The Rise of Molecules Able To Regenerate the Central Nervous System

Injury to the adult central nervous system (CNS) usually leads to permanent deficits of cognitive, sensory, and/or motor functions. The failure of axonal regeneration in the damaged CNS limits functional recovery. The lack of information concerning the biological mechanism of axonal regeneration and its complexity has delayed the process of drug discovery for many years compared to other drug classes. Starting in the early 2000s, the ability of many molecules to stimulate axonal regrowth was evaluated through automated screening techniques; many hits and some new mechanisms involved in axonal regeneration were identified. In this Perspective, we discuss the rise of the CNS regenerative drugs, the main biological techniques used to test these drug candidates, some of the most important screens performed so far, and the main challenges following the identification of a drug that is able to induce axonal regeneration in vivo.