Emerging clinical investigational drugs for the treatment of amyotrophic lateral sclerosis

IntroductionAmyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder caused by motoneuron death with a median survival time of 3-5 years since disease onset. There are no effective treatments to date. However, a variety of innovative investigational drugs and biological-based therapies are under clinical development.Areas coveredThis review provides an overview of the clinical investigational small molecules as well as a brief summary of the biological-based therapies that are currently undergoing clinical trials for the treatment of ALS. All the data were obtained from ClinicalTrials.gov (registered through November 1).Expert opinionDrug discovery for ALS is an active and evolving field, where many investigational clinical drugs are in different trials. There are several mechanisms of action supporting all these new therapies, although proteostasis is gaining stage. Probably, small orally bioavailable molecules able to recover functional TDP-43 homeostasis may have solid chances to modify ALS progression.

Automated summary

This review provides an overview of the clinical investigational small molecules and biological-based therapies that are currently undergoing clinical trials for the treatment of ALS. Despite the lack of effective treatments, there are many innovative drugs and therapies being developed. Proteostasis, particularly the recovery of functional TDP-43 homeostasis, may be a promising mechanism for modifying ALS progression.