Thursday, 1 June 2017

ONC201 expanded access for H3 K27M mutant gliomas

This is rather big news for H3 K27M mutant gliomas (including a high percentage of DIPG).

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.

A phase 2 study of the first imipridone ONC201, a selective DRD2 antagonist for oncology, administered every three weeks in recurrent glioblastoma  (click here)

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.


  1. Thanks for posting this!
    The second tumour regressed by 76% according to the abstract:
    "exhibiting regression by 85% in one lesion and 76% in the second lesion"

    1. The phrasing is confusing. In the main body of the text it says "The second lesion regressed to approximately 75% of the baseline size after 10.7
      months of therapy."
      If it regressed to 75% of the baseline, that would be a 25% regression, but this is probably just bad phrasing, so it probably was a 75% regression as it seems to be saying in the abstract.