Evidence map›Paper›PMID 39962287›Full record

ArticleNature medicine2025

Long-term outcomes of GD2-directed CAR-T cell therapy in patients with neuroblastoma.

Che-Hsing Li, Sandhya Sharma, Andras A Heczey, Mae L Woods, David H M Steffin, Chrystal U Louis, Bambi J Grilley, Sachin G Thakkar, Mengfen Wu, Tao Wang and 3 more

Registry-linked trialAbstract readClinical Trial, Phase I
PubMed Publisher
In one paragraph

Article in Nature medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00085930 (Administration of Peripheral Blood T-Cells and EBV Specific CTLs Transduced to Express GD-2 Specific Chimeric T Cell Receptors to Patients With Neuroblastoma), which is not on this map. Cited by 38 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
38citing papers in PubMed, 1 pooled it
–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.

NCT00085930 phase1completednot on this map

Administration of Peripheral Blood T-Cells and EBV Specific CTLs Transduced to Express GD-2 Specific Chimeric T Cell Receptors to Patients With Neuroblastoma

TypeinterventionalSponsorBaylor College of MedicineRan2003 to 2025Enrolled19ConditionsNeuroblastomaArmsEBV specific CTLs
3 · Its place in the literature

Who cites it

38 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Mapping the clinical landscape of multifunctional CAR T cells: Targets, trends, and synergies.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Pooled it
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  10. Advances in Cancer Immunotherapy for Solid Tumors.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026
    Review
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

13 authors.

Che-Hsing LiCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.ORCID http://orcid.org/0000-0003-0660-1125
Sandhya SharmaCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.
Andras A HeczeyCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.ORCID http://orcid.org/0000-0002-4263-9115
Mae L WoodsCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.
David H M SteffinCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.
Chrystal U LouisCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.
Bambi J GrilleyCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.
Sachin G ThakkarCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.
Mengfen WuDan L. Duncan Comprehensive Cancer Center, Baylor College of Medicine, Houston, TX, USA.
Tao WangDan L. Duncan Comprehensive Cancer Center, Baylor College of Medicine, Houston, TX, USA.ORCID http://orcid.org/0000-0003-1957-7160
Cliona M RooneyCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.
Malcolm K BrennerCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.
Helen E HeslopCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA. hheslop@bcm.edu.ORCID http://orcid.org/0000-0001-7049-7698

Funding

Tumor BiologyP30CA125123 · NCI · BAYLOR COLLEGE OF MEDICINE · PI Michael M Ittmann · 2007 to 2026
$73.9M
VECTOR PRODUCTIONP01CA094237 · NCI · BAYLOR COLLEGE OF MEDICINE · PI HESLOP, HELEN E, ROONEY, CLIONA M. · 2002 to 2017
$27.8M
NCI NIH HHS P01 CA094237U.S. Department of Health & Human Services | NIH | National Cancer Institute (NCI) P01CA094237U.S. Department of Health & Human Services | NIH | National Cancer Institute (NCI) P30CA125123
6 · The paper itself

Abstract

In a phase 1 clinical trial open to accrual from 2004 to 2009, we treated children with neuroblastoma with Epstein-Barr virus (EBV)-specific T lymphocytes and CD3-activated T cells-each expressing chimeric antigen receptors (CARs) targeting GD2 but without an embedded co-stimulatory sequence (first-generation CARs). These CARs incorporated barcoded sequences to track each infused population. We previously reported outcomes up to 5 years and now report long-term outcomes up to 18 years. Of 11 patients with active disease at infusion, three achieved a complete response that was sustained in two patients, one for 8 years until lost to follow-up and one for more than 18 years. Of eight patients with no evidence of disease at the time of CAR-T administration, five are disease free at their last follow-up between 10 years and 15 years after infusion. Intermittent low levels of transgene were detected during the follow-up period with significantly greater persistence in those who were long-term survivors. Despite using first-generation vectors that are no longer employed because of the lack of co-stimulatory domains, patients with relapsed/refractory neuroblastoma achieved long-term disease control after receiving GD2 CAR-T cell therapy, including one patient now in remission of relapsed disease for more than 18 years.ClinicalTrials.gov identifier: NCT00085930 .

Indexed as

GangliosidesImmunotherapy, AdoptiveNeuroblastomaReceptors, Antigen, T-CellReceptors, Chimeric AntigenT-LymphocytesAdolescentChildChild, PreschoolFemaleHerpesvirus 4, HumanHumansInfantMaleTreatment Outcomeganglioside, GD2GangliosidesReceptors, Antigen, T-CellReceptors, Chimeric Antigen

Identifiers

What OpenQuestion holds

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Registered trials

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.