Innovative PDK1‐Degrading PROTACs Transform Cancer Aerobic Glycolysis and Induce Immunogenic Cell Death in Breast Cancer

ABSTRACT Cancer cells are characterized by the Warburg effect, which hijacks glycolysis and hinders OXPHOS. Pyruvate dehydrogenase kinase 1 (PDK1) is a key modulator in the Warburg effect and is highly expressed in tumor cells. We utilize PROTAC technology to design compounds that could achieve long...

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Main Authors: Aohua Deng, Renming Fan, Jiakui Gou, Ruoxi Sang, Ruizhuo Lin, Ting Zhao, Junyan Zhuang, Yongrui Hai, Jialin Sun, Gaofei Wei
Format: Article
Language:English
Published: Wiley 2025-08-01
Series:Exploration
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Online Access:https://doi.org/10.1002/EXP.20240031
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author Aohua Deng
Renming Fan
Jiakui Gou
Ruoxi Sang
Ruizhuo Lin
Ting Zhao
Junyan Zhuang
Yongrui Hai
Jialin Sun
Gaofei Wei
author_facet Aohua Deng
Renming Fan
Jiakui Gou
Ruoxi Sang
Ruizhuo Lin
Ting Zhao
Junyan Zhuang
Yongrui Hai
Jialin Sun
Gaofei Wei
author_sort Aohua Deng
collection DOAJ
description ABSTRACT Cancer cells are characterized by the Warburg effect, which hijacks glycolysis and hinders OXPHOS. Pyruvate dehydrogenase kinase 1 (PDK1) is a key modulator in the Warburg effect and is highly expressed in tumor cells. We utilize PROTAC technology to design compounds that could achieve long‐lasting degradation on PDK1. After screening anti‐tumor activity in vitro, we selected a top compound A04, among 22 chemical candidates in various structures. Compared to a conventional PDK1 inhibitor, A04 dramatically improves over 1000‐fold proliferation inhibition efficacy. Besides, A04 reverses Warburg effect and causes tumor apoptosis. In vivo, A04 achieves potent therapeutic efficacy in tumor‐bearing mice and dramatically prolongs their lifetime after surgery resection. For the mechanism, A04 induces immunogenic cell death and reverses immunosuppression in the TME to enhance antitumor immunoreactivity. Further, transcriptome analysis verifies the mechanisms and uncovers fluctuation in cancer related pathways. Combination with αPD‐L1 improves therapeutic efficacy and promotes multiple immunocytes infiltration. In conclusion, we first utilize PROTAC technology on modulating aberrant expressed metabolic enzyme PDK1 in cancer cells and achieve a great pharmacological effect, rendering it promising for energy‐aberrant cancer therapy.
format Article
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institution Kabale University
issn 2766-8509
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publishDate 2025-08-01
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spelling doaj-art-e56154419f474a45bddbc98a63ec6bd32025-08-26T10:33:04ZengWileyExploration2766-85092766-20982025-08-0154n/an/a10.1002/EXP.20240031Innovative PDK1‐Degrading PROTACs Transform Cancer Aerobic Glycolysis and Induce Immunogenic Cell Death in Breast CancerAohua Deng0Renming Fan1Jiakui Gou2Ruoxi Sang3Ruizhuo Lin4Ting Zhao5Junyan Zhuang6Yongrui Hai7Jialin Sun8Gaofei Wei9Institute of Medical Research Northwestern Polytechnical University Xi'an Shaanxi ChinaInstitute of Medical Research Northwestern Polytechnical University Xi'an Shaanxi ChinaInstitute of Medical Research Northwestern Polytechnical University Xi'an Shaanxi ChinaInstitute of Medical Research Northwestern Polytechnical University Xi'an Shaanxi ChinaInstitute of Medical Research Northwestern Polytechnical University Xi'an Shaanxi ChinaInstitute of Medical Research Northwestern Polytechnical University Xi'an Shaanxi ChinaInstitute of Medical Research Northwestern Polytechnical University Xi'an Shaanxi ChinaInstitute of Medical Research Northwestern Polytechnical University Xi'an Shaanxi ChinaEdinburgh Kidney Research Group Centre for Cardiovascular Science Queen's Medical Research Institute University of Edinburgh Edinburgh UKInstitute of Medical Research Northwestern Polytechnical University Xi'an Shaanxi ChinaABSTRACT Cancer cells are characterized by the Warburg effect, which hijacks glycolysis and hinders OXPHOS. Pyruvate dehydrogenase kinase 1 (PDK1) is a key modulator in the Warburg effect and is highly expressed in tumor cells. We utilize PROTAC technology to design compounds that could achieve long‐lasting degradation on PDK1. After screening anti‐tumor activity in vitro, we selected a top compound A04, among 22 chemical candidates in various structures. Compared to a conventional PDK1 inhibitor, A04 dramatically improves over 1000‐fold proliferation inhibition efficacy. Besides, A04 reverses Warburg effect and causes tumor apoptosis. In vivo, A04 achieves potent therapeutic efficacy in tumor‐bearing mice and dramatically prolongs their lifetime after surgery resection. For the mechanism, A04 induces immunogenic cell death and reverses immunosuppression in the TME to enhance antitumor immunoreactivity. Further, transcriptome analysis verifies the mechanisms and uncovers fluctuation in cancer related pathways. Combination with αPD‐L1 improves therapeutic efficacy and promotes multiple immunocytes infiltration. In conclusion, we first utilize PROTAC technology on modulating aberrant expressed metabolic enzyme PDK1 in cancer cells and achieve a great pharmacological effect, rendering it promising for energy‐aberrant cancer therapy.https://doi.org/10.1002/EXP.20240031immunogenic cell deathPROTACpyruvate dehydrogenase kinaseWarburg effect
spellingShingle Aohua Deng
Renming Fan
Jiakui Gou
Ruoxi Sang
Ruizhuo Lin
Ting Zhao
Junyan Zhuang
Yongrui Hai
Jialin Sun
Gaofei Wei
Innovative PDK1‐Degrading PROTACs Transform Cancer Aerobic Glycolysis and Induce Immunogenic Cell Death in Breast Cancer
Exploration
immunogenic cell death
PROTAC
pyruvate dehydrogenase kinase
Warburg effect
title Innovative PDK1‐Degrading PROTACs Transform Cancer Aerobic Glycolysis and Induce Immunogenic Cell Death in Breast Cancer
title_full Innovative PDK1‐Degrading PROTACs Transform Cancer Aerobic Glycolysis and Induce Immunogenic Cell Death in Breast Cancer
title_fullStr Innovative PDK1‐Degrading PROTACs Transform Cancer Aerobic Glycolysis and Induce Immunogenic Cell Death in Breast Cancer
title_full_unstemmed Innovative PDK1‐Degrading PROTACs Transform Cancer Aerobic Glycolysis and Induce Immunogenic Cell Death in Breast Cancer
title_short Innovative PDK1‐Degrading PROTACs Transform Cancer Aerobic Glycolysis and Induce Immunogenic Cell Death in Breast Cancer
title_sort innovative pdk1 degrading protacs transform cancer aerobic glycolysis and induce immunogenic cell death in breast cancer
topic immunogenic cell death
PROTAC
pyruvate dehydrogenase kinase
Warburg effect
url https://doi.org/10.1002/EXP.20240031
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