Expanded Access Program for ONC201 to Treat Recurrent Histone H3 Mutant Glioma (click here)
In a phase two trial of ONC201 for recurrent GBM, a 22 year old female with secondary H3 K27M mutant GBM achieved a partial response to ONC201, which has been sustained for over 6 months. One of her two tumors regressed by 85% and the other tumor regressed by 75% after 8 and 11 months of therapy.
Note that the H3 K27M mutation (also known as H3F3A K27M) is most common in brainstem gliomas of young children and young adults, including diffuse intrinsic pontine glioma (DIPG). This treatment could turn out to be a breakthrough, as there is currently no known effective treatment for this tumor type.