Chapter 18 - New insights on granulocyte-macrophage colony-stimulating factor (GM-CSF) as potential therapeutic target for immune-mediated neurological disorders

Abstract

Multiple sclerosis (MS), the most prevalent nontraumatic disability in adults from western societies, is characterized as an inflammatory neurodegenerative disease of the central nervous system (CNS). Although several therapeutic approaches have been approved for its treatment, a continuous effort in the search of novel strategies is restlessly underway. This is supported by the identification of poor safety profiles in some available drugs and by the paucity of effective treatment for patients with progressive conditions. In recent years, the granulocyte-macrophage colony-stimulating factor (GM-CSF) has become one of the most attractive targets for drug discovery on this disease. Here, we review up-to-date data supporting the rationale for studying GM-CSF as a therapeutic target for MS, from its receptor expression in the CNS to current data from animal studies and clinical trials in MS. Finally, our discussion addresses future avenues aiming to exploit the GM-CSF pathway interference as therapeutic for immune-mediated neurological disorders.