Pathogenesis and therapeutic perspectives for amyotrophic lateral sclerosis (ALS)

After gene mutations of SOD1 were found in familial amyotrophic lateral sclerosis (ALS) in 1993, many studies have elucidated pathogenesis of this progressive motor neuron disease. Among them, oxidative stress, impaired axonal transport, imbalance of survival & death signals, organellic stress (for mitochondria, endoplasmic reticulum and proteasome) are the most important with linking each other through energy failure within the motor neuron. New therapeutic approaches have also been tried, such as free radical scavenger edaravone, a continuous intra-thecal injection of neurotrophic factor IGF-1, and methylcobalamine as well as gene therapy with GDNF and regenerative therapy with stem cell activation and stem cell transplantation.