People suffering from stiff person syndrome (SPS), which affects Celine Dion, the singer, could soon benefit from a new medicine which improves symptoms.
Patients in the US were given a one-off immunotherapy drug in a clinical trial, resulting in two individuals regaining function related to everyday life that had been lost to SPS, according to The Telegraph.
SPS is a rare neurological disorder that gets progressively worse, and symptoms include stiffness in the body, spine, arms and legs.
The Telegraph said patients were given chimeric antigen receptor T-cell (CAR-T) therapy, which teaches the body’s immune system to recognize and attack deadly cells causing disease.
CAR-T therapy is already approved on the NHS for some illnesses, including certain types of cancer, and research is ongoing.
One patient in the US trial, Jonathan Walden, had reached the point where walking and basic daily tasks required help from his family.
After CAR-T therapy, he has now returned to work and normal activities.
“I can go to work every single day. I can do everything I did before. I go hunting, I can go fishing,” he said.
Dr. Anastasia Zekeridou, a neurologist and researcher at Mayo Clinic in Rochester in the US, which led the trial, said: “What was most striking was the degree of improvement we saw in some patients.”
“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, adding that these early results are very encouraging, and the key question now is whether those improvements will be sustained over time.
Dr. Sean Pittock, chairman of the department of neurology at Mayo Clinic, said: “CAR-T therapy has already transformed the treatment of some cancers, and we are now beginning to see its potential in autoimmune neurologic diseases.”
Pittock also explained that 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.
“There is still much we need to learn, but this opens an exciting new avenue for the field,” he said.
Dion, the Canadian vocalist, announced she was diagnosed with stiff person syndrome in December 2022.
In an Instagram video, she told fans the condition had affected “every aspect of my daily life,” including harming her voice.
SPS affects an estimated 8,000 people worldwide. The British Neurological Surveillance Unit identified 119 confirmed cases in the UK across a five-year tracking period between 2000 and 2005.
Scientists believe it can become a widespread syndrome that affects multiple parts of the body and involves the brain, brainstem and spinal cord.
