Novel therapeutic approaches for motor neuron disease

Handb Clin Neurol. 2023:196:523-537. doi: 10.1016/B978-0-323-98817-9.00027-2.


Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that leads to the neurodegeneration and death of upper and lower motor neurons (MNs). Although MNs are the main cells involved in the process of neurodegeneration, a growing body of evidence points toward other cell types as concurrent to disease initiation and propagation. Given the current absence of effective therapies, the quest for other therapeutic targets remains open and still challenges the scientific community. Both neuronal and extra-neuronal mechanisms of cellular stress and damage have been studied and have posed the basis for the development of novel therapies that have been investigated on both animal models and humans. In this chapter, a thorough review of the main mechanisms of cellular damage and the respective therapeutic attempts targeting them is reported. The main areas covered include neuroinflammation, protein aggregation, RNA metabolism, and oxidative stress.

Keywords: ALS; Motor neurons; Neuroinflammation; Oxidative stress; Protein aggregation; RNA metabolism.

Publication types

  • Review

MeSH terms

  • Amyotrophic Lateral Sclerosis* / therapy
  • Animals
  • Cognition
  • Humans
  • Motor Neuron Disease* / therapy
  • Motor Neurons
  • Neurodegenerative Diseases*