Article

Downregulation of c-Myc expression confers sensitivity to CHK1 inhibitors in hematologic malignancies

Kai-long Jiang1,2, Le-xian Tong3, Tao Wang4, Han-lin Wang1,2,5, Xiao-bei Hu1,6, Gao-ya Xu1, Ting-ting Jin3, Wei-juan Kan1, Lei Xu1,2,6, Jia-nan Li1,7, Kai-xiang Zhang1,2, Ning Song1,2,5, Jie-yu Liu1,2, Meng-meng Zhang1, Wen-biao Wu1,2, Yu-qi Xiang1,2,5, An-hui Gao1, Yong-zhou Hu3, Yu-bo Zhou1,2,6, Tao Liu3, Jian-min Yang4, Jia Li1,2,5,6
1 National Center for Drug Screening, State Key Laboratory of Drug Research, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai 201203, China
2 University of Chinese Academy of Sciences, Beijing 100049, China
3 ZJU-ENS Joint Laboratory of Medicinal Chemistry, Zhejiang Province Key Laboratory of Anti-Cancer Drug Research, College of Pharmaceutical Sciences, Zhejiang University, Hangzhou 310058, China
4 Changhai Hospital, Naval Medical University, Shanghai 200433, China
5 Shanghai Tech University, Shanghai 201210, China
6 Zhongshan Institute for Drug Discovery, Institutes of Drug Discovery and Development, Chinese Academy of Sciences, Zhongshan 528400, China
7 College of Pharmacy, Nanjing University of Chinese Medicine, Nanjing 210033, China
Correspondence to: Yu-bo Zhou: ybzhou@simm.ac.cn, Tao Liu: lt601@zju.edu.cn, Jian-min Yang: chyangjianmin@163.com, Jia Li: jli@simm.ac.cn,
DOI: 10.1038/s41401-021-00652-1
Received: 26 December 2020
Accepted: 12 March 2021
Advance online: 29 March 2021

Abstract

Checkpoint kinase 1 inhibitors (CHK1i) have shown impressive single-agent efficacy in treatment of certain tumors, as monotherapy or potentiators of chemotherapy in clinical trials, but the sensitive tumor types and downstream effectors to dictate the therapeutic responses to CHK1i remains unclear. In this study we first analyzed GDSC (Genomics of Drug Sensitivity in Cancer) and DepMap database and disclosed that hematologic malignancies (HMs) were relatively sensitive to CHK1i or CHK1 knockdown. This notion was confirmed by examining PY34, a new and potent in-house selective CHK1i, which exhibited potent anti-HM effect in vitro and in vivo, as single agent. We demonstrated that the downregulation of c-Myc and its signaling pathway was the common transcriptomic profiling response of sensitive HM cell lines to PY34, whereas overexpressing c-Myc could partially rescue the anticancer effect of PY34. Strikingly, we revealed the significant correlations between downregulation of c-Myc and cell sensitivity to PY34 in 17 HM cell lines and 39 patient-derived cell (PDC) samples. Thus, our results demonstrate that HMs are more sensitive to CHK1i than solid tumors, and c-Myc downregulation could represent the CHK1i efficacy in HMs.
Keywords: CHK1 inhibitors; PY34; drug sensitivity; c-Myc; hematologic malignancies

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