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

PNOC015: Repeated convection enhanced delivery (CED) of MTX110 (aqueous panobinostat) in children with newly diagnosed diffuse intrinsic pontine glioma (DIPG)

(2023) Sabine Mueller et al.   NEURO-ONCOLOGY

Fifty years of DIPG: looking at the future with hope

(2023) Umberto Tosi et al.   CHILDS NERVOUS SYSTEM

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

(2023) Bahattin Tanrıkulu et al.   JOURNAL OF NEURO-ONCOLOGY

GD2-CAR T cell therapy for H3K27M-mutated diffuse midline gliomas

(2022) Robbie G. Majzner et al.   NATURE

Imipridones affect tumor bioenergetics and promote cell lineage differentiation in diffuse midline gliomas

(2022) Justyna M Przystal et al.   NEURO-ONCOLOGY

Serial H3K27M cell-free tumor DNA (cf-tDNA) tracking predicts ONC201 treatment response and progression in diffuse midline glioma

(2022) Evan Cantor et al.   NEURO-ONCOLOGY

Drug delivery across the blood-brain barrier for the treatment of pediatric brain tumors – An update

(2022) Erica A. Power et al.   ADVANCED DRUG DELIVERY REVIEWS

STAT3 is a biologically relevant therapeutic target in H3K27M-mutant diffuse midline glioma

(2022) Liang Zhang et al.   NEURO-ONCOLOGY

Short and Long-Term Toxicity in Pediatric Cancer Treatment: Central Nervous System Damage

(2022) Iside Alessi et al.   Cancers

IL-13Rα2 Status Predicts GB-13 (IL13.E13K-PE4E) Efficacy in High-Grade Glioma

(2022) Julian S. Rechberger et al.   Pharmaceutics

Murine CNS and bone marrow distribution of the aurora A kinase inhibitor alisertib: pharmacokinetics and exposure at the sites of efficacy and toxicity

(2022) Ju-Hee Oh et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

CBTRUS Statistical Report: Pediatric Brain Tumor Foundation Childhood and Adolescent Primary Brain and Other Central Nervous System Tumors Diagnosed in the United States in 2014–2018

(2022) Quinn T Ostrom et al.   NEURO-ONCOLOGY

CBTRUS Statistical Report: Primary Brain and Other Central Nervous System Tumors Diagnosed in the United States in 2015–2019

(2022) Quinn T Ostrom et al.   NEURO-ONCOLOGY

Intraventricular B7-H3 CAR T cells for diffuse intrinsic pontine glioma: preliminary first-in-human bioactivity and safety

(2022) Nicholas A. Vitanza et al.   Cancer Discovery

The landscape of tumor cell states and spatial organization in H3-K27M mutant diffuse midline glioma across age and location

(2022) Ilon Liu et al.   NATURE GENETICS

Cancer Statistics, 2021

(2021) Rebecca L. Siegel et al.   CA-A CANCER JOURNAL FOR CLINICIANS

Complex Factors and Challenges that Affect the Pharmacology, Safety and Efficacy of Nanocarrier Drug Delivery Systems

(2021) Joseph A. Piscatelli et al.   Pharmaceutics

The emerging role of nanotechnology in pursuit of successful drug delivery to H3K27M diffuse midline gliomas

(2021) Erica A Power et al.   Nanomedicine

CAR-T cell therapy: current limitations and potential strategies

(2021) Robert C. Sterner et al.   Blood Cancer Journal

Intraarterial delivery of bevacizumab and cetuximab utilizing blood-brain barrier disruption in children with high-grade glioma and diffuse intrinsic pontine glioma: results of a phase I trial

(2021) Heather J. McCrea et al.   Journal of Neurosurgery-Pediatrics

The 2021 WHO Classification of Tumors of the Central Nervous System: a summary

(2021) David N Louis et al.   NEURO-ONCOLOGY

Phase I/II trial of vorinostat and radiation and maintenance vorinostat in children with diffuse intrinsic pontine glioma: A Children’s Oncology Group report

(2021) Jack M Su et al.   NEURO-ONCOLOGY

IMMU-11. CLINICAL UPDATES AND CORRELATIVE FINDINGS FROM THE FIRST PATIENT WITH DIPG TREATED WITH INTRACRANIAL CAR T CELLS

(2021) Nicholas Vitanza et al.   NEURO-ONCOLOGY

Efflux Limits Tumor Drug Delivery Despite Disrupted BBB

(2021) Jessica I. Griffith et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Repurposing vandetanib plus everolimus for the treatment of ACVR1-mutant diffuse intrinsic pontine glioma

(2021) Diana M Carvalho et al.   Cancer Discovery

Dual IGF1R/IR inhibitors in combination with GD2-CAR T-cells display a potent anti-tumor activity in diffuse midline glioma H3K27M-mutant

(2021) Emmanuel de Billy et al.   NEURO-ONCOLOGY

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

(2021) Izac J. Findlay et al.   ONCOGENE

Intrathecal drug delivery in the era of nanomedicine

(2020) M. Fowler et al.   ADVANCED DRUG DELIVERY REVIEWS

MRI Patterns of Extrapontine Lesion Extension in Diffuse Intrinsic Pontine Gliomas

(2020) L. Makepeace et al.   AMERICAN JOURNAL OF NEURORADIOLOGY

Progress in diffuse intrinsic pontine glioma: advocating for stereotactic biopsy in the standard of care

(2020) John R. Williams et al.   Neurosurgical Focus

Clinical efficacy of ONC201 in thalamic H3 K27M-mutant glioma.

(2020) Abed Rahman Kawakibi et al.   JOURNAL OF CLINICAL ONCOLOGY

Diffuse Intrinsic Pontine Glioma (DIPG): breakthrough and clinical perspective

(2020) Maria Grazia Perrone et al.   CURRENT MEDICINAL CHEMISTRY

PET, image-guided HDAC inhibition of pediatric diffuse midline glioma improves survival in murine models

(2020) Umberto Tosi et al.   Science Advances

Everolimus improves the efficacy of dasatinib in PDGFRα-driven glioma

(2020) Zachary Miklja et al.   JOURNAL OF CLINICAL INVESTIGATION

A phase I/II study of ribociclib following radiation therapy in children with newly diagnosed diffuse intrinsic pontine glioma (DIPG)

(2020) Mariko DeWire et al.   JOURNAL OF NEURO-ONCOLOGY

Repeat convection-enhanced delivery for diffuse intrinsic pontine glioma

(2020) Evan D. Bander et al.   Journal of Neurosurgery-Pediatrics

Patient-derived orthotopic xenograft models of medulloblastoma lack a functional blood-brain barrier

(2020) Laura A Genovesi et al.   NEURO-ONCOLOGY

Addressing BBB Heterogeneity: A New Paradigm for Drug Delivery to Brain Tumors

(2020) Jessica I. Griffith et al.   Pharmaceutics

The application of nanoparticles in cancer immunotherapy: Targeting tumor microenvironment

(2020) Muyue Yang et al.   Bioactive Materials

Genetic labeling reveals temporal and spatial expression pattern of D2 dopamine receptor in rat forebrain

(2019) Qing Yu et al.   Brain Structure & Function

Treatment Strategies in Diffuse Midline Gliomas With the H3K27M Mutation: The Role of Convection-Enhanced Delivery in Overcoming Anatomic Challenges

(2019) Benjamin T. Himes et al.   Frontiers in Oncology

Phase I trial of convection-enhanced delivery of IL13-Pseudomonas toxin in children with diffuse intrinsic pontine glioma

(2019) John D. Heiss et al.   Journal of Neurosurgery-Pediatrics

Nimotuzumab and radiotherapy for treatment of newly diagnosed diffuse intrinsic pontine glioma (DIPG): a phase III clinical study

(2019) G. Fleischhack et al.   JOURNAL OF NEURO-ONCOLOGY

Reirradiation for diffuse intrinsic pontine glioma: a systematic review and meta-analysis

(2019) Victor M. Lu et al.   CHILDS NERVOUS SYSTEM

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

(2019) Matthew D. Hall et al.   Journal of Neurosurgery-Pediatrics

Radiosensitization by Histone H3 Demethylase Inhibition in Diffuse Intrinsic Pontine Glioma

(2019) Hiroaki Katagi et al.   CLINICAL CANCER RESEARCH

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

(2019) Nicholas A. Vitanza et al.   Current Treatment Options in Neurology

Brainstem biopsy in pediatric diffuse intrinsic pontine glioma in the era of precision medicine: the INFORM study experience

(2019) Elke Pfaff et al.   EUROPEAN JOURNAL OF CANCER

PFA ependymoma-associated protein EZHIP inhibits PRC2 activity through a H3 K27M-like mechanism

(2019) Siddhant U. Jain et al.   Nature Communications

Clinical trials for diffuse intrinsic pontine glioma: the current state of affairs

(2019) Julian S. Rechberger et al.   CHILDS NERVOUS SYSTEM

Towards Immunotherapy for Pediatric Brain Tumors

(2019) Stacie Shiqi Wang et al.   TRENDS IN IMMUNOLOGY

Defining optimal target volumes of conformal radiation therapy for diffuse intrinsic pontine glioma

(2019) Christopher L. Tinkle et al.   INTERNATIONAL JOURNAL OF RADIATION ONCOLOGY BIOLOGY PHYSICS

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

(2019) Andrew S. Chi et al.   JOURNAL OF NEURO-ONCOLOGY

Epigenetic reprogramming and chromatin accessibility in pediatric diffuse intrinsic pontine gliomas: a neural developmental disease

(2019) Flor M Mendez et al.   NEURO-ONCOLOGY

Hypofractionated radiotherapy in children with diffuse intrinsic pontine glioma

(2019) Akiko Hayashi et al.   PEDIATRICS INTERNATIONAL

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

(2019) Grant L. Lin et al.   Science Translational Medicine

Diffuse intrinsic pontine gliomas: Diagnostic approach and treatment strategies

(2019) M. Burhan Janjua et al.   JOURNAL OF CLINICAL NEUROSCIENCE

The intersect of neurosurgery with diffuse intrinsic pontine glioma

(2019) Claudia M. Kuzan-Fischer et al.   Journal of Neurosurgery-Pediatrics

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

(2018) Lindsey M. Hoffman et al.   JOURNAL OF CLINICAL ONCOLOGY

The distribution, clearance, and brainstem toxicity of panobinostat administered by convection-enhanced delivery

(2018) William G. B. Singleton et al.   Journal of Neurosurgery-Pediatrics

Convection-enhanced delivery for diffuse intrinsic pontine glioma: a single-centre, dose-escalation, phase 1 trial

(2018) Mark M Souweidane et al.   LANCET ONCOLOGY

Efficacy of the MDM2 inhibitor SAR405838 in glioblastoma is limited by poor distribution across the blood-brain barrier

(2018) Minjee Kim et al.   MOLECULAR CANCER THERAPEUTICS

Potent antitumor efficacy of anti-GD2 CAR T cells in H3-K27M+ diffuse midline gliomas

(2018) Christopher W. Mount et al.   NATURE MEDICINE

Prospective Feasibility and Safety Assessment of Surgical Biopsy for Patients with Newly Diagnosed Diffuse Intrinsic Pontine Glioma

(2018) Nalin Gupta et al.   NEURO-ONCOLOGY

Phase 1 trial, pharmacokinetics, and pharmacodynamics of dasatinib combined with crizotinib in children with recurrent or progressive high-grade and diffuse intrinsic pontine glioma

(2018) Alberto Broniscer et al.   PEDIATRIC BLOOD & CANCER

H3.3K27M mutant proteins reprogram epigenome by sequestering the PRC2 complex to poised enhancers

(2018) Dong Fang et al.   eLife

CDK4/6 and PDGFRA Signaling as Therapeutic Targets in Diffuse Intrinsic Pontine Glioma

(2018) Christine Hoeman et al.   Frontiers in Oncology

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

(2018) Cassie Kline et al.   JOURNAL OF NEURO-ONCOLOGY

Histone H3.3 K27M Accelerates Spontaneous Brainstem Glioma and Drives Restricted Changes in Bivalent Gene Expression

(2018) Jon D. Larson et al.   CANCER CELL

Nimotuzumab-containing regimen for pediatric diffuse intrinsic pontine gliomas: a retrospective multicenter study and review of the literature

(2018) Rejin Kebudi et al.   CHILDS NERVOUS SYSTEM

Integrated mapping of pharmacokinetics and pharmacodynamics in a patient-derived xenograft model of glioblastoma

(2018) Elizabeth C. Randall et al.   Nature Communications

Convection enhanced delivery of panobinostat (LBH589)-loaded pluronic nano-micelles prolongs survival in the F98 rat glioma model

(2017) WG Singleton et al.   International Journal of Nanomedicine

Novel and shared neoantigen derived from histone 3 variant H3.3K27M mutation for glioma T cell therapy

(2017) Zinal S. Chheda et al.   JOURNAL OF EXPERIMENTAL MEDICINE

EZH2 is a potential therapeutic target for H3K27M-mutant pediatric gliomas

(2017) Faizaan Mohammad et al.   NATURE MEDICINE

Combination of EZH2 inhibitor and BET inhibitor for treatment of diffuse intrinsic pontine glioma

(2017) Yaqin Zhang et al.   Cell and Bioscience

K27M-mutant histone-3 as a novel target for glioma immunotherapy

(2017) Katharina Ochs et al.   OncoImmunology

Pre-Clinical Study of Panobinostat in Xenograft and Genetically Engineered Murine Diffuse Intrinsic Pontine Glioma Models

(2017) Tammy Hennika et al.   PLoS One

The 2016 World Health Organization Classification of Tumors of the Central Nervous System: a summary

(2016) David N. Louis et al.   ACTA NEUROPATHOLOGICA

Nanotechnology Applications for Diffuse Intrinsic Pontine Glioma

(2016) Amy Bredlau et al.   Current Neuropharmacology

ABCG2 and ABCB1 Limit the Efficacy of Dasatinib in a PDGF-B-Driven Brainstem Glioma Model

(2016) R. K. Mittapalli et al.   MOLECULAR CANCER THERAPEUTICS

Lowered H3K27me3 and DNA hypomethylation define poorly prognostic pediatric posterior fossa ependymomas

(2016) J. Bayliss et al.   Science Translational Medicine

Structural basis of oncogenic histone H3K27M inhibition of human polycomb repressive complex 2

(2016) Neil Justin et al.   Nature Communications

Toxicity and management in CAR T-cell therapy

(2016) Challice L Bonifant et al.   Molecular Therapy-Oncolytics

Histone H3F3A and HIST1H3B K27M mutations define two subgroups of diffuse intrinsic pontine gliomas with different prognosis and phenotypes

(2015) David Castel et al.   ACTA NEUROPATHOLOGICA

Biopsy in a series of 130 pediatric diffuse intrinsic Pontine gliomas

(2015) Stephanie Puget et al.   CHILDS NERVOUS SYSTEM

A pilot study of bevacizumab-based therapy in patients with newly diagnosed high-grade gliomas and diffuse intrinsic pontine gliomas

(2015) Trent R. Hummel et al.   JOURNAL OF NEURO-ONCOLOGY

Functionally defined therapeutic targets in diffuse intrinsic pontine glioma

(2015) Catherine S Grasso et al.   NATURE MEDICINE

Genomic analysis of diffuse intrinsic pontine gliomas identifies three molecular subgroups and recurrent activating ACVR1 mutations

(2014) Pawel Buczkowicz et al.   NATURE GENETICS

Recurrent activating ACVR1 mutations in diffuse intrinsic pontine glioma

(2014) Kathryn R Taylor et al.   NATURE GENETICS

Pharmacologic inhibition of histone demethylation as a therapy for pediatric brainstem glioma

(2014) Rintaro Hashizume et al.   NATURE MEDICINE

Phase 2 study of safety and efficacy of nimotuzumab in pediatric patients with progressive diffuse intrinsic pontine glioma

(2014) U. Bartels et al.   NEURO-ONCOLOGY

Preclinical evaluation of dasatinib alone and in combination with cabozantinib for the treatment of diffuse intrinsic pontine glioma

(2014) Nathalène Truffaux et al.   NEURO-ONCOLOGY

Histone H3 lysine-to-methionine mutants as a paradigm to study chromatin signaling

(2014) H.-M. Herz et al.   SCIENCE

Reduced H3K27me3 and DNA Hypomethylation Are Major Drivers of Gene Expression in K27M Mutant Pediatric High-Grade Gliomas

(2013) Sebastian Bender et al.   CANCER CELL

Novel Oncogenic PDGFRA Mutations in Pediatric High-Grade Gliomas

(2013) B. S. Paugh et al.   CANCER RESEARCH

Phase I Trial, Pharmacokinetics, and Pharmacodynamics of Vandetanib and Dasatinib in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma

(2013) A. Broniscer et al.   CLINICAL CANCER RESEARCH

Brainstem Glioma: A Review

(2013) Sean A. Grimm et al.   Current Neurology and Neuroscience Reports

The histone H3.3K27M mutation in pediatric glioma reprograms H3K27 methylation and gene expression

(2013) K.-M. Chan et al.   GENES & DEVELOPMENT

Inhibition of PRC2 Activity by a Gain-of-Function H3 Mutation Found in Pediatric Glioblastoma

(2013) P. W. Lewis et al.   SCIENCE

K27M mutation in histone H3.3 defines clinically and biologically distinct subgroups of pediatric diffuse intrinsic pontine gliomas

(2012) Dong-Anh Khuong-Quang et al.   ACTA NEUROPATHOLOGICA

Breast Cancer Resistance Protein and P-Glycoprotein in Brain Cancer: Two Gatekeepers Team Up

(2012) Sagar Agarwal et al.   CURRENT PHARMACEUTICAL DESIGN

Co-administration strategy to enhance brain accumulation of vandetanib by modulating P-glycoprotein (P-gp/Abcb1) and breast cancer resistance protein (Bcrp1/Abcg2) mediated efflux with m-TOR inhibitors

(2012) Mukul Minocha et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

B7-H3, a potential therapeutic target, is expressed in diffuse intrinsic pontine glioma

(2012) Zhiping Zhou et al.   JOURNAL OF NEURO-ONCOLOGY

Active Efflux of Dasatinib from the Brain Limits Efficacy against Murine Glioblastoma: Broad Implications for the Clinical Use of Molecularly Targeted Agents

(2012) S. Agarwal et al.   MOLECULAR CANCER THERAPEUTICS

Driver mutations in histone H3.3 and chromatin remodelling genes in paediatric glioblastoma

(2012) Jeremy Schwartzentruber et al.   NATURE

Somatic histone H3 alterations in pediatric diffuse intrinsic pontine gliomas and non-brainstem glioblastomas

(2012) Gang Wu et al.   NATURE GENETICS

Diffuse intrinsic pontine glioma: poised for progress

(2012) Katherine E. Warren   Frontiers in Oncology

Diffuse intrinsic pontine gliomas: A systematic update on clinical trials and biology

(2011) M.H.A. Jansen et al.   CANCER TREATMENT REVIEWS

Bevacizumab in recurrent high-grade pediatric gliomas

(2010) A. Narayana et al.   NEURO-ONCOLOGY