Evidence map›Paper›PMID 34988452›Full record

ArticleNeuro-oncology advances

Preclinical and clinical evaluation of German-sourced ONC201 for the treatment of H3K27M-mutant diffuse intrinsic pontine glioma.

Ryan J Duchatel, Abdul Mannan, Ameha S Woldu, Tom Hawtrey, Phoebe A Hindley, Alicia M Douglas, Evangeline R Jackson, Izac J Findlay, Zacary P Germon, Dilana Staudt and 17 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Neuro-oncology advances. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.

0numbers the graph read from it
0cells of the map it votes in
34citing papers in PubMed
5.0field-weighted citation impact, top 4% 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.

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

34 citing papers in PubMed, 45 citations in OpenAlex.

  1. Article
  2. Review
  3. Targeted therapies in pediatric oncology: A start.Molecular therapy. Oncology · 2026
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  8. Article
  9. Case Report: Application ofFrontiers in oncology · 2025
    Article
  10. Article
  11. Article
  12. Review
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  14. Article
  15. Review
  16. Article
  17. Article
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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

27 authors at 11 institutions in 6 countries.

Ryan J DuchatelCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Abdul MannanCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Ameha S WolduCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Tom HawtreySchool of Chemistry, University of New South Wales, Sydney, New South Wales, Australia.
Phoebe A HindleyCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Alicia M DouglasCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Evangeline R JacksonCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Izac J FindlayCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Zacary P GermonCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Dilana StaudtCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Padraic S KearneyCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Nathan D SmithAnalytical and Biomolecular Research Facility, Advanced Mass Spectrometry Unit, University of Newcastle, Callaghan, New South Wales, Australia.
Kate E HindleyCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
Jason E CainHudson Institute of Medical Research, Clayton, Victoria, Australia.
Nicolas AndréDepartment of Pediatric Oncology, La Timone Children's Hospital, AP-HM, Marseille, France.
Andres Morales La MadridLaboratory of Developmental Cancer, Institut de Recerca Sant Joan de Déu, Barcelona, Spain.
Brett NixonReproductive Science Group, College of Engineering, Science and Environment, University of Newcastle, Callaghan, New South Wales, Australia.ORCID https://orcid.org/0000-0003-2745-8188
Geoffry N De IuliisReproductive Science Group, College of Engineering, Science and Environment, University of Newcastle, Callaghan, New South Wales, Australia.
Javad NazarianChildren's National Medical Center, Washington, District of Columbia., USA.ORCID https://orcid.org/0000-0002-1951-9828
Kathleen IrishHunter Medical Research Institute, New Lambton Heights, New South Wales, Australia.
Frank AlvaroCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.
David D EisenstatChildren's Cancer Centre, The Royal Children's Hospital Melbourne, Parkville, Victoria, Australia.
Alexander BeckCenter for Neuropathology, Ludwig Maximilian University of Munich, Munich, Germany.
Nicholas A VitanzaBen Towne Center for Childhood Cancer Research, Seattle Children's Research Institute, Seattle, Washington, USA.
Sabine MuellerUniversity Children's Hospital Zurich, Zurich, Switzerland.ORCID https://orcid.org/0000-0002-3452-5150
Jonathan C MorrisSchool of Chemistry, University of New South Wales, Sydney, New South Wales, Australia.
Matthew D DunCancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine and Wellbeing, University of Newcastle, Callaghan, New South Wales, Australia.ORCID https://orcid.org/0000-0002-9063-5370
Hunter Medical Research Institute · AUUniversity of Newcastle Australia · AUUNSW Sydney · AUChildren's National · USHospital Sant Joan de Déu Barcelona · ESHudson Institute of Medical Research · AUInserm · FRLudwig-Maximilians-Universität München · DERoyal Children's Hospital · AUSeattle Children's Hospital · USUniversity of California, San Francisco · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundDiffuse intrinsic pontine glioma (DIPG) is a fatal childhood brainstem tumor for which radiation is the only treatment. Case studies report a clinical response to ONC201 for patients with H3K27M-mutant gliomas. Oncoceutics (ONC201) is only available in the United States and Japan; however, in Germany, DIPG patients can be prescribed and dispensed a locally produced compound-ONC201 German-sourced ONC201 (GsONC201). Pediatric oncologists face the dilemma of supporting the administration of GsONC201 as conjecture surrounds its authenticity. Therefore, we compared GsONC201 to original ONC201 manufactured by Oncoceutics Inc.

methodsAuthenticity of GsONC201 was determined by high-resolution mass spectrometry and nuclear magnetic resonance spectroscopy. Biological activity was shown via assessment of on-target effects,

resultsGsONC201 harbored the authentic structure, however, was formulated as a free base rather than the dihydrochloride salt used in clinical trials. GsONC201

conclusionsThis study confirms the biological activity of GsONC201 and documents the OS of patients who received the drug; however, GsONC201 was never used as a monotherapy.

Indexed as

diffuse intrinsicdiffuse midline gliomaONC201pontine glioma

Identifiers

PMID34988452
PMCPMC8709907
OpenAlexW4206071669

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.