Review/Advances in Neurological Therapeutics (2016). Motor neuron disease

Abstract

Amyotrophic lateral sclerosis (ALS) is the progressive motor neuron disease (MND) in adults, characterized by the selective death of motor neurons in the motor cortex, brainstem and spinal cord. This review provides a general overview of preclinical and clinical advances in 2016 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, neuroinflammation, autophagy, growth factor supplementation, axonal pathology in ALS. Clinical trials for ALS targeting on these pathomechanisms are on–going including intrathecal administration of hepatocyte growth factor (HGF).