Identification of potent inhibitors of Leishmania donovani and Leishmania infantum chagasi, the causative agents of Old and New World visceral leishmaniasis

Abstract The drug discovery pipeline for neglected kinetoplastid diseases remains sparse. In particular, the field of leishmaniasis drug discovery has had limited success in translating potential drug candidates into viable therapies. Here, we describe the development of two lead compounds, BZ-1 and...

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Main Authors: Bilal Zulfiqar, Fabio Antonio Colombo, Juliana Barbosa Nunes, Patricia Ferreira Espuri, Aurea Favero Ferreira, Sujatha Manthri, Manu De Rycker, Marcos Jose Marques, Vicky M. Avery
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Communications Biology
Online Access:https://doi.org/10.1038/s42003-025-08386-0
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author Bilal Zulfiqar
Fabio Antonio Colombo
Juliana Barbosa Nunes
Patricia Ferreira Espuri
Aurea Favero Ferreira
Sujatha Manthri
Manu De Rycker
Marcos Jose Marques
Vicky M. Avery
author_facet Bilal Zulfiqar
Fabio Antonio Colombo
Juliana Barbosa Nunes
Patricia Ferreira Espuri
Aurea Favero Ferreira
Sujatha Manthri
Manu De Rycker
Marcos Jose Marques
Vicky M. Avery
author_sort Bilal Zulfiqar
collection DOAJ
description Abstract The drug discovery pipeline for neglected kinetoplastid diseases remains sparse. In particular, the field of leishmaniasis drug discovery has had limited success in translating potential drug candidates into viable therapies. Here, we describe the development of two lead compounds, BZ-1 and BZ1-I, which have potent in vitro anti-leishmanial activity against Leishmania donovani DD8 intracellular amastigotes (0.59 ± 0.13 µM and 0.40 ± 0.38 µM) with corresponding selectivity ( > 33.89 and > 49.12) for differentiated THP-1 cells (Human monocytic cell line), respectively. Further characterization and biological profiling identified that in addition to the activity against L. donovani DD8 (Old World - Indian strain), compounds were active against intracellular parasites from other species and strains of the Old and New World, namely L. donovani (Old World - Sudanese strain) and L. infantum chagasi (New World-South American strain). In vivo evaluation using the hamster model illustrated that the activity observed in vitro was translated in vivo, with outstanding results. Our data suggests that these compounds represent a promising starting point for developing a novel lead series for future anti-leishmanial therapeutics.
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spelling doaj-art-70e53cfb4b9d42bab5599299a3ec47eb2025-08-20T03:06:01ZengNature PortfolioCommunications Biology2399-36422025-07-018112010.1038/s42003-025-08386-0Identification of potent inhibitors of Leishmania donovani and Leishmania infantum chagasi, the causative agents of Old and New World visceral leishmaniasisBilal Zulfiqar0Fabio Antonio Colombo1Juliana Barbosa Nunes2Patricia Ferreira Espuri3Aurea Favero Ferreira4Sujatha Manthri5Manu De Rycker6Marcos Jose Marques7Vicky M. Avery8Discovery Biology, Centre for Cellular Phenomics, Griffith UniversityDepartment of Clinical and Toxicological Analysis, Faculty of Pharmaceutical Sciences, Federal University of AlfenasDepartment of Pathology, University of São PauloDepartment of Pathology and Parasitology, Institute of Biomedical Sciences, Federal University of AlfenasDepartment of Pathology, University of São PauloDrug Discovery Unit, University of DundeeDrug Discovery Unit, University of DundeeDepartment of Pathology and Parasitology, Institute of Biomedical Sciences, Federal University of AlfenasDiscovery Biology, Centre for Cellular Phenomics, Griffith UniversityAbstract The drug discovery pipeline for neglected kinetoplastid diseases remains sparse. In particular, the field of leishmaniasis drug discovery has had limited success in translating potential drug candidates into viable therapies. Here, we describe the development of two lead compounds, BZ-1 and BZ1-I, which have potent in vitro anti-leishmanial activity against Leishmania donovani DD8 intracellular amastigotes (0.59 ± 0.13 µM and 0.40 ± 0.38 µM) with corresponding selectivity ( > 33.89 and > 49.12) for differentiated THP-1 cells (Human monocytic cell line), respectively. Further characterization and biological profiling identified that in addition to the activity against L. donovani DD8 (Old World - Indian strain), compounds were active against intracellular parasites from other species and strains of the Old and New World, namely L. donovani (Old World - Sudanese strain) and L. infantum chagasi (New World-South American strain). In vivo evaluation using the hamster model illustrated that the activity observed in vitro was translated in vivo, with outstanding results. Our data suggests that these compounds represent a promising starting point for developing a novel lead series for future anti-leishmanial therapeutics.https://doi.org/10.1038/s42003-025-08386-0
spellingShingle Bilal Zulfiqar
Fabio Antonio Colombo
Juliana Barbosa Nunes
Patricia Ferreira Espuri
Aurea Favero Ferreira
Sujatha Manthri
Manu De Rycker
Marcos Jose Marques
Vicky M. Avery
Identification of potent inhibitors of Leishmania donovani and Leishmania infantum chagasi, the causative agents of Old and New World visceral leishmaniasis
Communications Biology
title Identification of potent inhibitors of Leishmania donovani and Leishmania infantum chagasi, the causative agents of Old and New World visceral leishmaniasis
title_full Identification of potent inhibitors of Leishmania donovani and Leishmania infantum chagasi, the causative agents of Old and New World visceral leishmaniasis
title_fullStr Identification of potent inhibitors of Leishmania donovani and Leishmania infantum chagasi, the causative agents of Old and New World visceral leishmaniasis
title_full_unstemmed Identification of potent inhibitors of Leishmania donovani and Leishmania infantum chagasi, the causative agents of Old and New World visceral leishmaniasis
title_short Identification of potent inhibitors of Leishmania donovani and Leishmania infantum chagasi, the causative agents of Old and New World visceral leishmaniasis
title_sort identification of potent inhibitors of leishmania donovani and leishmania infantum chagasi the causative agents of old and new world visceral leishmaniasis
url https://doi.org/10.1038/s42003-025-08386-0
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