Evidence map›Paper›PMID 39815015›Full record

ArticleNature medicine2025

Donor-derived GD2-specific CAR T cells in relapsed or refractory neuroblastoma.

Concetta Quintarelli, Francesca Del Bufalo, Maria Antonietta De Ioris, Marika Guercio, Mattia Algeri, Daria Pagliara, Domenico Alessandro Silvestris, Matteo Di Nardo, Matilde Sinibaldi, Stefano Di Cecca and 19 more

Abstract read
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Article in Nature medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

22 citing papers in PubMed.

  1. Trial
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  10. Review
  11. Immunotherapeutic Strategies Targeting GD2-Expressing Malignancies.Advances in experimental medicine and biology · 2026
    Review
  12. Article
  13. Challenges and opportunities of human iPSC-derived NK as "Off-the-shelf" cellular therapies.Journal of experimental & clinical cancer research : CR · 2025
    Review
  14. Review
  15. Inducing ferroptosis to improve cancer therapy: a promising tool for enhancing immunotherapy.Journal of experimental & clinical cancer research : CR · 2025
    Review
  16. Review
  17. CAR T cell persistence in cancer.Trends in cancer · 2025
    Review
  18. Review
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  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

29 authors.

Concetta Quintarelli *Department of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Francesca Del Bufalo *Department of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0001-9643-3465
Maria Antonietta De IorisDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Marika GuercioDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0002-1356-5874
Mattia AlgeriDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Daria PagliaraDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Domenico Alessandro SilvestrisDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Matteo Di NardoPediatric Intensive Care Unit, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Matilde SinibaldiDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Stefano Di CeccaDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Laura IaffaldanoDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Simona ManniDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Valentina FustainoDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0002-5708-8715
Maria Carmen GarganeseNuclear Medicine Unit/Imaging Department, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Giovanna Stefania ColafatiImaging Department, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Valentina BertainaDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Marco BecilliDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Angela MastronuzziDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0002-4408-2373
Francesco FabozziDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0002-3906-4410
Monica GunettiDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Stefano IacovelliDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Rossana BugianesiDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0001-8565-1539
Stefania MacchiaDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Giuseppina Li PiraDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Maria Giuseppina CefaloDepartment of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Giovanna LeoneTransfusion Unit, Department of Laboratories, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Giada Del Baldo *Department of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.
Biagio De Angelis *Department of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0002-7938-737X
Franco Locatelli *Department of Hematology/Oncology, Cell and Gene Therapy, Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), Bambino Gesù Children's Hospital, Rome, Italy. franco.locatelli@opbg.net.ORCID http://orcid.org/0000-0002-7976-3654

Funding

Associazione Italiana per la Ricerca sul Cancro (Italian Association for Cancer Research) AIRC IG 2018 id. 21724Associazione Italiana per la Ricerca sul Cancro (Italian Association for Cancer Research) fellowship ID25316Associazione Italiana per la Ricerca sul Cancro (Italian Association for Cancer Research) IG2023-ID29057Associazione Italiana per la Ricerca sul Cancro (Italian Association for Cancer Research) MFAG21979Associazione Italiana per la Ricerca sul Cancro (Italian Association for Cancer Research) Special Project 5×1000 no. 9962Ministero della Salute (Ministry of Health, Italy) LSH-TA Ecosistema innovativo della SaluteMinistero della Salute (Ministry of Health, Italy) RF2021-12374120Ministero dell'Istruzione, dell'Università e della Ricerca (Ministry of Education, University and Research) Italian PNRR CN3 "National Center for Gene Therapy and Drugs based on RNA Technology"Ministero dell'Istruzione, dell'Università e della Ricerca (Ministry of Education, University and Research) PRIN2022
6 · The paper itself

Abstract

Allogeneic chimeric antigen receptor (CAR) T cells targeting disialoganglioside-GD2 (ALLO_GD2-CART01) could be a therapeutic option for patients with relapsed or refractory, high-risk neuroblastoma (r/r HR-NB) whose tumors did not respond to autologous GD2-CART01 or who have profound lymphopenia. We present a case series of five children with HR-NB refractory to more than three different lines of therapy who received ALLO_GD2-CART01 in a hospital exemption setting. Four of them had previously received allogeneic hematopoietic stem cell transplantation. All patients experienced grade 2 or 3 cytokine release syndrome and one grade 2 neurotoxicity. Moderate acute graft-versus-host-disease occurred in four patients. ALLO_GD2-CART01 persisted for >6 weeks. Post-treatment, two complete responses were achieved and one maintained; in addition, one partial response and one stable disease were observed. Comparing the transcriptomic profiles obtained by RNA sequencing analyses of drug products with patient-matched, peripheral blood ALLO_GD2-CART01 collected at expansion, we found upregulation of genes associated with T cell activation and migration. In addition, after infusion, transcriptomic signaling analysis showed enrichment of genes involved in response to decreased oxygen levels, humoral immune response, cell polarization and immune-synapse formation. In comparison to autologous CAR T cells, ALLO_GD2-CAR T cells were characterized by pathways associated with T cell proliferation, immune-synapse formation and cell chemotaxis. The safety and efficacy of ALLO_GD2-CART01 in children with r/r HR-NB deserve further investigation in a prospective trial.

Indexed as

GangliosidesImmunotherapy, AdoptiveNeuroblastomaReceptors, Chimeric AntigenT-LymphocytesChildChild, PreschoolFemaleGraft vs Host DiseaseHematopoietic Stem Cell TransplantationHumansInfantMaleNeoplasm Recurrence, LocalReceptors, Antigen, T-Cellganglioside, GD2GangliosidesReceptors, Antigen, T-CellReceptors, Chimeric Antigen

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.