Article

Inhibiting the Otub1/phosphorylated STAT3 axis for the treatment of non-small cell lung cancer

Zi-yang Liu1, Ya-wen Zhang2, Hai-xia Zhuang1, Yu-jie Ou1, Qiu-yun Jiang1, Ping-fei Li1, Yuan-ming He1, Ying Ren2,3, Xin-liang Mao1
1 The Key Laboratory of Advanced Interdisciplinary Studies, The First Affiliated Hospital of Guangzhou Medical University & Guangdong Provincial Key Laboratory of Protein Modification and Degradation, School of Basic Medical Sciences, Guangzhou Medical University, Guangzhou 511436, China
2 Department of Pharmacology, College of Pharmaceutical Sciences, Soochow University, Suzhou 215123, China
3 Jiangsu Key Laboratory of New Drug Research and Clinical Pharmacy, Xuzhou Medical University, 209 Tongshan Road, Xuzhou 221004, China
Correspondence to: Xin-liang Mao: xinliangmao@gzhmu.edu.cn,
DOI: 10.1038/s41401-024-01366-w
Received: 28 January 2024
Accepted: 22 July 2024
Advance online: 28 August 2024

Abstract

The transcription factor STAT3 is a promising target for the treatment of non-small cell lung cancer (NSCLC). STAT3 activity is mainly dependent on phosphorylation at tyrosine 705 (pSTAT3-Y705), but the modulation on pSTAT3-Y705 is elusive. By screening a library of deubiquitinases (Dubs), we found that the Otub1 increases STAT3 transcriptional activity. As a Dub, Otub1 binds to pSTAT3-Y705 and specifically abolishes its K48-linked ubiquitination, therefore preventing its degradation and promoting NSCLC cell survival. The Otub1/pSTAT3-Y705 axis could be a potential target for the treatment of NSCLC. To explore this concept, we screen libraries of FDA-approved drugs and natural products based on STAT3-recognition element-driven luciferase assay, from which crizotinib is found to block pSTAT3-Y705 deubiquitination and promotes its degradation. Different from its known action to induce ALK positive NSCLC cell apoptosis, crizotinib suppresses ALK-intact NSCLC cell proliferation and colony formation but not apoptosis. Furthermore, crizotinib also suppresses NSCLC xenograft growth in mice. Taken together, these findings identify Otub1 as the first deubiquitinase of pSTAT3-Y705 and provide that the Otub1/pSTAT3-Y705 axis is a promising target for the treatment of NSCLC.
Keywords: Otub1; STAT3; non-small cell lung cancer; Crizotinib

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