Article
Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways.
Cancer discovery - 1 Nov 2023
Venneti Sriram, Kawakibi Abed Rahman, Ji Sunjong, Waszak Sebastian M, Sweha Stefan R, Mota Mateus, Pun Matthew, Deogharkar Akash, Chung Chan, Tarapore Rohinton S, Ramage Samuel, Chi Andrew, Wen Patrick Y, Arrillaga-Romany Isabel, Batchelor Tracy T, Butowski Nicholas A, Sumrall Ashley, Shonka Nicole, Harrison Rebecca A, de Groot John, Mehta Minesh, Hall Matthew D, Daghistani Doured, Cloughesy Timothy F, Ellingson Benjamin M, Beccaria Kevin, Varlet Pascale, Kim Michelle M, Umemura Yoshie, Garton Hugh, Franson Andrea, Schwartz Jonathan, Jain Rajan, Kachman Maureen, Baum Heidi, Burant Charles F, Mottl Sophie L, Cartaxo Rodrigo T, John Vishal, Messinger Dana, Qin Tingting, Peterson Erik, Sajjakulnukit Peter, Ravi Karthik, Waugh Alyssa, Walling Dustin, Ding Yujie, Xia Ziyun, Schwendeman Anna, Hawes Debra, Yang Fusheng, Judkins Alexander R, Wahl Daniel, Lyssiotis Costas A, de la Nava Daniel, Alonso Marta M, Eze Augustine, Spitzer Jasper, Schmidt Susanne V, Duchatel Ryan J, Dun Matthew D, Cain Jason E, Jiang Li, Stopka Sylwia A, Baquer Gerard, Regan Michael S, Filbin Mariella G, Agar Nathalie Y R, Zhao Lili, Kumar-Sinha Chandan, Mody Rajen, Chinnaiyan Arul, Kurokawa Ryo, Pratt Drew, Yadav Viveka N, Grill Jacques, Kline Cassie, Mueller Sabine, Resnick Adam, Nazarian Javad, Allen Joshua E, Odia Yazmin, Gardner Sharon L, Koschmann Carl
Abstract excerpt
Patients with H3K27M-mutant diffuse midline glioma (DMG) have no proven effective therapies. ONC201 has recently demonstrated efficacy in these patients, but the mechanism behind this finding remains unknown. We assessed clinical outcomes, tumor sequencing, and tissue/cerebrospinal fluid (CSF) correlate samples from patients treated in two completed multisite clinical studies. Patients treated with ONC201...
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