Neddylation status determines the therapeutic sensitivity of tyrosine kinase inhibitors in chronic myeloid leukemia

Abstract BCR::ABL1-targeting tyrosine kinase inhibitors (TKIs) dominate the treatment of chronic myeloid leukemia (CML) over the past decades. In this study, we reported an unexpected role of neddylation inhibitors in desensitizing the therapeutic efficacy of BCR::ABL1-targeting TKIs in CML. Unlike...

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Main Authors: Congyi Zhang, Yikai Yao, Qiuting Qian, Xiongyu Han, Yunkun Lu, Xinyi Jiang, Hongqiang Cheng, Xue Zhang, Ying Chi, Yuehai Ke, Peng Xiao
Format: Article
Language:English
Published: Nature Portfolio 2025-05-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-04153-7
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author Congyi Zhang
Yikai Yao
Qiuting Qian
Xiongyu Han
Yunkun Lu
Xinyi Jiang
Hongqiang Cheng
Xue Zhang
Ying Chi
Yuehai Ke
Peng Xiao
author_facet Congyi Zhang
Yikai Yao
Qiuting Qian
Xiongyu Han
Yunkun Lu
Xinyi Jiang
Hongqiang Cheng
Xue Zhang
Ying Chi
Yuehai Ke
Peng Xiao
author_sort Congyi Zhang
collection DOAJ
description Abstract BCR::ABL1-targeting tyrosine kinase inhibitors (TKIs) dominate the treatment of chronic myeloid leukemia (CML) over the past decades. In this study, we reported an unexpected role of neddylation inhibitors in desensitizing the therapeutic efficacy of BCR::ABL1-targeting TKIs in CML. Unlike their function in reducing drug resistance in many solid tumors, we revealed that neddylation inhibitors counteracted the cytotoxicity of TKIs against CML cells, both in cellular experiments and in animal model. Conversely, neddylation agonist sensitized the function of TKIs. RNA sequencing data revealed that neddylation inhibitor reversed the transcriptomic changes induced by TKI. Co-immunoprecipitation (co-IP) assay identified ABL1 kinase domain as a novel substrate for neddylation. Furthermore, an artificial intelligence (AI) 3-Dimensional spatial structure binding technology was employed to predict the impact of neddylation on the structure of ABL1 kinase domain. Finally, we provided potential evidence showing that TKI therapy decreased the expression of neddylation enzymes in the bone marrow of CML patients. Hence, our study offers new insights into the post-translational modification (PTM)-mediated drug resistance, and highlights the potential clinical benefits of neddylation agonists in improving the responsiveness of BCR::ABL1 TKIs in CML.
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spelling doaj-art-4bbac47e00b84809a6b736085b9dd5002025-08-20T02:03:31ZengNature PortfolioScientific Reports2045-23222025-05-0115111010.1038/s41598-025-04153-7Neddylation status determines the therapeutic sensitivity of tyrosine kinase inhibitors in chronic myeloid leukemiaCongyi Zhang0Yikai Yao1Qiuting Qian2Xiongyu Han3Yunkun Lu4Xinyi Jiang5Hongqiang Cheng6Xue Zhang7Ying Chi8Yuehai Ke9Peng Xiao10Department of Pathology and Pathophysiology, and Department of Respiratory Medicine at Sir Run Run Shaw Hospital, Zhejiang University School of MedicineDepartment of Pathology and Pathophysiology, and Department of Respiratory Medicine at Sir Run Run Shaw Hospital, Zhejiang University School of MedicineZhejiang University-University of Edinburgh Joint Institute, ZJU Haining International CampusZhejiang University-University of Edinburgh Joint Institute, ZJU Haining International CampusSir Run Run Shaw Hospital, Zhejiang University School of MedicineDepartment of Pathology and Pathophysiology, and Department of Respiratory Medicine at Sir Run Run Shaw Hospital, Zhejiang University School of MedicineDepartment of Pathology and Pathophysiology, and Department of Respiratory Medicine at Sir Run Run Shaw Hospital, Zhejiang University School of MedicineDepartment of Pathology and Pathophysiology, and Department of Respiratory Medicine at Sir Run Run Shaw Hospital, Zhejiang University School of MedicineZhejiang University-University of Edinburgh Joint Institute, ZJU Haining International CampusDepartment of Pathology and Pathophysiology, and Department of Respiratory Medicine at Sir Run Run Shaw Hospital, Zhejiang University School of MedicineSir Run Run Shaw Hospital, Zhejiang University School of MedicineAbstract BCR::ABL1-targeting tyrosine kinase inhibitors (TKIs) dominate the treatment of chronic myeloid leukemia (CML) over the past decades. In this study, we reported an unexpected role of neddylation inhibitors in desensitizing the therapeutic efficacy of BCR::ABL1-targeting TKIs in CML. Unlike their function in reducing drug resistance in many solid tumors, we revealed that neddylation inhibitors counteracted the cytotoxicity of TKIs against CML cells, both in cellular experiments and in animal model. Conversely, neddylation agonist sensitized the function of TKIs. RNA sequencing data revealed that neddylation inhibitor reversed the transcriptomic changes induced by TKI. Co-immunoprecipitation (co-IP) assay identified ABL1 kinase domain as a novel substrate for neddylation. Furthermore, an artificial intelligence (AI) 3-Dimensional spatial structure binding technology was employed to predict the impact of neddylation on the structure of ABL1 kinase domain. Finally, we provided potential evidence showing that TKI therapy decreased the expression of neddylation enzymes in the bone marrow of CML patients. Hence, our study offers new insights into the post-translational modification (PTM)-mediated drug resistance, and highlights the potential clinical benefits of neddylation agonists in improving the responsiveness of BCR::ABL1 TKIs in CML.https://doi.org/10.1038/s41598-025-04153-7Chronic myeloid leukemiaTyrosine kinase inhibitorNeddylationPost-translational modificationResistance
spellingShingle Congyi Zhang
Yikai Yao
Qiuting Qian
Xiongyu Han
Yunkun Lu
Xinyi Jiang
Hongqiang Cheng
Xue Zhang
Ying Chi
Yuehai Ke
Peng Xiao
Neddylation status determines the therapeutic sensitivity of tyrosine kinase inhibitors in chronic myeloid leukemia
Scientific Reports
Chronic myeloid leukemia
Tyrosine kinase inhibitor
Neddylation
Post-translational modification
Resistance
title Neddylation status determines the therapeutic sensitivity of tyrosine kinase inhibitors in chronic myeloid leukemia
title_full Neddylation status determines the therapeutic sensitivity of tyrosine kinase inhibitors in chronic myeloid leukemia
title_fullStr Neddylation status determines the therapeutic sensitivity of tyrosine kinase inhibitors in chronic myeloid leukemia
title_full_unstemmed Neddylation status determines the therapeutic sensitivity of tyrosine kinase inhibitors in chronic myeloid leukemia
title_short Neddylation status determines the therapeutic sensitivity of tyrosine kinase inhibitors in chronic myeloid leukemia
title_sort neddylation status determines the therapeutic sensitivity of tyrosine kinase inhibitors in chronic myeloid leukemia
topic Chronic myeloid leukemia
Tyrosine kinase inhibitor
Neddylation
Post-translational modification
Resistance
url https://doi.org/10.1038/s41598-025-04153-7
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