Promoting remyelination: A case study in regenerative medicine

Therapeutics that modulate regenerative mechanisms by targeting the activity of endogenous (adult) stem cell populations have the potential to revolutionize medicine. In many human disease states, capacity to repair damaged tissue underlies progressive decline and disease progression. Recent insights derived from efforts aimed at promoting remyelination for the treatment of multiple sclerosis (MS) highlight the importance of considering the limiting factors and underlying mechanisms associated with all aspects of disease onset, progression and recovery, during both the discovery and clinical stages of developing a regenerative medicine. This perspective presents general considerations for the development of regenerative therapies, using remyelination as a case study. •Remyelination is a spontaneous regenerative response to demyelination.•New oligodendrocytes derived from a progenitor cell population define and facilitate remyelination.•Phenotypic screening has led to the discovery of remyelinating small molecules.•Age and disease progression negatively impact remyelination capacity.•The underlying causes of remyelination failure informs regenerative drug development.