CAR-T cell therapy shows promising early results for stiff person syndrome
· News-MedicalA 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.
Anastasia Zekeridou, M.D., Ph.D., neurologist and researcher at Mayo Clinic, RochesterWhat 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."
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.
For two patients in the trial, improvement meant regaining parts of everyday life the disease had taken away.
Shari St. Germain developed severe leg cramps and difficulty walking in 2021. As her disease progressed, sudden noises could trigger muscle rigidity and falls, and by 2024 standard treatments were no longer controlling her symptoms. Months after receiving CAR-T cell therapy, Shari began noticing improvements. Once an avid walker who routinely covered 7 miles a day, she reached an important milestone when she was able to walk on a treadmill again.
Jonathan Walden had reached the point where walking and basic daily tasks required help from his family. Following treatment, he says he has returned to work and activities including hunting and fishing.
"I can go to work every single day. I can do everything I did before. I go hunting; I can go fishing," Jonathan says. "I have not had one episode since receiving treatment."
"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. There is still much we need to learn, but this opens an exciting new avenue for the field," says Sean Pittock, M.D., chair of the Department of Neurology at Mayo Clinic in Rochester.
Mayo Clinic is one of three participating sites in the trial and played a key role in the research, enrolling patients and providing scientific leadership, including authorship of the publication.
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