Development of a 3D Tissue Culture-Based High-Content Screening Platform That Uses Phenotypic Profiling to Discriminate Selective Inhibitors of Receptor Tyrosine Kinases

3D tissue cultures provide a more physiologically relevant context for the screening of compounds, compared with 2D cell cultures. Cells cultured in 3D hydrogels also show complex phenotypes, increasing the scope for phenotypic profiling. Here we describe a high-content screening platform that uses invasive human prostate cancer cells cultured in 3D in standard 384-well assay plates to study the activity of potential therapeutic small molecules and antibody biologics. Image analysis tools were developed to process 3D image data to measure over 800 phenotypic parameters. Multiparametric analysis was used to evaluate the effect of compounds on tissue morphology. We applied this screening platform to measure the activity and selectivity of inhibitors of the c-Met and epidermal growth factor (EGF) receptor (EGFR) tyrosine kinases in 3D cultured prostate carcinoma cells. c-Met and EGFR activity was quantified based on the phenotypic profiles induced by their respective ligands, hepatocyte growth factor and EGF. The screening method was applied to a novel collection of 80 putative inhibitors of c-Met and EGFR. Compounds were identified that induced phenotypic profiles indicative of selective inhibition of c-Met, EGFR, or bispecific inhibition of both targets. In conclusion, we describe a fully scalable high-content screening platform that uses phenotypic profiling to discriminate selective and nonselective (off-target) inhibitors in a physiologically relevant 3D cell culture setting.

Journal of biomolecular screening. 2016 Jul 07 [Epub ahead of print]

Tijmen H Booij, Maarten J D Klop, Kuan Yan, Csaba Szántai-Kis, Balint Szokol, Laszlo Orfi, Bob van de Water, Gyorgy Keri, Leo S Price

Division of Toxicology, Leiden Academic Centre for Drug Research, Leiden University, Leiden, The Netherlands., OcellO B.V., Leiden, The Netherlands., OcellO B.V., Leiden, The Netherlands., Vichem Chemie Research Ltd., Budapest, Hungary., Vichem Chemie Research Ltd., Budapest, Hungary., Vichem Chemie Research Ltd., Budapest, Hungary Department of Pharmaceutical Chemistry, Semmelweis University, Budapest, Hungary., Division of Toxicology, Leiden Academic Centre for Drug Research, Leiden University, Leiden, The Netherlands., Vichem Chemie Research Ltd., Budapest, Hungary MTA-SE Pathobiochemistry Research Group, Department of Medical Chemistry, Semmelweis University, Budapest, Hungary., Division of Toxicology, Leiden Academic Centre for Drug Research, Leiden University, Leiden, The Netherlands OcellO B.V., Leiden, The Netherlands .