Understanding ALS: new therapeutic approaches

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease associated with motor neuron degeneration, muscle atrophy and paralysis. Although numerous pathological mechanisms have been elucidated, ALS remains an invariably fatal disease in the absence of any effective therapy. The heterogeneity of the disease and the failure to develop satisfactory therapeutic protocols reinforce the view that ALS is a multi-factorial and multi-systemic disease. Thus, a better understanding of the pathogenic mechanisms and study of the potential pathological relationship between the various cellular processes is required to ensure efficacious therapy. The pathogenic mechanisms associated with ALS are reviewed, and the strengths and limitations of some new therapeutic approaches are discussed.

Keywords: ALS-associated genes; amyotrophic lateral sclerosis; complexity of ALS pathogenesis; growth factors; mutant SOD1 toxicity; neuromuscular disease; oxidative stress; skeletal muscle; stem cells; therapeutic approaches.