Journal
NATURE MEDICINE
Volume 21, Issue 6, Pages 555-559Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nm.3855
Keywords
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Funding
- Lyla Nsouli Foundation
- Children's Oncology Group (COG) Central Nervous System Committee
- DIPG Collaborative (The Cure Starts Now Foundation)
- DIPG Collaborative (Reflections of Grace Foundation)
- DIPG Collaborative (Smiles for Sophie Foundation)
- DIPG Collaborative (Cancer-Free Kids Foundation)
- DIPG Collaborative (Carly's Crusade Foundation)
- DIPG Collaborative (Jeffrey Thomas Hayden Foundation)
- DIPG Collaborative (Soar with Grace Foundation)
- Accelerate Brain Cancer Cures Foundation (ABC2)
- CureSearch for Childhood Cancer
- Team Julian Foundation
- COG Chair's Grant [5UOCA098543]
- US National Institutes of Health [K08NS070926]
- Alex's Lemonade Stand Foundation
- McKenna Claire Foundation
- Connor Johnson Memorial Fund
- Dylan Jewett Memorial Fund
- Elizabeth Stein Memorial Fund
- Dylan Frick Memorial Fund
- Abigail Jensen Memorial Fund
- Zoey Ganesh Memorial Fund
- Wayland Villars DIPG Foundation
- Jennifer Kranz Memorial Fund
- Unravel Pediatric Cancer
- Virginia & D.K. Ludwig Fund for Cancer Research
- Price Family Charitable Fund
- Matthew Larson Foundation
- Godfrey Family Fund in Memory of Fiona Penelope
- Child Health Research Institute at Stanford
- Anne T. and Robert M. Bass Endowed Faculty Scholarship in Pediatric Cancer and Blood Diseases
- Etoile de Martin
- Fondation Lemos
- Le Defi de Fortunee
- Scott Carter Foundation
- Semmy Foundation
- US Department of Defense
- National Science Foundation [CCF0953366]
- Marie Curie grant [IRG270459]
- Spanish Ministry of Health [PI13/0125]
- St. Baldrick's Foundation
- Iron Matt Foundation
- Division of Computing and Communication Foundations
- Direct For Computer & Info Scie & Enginr [0953366] Funding Source: National Science Foundation
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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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