Evidence map›Paper›PMID 32152626›Full record

ArticleNeuro-oncology2020

Phase I study of intraventricular infusions of autologous ex vivo expanded NK cells in children with recurrent medulloblastoma and ependymoma.

Soumen Khatua, Laurence J N Cooper, David I Sandberg, Leena Ketonen, Jason M Johnson, Michael E Rytting, Diane D Liu, Heather Meador, Prashant Trikha, Robin J Nakkula and 11 more

Abstract readClinical Trial, Phase I
In one paragraph

Article in Neuro-oncology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
62citing papers in PubMed, 2 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

62 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
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  3. Trial
  4. From developmental origins to relapse: molecular and therapeutic insights in medulloblastoma.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026
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  11. National multicentered retrospective review of salvage therapy for pediatric medulloblastoma: a Canadian experience.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2025
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2 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

21 authors.

Soumen KhatuaDepartment of Pediatrics, MD Anderson Cancer Center, Houston.
Laurence J N CooperZiopharm Oncology and MD Anderson Cancer Center, Houston.
David I SandbergDepartment of Neurosurgery, MD Anderson Cancer Center, Houston.
Leena KetonenDepartment of Diagnostic Imaging, MD Anderson Cancer Center, Houston.
Jason M JohnsonDepartment of Diagnostic Imaging, MD Anderson Cancer Center, Houston.
Michael E RyttingDepartment of Pediatrics, MD Anderson Cancer Center, Houston.
Diane D LiuDepartment of Biostatistics, University of Texas MD Anderson Cancer center.
Heather MeadorDepartment of Pediatrics, MD Anderson Cancer Center, Houston.
Prashant TrikhaDepartment of Hematology, Oncology and BMT, Nationwide Children's Hospital, Columbus, Ohio and Department of Hematology, The Ohio State University Comprehensive Cancer Center, Columbus, Ohio.
Robin J NakkulaDepartment of Hematology, Oncology and BMT, Nationwide Children's Hospital, Columbus, Ohio and Department of Hematology, The Ohio State University Comprehensive Cancer Center, Columbus, Ohio.
Gregory K BehbehaniDepartment of Hematology, Oncology and BMT, Nationwide Children's Hospital, Columbus, Ohio and Department of Hematology, The Ohio State University Comprehensive Cancer Center, Columbus, Ohio.
Dristhi RagoonananDepartment of Pediatrics, MD Anderson Cancer Center, Houston.
Sumit GuptaDepartment of Pediatrics, MD Anderson Cancer Center, Houston.
Aikaterini KotrotsouDepartment of Cancer Systems Imaging, MD Anderson Cancer Center, Houston.
Tagwa IdrisDepartment of Radiology, Harvard Medical School.
Elizabeth J ShpallDepartment of Stem Cell Transplantation and Cellular Therapy, MD Anderson Cancer Center, Houston.
Katy RezvaniDepartment of Stem Cell Transplantation and Cellular Therapy, MD Anderson Cancer Center, Houston.
Rivka ColenHillman Cancer Center, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania.
Wafik ZakyDepartment of Pediatrics, MD Anderson Cancer Center, Houston.
Dean A LeeDepartment of Hematology, Oncology and BMT, Nationwide Children's Hospital, Columbus, Ohio and Department of Hematology, The Ohio State University Comprehensive Cancer Center, Columbus, Ohio.
Vidya GopalakrishnanDepartment of Pediatrics, MD Anderson Cancer Center, Houston.

Funding

Tumor Evolution and Metastasis ProgramP30CA016672 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI Fabien Delerue · 1985 to 2026
$290.8M
NCI NIH HHS P30 CA016672
6 · The paper itself

Abstract

backgroundRecurrent pediatric medulloblastoma and ependymoma have a grim prognosis. We report a first-in-human, phase I study of intraventricular infusions of ex vivo expanded autologous natural killer (NK) cells in these tumors, with correlative studies.

methodsTwelve patients were enrolled, 9 received protocol therapy up to 3 infusions weekly, in escalating doses from 3 × 106 to 3 × 108 NK cells/m2/infusion, for up to 3 cycles. Cerebrospinal fluid (CSF) was obtained for cellular profile, persistence, and phenotypic analysis of NK cells. Radiomic characterization on pretreatment MRI scans was performed in 7 patients, to develop a non-invasive imaging-based signature.

resultsPrimary objectives of NK cell harvest, expansion, release, and safety of 112 intraventricular infusions of NK cells were achieved in all 9 patients. There were no dose-limiting toxicities. All patients showed progressive disease (PD), except 1 patient showed stable disease for one month at end of study follow-up. Another patient had transient radiographic response of the intraventricular tumor after 5 infusions of NK cell before progressing to PD. At higher dose levels, NK cells increased in the CSF during treatment with repetitive infusions (mean 11.6-fold). Frequent infusions of NK cells resulted in CSF pleocytosis. Radiomic signatures were profiled in 7 patients, evaluating ability to predict upfront radiographic changes, although they did not attain statistical significance.

conclusionsThis study demonstrated feasibility of production and safety of intraventricular infusions of autologous NK cells. These findings support further investigation of locoregional NK cell infusions in children with brain malignancies.

Indexed as

Brain NeoplasmsCerebellar NeoplasmsEpendymomaMedulloblastomaAdolescentChildFemaleHumansInfusions, IntraventricularKiller Cells, NaturalMaleNeoplasm Recurrence, Localimmunotherapyintraventricular infusionsnatural killer cellsrecurrent brain tumors

Identifiers

PMID32152626
PMCPMC7594549

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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.