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

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 mSOD1(G93A) 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.

Automated summary

This article introduces the basics of amyotrophic lateral sclerosis (ALS) and G-CSF, and comprehensively reviews the applications of G-CSF in clinical and pre-clinical studies, emphasizing the significant efficacy in pre-clinical studies and the failure to replicate the results in clinical studies.