According to a population-based estimate (2019), 300,000 to 400,000 individuals are affected by multiple sclerosis (MS): the most common progressive neurologic disease in the United States. The disease is considered a chronic inflammatory disease of the central nervous system (CNS) that leads to demyelination and axonal loss. The disease is complex and multi-factorial with the involvement of an abnormal response of the immune system cells (B cells & T cells). Close to 80% of the patients show the relapsing-remitting form (RRMS) while two-thirds of them develop a secondary progressive form. The remaining patients show primary progressive multiple sclerosis (PPMS).
The major goals of the research are to target the inflammation and hence protect the nervous system. A 2010-published research article described the “International Stem Cells Transplantation Study Group” (IMSCTSG) of International experts in multiple sclerosis and stem cells. A phase 1/2 open-safety clinical trial published in 2010 (Karussis et al, 2010) reported the safety of mesenchymal stem cell transplantation in patients with multiple sclerosis. MSCs isolated from the bone marrow were administered into 15 patients with intrathecally/intravenously. There were no reports of any adverse side effects while the Expanded Disability Status Scale [EDSS] score improved from 6.7 to 5.9 during the 6- to 25-month observation.
In another 2011-published phase II open-label clinical trial (ClinicalTrials.gov-NCT00395200), 10 patients with average disease duration with secondary progressive multiple sclerosis (SPMS) received MSCs intravenously. The neuroprotective effects of MSCs were shown by improvements in visual functions such as visual acuity and increased optic nerve area (Connick et al, 2011).
Genomics and epidemiological data show that the aberrant activation of the immune system accounts for the initiation of MS. Apart from the role of genetics, there is also an environmental influence such as the involvement of vitamin D deficiency and smoking. MSCs are particularly known for their ability to modulate immune responses. This point coupled with their ease of isolation, minimal ethical issues and their safety shown by studies makes them a valuable treatment strategy for MS.
Dulamea A. (2015). Mesenchymal stem cells in multiple sclerosis – translation to clinical trials. Journal of medicine and life, 8(1), 24–27.
Mitchell T. Wallin, William J. Culpepper, Jonathan D. Campbell, et al. The prevalence of MS in the United States. A population-based estimate using health claims data. Neurology Mar 2019, 92 (10) e1029-e1040; DOI: 10.1212/WNL.0000000000007035.
Freedman MS, Bar-Or A, Atkins HL, et al. The therapeutic potential of mesenchymal stem cell transplantation as a treatment for multiple sclerosis: consensus report of the International MSCT Study Group. Mult Scler. 2010;16(4):503‐510. doi:10.1177/1352458509359727.
Karussis D, Karageorgiou C, Vaknin-Dembinsky A, et al. Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis. Arch Neurol. 2010 Oct; 67(10):1187-94.
Connick P, Kolappan M, Patani R, et al. The mesenchymal stem cells in multiple sclerosis (MSCIMS) trial protocol and baseline cohort characteristics: an open-label pre-test: post-test study with blinded outcome assessments. Trials. 2011 Mar 2; 12():62.