Evidence map›Paper›PMID 40841488›Full record

Trial reportNature medicine2025

GD2-targeting CAR T cells in high-risk neuroblastoma: a phase 1/2 trial.

Franco Locatelli, Daria Pagliara, Maria A De Ioris, Marco Becilli, Giada Del Baldo, Annalisa Serra, Angela Mastronuzzi, Maria G Cefalo, Giuseppina Li Pira, Giovanna Leone and 18 more

Erratum issuedAbstract readClinical Trial, Phase IClinical Trial, Phase II
PubMed Publisher
In one paragraph

Trial report in Nature medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 37 papers.

0numbers the graph read from it
0cells of the map it votes in
37citing 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

37 citing papers in PubMed.

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  15. Cellular immunotherapy in melanoma: the next frontier in cancer treatment.Journal of experimental & clinical cancer research : CR · 2026
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

28 authors.

Franco LocatelliDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy. franco.locatelli@opbg.net.ORCID http://orcid.org/0000-0002-7976-3654
Daria PagliaraDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Maria A De IorisDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Marco BecilliDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Giada Del BaldoDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Annalisa SerraDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0003-2505-9888
Angela MastronuzziDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0002-4408-2373
Maria G CefaloDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Giuseppina Li PiraDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Giovanna LeoneTransfusion Unit, Department of Laboratories, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Valentina BertainaDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Francesco FabozziDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0002-3906-4410
Matteo Di NardoPediatric Intensive Care Unit, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Chiara RosignoliDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Maria Luisa D'AndreaImaging Department, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Alessandro CrocoliSurgical Oncology Unit, Bambino Gesù Pediatric Hospital IRCCS, Rome, Italy.
Sabina VennariniPediatric Radiotherapy Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy.
Matilde SinibaldiDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Stefano Di CeccaDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Marika GuercioDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0002-1356-5874
Laura IaffaldanoDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Barbarella LucarelliDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Mattia AlgeriDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Pietro MerliDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0001-6426-4046
Giovanna S ColafatiImaging Department, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Biagio De AngelisDepartment of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0002-7938-737X
Concetta Quintarelli *Department of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.
Francesca Del Bufalo *Department of Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.ORCID http://orcid.org/0000-0001-9643-3465

Funding

Associazione Italiana per la Ricerca sul Cancro (Italian Association for Cancer Research) AIRC IG 2023 id. 29057Associazione Italiana per la Ricerca sul Cancro (Italian Association for Cancer Research) MFAG 25106Associazione Italiana per la Ricerca sul Cancro (Italian Association for Cancer Research) Special Project 5×1000 no. 9962 (F.L.), AIRC IG 2018 id. 21724 (F.L.)Ministero della Salute (Ministry of Health, Italy) GR-2021-12372614; Ricerca Corrente/5x1000Ministero della Salute (Ministry of Health, Italy) RF-2021-12374120Ministero della Salute (Ministry of Health, Italy) Ricerca Corrente/5x1000Ministero dell'Istruzione, dell'Università e della Ricerca (Ministry of Education, University and Research) Grant PRIN 2020 to F.L.; Grant PRIN 2022 to F.L.Ministero dell'Istruzione, dell'Università e della Ricerca (Ministry of Education, University and Research) Grant PRIN 2022Ministero dello Sviluppo Economico (Ministry of Economic Development) Bando 2023 Filiere produttiveMinistry of Health, Italy | Agenzia Italiana del Farmaco, Ministero della Salute (Italian Medicines Agency) Ricerca Indipendete 2016
6 · The paper itself

Abstract

Antidisialoganglioside (GD2), third-generation chimeric antigen receptor (CAR) T cells (GD2-CART01) have shown encouraging efficacy in children with high-risk metastatic, relapsed, or refractory neuroblastoma in the interim analysis of a phase 1/2 clinical trial. We now present the final results obtained in all 35 patients enrolled and in 19 additional children selected with the same criteria of the trial and treated in a hospital exemption setting. Primary endpoints for the trial were safety, maximum tolerated dose, overall response rate (ORR) and complete remission rate at various timepoints. Secondary endpoints included 5-year overall survival (OS) and persistence of GD2-CART01. No new safety signals were observed. Grade 3 immune effector cell-associated neurotoxicity syndrome was diagnosed in four children and rapidly controlled with the activation of the inducible caspase-9 suicide gene by rimiducid. The maximum tolerated dose was 10 × 10

Indexed as

GangliosidesImmunotherapy, AdoptiveNeuroblastomaReceptors, Chimeric AntigenT-LymphocytesAdolescentChildChild, PreschoolFemaleHumansInfantMaleMaximum Tolerated DoseTreatment Outcomeganglioside, GD2GangliosidesReceptors, Chimeric Antigen

Identifiers

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.