High-Throughput Small Molecule Screens Reveal Therapeutic Opportunities Against Tfe3-Fusion Renal Cell Carcinoma

Background: Accounting for 1–5% of all renal cell carcinoma (RCC) cases, TFE3-fusion RCC comprise an aggressive subset of neoplasms typically diagnosed in early and middle adulthood, for which treatment options are currently scarce. We sought to describe the distinctive TFE3-fusion RCC transcriptomic landscape, to perform high-throughput drug screening and to validate potential therapeutic targets using a combination of in vitro and in vivo experimental approaches.