Published Medical Research

Making Headway has funded medical research grants and programs that have directly led to important new discoveries. Below are just some of the papers that have been published due to these investments.

Clinical utility of whole genome DNA methylation profiling, Neuro-Oncology Advances June 2023

To view our currently funded research projects, click here.

• Pediatric Posterior Fossa Tumor Treatment Affects Thalamic Volume, Memory, and Processing Speed, Neuro-Oncology April 2024.
• Confronting a Rare and Deadly Cancer in Children March 2024.
• Safety and pharmacokinetics of ONC201 Neuro-Oncology Feb 2024.
• Clinical utility of whole genome DNA methylation profiling, Neuro-Oncology Advances June 2023 .
• Phase II Study of Everolimus For Recurrent Or Progressive Pediatric Ependymoma February 2023.
• Reversal of cancer gene expression identifies repurposed drugs for diffuse intrinsic pontine glioma, Acta Neuropathologica, October 2022.
• Phase I dose escalation and expansion trial of single agent ONC201, Neuro-Oncology Advances, September 2022.
• Clinical, Pathological, and Molecular Characteristics of Diffuse Spinal Cord Gliomas, Journal of Neuropathology, September 2022.
• Tumor-Educated Platelets: A Review of Current and Potential Applications in Solid Tumors, Cureus, November 2021.
• Histone H3K36I mutation in a metastatic histiocytic tumor of the skull and response to sarcoma chemotherapy, CSH September 2021.
• Deconvoluting mechanisms of acquired resistance to RAF inhibitors in BRAF V600E mutant human glioma, RAF September 2021.
• A Novel Blood-Brain Barrier-Permeable Chemotherapeutic Agent for the Treatment of Glioblastoma, Cereus August 2021.
• PATZ1 fusions define a novel molecularly distinct neuroepithelial tumor entity with a broad histological spectrum, Acta Neuropathol, July 2021.
• Anaplastic Transformation in Myxopapillary Ependymoma, J Neuropathol, July 2020.
• Spatial progression and molecular heterogeneity of IDH‑mutant glioblastoma determined by DNA methylation‑based mapping, Acta Neuropathol, June 2021.
• Long-term neuropsychological outcomes of survivors of young childhood brain tumors treated on the Head Start II protocol, Neuro-Oncology Practice, May 2021.
• Glioblastomas with primitive neuronal component harbor a distinct methylation and copy‑number profile with inactivation of TP53, PTEN, and RB1, Acta Neuropathologica, April 2021. 
• The H3K36me2 writer-reader dependency in H3K27M-DIPG, January 2021.
• Primary mismatch repair deficient IDH‑mutant astrocytoma (PMMRDIA) is a distinct type with a poor prognosis, Acta Neuropathol, September 2020.
• Exploring DNA Methylation for Prognosis & Analyzing the Tumor Microenvironment in Pleomorphic Xanthoastrocytoma, Journal of Neuropathology, Aug 2020
• Molecular Correlates of Long Survival in IDH-Wildtype Glioblastoma Cohorts, Neuropathol, August 2020.
• Exploring DNA Methylation for Prognosis and Analyzing the Tumor Microenvironment in Pleomorphic Xanthoastrocytoma, J Neuropathol, May 2020.
• Genomics of MPNST (GeM) Consortium: Rationale and Study Design for Multi-Omic Characterization of NF1-Associated and Sporadic MPNSTs, Genes April 2020.
• Infant high grade gliomas comprise multiple subgroups characterized by novel targetable gene fusions and favorable outcomes, April 2020.
• Clinical impact of combined epigenetic and molecular analysis of pediatric low grade gliomas, Oxford Press, April 2020.
• GOPC-ROS1 Fusion Due to Microdeletion at 6q22 Is an Oncogenic Driver in a Subset of Pediatric Gliomas and Glioneuronal Tumors, Journal of Neuropathology, December 2019.
• Functional and topographic effects on DNA methylation in IDH1/2 mutant cancers, Nature, November 2019.
• LEDGF and HDGF2 relieve the nucleosome-induced barrier to transcription in differentiated cells, Molecular Biology, October 2019.
• Molecular subgrouping of primary pineal parenchymal tumors reveals distinct subtypes correlated with clinical parameters and genetic alterations, October 2019.
• Pediatric and adult H3 K27M‑mutant diffuse midline glioma treated with the selective DRD2 antagonist ONC201, Journal of Neuro-Oncology, August 2019
• Subgroup-specific outcomes of children with malignant childhood brain tumors treated with an irradiation-sparing protocol, Child’s Nervous System, July 2019.
• Automethylation of PRC2 promotes H3K27 methylation and is impaired in H3K27M pediatric glioma, CHS, May 2019.
• Quality of life, hypothalamic obesity, and sexual function in adulthood two decades after primary gross-total resection for childhood craniopharyngioma, Child’s Nervous System, April 2019.
• Cell Surface Notch Ligand DLL3 is a Therapeutic Glioma, February 2019.
• Long-term clinical and visual outcomes after surgical resection of pediatric pilocytic/pilomyxoid optic pathway gliomas – JNS Pediatrics, 2019
• LEDGF and HDGF2 relieve the nucleosome-induced barrier – Biorxiv, December 2018.
• Multiple modes of PRC2 inhibition elicit global chromatin alterations in H3K27M pediatric glioma – Science, October 2018.
• Recurrent homozygous deletion of DROSHA and microduplication of PDE4DIP in pineoblastoma – Nature Communication, July 2018.
• Distinct stimulatory mechanisms regulate the catalytic activity of Polycomb Repressive Complex 2 (PRC2) – Molecular Cell May 2018.
• Allosteric Activation Dictates PRC2 Activity Independent of Its Recruitment to Chromatin, Molecular Cell, May 2018.
• Pre-treatment lymphopenia and indication of tumor-induced systemic immunosuppression in medulloblastoma, November 2017.
• Endothelium-Independent Primitive Myxoid Vascularization Creates Invertebrate-Like Channels to Maintain Blood Supply in Optic Gliomas, August 2017.
• Therapeutic and Prognostic Implications of BRAF V600E in Pediatric Low-Grade Gliomas. – Journal of Clinical Oncology, July 2017.
• Immunohistochemical analysis of H3K27me3 demonstrates global reduction in group‑A childhood posterior fossa ependymoma and is a powerful predictor of outcome, Acta Neuropathol
• Comparison of self-report and electronic monitoring of 6MP intake in childhood ALL: a Children’s Oncology Group study, April 2017
• Splice site mutation in factor X gene manifesting as severe intracranial haemorrhage in neonatal period with a challenging treatment course, Dec 2016. 
• Polymorphous low-grade neuroepithelial tumor of the young (PLNTY): an epileptogenic neoplasm with oligodendroglioma-like components, aberrant CD34 expression, and genetic alterations involving the MAP kinase pathway, November 2016
• Mutant IDH1 and thrombosis in gliomas, Acta Neuropathologica, September 2016
• MLL Rearrangements Impact Outcome in HOXA-deregulated T-lineage Acute Lymphoblastic Leukemia: A Children’s Oncology Group Study, September 2016
• Vascularization of optic gliomas: primitive invertebrate-like channels – Clinical and Therapeutic implications, 2016.
• Recurrent MET fusion genes represent a drug target in pediatric glioblastoma – Nature Medicine, October 2016.
• Utility of MRI versus tumor markers for post-treatment surveillance of marker-positive CNS germ cell tumors, Neurooncol, September 2016.
• Therapeutic Impact of Cytoreductive Surgery and Irradiation – Journal of Clinical Oncology, July 2016.
• High Incidence of Veno-Occlusive Disease With Myeloablative Chemotherapy Following Craniospinal Irradiation in Children With Newly Diagnosed High-Risk CNS Embryonal Tumors: A Report From the Children’s Oncology Group, May 2016.
• The influence of central review on outcome in malignant gliomas – DHH, April 2016.
• New Brain Tumor Entities Emerge from Molecular Classification of CNS-PNETs – Cell, February 2016 
• Molecular Classification of Ependymal Tumors across All CNS Compartments, Histopathological Grades, and Age Groups – Cancer Cell, May 2015
• Phase II study of everolimus in children and adults with neurofibromatosis type 2 and progressive vestibular schwannomas – Neuro-Oncology, 2014.
• Recurrent Ascites in a Patient With Low-grade Astrocytoma and Ventriculo-Peritoneal Shunt Treated With the Multikinase Inhibitor Sorafenib, Pediatr Hematol, November 2014.
• Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas – Neuro-Oncology, May 2014.
• Recommendations for imaging tumor response in neurofibromatosis clinical trials – Neurology, August 2013.
• Potential Role of Ventricular Tumor Markers in CNS Germ Cell Tumors, Pediatr Blood Cancer, June 2013.
• BRAF Alterations in Primary Glial and Glioneuronal Neoplasms of the Central Nervous System With Identification of 2 Novel KIAA1549:BRAF Fusion Variants – Journal of Neuropathology & Experimental neurology, January 2012.
• Imaging CXCR4 Expression in Human Cancer Xenografts: Evaluation of Monocyclam Cu-64-AMD3465, Nuclear Medicine, June 2011.
• Regulation of the Unfolded Protein Response by eIF2Bδ Isoforms – Journal of Biological Chemistry, August 2010.
• Hypoxic regulation of mRNA expression, Cell Cycle July 2008.
• Hypoxic Inhibition of Nonsense-Mediated RNA Decay Regulates Gene Expression and the Integrated Stress Response, Molecular Biology, June 2008.
• Tumor antigen precursor protein profiles of adult and pediatric brain tumors identify potential targets for immunotherapy – National Institute of Health, May 2008.
• Tenascin-C Promotes Microvascular Cell Migration and Phosphorylation of Focal Adhesion Kinase – Cancer Research, May 2002.
• Initial Management of Children with Hypothalamic and Thalamic Tumors and the Modifying Role of Neurofibromatosis-1, January 2000.