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A Covalent PFKL Activator Suppresses Tumor Growth

Jiang, X.; Lynch, E. M.; Lyu, C.; Wilson, C.; Salay, L. E.; Lyons, S.; Lu, M.-J.; Luo, S.; Kim, G.; Chan, H.-R.; Wolfe, W.; Lin, Y.-C.; Kollman, J. M.; Cambronne, X. A.; Hsu, K.-L.

2025-10-03 biochemistry
10.1101/2025.10.02.680014 bioRxiv
Show abstract

Glycolysis fuels vital cellular functions and its dysregulation is implicated in cancer, neurodegeneration, antibiotic resistance and diabetes. The glycolytic dependency of cancer, known as the Warburg effect, presents a key vulnerability for developing targeted anticancer agents but remains challenging due to metabolic heterogeneity and resistance. Here, we developed a first-in-class covalent phosphofructokinase-1 liver type (PFKL) activator that induces metabolic imbalance coupled to delivery of a cytotoxic payload to cancer cells in vitro and in vivo. The electrophile-drug conjugate (EDC) site-specifically and proteome-wide selectively modifies K677 in the allosteric effector site to stabilize the R-state tetramer of PFKL and destabilize cell metabolism. We introduce EDCs as a new delivery mechanism analogous to antibody-drug conjugates but differentiated by selective covalent targeting of intracellular proteins.

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