Novel Therapeutic Approaches for Inclusion Body Myositis

Thomas E. Lloyd

Disclosures

Curr Opin Rheumatol. 2010;22(6):658-664. 

In This Article

Stem Cell Therapy

In addition to stimulation of endogenous muscle stem cells with growth factors, enhancing muscle growth with exogenous stem cells is a promising area of therapeutic development.[20•] In particular, the possibility of converting patient's own cells into muscle progenitors using induced pluripotent stem (iPS) cell technology is a new and exciting field of research,[21] although many obstacles to clinical application remain. Satellite cells have long been known to lie quiescent within the myofiber basal lamina, proliferate in response to muscle injury, and replace degenerating myofibers. In recent years, improvements in isolating satellite cells from muscle have been made, but the utility of satellite cells in cell-based therapy is severely limited by their poor expansion in culture and inability to cross the endothelial wall, prohibiting systemic delivery. Embryonic stem cells, hematopoietic stem cells, mesoangioblasts (MABs), and other myoblast progenitors may be able to circumvent these problems and are capable of differentiating into myofibers in vitro or in vivo.[22,23] MABs, isolated from blood vessel walls, have shown particular promise, leading to improved muscle regeneration and strength in animal models of muscular dystrophy when delivered intraarterially.[24,25] An improved understanding of the efficacy and safety of these stem cells will likely lead to clinical trials for cell-based therapy of skeletal muscle diseases in the near future.

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