Abstract

Medicinal Chemistry In Regenerative Medicine: Discovery Of In Vivo-Active Small Molecule Modulators Of Endogenous Stem Cells

Angela Russell, Professor, University of Oxford

The ability to harness the potential of adult stem and precursor cells would be a major advance in the treatment of human disease. They are remarkable cells characterized by their ability to divide and to differentiate via a number of steps to embryonic and adult somatic cell lineages. Such cells thus hold enormous promise both for in vitro screening tools for drug efficacy and toxicity testing, and especially for regenerative therapies treating a wide range of disorders with high unmet medical need such as neurodegenerative diseases, diabetes, heart disease, and vision loss. ?The discovery of small molecules to control cell fate has attracted immense interest in recent years. The ability to control each step in proliferation, differentiation and dedifferentiation/ reprogramming processes would allow control of the selective production of different tissue types both in vitro and, importantly in many instances, directly in vivo. The use of chemicals to manipulate cell fate offers many significant advantages over other techniques in terms of speed, cost, reproducibility and the ability to influence cell fate reversibly.?