Evidence map›Paper›PMID 41458919›Full record

ArticleNeuro-oncology practice2025

Phase 1 study of mebendazole therapy for refractory/progressive or recurrent pediatric brain tumors.

Paul Phan, Stacie L Stapleton, Gregory J Riggins, Michael A Koldobskiy, Eric Raabe, Kenneth J Cohen

Abstract read
In one paragraph

Article in Neuro-oncology practice, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Review
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

6 authors.

Paul PhanJohns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Stacie L StapletonCancer and Blood Disorders Institute, Johns Hopkins All Children's Hospital, St. Petersburg, Florida, USA.
Gregory J RigginsDepartment of Neurosurgery, The Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Michael A KoldobskiyDepartment of Oncology, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA.
Eric RaabeDepartment of Oncology, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA.ORCID https://orcid.org/0000-0002-9368-7037
Kenneth J CohenDepartment of Oncology, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA.ORCID https://orcid.org/0000-0001-9193-9344

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Mebendazole (MBZ) is an anti-helminthic that has shown antitumor activity in mice with gliomas and subsequently in medulloblastoma models. Safety and tolerability have been demonstrated in adults with brain tumors but not explored in children as a monotherapy. We characterized the safety and maximum tolerated dose of oral MBZ in pediatric patients with refractory or progressive brain tumors and assessed progression-free survival (PFS) as a secondary objective. Methods: Patients up to 21 years of age with refractory or progressive brain tumors were enrolled at 2 centers in a 3 + 3 design with 3 doses of MBZ (1250, 1875, or 2500 mg/m Results: There were 17 patients enrolled between 2017 and 2022 with diffuse intrinsic pontine gliomas, high-grade astrocytomas, diffuse midline gliomas, glioblastoma multiform, ependymoma, and nonspecific gliomas. At all 3 dose levels, MBZ was well-tolerated with no dose-limiting toxicities. 121 adverse events (AE) including 69 AEs possibly/probably related to MBZ occurred-the most common being decreased lymphocyte count ( Conclusions: MBZ is safe and tolerable in treating refractory or progressive pediatric brain tumors, with doses up to 2500 mg/m

Indexed as

brain tumorsmebendazolepediatricphase 1

Identifiers

PMID41458919
PMCPMC12741831

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.