Bioorthogonal Probes for the Study of MDM2-p53 Inhibitors in Cells and Development of High-Content Screening Assays for Drug Discovery

Angew Chem Int Ed Engl. 2016 Dec 23;55(52):16026-16030. doi: 10.1002/anie.201608568. Epub 2016 Nov 22.

Abstract

To study the behavior of MDM2-p53 inhibitors in a disease-relevant cellular model, we have developed and validated a set of bioorthogonal probes that can be fluorescently labeled in cells and used in high-content screening assays. By using automated image analysis with single-cell resolution, we could visualize the intracellular target binding of compounds by co-localization and quantify target upregulation upon MDM2-p53 inhibition in an osteosarcoma model. Additionally, we developed a high-throughput assay to quantify target occupancy of non-tagged MDM2-p53 inhibitors by competition and to identify novel chemical matter. This approach could be expanded to other targets for lead discovery applications.

Keywords: bioorthogonal chemistry; drug discovery; fluorescent probes; protein-protein interactions; target engagement.

MeSH terms

  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology*
  • Biosensing Techniques
  • Cell Line, Tumor
  • Drug Evaluation, Preclinical / methods*
  • Fluorescent Dyes / analysis*
  • Fluorescent Dyes / chemistry
  • Humans
  • Indoles / chemistry
  • Indoles / pharmacology*
  • Models, Molecular
  • Molecular Structure
  • Osteosarcoma / drug therapy*
  • Osteosarcoma / pathology
  • Proto-Oncogene Proteins c-mdm2 / antagonists & inhibitors*
  • Single-Cell Analysis
  • Tumor Suppressor Protein p53 / antagonists & inhibitors*

Substances

  • Antineoplastic Agents
  • Fluorescent Dyes
  • Indoles
  • Tumor Suppressor Protein p53
  • MDM2 protein, human
  • Proto-Oncogene Proteins c-mdm2