Ron Firestein
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View article: Supplementary Data from ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma
Supplementary Data from ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma Open
All Supplementary Tables
View article: EPCO-16. CHROMATIN REMODELING WITH COMBINED FACT AND BET INHIBITION DISRUPTS ONCOGENIC TRANSCRIPTION IN DIFFUSE MIDLINE GLIOMA
EPCO-16. CHROMATIN REMODELING WITH COMBINED FACT AND BET INHIBITION DISRUPTS ONCOGENIC TRANSCRIPTION IN DIFFUSE MIDLINE GLIOMA Open
Aberrant epigenetic regulation is a hallmark of Diffuse Midline Glioma (DMG), an incurable pediatric brain tumor. The H3K27M driver histone mutation leads to transcriptional dysregulation, indicating that targeting the epigenome and transc…
View article: EPCO-06. ESTABLISHING THE EPIGENETIC LANDSCAPE OF THE UNEXPLORED EZHIP-EXPRESSING DIFFUSE MIDLINE GLIOMA SUBTYPE
EPCO-06. ESTABLISHING THE EPIGENETIC LANDSCAPE OF THE UNEXPLORED EZHIP-EXPRESSING DIFFUSE MIDLINE GLIOMA SUBTYPE Open
INTRODUCTION Diffuse midline glioma (DMG) is a terminal paediatric brain cancer which has no current cure, leading to an urgent demand to re-evaluate therapeutic strategies. DMG is an ‘epigenetically driven’ cancer, as most patients harbor…
View article: Resource: A compendium of HLA types and expression in pediatric cancer models
Resource: A compendium of HLA types and expression in pediatric cancer models Open
Cancer immunotherapy has revolutionized treatment by leveraging the immune system to recognize and destroy tumor cells, offering a promising, less toxic option for pediatric patients. A key component of this response is antigen presentatio…
View article: The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science
The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science Open
View article: Model systems and unique biological features of high and low-grade colorectal cancer (CRC) revealed by xenografting 84 human CRC cell lines
Model systems and unique biological features of high and low-grade colorectal cancer (CRC) revealed by xenografting 84 human CRC cell lines Open
View article: Integrative Multi-Omics Analysis Identifies Nuclear Factor I as a Key Driver of Dysregulated Purine Metabolism in DIPG
Integrative Multi-Omics Analysis Identifies Nuclear Factor I as a Key Driver of Dysregulated Purine Metabolism in DIPG Open
Diffuse intrinsic pontine glioma (DIPG) is a devastating brainstem cancer in children, with a median survival of under one year and limited treatment options. Over 80% of DIPGs possess a H3K27M mutation. To identify metabolic vulnerabiliti…
View article: H3K27M Mutation Reshapes the Antigenic Landscape in Diffuse Midline Glioma
H3K27M Mutation Reshapes the Antigenic Landscape in Diffuse Midline Glioma Open
Simple Summary Diffuse midline gliomas (DMGs) are aggressive childhood brain tumours with no effective treatments. Over 80% of cases carry the histone H3K27M mutation, which alters chromatin structure and gene regulation and induce tumours…
View article: Mediator kinase inhibition drives myometrial stem cell differentiation and the uterine fibroid phenotype through super-enhancer reprogramming
Mediator kinase inhibition drives myometrial stem cell differentiation and the uterine fibroid phenotype through super-enhancer reprogramming Open
View article: Multidimensional, integrative profiling identifies BCL2L1 methylation as a predictor of MCL1 dependency in pediatric malignancies
Multidimensional, integrative profiling identifies BCL2L1 methylation as a predictor of MCL1 dependency in pediatric malignancies Open
Pediatric high-grade gliomas (pHGGs) are the most aggressive brain tumors in children, necessitating innovative therapies to improve outcomes. Unlike adult gliomas, recent research reveals that childhood gliomas have distinct biological fe…
View article: Mediator kinase inhibition drives myometrial stem cell differentiation and the uterine fibroid phenotype through super-enhancer reprogramming
Mediator kinase inhibition drives myometrial stem cell differentiation and the uterine fibroid phenotype through super-enhancer reprogramming Open
View article: Supplementary Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma
Supplementary Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma Open
All Supplementary Tables
View article: Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma
Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma Open
Diffuse midline gliomas (DMG), including diffuse intrinsic pontine gliomas (DIPGs), are the most lethal of childhood cancers. Palliative radiotherapy is the only established treatment, with median patient survival of 9-11 months. ONC201 is…
View article: Supplementary Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma
Supplementary Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma Open
All Supplementary Figures and their captions.
View article: Supplementary Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma
Supplementary Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma Open
All Supplementary Tables
View article: Supplementary Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma
Supplementary Data from ONC201 in combination with paxalisib for the treatment of H3K27-altered diffuse midline glioma Open
All Supplementary Figures and their captions.
View article: Emerging and Biological Concepts in Pediatric High-Grade Gliomas
Emerging and Biological Concepts in Pediatric High-Grade Gliomas Open
Primary central nervous system tumors are the most frequent solid tumors in children, accounting for over 40% of all childhood brain tumor deaths, specifically high-grade gliomas. Compared with pediatric low-grade gliomas (pLGGs), pediatri…
View article: HGG-23. MAPPING GENETIC DEPENDENCIES IN PAEDIATRIC HIGH GRADE GLIOMAS AT GENOME-SCALE
HGG-23. MAPPING GENETIC DEPENDENCIES IN PAEDIATRIC HIGH GRADE GLIOMAS AT GENOME-SCALE Open
BACKGROUND Pediatric high-grade gliomas (HGGs) present a therapeutic challenge due to their aggressive behavior and limited treatment options. Traditional treatments often fall short in addressing the genetic complexity and aggressiveness …
View article: DIPG-22. A NOVEL ROLE FOR ACVR1/ALK2 IN REGULATING CHOLESTEROL BIOSYNTHESIS PROVIDES A NEW COMBINATORIAL THERAPEUTIC APPROACH FOR PATIENTS WITH DMG
DIPG-22. A NOVEL ROLE FOR ACVR1/ALK2 IN REGULATING CHOLESTEROL BIOSYNTHESIS PROVIDES A NEW COMBINATORIAL THERAPEUTIC APPROACH FOR PATIENTS WITH DMG Open
BACKGROUND The discovery of somatic mutations in ACVR1, which encodes the serine/threonine kinase ALK2, in 20-25% of diffuse midline glioma H3K27-altered (DMG-H3K27) patients has led to the development of numerous chemotypes of ALK2 inhibi…
View article: CNSC-03. LINKING CELL FATE DETERMINATION IN THE EMBRYONIC FOREBRAIN TO PEDIATRIC HIGH-GRADE GLIOMA
CNSC-03. LINKING CELL FATE DETERMINATION IN THE EMBRYONIC FOREBRAIN TO PEDIATRIC HIGH-GRADE GLIOMA Open
BACKGROUND Pediatric high-grade gliomas are treatment resistant, usually fatal cancers encompassing diffuse midline glioma, K27-altered (DMG), and diffuse hemispheric glioma, G34R/V (DHG). Most patients harbour canonical and non-canonical …
View article: HGG-53. MULTI-DIMENSIONAL INTEGRATIVE PROFILING IDENTIFIES BCL2L1 METHYLATION AS A PREDICTIVE BIOMARKER FOR MCL-1 ACTIVITY IN PEDIATRIC HIGH-GRADE GLIOMAS
HGG-53. MULTI-DIMENSIONAL INTEGRATIVE PROFILING IDENTIFIES BCL2L1 METHYLATION AS A PREDICTIVE BIOMARKER FOR MCL-1 ACTIVITY IN PEDIATRIC HIGH-GRADE GLIOMAS Open
BACKGROUND Pediatric high-grade gliomas (pHGGs) pose a significant challenge as the most aggressive central nervous system tumors in children. The lack of effective treatment and poor survival rates emphasize the critical need for innovati…
View article: The Landscape of Pediatric High-Grade Gliomas: The Virtues and Pitfalls of Pre-Clinical Models
The Landscape of Pediatric High-Grade Gliomas: The Virtues and Pitfalls of Pre-Clinical Models Open
Pediatric high-grade gliomas (pHGG) are malignant and usually fatal central nervous system (CNS) WHO Grade 4 tumors. The majority of pHGG consist of diffuse midline gliomas (DMG), H3.3 or H3.1 K27 altered, or diffuse hemispheric gliomas (D…
View article: Correction: The SIRT1 Deacetylase Suppresses Intestinal Tumorigenesis and Colon Cancer Growth
Correction: The SIRT1 Deacetylase Suppresses Intestinal Tumorigenesis and Colon Cancer Growth Open
[This corrects the article DOI: 10.1371/journal.pone.0002020.].
View article: PI3K/mTOR is a therapeutically targetable genetic dependency in diffuse intrinsic pontine glioma
PI3K/mTOR is a therapeutically targetable genetic dependency in diffuse intrinsic pontine glioma Open
Diffuse midline glioma (DMG), including tumors diagnosed in the brainstem (diffuse intrinsic pontine glioma; DIPG), are uniformly fatal brain tumors that lack effective treatment. Analysis of CRISPR/Cas9 loss-of-function gene deletion scre…
View article: Pediatric glioma histone H3.3 K27M/G34R mutations drive abnormalities in PML nuclear bodies
Pediatric glioma histone H3.3 K27M/G34R mutations drive abnormalities in PML nuclear bodies Open
View article: Integrin-linked kinase expression in myeloid cells promotes colon tumorigenesis
Integrin-linked kinase expression in myeloid cells promotes colon tumorigenesis Open
Colorectal cancer (CRC) is one of the most common forms of cancer worldwide and treatment options for advanced CRC, which has a low 5-year survival rate, remain limited. Integrin-linked kinase (ILK), a multifunctional, scaffolding, pseudo-…
View article: EXTH-31. EXPLOITING THE GENETIC DEPENDENCY ON PI3K/MTOR SIGNALING FOR THE TREATMENT OF H3-ALTERED DIFFUSE MIDLINE GLIOMA
EXTH-31. EXPLOITING THE GENETIC DEPENDENCY ON PI3K/MTOR SIGNALING FOR THE TREATMENT OF H3-ALTERED DIFFUSE MIDLINE GLIOMA Open
Recurring activating mutations, amplifications and allelic loss of the negative repressors of the phosphatidylinositol-4,5-bisphosphate 3-kinase (PI3K) signaling genes are overarching contributors to the poor survival of patients with H3K2…
View article: EXTH-36. COMBINING ONC201 AND PAXALISIB FOR THE TREATMENT OF DIFFUSE MIDLINE GLIOMA (DMG); THE PRECLINICAL RESULTS UNDERPINNING THE INTERNATIONAL PHASE II CLINICAL TRIAL (NCT05009992)
EXTH-36. COMBINING ONC201 AND PAXALISIB FOR THE TREATMENT OF DIFFUSE MIDLINE GLIOMA (DMG); THE PRECLINICAL RESULTS UNDERPINNING THE INTERNATIONAL PHASE II CLINICAL TRIAL (NCT05009992) Open
Diffuse midline gliomas (DMGs) diagnosed in the pons (DIPG) are universally fatal central nervous system tumors and are the leading cause of cancer-related death in children. Palliative radiotherapy is the only recognized treatment, with m…
View article: MODL-12. REGULATION OF OLIGODENDROGLIAL PROGENITOR CELL (OPC) CELL FATE IN DIFFUSE MIDLINE GLIOMA (DMG) BY THE HOMEOBOX TRANSCRIPTION FACTOR DLX2
MODL-12. REGULATION OF OLIGODENDROGLIAL PROGENITOR CELL (OPC) CELL FATE IN DIFFUSE MIDLINE GLIOMA (DMG) BY THE HOMEOBOX TRANSCRIPTION FACTOR DLX2 Open
Diffuse midline glioma, K27-altered (DMG) is a treatment resistant, uniformly fatal cancer that arises predominantly in children. 80% of patients with DMG harbor K27M mutations in canonical histone variants H3.3 and H3.1, leading to global…
View article: A protocol to establish cell line models from rare pediatric solid tumors
A protocol to establish cell line models from rare pediatric solid tumors Open