Barriers to novel therapeutics in amyotrophic lateral sclerosis

Abstract

SUMMARY Amyotrophic lateral sclerosis is a devastating neurodegenerative condition primarily involving the motor system in the cerebral cortex, brain stem and spinal cord, but can, in later disease stages, also affect distinct extramotor brain regions. In this article, we discuss the prevalent barriers, including clinical and genetic variability of amyotrophic lateral sclerosis, frailty of the current mouse model and inadequateness of clinical trials, in the search for novel therapeutics. Approaches in terms of understanding the pathogenesis, and the search for biomarkers to initiate early or even presymptomatic treatment and monitor treatment effects are highlighted.