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Treatment for Celine Dion's diagnosis shows promising early results

A Mayo Clinic trial of CAR-T cell therapy has produced dramatic improvements for patients with stiff person syndrome, the rare autoimmune condition affecting Celine Dion.

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The brief

Doctors at the Mayo Clinic in the United States have reported promising early results from a trial of CAR-T cell immunotherapy for stiff person syndrome, a rare autoimmune disease that attacks the central nervous system and causes severe rigidity and painful muscle spasms. The Guardian reported that some patients who previously needed help with basic daily tasks and struggled to walk have returned to work and resumed hobbies after receiving the treatment.

CAR-T cell therapy is already used to treat cancer. In the study, patients’ own T cells were extracted and engineered to wipe out disease-causing cells. The results were published in the medical journal Jama Neurology.

Dr Anastasia Zekeridou, a neurologist and researcher at Mayo Clinic in Rochester, 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. These early results are very encouraging, and the key question now is whether those improvements will be sustained over time.”

Patient Jonathan Walden had reached the point where walking and basic daily tasks required help from his family. After CAR-T, he returned to work and said: “I can go to work every single day. I can do everything I did before. I go hunting; I can go fishing. 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. She said she walked for seven minutes on a treadmill after treatment and described each day as feeling “better and better.”

Dr Sean Pittock, chair of the department of neurology at Mayo Clinic, said CAR-T therapy has transformed treatment for some cancers and is now showing potential in autoimmune neurologic diseases. He added that there is still much to learn, but the results open an exciting new avenue for the field.

Celine 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 harming her voice. Yasmin Sheikh of Anthony Nolan, a blood cancer charity not involved in the trial, called the research an exciting development that suggests CAR-T therapies could have applications beyond blood cancer.

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Quick answers

What is stiff person syndrome?

Stiff person syndrome is a rare autoimmune disease that attacks the central nervous system and can cause devastating rigidity throughout the body and painful muscle spasms. There is no cure.

What treatment showed promising results?

CAR-T cell therapy, a form of immunotherapy already used to treat cancer, in which patients’ own T cells are extracted and engineered to wipe out disease-causing cells.

How did patients respond to the treatment?

Some patients who previously needed help with basic daily tasks and struggled to walk returned to work and resumed hobbies. One patient said he had not had one episode since receiving treatment, and another walked for seven minutes on a treadmill.

Where were the results published?

The results were published in the medical journal Jama Neurology.

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