CAR T cell therapy for children with rheumatic disease: the time is now

Holly Wobma, Division of Immunology, Boston Children's Hospital, Boston, MA, USA.
Stacy P. Ardoin, Department of Paediatrics, Division of Rheumatology, Ohio State University, Columbus, OH, USA.
Challice L. Bonifant, The Sidney Kimmel Comprehensive Cancer Center, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Jennifer C. Cooper, Department of Paediatrics, Section of Rheumatology, University of Colorado School of Medicine, Children's Hospital Colorado, Aurora, CO, USA.
Hanna Kim, Juvenile Myositis Pathogenesis and Therapeutics Unit, National Institute of Arthritis Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.
Rebecca E. Sadun, Departments of Medicine and Paediatrics (Rheumatology), Duke University School of Medicine, Durham, NC, USA.
Laura Lewandowski, Lupus Genomics and Global Health Disparities Unit, National Institute of Arthritis Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.
Michael Keller, Center for Cancer and Immunology Research and Division of Allergy and Immunology, Children's National Hospital, Washington, DC, USA.
Robert A. Colbert, Paediatric Translational Research Branch, National Institute of Arthritis, Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.
Cuoghi Edens, Departments of Internal Medicine and Paediatrics, Sections of Rheumatology and Paediatric Rheumatology, University of Chicago Medicine, Chicago, IL, USA.
Kimberly DeQuattro, Department of Medicine, Division of Rheumatology, University of Pennsylvania, Philadelphia, PA, USA.
Kyla Driest, Department of Paediatrics, Division of Rheumatology, Ohio State University, Columbus, OH, USA.
Julia Shalen, Department of Paediatrics, Division of Paediatric Allergy, Immunology and Rheumatology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Ivana Stojkic, Department of Paediatrics, Division of Rheumatology, Ohio State University, Columbus, OH, USA.
Andrea Knight, Division of Rheumatology, Hospital for Sick Children, Department of Paediatrics, Temerty Faculty of Medicine, University of Toronto, Toronto, Canada.
Colleen Annesley, Department of Paediatrics, Division of Paediatric Oncology, University of Washington, Seattle, WA, USA.
Kathryn S. Torok, Department of Paediatrics, Division of Rheumatology, University of Pittsburgh Medical Center, Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
Caitlin W. Elgarten, Division of Oncology, Cellular Therapy and Transplantation Section, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Toshihiro Onishi, Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ, USA.
Shaun W. Jackson, Seattle Children's Research Institute, Seattle, WA, USA.
Susan Prockop, Dana Farber Cancer Institute/Boston Children's Hospital Center for Cancer and Blood Disorders, Boston, MA, USA.
Nirali N. Shah, Paediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Kaveh Ardalan, Department of Paediatrics, Division of Paediatric Rheumatology, Duke University School of Medicine, Durham, NC, USA.
Margaret Lamb, Department of Paediatrics, Division of Hematology, Oncology, Blood and Marrow Transplant, The Ohio State University, Nationwide Children's Hospital, Columbus, OH, USA. margaret.lamb2@nationwidechildrens.org.

Document Type

Journal Article

Publication Date

8-1-2025

Journal

Nature reviews. Rheumatology

Volume

21

Issue

8

DOI

10.1038/s41584-025-01272-3

Abstract

Initial success with B cell-targeted chimeric antigen receptor (CAR) T cells for the treatment of systemic lupus erythematosus and other rheumatic diseases has generated enthusiasm for the broad application of this technology outside of the field of oncology. Paediatric patients with severe rheumatic diseases require lifelong therapy with a substantial toxicity burden and a high cost of care. Paradigm-shifting treatments, including CAR T cells, are desperately needed. Although CAR T cell therapy shows promise for paediatric rheumatic diseases, there are unique aspects of care compared with adults, which require careful consideration and expertise. In response, we established the Integrated Multidisciplinary Paediatric Autoimmunity and Cell Therapy (IMPACT) working group, comprising international experts in the fields of paediatric rheumatology, oncology and cellular therapy, immunology and nephrology, to address the challenges of introducing cell therapies to patients with paediatric-onset autoimmune diseases. Given the possible benefits, we advocate for the study of CAR T cells in paediatric patients with rheumatic diseases who carry a lifelong risk of morbidity and mortality from chronic illness and medication toxicity. As this patient population is relatively small, consensus around definitions of success, robust study of predictors of response and uniform assessment and reporting of toxicities are critical to advancing the field.

Department

Pediatrics

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