Preliminary results from a Phase 1 clinical trial suggest that a CAR T-cell therapy called equecabtagene autoleucel may offer benefit to patients with progressive forms of multiple sclerosis (MS). The therapy, developed by Iaso Biotherapeutics, was tested in three individuals—two with secondary progressive MS (SPMS) and one with primary progressive MS (PPMS)—as part of the ongoing CARTinNS study at Tongji Hospital in China.
Each patient received a single dose of the therapy, which is derived from the patient’s own T-cells and engineered to target BCMA, a protein on B-cells involved in MS-related inflammation. All three participants had previously tried multiple approved MS treatments.
After three months:
- Both SPMS patients showed improvements in their Expanded Disability Status Scale (EDSS) scores, with reductions from 6 to 5.5 and 5, allowing walking without assistance.
- The PPMS patient’s score improved from 7 to 5.5, marking a transition from wheelchair dependence to more independent movement.
- All three patients exhibited better hand and walking function, and no new or enlarged brain lesions were reported.
- Importantly, oligoclonal bands, markers of inflammation in spinal fluid, were no longer present in any of the participants.
The treatment was generally well tolerated. Mild and temporary cytokine release syndrome, a common risk of CAR T-cell therapy, occurred in all three patients but resolved without complications. There were no reported cases of neurological toxicity, although transient low white blood cell counts were observed within the first month.
While encouraging, these early findings are based on a limited sample size and focus primarily on safety. Additional research is needed to determine long-term efficacy and broader applicability for people with progressive MS, a population with limited treatment options.