A single infusion of the CAR-T therapy zola-cel showed zero new disease activity in patients with highly active relapsing multiple sclerosis and stabilized disability in most progressive MS patients, per updated Phase 1 data. All patients at the expansion dose achieved NEDA-3 status at week 26, and nearly all stayed off disease-modifying therapy. The safety profile was manageable, with no severe cytokine release syndrome events reported.
A single infusion of zolacabtagene autoleucel (zola-cel), an autologous CD19-directed CAR-T cell therapy developed by Bristol Myers Squibb, is showing striking early results in multiple sclerosis. Updated data from the Phase 1 Breakfree-2 trial, presented at MSToronto 2026, revealed that all 4 patients with highly active relapsing MS (haRMS) treated at the expansion dose achieved no evidence of disease activity (NEDA-3) at week 26 — meaning no new lesions, no relapses, and no disability progression. Among progressive MS patients, 83% had no confirmed disability progression, and all participants at the expansion dose remained off disease-modifying therapy at last follow-up.
The therapy also showed signs of working deep within the central nervous system: 70% of participants with detectable oligoclonal bands at baseline showed a reduction by week 12, suggesting B-cell depletion inside the CNS itself. Safety was largely manageable, with cytokine release syndrome (CRS) occurring in some patients but graded only 1 or 2 in all but one case.
By the Numbers:
Why it matters: MS affects nearly 3 million people worldwide, and many patients exhaust available therapies. A one-time CAR-T infusion that could reset the immune system and eliminate the need for ongoing treatment would be a significant advance — and these early results suggest that possibility is worth pursuing.
