US doctors report improvements in stiff person syndrome patients using cell therapy
Neurologists at the Mayo Clinic in the US report encouraging results from treating stiff person syndrome, a rare autoimmune disease, with CAR-T cell therapy. Singer Céline Dion also has the condition.
Doctors at the Mayo Clinic in the United States have reported a potentially significant breakthrough in treating stiff person syndrome (SPS). The rare autoimmune disease attacks the central nervous system and can cause severe body rigidity and painful muscle spasms. Singer Céline Dion also lives with it. There is no cure, and a team of neurologists has now tested a new form of immunotherapy that has led to marked improvements in patients.
The results were published in the medical journal Jama Neurology. The method, CAR-T cell therapy, is already used against cancer and reprogrammes a patient's immune cells to help fight disease. In the study, patients' own T cells, a type of immune cell, were taken out and engineered to destroy the cells that cause the disease.
According to the doctors, patients who previously needed help with basic daily tasks and struggled to walk have regained the ability to take part in everyday life. Some have returned to work and resumed hobbies.
Dr Anastasia Zekeridou, a neurologist and researcher at the Mayo Clinic in Rochester, said the degree of improvement in some patients was the most striking finding. She said existing treatments may ease symptoms but only very rarely do patients return to their previous level of function. The key question now, she added, is whether the improvements will last.
Trial participant Jonathan Walden had reached the point where walking and basic daily tasks required his family's help. He has since returned to work, goes hunting and fishing, and says he has not had an episode since the treatment. Another patient, Shari St Germain, had suffered severe leg cramps and difficulty walking, later followed by muscle rigidity and falls. After treatment she said every day feels "better and better"; she once walked for seven minutes on a treadmill.
Dr Sean Pittock, chair of the Mayo Clinic's department of neurology, said CAR-T therapy has already transformed the treatment of some cancers and its potential is now being seen in autoimmune neurological diseases. He said the results offer hope for people with other immune-mediated neurological conditions where effective treatments remain limited.
Dion announced her SPS diagnosis in December 2022. In an Instagram video she said the condition had affected every aspect of her daily life, including her voice. Yasmin Sheikh of the blood cancer charity Anthony Nolan, who was not involved in the trial, said the research provides more evidence that CAR-T therapies could have uses beyond blood cancer.
