell immunotherapies,” the agency announced. “Reports were received from clinical trials and/or postmarketing adverse event (AE) data sources.”

CAR-T was approved in 2017 and is used to treat blood cancers.

“Although the overall benefits of these products continue to outweigh their potential risks for their approved uses, FDA is investigating the identified risk of T cell malignancy with serious outcomes, including hospitalization and death, and is evaluating the need for regulatory action,” the FDA’s statement added.

“Patients and clinical trial participants receiving treatment with these products should be monitored life-long for new malignancies. In the event that a new malignancy occurs following treatment with these products, contact the manufacturer to report the event and obtain instructions on collection of patient samples for testing for the presence of the Chimeric Antigen Receptor (CAR) transgene.”

The approved CAR-T products that have now been discovered to have risks include:

  • Kymriah
  • Yescarta
  • Carvykti
  • Breyanzi
  • Abecma
  • Tecartus

A Gilead spokesperson, the company behind Yescarta and Tecartus, told medical news outlet Fierce Pharma, “We have a rigorous process in place to continuously monitor for and report adverse events to regulatory authorities. We have fully cooperated with the FDA’s request for an analysis of our data related to this inquiry.”

FDA Adverse Event Reporting System (FAERS) data show:

  • Kymriah- 2,470 adverse events and 662 deaths
  • Yescarta- 3,729 adverse events and 746 deaths
  • Carvykti- 408 adverse events and 28 deaths
  • Breyanzi- 202 adverse events and 38 deaths
  • Abecma- 528 adverse events and 60 deaths
  • Tecartus- 609 adverse events and 136 deaths

Not all the adverse effects are exclusively blood and lymphatic system disorders, but may be nervous system disorders, immune system disorders, infections, cardiac disorders, vascular disorders, gastrointestinal disorders, and others.