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Functionally-defined Therapeutic Targets in Diffuse Intrinsic Pontine Glioma
dbGaP
Data Resource:  dbGaP
Point of Contact:  
Charles Keller,  
Project

About This Dataset
Diffuse Intrinsic Pontine Glioma (DIPG) is a universally fatal childhood cancer. Here, we performed a chemical screen in patient-derived DIPG cell cultures along with RNAseq expression analysis and integrated computational modeling to identify potentially effective therapeutic strategies. Panobinostat, among the more promising agents identified, demonstrated efficacy in pontine orthotopic xenograft models of both H3K27M and histone WT DIPG. These data suggest the potential utility of specific drug combinations and provides evidence of in vivo treatment efficacy of the multi-histone deacetylase inhibitor panobinostat.
Core Data Elements
Number of Cases
31
Case Sex
Female (11); Male (12); Not Reported (8)
Case Age At Diagnosis
0 to 4 years (7); 5 to 9 years (16); 10 to 14 years (6); 15 to 19 years (1); 20 to 24 years (1)
Case Race
Not Reported (31)
Case Ethnicity
Not Reported (31)
Case Disease Diagnosis
Diffuse Intrinsic Pontine Glioma (31)
Case Tumor Site
Brain (31)
Number of Samples
75
Sample Assay Method
RNA Sequencing (40); Whole Genome Sequencing (35)
Sample Is Normal
Yes (20); No (55)
Additional Data Elements
DBGAP STUDY IDENTIFIER
Grant Information
K08NS070926
Precursor cell-niche interactions and the genesis of brainstem gliomas
Published In
https://doi.org/10.1038/nm.3855
Charts
Male (12); Female (11); Not Reported (8)
12Male