Paving the Way for Autoimmune Neurology Treatment With CAR T-Cell Therapy: Amanda Piquet, MD, FAAN

WATCH TIME: 3 minutes

“In hematologic malignancies, the purpose is to eliminate the malignant B-cells in hopes of curing the cancer. In autoimmune diseases, we're looking for that deep B-cell depletion in order to hopefully reset the immune system.”

CAR T-cell therapy has revolutionized oncology by providing a novel approach to treating certain hematologic cancers, including leukemia and lymphoma. This immunotherapy involves genetically modifying a patient’s T-cells to better recognize and attack cancer cells. To date, this approach has shown remarkable success, especially in cases where traditional treatments like chemotherapy have failed. In this process, CAR T-cells are designed to target specific antigens present on the surface of cancer cells, allowing for more precise and potent eradication of the tumor. Despite its efficacy, the therapy is not without risks, such as cytokine release syndrome (CRS) and neurotoxicity, which may require careful monitoring and management.¹

In neurology, CAR T-cell therapy is being explored as a potential treatment for autoimmune neurological disorders like multiple sclerosis, myasthenia gravis, and Stiff-Person syndrome. Unlike in oncology, where the goal is to eliminate malignant cells, the focus in neurology is on resetting the immune system by targeting and depleting specific B cells responsible for autoimmune attacks. By targeting these B cells, CAR T therapy may offer more durable remission and improved outcomes compared with current treatments. However, applying this therapy in neurological conditions presents new challenges, including managing adverse effects such as CRS and neurotoxicity, which are less common in the context of autoimmune diseases.²

Amanda Piquet, MD, FAAN, recently presented a session on the evolving role of CAR T therapy in 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. In a recent interview with NeurologyLive^®, Piquet, director of the autoimmune neurology program and associate professor of neurology at the University of Colorado Anschutz Medical Campus, addressed key questions about how CAR T’s approach to targeting B-cells differs between autoimmune neurological diseases and hematologic malignancies. Piquet also highlighted the challenges of applying CAR T therapy to autoimmune conditions and emphasized the potential for more precise targeting to improve treatments for diseases.

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. Schett G, Müller F, Taubmann J, et al. Advancements and challenges in CAR T cell therapy in autoimmune diseases. Nat Rev Rheumatol. 2024;20(9):531-544. doi:10.1038/s41584-024-01139-z
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.