Modeyso™ (formerly known as ONC201) is an oral, well-tolerated drug that has just (On 8/6/2025) received FDA approval for the treatment of recurrent H3K27M diffuse midline glioma, including DIPG - a rare and aggressive brain tumor primarily affecting children and young adults. Until now, this tumor type had no FDA-approved treatments, and the average survival was only 9 months.
In clinical trials, Modeyso has demonstrated very few safety-related side effects in over 350 patients. In a small trial specifically focused on this tumor type, Modeyso resulted in at least a doubling of expected survival time, with many patients still doing well long-term. The patient benefit rate exceeded 50%, which is unprecedented in this type of brain tumor.
There are a few clinical trials going on with this drug. Click here to see them.
(Added 8/6/25) Modeys™ will soon (hopefully a few weeks) be available by prescription anywhere in the USA. Jazz Pharmaceuticals will start patient assistance programs hopefully as soon as the drug becomes available! Contact them for help!
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!)
Clinical efficacy of ONC-201 in H3K27M mutated DMG is driven by disruption of integrated metatbolic and epigenetic pathways (Added 8/26/23). This peer reviewed paper discussed the results of two clinical trials showing that Onc-201 (by itself) doubles survival in these patients! Hopefully will do much better in combinations!(We helped fund this project!)
We had a webinar about combination therapy for DIPG / DMG which is based on Onc-201.