Progressive multiple sclerosis is a significant disruptive neurodegenerative disease that interferes with the brain’s ability to control the body; the condition continues to get worse over time and, to date, has no known therapeutic treatment or cure. 

Petrou Et. Al’s double-blind clinical trial examined the therapeutic efficacy of mesenchymal stem cell (MSC) transplantation in active progressive multiple sclerosis and explored the most favorable route of cell delivery (intravenous or intrathecal injections).

Prior to this study, previous trials examining various types of MSC administration in the therapeutic treatment of multiple sclerosis have demonstrated the clinical safety of MSC administration but have not identified treatments to suppress central nervous system (CNS) inflammation associated with the progression of diseases like progressive multiple sclerosis. 

Several studies have also demonstrated that CNS loses the ability to repair and regenerate over time. Considering that stem cells, and specifically MSCs, have demonstrated to provide additional benefits, including immunomodulatory and neurotrophic effects, when used in the treatment of stroke and multi-system atrophy, they appear to be a viable potential therapeutic treatment for active progressive multiple sclerosis.  

For the purposes of Petrou Et. Al’s study, a total of 48 participants with a mean disease (active progressive multiple sclerosis) duration of 12.70 years were included as part of this study either as part of a placebo group, MSC-IV group, or MSC-IT group; selected treatment was applied at 3-month and 6-month marks of the study.

At the conclusion of this study, the authors report no serious, treatment-related adverse effects were observed and significantly fewer patients in the MSC-IT and MSC-IV groups experienced treatment failure when compared to the placebo group. 

By reviewing changes observed in ambulation index, the sum of functional scores, 25-foot timed walking test, PASAT and OWAT/KAVE cognitive test, and the rate of change in T2 lesion load on MRI observed after the 6th-month treatment, researchers also found beneficial effects in both the MSC-IT and MSC-IV groups.

It appears that repeated intrathecal injection of MSC during the second round of treatment (Month 6) significantly improved the effects measured during the first round of similar treatment (Month 3); similar, but less significant benefits were also observed in the MSC-IV group.  Specifically, researchers report that these observed benefits may indicate the involvement of immunomodulatory and neuroprotective mechanisms.

Of particular interest is the fact that the benefits with clinical significance were observed in participants with progressive multiple sclerosis found to be previously unresponsive to conventional immunotherapies and currently with limited treatment options.

In conclusion, this study found short-term clinical efficacy and perhaps neuroprotection by administration of MSCs to participants with progressive multiple sclerosis. The researchers also found that while repeated injections of both MSC-IT and MSC-IV produced beneficial effects, intrathecal administration appears to produce more clinically significant and observable benefits than MSC-IV. 

These findings are recommended for use in the design of future studies examining the impact of cell therapy on neurodegeneration and neuronal regeneration and warrant Phase III study to confirm the therapeutic potential of cellular therapy in neurodegenerative and neuroinflammatory diseases, including multiple sclerosis.

Source: (2020, December 1). Beneficial effects of autologous mesenchymal stem … – PubMed., from https://pubmed.ncbi.nlm.nih.gov/33253391/

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