Stem cell transplantation is a promising therapy for neuromyelitis optica (NMO). Among stem cell varieties,
human umbilical cord-derived mesenchymal stem cells (hUC-MSCs) possess many advantages, add a differential
potential into neural cells, secrete a set of trophic factors and cytokines, regulate immunological function, and have
therapeutic potential for neurological diseases. In this study, hUC-MSCs transplantation was used to treat five NMO
patients with follow-up for 18 months including evaluation of Expanded Disability Status Scale (EDSS) levels, clinical
course, magnetic resonance imaging (MRI) characteristics, adverse events, and so on. Among the five cases, four showed
therapeutic improvement after hUC-MSCs treatment. Both symptoms and signs improved and relapse frequencies were
reduced. MRI characteristics also showed decreased volume and severity of lesions, while few adverse events occurred.
The results suggest that hUC-MSCs transplantation appear safe and might be effective for NMO treatment in the near
future. In addition, according to flow cytometry assay (FACS) results, B cells of blood were inhibited while T cells
increased after treatment, indicating an immune-related mechanism.