Doctors at the Mayo Clinic have reported encouraging results from a clinical trial testing CAR-T cell therapy on patients with stiff person syndrome, the rare autoimmune condition that affects singer Céline Dion, according to The Guardian.
Stiff person syndrome is a rare disease attacking the central nervous system, causing severe muscle rigidity and painful spasms without a current cure. The trial tested a novel form of immunotherapy using CAR-T cell therapy, which reprograms a patient’s own T cells to wipe out disease-causing cells. The findings were published in JAMA Neurology.
Dr. Anastasia Zekeridou stated that the degree of improvement seen in patients was striking. She noted that while existing treatments can improve symptoms, patients very rarely return to their previous level of function.
She also added, These early results are very encouraging, and the key question now is whether those improvements will be sustained over time.
Dr. Sean Pittock added that the results offer tremendous hope for people living with the disease, and stated, There is still much we need to learn, but this opens an exciting new avenue for the field.
Participants in the trial who previously required assistance with daily tasks regained mobility and returned to work. Jonathan Walden reported no episodes since receiving the treatment and returned to activities like hunting and fishing. Shari St. Germain described feeling better each day and managed to walk on a treadmill. Germain developed severe leg cramps and difficulty walking in 2021, and later experienced muscle rigidity and falls.
She recalled getting up one day after the treatment feeling really good and deciding to walk on the treadmill, describing how she cried and told her husband she had just walked for seven minutes.
Walden stated that he can go to work every single day, do everything he did before, and go hunting and fishing, noting, I have not had one episode since receiving treatment.
St Germain similarly stated that every day feels better and better
.
The trial evaluated miv-cel, a treatment aimed at becoming the first approved therapy for the condition. Data showed that patients stopped chronic immunotherapies following the intervention. Potential side effects of the underlying therapy include cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome.
| Trial Detail | Clinical Finding |
|---|---|
| Study Institution | Mayo Clinic |
| Intervention | CAR-T Cell Therapy (Miv-cel) |
| Target Condition | Stiff Person Syndrome (SPS) |
| Publication Venue | JAMA Neurology |
- The Guardian: Medical breakthrough offers hope to people with same stiff person syndrome as Céline Dion
- News-Medical: CAR-T cell therapy shows promising early results for stiff person syndrome
