Functionally defined therapeutic targets in diffuse intrinsic pontine glioma

CS Grasso, Y Tang, N Truffaux, NE Berlow, L Liu… - Nature medicine, 2015 - nature.com
CS Grasso, Y Tang, N Truffaux, NE Berlow, L Liu, MA Debily, MJ Quist, LE Davis, EC Huang…
Nature medicine, 2015nature.com
Diffuse intrinsic pontine glioma (DIPG) is a fatal childhood cancer. We performed a chemical
screen in patient-derived DIPG cultures along with RNA-seq analyses and integrated
computational modeling to identify potentially effective therapeutic strategies. The multi–
histone deacetylase inhibitor panobinostat demonstrated therapeutic efficacy both in vitro
and in DIPG orthotopic xenograft models. Combination testing of panobinostat and the
histone demethylase inhibitor GSK-J4 revealed that the two had synergistic effects …
Abstract
Diffuse intrinsic pontine glioma (DIPG) is a fatal childhood cancer. We performed a chemical screen in patient-derived DIPG cultures along with RNA-seq analyses and integrated computational modeling to identify potentially effective therapeutic strategies. The multi–histone deacetylase inhibitor panobinostat demonstrated therapeutic efficacy both in vitro and in DIPG orthotopic xenograft models. Combination testing of panobinostat and the histone demethylase inhibitor GSK-J4 revealed that the two had synergistic effects. Together, these data suggest a promising therapeutic strategy for DIPG.
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