Last updated: 8/24/2020

Note - this is an experimental drug - and things may change rapidly. This page is current as of 8/24/2020 but everything can change - contact us if you need to know for sure!

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Overview: Onc-201 is an oral, well tolerated experimental drug used to treat a serious rare pediatric and young adult brain tumor: H3K27M diffuse midline glioma, including DIPG. This type of tumor has no approved treatments and average survival is only 9 months. Onc-201 has been shown to have very few safety related side effects in over 350 patients, and in a small trial of this specific brain tumor type showed at least a doubling of expected survival time with many patients still doing well, and a 27%-46% response rate, which is unheard of in this tumor type.

Clinicl Trials There are a few clinical trials going on with this drug. Click HERE to see them.

Compassionate use in the USA We used to fund and run the compassionate use program in the USA for Onc-201 but we ran out of funding and sadly the program had to close. We are trying to reopen it. Contact us for details if you are interested.


There was an FDA ODAC meeting on June 20, 2019 to discuss the development process of Onc-201. Here are some important files from that meeting: