Eslam Roshdy, Eva Řezníčková, Mostafa M. Elbadawi, İsmail Çeli̇k, Simone Giovannuzzi, Denisa Veselá, Veronika Vojáčková, Petra Krňávková, Alessio Nocentini, Claudiu T. Supuran, Vladimír Kryštof, Wagdy M. Eldehna, Manabu Abe
High Resolution Image Download MS PowerPoint Slide Leukemia remains a challenging hematological malignancy, with limited therapeutic options. To address this unmet need, we report quinoline–sulfonamide hybrids as first-in-class dual inhibitors of platelet-derived growth factor receptor (PDGFR) and carbonic anhydrase (CA) IX/XII. Structure–activity relationship studies identified compound 9d as a potent lead, exhibiting strong inhibition of PDGFRA (IC 50 = 20 nM) and CA IX/XII ( K I = 93.3 and 80.0 nM, respectively), along with exceptional antiproliferative activity in FIP1L1–PDGFRA-driven EOL-1 cells (GI 50 = 2 nM), comparable to clinical agents. Mechanistic analyses revealed that 9d effectively abrogates PDGFRA signaling, induces G0/G1 cell-cycle arrest, and triggers apoptosis. Molecular docking and 200 ns molecular dynamics simulations supported stable dual binding of 9d within the ATP-binding pocket of PDGFR and the catalytic cleft of CA IX. By simultaneously targeting oncogenic PDGFRA signaling and hypoxia-driven pH regulation (CA IX/XII), 9d represents a promising lead for preclinical development in PDGFR/CA IX/XII-driven leukemias.