Acetyl-L-carnitine and Amyotrophic Lateral Sclerosis: Current Evidence and Potential use

CNS Neurol Disord Drug Targets. 2024;23(5):588-601. doi: 10.2174/1871527322666230330083757.

Abstract

Background: The management of neurodegenerative diseases can be frustrating for clinicians, given the limited progress of conventional medicine in this context.

Aim: For this reason, a more comprehensive, integrative approach is urgently needed. Among various emerging focuses for intervention, the modulation of central nervous system energetics, oxidative stress, and inflammation is becoming more and more promising.

Methods: In particular, electrons leakage involved in the mitochondrial energetics can generate reactive oxygen-free radical-related mitochondrial dysfunction that would contribute to the etiopathology of many disorders, such as Alzheimer's and other dementias, Parkinson's disease, multiple sclerosis, stroke, and amyotrophic lateral sclerosis (ALS).

Results: In this context, using agents, like acetyl L-carnitine (ALCAR), provides mitochondrial support, reduces oxidative stress, and improves synaptic transmission.

Conclusion: This narrative review aims to update the existing literature on ALCAR molecular profile, tolerability, and translational clinical potential use in neurodegeneration, focusing on ALS.

Keywords: ALCAR; Amyotrophic lateral sclerosis; MNDs.; TDP-43; central nervous system; motor neuron diseases.

Publication types

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

MeSH terms

  • Acetylcarnitine* / pharmacology
  • Acetylcarnitine* / therapeutic use
  • Amyotrophic Lateral Sclerosis* / drug therapy
  • Amyotrophic Lateral Sclerosis* / metabolism
  • Animals
  • Humans
  • Mitochondria / drug effects
  • Mitochondria / metabolism
  • Oxidative Stress* / drug effects

Substances

  • Acetylcarnitine