Stem and Progenitor Cell-Based Therapy of the Central Nervous System: Hopes, Hype, and Wishful Thinking

Abstract

A variety of neurological disorders are attractive targets for stem and progenitor cell-based therapy. Yet many conditions are not, whether by virtue of an inhospitable disease environment, poorly understood pathophysiology, or poor alignment of donor cell capabilities with patient needs. Moreover, some disorders may be medically feasible targets but are not practicable, in light of already available treatments, poor risk-benefit and cost-benefit profiles, or resource limitations. This Perspective seeks to define those neurological conditions most appropriate for cell replacement therapy by considering its potential efficacy and clinical feasibility in those disorders, as well as potential impediments to its application.

Keywords: astrocyte; cell therapy; glial progenitor cell; myelin disease; neural stem cell; neurodegenerative disease; neurological therapeutics; oligodendrocyte progenitor cell.

Figure 1. Neural and glial cell therapeutics and their disease targets

This schematic illustrates the principal sources of transplantable human neural stem cells and phenotypically-restricted neuronal and glial progenitor cells, and highlights the most feasible current opportunities for their use in treating disorders of the brain.