A team of neurologists in the United States has published findings from a clinical trial of CAR-T cell therapy for stiff person syndrome, a rare autoimmune condition affecting the central nervous system. The disease causes severe muscle rigidity and spasms throughout the body and has no known cure despite decades of research. According to results reported in the journal Jama Neurology, the novel immunotherapy approach produced what researchers described as dramatic and encouraging outcomes in trial participants.
CAR-T cell therapy works by extracting a patient’s own T cells, a type of immune cell, and engineering them to target disease-causing cells. The treatment is already established in cancer care but represents a new application for autoimmune neurological conditions. Patients in the trial who had previously required assistance with basic activities and experienced significant mobility challenges showed substantial functional recovery. Some participants returned to employment and resumed recreational activities they had believed lost to the disease.
Dr. Anastasia Zekeridou of Mayo Clinic in Rochester emphasized that the degree of improvement observed distinguished these results from existing treatment options, which typically manage symptoms without restoring patients to previous functional levels. Among trial participants, Jonathan Walden regained the ability to work and engage in activities such as hunting and fishing, while reporting no symptom episodes following treatment. Another patient, Shari St Germain, experienced relief from severe leg cramps and muscle rigidity that had previously caused falls and mobility difficulties.
Dr. Sean Pittock, chair of the neurology department at Mayo Clinic, noted that while CAR-T therapy has transformed cancer treatment outcomes, the SPS trial demonstrates potential applications in autoimmune neurological diseases where treatment options remain limited. Researchers indicated that sustained improvements and long-term outcomes require further investigation. The findings have prompted discussion about the therapy’s potential for other immune-mediated neurological conditions.
Stiff person syndrome gained increased public attention when singer Céline Dion announced her diagnosis in December 2022, describing how the condition affected her daily functioning and vocal abilities. The trial results have been characterized as offering new hope to individuals living with the disease and potentially other related conditions.
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