Mayo Clinic trials CAR-T cell therapy for stiff person syndrome

Patients regain daily function after treatment

Doctors at the Mayo Clinic in Rochester described dramatic improvements for people living with stiff person syndrome. Patients who previously needed help walking have regained the ability to perform everyday tasks and return to work. Jonathan Walden, a trial participant, stated he can now go hunting and fishing without assistance. Another patient named Shari St Germain walked for seven minutes on a treadmill after suffering severe leg cramps. Dr Anastasia Zekeridou noted that existing treatments rarely allow patients to reach their previous level of function.

Novel immunotherapy targets immune cells

The novel treatment involves CAR-T cell therapy which reprogrammes a patient's own T cells to fight disease-causing cells. This approach extracts immune cells and engineers them to wipe out the specific cells causing the autoimmune condition. The results were published in the medical journal Jama Neurology by researchers studying this rare central nervous system disorder. Dr Sean Pittock explained that CAR-T therapy has already transformed cancer treatment but is now showing potential for neurologic diseases.

Hope extends to other conditions

Singer Céline Dion announced her diagnosis in December 2022 after the condition harmed her voice and daily life. Dr Pittock suggested these results offer tremendous hope for patients with other immune-mediated neurologic conditions where options remain limited. The key question now is whether these improvements will be sustained over time for all participants. Yasmin Sheikh from Anthony Nolan charity noted this research represents a significant step forward in the field.