CAR-T Cell Therapy Shows Promise Against Stiff Person Syndrome, the Condition Affecting Céline Dion

Doctors in the United States have reported a potentially significant breakthrough in treating stiff person syndrome (SPS), the rare autoimmune condition that affects the singer Céline Dion, according to the Guardian [1]. SPS is a rare disease that attacks the central nervous system, causing severe rigidity and painful muscle spasms throughout the body. Despite decades of research, no cure currently exists, according to [1]. A team of neurologists at the Mayo Clinic trialling a novel form of immunotherapy has reported what it described as "exciting" and "very encouraging" results, with some patients experiencing dramatic improvements, according to [1]. The treatment, CAR-T cell therapy, is already used to treat cancer and works by reprogramming a patient's immune cells to fight disease. In the study, SPS patients' own T cells were extracted and engineered to eliminate disease-causing cells, according to [1]. Patients who had previously needed help with basic daily tasks and struggled to walk have regained the ability to take part in everyday life, with some returning to work and resuming hobbies, according to doctors at the Mayo Clinic cited by [1]. The results were published in the journal Jama Neurology, according to [1]. Dr Anastasia Zekeridou, a neurologist and researcher at Mayo Clinic in Rochester, said the degree of improvement seen in some patients was striking. "With existing treatments, we may be able to improve symptoms but we very rarely see patients return to their previous level of function," she said, according to [1]. "These early results are very encouraging and the key question now is whether those improvements will be sustained over time." Among the trial participants, Jonathan Walden had reached the point where walking and basic daily tasks required help from his family. After receiving CAR-T therapy, he returned to work. "I can go to work every single day. I can do everything I did before. I go hunting; I can go fishing," he said, according to [1]. "I have not had one episode since receiving treatment." Another patient, Shari St Germain, had suffered severe leg cramps, difficulty walking, muscle rigidity and falls before the treatment. Following CAR-T therapy, she described major improvements, telling the Guardian that every day feels "better and better" [1]. She recounted walking on a treadmill for seven minutes for the first time, an experience that left her in tears, according to [1]. Dr Sean Pittock, chair of the department of neurology at Mayo Clinic, said CAR-T therapy had already transformed treatment for some cancers and was now showing potential in autoimmune neurological 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," he said, according to [1]. He added that there was "still much we need to learn" but that the findings opened "an exciting new avenue for the field". Dion announced her SPS diagnosis in December 2022, telling fans in an Instagram video that the condition had affected "every aspect of my daily life", including her voice, according to [1]. Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, a blood cancer charity not involved in the trial, said the research provided further evidence that CAR-T cell therapies could have applications beyond blood cancer. "This research is an exciting development, providing more evidence that CAR-T cell therapies could have applications beyond blood cancer and offer new hope to people living with other conditions, including SPS," she said, according to [1].