Repurposing MDM2 inhibitor RG7388 for TP53-mutant NSCLC: a p53-independent pyroptotic mechanism via ROS/p-p38/NOXA/caspase-3/GSDME axis

Abstract Non-small cell lung cancer (NSCLC) is highly malignant with limited treatment options, largely due to the inherent tumoral heterogeneity and acquired resistance towards chemotherapy and immunotherapy. RG7388, a known MDM2 inhibitor, exhibited anticancer activity in TP53-wild-type (TP53 WT)...

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Main Authors: Gaoyan Tang, Xuelei Cao, Jiaqi Chen, Fu Hui, Na Xu, Yiqing Jiang, Hongmin Lu, Haifeng Xiao, Xiuming Liang, Mingzhe Ma, Yu Qian, Dongli Liu, Zhenlu Wang, Shuzhen Liu, Guohua Yu, Lei Sun
Format: Article
Language:English
Published: Nature Publishing Group 2025-06-01
Series:Cell Death and Disease
Online Access:https://doi.org/10.1038/s41419-025-07770-2
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author Gaoyan Tang
Xuelei Cao
Jiaqi Chen
Fu Hui
Na Xu
Yiqing Jiang
Hongmin Lu
Haifeng Xiao
Xiuming Liang
Mingzhe Ma
Yu Qian
Dongli Liu
Zhenlu Wang
Shuzhen Liu
Guohua Yu
Lei Sun
author_facet Gaoyan Tang
Xuelei Cao
Jiaqi Chen
Fu Hui
Na Xu
Yiqing Jiang
Hongmin Lu
Haifeng Xiao
Xiuming Liang
Mingzhe Ma
Yu Qian
Dongli Liu
Zhenlu Wang
Shuzhen Liu
Guohua Yu
Lei Sun
author_sort Gaoyan Tang
collection DOAJ
description Abstract Non-small cell lung cancer (NSCLC) is highly malignant with limited treatment options, largely due to the inherent tumoral heterogeneity and acquired resistance towards chemotherapy and immunotherapy. RG7388, a known MDM2 inhibitor, exhibited anticancer activity in TP53-wild-type (TP53 WT) NSCLC by triggering the p53/PUMA axis-dependent apoptosis. However, our study uncovered previously unrecognized p53-independent anticancer effects of RG7388 in TP53-mutant (TP53 mutant) NSCLC, although the underlying mechanisms remained elusive. Here, we demonstrated that RG7388 specifically induced the NOXA/caspase-3 axis-dependent apoptosis and gasdermin E (GSDME)-mediated secondary pyroptosis in TP53 mutant NSCLC, as validated through in silico analyses and multiple biological assays. Mechanically, we identified reactive oxygen species (ROS) as the critical mediator in NOXA upregulation and p38 MAPK pathway activation in RG7388 treated TP53 mutant NSCLC. This was further supported by the use of ROS scavengers, N-acetylcysteine (NAC), and Ferrostatin-1 (Fer-1), which attenuated these effects. Pharmacologic inhibition of p38 MAPK signaling by SB203580 rescued RG7388-induced ROS-dependent NOXA accumulation and subsequent apoptosis and pyroptosis, highlighting the central role of the ROS/phosphorylated p38 MAPK (p-p38)/NOXA/caspase-3 axis in RG7388-induced TP53 mutant NSCLC cell death. Our findings revealed a novel mechanism for selectively targeting mutant p53-derived cancer through ROS/p-p38-mediated NOXA accumulation, offering potential therapeutic implications given the current lack of direct mutant p53 targeting strategies in cancer. Furthermore, immunohistochemical (IHC) analysis of an NSCLC tissue microarray confirmed a strong positive correlation between p-p38 and NOXA expression. Clinical data analysis further suggested that the p-p38/NOXA axis might be a potential prognostic biomarker for overall survival (OS) in NSCLC patients.
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spelling doaj-art-59153e594c1942d2b5d5dfcaa3aaa7ba2025-08-20T02:10:38ZengNature Publishing GroupCell Death and Disease2041-48892025-06-0116111710.1038/s41419-025-07770-2Repurposing MDM2 inhibitor RG7388 for TP53-mutant NSCLC: a p53-independent pyroptotic mechanism via ROS/p-p38/NOXA/caspase-3/GSDME axisGaoyan Tang0Xuelei Cao1Jiaqi Chen2Fu Hui3Na Xu4Yiqing Jiang5Hongmin Lu6Haifeng Xiao7Xiuming Liang8Mingzhe Ma9Yu Qian10Dongli Liu11Zhenlu Wang12Shuzhen Liu13Guohua Yu14Lei Sun15Department of Oncology, Weifang People’s Hospital, the First Affiliated Hospital of Shandong Second Medical UniversityDepartment of Clinical Laboratory, Qilu Hospital of Shandong UniversityCancer Prevention and Treatment Center, The Second Hospital of Shandong UniversityDepartment of Oncology, Weifang People’s Hospital, the First Affiliated Hospital of Shandong Second Medical UniversityDepartment of Oncology, Weifang People’s Hospital, the First Affiliated Hospital of Shandong Second Medical UniversityDepartment of Oncology, Weifang People’s Hospital, the First Affiliated Hospital of Shandong Second Medical UniversityDepartment of Oncology, Shanghai Renji Hospital, Shanghai Jiaotong University medical schoolDepartment of Oncology, Weifang People’s Hospital, the First Affiliated Hospital of Shandong Second Medical UniversityDivision for Biomolecular and Cellular Medicine, Department of Laboratory Medicine, Karolinska InstitutetDepartment of Gastric Surgery, Fudan University Shanghai Cancer CenterDepartment of thoracic head and neck medical oncology, University of Texas MD Anderson Cancer CenterDepartment of Radiation Oncology, Shanghai General Hospital, Shanghai Jiaotong University medical schoolDepartment of Oncology, Weifang People’s Hospital, the First Affiliated Hospital of Shandong Second Medical UniversityDepartment of Oncology, Weifang People’s Hospital, the First Affiliated Hospital of Shandong Second Medical UniversityDepartment of Oncology, Weifang People’s Hospital, the First Affiliated Hospital of Shandong Second Medical UniversityDepartment of Oncology, Weifang People’s Hospital, the First Affiliated Hospital of Shandong Second Medical UniversityAbstract Non-small cell lung cancer (NSCLC) is highly malignant with limited treatment options, largely due to the inherent tumoral heterogeneity and acquired resistance towards chemotherapy and immunotherapy. RG7388, a known MDM2 inhibitor, exhibited anticancer activity in TP53-wild-type (TP53 WT) NSCLC by triggering the p53/PUMA axis-dependent apoptosis. However, our study uncovered previously unrecognized p53-independent anticancer effects of RG7388 in TP53-mutant (TP53 mutant) NSCLC, although the underlying mechanisms remained elusive. Here, we demonstrated that RG7388 specifically induced the NOXA/caspase-3 axis-dependent apoptosis and gasdermin E (GSDME)-mediated secondary pyroptosis in TP53 mutant NSCLC, as validated through in silico analyses and multiple biological assays. Mechanically, we identified reactive oxygen species (ROS) as the critical mediator in NOXA upregulation and p38 MAPK pathway activation in RG7388 treated TP53 mutant NSCLC. This was further supported by the use of ROS scavengers, N-acetylcysteine (NAC), and Ferrostatin-1 (Fer-1), which attenuated these effects. Pharmacologic inhibition of p38 MAPK signaling by SB203580 rescued RG7388-induced ROS-dependent NOXA accumulation and subsequent apoptosis and pyroptosis, highlighting the central role of the ROS/phosphorylated p38 MAPK (p-p38)/NOXA/caspase-3 axis in RG7388-induced TP53 mutant NSCLC cell death. Our findings revealed a novel mechanism for selectively targeting mutant p53-derived cancer through ROS/p-p38-mediated NOXA accumulation, offering potential therapeutic implications given the current lack of direct mutant p53 targeting strategies in cancer. Furthermore, immunohistochemical (IHC) analysis of an NSCLC tissue microarray confirmed a strong positive correlation between p-p38 and NOXA expression. Clinical data analysis further suggested that the p-p38/NOXA axis might be a potential prognostic biomarker for overall survival (OS) in NSCLC patients.https://doi.org/10.1038/s41419-025-07770-2
spellingShingle Gaoyan Tang
Xuelei Cao
Jiaqi Chen
Fu Hui
Na Xu
Yiqing Jiang
Hongmin Lu
Haifeng Xiao
Xiuming Liang
Mingzhe Ma
Yu Qian
Dongli Liu
Zhenlu Wang
Shuzhen Liu
Guohua Yu
Lei Sun
Repurposing MDM2 inhibitor RG7388 for TP53-mutant NSCLC: a p53-independent pyroptotic mechanism via ROS/p-p38/NOXA/caspase-3/GSDME axis
Cell Death and Disease
title Repurposing MDM2 inhibitor RG7388 for TP53-mutant NSCLC: a p53-independent pyroptotic mechanism via ROS/p-p38/NOXA/caspase-3/GSDME axis
title_full Repurposing MDM2 inhibitor RG7388 for TP53-mutant NSCLC: a p53-independent pyroptotic mechanism via ROS/p-p38/NOXA/caspase-3/GSDME axis
title_fullStr Repurposing MDM2 inhibitor RG7388 for TP53-mutant NSCLC: a p53-independent pyroptotic mechanism via ROS/p-p38/NOXA/caspase-3/GSDME axis
title_full_unstemmed Repurposing MDM2 inhibitor RG7388 for TP53-mutant NSCLC: a p53-independent pyroptotic mechanism via ROS/p-p38/NOXA/caspase-3/GSDME axis
title_short Repurposing MDM2 inhibitor RG7388 for TP53-mutant NSCLC: a p53-independent pyroptotic mechanism via ROS/p-p38/NOXA/caspase-3/GSDME axis
title_sort repurposing mdm2 inhibitor rg7388 for tp53 mutant nsclc a p53 independent pyroptotic mechanism via ros p p38 noxa caspase 3 gsdme axis
url https://doi.org/10.1038/s41419-025-07770-2
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