Patients living with stiff person syndrome, a rare autoimmune disease that causes severe muscle rigidity and painful spasms, have experienced dramatic improvements in a small clinical trial. Some patients who previously struggled to walk or required daily assistance from their families have regained enough mobility to return to work, exercise, and resume everyday hobbies.
The experimental approach uses CAR-T cell therapy, a form of immunotherapy already established in cancer care. Neurologists at the Mayo Clinic extracted T cells—a type of immune cell—from patients and genetically reprogrammed them to target and destroy the harmful cells driving the condition. The findings, published in the journal JAMA Neurology, offer new hope for a disorder that attacks the central nervous system and has long lacked a cure.
Among the participants was Jonathan Walden, who had previously depended on his family for basic daily tasks. Following the treatment, Walden returned to full-time work and resumed outdoor activities such as hunting and fishing, reporting that he had not suffered a single episode since receiving the therapy. Another patient, Shari St Germain, who had endured severe leg cramps, muscle stiffness, and frequent falls, reported steady progress that allowed her to walk on a treadmill again.
Medical experts emphasize that the results remain preliminary and that further research is required to determine whether the improvements will last over time. Dr. Anastasia Zekeridou, a neurologist and researcher at the Mayo Clinic, noted that while existing therapies may reduce symptoms, patients rarely return to their previous level of physical function. If supported by future studies, researchers believe the technique could open new therapeutic avenues for other challenging autoimmune neurological disorders.