Review: Evidence-based drug treatment in amyotrophic lateral sclerosis and upcoming clinical trials

Abstract

Amyotrophic lateral sclerosis/motor neuron disease is a severe neurodegenerative disease characterized by upper and lower motor neuron degeneration for which there is no truly effective treatment. Several therapies have shown promise in preclinical models of motor neuron disease; however, most of them failed in human studies, so that the noticeable progress in understanding the cellular mechanisms of motor neuron degeneration has not been matched with the development of therapeutic strategies to prevent disease progression or to extend survival longer than achieved by riluzole. We review treatment development in motor neuron disease and discuss the strengths and limitations of past as well as upcoming clinical trials.