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.
There are a few clinical trials going on with this drug. Click here to see them.
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 interested.