2026
2025

A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low grarde gliomas.
Apfelbaum April, Morin E, et al. Nature Commun. 2025 July 31;16(1):7018.

Deep learning volumetrics reveals distinct clinical trajectories for pediatric low-grade gliomas under surveillance: A multicenter study.
Climent Pardo JC, et al. Neuroncol Advances. 2025 July 1;7(1):vdaf145.

Longitudinal risk prediction for pediatric glioma with temporal deep learning
Tak, et al. NEJM AI. 2025 May;2(5):10.1056/aioa2400703

Incidental Diffuse Midline Glioma, H3 K27-altered of the pons without significant coalterations
Yeo, et al. JCO Precis Oncol. 2025 June: 9:e2400944

Recurrent breakpoints in the BRD4 locus reduce toxicity associated with gene amplification
Wala, et al. Cell Genom. 2025 April 9; 5(4):100815.

Effective targeting of PDGFRA-altered-high-grade glioma with avapritinib
Mayr L, et al. Cancer Cell. 2025 April 14;43(4):740-756.

A compendium of amplification-related gain of sensitivity genes in human cancer.
Rendo V, et al. Nature Commun. 2025 Jan 27;16(1):1077. 
Characterization and inhibitor sensitivity of ARAF, BRAF, and CRAF kinases.J Biol Chem. 2025 Dec;301(12):110800.

Cryo-EM structures of CRAF/MEK1/14-3-3 complexes in autoinhibited and open-monomer states reveal features of RAF regulation.Nat Commun. 2025 Sep 1;16(1):8150.

2024

Pediatric neuro-oncology: Highlights of the last quarter-century.
Neoplasia. 2025 Jan: 59:101098.

FOXR2 targets LHX6+/DLX+ neural lineages to drive CNS neuroblastoma.
Cancer Res. 2024 Nov 4

Multimodal deep learning improves recurrence risk prediction in pediatric low-grade gliomas.
Neuro Oncol. 2024 Aug 30: noae173.

Genome-wide CRISPR-Cas9 knockout screens identify DNMTA1 as a druggable dependency in sonic hedgehog medulloblastoma.
Acta Neuropathol Commun. 2024 August 7;12(1):125.

Molecular profiling of 888 pediatric tumors informs future precision trials and data-sharing initiatives in pediatric cancer.
Nat Commun. 2024 July 11;15(1):5837.

Resistance, rebound, and recurrence growth patterns in pediatric low-grade glioma treated by MAPK inhibition: A modified Delphi approach to build international consensus-based definitions-International Pediatric Low-Grade Glioma Coalition
Neuro Oncol. 2024 Aug 5;26(8):1357-1366.

Noninvasive molecular subtyping of pediatric low-grade glioma with self-supervised transfer learning
Radiol Artif Intell. 2024 May;6(3):e230333.

Aberrant DNA repair reveals a vulnerability in histone H3.3-mutant brain tumors.
Nucleic Acids Res. 2024 Mar 21;52(5):2372-2388.

Translation of non-canonical open reading frames as a cancer cell survival mechanism in childhood medulloblastoma
Mol Cell. 2024 Jan 18;84(2):261-276.e18. 

2023

Consensus framework for conducting phase I/II clinical trials for children, adolescents, and young adults with pediatric low-grade glioma: Guidelines established by the International Pediatric Low-Grade Glioma Coalition Clinical Trial Working Group
Neuro Oncol. 2023 Dec 26

A road map for the treatment of pediatric diffuse midline glioma
Cancer Cell. 2023 Nov 20: S1535-6108(23)00393-8.

Pediatric low-grade glioma: State-of-the-art and ongoing challenges
Neuro Oncol. 2023 Nov 7; noad195.

Indoximod-based chemo-immunotherapy for pediatric brain tumors: a first-in-children phase 1 trial
Neuro Oncol. 2023 Sep 16; noad176.

The ‘LOGGIC’ of RNA-sequencing in enhancing diagnoses of pediatric low-grade gliomas
Neuro Oncol. 2023 Aug 2; noad142.

A pilot study omitting radiation in the treatment of children with newly diagnosed WNT-activated medulloblastoma.
Clin Cancer Res. 2023 July 27, CCR-23–348.

Genome-wide pooled CRISPR screening in neurospheres.
Nat Protocols. 2023 June 7.

Accurate diagnosis of pediatric brain cancers.
Nat Medicine. 2023 Mar 17.

Integrated response analysis of pediatric low-grade gliomas during and after targeted therapy treatment.
Neurooncol Advances. 2022 Dec 18;5(1):vdac182.

ALK amplification and rearrangements are recurrent targetable events in congenital and adult glioblastoma. 
Clin Cancer Research. 2023 Feb 13; CCR-21-3521.

Pediatric low-grade glioma: Targeted therapeutics and clinical trials in the molecular era.
Neoplasia. 2023 Feb; 36:100857.

K27M in canonical and noncanonical H3 variants occurs in distinct oligodendroglial cell lineages in brain midbrain gliomas
Nat Genetics. 2022 Dec; 54(12); 1865-1880

Cryo-EM structure of a RAS/RAF recruitment complex. Nat Commun14, 5517 (2023).

Structure of the MRAS-SHOC2-PP1C phosphatase complex. Nature. 2022 Sep;609(7926):416-423.

Allosteric MEK inhibitors act on BRAF/MEK complexes to block MEK activation. Proc Natl Acad Sci U S A. 2021 Sep 7;118(36):e2107207118.

Architecture of autoinhibited and active BRAF-MEK-14-3-3 complexes. Nature. 2019 Nov;575(7783):545-550.

2022

Paediatric Strategy Forum for medicinal product development in mitogen-activated protein kinase pathway inhibitors: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. 
Eur J Cancer. 2022 Oct 14;177:120-142.

Molecular genetics of paediatric brain tumours and opportunities for precision medicine – a focus on infant brain tumors. 
Curr Opin Neurol. 2022 Oct 5.

BH3 mimetics targeting BCL-XL impact the senescent compartment of pilocytic astrocytoma
Neuro Oncol. 2022 Aug 17; noac199.

Single cell spatial analysis reveals the topology of immunomodulatory purinergic signaling in glioblastoma
Nat Commun Aug 16;13(1):4814.

FOXR2 is an epigenetically regulated pan-cancer oncogene that activates ETS transcriptional circuits
Cancer Research 2022 July 8.

Featured in the Broad Blog here.

Structural variants shape driver combinations and outcomes in pediatric high-grade glioma
Nat Cancer 2022 July 4.

Featured in the DFCI Blog here.

Pediatric pineoblastoma: A pooled outcome study of North American and Australian therapeutic data
Neurooncol Adv 2022 April 14; 4(1):vdac056.

PPM1D mutations are oncogenic drivers of de novo diffuse midline glioma formation
Nature Commun 2022 Feb 1; 13(1):604.

Featured on this Broad News Update.

Liquid biopsy detection of genomic alterations in pediatric brain tumors from cell-free DNA in peripheral blood, CSF and Urine.
Neuro Oncol 2022 Jan 4

Accuracy of central neuro-imaging review of DIPG compared with histopathology in the International DIPG Registry
Neuro Oncol 2021 Oct 20

Molecular alterations in pediatric low-grade gliomas that led to death
J Neuropathol Exp Neurol 2021  Nov 19; 80(11):1052-1059

The challenges in treating embryonal tumors with multilayered rosettes (ETMR) and other infant brain tumors
Neuro Oncol. 2022  Jan 5; 24(1):138-140

Characteristics of patients greater than 10 years of age with diffuse intrinsic pontine glioma: a report from the International DIPG/DMG Registry
Neuro Oncol. 2022  Jan 5;24(1):141-152

2021
2020

Advances in the classification and treatment of pediatric brain tumors
Curr Opin Pediatr. 2020 Dec 4.

Histone H3.3G34-Mutant Interneuron Progenitors Co-opt PDGFRA for Gliomagenesis
Cell (2020); S0092-8674(20)31529-4

CloneSifter: enrichment of rare clones from heterogenous cell populations
BMC Biology (2020); Nov 24;18(1):177.

Retrospective evaluation of whole exome and genome mutation calls in 746 cancer samples
Nature Commun (2020); Sept 21;11(1):4748.

Sex differences in oncogenic mutational processes
Nature Commun (2020); Aug 28;11(1):4330.

Trametinib for the treatment of recurrent/progressive pediatric low-grade glioma
J Neuro-Onc (2020); Sept;149(2):253-262.

Mechanisms and therapeutic implications of hypermutation in gliomas
Nature (2020) Pub online 15 April 2020.

The “Risk” in Pediatric Low-Grade Glioma
Cancer Cell (2020); Apr 13;37(4): 424-425.

Analyses of non-coding somatic drivers in 2,658 cancer whole genomes
Nature (2020); 578 (7793): 102-111.

Pan-cancer Analysis of whole genomes
Nature (2020); 578 (7793): 82-93.

Pathway and Network Analysis of more than 2500 whole cancer genomes
Nature Commun (2020); 11(1):729. Published 2020 Feb 5.

Combined burden and functional impact tests for cancer driver discovery using DriverPower
Nature Commun (2020); 11(1):734. Published 2020 Feb 5.

Treatment-induced tumor dormancy through YAP-mediated transcriptional reprogramming of the apoptotic pathway
Cancer Cell (2020) Jan 13; 37(1):104-122.e12.

The power of human cancer genetics as revealed by low-grade gliomas.
Annual Review of Genetics. Dec 2019/53;483-503.
Management of pediatric low-grade glioma.
Current Opinions in Pediatrics. Feb 2019/31 (1);21-27.
2018
2017
Brainstem angiocentric gliomas with MYB-QKI rearrangements.
Acta Neuropathology. 2017; 134(4):667-669.
2016
2015
2014
Diffuse intrinsic pontine glioma: a reassessment
J Neurooncol. 2014 Aug;119(1):7-15.
Targeting BRAF in pediatric brain tumors
Am Soc Clin Oncol Educ Book. 2014:e436-40.
2013
Recurrent somatic alterations of FGFR1 and NTRK2 in pilocytic astrocytoma
International Cancer Genome Consortium PedBrain Tumor Project. Nat Genet. 2013 Aug;45(8):927-32. doi: 10.1038/ng.2682. Epub 2013 Jun 30. PMID:23817572.
PDGFRA amplification is common in pediatric and adult high-grade astrocytomas and identifies a poor prognostic group in IDH1 mutant glioblastoma
Brain Pathol. 2013 Sep;23(5):565-73. doi: 10.1111/bpa.12043. Epub 2013 Mar 18. PMID:23438035.

2012
Absence of oncogenic canonical pathway mutations in aggressive pediatric rhabdoid tumors
Blood Cancer. 2012 Dec 15;59(7):1155-7. doi: 10.1002/pbc.24315. Epub 2012 Sep 19. PMID:2997201.
Separated at birth? The functional and molecular divergence of OLIG1 and OLIG2
Nat Rev Neurosci. 2012 Dec;13(12):819-31. PMID:23165259.

2011

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