Harnessing artificial intelligence to identify Bufalin as a molecular glue degrader of estrogen receptor alpha

Abstract Target identification in natural products plays a critical role in the development of innovative drugs. Bufalin, a compound derived from traditional medicines, has shown promising anti-cancer activity; however, its precise molecular mechanism of action remains unclear. Here, we employ artif...

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Main Authors: Shilong Jiang, Keyi Liu, Ting Jiang, Hui Li, Xiao Wei, Xiaoya Wan, Changxin Zhong, Rong Gong, Zonglin Chen, Chan Zou, Qing Zhang, Yan Cheng, Dongsheng Cao
Format: Article
Language:English
Published: Nature Portfolio 2025-08-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-62288-7
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author Shilong Jiang
Keyi Liu
Ting Jiang
Hui Li
Xiao Wei
Xiaoya Wan
Changxin Zhong
Rong Gong
Zonglin Chen
Chan Zou
Qing Zhang
Yan Cheng
Dongsheng Cao
author_facet Shilong Jiang
Keyi Liu
Ting Jiang
Hui Li
Xiao Wei
Xiaoya Wan
Changxin Zhong
Rong Gong
Zonglin Chen
Chan Zou
Qing Zhang
Yan Cheng
Dongsheng Cao
author_sort Shilong Jiang
collection DOAJ
description Abstract Target identification in natural products plays a critical role in the development of innovative drugs. Bufalin, a compound derived from traditional medicines, has shown promising anti-cancer activity; however, its precise molecular mechanism of action remains unclear. Here, we employ artificial intelligence, molecular docking, and molecular dynamics simulations to elucidate the molecular mechanism of Bufalin. Using an integrated multi-predictive strategy, we identify CYP17A1, ESR1, mTOR, AR, and PRKCD as the potential targets of Bufalin. Subsequent validation via surface plasmon resonance, biotin pulldown, and thermal shift assays confirms Bufalin’s direct binding to ESR1, which encodes estrogen receptor alpha (ERα). Molecular docking analyses pinpoint Bufalin’s selective interaction with Arg394 on ERα. Molecular dynamic simulations further show that Bufalin acts as a molecular glue, enhancing the interaction between ERα and the E3 ligase STUB1, thereby promoting proteasomal degradation of ERα. Given the therapeutic potential of ERα degradation in overcoming endocrine resistance, we investigate the inhibitory effect of Bufalin on endocrine-resistant models and prove Bufalin reverses Tamoxifen resistance in vitro, in vivo, and in patient-derived breast cancer organoids from tamoxifen-relapsed cases. Collectively, our findings indicate that Bufalin functions as a molecular glue to degrade ERα, offering a potential therapeutic strategy for reversing Tamoxifen resistance.
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spelling doaj-art-64b59f227c774ab0a93aaf4c939e637a2025-08-24T11:39:51ZengNature PortfolioNature Communications2041-17232025-08-0116111910.1038/s41467-025-62288-7Harnessing artificial intelligence to identify Bufalin as a molecular glue degrader of estrogen receptor alphaShilong Jiang0Keyi Liu1Ting Jiang2Hui Li3Xiao Wei4Xiaoya Wan5Changxin Zhong6Rong Gong7Zonglin Chen8Chan Zou9Qing Zhang10Yan Cheng11Dongsheng Cao12Department of Pharmacy, Xiangya Hospital, Central South UniversityDepartment of Cardiology, West China Hospital, Sichuan UniversityDepartment of Pharmacy, The Second Xiangya Hospital, Central South UniversityXiangya School of Pharmaceutical Sciences, Central South UniversityXiangya School of Pharmaceutical Sciences, Central South UniversityDepartment of Pharmacy, The Second Xiangya Hospital, Central South UniversityDepartment of Pharmacy, The Second Xiangya Hospital, Central South UniversityDepartment of Pharmacy, The Second Xiangya Hospital, Central South UniversityDepartment of Pharmacy, The Second Xiangya Hospital, Central South UniversityCenter for Clinical Pharmacology, the Third Xiangya Hospital, Central South UniversityDepartment of Cardiology, West China Hospital, Sichuan UniversityDepartment of Pharmacy, The Second Xiangya Hospital, Central South UniversityXiangya School of Pharmaceutical Sciences, Central South UniversityAbstract Target identification in natural products plays a critical role in the development of innovative drugs. Bufalin, a compound derived from traditional medicines, has shown promising anti-cancer activity; however, its precise molecular mechanism of action remains unclear. Here, we employ artificial intelligence, molecular docking, and molecular dynamics simulations to elucidate the molecular mechanism of Bufalin. Using an integrated multi-predictive strategy, we identify CYP17A1, ESR1, mTOR, AR, and PRKCD as the potential targets of Bufalin. Subsequent validation via surface plasmon resonance, biotin pulldown, and thermal shift assays confirms Bufalin’s direct binding to ESR1, which encodes estrogen receptor alpha (ERα). Molecular docking analyses pinpoint Bufalin’s selective interaction with Arg394 on ERα. Molecular dynamic simulations further show that Bufalin acts as a molecular glue, enhancing the interaction between ERα and the E3 ligase STUB1, thereby promoting proteasomal degradation of ERα. Given the therapeutic potential of ERα degradation in overcoming endocrine resistance, we investigate the inhibitory effect of Bufalin on endocrine-resistant models and prove Bufalin reverses Tamoxifen resistance in vitro, in vivo, and in patient-derived breast cancer organoids from tamoxifen-relapsed cases. Collectively, our findings indicate that Bufalin functions as a molecular glue to degrade ERα, offering a potential therapeutic strategy for reversing Tamoxifen resistance.https://doi.org/10.1038/s41467-025-62288-7
spellingShingle Shilong Jiang
Keyi Liu
Ting Jiang
Hui Li
Xiao Wei
Xiaoya Wan
Changxin Zhong
Rong Gong
Zonglin Chen
Chan Zou
Qing Zhang
Yan Cheng
Dongsheng Cao
Harnessing artificial intelligence to identify Bufalin as a molecular glue degrader of estrogen receptor alpha
Nature Communications
title Harnessing artificial intelligence to identify Bufalin as a molecular glue degrader of estrogen receptor alpha
title_full Harnessing artificial intelligence to identify Bufalin as a molecular glue degrader of estrogen receptor alpha
title_fullStr Harnessing artificial intelligence to identify Bufalin as a molecular glue degrader of estrogen receptor alpha
title_full_unstemmed Harnessing artificial intelligence to identify Bufalin as a molecular glue degrader of estrogen receptor alpha
title_short Harnessing artificial intelligence to identify Bufalin as a molecular glue degrader of estrogen receptor alpha
title_sort harnessing artificial intelligence to identify bufalin as a molecular glue degrader of estrogen receptor alpha
url https://doi.org/10.1038/s41467-025-62288-7
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