Novel dual action chimera doxorubizen demonstrates superior efficacy to doxorubicin in acute leukemia

Abstract Modifying existing drugs to enhance their activity and reduce toxicity is a major focus of drug development. We developed a novel class of dual-action chimeric molecules for cancer therapy, linking known drugs to a DNA-methylating monomethyl triazene moiety (azene) via nucleophilic substitu...

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Main Authors: Dipak Walunj, Katarina Egarmina, Adi Zipin-Roitman, Siva Sai Naga Anurag Muddineni, Iryna Tkachenko, Pousali Mitra, Dror Tobi, Andrii Bazylevich, Ofer Shpilberg, Michael Milyavsky, Oshrat Hershkovitz-Rokah, Gary Gellerman
Format: Article
Language:English
Published: Nature Portfolio 2025-03-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-94373-8
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author Dipak Walunj
Katarina Egarmina
Adi Zipin-Roitman
Siva Sai Naga Anurag Muddineni
Iryna Tkachenko
Pousali Mitra
Dror Tobi
Andrii Bazylevich
Ofer Shpilberg
Michael Milyavsky
Oshrat Hershkovitz-Rokah
Gary Gellerman
author_facet Dipak Walunj
Katarina Egarmina
Adi Zipin-Roitman
Siva Sai Naga Anurag Muddineni
Iryna Tkachenko
Pousali Mitra
Dror Tobi
Andrii Bazylevich
Ofer Shpilberg
Michael Milyavsky
Oshrat Hershkovitz-Rokah
Gary Gellerman
author_sort Dipak Walunj
collection DOAJ
description Abstract Modifying existing drugs to enhance their activity and reduce toxicity is a major focus of drug development. We developed a novel class of dual-action chimeric molecules for cancer therapy, linking known drugs to a DNA-methylating monomethyl triazene moiety (azene) via nucleophilic substitution. In-vitro screening of these chimeras on various leukemia cell lines identified a potent chimera, doxorubizen, a sequel of the known DNA intercalator and topoisomerase 2 (Topo-II) inhibitor doxorubicin (Dox) and azene. Molecular docking and dynamic simulations showed doxorubizen as a more potent Topo-II inhibitor than Dox as it binds to major grooves in DNA. Moreover, the monomethyl triazene portion is positioned favorably through tetracene core intercalation, potentially facilitating methylation at nearby guanine bases. Doxorubizen demonstrated significantly higher cytotoxicity, mitochondrial depolarization, DNA intercalation, and cell death than Dox. A Topo-II activity assay confirmed potent enzyme inhibition by doxorubizen. The mechanism of action of doxorubizen involves the inhibition of DNA repair in proximity to double-strand breaks by guanine methylation, enhanced mitochondrial depolarization, and increased apoptosis. Furthermore, in an acute leukemia xenograft model, doxorubizen significantly reduced the leukemia burden compared to Dox while preserving body weight and liver function. This work underscores the therapeutic potential of doxorubizen in leukemia treatment.
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spelling doaj-art-63718f8cba4e490e8d765d046e98ecc82025-08-20T02:10:23ZengNature PortfolioScientific Reports2045-23222025-03-0115111710.1038/s41598-025-94373-8Novel dual action chimera doxorubizen demonstrates superior efficacy to doxorubicin in acute leukemiaDipak Walunj0Katarina Egarmina1Adi Zipin-Roitman2Siva Sai Naga Anurag Muddineni3Iryna Tkachenko4Pousali Mitra5Dror Tobi6Andrii Bazylevich7Ofer Shpilberg8Michael Milyavsky9Oshrat Hershkovitz-Rokah10Gary Gellerman11Department of Chemical Sciences, Ariel UniversityDepartment of Molecular Biology, Ariel UniversityDepartment of Pathology, Faculty of Medical & Health Sciences, Tel-Aviv UniversityDepartment of Chemical Sciences, Ariel UniversityDepartment of Chemical Sciences, Ariel UniversityDepartment of Chemical Sciences, Ariel UniversityDepartment of Molecular Biology, Ariel UniversityDepartment of Chemical Sciences, Ariel UniversityAdelson School of Medicine, Ariel UniversityDepartment of Pathology, Faculty of Medical & Health Sciences, Tel-Aviv UniversityDepartment of Molecular Biology, Ariel UniversityDepartment of Chemical Sciences, Ariel UniversityAbstract Modifying existing drugs to enhance their activity and reduce toxicity is a major focus of drug development. We developed a novel class of dual-action chimeric molecules for cancer therapy, linking known drugs to a DNA-methylating monomethyl triazene moiety (azene) via nucleophilic substitution. In-vitro screening of these chimeras on various leukemia cell lines identified a potent chimera, doxorubizen, a sequel of the known DNA intercalator and topoisomerase 2 (Topo-II) inhibitor doxorubicin (Dox) and azene. Molecular docking and dynamic simulations showed doxorubizen as a more potent Topo-II inhibitor than Dox as it binds to major grooves in DNA. Moreover, the monomethyl triazene portion is positioned favorably through tetracene core intercalation, potentially facilitating methylation at nearby guanine bases. Doxorubizen demonstrated significantly higher cytotoxicity, mitochondrial depolarization, DNA intercalation, and cell death than Dox. A Topo-II activity assay confirmed potent enzyme inhibition by doxorubizen. The mechanism of action of doxorubizen involves the inhibition of DNA repair in proximity to double-strand breaks by guanine methylation, enhanced mitochondrial depolarization, and increased apoptosis. Furthermore, in an acute leukemia xenograft model, doxorubizen significantly reduced the leukemia burden compared to Dox while preserving body weight and liver function. This work underscores the therapeutic potential of doxorubizen in leukemia treatment.https://doi.org/10.1038/s41598-025-94373-8Anticancer drugDNA intercalationDNA double-strand breaksMolecular
spellingShingle Dipak Walunj
Katarina Egarmina
Adi Zipin-Roitman
Siva Sai Naga Anurag Muddineni
Iryna Tkachenko
Pousali Mitra
Dror Tobi
Andrii Bazylevich
Ofer Shpilberg
Michael Milyavsky
Oshrat Hershkovitz-Rokah
Gary Gellerman
Novel dual action chimera doxorubizen demonstrates superior efficacy to doxorubicin in acute leukemia
Scientific Reports
Anticancer drug
DNA intercalation
DNA double-strand breaks
Molecular
title Novel dual action chimera doxorubizen demonstrates superior efficacy to doxorubicin in acute leukemia
title_full Novel dual action chimera doxorubizen demonstrates superior efficacy to doxorubicin in acute leukemia
title_fullStr Novel dual action chimera doxorubizen demonstrates superior efficacy to doxorubicin in acute leukemia
title_full_unstemmed Novel dual action chimera doxorubizen demonstrates superior efficacy to doxorubicin in acute leukemia
title_short Novel dual action chimera doxorubizen demonstrates superior efficacy to doxorubicin in acute leukemia
title_sort novel dual action chimera doxorubizen demonstrates superior efficacy to doxorubicin in acute leukemia
topic Anticancer drug
DNA intercalation
DNA double-strand breaks
Molecular
url https://doi.org/10.1038/s41598-025-94373-8
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