Engineered immune cells show early promise against stiff person syndrome

A Mayo Clinic trial of CAR-T therapy, already used in cancer, helped some stiff person syndrome patients regain daily function. Durability is unproven.

Neurologists at the Mayo Clinic in the US have tested CAR-T cell therapy in patients with stiff person syndrome, a rare autoimmune disease of the central nervous system. The results appeared in JAMA Neurology. The condition causes body rigidity and painful muscle spasms, and it has no cure. Céline Dion announced her own diagnosis in December 2022.

CAR-T is already used against cancer. Doctors take a patient's own T cells, engineer them, and return them to the body to destroy the cells that drive the disease. Here the target is the immune activity behind SPS. Some patients who had needed help with daily tasks and struggled to walk regained everyday function. A few went back to work and hobbies. Jonathan Walden is working again, says he can hunt and fish, and reports no episodes since treatment. Shari St Germain, who had cramps, rigidity and falls, walked seven minutes on a treadmill afterward.

Why it matters: existing treatments, according to Dr Anastasia Zekeridou, rarely return patients to their earlier level of function. A therapy that restores independence would reduce the need for daily assistance, which is both a health gain and a care gain. Mayo's Dr Sean Pittock suggested the approach could matter for other immune-mediated neurological conditions.

The caveats are real. The results are early, and the account gives no trial size or follow-up length. The patient stories come from only some participants. Zekeridou named the key open question: whether the improvements last. CAR-T is also a bespoke, individually manufactured treatment, so cost and access remain far from settled. Watch for larger studies and longer follow-up.

Source: Guardian