Most of the tissues in the human body are capable of regeneration to maintain their normal function and repair damage caused by injury and disease. Progenitor aims to discover small molecules that selectively stimulate this regenerative process.
Repair and regeneration is dependent on stem cells resident in the adult body, that give rise to a hierarchy of progenitor cells and finally to terminally differentiated cells that perform tissue-specific functions. Differentiation is largely regulated by proteins such as growth factors that stimulate specific biochemical signalling pathways. Examples of protein growth factors are erythropoietin (EPO), which promotes the formation of red blood cells, and thrombopoietin (TPO), which stimulates the production of platelets.
While many molecules with regenerative properties are natural proteins, it is possible to use traditional pharmaceutical drug screening technologies to discover synthetic small molecule modulators of regenerative pathways. For example, a TPO small molecule mimetic, Promacta® (GlaxoSmithKline), was discovered in this way and is now used to treat blood clotting disorders.