Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

ONC201 对 H3K27M 突变型弥漫性中线胶质瘤的临床疗效是由综合代谢和表观遗传途径的破坏所驱动

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作者：Sriram Venneti #, Abed Rahman Kawakibi #, Sunjong Ji #, Sebastian M Waszak #, Stefan R Sweha #, Mateus Mota #, Matthew Pun #, Akash Deogharkar, Chan Chung, Rohinton S Tarapore, Samuel Ramage, Andrew Chi, Patrick Y Wen, Isabel Arrillaga-Romany, Tracy T Batchelor, Nicholas A Butowski, Ashley Sumrall,

期刊：

Cancer Discovery

影响因子：

29.700

时间：

2023

起止号：

2023 Nov 1;13(11):2370-2393.

doi：

10.1158/2159-8290.CD-23-0131

方法学：

ELISA、IF、IHC-P、IP、WB

研究方向：

代谢、神经、肿瘤

疾病类型：

胶质瘤

Significance

The clinical, radiographic, and molecular analyses included in this study demonstrate the efficacy of ONC201 in H3K27M-mutant DMG and support ONC201 as the first monotherapy to improve outcomes in H3K27M-mutant DMG beyond radiation. Mechanistically, ONC201 disrupts integrated metabolic and epigenetic pathways and reverses pathognomonic H3K27me3 reduction. This article is featured in Selected Articles from This Issue, p. 2293.

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