Targeting the CSF-1/CSF-1R Axis: Exploring the Potential of CSF1R Inhibitors in Neurodegenerative Diseases

J Med Chem. 2024 Apr 11;67(7):5699-5720. doi: 10.1021/acs.jmedchem.3c02366. Epub 2024 Mar 26.

Abstract

We report herein the potential of colony-stimulating factor-1 receptor (CSF1R) inhibitors as therapeutic agents in neuroinflammatory diseases, with a focus on Alzheimer's disease (AD). Employing a carefully modified scaffold, N-(4-heterocycloalkyl-2-cycloalkylphenyl)-5-methylisoxazole-3-carboxamide, we identify highly selective and potent CSF1R inhibitors─7dri and 7dsi. Molecular docking studies shed light on the binding modes of these key compounds within the CSF1R binding site. Remarkably, kinome-wide selectivity assessment underscores the impressive specificity of 7dri for CSF-1R. Notably, 7dri emerges as a potent CSF-1R inhibitor with favorable cellular activity and minimal cytotoxicity among the synthesized compounds. Demonstrating efficacy in inhibiting CSF1R phosphorylation in microglial cells and successfully mitigating neuroinflammation in an in vivo LPS-induced model, 7dri establishes itself as a promising antineuroinflammatory agent.

MeSH terms

  • Humans
  • Macrophage Colony-Stimulating Factor
  • Molecular Docking Simulation
  • Neurodegenerative Diseases* / drug therapy
  • Phosphorylation
  • Receptor Protein-Tyrosine Kinases / metabolism
  • Receptors, Granulocyte-Macrophage Colony-Stimulating Factor

Substances

  • Macrophage Colony-Stimulating Factor
  • Receptors, Granulocyte-Macrophage Colony-Stimulating Factor
  • Receptor Protein-Tyrosine Kinases