Potent β-lactam-based tyrosyl-DNA phosphodiesterase 1 inhibitors identified by a virtual screen

Abstract Tyrosyl-DNA phosphodiesterase 1 (TDP1) is an important therapeutic target. We recently reported several crystal structures of TDP1 with small molecules bound within the catalytic pocket. These molecules bind by forming hydrogen bonds with residues of the catalytic HKN motifs. Guided by thes...

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Main Authors: Xue Zhi Zhao, Wenjie Wang, Kiall F. Suazo, Md Rasel Al Mahmud, Keli Agama, George T. Lountos, Thorkell Andresson, Yves Pommier, Terrence R. Burke
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-12503-8
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author Xue Zhi Zhao
Wenjie Wang
Kiall F. Suazo
Md Rasel Al Mahmud
Keli Agama
George T. Lountos
Thorkell Andresson
Yves Pommier
Terrence R. Burke
author_facet Xue Zhi Zhao
Wenjie Wang
Kiall F. Suazo
Md Rasel Al Mahmud
Keli Agama
George T. Lountos
Thorkell Andresson
Yves Pommier
Terrence R. Burke
author_sort Xue Zhi Zhao
collection DOAJ
description Abstract Tyrosyl-DNA phosphodiesterase 1 (TDP1) is an important therapeutic target. We recently reported several crystal structures of TDP1 with small molecules bound within the catalytic pocket. These molecules bind by forming hydrogen bonds with residues of the catalytic HKN motifs. Guided by these interactions, in our current work, we used the MolSoft ICM Pro suite of software to perform a virtual screen of the publicly available DrugBank 5.0 (3449 structures) for the ability to bind to the TDP1 catalytic pocket. Among compounds identified as giving good binding scores were several β-lactams. The β-lactam pharmacophore serves as a key component in a range of antibiotics. We subjected a subset of the β-lactam hits to gel-based TDP1 fluorescence catalytic assays and established that certain members showed micromolar TDP1 inhibition. In follow-up, we evaluated a commercially available library of 90 β-lactam antibiotics. This led to our identification of additional β-lactams having micromolar TDP1 inhibitory potencies. In particular, cephalosporin C showed single-digit micromolar TDP1 IC50 values. Since β-lactams can form covalent bonds with serine residues in target penicillin-binding proteins (PBPs), we performed docking studies with cephalosporin C, which showed that it bound within the catalytic pocket and extended into the DNA substrate binding channel. Importantly, the modeling indicated that both noncovalent and covalent binding modes were theoretically possible. Surface plasmon resonance analysis demonstrated its non-covalent binding mode. Thus, β-lactams may serve as a new and potentially useful platform to design TDP1-binding ligands that interact with the catalytic pocket and extend into the DNA substrate binding channel.
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spelling doaj-art-0ebcafa1c7454a0e85c7571601648f4e2025-08-20T03:45:55ZengNature PortfolioScientific Reports2045-23222025-07-0115111210.1038/s41598-025-12503-8Potent β-lactam-based tyrosyl-DNA phosphodiesterase 1 inhibitors identified by a virtual screenXue Zhi Zhao0Wenjie Wang1Kiall F. Suazo2Md Rasel Al Mahmud3Keli Agama4George T. Lountos5Thorkell Andresson6Yves Pommier7Terrence R. Burke8Chemical Biology Laboratory, Center for Cancer Research, National Cancer Institute, National Institutes of HealthDevelopmental Therapeutics Branch & Laboratory of Molecular Pharmacology, Center for Cancer Research, National Cancer Institute, National Institutes of HealthChemical Biology Laboratory, Center for Cancer Research, National Cancer Institute, National Institutes of HealthDevelopmental Therapeutics Branch & Laboratory of Molecular Pharmacology, Center for Cancer Research, National Cancer Institute, National Institutes of HealthDevelopmental Therapeutics Branch & Laboratory of Molecular Pharmacology, Center for Cancer Research, National Cancer Institute, National Institutes of HealthBasic Science Program, Frederick National Laboratory for Cancer ResearchProtein Characterization Laboratory, Cancer Research Technology Program, Frederick National Laboratory for Cancer ResearchDevelopmental Therapeutics Branch & Laboratory of Molecular Pharmacology, Center for Cancer Research, National Cancer Institute, National Institutes of HealthChemical Biology Laboratory, Center for Cancer Research, National Cancer Institute, National Institutes of HealthAbstract Tyrosyl-DNA phosphodiesterase 1 (TDP1) is an important therapeutic target. We recently reported several crystal structures of TDP1 with small molecules bound within the catalytic pocket. These molecules bind by forming hydrogen bonds with residues of the catalytic HKN motifs. Guided by these interactions, in our current work, we used the MolSoft ICM Pro suite of software to perform a virtual screen of the publicly available DrugBank 5.0 (3449 structures) for the ability to bind to the TDP1 catalytic pocket. Among compounds identified as giving good binding scores were several β-lactams. The β-lactam pharmacophore serves as a key component in a range of antibiotics. We subjected a subset of the β-lactam hits to gel-based TDP1 fluorescence catalytic assays and established that certain members showed micromolar TDP1 inhibition. In follow-up, we evaluated a commercially available library of 90 β-lactam antibiotics. This led to our identification of additional β-lactams having micromolar TDP1 inhibitory potencies. In particular, cephalosporin C showed single-digit micromolar TDP1 IC50 values. Since β-lactams can form covalent bonds with serine residues in target penicillin-binding proteins (PBPs), we performed docking studies with cephalosporin C, which showed that it bound within the catalytic pocket and extended into the DNA substrate binding channel. Importantly, the modeling indicated that both noncovalent and covalent binding modes were theoretically possible. Surface plasmon resonance analysis demonstrated its non-covalent binding mode. Thus, β-lactams may serve as a new and potentially useful platform to design TDP1-binding ligands that interact with the catalytic pocket and extend into the DNA substrate binding channel.https://doi.org/10.1038/s41598-025-12503-8Tyrosyl-DNA phosphodiesterase 1 (TDP1) inhibitorsβ-LactamVirtual screenDrugBankFluorescence assaySurface plasmon resonance
spellingShingle Xue Zhi Zhao
Wenjie Wang
Kiall F. Suazo
Md Rasel Al Mahmud
Keli Agama
George T. Lountos
Thorkell Andresson
Yves Pommier
Terrence R. Burke
Potent β-lactam-based tyrosyl-DNA phosphodiesterase 1 inhibitors identified by a virtual screen
Scientific Reports
Tyrosyl-DNA phosphodiesterase 1 (TDP1) inhibitors
β-Lactam
Virtual screen
DrugBank
Fluorescence assay
Surface plasmon resonance
title Potent β-lactam-based tyrosyl-DNA phosphodiesterase 1 inhibitors identified by a virtual screen
title_full Potent β-lactam-based tyrosyl-DNA phosphodiesterase 1 inhibitors identified by a virtual screen
title_fullStr Potent β-lactam-based tyrosyl-DNA phosphodiesterase 1 inhibitors identified by a virtual screen
title_full_unstemmed Potent β-lactam-based tyrosyl-DNA phosphodiesterase 1 inhibitors identified by a virtual screen
title_short Potent β-lactam-based tyrosyl-DNA phosphodiesterase 1 inhibitors identified by a virtual screen
title_sort potent β lactam based tyrosyl dna phosphodiesterase 1 inhibitors identified by a virtual screen
topic Tyrosyl-DNA phosphodiesterase 1 (TDP1) inhibitors
β-Lactam
Virtual screen
DrugBank
Fluorescence assay
Surface plasmon resonance
url https://doi.org/10.1038/s41598-025-12503-8
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