Drug screening for Huntington's disease and other neurodegenerative disorders

Abstract

Devising therapies for neurodegenerative diseases remains a major challenge due to the complex etiology, prolonged disease course and the paucity of validated targets. These factors make it difficult to model neurodegenerative diseases in a manner amenable to large-scale screening. However recent developments in automation, combinatorial chemistry and high-throughput phenotypic assays have presented new opportunities for discovering small molecule therapeutics for neurodegenerative diseases. This review focuses on novel in vitro and phenotypic screens for Huntington's disease and a few other neurodegenerative diseases. The lessons learned from these screens and the potential of the small molecules identified as therapeutic leads are discussed.