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A multicenter clinical trial evaluating chimeric antigen receptor-T cell therapy (CAR-T cell therapy) for stiff person syndrome (SPS), a rare autoimmune neurologic disorder, has produced promising early results.

The study, published in JAMA Neurology, found early evidence of substantial clinical improvement among participants treated with CAR-T cell therapy. Researchers say the findings warrant further investigation in larger studies.

"What was most striking was the degree of improvement we saw in some patients. With existing treatments, we may be able to improve symptoms, but we very rarely see patients return to their previous level of function. These early results are very encouraging, and the key question now is whether those improvements will be sustained over time," says Anastasia Zekeridou, M.D., Ph.D., a neurologist and researcher at Mayo Clinic in Rochester.

CAR-T cell therapy reengineers a patient's own immune cells to target other cells contributing to disease. Study participants received a single treatment designed to reset the immune system and reduce symptoms that can profoundly limit daily life.

SPS causes progressive muscle stiffness and painful spasms that can make walking, driving, working and socializing increasingly difficult. Mayo Clinic first described the disease in 1956.

" CAR-T therapy has already transformed the treatment of some cancers, and we are now beginning to see its potential in autoimmune neurologic diseases. The results in stiff person syndrome offer tremendous hope, not only for people living with this disease but potentially for patients with other immune-mediated neurologic conditions where effective treatment options remain limited.

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