Stem cell modulators (SCMs): a small molecule and biologic approach to stem cell therapeutics

The scientific advancements of stem cell and developmental biology over the past decade have formed a solid foundation to pursue regenerative medicines for diseases with little to no treatment options. These discoveries have contributed to a more comprehensive understanding of stem cell niches across many tissues of the body and of the key stem cell pathways, such as Wnt, Hedgehog and Notch, which influence these niches. The quickest way to bring in the horizon for stem cell medicines might be through the use of conventional pharmaceuticals to modulate key stem cell pathways. The premise of this approach is to intervene using small molecules and biologics, modulating stem cell biology for a desired outcome such as cell proliferation and differentiation for therapeutic benefit. As each year passes, more stem and progenitor cell populations are being identified in adult tissues, and myriad proof of concepts for treating diseases have emerged on the basis of activating stem and progenitor cells. With a dynamic and ever-growing foundation of novel biology, we explore in this review stem cell modulators (SCMs), small molecules and biologics that seek to modulate cells in vivo to harness stem cell-related biology for therapeutic applications.

Section editor:Randall Moon – University of Washington, Pharmacology/HHMI, Seattle, WA, USA