Authors
Sebastian Brabetz, Sarah ES Leary, Susanne N Gröbner, Madison W Nakamoto, Huriye Şeker-Cin, Emily J Girard, Bonnie Cole, Andrew D Strand, Karina L Bloom, Volker Hovestadt, Norman L Mack, Fiona Pakiam, Benjamin Schwalm, Andrey Korshunov, Gnana Prakash Balasubramanian, Paul A Northcott, Kyle D Pedro, Joyoti Dey, Stacey Hansen, Sally Ditzler, Peter Lichter, Lukas Chavez, David TW Jones, Jan Koster, Stefan M Pfister, Marcel Kool, James M Olson
Publication date
2018/11
Journal
Nature medicine
Volume
24
Issue
11
Pages
1752-1761
Publisher
Nature Publishing Group US
Description
Brain tumors are the leading cause of cancer-related death in children. Genomic studies have provided insights into molecular subgroups and oncogenic drivers of pediatric brain tumors that may lead to novel therapeutic strategies. To evaluate new treatments, better preclinical models adequately reflecting the biological heterogeneity are needed. Through the Children’s Oncology Group ACNS02B3 study, we have generated and comprehensively characterized 30 patient-derived orthotopic xenograft models and seven cell lines representing 14 molecular subgroups of pediatric brain tumors. Patient-derived orthotopic xenograft models were found to be representative of the human tumors they were derived from in terms of histology, immunohistochemistry, gene expression, DNA methylation, copy number, and mutational profiles. In vivo drug sensitivity of targeted therapeutics was associated with distinct molecular …
Scholar articles
S Brabetz, SES Leary, SN Gröbner, MW Nakamoto… - Nature medicine, 2018