ArticleNature medicine2020
Locoregional delivery of CAR T cells to the cerebrospinal fluid for treatment of metastatic medulloblastoma and ependymoma.
Erratum issuedOpen access · greenAbstract readValidation Study
In one paragraph
Article in Nature medicine, 2020. 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 169 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
169citing papers in PubMed, 2 pooled it
12.6field-weighted citation impact, top 1% of its field
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.
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.
Who cites it
169 citing papers in PubMed, 2 syntheses or guidelines pooled it, 235 citations in OpenAlex.
- Locoregional and systemic adoptive cellular therapies for pediatric brain tumors: a systematic review of CAR‑T, TCR‑engineered T cells, and NK cell strategies.Neurosurgical review · 2026Pooled it
- Locoregional delivery of CAR T cells in high-grade gliomas: a systematic analysis of safety, efficacy, and emerging biomarkers of response.Journal for immunotherapy of cancer · 2026Pooled it
- Paediatric therapeutic development workshop on medulloblastoma.British journal of cancer · 2026Review
- Chimeric antigen receptor (CAR)-T cell therapy for solid tumors in pediatric patients: current breakthroughs, dilemmas, and strategies.Experimental hematology & oncology · 2026Review
- CAR-T cell therapy: potential for paediatric brain tumours-an update.Journal of neuro-oncology · 2026Review
- Phase 1 study of intracranial T cell therapy with pemetrexed for refractory brain metastases and glioblastomas.Cell reports. Medicine · 2026Article
- Potent anti-tumor preclinical efficacy of B7-H3 CAR-T cells in H3G34-mutant, Diffuse Hemispheric Glioma.Neuro-oncology · 2026Article
- PCK2 as a potential therapeutic target for aggressive MYC-amplified non-WNT/non-SHH medulloblastoma based on tumor continuum.Acta neuropathologica communications · 2026Article
- New developments and prospects for drug delivery in medulloblastoma.Molecular therapy. Oncology · 2026Review
- MIF-CD74 signaling drives immune modulation in medulloblastoma.Neuro-oncology · 2026Article
- Allogeneic CD19 CAR T cells armed with an anti-rejection CD70 CAR overcome antigen escape and evade alloimmune responses.Nature communications · 2026Article
- Article
- The quintessential role for CAR T cell therapy in children, adolescents and young adults with cancer.Nature reviews. Clinical oncology · 2026Review
- The promise of immunotherapy for central nervous system tumours.Nature reviews. Immunology · 2026Review
- Antigen Specificity and Cell Engineering Determine CAR T Cell Efficacy in Group 3 Medulloblastoma.Research square · 2026Article
- Ibrutinib and PD-1 Blockade Potentiate Mesothelin-Targeting CAR T-cell Therapy in Preclinical Models of Pancreatic Cancer.Clinical cancer research : an official journal of the American Association for Cancer Research · 2026Article
- Immune checkpoint inhibitors in medulloblastoma: current updates in preclinical and clinical developments.Frontiers in oncology · 2026Review
- Overcoming immunotherapy barriers in pediatric brain tumors: epigenetic strategies.Frontiers in oncology · 2026Review
- Decoding the immune microenvironment: precision immunotherapy for medulloblastoma subtypes.Frontiers in immunology · 2026Review
- CAR T-cells for the treatment of CNS malignancies.Frontiers in immunology · 2026Review
109 more citing papers are in PubMed but not listed here.
Corrections and comments
- Erratum issued
Authors and funding
56 authors at 11 institutions in 4 countries.
Laura K DonovanThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Alberto DelaidelliDepartment of Molecular Oncology, British Columbia Cancer Research Centre, Vancouver, British Columbia, Canada.
Sujith K JosephTexas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.
Kevin BielamowiczTexas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.
Kristen FousekTexas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.ORCID http://orcid.org/0000-0002-2307-5000
Borja L HolgadoThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Alex MannoThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Dilakshan SrikanthanThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Ahmed Z GadTexas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.ORCID http://orcid.org/0000-0002-6149-3414
Randy Van OmmerenThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0002-7948-7530
David PrzelickiThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Cory RichmanThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Vijay RamaswamyThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0002-6557-895X
Craig DanielsThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Jonelle G PallotaThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Tajana DouglasThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Alyssa C M JoyntThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Joonas HaapasaloThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Carolina NorThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Maria C VladoiuThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Claudia M Kuzan-FischerThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Livia GarziaCancer Research Program, McGill University Health Centre Research Institute, Montreal, Quebec, Canada.
Stephen C MackBrain Tumour Program, Children's Cancer Centre and Department of Paediatrics, Baylor College of Medicine, Houston, TX, USA.
Srinidhi VaradharajanBrain Tumour Program, Children's Cancer Centre and Department of Paediatrics, Baylor College of Medicine, Houston, TX, USA.ORCID http://orcid.org/0000-0002-2033-0758
Matthew L BakerCentre for Cell and Gene Therapy, Texas Children's Hospital, Houston Methodist Hospital, Baylor College of Medicine, Houston, TX, USA.
Liam HendrikseThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0002-0253-8512
Michelle LyThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Kaitlin KharasThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Polina BalinThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Xiaochong WuThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Lei QinThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Ning HuangThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Ana Guerreiro StucklinThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0003-3136-9241
A Sorana MorrissyDepartment of Biochemistry & Molecular Biology, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada.ORCID http://orcid.org/0000-0001-8526-562X
Florence M G CavalliThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Betty LuuThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Raul SuarezThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Pasqualino De AntonellisThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Antony MichealrajThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Avesta RastanThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Meenakshi HegdeTexas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.
Martin KomosaThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Olga SirbuThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Sachin A KumarThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0002-4853-3864
Zied AbdullaevLaboratory of Pathology, National Cancer Institute Centre for Cancer Research, Bethesda, MD, USA.
Claudia C FariaDivision of Neurosurgery, Centro Hospitalar Lisboa Norte, Hospital de Santa Maria, Lisbon, Portugal.
Stephen YipDepartment of Pathology and Laboratory Medicine, University of British Columbia, Vancouver, British Columbia, Canada.ORCID http://orcid.org/0000-0002-8514-9861
Juliette HukinDivision of Neurology, British Columbia Children's Hospital, Vancouver, British Columbia, Canada.ORCID http://orcid.org/0000-0002-8727-8456
Uri TaboriThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Cynthia HawkinsThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
Ken AldapeLaboratory of Pathology, National Cancer Institute Centre for Cancer Research, Bethesda, MD, USA.
Mads DaugaardDepartment of Urologic Sciences, University of British Columbia, Vancouver, British Columbia, Canada.ORCID http://orcid.org/0000-0001-8383-055X
John M MarisDivision of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Poul H SorensenDepartment of Molecular Oncology, British Columbia Cancer Research Centre, Vancouver, British Columbia, Canada.
Nabil AhmedTexas Children's Hospital, Baylor College of Medicine, Houston, TX, USA. nmahmed@texaschildrens.org.ORCID http://orcid.org/0000-0002-0982-8424
Michael D TaylorThe Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada. mdt.cns@gmail.com.ORCID http://orcid.org/0000-0001-7009-3466
Hospital for Sick Children · CAUniversity of Toronto · CAHouston Methodist · USBaylor College of Medicine · USNational Cancer Institute · USUniversity of British Columbia · CABC Children's Hospital · CACentro Hospitalar Lisboa Norte · PTChildren's Hospital of Philadelphia · USMcGill University Health Centre · CAUniversity of Calgary · CA
Funding
Understanding and Overcoming Resistance to Immunotherapies in Childhood CancersU54CA232568 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI MACKALL, CRYSTAL, MARIS, JOHN M · 2018 to 2022
$13.1MMouse models to discover new drivers of medulloblastomaR01CA159859 · NCI · SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE · PI Michael D. Taylor, WILLIAM A WEISS · 2011 to 2026
$10.4MGenomic Biomarkers of CancerZIABC011850 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI ALDAPE, KENNETH · 2019 to 2025
$5.1MMedulloblastoma and MetastasesR01CA148699 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI TAYLOR, MICHAEL D., WEISS, WILLIAM A · 2010 to 2014
$2.4MCIHRIntramural NIH HHS ZIA BC011850NCI NIH HHS R01 CA148699NCI NIH HHS R01 CA159859NCI NIH HHS U54 CA232568
Abstract
Recurrent medulloblastoma and ependymoma are universally lethal, with no approved targeted therapies and few candidates presently under clinical evaluation. Nearly all recurrent medulloblastomas and posterior fossa group A (PFA) ependymomas are located adjacent to and bathed by the cerebrospinal fluid, presenting an opportunity for locoregional therapy, bypassing the blood-brain barrier. We identify three cell-surface targets, EPHA2, HER2 and interleukin 13 receptor α2, expressed on medulloblastomas and ependymomas, but not expressed in the normal developing brain. We validate intrathecal delivery of EPHA2, HER2 and interleukin 13 receptor α2 chimeric antigen receptor T cells as an effective treatment for primary, metastatic and recurrent group 3 medulloblastoma and PFA ependymoma xenografts in mouse models. Finally, we demonstrate that administration of these chimeric antigen receptor T cells into the cerebrospinal fluid, alone or in combination with azacytidine, is a highly effective therapy for multiple metastatic mouse models of group 3 medulloblastoma and PFA ependymoma, thereby providing a rationale for clinical trials of these approaches in humans.
Indexed as
AnimalsBrain NeoplasmsCancer VaccinesCerebellar NeoplasmsCerebrospinal FluidChildChild, PreschoolDrug Delivery SystemsEpendymomaFemaleHEK293 CellsHumansImmunotherapy, AdoptiveInfantInjections, IntraventricularMaleCancer VaccinesReceptors, 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.