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Diffuse midline glioma | Treatment | Targeted therapy | Dordaviprone (ONC201)

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Hansford JR, Manoharan N, Bouche G, Ramaswamy V, Jabado N, Bouffet E.
ONC201 (Dordaviprone): review of evidence to date in diffuse midline glioma, hope or hype?
Expert Opin Emerg Drugs. 2024 Nov 21:1-5. doi: 10.1080/14728214.2024.2426649. PMID: 39548664. Perspective. ˍ

Arrillaga-Romany I, Gardner SL, Odia Y, Aguilera D, Allen JE, Batchelor T, Butowski N, Chen C, Cloughesy T, Cluster A, de Groot J, Dixit KS, Graber JJ, Haggiagi AM, Harrison RA, Kheradpour A, Kilburn L, Kurz SC, Lu G, MacDonald TJ, Mehta M, Melemed AS, Nghiemphu PL, Ramage SC, Shonka N, Sumrall A, Tarapore R, Taylor L, Umemura Y, Wen PY.
ONC201 (Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline Glioma.
J Clin Oncol. 2024 Feb 9:JCO2301134. doi: 10.1200/JCO.23.01134. PMID: 38335473. Interventional study. ˍ

Di Carlo D, Annereau M, Vignes M, Denis L, Epaillard N, Dumont S, Guyon D, Rieutord A, Jacobs S, Salomon V, Yoldjian I, Duperray F, Brunel L, Baiao X, Lemos F, Vauleon E, Capra M, Abbou S, Touat M, Sanson M, Gandemer V, De Carli E, Bourdeaut F, Hezam I, Vassal G, Grill J.
Real life data of ONC201 (dordaviprone) in pediatric and adult H3K27-altered recurrent diffuse midline glioma: Results of an international academia-driven compassionate use program.
Eur J Cancer. 2024 Dec 11;216:115165. doi: 10.1016/j.ejca.2024.115165. PMID: 39700833. Compassionate use. ˍ

Faison SL, Batonga J, Arumugham T, Bartkus A, Morrison M, Mullin MJ, Tippin T, Naderer O.
A Phase 1 Randomized Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Escalating Oral Doses of Dordaviprone and the Effects of Food on the Bioavailability of Dordaviprone in Healthy Adult Subjects.
Clin Pharmacol Drug Dev. 2025 Jan 16. doi: 10.1002/cpdd.1512. PMID: 39817482. Interventional study. ˍ