Colony-stimulating factor 3 as a key mediator in the progression of idiopathic pulmonary fibrosis: a novel therapeutic target

Signal Transduct Target Ther. 2025 Oct 2;10(1):322. doi: 10.1038/s41392-025-02421-6.

Abstract

Idiopathic pulmonary fibrosis (IPF) is a fatal lung disease characterized by excessive ECM deposition and myofibroblast accumulation driven by cytokine dysregulation. This study identified granulocyte colony-stimulating factor 3 (CSF3) as a key mediator of IPF progression. Elevated CSF3 expression was observed in the lung tissues of IPF patients. Recombinant CSF3 promoted myofibrogenesis in lung fibroblasts, whereas CSF3-deficient mice were protected from bleomycin-induced pulmonary fibrosis. Treatment with novel CSF3-neutralizing antibodies significantly restored fibrosis in IPF mice by suppressing myofibroblast differentiation and reducing ECM deposition. Here, we demonstrated a reciprocal regulatory relationship between CSF3 and TGF-β that amplifies pro-fibrotic signaling. Our mechanistic studies revealed that CSF3 acts as an upstream regulator of TGF-β, forming a positive feedback loop that significantly accelerates the fibrotic process. Knockout or neutralization of CSF3 suppressed fibrosis by reducing TGF-β levels, whereas treatment with recombinant CSF3 promoted fibrosis with increased TGF-β expression. Notably, while CSF3 inhibition reduced TGF-β expression levels, it did not decrease them below normal levels. This finding suggests that inhibiting CSF3 could simultaneously reduce fibrosis by suppressing excessive TGF-β expression while also minimizing side effects by maintaining TGF-β homeostasis. Taken together, these results provide strong evidence that CSF3 is a critical driver of IPF pathogenesis and that targeting CSF3 may provide a therapeutic strategy by modulating TGF-β signaling and restoring the ECM and cellular homeostasis.

MeSH terms

  • Animals
  • Antibodies, Neutralizing
  • Bleomycin
  • Cell Differentiation / genetics
  • Disease Progression
  • Granulocyte Colony-Stimulating Factor* / genetics
  • Humans
  • Idiopathic Pulmonary Fibrosis* / chemically induced
  • Idiopathic Pulmonary Fibrosis* / drug therapy
  • Idiopathic Pulmonary Fibrosis* / genetics
  • Idiopathic Pulmonary Fibrosis* / metabolism
  • Idiopathic Pulmonary Fibrosis* / pathology
  • Mice
  • Mice, Knockout
  • Myofibroblasts / drug effects
  • Myofibroblasts / metabolism
  • Myofibroblasts / pathology
  • Signal Transduction / genetics
  • Transforming Growth Factor beta* / genetics

Substances

  • Bleomycin
  • Transforming Growth Factor beta
  • Granulocyte Colony-Stimulating Factor
  • Antibodies, Neutralizing