Selective binding and oligomerization of the murine granulocyte colony-stimulating factor receptor by a low molecular weight, nonpeptidyl ligand

J Biol Chem. 2003 Mar 14;278(11):9426-34. doi: 10.1074/jbc.M209220200. Epub 2003 Jan 10.

Abstract

Granulocyte colony-stimulating factor regulates neutrophil production by binding to a specific receptor, the granulocyte colony-stimulating factor receptor, expressed on cells of the granulocytic lineage. Recombinant forms of granulocyte colony-stimulating factor are used clinically to treat neutropenias. As part of an effort to develop granulocyte colony-stimulating factor mimics with the potential for oral bioavailability, we previously identified a nonpeptidyl small molecule (SB-247464) that selectively activates murine granulocyte colony-stimulating factor signal transduction pathways and promotes neutrophil formation in vivo. To elucidate the mechanism of action of SB-247464, a series of cell-based and biochemical assays were performed. The activity of SB-247464 is strictly dependent on the presence of zinc ions. Titration microcalorimetry experiments using a soluble murine granulocyte colony-stimulating factor receptor construct show that SB-247464 binds to the extracellular domain of the receptor in a zinc ion-dependent manner. Analytical ultracentrifugation studies demonstrate that SB-247464 induces self-association of the N-terminal three-domain fragment in a manner that is consistent with dimerization. SB-247464 induces internalization of granulocyte colony-stimulating factor receptor on intact cells, consistent with a mechanism involving receptor oligomerization. These data show that small nonpeptidyl compounds are capable of selectively binding and inducing productive oligomerization of cytokine receptors.

MeSH terms

  • Animals
  • Benzimidazoles / pharmacology
  • Bone Marrow Cells / metabolism
  • Calorimetry
  • Cell Line
  • Circular Dichroism
  • Cytokines / metabolism
  • Dimerization
  • Dose-Response Relationship, Drug
  • Edetic Acid / pharmacology
  • Guanidines / pharmacology
  • Ions
  • Ligands
  • Mice
  • Models, Chemical
  • Peptides / chemistry
  • Protein Binding
  • Protein Structure, Tertiary
  • Receptors, Granulocyte Colony-Stimulating Factor / chemistry*
  • Receptors, Granulocyte Colony-Stimulating Factor / metabolism*
  • Recombinant Fusion Proteins / metabolism
  • Signal Transduction
  • Transfection
  • Ultracentrifugation
  • Zinc

Substances

  • Benzimidazoles
  • Cytokines
  • Guanidines
  • Ions
  • Ligands
  • Peptides
  • Receptors, Granulocyte Colony-Stimulating Factor
  • Recombinant Fusion Proteins
  • SB 247464
  • Edetic Acid
  • Zinc