Mesenchymal Stem Cell-Derived Neural Progenitors Aid MS

On Thursday, June 13, 2024, a significant study suggested that specific patients with progressive multiple sclerosis (MS) might benefit from mesenchymal stem cell-neural progenitors (MSC-NPs). The research, published online on May 23 in Stem Cell Research & Therapy, was led by Dr. Violaine K. Harris and her team at the Tisch Multiple Sclerosis Research Center of New York, based in New York City.

In this study, a randomized, double-blind trial with a compassionate crossover design was conducted at a single site. The participants were divided based on their initial Expanded Disability Status Scale (EDSS, ranging from 3.0 to 6.5) scores and the type of progressive MS they had, either secondary or primary. They were then randomly assigned to receive either six intrathecal (IT) injections of autologous MSC-NPs or a placebo (saline solution) every two months, with 27 individuals in each group.

The researchers aimed to measure the efficacy of the MSC-NP treatment by comparing it to the placebo. They found no significant differences between the MSC-NP group and the placebo group regarding the EDSS Plus, which includes improvements in the EDSS, a timed 25-foot walk (T25FW), or the nine-hole peg test (with improvements seen in 33% of the MSC-NP group and 37% of the placebo group). However, in an exploratory subgroup analysis focusing on participants who needed assistance for ambulation (EDSS scores from 6.0 to 6.5), there were notable improvements in the T25FW and six-minute walk test in the MSC-NP group compared to the placebo group.

Additionally, the MSC-NP treatment group exhibited improved bladder function and a reduced rate of grey matter atrophy as seen on brain magnetic resonance imaging (MRI). Another interesting finding was the post-treatment increase in MMP9 levels and decrease in CCL2 levels in the cerebrospinal fluid of those treated with MSC-NPs.

The authors of the study highlighted the clinical and laboratory evidence supporting the efficacy of IT MSC-NP therapy in progressive MS. They emphasized the need for further research focusing on ambulatory measures as primary endpoints and exploring the optimal dosing of MSC-NPs to enhance treatment outcomes.

Two of the study's authors are noted as inventors on a U.S. patent related to this research. This study represents a potential step forward in the treatment of progressive MS, offering hope for improved mobility and quality of life for patients suffering from this condition.