A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed?

Transl Neurodegener. 2021 Aug 10;10(1):29. doi: 10.1186/s40035-021-00250-5.

Abstract

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease involving both upper and lower motor neurons, leading to paralysis and eventually death. Symptomatic treatments such as inhibition of salivation, alleviation of muscle cramps, and relief of spasticity and pain still play an important role in enhancing the quality of life. To date, riluzole and edaravone are the only two drugs approved by the Food and Drug Administration for the treatment of ALS in a few countries. While there is adequate consensus on the modest efficacy of riluzole, there are still open questions concerning the efficacy of edaravone in slowing the disease progression. Therefore, identification of novel therapeutic strategies is urgently needed. Impaired autophagic process plays a critical role in ALS pathogenesis. In this review, we focus on therapies modulating autophagy in the context of ALS. Furthermore, stem cell therapies, gene therapies, and newly-developed biomaterials have great potentials in alleviating neurodegeneration, which might halt the disease progression. In this review, we will summarize the current and prospective therapies for ALS.

Keywords: Amyotrophic lateral sclerosis; Autophagy; Gene editing; Motor neurons; Stem cells.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Amyotrophic Lateral Sclerosis / diagnosis*
  • Amyotrophic Lateral Sclerosis / therapy*
  • Clinical Trials as Topic / methods*
  • Disease Management*
  • Disease Progression*
  • Edaravone / therapeutic use
  • Free Radical Scavengers / therapeutic use
  • Humans
  • Neuroprotective Agents / therapeutic use
  • Riluzole / therapeutic use
  • Stem Cell Transplantation / methods
  • Stem Cell Transplantation / trends

Substances

  • Free Radical Scavengers
  • Neuroprotective Agents
  • Riluzole
  • Edaravone