An academia-driven compassionate use program for patients with recurrent H3K27-altered diffuse midline glioma: a new way to access innovation when clinical trials …

D Di Carlo, M Annereau, I Yoldjian… - Annals of …, 2023 - annalsofoncology.org
Diffuse midline gliomas H3K27-altered have a very poor prognosis both in adults and
children (1). The current therapeutic strategy is based on radiotherapy, that can only slow
down the progression of the disease (2). In this context, it is a priority to find new agents to
be tested in clinical trials. In the last years, a new drug developed by OncoceuticsTM and
then ChimerixTM, ONC201/dordaviprone, has emerged as a promising agent in a phase II
clinical trial in the USA (3). The drug was well tolerated with just grade 1 or 2 toxicities like …