A new CAR-T immunotherapy is showing significant improvement in people with stiff-person syndrome – a rare autoimmune disease that affects the central nervous system and causes severe stiffness and painful muscle spasms. Some patients who participated in the study regained the ability to perform everyday activities, while others returned to work and resumed their hobbies.
The results were published in the medical journal JAMA Neurology and were described by neurologists as “exciting” and “very encouraging”. The study was conducted by a team in the US, and the treatment was tested at the Mayo Clinic.
How CAR-T therapy works
CAR-T is a cellular immunotherapy already used to treat some types of cancer. The method involves extracting the patient’s own T cells – a type of immune cell that is then genetically modified. The aim is for them to recognize and destroy the cells associated with the autoimmune process.
Unlike conventional approaches, which mainly relieve symptoms, the new therapy is associated with the restoration of greater functionality. Doctors at the Mayo Clinic report that patients who previously needed help with basic daily activities and had difficulty walking have been able to lead fuller lives.
“With the treatments available so far, we can relieve symptoms, but it is very rare for patients to regain their previous level of functionality. These initial results are extremely encouraging, and the main question now is whether the improvements will be sustained over time,” says Dr. Anastasia Zekeridou, a neurologist at the Mayo Clinic, quoted by The Guardian.
Jonathan Walden, a participant in the clinical trial, had relied on his family’s help with even the most basic daily activities before treatment. After CAR-T therapy, he returned to work.
“I can go to work every day. I can do everything I did before. I go hunting, and I can go fishing too,” Walden said, quoted by The Guardian. He added: “Since undergoing the treatment, I haven’t had a single episode of symptoms.”
Dr. Sean Pittock, chair of the Department of Neurology at the Mayo Clinic, noted that the potential of CAR-T therapy is already expanding beyond oncology.
“CAR-T therapy has already transformed the treatment of some types of cancer, and now we are beginning to see its potential in autoimmune neurological diseases as well,” Pittock said. According to him, the results also offer hope to patients with other neurological conditions for which effective treatment options are limited.
Celine Dion lives with the disease
Celine Dion announced in December 2022 that she had been diagnosed with stiff-person syndrome. The singer said that the condition had affected “every aspect of her daily life” and impaired her voice.
In September, Dion returned to the stage in front of tens of thousands of fans in Paris – her first concert after a six-year absence. Despite the encouraging results from CAR-T therapy, there is still no cure for the syndrome, and doctors have yet to determine whether the improvements will be sustained over time.