Evidence map›Paper›PMID 28030841›Full record

ArticleOncotarget2017

Vamorolone, a dissociative steroidal compound, reduces pro-inflammatory cytokine expression in glioma cells and increases activity and survival in a murine model of cortical tumor.

Elizabeth Wells, Madhuri Kambhampati, Jesse M Damsker, Heather Gordish-Dressman, Sridevi Yadavilli, Oren J Becher, Jamila Gittens, Mojca Stampar, Roger J Packer, Javad Nazarian

2 registry-linked trialsOpen access · diamondAbstract readComparative Study
In one paragraph

Article in Oncotarget, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
1.4field-weighted citation impact, top 18% of its field
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.

NCT05166109 phase2completednot on this mapstarted 2022, after this paper: background citation

A Phase II Pilot Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy

TypeinterventionalSponsorReveraGen BioPharma, Inc.Ran2022 to 2025Enrolled46ConditionsBecker Muscular DystrophyArmsVamorolone, Placebo
NCT05185622 phase2completednot on this mapstarted 2022, after this paper: background citation

A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

TypeinterventionalSponsorSanthera PharmaceuticalsRan2022 to 2024Enrolled54ConditionsDuchenne Muscular DystrophyArmsVamorolone
3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 1 synthesis or guideline pooled it, 11 citations in OpenAlex.

  1. Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
    Pooled it
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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

10 authors at 4 institutions in 1 country.

Elizabeth WellsResearch Center for Genetic Medicine, Children's National Health System, Washington, DC, USA.
Madhuri KambhampatiResearch Center for Genetic Medicine, Children's National Health System, Washington, DC, USA.
Jesse M DamskerReveraGen BioPharma, Rockville, MD, USA.
Heather Gordish-DressmanResearch Center for Genetic Medicine, Children's National Health System, Washington, DC, USA.
Sridevi YadavilliResearch Center for Genetic Medicine, Children's National Health System, Washington, DC, USA.
Oren J BecherDuke University Medical Center, Durham, NC, USA.
Jamila GittensResearch Center for Genetic Medicine, Children's National Health System, Washington, DC, USA.
Mojca StamparResearch Center for Genetic Medicine, Children's National Health System, Washington, DC, USA.
Roger J PackerBrain Tumor Institute, Center for Neuroscience and Behavioral Medicine, Children's National Health System, Washington, DC, USA.
Javad NazarianResearch Center for Genetic Medicine, Children's National Health System, Washington, DC, USA.
Children's National · USDuke Medical Center · USGeorge Washington University · USReveraGen BioPharma (United States) · US

Funding

Research_ProjectU54HD090257 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI GUAY-WOODFORD, LISA MARIE · 2016 to 2020
$5.9M
NICHD NIH HHS U54 HD090257
6 · The paper itself

Abstract

Corticosteroids, such as dexamethasone, are routinely used as palliative care in neuro-oncology for their anti-inflammatory benefits, however many patients experience dose limiting side effects caused by glucocorticoid response element (GRE)-mediated transcription. The purpose of this study was to use a murine model to investigate a new steroid alternative, vamorolone, which promises to reduce side effects through dissociating GRE-mediated transcription and NF-κB -mediated anti-inflammatory actions. To compare vamorolone to dexamethasone in reducing pro-inflammatory signals in vitro, murine glioma cells were treated with dexamethasone, vamorolone or vehicle control. Changes in mRNA expression were assessed using the nanostring inflammatory platform. Furthermore, drug efficacy, post-treatment behavioral activity and side effects were assessed by treating two cohorts of brain tumor bearing mice with dexamethasone, vamorolone, or vehicle control. Our investigation showed that treatment with vamorolone resulted in a reduction of pro-inflammatory signals in tumor cells in vitro similar to treatment with dexamethasone. Treatment with vamorolone resulted in a better safety profile in comparison to dexamethasone treatment. Vamorolone- treated mice showed similar or better activity and survival when compared to dexamethasone-treated mice. Our data indicate vamorolone is a potential steroid-sparing alternative for treating patients with brain tumors.

Indexed as

AnimalsAnti-Inflammatory AgentsBrain NeoplasmsCell Line, TumorCytokinesDexamethasoneDown-RegulationGene Expression Regulation, NeoplasticGliomaInflammation MediatorsMice, Inbred NODMice, SCIDMotor ActivityNF-kappa BRNA, MessengerSignal TransductionAnti-Inflammatory AgentsCytokinesDexamethasoneInflammation MediatorsNF-kappa BRNA, MessengerSteroidsanti-inflammatorycytokinesglucocorticoidspediatric brain tumorspre-clinical testing

Identifiers

PMID28030841
PMCPMC5354737
OpenAlexW2561862500

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.