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

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What this work says

Outgoing claims: the papers this work cites, builds on, or argues with.

inferredcites →https://doi.org/10.1002/anie.201402133openalex-snapshot

Pharmacophore Reassignment for Induction of the Immunosurveillance Cytokine TRAIL

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Signal Transduction in Diffuse Intrinsic Pontine Glioma

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Improved protein–ligand docking using GOLD

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Development and validation of a genetic algorithm for flexible docking 1 1Edited by F. E. Cohen

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Treatment of recurrent diffuse intrinsic pontine glioma: the MD Anderson Cancer Center experience

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Clinico-radiologic characteristics of long-term survivors of diffuse intrinsic pontine glioma

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Diffuse intrinsic pontine glioma: a reassessment

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Reirradiation and PD-1 inhibition with nivolumab for the treatment of recurrent diffuse intrinsic pontine glioma: a single-institution experience

inferredcites →https://doi.org/10.1007/s11060-019-03271-3openalex-snapshot

Pediatric and adult H3 K27M-mutant diffuse midline glioma treated with the selective DRD2 antagonist ONC201

inferredcites →https://doi.org/10.1007/s11940-019-0577-yopenalex-snapshot

Diffuse Intrinsic Pontine Glioma: From Diagnosis to Next-Generation Clinical Trials

inferredcites →https://doi.org/10.1016/j.ccell.2017.03.011openalex-snapshot

Transcriptional Dependencies in Diffuse Intrinsic Pontine Glioma

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Mitochondrial ClpP-Mediated Proteolysis Induces Selective Cancer Cell Lethality

inferredcites →https://doi.org/10.1016/j.ejca.2016.12.007openalex-snapshot

Survival benefit for patients with diffuse intrinsic pontine glioma (DIPG) undergoing re-irradiation at first progression: A matched-cohort analysis on behalf of the SIOP-E-HGG/DIPG working group

inferredcites →https://doi.org/10.1016/s0016-5085(85)80133-5openalex-snapshot

Omeprazole: A Study of Its Inhibition of Gastric pH and Oral Pharmacokinetics After Morning or Evening Dosage

inferredcites →https://doi.org/10.1016/s1470-2045(21)00009-7openalex-snapshot

Diffuse intrinsic pontine glioma: a clinic in Mexico, social media, and unpublishable data

inferredcites →https://doi.org/10.1021/acschembio.9b00222openalex-snapshot

Mitochondrial Protease ClpP is a Target for the Anticancer Compounds ONC201 and Related Analogues

inferredcites →https://doi.org/10.1038/cdd.2009.59openalex-snapshot

Caspase activation pathways: some recent progress

inferredcites →https://doi.org/10.1038/s41375-020-0814-0openalex-snapshot

Shwachman–Bodian–Diamond syndrome (SBDS) protein is a direct inhibitor of protein phosphatase 2A (PP2A) activity and overexpressed in acute myeloid leukaemia

inferredcites →https://doi.org/10.1038/s41388-021-02102-yopenalex-snapshot

Pharmaco-proteogenomic profiling of pediatric diffuse midline glioma to inform future treatment strategies

inferredcites →https://doi.org/10.1038/s41467-019-12928-6openalex-snapshot

A Bayesian machine learning approach for drug target identification using diverse data types

inferredcites →https://doi.org/10.1038/s41467-020-16682-yopenalex-snapshot

The integrated genomic and epigenomic landscape of brainstem glioma

inferredcites →https://doi.org/10.1039/c6ob00556jopenalex-snapshot

Development of novel PP2A activators for use in the treatment of acute myeloid leukaemia

inferredcites →https://doi.org/10.1053/j.gastro.2008.03.006openalex-snapshot

Intragastric pH With Oral vs Intravenous Bolus Plus Infusion Proton-Pump Inhibitor Therapy in Patients With Bleeding Ulcers

inferredcites →https://doi.org/10.1073/pnas.1101657108openalex-snapshot

Hedgehog-responsive candidate cell of origin for diffuse intrinsic pontine glioma

inferredcites →https://doi.org/10.1073/pnas.91.6.2076openalex-snapshot

Convection-enhanced delivery of macromolecules in the brain.

inferredcites →https://doi.org/10.1080/03639045.2019.1698597openalex-snapshot

Development and optimization of levodopa and benzylhydrazine orally disintegrating tablets by direct compression and response surface methodology

inferredcites →https://doi.org/10.1091/mbc.01-05-0272openalex-snapshot

Activation and Caspase-mediated Inhibition of PARP: A Molecular Switch between Fibroblast Necrosis and Apoptosis in Death Receptor Signaling

inferredcites →https://doi.org/10.1093/neuonc/noz164openalex-snapshot

Biological activity of weekly ONC201 in adult recurrent glioblastoma patients

inferredcites →https://doi.org/10.1111/j.1365-2605.2011.01235.xopenalex-snapshot

Investigation of the expression and functional significance of the novel mouse sperm protein, a disintegrin and metalloprotease with thrombospondin type 1 motifs number 10 (ADAMTS10)

inferredcites →https://doi.org/10.1124/jpet.107.119560openalex-snapshot

Pharmacokinetics and Pharmacodynamics of Seven Opioids in P-Glycoprotein-Competent Mice: Assessment of Unbound Brain EC50,u and Correlation of in Vitro, Preclinical, and Clinical Data

inferredcites →https://doi.org/10.1126/scisignal.aac4380openalex-snapshot

ATF4 induction through an atypical integrated stress response to ONC201 triggers p53-independent apoptosis in hematological malignancies

inferredcites →https://doi.org/10.1126/scitranslmed.3004828openalex-snapshot

Dual Inactivation of Akt and ERK by TIC10 Signals Foxo3a Nuclear Translocation, TRAIL Gene Induction, and Potent Antitumor Effects

inferredcites →https://doi.org/10.1126/scitranslmed.aaw0064openalex-snapshot

Therapeutic strategies for diffuse midline glioma from high-throughput combination drug screening

inferredcites →https://doi.org/10.1136/bmjopen-2013-004587openalex-snapshot

Corticosteroids and risk of gastrointestinal bleeding: a systematic review and meta-analysis

inferredcites →https://doi.org/10.1186/s40425-019-0599-8openalex-snapshot

Safety and enhanced immunostimulatory activity of the DRD2 antagonist ONC201 in advanced solid tumor patients with weekly oral administration

inferredcites →https://doi.org/10.1200/jco.2017.75.9308openalex-snapshot

Clinical, Radiologic, Pathologic, and Molecular Characteristics of Long-Term Survivors of Diffuse Intrinsic Pontine Glioma (DIPG): A Collaborative Report From the International and European Society for Pediatric Oncology DIPG Registries

inferredcites →https://doi.org/10.1371/journal.pone.0174072openalex-snapshot

Real-time monitoring of human blood-brain barrier disruption

inferredcites →https://doi.org/10.18632/oncotarget.17837openalex-snapshot

A phase 2 study of the first imipridone ONC201, a selective DRD2 antagonist for oncology, administered every three weeks in recurrent glioblastoma

inferredcites →https://doi.org/10.18632/oncotarget.1801openalex-snapshot

Genome-wide shRNA screen revealed integrated mitogenic signaling between dopamine receptor D2 (DRD2) and epidermal growth factor receptor (EGFR) in glioblastoma

inferredcites →https://doi.org/10.18632/oncotarget.2890openalex-snapshot

The angular structure of ONC201, a TRAIL pathway-inducing compound, determines its potent anti-cancer activity

inferredcites →https://doi.org/10.3171/2019.2.peds18480openalex-snapshot

First clinical experience with DRD2/3 antagonist ONC201 in H3 K27M–mutant pediatric diffuse intrinsic pontine glioma: a case report

inferredcites →https://doi.org/10.3389/fonc.2015.00237openalex-snapshot

DIPG in Children – What Can We Learn from the Past?

inferredcites →https://doi.org/10.3389/fonc.2021.659800openalex-snapshot

World Cancer Day 2021 - Perspectives in Pediatric and Adult Neuro-Oncology

inferredcites →https://doi.org/10.5966/sctm.2015-0023openalex-snapshot

Intracarotid Infusion of Mesenchymal Stem Cells in an Animal Model of Parkinson's Disease, Focusing on Cell Distribution and Neuroprotective and Behavioral Effects

inferredcites →https://doi.org/10.7150/thno.50598openalex-snapshot

Correlation of multimodal 18F-DOPA PET and conventional MRI with treatment response and survival in children with diffuse intrinsic pontine gliomas

What points at this work

Incoming claims: where this work appears in other papers’ reference lists and arguments.

inferred← citeshttps://doi.org/10.2139/ssrn.4180012openalex-extracted

Real-Time Drug Testing of Pediatric Diffuse Midline Glioma to Support Clinical Decision Making: The Zurich DIPG/DMG Center Experience

inferred← citeshttps://doi.org/10.1002/cph4.70105openalex-snapshot

The Integrated Stress Response in Cancer: Paradox and Therapeutic Promise

inferred← citeshttps://doi.org/10.1002/pbc.30929openalex-snapshot

Hypofractionated re‐irradiation for diffuse intrinsic pontine glioma

inferred← citeshttps://doi.org/10.1007/978-3-031-62017-1_29openalex-snapshot

The Use of Complementary and Alternative Medicine in Pediatric Neuro-Oncology

inferred← citeshttps://doi.org/10.1007/s11060-023-04347-xopenalex-snapshot

Preliminary findings of German-sourced ONC201 treatment in H3K27 altered pediatric pontine diffuse midline gliomas

inferred← citeshttps://doi.org/10.1016/j.annonc.2023.06.012openalex-snapshot

An academia-driven compassionate use program for patients with recurrent H3K27-altered diffuse midline glioma: a new way to access innovation when clinical trials are lacking

inferred← citeshttps://doi.org/10.1016/j.biopha.2023.114630openalex-snapshot

Engineered extracellular vesicles (EVs): Promising diagnostic/therapeutic tools for pediatric high-grade glioma

inferred← citeshttps://doi.org/10.1016/j.ccell.2023.11.002openalex-snapshot

A road map for the treatment of pediatric diffuse midline glioma

inferred← citeshttps://doi.org/10.1016/j.ejca.2022.10.014openalex-snapshot

Real-time drug testing of paediatric diffuse midline glioma to support clinical decision making: The Zurich DIPG/DMG centre experience

inferred← citeshttps://doi.org/10.1016/j.ejca.2025.115230openalex-snapshot

Paediatric strategy forum for medicinal product development in diffuse midline gliomas in children and adolescents ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration

inferred← citeshttps://doi.org/10.1016/j.jconrel.2024.05.018openalex-snapshot

Current status and advances to improving drug delivery in diffuse intrinsic pontine glioma

inferred← citeshttps://doi.org/10.1016/j.lanwpc.2023.100739openalex-snapshot

National brain tumour registry: a new era of research collaboration with China

inferred← citeshttps://doi.org/10.1016/j.neo.2023.100899openalex-snapshot

Therapeutic avenues for targeting treatment challenges of diffuse midline gliomas

inferred← citeshttps://doi.org/10.1016/j.neo.2023.100909openalex-snapshot

Translational considerations for immunotherapy clinical trials in pediatric neuro-oncology

inferred← citeshttps://doi.org/10.1016/j.neo.2024.101015openalex-snapshot

ONC206 targeting ClpP induces mitochondrial dysfunction and protective autophagy in hepatocellular carcinoma cells

inferred← citeshttps://doi.org/10.1016/j.str.2022.12.002openalex-snapshot

Potent ClpP agonists with anticancer properties bind with improved structural complementarity and alter the mitochondrial N-terminome

inferred← citeshttps://doi.org/10.1016/s1470-2045(23)00070-0openalex-snapshot

Access to new drugs in paediatric oncology: can we learn from the ongoing ONC201 saga?

inferred← citeshttps://doi.org/10.1021/acs.jmedchem.6c00777openalex-snapshot

Dysregulation of Human ClpP Using Small Molecules with Piperazine-Based Scaffold for Diffuse Intrinsic Pontine Glioma Therapy Validated by Patient-Derived Tumor Organoids

inferred← citeshttps://doi.org/10.1038/s41388-025-03533-7openalex-snapshot

Multicellular tumor-stromal interactions recapitulate aspects of therapeutic response and human oncogenic signaling in a 3D disease model for H3K27M-altered DIPG

inferred← citeshttps://doi.org/10.1080/14728214.2024.2426649openalex-snapshot

ONC201 (Dordaviprone): review of evidence to date in diffuse midline glioma, hope or hype?

inferred← citeshttps://doi.org/10.1080/14728222.2023.2177531openalex-snapshot

H3K27-altered diffuse midline glioma: a paradigm shifting opportunity in direct delivery of targeted therapeutics

inferred← citeshttps://doi.org/10.1080/14728222.2023.2277232openalex-snapshot

Bench-to-bedside investigations of H3 K27-altered diffuse midline glioma: drug targets and potential pharmacotherapies

inferred← citeshttps://doi.org/10.1080/17568919.2025.2557175openalex-snapshot

Recent advances in the design of small molecules targeting human ClpP

inferred← citeshttps://doi.org/10.1093/neuonc/noac117openalex-snapshot

The intrinsic and microenvironmental features of diffuse midline glioma: Implications for the development of effective immunotherapeutic treatment strategies

inferred← citeshttps://doi.org/10.1093/neuonc/noad144openalex-snapshot

A review of current therapeutics targeting the mitochondrial protease ClpP in diffuse midline glioma, H3 K27-altered

inferred← citeshttps://doi.org/10.1093/neuonc/noad222openalex-snapshot

Chaetocin-mediated SUV39H1 inhibition targets stemness and oncogenic networks of diffuse midline gliomas and synergizes with ONC201

inferred← citeshttps://doi.org/10.1093/neuonc/noae066openalex-snapshot

The oncolytic adenovirus Delta-24-RGD in combination with ONC201 induces a potent antitumor response in pediatric high-grade and diffuse midline glioma models

inferred← citeshttps://doi.org/10.1093/neuonc/noaf007openalex-snapshot

Establishing a living biobank of pediatric high-grade glioma and ependymoma suitable for cancer pharmacology

inferred← citeshttps://doi.org/10.1093/neuonc/noag105openalex-snapshot

ClpP activation in high-risk medulloblastoma: ONC206 as a potent inducer of the integrated stress response and mitochondrial damage for a broader therapeutic window

inferred← citeshttps://doi.org/10.1093/neuonc/noag117openalex-snapshot

Point/Counterpoint: Dordaviprone for diffuse midline glioma - A landmark approval or a premature step?

inferred← citeshttps://doi.org/10.1093/neuonc/noag119openalex-snapshot

Single-nucleus profiling of postmortem diffuse midline gliomas identifies mitochondrial biogenesis as a resistance mechanism to imipridone therapy

inferred← citeshttps://doi.org/10.1093/noajnl/vdag074openalex-snapshot

Outcomes of H3K27M-altered diffuse midline glioma adult Chinese patients and real-world experience with German-sourced ONC201 targeted therapy: A multi-center study in Hong Kong

inferred← citeshttps://doi.org/10.1111/cns.14225openalex-snapshot

Radiotherapy and radio‐sensitization in H3K27M ‐mutated diffuse midline gliomas

inferred← citeshttps://doi.org/10.1158/0008-5472.can-23-0186openalex-snapshot

ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma

inferred← citeshttps://doi.org/10.1158/1541-7786.mcr-22-0567openalex-snapshot

B-cell Lymphoma 6 (BCL6): From Master Regulator of Humoral Immunity to Oncogenic Driver in Pediatric Cancers

inferred← citeshttps://doi.org/10.1158/2159-8290.cd-23-0131openalex-snapshot

Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

inferred← citeshttps://doi.org/10.1172/jci170329openalex-snapshot

PI3K/mTOR is a therapeutically targetable genetic dependency in diffuse intrinsic pontine glioma

inferred← citeshttps://doi.org/10.1177/03008916221099067openalex-snapshot

A homogeneous treatment for non-DIPG diffuse midline glioma

inferred← citeshttps://doi.org/10.1186/s41984-023-00242-9openalex-snapshot

Diffuse intrinsic pontine gliomas in pediatric patients: management updates

inferred← citeshttps://doi.org/10.21294/1814-4861-2023-22-3-108-118openalex-snapshot

Non-invasive methods of molecular diagnosis, clinical monitoring and approaches to the personalized therapy of diffuse midline glioma

inferred← citeshttps://doi.org/10.3389/fonc.2025.1606575openalex-snapshot

Case Report: Application of ex-vivo drug sensitivity testing to identify personalized treatment options for an adolescent with diffuse midline glioma

inferred← citeshttps://doi.org/10.3390/biomedicines10061311openalex-snapshot

The Intricate Epigenetic and Transcriptional Alterations in Pediatric High-Grade Gliomas: Targeting the Crosstalk as the Oncogenic Achilles’ Heel

inferred← citeshttps://doi.org/10.3390/brainsci13071106openalex-snapshot

Pediatric Central Nervous System Tumor Overview and Emerging Treatment Considerations

inferred← citeshttps://doi.org/10.3390/cancers17183062openalex-snapshot

Targeting the Mitochondria in High-Grade Gliomas

inferred← citeshttps://doi.org/10.3390/cells13131122openalex-snapshot

H3K27-Altered Diffuse Midline Glioma of the Brainstem: From Molecular Mechanisms to Targeted Interventions

inferred← citeshttps://doi.org/10.3390/jpm12050840openalex-snapshot

Diffuse Intrinsic Pontine Glioma: Molecular Landscape, Evolving Treatment Strategies and Emerging Clinical Trials

inferred← citeshttps://doi.org/10.3390/neuroglia4010003openalex-snapshot

ONC201 for Glioma Treatment: Adding an Important Weapon to Our Arsenal

inferred← citeshttps://doi.org/10.5306/wjco.v15.i4.482openalex-snapshot

Focus on current and emerging treatment options for glioma: A comprehensive review

inferred← citeshttps://doi.org/10.5694/mja2.52295openalex-snapshot

Management of patients with diffuse intrinsic pontine glioma in Australia and New Zealand: Australian and New Zealand Children's Haematology/Oncology Group position statement

inferred← citeshttps://doi.org/10.70675/be0bde8bzaab7z4b1fz8704zbcdc0c236054openalex-snapshot

Rôle et ciblage de HIF-2a dans les gliomes de haut-grade pédiatrique de la ligne médiane

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