High throughput screening identified a substituted imidazole as a novel RANK pathway-selective osteoclastogenesis inhibitor

Assay Drug Dev Technol. 2006 Aug;4(4):387-96. doi: 10.1089/adt.2006.4.387.

Abstract

Receptor activator of nuclear factor-kappaB (NF-kappaB) (RANK) plays a key role in the differentiation, activation, and survival of osteoclasts. Upon activation of RANK with RANK ligand (RANKL), osteoclast precursor cells differentiate into tartrate-resistant acid phosphatase (TRAP)-positive, multinucleated osteoclasts. To identify compounds that block osteoclastogenesis, a cell-based assay was developed using RAW264.7 cells stably transfected with a TRAP promoter-dependent reporter gene as a surrogate readout for differentiation. Described herein is the strategy for high throughput screening and subsequent secondary biological assays for hit triage, which resulted in the identification of compound 1, a 4-nitroimidazole derivative, that specifically inhibited RANKL-induced TRAP gene and protein expression. Compound 1 did not affect the tumor necrosis factor-alpha- or lipopolysaccharide-induced TRAP-luciferase response, suggesting selective inhibition of the RANKL-induced pathway. Reverse transcription polymerase chain reaction analysis confirmed the inhibition of expression of osteoclast marker genes, such as TRAP, cathepsin K, and carbonic anhydrase type II. Compound 1 did not inhibit the RANKL-induced activation of a NF-kappaB reporter gene, or p38 kinase activity, suggesting a mechanism of action downstream of NF-kappaB. Together, these results suggest that we have identified a RANK pathway-specific inhibitor able to block the RANKL-induced osteoclast differentiation process. The hit identification strategy described here can be applied to other cell-based assays using an indirect surrogate readout to improve success rates.

MeSH terms

  • Acid Phosphatase / genetics
  • Acid Phosphatase / metabolism
  • Animals
  • Cell Line
  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Dimethyl Sulfoxide / pharmacology
  • Dose-Response Relationship, Drug
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology
  • Gene Expression / drug effects
  • Humans
  • Imidazoles / chemistry
  • Imidazoles / pharmacology
  • Isoenzymes / genetics
  • Isoenzymes / metabolism
  • Lipopolysaccharides / pharmacology
  • Luciferases / genetics
  • Luciferases / metabolism
  • Nitroimidazoles / chemistry
  • Nitroimidazoles / pharmacology*
  • Osteoclasts / cytology
  • Osteoclasts / drug effects
  • Osteoclasts / metabolism
  • Osteoprotegerin / chemistry
  • Osteoprotegerin / pharmacology*
  • Pyridines / chemistry
  • Pyridines / pharmacology
  • RANK Ligand / genetics
  • RANK Ligand / metabolism*
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Reproducibility of Results
  • Reverse Transcriptase Polymerase Chain Reaction
  • Signal Transduction / drug effects*
  • Tartrate-Resistant Acid Phosphatase
  • Tumor Necrosis Factor-alpha / metabolism
  • Tumor Necrosis Factor-alpha / pharmacology

Substances

  • Enzyme Inhibitors
  • Imidazoles
  • Isoenzymes
  • Lipopolysaccharides
  • Nitroimidazoles
  • Osteoprotegerin
  • Pyridines
  • RANK Ligand
  • RNA, Messenger
  • Tumor Necrosis Factor-alpha
  • Luciferases
  • Acid Phosphatase
  • Tartrate-Resistant Acid Phosphatase
  • SB 203580
  • 4-nitroimidazole
  • Dimethyl Sulfoxide