Exploring the Potential of CAR T-Cell Therapy in Autoimmune Neurologic Diseases: Amanda Piquet, MD, FAAN

WATCH TIME: 3 minutes

“Clearly, there's an unmet need for better therapies, and I believe CAR T has that potential. Obviously, this needs to be studied further in clinical trials, and if we achieve success with treatment in our clinical trials, we have yet to see how long patients maintain disease freedom.”

CAR T-cell therapy has transformed cancer treatment by offering a groundbreaking method for addressing certain blood cancers, such as leukemia and lymphoma. This form of immunotherapy involves modifying a patient's T-cells to enhance their ability to detect and destroy cancer cells. It has proven highly effective, particularly in cases where conventional treatments, like chemotherapy, have been unsuccessful. The engineered T-cells are designed to recognize specific antigens on the surface of cancer cells, allowing for a more targeted and powerful elimination of tumors. However, despite its success, the therapy carries risks, including cytokine release syndrome (CRS) and neurotoxicity, which necessitate careful oversight and management.¹

In the field of neurology, CAR T-cell therapy is being investigated as a potential treatment for autoimmune neurological diseases, such as multiple sclerosis, myasthenia gravis, and Stiff-Person syndrome. Unlike oncology, where the aim is to destroy cancerous cells, the focus in neurology is on resetting the immune system by targeting and eliminating specific B cells that cause autoimmune responses. By depleting these B -ells, CAR T therapy may offer longer-lasting remissions and improved outcomes compared with current therapies. However, applying this treatment to neurological conditions introduces new challenges, particularly in managing adverse effects like CRS and neurotoxicity, which may be less common in autoimmune diseases.²

Amanda Piquet, MD, FAAN, recently discussed the expanding role of CAR T-cell therapy in treating autoimmune neurological diseases at the 2025 Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum, held February 27 to March 1, in West Palm Beach, Florida.³ In an interview with NeurologyLive^®, Piquet, director of the autoimmune neurology program and associate professor of neurology at the University of Colorado Anschutz Medical Campus, addressed several key topics, including how CAR T therapy could reshape treatment for autoimmune neurological conditions, the challenges in managing neurotoxicity associated with the therapy, and the importance of multidisciplinary collaboration in enhancing patient outcomes.

Click here for coverage of 2025 ACTRIMS Forum.

REFERENCES
1. Neelapu SS, Tummala S, Kebriaei P, et al. Chimeric antigen receptor T-cell therapy - assessment and management of toxicities. Nat Rev Clin Oncol. 2018;15(1):47-62. doi:10.1038/nrclinonc.2017.148
2. Hu C, Liu M, Li Y, et al. Recent advances and future perspectives of CAR-T cell therapy in head and neck cancer. Front Immunol. 2023;14:1213716. Published 2023 Jun 29. doi:10.3389/fimmu.2023.1213716
3. Piquet A, Cree B, Seshadri M. The (R)Evolution of CAR T-Cell Therapy: A Transformative Path from Oncology to Neurology. Presented at ACTRIMS Forum 2025; February 27 to March 1; West Palm Beach, Florida.