Those who treat autoimmune diseases such as MS, much has been expected from stem cell research, and an ongoing study at the University of Cambridge, UK, has become a focus of high hopes.
Among those who treat autoimmune diseases such as MS, much has been expected from stem cell research, and an ongoing study at the University of Cambridge, UK, has become a focus of high hopes. Now in phase II trials, a small proof-ofconcept trial of 10 MS patients and eight healthy controls showed a neuroprotective effect of treatment with a single injection of autologous mesenchymal stem cells. The cells were obtained through aspiration of the patients’ bone marrow.
MS patients selected for the 18-month trial had secondary progressive MS and showed objective evidence of MS-caused disability of their visual pathways (an average expanded disability status score [EDSS] of 5.5 to 6.5). After treatment, MS patients had a significant improvement in visual acuity and an increase in optic nerve area. These results indicated that stem cell therapy had a neuroprotective, possibly regenerative, effect on myelin.
A primary goal of the study was to assess feasibility and safety of the procedure; the secondary outcome was to assess efficacy on a “sentinel lesion”—in this case, damage to the optic nerve—as a sign that the treatment would have a similar effect on the patients’ other MS-caused conditions Over the course of 6 to 10 months after a single infusion of autologous mesenchymal stem cells in the central nervous system, researchers saw evidence of functional, neurophysiological, and structural improvement in vision without evidence of significant adverse effects.
Peter Connick, MRCP, from the Department of Clinical Neurosciences, University of Cambridge, and colleagues reported the results in the February 2012 issue of Lancet Neurology. The authors noted that despite major advances in immunomodulatory therapies for patients with relapsing-remitting MS, there are no proven or available treatments to slow, stop, or reverse the accumulation of fixed disability that is typical in patients with secondary progressive disease.
• 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;12:62-72.
• Connick P, Kolappan M, Crawley C, et al. Autologous mesenchymal stem cells for the treatment of secondary progressive multiple sclerosis: an open-label phase 2a proofof- concept study. Lancet Neurol. 2012;11:150-156.