This is an experimental drug - and things may rapidly change.
This page is current as of 11/24/21, but everything can change - contact us if you definitely need to know.
Disclaimer: (added 10/22/2021) The Musella Foundation has made a few grants to Oncoceutics (the company that was developing Onc-201 and which was later sold to Chimerix), some of which were of the venture philanthropy type. This means that if the drug ever gets approved and makes a profit, the Musella Foundation will get a return of more than the amount we gave them. If the drug is never profitable, the Musella Foundation gets nothing. Dr. Al Musella will not personally benefit from this arrangement. Dr. Musella will not participate in the patient navigation process for patients eligible for Onc-201 to avoid any possibility (or appearance) of conflict of interest. There was never a shortage of patients for the Onc-201 program - patients reached out to us specifically to get help getting access to the drug. At the time we ran the expanded access program for Onc-201, there were literally no other promising treatments available for DIPG and DMG. (There are a few now!) xCures ran the patient registry and did all of the regulatory paperwork for us and did not charge us yet. If we do get a return, we will then pay xCures for the services they provided. The remainder will be used to fund brain tumor research. The scientists and doctors who run our patient navigation program ( at Cancer Commons and xCures ) will not personally benefit from any return either. The one exception to this is that Dr. Musella does own stock in xCures and it's value may go up if Onc-201 gets approved based on help from data xCures collected. However, the Onc-201 project at xCures is just a small portion of what xCures handles and they will do well even if Onc-201 doesn't get approved.


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 patient benefit rate of over 50%, which is unheard of in this tumor type.

Clinical 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. A smaller expanded access program is now open. See https://oncoceutics.com/expanded-access/ for details!.

Access outside of the USA

France now supplies Onc-201 for free for those who need it!
The DMG-ACT trial is now open in Europe and Austrailia! (as well as all over the USA!)

Latest News about Onc-201

We had a webinar about combination therapy for DIPG / DMG which is based on Onc-201.

Chimerix Announces Positive ONC201 Data in Recurrent H3 K27M-mutant Diffuse Midline Glioma to Be Presented at the Society for Neuro-Oncology Annual Meeting

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:

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