IL-36 signaling as a drug target in Crohn’s disease patients with IL36RN mutations

Abstract The IL-36 signaling pathway has recently been identified as a key regulator of intestinal homeostasis and inflammation. However, the role of mutations in the IL-36R signaling pathway in the pathogenesis of inflammatory bowel disease remains unclear. We here identified four Crohn’s disease p...

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Main Authors: Julia Hecker, Christina Plattner, Camila A Cancino, Britt-Sabina Löscher, Judith Saurenbach, Marilena Letizia, Dietmar Rieder, TRR241 IBDome Consortium, Inka Freise, Kristina Koop, Clemens Neufert, Désirée Kunkel, Zainab Al Khatim, Lars-Arne Schaafs, Anja Schütz, Christoph Becker, Raja Atreya, Zlatko Trajanoski, Andre Franke, Elena Sonnenberg, Ahmed N Hegazy, Britta Siegmund, Carl Weidinger
Format: Article
Language:English
Published: Springer Nature 2025-05-01
Series:EMBO Molecular Medicine
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Online Access:https://doi.org/10.1038/s44321-025-00245-z
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author Julia Hecker
Christina Plattner
Camila A Cancino
Britt-Sabina Löscher
Judith Saurenbach
Marilena Letizia
Dietmar Rieder
TRR241 IBDome Consortium
Inka Freise
Kristina Koop
Clemens Neufert
Désirée Kunkel
Zainab Al Khatim
Lars-Arne Schaafs
Anja Schütz
Christoph Becker
Raja Atreya
Zlatko Trajanoski
Andre Franke
Elena Sonnenberg
Ahmed N Hegazy
Britta Siegmund
Carl Weidinger
author_facet Julia Hecker
Christina Plattner
Camila A Cancino
Britt-Sabina Löscher
Judith Saurenbach
Marilena Letizia
Dietmar Rieder
TRR241 IBDome Consortium
Inka Freise
Kristina Koop
Clemens Neufert
Désirée Kunkel
Zainab Al Khatim
Lars-Arne Schaafs
Anja Schütz
Christoph Becker
Raja Atreya
Zlatko Trajanoski
Andre Franke
Elena Sonnenberg
Ahmed N Hegazy
Britta Siegmund
Carl Weidinger
author_sort Julia Hecker
collection DOAJ
description Abstract The IL-36 signaling pathway has recently been identified as a key regulator of intestinal homeostasis and inflammation. However, the role of mutations in the IL-36R signaling pathway in the pathogenesis of inflammatory bowel disease remains unclear. We here identified four Crohn’s disease patients with heterozygous missense mutations in the IL-36 receptor antagonist (IL36RN, IL-36RA). Experimental overexpression and functional assays demonstrated that two identified mutations resulted in reduced expression of IL-36RA. In-depth immune profiling of one IL36RN-mutated patient revealed an increased response of PBMCs to IL-36 stimulation and elevated serum levels of IL-36-regulated cytokines. Administration of the IL-36R-blocking antibody spesolimab to this patient resulted in a reduction of intestinal inflammation and alterations in immune cell composition and function. Our findings indicate that pathogenic IL36RN mutations may contribute to the pathogenesis of Crohn’s disease in a subset of patients and that inhibiting IL-36 signaling could offer a personalized therapeutic approach for these patients.
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spelling doaj-art-e688a17418c8430caa7e4217cb01ab3a2025-08-20T03:05:53ZengSpringer NatureEMBO Molecular Medicine1757-46842025-05-011771539155510.1038/s44321-025-00245-zIL-36 signaling as a drug target in Crohn’s disease patients with IL36RN mutationsJulia Hecker0Christina Plattner1Camila A Cancino2Britt-Sabina Löscher3Judith Saurenbach4Marilena Letizia5Dietmar Rieder6TRR241 IBDome ConsortiumInka Freise7Kristina Koop8Clemens Neufert9Désirée Kunkel10Zainab Al Khatim11Lars-Arne Schaafs12Anja Schütz13Christoph Becker14Raja Atreya15Zlatko Trajanoski16Andre Franke17Elena Sonnenberg18Ahmed N Hegazy19Britta Siegmund20Carl Weidinger21Department of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinBiocenter, Institute of Bioinformatics, Medical University of InnsbruckDepartment of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinInstitute of Clinical Molecular Biology, Kiel University and University Medical CenterDepartment of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinDepartment of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinBiocenter, Institute of Bioinformatics, Medical University of InnsbruckDepartment of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinDepartment of Medicine 1, Friedrich-Alexander-Universität Erlangen-NürnbergDepartment of Medicine 1, Friedrich-Alexander-Universität Erlangen-NürnbergBerlin Institute of Health at Charité - Universitätsmedizin Berlin, Flow & Mass Cytometry Core FacilityDepartment of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinDepartment of Radiology, Charité - Universitätsmedizin BerlinMax-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC)Department of Medicine 1, Friedrich-Alexander-Universität Erlangen-NürnbergDepartment of Medicine 1, Friedrich-Alexander-Universität Erlangen-NürnbergBiocenter, Institute of Bioinformatics, Medical University of InnsbruckInstitute of Clinical Molecular Biology, Kiel University and University Medical CenterDepartment of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinDepartment of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinDepartment of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinDepartment of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Campus Benjamin FranklinAbstract The IL-36 signaling pathway has recently been identified as a key regulator of intestinal homeostasis and inflammation. However, the role of mutations in the IL-36R signaling pathway in the pathogenesis of inflammatory bowel disease remains unclear. We here identified four Crohn’s disease patients with heterozygous missense mutations in the IL-36 receptor antagonist (IL36RN, IL-36RA). Experimental overexpression and functional assays demonstrated that two identified mutations resulted in reduced expression of IL-36RA. In-depth immune profiling of one IL36RN-mutated patient revealed an increased response of PBMCs to IL-36 stimulation and elevated serum levels of IL-36-regulated cytokines. Administration of the IL-36R-blocking antibody spesolimab to this patient resulted in a reduction of intestinal inflammation and alterations in immune cell composition and function. Our findings indicate that pathogenic IL36RN mutations may contribute to the pathogenesis of Crohn’s disease in a subset of patients and that inhibiting IL-36 signaling could offer a personalized therapeutic approach for these patients.https://doi.org/10.1038/s44321-025-00245-zIL-36 SignalingCrohn’s DiseasePersonalized TherapyGenetics
spellingShingle Julia Hecker
Christina Plattner
Camila A Cancino
Britt-Sabina Löscher
Judith Saurenbach
Marilena Letizia
Dietmar Rieder
TRR241 IBDome Consortium
Inka Freise
Kristina Koop
Clemens Neufert
Désirée Kunkel
Zainab Al Khatim
Lars-Arne Schaafs
Anja Schütz
Christoph Becker
Raja Atreya
Zlatko Trajanoski
Andre Franke
Elena Sonnenberg
Ahmed N Hegazy
Britta Siegmund
Carl Weidinger
IL-36 signaling as a drug target in Crohn’s disease patients with IL36RN mutations
EMBO Molecular Medicine
IL-36 Signaling
Crohn’s Disease
Personalized Therapy
Genetics
title IL-36 signaling as a drug target in Crohn’s disease patients with IL36RN mutations
title_full IL-36 signaling as a drug target in Crohn’s disease patients with IL36RN mutations
title_fullStr IL-36 signaling as a drug target in Crohn’s disease patients with IL36RN mutations
title_full_unstemmed IL-36 signaling as a drug target in Crohn’s disease patients with IL36RN mutations
title_short IL-36 signaling as a drug target in Crohn’s disease patients with IL36RN mutations
title_sort il 36 signaling as a drug target in crohn s disease patients with il36rn mutations
topic IL-36 Signaling
Crohn’s Disease
Personalized Therapy
Genetics
url https://doi.org/10.1038/s44321-025-00245-z
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