Stem and progenitor cell-based therapy of myelin disorders

Much of clinical neurology is concerned with diseases of-or involving-the brain's subcortical white matter. Common to these disorders is the loss of myelin, reflecting the elimination or dysfunction of oligodendrocytes and fibrous astrocytes. As such, the introduction of glial progenitor cells, which can give rise to new oligodendrocytes and astrocytes alike, may be a feasible strategy for treating a broad variety of conditions in which white matter loss is causally involved. This review first covers the sourcing and production of human glial progenitor cells, and the preclinical evidence for their efficacy in achieving myelin restoration in vivo. It then discusses both pediatric and adult disease targets for which transplanted glial progenitors may prove of therapeutic value, those challenges that remain in the clinical application of a glial cell replacement strategy, and the clinical endpoints by which the efficacy of this approach may be assessed.