New models for the development of and access to CAR T-cell therapies for children and adolescents with cancer: an ACCELERATE multistakeholder analysis

Andrew D J Pearson¹, Claudia Rossig², Crystal L Mackall³, Nirali N Shah⁴, André Baruchel⁵, Sam Daems⁶, John Anderson⁷, Andrea Biondi⁸, Nicholas Bird⁹, Nicole Bodmer¹⁰, Erica Brivio¹¹, Jochen Buechner¹², Friso G Calkoen¹¹, Todd Cooper¹³, Teresa de Rojas¹⁴, Elizabeth Fox¹⁵, Rebecca Gardner¹⁵, Sara Ghorashian⁷, Delphine Heenen¹⁶, Marianne Ifversen¹⁷, Elad Jacoby¹⁸, Manel Juan¹⁹, Leona Knox⁹, Krishna Komanduri²⁰, Jerome Larghero⁵, Franco Locatelli²¹, Donna Ludwinski²², Robbie G Majzner²³, Joe McDonough²⁴, Veronique Minard-Colin²⁵, Karsten Nysom¹⁷, Alberto Pappo¹⁵, Julie R Park¹⁵, Waseem Qasim²⁶, Concetta Quintarelli²¹, Susana Rives²⁷, Rayne H Rouce²⁸, Nicole Scobie²⁹, Christian Seitz³⁰, Sarah K Tasian³¹, Brenda Weigel³², Susan Weiner³³, C Michel Zwaan¹¹, Gilles Vassal³⁴

Affiliations

¹ ACCELERATE, Brussels, Belgium. Electronic address: andy1pearson@btinternet.com.
² University Children's Hospital Muenster, Muenster, Germany.
³ Stanford Cancer Institute, Stanford, CA, USA.
⁴ Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
⁵ Hôpital Universitaire Robert Debré and Saint-Louis, Paris, France.
⁶ Waterland Private Equity Investments, Antwerp, Belgium; I3h Institute, Université libre de Bruxelles, Brussels, Belgium.
⁷ University College London Great Ormond Street Institute of Child Health, Great Ormond Street Hospital, London, UK.
⁸ University of Milano-Bicocca, Monza, Italy.
⁹ Solving Kids' Cancer UK, London, UK.
¹⁰ University Children's Hospital, Zurich, Switzerland.
¹¹ Princess Máxima Center for Pediatric Oncology, Utrecht and Erasmus MC-Sophia Children's Hospital, Rotterdam, Netherlands.
¹² Oslo University Hospital, Oslo, Norway.
¹³ Cancer and Blood Disorders Service, Seattle Children's Hospital, Seattle, WA, USA.
¹⁴ ACCELERATE, Brussels, Belgium.
¹⁵ St Jude Children's Research Hospital, Memphis, TN, USA.
¹⁶ KickCancer, Brussels, Belgium.
¹⁷ Rigshospitalet, Copenhagen, Denmark.
¹⁸ Sheba Medical Center, Tel-Hashomer, Sackler School of Medicine, Tel Aviv University, Tel Aviv-Yafo, Israel.
¹⁹ Hospital Clínic de Barcelona, IDIBAPS, Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, Spain.
²⁰ University of California San Francisco, San Francisco, CA, USA.
²¹ IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy; Catholic University of the Sacred Heart, Rome, Italy.
²² Solving Kids' Cancer, New York, NY, USA.
²³ Dana-Farber Cancer Institute, Boston, MA, USA.
²⁴ The Andrew McDonough B+ Foundation, Wilmington, DE, USA.
²⁵ Gustave Roussy Cancer Centre, Paris, France.
²⁶ University College London, London, UK.
²⁷ Hospital Sant Joan de Déu, Barcelona, Spain; Institut de Recerca Sant Joan de Déu, Barcelona, Spain.
²⁸ Baylor College of Medicine, Houston, TX, USA.
²⁹ Zoe4Life, Sullens, Switzerland.
³⁰ Hopp Children's Cancer Center, Heidelberg, Germany.
³¹ Children's Hospital of Philadelphia, Division of Oncology and Center for Childhood Cancer Research, Philadelphia, PA, USA; Department of Pediatrics and Abramson Cancer Center, School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
³² University of Minnesota, Minneapolis, MN, USA.
³³ Children's Cancer Cause, Washington, DC, USA.
³⁴ ACCELERATE, Brussels, Belgium; Gustave Roussy Cancer Centre, Paris, France.

PMID: 40179917
DOI: 10.1016/S1470-2045(24)00736-8

Review

New models for the development of and access to CAR T-cell therapies for children and adolescents with cancer: an ACCELERATE multistakeholder analysis

Andrew D J Pearson et al. Lancet Oncol. 2025 Apr.

. 2025 Apr;26(4):e214-e224.

doi: 10.1016/S1470-2045(24)00736-8.

Authors

Affiliations

¹ ACCELERATE, Brussels, Belgium. Electronic address: andy1pearson@btinternet.com.
² University Children's Hospital Muenster, Muenster, Germany.
³ Stanford Cancer Institute, Stanford, CA, USA.
⁴ Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
⁵ Hôpital Universitaire Robert Debré and Saint-Louis, Paris, France.
⁶ Waterland Private Equity Investments, Antwerp, Belgium; I3h Institute, Université libre de Bruxelles, Brussels, Belgium.
⁷ University College London Great Ormond Street Institute of Child Health, Great Ormond Street Hospital, London, UK.
⁸ University of Milano-Bicocca, Monza, Italy.
⁹ Solving Kids' Cancer UK, London, UK.
¹⁰ University Children's Hospital, Zurich, Switzerland.
¹¹ Princess Máxima Center for Pediatric Oncology, Utrecht and Erasmus MC-Sophia Children's Hospital, Rotterdam, Netherlands.
¹² Oslo University Hospital, Oslo, Norway.
¹³ Cancer and Blood Disorders Service, Seattle Children's Hospital, Seattle, WA, USA.
¹⁴ ACCELERATE, Brussels, Belgium.
¹⁵ St Jude Children's Research Hospital, Memphis, TN, USA.
¹⁶ KickCancer, Brussels, Belgium.
¹⁷ Rigshospitalet, Copenhagen, Denmark.
¹⁸ Sheba Medical Center, Tel-Hashomer, Sackler School of Medicine, Tel Aviv University, Tel Aviv-Yafo, Israel.
¹⁹ Hospital Clínic de Barcelona, IDIBAPS, Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, Spain.
²⁰ University of California San Francisco, San Francisco, CA, USA.
²¹ IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy; Catholic University of the Sacred Heart, Rome, Italy.
²² Solving Kids' Cancer, New York, NY, USA.
²³ Dana-Farber Cancer Institute, Boston, MA, USA.
²⁴ The Andrew McDonough B+ Foundation, Wilmington, DE, USA.
²⁵ Gustave Roussy Cancer Centre, Paris, France.
²⁶ University College London, London, UK.
²⁷ Hospital Sant Joan de Déu, Barcelona, Spain; Institut de Recerca Sant Joan de Déu, Barcelona, Spain.
²⁸ Baylor College of Medicine, Houston, TX, USA.
²⁹ Zoe4Life, Sullens, Switzerland.
³⁰ Hopp Children's Cancer Center, Heidelberg, Germany.
³¹ Children's Hospital of Philadelphia, Division of Oncology and Center for Childhood Cancer Research, Philadelphia, PA, USA; Department of Pediatrics and Abramson Cancer Center, School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
³² University of Minnesota, Minneapolis, MN, USA.
³³ Children's Cancer Cause, Washington, DC, USA.
³⁴ ACCELERATE, Brussels, Belgium; Gustave Roussy Cancer Centre, Paris, France.

PMID: 40179917
DOI: 10.1016/S1470-2045(24)00736-8

Abstract

Realising the potentially substantial benefits of chimeric antigen receptor (CAR) T-cell therapy for children with cancer is hindered by non-scientific barriers that are also relevant for other rare diseases. A solely commercial development model will not deliver optimally due to insufficient return on investment for pharmaceutical companies. Access to therapies is restricted for patients who might benefit and advancing innovation in the academic research setting is difficult. Challenges relating to CAR T-cell therapies in paediatric malignancies and how they might be addressed were discussed in a meeting convened by ACCELERATE-an international multistakeholder organisation aiming to advance the timely investigation of new anticancer drugs. New academic and biopharma hybrid development models could benefit rare populations and coordination of early development can promote synergy and avoid duplicative efforts. Following promising first-in-child trials, new models are needed to support pivotal trials, decentralised manufacturing, registration, and reduced costs. The European Medicines Agency and the US Food and Drug Administration encourage academic development and early discussions. A biotech company funded via a pooled investment vehicle could provide access to safe and effective products for children and adolescents with cancer through registration and reimbursement.

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Conflict of interest statement

Declaration of interests CR has received honoraria from Amgen and Novartis for advisory board participation and speaking commitments. CLM received research funding from Tune Therapeutics and Lyell Immunopharma; receives consulting fees from Immatics, Ensoma, Mammoth, Lyell, CARGO Therapeutics, Link Cell Therapies, and GBM NewCo; receives royalties from CARGO Therapeutics via the National Institutes of Health and Stanford; and has equity in Lyell Immunopharma, CARGO Therapeutics, Link Cell Therapies, and Ensoma. NNS has received research funding from Lentigen, VOR Bio, and CARGO Therapeutics; has attended advisory board meetings (no honoraria) for VOR Bio, ImmunoACT, and Sobi; and receives royalties from CARGO Therapeutics. ABa has received research funding and honoraria from Servier; has participated in advisory boards for CARGO Therapeutics, JAZZ Pharmaceuticals, and Wugen; and received institutional funding from AstraZeneca. JB has served on advisory board, steering committees, or both (with honoraria), speakers’ bureaus, and has received non-financial support from Novartis and advisory board honoraria from Pfizer, Kite, and Janssen. SG has received honoraria from Novartis; patents, trial management, and conference support from Autolus; and holds patents with UCLB. SD owns stock in Waterland managed funds. MJ is a member of an academic institution (Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, Spain) that has two CAR T-cell products authorised for use but without economic profit and is one of the patent's inventors. FL has received speaker bureau fees from Amgen, Bluebird Bio, Gilead, Jazz Pharmaceuticals, Medac, Miltenyi, Neovii, Novartis, Sanofi, and SOBI and received honoraria from Amgen, Neovii, Novartis, Sanofi, and Vertex for participation in advisory boards. RGM is a co-founder of, consultant for, and holds equity in Link Cell Therapies and CARGO Therapeutics; has served as a consultant for and holds equity in Lyell Immunopharma, Innervate Radiopharmaceuticals, and Waypoint Bio; and has consulted for NKarta, Arovella Pharmaceuticals, GammaDelta Therapeutics, Aptorum Group, Zai Labs, Immunai, Gadeta, and FATE Therapeutics. VM-C received institutional funding from AbbVie, Miltenyi, Roche, Adaptimmune, and Novartis. KN received institutional payments from Novartis for clinical trial conduct. JL has received honoraria from Novartis, Kite/Gilead, BMS, Miltenyi, and Janssen. CQ is a named inventor on multiple patent applications related to CAR T and CAR NK technologies. SR has received honoraria, travel support, or both from Novartis and OneChain Immunotherapeutics; honoraria from Novartis; and for participation in data safety monitoring boards. RHR has received honoraria from Novartis and consulting fees from Pfizer. SKT has received research funding from Beam Therapeutics, Incyte Corporation, and Kura Oncology; has served on scientific advisory boards for Aleta Biotherapeutics, AstraZeneca, Jazz Pharmaceuticals, Kestrel Therapeutics, Syndax Pharmaceuticals, and Wugen; and has received travel support from Amgen and Jazz Pharmaceuticals. CMZ received institutional funding from Pfizer, Daiichi Sankyo, Kura Oncology, AbbVie, Jazz Pharmaceuticals, and Takeda, and has consulted for Syndax, Beigene, Janssen, Novartis, Incyte, Sanofi, Roche, and Sutro. JRP has intellectual property held in conjunction with Umoja Biopharma. All other authors declare no competing interests.

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New models for the development of and access to CAR T-cell therapies for children and adolescents with cancer: an ACCELERATE multistakeholder analysis

Affiliations

New models for the development of and access to CAR T-cell therapies for children and adolescents with cancer: an ACCELERATE multistakeholder analysis

Authors

Affiliations

Abstract

Conflict of interest statement

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