CAR-T cell therapy for juvenile-onset autoimmune diseases: a promising future?

Abstract Chimeric antigen receptor (CAR) T-cell therapy targeting B cells has shown promising results, including drug-free remission, in adult-onset autoimmune diseases. Extending this therapeutic approach to the pediatric population, particularly for juvenile autoimmune diseases, presents an exciti...

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Main Authors: Maurine Jouret, Sebastien Viel, Benjamin Fournier, Sarah Benezech, Jérome Avouac, Marc Scherlinger, Alexandre Belot, for the C3I consortium
Format: Article
Language:English
Published: BMC 2025-05-01
Series:Arthritis Research & Therapy
Subjects:
Online Access:https://doi.org/10.1186/s13075-025-03564-1
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author Maurine Jouret
Sebastien Viel
Benjamin Fournier
Sarah Benezech
Jérome Avouac
Marc Scherlinger
Alexandre Belot
for the C3I consortium
author_facet Maurine Jouret
Sebastien Viel
Benjamin Fournier
Sarah Benezech
Jérome Avouac
Marc Scherlinger
Alexandre Belot
for the C3I consortium
author_sort Maurine Jouret
collection DOAJ
description Abstract Chimeric antigen receptor (CAR) T-cell therapy targeting B cells has shown promising results, including drug-free remission, in adult-onset autoimmune diseases. Extending this therapeutic approach to the pediatric population, particularly for juvenile autoimmune diseases, presents an exciting opportunity. However, challenges specific to juvenile-onset autoimmune conditions, such as long-term adverse events, heightened disease activity, and the imperative to reduce steroid exposure, must be considered. While this strategy appears viable for these severe conditions, the limited data available for this population and the absence of evidence on cases with a high genetic component, such as monogenic lupus, represent significant challenges. Most monogenic lupus cases are associated with innate immune defects, and the involvement of B cells in these genetic anomalies remains poorly understood. In this review, we examine the potential indications, current knowledge, and limitations of CAR-T cell therapy in juvenile-onset autoimmune diseases, extending the discussion beyond early-onset lupus.
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publisher BMC
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series Arthritis Research & Therapy
spelling doaj-art-11fce15db94b4dd79a145ef740bf762a2025-08-20T03:09:19ZengBMCArthritis Research & Therapy1478-63622025-05-0127111410.1186/s13075-025-03564-1CAR-T cell therapy for juvenile-onset autoimmune diseases: a promising future?Maurine Jouret0Sebastien Viel1Benjamin Fournier2Sarah Benezech3Jérome Avouac4Marc Scherlinger5Alexandre Belot6for the C3I consortiumNational Referee Centre for Pediatric-Onset Rheumatic and Autoimmune Diseases (RAISE), Pediatric Nephrology, Rheumatology, Dermatology Unit, HFME, Hospices Civils de LyonInternational Center of Research in Infectiology, Lyon University, INSERM U1111, CNRS UMR 5308, ENS, UCBLDepartment for Immunology, Hematology and Pediatric Rheumatology, Necker Hospital, APHP, Institut IMAGINEInternational Center of Research in Infectiology, Lyon University, INSERM U1111, CNRS UMR 5308, ENS, UCBLRheumatology Department, Cochin Hospital, AP-HP. Centre Université Paris-CitéRheumatology Department, Strasbourg University HospitalNational Referee Centre for Pediatric-Onset Rheumatic and Autoimmune Diseases (RAISE), Pediatric Nephrology, Rheumatology, Dermatology Unit, HFME, Hospices Civils de LyonAbstract Chimeric antigen receptor (CAR) T-cell therapy targeting B cells has shown promising results, including drug-free remission, in adult-onset autoimmune diseases. Extending this therapeutic approach to the pediatric population, particularly for juvenile autoimmune diseases, presents an exciting opportunity. However, challenges specific to juvenile-onset autoimmune conditions, such as long-term adverse events, heightened disease activity, and the imperative to reduce steroid exposure, must be considered. While this strategy appears viable for these severe conditions, the limited data available for this population and the absence of evidence on cases with a high genetic component, such as monogenic lupus, represent significant challenges. Most monogenic lupus cases are associated with innate immune defects, and the involvement of B cells in these genetic anomalies remains poorly understood. In this review, we examine the potential indications, current knowledge, and limitations of CAR-T cell therapy in juvenile-onset autoimmune diseases, extending the discussion beyond early-onset lupus.https://doi.org/10.1186/s13075-025-03564-1LupusJuvenile dermatomyositisMonogenic lupusCAR-T cellsCD19B cells
spellingShingle Maurine Jouret
Sebastien Viel
Benjamin Fournier
Sarah Benezech
Jérome Avouac
Marc Scherlinger
Alexandre Belot
for the C3I consortium
CAR-T cell therapy for juvenile-onset autoimmune diseases: a promising future?
Arthritis Research & Therapy
Lupus
Juvenile dermatomyositis
Monogenic lupus
CAR-T cells
CD19
B cells
title CAR-T cell therapy for juvenile-onset autoimmune diseases: a promising future?
title_full CAR-T cell therapy for juvenile-onset autoimmune diseases: a promising future?
title_fullStr CAR-T cell therapy for juvenile-onset autoimmune diseases: a promising future?
title_full_unstemmed CAR-T cell therapy for juvenile-onset autoimmune diseases: a promising future?
title_short CAR-T cell therapy for juvenile-onset autoimmune diseases: a promising future?
title_sort car t cell therapy for juvenile onset autoimmune diseases a promising future
topic Lupus
Juvenile dermatomyositis
Monogenic lupus
CAR-T cells
CD19
B cells
url https://doi.org/10.1186/s13075-025-03564-1
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AT sarahbenezech cartcelltherapyforjuvenileonsetautoimmunediseasesapromisingfuture
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