US neurologists trialling a novel form of immunotherapy have reported exciting and very encouraging results in treating stiff person syndrome.
Stiff person syndrome is a rare autoimmune disease attacking the central nervous system. It causes severe rigidity and painful muscle spasms.
Patients who previously needed help with basic daily tasks regained their ability to take part in everyday life. Some participants even returned to work and resumed hobbies, according to doctors at the Mayo Clinic.
The treatment uses CAR-T cell therapy, which already treats cancer. The process reprogrammes a patient’s immune cells to fight disease by extracting T cells and engineering them to wipe out disease-causing cells.
Results from the study were published in the medical journal Jama Neurology. Dr Anastasia Zekeridou, a neurologist and researcher at the Mayo Clinic in Rochester, noted the unusual level of recovery.
“What was most striking was the degree of improvement we saw in some patients,” Dr Zekeridou said.
She added that existing treatments rarely help patients return to their previous level of function. The key question now is whether those improvements will last over time.
Jonathan Walden reached a point where walking and basic daily tasks required help from his family. After receiving CAR-T cell 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,” Walden said. “I have not had one episode since receiving treatment.”
Shari St Germain suffered severe leg cramps, difficulty walking, muscle rigidity, and falls. Following the treatment, she experienced major improvements.
“After the treatment, I got up one day and I’m like ‘I’m feeling really good’, and I went to walk on the treadmill,” St Germain said. “My husband comes running in because I’m crying and he’s like: ‘Are you OK?’ And I’m like: ‘I just walked for seven minutes on the treadmill’.”
Dr Sean Pittock, chair of the department of neurology at the Mayo Clinic, said the therapy has already transformed cancer treatment. He added that researchers are now beginning to see its potential in autoimmune neurologic diseases.
The results offer tremendous hope for people living with stiff person syndrome. Dr Pittock noted the approach could potentially help patients with other immune-mediated neurologic conditions where effective treatment options remain limited.
Singer Céline Dion announced her diagnosis with the condition in December 2022. She told fans in an Instagram video that the illness affected every aspect of her daily life, including harming her voice.
Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, a blood cancer charity not involved with the trial, spoke about the broader impact. Sheikh said the research provides more evidence that CAR-T cell therapies could offer new hope to people living with other conditions beyond blood cancer.
Stiff person syndrome is a rare autoimmune condition that attacks the central nervous system. It triggers severe rigidity throughout the body and painful muscle spasms.
Céline Dion announced she had been diagnosed with stiff person syndrome in December 2022 via an Instagram video.
The trial was conducted by neurologists at the Mayo Clinic in Rochester, and the results were published in the medical journal Jama Neurology.
Worth a look