Nature Reviews Neurology 6, 247-255 (May 2010) | doi:10.1038/nrneurol.2010.35

Stem cell transplantation in multiple sclerosis: current status and future prospects

Gianvito Martino, Robin J. M. Franklin, Anne Baron Van Evercooren, Douglas A. Kerr  About the authors


This article provides an overview of the current knowledge relating to the potential use of transplanted stem cells in the treatment of patients with multiple sclerosis (MS). Two types of stem cells, CNS-derived neural stem/precursor cells (NPCs) and bone marrow-derived mesenchymal stem cells (MSCs) are considered to provide reproducible and robust therapeutic effects when intravenously or intrathecally injected into both rodents and primates with experimental autoimmune encephalomyelitis. Furthermore, preliminary safety data concerning the use of intrathecally injected autologous MSCs in patients with progressive MS are available. We discuss how the data gathered to date challenge the narrow view that the therapeutic effects of NPCs and MSCs observed in the treatment of MS are accomplished solely by cell replacement. Both types of stem cell, when transplanted systemically, might instead influence disease outcome by releasing a plethora of factors that are immunomodulatory or neuroprotective, thereby directly or indirectly influencing the regenerative properties of intrinsic CNS stem/precursor cells.

Author affiliations

G. Martino, R. J. M. Franklin, A. B. Van Evercooren, D. A. Kerr
Institute of Experimental Neurology–DIBIT 2, Division of Neuroscience, San Raffaele Scientific Institute, Via Olgettina 58, 20132 Milan, Italy (G. Martino). MRC Center for Stem Cell Biology and Regenerative Medicine, Department of Veterinary Medicine, University of Cambridge, Madingley Road, Cambridge CB3 0ES, UK (R. J. M. Franklin). INSERM U975, Université Pierre et Marie Curie-Paris, and AP-HP, Hôpital Pitié-Salpétrière, Fédération de Neurologie, Paris, F-75013, France (A. B. Van Evercooren). Johns Hopkins TM Center, Johns Hopkins Hospital, Broadway Research Building, Room 759, Baltimore, MD 21287-6965, USA (D. A. Kerr).

Correspondence to: G. Martino

Published online 20 April 2010