Drug Response Profiling - Leukemia: Empowering Precision Medicine in Under-represented European Regions

Precision medicine in hematologic malignancies remains unevenly accessible across Europe, especially in underserved regions where advanced molecular and functional diagnostics are lacking. A key limitation of current approaches is the difficulty in reliably translating genotype to phenotype—genomic profiling alone often fails to predict actual drug activity or clinical response. This project aims to address this challenge by expanding the application of ex vivo drug response profiling (DRP) in blood cancers—such as acute leukemias: AML, ALL. By establishing a collaborative network to collect patient samples from EU countries with limited access to high-throughput functional testing, we seek to complement molecular data with functional evidence of drug sensitivity, enabling more effective and equitable precision medicine strategies.

 Ex vivo DRP integrates cellular drug sensitivity with molecular phenotypes and can serve as a powerful complement to genomic profiling, particularly when no actionable mutations are found. Studies such as EXALT and SMARTrial have shown that DRP-based strategies can improve patient stratification and guide therapeutic decisions, even in highly refractory disease. However, a systematic application and validation of this approach across diverse geographic and clinical settings is still lacking.

 Our project will address this gap by collecting and analyzing samples from underrepresented EU regions, implementing DRP workflows (microscopy-based and ATP-based, pre-loaded tests), and correlating ex vivo drug sensitivity with clinical outcomes. We will also work to build local capacity and share protocols, enabling participating centers to develop functional diagnostic capabilities.