Review/Advances in Neurological Therapeutics (2015). Motor neuron disease

Abstract

Amyotrophic lateral sclerosis (ALS) is the most rapidly progressive motor neuron disease (MND) in adults, characterized by the selective death of motor neurons in the motor cortex, brainstem and spinal cord. Only supportive care and riluzole are available worldwide to date. This review provides a general overview of preclinical and clinical advances in 2015 and summarizes the literature regarding emerging therapeutic approaches. The topics include research using next–generation sequencing, progress in the pathomechanism of C9ORF72–mutated ALS, therapeutic strategies on mitochondrial pathology, novel investigation of therapies from Japan, strategies focusing on growth factors and neuro–inflammations, therapeutic strategies using iPS cells. We also discuss about the biomarkers and the problems of the design of clinical trials.