Department of Cancer Biology, Dana-Farber Cancer Institute, Boston, MA, USA.
Department of Cell Biology, Harvard Medical School, Boston, MA, USA.
Nat Chem Biol. 2023 Jul;19(7):815-824. doi: 10.1038/s41589-023-01273-x. Epub 2023 Feb 23.
Creatine kinases (CKs) provide local ATP production in periods of elevated energetic demand, such as during rapid anabolism and growth. Thus, creatine energetics has emerged as a major metabolic liability in many rapidly proliferating cancers. Whether CKs can be targeted therapeutically is unknown because no potent or selective CK inhibitors have been developed. Here we leverage an active site cysteine present in all CK isoforms to develop a selective covalent inhibitor of creatine phosphagen energetics, CKi. Using deep chemoproteomics, we discover that CKi selectively engages the active site cysteine of CKs in cells. A co-crystal structure of CKi with creatine kinase B indicates active site inhibition that prevents bidirectional phosphotransfer. In cells, CKi and its analogs rapidly and selectively deplete creatine phosphate, and drive toxicity selectively in CK-dependent acute myeloid leukemia. Finally, we use CKi to uncover an essential role for CKs in the regulation of proinflammatory cytokine production in macrophages.
肌酸激酶(CKs)在能量需求增加的时期提供局部 ATP 产生,例如在快速合成代谢和生长期间。因此,肌酸代谢已成为许多快速增殖的癌症的主要代谢负担。由于尚未开发出有效的或选择性的 CK 抑制剂,因此 CK 是否可以作为治疗靶点尚不清楚。在这里,我们利用所有 CK 同工酶中存在的活性位点半胱氨酸来开发肌酸磷酸原能量的选择性共价抑制剂 CKi。通过深入的化学蛋白质组学研究,我们发现 CKi 可选择性地与细胞中 CK 的活性位点半胱氨酸结合。CKi 与肌酸激酶 B 的共晶结构表明,活性位点抑制阻止了双向磷酸转移。在细胞中,CKi 及其类似物可快速且选择性地耗尽肌酸磷酸,并选择性地驱动 CK 依赖性急性髓细胞性白血病的毒性。最后,我们使用 CKi 来揭示 CK 在调节巨噬细胞中前炎性细胞因子产生中的重要作用。