Isabel Arrillaga-Romany, Sharon L Gardner, Yazmin Odia, Dolly Aguilera, Joshua E Allen, Tracy Batchelor, Nicholas Butowski, Clark Chen, Timothy Cloughesy, Andrew Cluster, John de Groot, Karan S Dixit, Jerome J Graber, Aya M Haggiagi, Rebecca A Harrison, Albert Kheradpour, Lindsay Kilburn, Sylvia C Kurz, Guangrong Lu, Tobey J MacDonald, Minesh Mehta, Allen S Melemed, Phioanh Leia Nghiemphu, Samuel C Ramage, Nicole Shonka, Ashley Sumrall, Rohinton Tarapore, Lynne Taylor, Yoshie Umemura, Patrick Y Wen
PURPOSE: Histone 3 (H3) K27M-mutant diffuse midline glioma (DMG) has a dismal prognosis with no established effective therapy beyond radiation. This integrated analysis evaluated single-agent ONC201 (dordaviprone), a first-in-class imipridone, in recurrent H3 K27M-mutant DMG. METHODS: Fifty patients (pediatric, n = 4; adult, n = 46) with recurrent H3 K27M-mutant DMG who received oral ONC201 monotherapy in four clinical trials or one expanded access protocol were included...
February 9, 2024: Journal of Clinical Oncology