Mesenchymal Stem Cell Transplantation as a Therapeutic Agent for Multiple Sclerosis

Posted June 1, 2018

Jeffrey Cohen, M.D., Cleveland Clinic Foundation, Mellen Center for Multiple Sclerosis Treatment and Research

Jeffrey Cohen, M.D., Cleveland Clinic Foundation, Mellen Center for Multiple Sclerosis Treatment and Research
Dr. Jeffrey Cohen

Over two million people worldwide are affected by multiple sclerosis (MS), a chronic disease in which the immune system attacks the protective myelin covering on nerve fibers of the central nervous system, causing damage to both the myelin and the nerve fibers. The diagnosis is based on characteristic clinical manifestations, magnetic resonance imaging (MRI) findings and, in some cases, additional testing. While approved therapies effectively prevent accumulation of focal inflammatory damage seen in relapsing MS, there are no approved therapies that directly promote repair of the myelin sheath.

Mesenchymal stem cells (MSCs) possess several properties that make them an attractive therapeutic agent, especially for conditions that result in tissue damage or inflammation. MSCs can be isolated from several types of adult tissues, expanded in culture ex vivo, induced to differentiate into cells of the mesodermal lineage, and then returned to the patient to traffic to particular areas of the body. With support from a fiscal year 2009 Clinical Trial Award, Dr. Jeffrey Cohen and his team sought to test MSC transplantation as a potential approach to neurorepair in MS. Results from a pilot study conducted to test the feasibility, safety, and tolerability of autologous MSC cell transplantation in patients with relapsing forms of MS were recently published in the Multiple Sclerosis Journal. This was a single-site, investigator-run, open label, Phase I study, which included 24 patients ranging in age from 18 to 55. Patients were diagnosed with relapse-remitting MS or secondary progressive MS and fulfilled the 2010 McDonald diagnostic criteria for MS. MSCs were isolated and expanded in culture from each patient after bone marrow aspiration. Patients were subsequently infused with 1 to 2 million MSCs per kilogram of body weight, and safety and tolerability were monitored up to 6 months post-infusion.

Data collected during the study indicate that autologous MSC transplantation is safe and well tolerated, as no severe or serious adverse events related to the treatment were reported during this trial. While inhibition of the gadolinium-enhancing lesions was not observed during this study, a possible benefit was documented on several MRI measures and on the Expanded Disability Status Scale, related to the distance a participant walks. Results from this pilot study conducted by Dr. Cohen and his team support the need for further exploration of MSCs as a therapeutic agent for MS.

Several other pilot trials of MSC transplantation in MS by other groups of investigators are ongoing. Based on the results of the above trial and these other trials, Dr. Cohen and his team are planning future, more definitive trials.


Cohen JA, Imrey PB, Planchon SM, Bermel RA, Fisher E, Fox RJ, Bar-Or A, Sharp SL, Skaramagas TT, Jagodnik P, Karafa M, Morrison S, Reese Koc J, Gerson SL, Lazarus HM. 2018. Pilot trial of intravenous autologous culture-expanded mesenchymal stem cell transplantation in multiple sclerosis. Mult. Scler. 24(2): 501-511.

Planchon SM, Lingas KT, Reese KoƧ J, Hooper BM, Maitra B, Fox RM, Imrey PB, Drake KM, Aldred MA, Lazarus HM, Cohen JA. 2018. Feasibility of mesenchymal stem cell culture expansion for a phase I clinical trial in multiple sclerosis. Mult. Scler. J. Exp. Transl. Clin. 4(1):2055217318765288.


Public and Technical Abstracts: A Phase I Assessment of Mesenchymal Stem Cells for the Treatment of Multiple Sclerosis

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Last updated Thursday, May 26, 2022