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Review
. 2025 Oct 30:S2666-6367(25)01527-1.
doi: 10.1016/j.jtct.2025.10.023. Online ahead of print.

Opportunities and Challenges with CAR T-cell Treatment of Children and Young Adults with B-Cell Acute Lymphoblastic Leukemia: Review and Recommendations from the Westhafen Intercontinental Group

Candelaria Deimundo Roura  1 , Persis Amrolia  2 , Marc Ansari  3 , Peter Bader  4 , Adriana Balduzzi  5 , Jochen Buechner  6 , Emily M Hsieh  7 , Regina M Myers  8 , Giorgio Ottaviano  9 , Christina Peters  10 , Rayne H Rouce  11 , Nirali N Shah  12 , Michael R Verneris  13 , Krzysztof Kalwak  14 , Michael A Pulsipher  15
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Free article
Review

Opportunities and Challenges with CAR T-cell Treatment of Children and Young Adults with B-Cell Acute Lymphoblastic Leukemia: Review and Recommendations from the Westhafen Intercontinental Group

Candelaria Deimundo Roura et al. Transplant Cell Ther. .
Free article

Abstract

Chimeric Antigen-Receptor T-cells (CAR T-cells) targeted at pediatric B-cell precursor acute lymphoblastic leukemia (B-ALL) have changed the paradigm for treatment of relapsed and refractory B-ALL. We present a comprehensive review and recommendations approaching this topic from the Westhafen Intercontinental Group, which is comprised of leaders from the International Berlin Frankfurt, Muenster (iBFM) Stem Cell Transplantation Committee, the Center for International Blood and Marrow Transplant Research (CIBMTR) Pediatric Cancer Working Committee, the Children's Oncology Group (COG) Cellular Therapy Committee, the Pediatric Diseases Working Party (PDWP) of the European Society for Bone and Marrow Transplantation (EBMT) and the Pediatric Transplantation and Cellular Therapy Consortium (PTCTC). In this paper we examine the current state of CAR T-cell therapy in pediatric B-ALL, assess current and emerging integration of CAR T-cells into treatment algorithms, and discuss emerging strategies to overcome existing challenges.

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Conflict of interest statement

Declaration of competing interest This research was supported [in part] by the Intramural Research Program of the National Institutes of Health (NIH). The contributions of the NIH author(s) were made as part of their official duties as NIH federal employees, are in compliance with agency policy requirements, and are considered Works of the United States Government. However, the findings and conclusions presented in this paper are those of the author(s) and do not necessarily reflect the views of the NIH or the U.S. Department of Health and Human Services. P.A. reports receiving patents and/or royalties from Autolus PLC. A.B. has received speaker’s bureau fees from Novartis, Medac, Amgen, and Neovii. J.B. has participated on advisory boards, study steering committees, and speaker’s bureaus for Novartis, Medac, Pfizer, Amgen, and Janssen. E.M.H. reports consulting for Novartis. R.H.R. has received honoraria from Pierre Fabre for advisory board participation, from Novartis for advisory board work in the last two years, and has provided consulting services to Pfizer during the same period. N.N.S. receives research funding from Lentigen, VOR Bio, and CARGO Therapeutics; has attended advisory board meetings without honoraria for VOR, ImmunoACT, and Sobi; and receives royalties from CARGO Therapeutics. K.K. reports participation in Novartis’ speaker’s bureau. M.A.P. Advisory boards—Novartis, Cargo, Pfizer, Autolous, Garuda, Mesoblast. Educational lecture—Sanofi. Study support Miltenyi, Adaptive. All other authors have no other financial conflict of interests.

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