Title

Recent advances in understanding amyotrophic lateral sclerosis and emerging therapies

Document Type

Article

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that is characterized by degeneration of both upper and lower motor neurons and subsequent progressive loss of muscle function. Within the last decade, significant progress has been made in the understanding of the etiology and pathobiology of the disease; however, treatment options remain limited and only two drugs, which exert a modest effect on survival, are approved for ALS treatment in the US. Therefore, the search for effective ALS therapies continues, and over 60 clinical trials are in progress for patients with ALS and other therapeutics are at the pre-clinical stage of development. Recent advances in understanding the genetics, pathology, and molecular mechanisms of ALS have led to the identification of novel targets and strategies that are being used in emerging ALS therapeutic interventions. Here, we review the current status and mechanisms of action of a selection of emerging ALS therapies in pre-clinical or early clinical development, including gene therapy, immunotherapy, and strategies that target neuroinflammation, phase separation, and protein clearance.

Keywords

ALS, ALS therapeutics, Amyotrophic lateral sclerosis, clinical trials, gene therapy, immunotherapy, platform trials

Publication Date

1-1-2020

Publication Title

Faculty reviews

E-ISSN

2732-432X

Volume

9

First Page

12

PubMed ID

33659944

Digital Object Identifier (DOI)

10.12703/b/9-12

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