Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis

Currently, treatment with disease-modifying therapies has limited impact on the progressive stages of multiple sclerosis. Stem-cell therapies have been claimed to be a possible treatment for patients with secondary progressive multiple sclerosis (SPMS). Leone MA et Al. in this phase I, open-label, multicenter, dose-escalation clinical trial of intracerebroventricular transplantation of allogeneic human neural stem/progenitor cells (NSCs) (from a single donor) in 15 patients with SPMS, demonstrated that:

  • the treatment is feasible and well-tolerated in humans
  • the disease in the treated patients remained stable during the 12-month follow-up period
  • in a subgroup analysis, there was an inverse correlation between the doses of the injected NSCs and the parenchymal brain volume changes
  • the treatment presented a metabolic impact  with an increase of CSF acyl-carnitines and fatty acids at follow-up

Although this highly relevant study has several limitations, it is an encouraging and essential step forward for the use of cell-therapy in multiple sclerosis.