Autologous bone marrow-derived MSC-NPs are currently being investigated as a novel cell therapy aimed at mitigating the neurologic disability associated with progressive MS. Pre-clinical studies have elucidated some of the trophic and immunomodulatory effects of MSC-NPs in models of MS. We recently completed a phase I open-label clinical trial in 20 patients with progressive MS, which demonstrated the safety and tolerability of 3 separate IT doses of MSC-NPs spaced 3 months apart. Preliminary efficacy data from the study suggested that treatment with MSC-NPs was associated with improved motor strength and bladder function in a subset of patients with MS. The exact therapeutic mechanism of action remains unknown.

Treatment with intrathecal MSC-NPs was associated with reduced CCL2 levels in CSF post-treatment, which may potentially reflect MSC-NP action on activated microglia. Conversely, TGF-β2, a marker of anti-inflammatory microglia, was increased and inversely correlated with CCL2. An additional marker of activated microglia, chitinase-3 like 1 protein (CHI3L1), demonstrated a higher baseline level in patients classified as non-responders.