Evidence map›Paper›PMID 39775044›Full record

ArticleNature medicine2025

Intracerebroventricular B7-H3-targeting CAR T cells for diffuse intrinsic pontine glioma: a phase 1 trial.

Nicholas A Vitanza, Rebecca Ronsley, Michelle Choe, Kristy Seidel, Wenjun Huang, Stephanie D Rawlings-Rhea, Madison Beam, Leonel Steinmetzer, Ashley L Wilson, Christopher Brown and 32 more

Abstract readClinical Trial, Phase I
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. Cited by 128 papers, 6 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
128citing papers in PubMed, 6 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.

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

128 citing papers in PubMed, 6 syntheses or guidelines pooled it.

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  17. Immunotherapy for Glioma: A compartmental framework for resistance and rational combination design.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026
    Review
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68 more citing papers are in PubMed but not listed here.

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

42 authors.

Nicholas A VitanzaBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA. nicholas.vitanza@seattlechildrens.org.ORCID http://orcid.org/0000-0002-3966-4985
Rebecca RonsleyBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.
Michelle ChoeBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.ORCID http://orcid.org/0000-0001-9850-9435
Kristy SeidelSeattle Children's Therapeutics, Seattle, WA, USA.
Wenjun HuangSeattle Children's Therapeutics, Seattle, WA, USA.ORCID http://orcid.org/0000-0001-8948-7262
Stephanie D Rawlings-RheaSeattle Children's Therapeutics, Seattle, WA, USA.
Madison BeamSeattle Children's Therapeutics, Seattle, WA, USA.ORCID http://orcid.org/0009-0006-5868-1939
Leonel SteinmetzerSeattle Children's Therapeutics, Seattle, WA, USA.
Ashley L WilsonSeattle Children's Therapeutics, Seattle, WA, USA.ORCID http://orcid.org/0000-0002-4440-001X
Christopher BrownSeattle Children's Therapeutics, Seattle, WA, USA.
Adam BeebeSeattle Children's Therapeutics, Seattle, WA, USA.
Catherine LindgrenSeattle Children's Therapeutics, Seattle, WA, USA.
Joshua A GustafsonSeattle Children's Therapeutics, Seattle, WA, USA.
Amy WeinDepartment of Pediatrics, Seattle Children's Hospital, University of Washington, Seattle, WA, USA.
Susan HoltzclawDepartment of Pediatrics, Seattle Children's Hospital, University of Washington, Seattle, WA, USA.
Corrine HoeppnerDepartment of Pediatrics, Seattle Children's Hospital, University of Washington, Seattle, WA, USA.
Hannah E GoldsteinDivision of Neurosurgery, Seattle Children's Hospital and Department of Neurological Surgery, University of Washington, Seattle, WA, USA.
Samuel R BrowdDivision of Neurosurgery, Seattle Children's Hospital and Department of Neurological Surgery, University of Washington, Seattle, WA, USA.
Jason S HauptmanDivision of Neurosurgery, Seattle Children's Hospital and Department of Neurological Surgery, University of Washington, Seattle, WA, USA.
Amy LeeDivision of Neurosurgery, Seattle Children's Hospital and Department of Neurological Surgery, University of Washington, Seattle, WA, USA.
Jeffrey G OjemannDivision of Neurosurgery, Seattle Children's Hospital and Department of Neurological Surgery, University of Washington, Seattle, WA, USA.
Erin E CrottyBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.ORCID http://orcid.org/0000-0002-2362-695X
Sarah E S LearyBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.ORCID http://orcid.org/0000-0003-0225-6184
Francisco A PerezDepartment of Radiology, Seattle Children's Hospital, Seattle, WA, USA.
Jason N WrightDepartment of Radiology, Seattle Children's Hospital, Seattle, WA, USA.
Marta M AlonsoDepartment of Pediatrics, Program of Solid Tumors, University Clinic of Navarra, CIMA-Universidad de Navarra, Pamplona, Spain.ORCID http://orcid.org/0000-0002-7520-7351
Matthew D DunCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Newcastle, New South Wales, Australia.ORCID http://orcid.org/0000-0002-9063-5370
Jessica B FosterDivision of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Diana HurstDepartment of Pediatrics, Seattle Children's Hospital, University of Washington, Seattle, WA, USA.
Ada KongDepartment of Pharmacy, Seattle Children's Hospital, Seattle, WA, USA.
Alison ThomsenDepartment of Pediatrics, Seattle Children's Hospital, University of Washington, Seattle, WA, USA.
Rimas J OrentasBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.
Catherine M AlbertBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.
Navin PintoBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.ORCID http://orcid.org/0000-0002-8775-6710
Colleen AnnesleyBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.
Rebecca A GardnerBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.ORCID http://orcid.org/0000-0003-1532-3200
On HoSeattle Children's Therapeutics, Seattle, WA, USA.
Sowmya PattabhiSeattle Children's Therapeutics, Seattle, WA, USA.
Juliane GustCenter for Integrative Brain Research, Seattle Children's Research Institute, Seattle, WA, USA.ORCID http://orcid.org/0000-0001-5679-1989
Jason P WendlerSeattle Children's Therapeutics, Seattle, WA, USA.
Julie R ParkBen Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children's Research Institute, Seattle, WA, USA.ORCID http://orcid.org/0000-0002-6777-9165
Michael C JensenSeattle Children's Therapeutics, Seattle, WA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Diffuse intrinsic pontine glioma (DIPG) is a fatal central nervous system (CNS) tumor that confers a median survival of 11 months. As B7-H3 is expressed on pediatric CNS tumors, we conducted BrainChild-03, a single-center, dose-escalation phase 1 clinical trial of repetitive intracerebroventricular (ICV) dosing of B7-H3-targeting chimeric antigen receptor T cells (B7-H3 CAR T cells) for children with recurrent or refractory CNS tumors and DIPG. Here we report results from Arm C, restricted to patients with DIPG. The primary objectives were to assess feasibility and tolerability, which were both met. Secondary objectives included assessments of CAR T cell distribution and survival. A total of 23 patients with DIPG enrolled, and 21 were treated with repeated doses of ICV B7-H3 CAR T cells using intra-patient dose-escalation regimens without previous lymphodepletion. Concurrent tumor-directed therapy, including re-irradiation, was not allowed while on protocol therapy. We delivered a total of 253 ICV doses and established the highest planned dose regimen, DR4, which escalated up to 10 × 10

Indexed as

B7 AntigensBrain Stem NeoplasmsDiffuse Intrinsic Pontine GliomaImmunotherapy, AdoptiveReceptors, Chimeric AntigenT-LymphocytesAdolescentChildChild, PreschoolFemaleHumansMaleB7 AntigensCD276 protein, humanReceptors, Chimeric Antigen

Identifiers

PMID39775044
PMCPMC11922736

What OpenQuestion holds

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Registered trials

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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.