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cc by nc-nd (c) Aragó, Marc et al., 2020
Si us plau utilitzeu sempre aquest identificador per citar o enllaçar aquest document: https://hdl.handle.net/2445/174403

Pharmacology and preclinical validation of a novel anticancer compound targeting PEPCK-M

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Background: Phosphoenolpyruvate carboxykinase (PEPCK) catalyzes the decarboxylation of oxaloacetate to phosphoenolpyruvate. The mitochondrial isozyme, PEPCK-M is highly expressed in cancer cells, where it plays a role in nutrient stress response. To date, pharmacological strategies to target this pathway have not been pursued. Methods: A compound embodying a 3-alkyl-1,8-dibenzylxanthine nucleus (iPEPCK-2), was synthesized and successfully probed in silico on a PEPCK-M structural model. Potency and target engagement in vitro and in vivo were evaluated by kinetic and cellular thermal shift assays (CETSA). The compound and its target were validated in tumor growth models in vitro and in murine xenografts. Results: Cross-inhibitory capacity and increased potency as compared to 3-MPA were confirmed in vitro and in vivo. Treatment with iPEPCK-2 inhibited cell growth and survival, especially in poor-nutrient environment, consistent with an impact on colony formation in soft agar. Finally, daily administration of the PEPCK-M inhibitor successfully inhibited tumor growth in two murine xenograft models as compared to vehicle, without weight loss, or any sign of apparent toxicity. Conclusion: We conclude that iPEPCK-2 is a compelling anticancer drug targeting PEPCK-M, a hallmark gene product involved in metabolic adaptations of the tumor.

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ARAGÓ, Marc, MORENO FELICI, Juan, ABÁS PRADES, Sònia, RODRÍGUEZ ARÉVALO, Sergio, HYROSSOVÁ, Petra, FIGUERAS, Agnes, VIÑALS CANALS, Francesc, PÉREZ, Belén, LOZA, María isabel, BREA, José, LATORRE, Pedro, CARRODEGUAS, Jose a., GARCÍA-ROVES, Pablo m. (pablo miguel), GALDEANO CANTADOR, Carlos, GINEX, Tiziana, LUQUE GARRIGA, F. xavier, ESCOLANO MIRÓN, Carmen, PERALES LOSA, Carlos. Pharmacology and preclinical validation of a novel anticancer compound targeting PEPCK-M. _Biomedicine & Pharmacotherapy_. 2020. Vol. 121, núm. 109601. [consulta: 24 de gener de 2026]. ISSN: 0753-3322. [Disponible a: https://hdl.handle.net/2445/174403]

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