For this type of tumor, recurrent H3K27M mutant Diffuse Midline Glioma, there are no good historical controls - as it was only recently created as a new classification of tumor. It is thought to be worse than a regular Glioblastoma. Historically, recurrent glioblastomas has an average survival of 6 months (which can be read as 50% survival at 6 months) and we would expect recurrent H3K27M mutant DMG to be less than that. This report showed that with Onc201, 57% of patients were alive at 12 months and 35% alive at 2 years – more than doubling what I feel is the correct control. Objective response rates are hard to determine in this area - but they reported 20% response rate including one complete response, using one grading system, and 26% using a different grading system. Doesn't sound like much but there is nothing that has been working for this tumor type.. the jump from 0 to 20 or 30% is major, and I think should be good enough to get FDA approval. Once it gets approved, we can try combinations of treatment and find the best way to use it and significantly improve those numbers. The DMG-ACT trial recently opened which will try a few combinations.
We funded and helped run the first expanded access program for Onc-201 along with Cancer Commons, xCures, The Cure Starts Now foundation, the Michael Mosier Defeat DIPG Foundation, Dragon Masters Foundation and the Finn Family Foundation. Some of the patients from our program were including in this analysis. They included the first 50 patients who met the inclusion criteria from all of the trials and the expanded access program! This is your donations at work!