Granulocyte-colony stimulating factor (G-CSF): an emerging therapeutic approach for amyotrophic lateral sclerosis (ALS)

Acta Neurol Belg. 2023 Jun;123(3):763-771. doi: 10.1007/s13760-022-01996-z. Epub 2022 Jun 23.

Abstract

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by neuronal degeneration and inflammation in the nerves. G-CSF is a 19.6-kDa hematopoietic growth factor which is essential for the proliferation and differentiation of granulocyte hematopoietic progenitors. G-CSF exerts neuroprotective activities by induction of neuronal regeneration, inhibition of neuronal apoptosis, mobilization of Hematopoietic stem cells (HSCs), regulation of pro and anti-inflammatory cytokines, and activation of angiogenesis. Pre-clinical studies have shown significant efficacy of G-CSF therapy in mSOD1G93A mice models. G-CSF treatments were able to increase the survival of mice. However, clinical studies on ALS patients failed to clone pre-clinical results. Considering the potential role of G-CSF in nervous system regeneration, this study aimed to comprehensively review the clinical and pre-clinical studies addressing G-CSF in ALS treatment.

Keywords: Amyotrophic lateral sclerosis (ALS); Granulocyte-colony stimulating factor (G-CSF).

Publication types

  • Review

MeSH terms

  • Amyotrophic Lateral Sclerosis* / drug therapy
  • Animals
  • Cytokines / metabolism
  • Granulocyte Colony-Stimulating Factor / metabolism
  • Granulocyte Colony-Stimulating Factor / pharmacology
  • Granulocyte Colony-Stimulating Factor / therapeutic use
  • Granulocytes / metabolism
  • Hematopoietic Stem Cells / metabolism
  • Mice

Substances

  • Granulocyte Colony-Stimulating Factor
  • Cytokines