A dual-targeting CAR-T cell therapy called CTA313 showed early signs of stabilizing disability in patients with progressive multiple sclerosis. In a phase 1 trial of just 3 patients, two saw disability score improvements and all showed better functional test results at 3 months. The therapy was well-tolerated, with no cases of neurotoxicity or graft-versus-host disease reported.
A novel allogeneic CAR-T cell therapy, CTA313, is showing early encouraging results in one of neurology's toughest-to-treat conditions: progressive multiple sclerosis (MS). Developed by Imviva Biotech, CTA313 simultaneously targets two immune proteins — CD19 and BCMA — and is derived from healthy donor cells, making it an "off-the-shelf" option that could reach patients faster than therapies requiring a patient's own cells.
Early phase 1 data from 3 patients (2 with secondary progressive MS, 1 with primary progressive MS) were presented at the 10th Joint ACTRIMS-ECTRIMS Meeting in Toronto. After a single infusion, 2 patients showed disability score improvements at 3 months, all 3 improved on timed functional tests, and 2 of 3 had cerebrospinal fluid markers of inflammation turn negative by day 28. Side effects were mild, with only grade 1 cytokine release syndrome observed and no neurotoxicity or graft-versus-host disease.
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Why it matters: Progressive MS has very few effective treatment options, partly because the inflammation driving it is locked inside the central nervous system. CAR-T cells can cross the blood-brain barrier, potentially reaching immune cells that current therapies can't. While these results are very preliminary, they hint at a new therapeutic frontier for a condition that has long been resistant to treatment.
