Oncotarget

Research Papers:

A novel drug discovery strategy: Mechanistic investigation of an enantiomeric antitumor agent targeting dual p53 and NF-κB pathways

Chunlin Zhuang _, Chunquan Sheng, Woo Shik Shin, Yuelin Wu, Jin Li, Jianzhong Yao, Guoqiang Dong, Wen Zhang, Yuk Yin Sham, Zhenyuan Miao and Wannian Zhang

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Oncotarget. 2014; 5:10830-10839. https://doi.org/10.18632/oncotarget.2521

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Abstract

Chunlin Zhuang1,2,*, Chunquan Sheng1,*, Woo Shik Shin3, Yuelin Wu1, Jin Li1, Jianzhong Yao1, Guoqiang Dong1, Wen Zhang2, Yuk Yin Sham3, Zhenyuan Miao1, Wannian Zhang1

1 Department of Medicinal Chemistry, School of Pharmacy, Second Military Medical University, Shanghai, 200433, People’s Republic of China

2 Research Center for Marine Drugs, School of Pharmacy, Second Military Medical University, Shanghai, 200433, People’s Republic of China

3 Center for Drug Design, Academic Health Center, University of Minnesota, Minneapolis, 55455, Minnesota

*These authors contributed equally to this work

Correspondence to:

Zhenyuan Miao, e-mail: [email protected]

Wannian Zhang, e-mail: [email protected]

Yuk Yin Sham, e-mail: [email protected]

Keywords: p53-MDM2, NF-κB, antitumor activity, dual inhibitors, enantiomer, molecular dynamics, molecular recognition

Received: June 30, 2014     Accepted: September 24, 2014     Published: October 07, 2014

ABSTRACT

The p53 and nuclear factor κB (NF-κB) pathways play crucial roles in human cancer development. Simultaneous targeting of both pathways is an attractive therapeutic strategy against cancer. In this study, we report an antitumor molecule that bears a pyrrolo[3,4-c]pyrazole scaffold and functions as an enantiomeric inhibitor against both the p53-MDM2 interaction and the NF-κB activation. It is a first-in-class enantiomeric inhibitor with dual efficacy for cancer therapy. Synergistic effect was observed in vitro and in vivo. Docking and molecular dynamics simulation studies further provided insights into the nature of stereoselectivity.


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