Olszewski Kellen, Barsotti Anthony, Feng Xiao-Jiang, Momcilovic Milica, Liu Kevin G, Kim Ji-In, Morris Koi, Lamarque Christophe, Gaffney Jack, Yu Xuemei, Patel Jeegar P, Rabinowitz Joshua D, Shackelford David B, Poyurovsky Masha V
Kadmon Corporation, LLC., New York, NY 10016, USA.
Division of Pulmonary and Critical Care Medicine, Department of Medicine, David Geffen School of Medicine at the University of California, Los Angeles, CA 90095, USA.
Cell Chem Biol. 2022 Mar 17;29(3):423-435.e10. doi: 10.1016/j.chembiol.2021.10.007. Epub 2021 Oct 28.
Efforts to target glucose metabolism in cancer have been limited by the poor potency and specificity of existing anti-glycolytic agents and a poor understanding of the glucose dependence of cancer subtypes in vivo. Here, we present an extensively characterized series of potent, orally bioavailable inhibitors of the class I glucose transporters (GLUTs). The representative compound KL-11743 specifically blocks glucose metabolism, triggering an acute collapse in NADH pools and a striking accumulation of aspartate, indicating a dramatic shift toward oxidative phosphorylation in the mitochondria. Disrupting mitochondrial metabolism via chemical inhibition of electron transport, deletion of the malate-aspartate shuttle component GOT1, or endogenous mutations in tricarboxylic acid cycle enzymes, causes synthetic lethality with KL-11743. Patient-derived xenograft models of succinate dehydrogenase A (SDHA)-deficient cancers are specifically sensitive to KL-11743, providing direct evidence that TCA cycle-mutant tumors are vulnerable to GLUT inhibitors in vivo.
针对癌症中葡萄糖代谢的研究工作一直受到现有抗糖酵解药物效力和特异性不佳以及对体内癌症亚型葡萄糖依赖性了解不足的限制。在此,我们展示了一系列经过广泛表征的强效口服生物可利用的I类葡萄糖转运蛋白(GLUTs)抑制剂。代表性化合物KL-11743特异性阻断葡萄糖代谢,引发NADH池的急性崩溃和天冬氨酸的显著积累,表明线粒体向氧化磷酸化发生了显著转变。通过化学抑制电子传递、缺失苹果酸-天冬氨酸穿梭成分GOT1或三羧酸循环酶的内源性突变来破坏线粒体代谢,会导致与KL-11743的合成致死性。琥珀酸脱氢酶A(SDHA)缺陷型癌症的患者来源异种移植模型对KL-11743特别敏感,这提供了直接证据,表明三羧酸循环突变型肿瘤在体内易受GLUT抑制剂的影响。