Targeting PRDX1 impairs acute myeloid leukemic blasts and stem cells by disrupting redox homeostasis

Abstract Acute myeloid leukemia (AML) is an aggressive hematologic malignancy with a poor prognosis and limited therapeutic options. Leukemic stem cells (LSCs), which drive disease progression and confer resistance to therapy, pose a significant challenge to conventional treatment strategies. In thi...

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Main Authors: Zhenghao Li, Guangci Liu, Ziren Chen, Keming Li, Zhe Yu, Chao He, Xinyu Ying, Danling Huang, Chengtian Tao, Sajid Khan, Yimeng Wang, Fang-Lin Zhang, Huan Li, Yun Chen, Jingfeng Zhou, Li Yu, Thomas J. Kipps, Yongxian Cheng, Suping Zhang
Format: Article
Language:English
Published: Nature Publishing Group 2025-08-01
Series:Cell Death and Disease
Online Access:https://doi.org/10.1038/s41419-025-07831-6
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author Zhenghao Li
Guangci Liu
Ziren Chen
Keming Li
Zhe Yu
Chao He
Xinyu Ying
Danling Huang
Chengtian Tao
Sajid Khan
Yimeng Wang
Fang-Lin Zhang
Huan Li
Yun Chen
Jingfeng Zhou
Li Yu
Thomas J. Kipps
Yongxian Cheng
Suping Zhang
author_facet Zhenghao Li
Guangci Liu
Ziren Chen
Keming Li
Zhe Yu
Chao He
Xinyu Ying
Danling Huang
Chengtian Tao
Sajid Khan
Yimeng Wang
Fang-Lin Zhang
Huan Li
Yun Chen
Jingfeng Zhou
Li Yu
Thomas J. Kipps
Yongxian Cheng
Suping Zhang
author_sort Zhenghao Li
collection DOAJ
description Abstract Acute myeloid leukemia (AML) is an aggressive hematologic malignancy with a poor prognosis and limited therapeutic options. Leukemic stem cells (LSCs), which drive disease progression and confer resistance to therapy, pose a significant challenge to conventional treatment strategies. In this study, we identified and characterized the inhibitory mechanisms of TH37, a small molecule derived from traditional Chinese medicine, which selectively targets AML blasts and LSCs. Our analyses identified peroxiredoxin 1 (PRDX1), an enzyme that catalyzes the breakdown of hydrogen peroxide (a reactive oxygen species), as the primary molecular target of TH37. We demonstrated that TH37 directly interacts with PRDX1, inhibiting its enzymatic activity and thereby elevating intracellular reactive oxygen species levels in AML cells. PRDX1 was found to be overexpressed in AML, and its expression correlated with poor prognosis and the activation of AML- and cancer-associated pathways. Targeting PRDX1, either through lentiviral short-hairpin RNA-mediated silencing or TH37 treatment, induced apoptosis, reduced colony formation, and impaired the engraftment and growth of AML cells in immunodeficient mouse models. Furthermore, TH37 synergized with conventional chemotherapeutic agent to significantly reduce the viability and colony-forming capacity of AML cells. These findings demonstrate the critical role of PRDX1 in AML pathogenesis and highlight its potential as a key therapeutic target to improve clinical outcomes for AML patients.
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spelling doaj-art-1583dc14361d496bacb072cc7cc492dd2025-08-24T11:54:22ZengNature Publishing GroupCell Death and Disease2041-48892025-08-0116111210.1038/s41419-025-07831-6Targeting PRDX1 impairs acute myeloid leukemic blasts and stem cells by disrupting redox homeostasisZhenghao Li0Guangci Liu1Ziren Chen2Keming Li3Zhe Yu4Chao He5Xinyu Ying6Danling Huang7Chengtian Tao8Sajid Khan9Yimeng Wang10Fang-Lin Zhang11Huan Li12Yun Chen13Jingfeng Zhou14Li Yu15Thomas J. Kipps16Yongxian Cheng17Suping Zhang18Shenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityDepartment of Hematology, Shenzhen University General HospitalShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityDepartment of Hematology, Shandong Provincial Third Hospital, Shandong UniversityCentral Laboratory, Jiangsu University Affiliated Fourth HospitalDepartment of Clinical Laboratory, Ningbo Medical Centre Lihuili Hospital, Affiliated Hospital of Ningbo UniversityShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversitySchool of Chemistry, Chemistry Engineering and Life Sciences, Wuhan University of TechnologyShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityDepartment of Immunology, Nanjing Medical UniversityShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityDepartment of Hematology, Shenzhen University General HospitalMoores Cancer Center, University of California San DiegoShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityShenzhen University International Cancer Center, Shenzhen Key Laboratory of Precision Medicine for Hematological Malignancies, Guangdong Key Laboratory for Genome Stability and Human Disease Prevention, Guangdong Provincial Key Laboratory of Chinese Medicine Ingredients and Gut Microbiomics, Marshall Laboratory of Biomedical Engineering, Institute for Inheritance-Based Innovation of Chinese Medicine, Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, School of Basic Medical Sciences, School of Pharmacy, Shenzhen University Medical School, Shenzhen UniversityAbstract Acute myeloid leukemia (AML) is an aggressive hematologic malignancy with a poor prognosis and limited therapeutic options. Leukemic stem cells (LSCs), which drive disease progression and confer resistance to therapy, pose a significant challenge to conventional treatment strategies. In this study, we identified and characterized the inhibitory mechanisms of TH37, a small molecule derived from traditional Chinese medicine, which selectively targets AML blasts and LSCs. Our analyses identified peroxiredoxin 1 (PRDX1), an enzyme that catalyzes the breakdown of hydrogen peroxide (a reactive oxygen species), as the primary molecular target of TH37. We demonstrated that TH37 directly interacts with PRDX1, inhibiting its enzymatic activity and thereby elevating intracellular reactive oxygen species levels in AML cells. PRDX1 was found to be overexpressed in AML, and its expression correlated with poor prognosis and the activation of AML- and cancer-associated pathways. Targeting PRDX1, either through lentiviral short-hairpin RNA-mediated silencing or TH37 treatment, induced apoptosis, reduced colony formation, and impaired the engraftment and growth of AML cells in immunodeficient mouse models. Furthermore, TH37 synergized with conventional chemotherapeutic agent to significantly reduce the viability and colony-forming capacity of AML cells. These findings demonstrate the critical role of PRDX1 in AML pathogenesis and highlight its potential as a key therapeutic target to improve clinical outcomes for AML patients.https://doi.org/10.1038/s41419-025-07831-6
spellingShingle Zhenghao Li
Guangci Liu
Ziren Chen
Keming Li
Zhe Yu
Chao He
Xinyu Ying
Danling Huang
Chengtian Tao
Sajid Khan
Yimeng Wang
Fang-Lin Zhang
Huan Li
Yun Chen
Jingfeng Zhou
Li Yu
Thomas J. Kipps
Yongxian Cheng
Suping Zhang
Targeting PRDX1 impairs acute myeloid leukemic blasts and stem cells by disrupting redox homeostasis
Cell Death and Disease
title Targeting PRDX1 impairs acute myeloid leukemic blasts and stem cells by disrupting redox homeostasis
title_full Targeting PRDX1 impairs acute myeloid leukemic blasts and stem cells by disrupting redox homeostasis
title_fullStr Targeting PRDX1 impairs acute myeloid leukemic blasts and stem cells by disrupting redox homeostasis
title_full_unstemmed Targeting PRDX1 impairs acute myeloid leukemic blasts and stem cells by disrupting redox homeostasis
title_short Targeting PRDX1 impairs acute myeloid leukemic blasts and stem cells by disrupting redox homeostasis
title_sort targeting prdx1 impairs acute myeloid leukemic blasts and stem cells by disrupting redox homeostasis
url https://doi.org/10.1038/s41419-025-07831-6
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