CAR-T Cell Therapy Shows Promising Early Results for Stiff Person Syndrome
In a phase II trial, 21 of 26 patients improved walking ability after one infusion, and some regained daily function.
- A Phase II trial published Monday in JAMA Neurology found that mivocabtagene autoleucel, an experimental CAR-T cell therapy, significantly improved mobility in patients with stiff person syndrome who previously failed standard treatments.
- Stiff person syndrome, which affects an estimated 8,000 people and gained prominence after Celine Dion's 2022 diagnosis, is a rare autoimmune disorder causing progressive muscle rigidity and spasms resistant to conventional treatments.
- In the study of 26 patients, 80.8% experienced clinically meaningful improvements in walking ability, while participants completed a timed 25-foot walk a median 45.6% faster than before treatment.
- Jonathan Walden returned to work and hunting, while Shari St. Germain walked on a treadmill again, as participants reported regaining everyday capabilities previously lost to the disease.
- Researchers cautioned that the study lacked a control group and median follow-up was just 6.5 months, though most patients experienced only manageable treatment-related adverse events.
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11 Articles
CAR T-Cell Therapy Restores Mobility in Patients with Treatment-Resistant Stiff Person Syndrome
An experimental CAR T-cell therapy significantly improved mobility and reduced stiffness in patients with stiff person syndrome who had responded inadequately to previous immunotherapies. In a Phase II trial, 21 of 26 patients achieved clinically meaningful improvements in walking ability after a single infusion, raising the possibility of sustained relief without ongoing immunotherapy.
CAR-T cell therapy shows promising early results for stiff person syndrome
A multicenter clinical trial evaluating chimeric antigen receptor-T cell therapy (CAR-T cell therapy) for stiff person syndrome (SPS), a rare autoimmune neurologic disorder, has produced promising early results.
A treatment used so far against certain forms of cancer could change the lives of people suffering from rigid person syndrome, the rare disease with which she has been diagnosed and the singer Celine...
Early trial results suggest potential new treatment for stiff person syndrome
A multicenter clinical trial evaluating chimeric antigen receptor-T cell therapy (CAR-T cell therapy) for stiff person syndrome (SPS), a rare autoimmune neurologic disorder, has produced promising early results.
Breakthrough treatment for Celine Dion's condition sees patients walk again
Canadian singer Celine Dion has suffered since 2022 from stiff person syndrome, a rare and incurable neurological condition
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