Human umbilical cord mesenchymal stromal cells therapy for neuromyelitis optica spectrum disorder: a phase 1/2a trial - PubMed

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Human umbilical cord-derived mesenchymal stromal cells (hUC-MSCs) therapy was evaluated for safety and efficacy in treating neuromyelitis optica spectrum disorder (NMOSD).
The trial included 31 NMOSD patients across three dose groups, receiving four infusions every three months with a 15-month follow-up.
Primary outcome was time to first recurrence, with secondary outcomes focusing on clinical scores, MRI lesions, and exploratory findings.
hUC-MSC infusion was well tolerated, with mostly mild adverse events and rare severe adverse events unrelated to treatment.
Median relapse-free interval increased significantly post-treatment, especially in medium- and high-dose groups.
Annualized Relapse Rate (ARR) dropped considerably from 1 to 0 during the two years before and after therapy.
Clinical scores improved in low and medium-dose groups, and total volume of high-signal white matter lesions in the brain decreased significantly.
Exploratory findings suggested potential involvement of thioredoxin and oxidative phosphorylation (OXPHOS) in boosting Treg differentiation and suppressive capacity.
The trial concluded that intravenous hUC-MSC administration is safe and shows potential efficacy, with medium dose being the best compromise between safety and effectiveness.

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