Implications for CMT: effects of mesenchymal stem cells

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Cell Immunol. 2008 Jul 14. [Epub ahead of print]

Immune effects of mesenchymal stem cells: Implications for Charcot-

Marie-Tooth disease.Leal A, Ichim TE, Marleau AM, Lara F, Kaushal S,

Riordan NH.

School of Biology and Neuroscience Research Program, University of

Costa Rica, San José, Costa Rica, USA.

Mesenchymal stem cell (MSC) therapy is the most clinically advanced

form of cell therapy, second to hematopoietic stem cell transplants.

To date, MSC have been used for immune modulation in conditions such

as Graft Versus Host Disease (GVHD) and Crohn's Disease, for which

Phase III clinical trials are currently in progress.

Here, we review the immunological properties of MSC and make a case

for their use in treatment of Charcot-Marie-Tooth disease type 1

(CMT1), a group of inherited peripheral neuropathies. CMT1 is

characterized by demyelination and aberrant immune activation making

this condition an ideal target for exploration of MSC therapy, given

the ability of these cells to promote sheath regeneration as well as

suppress inflammation.

Studies supporting this hypothesis will be presented and placed into

the context of other cell-based approaches that are theoretically

feasible. Given that MSCs selectively home to areas of inflammation,

as well as exert effects in an allogeneic manner, the possibility of

an " off the shelf " therapy for CMT1 will be discussed.