Last updated: 5/8/2020
Note - this is an experimental drug - and things may change rapidly. This page is current as of 5/8/2020 but everything can change - contact us if you need to know for sure!
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 are helping to run the compassionate use program, in collaboration with xCures, The Cure Starts Now Foundation, Michael Mosier Defeat DIPG Foundation, and Oncoceutics. Patients must not be eligible for the clinical trial before they can apply for compassionate use and unfortunately there are a few qualifications: Click HERE to see the list. To get started, first email or call us, then fill out the form at Virtualtrials.com/ask - and make sure to mention that you want to get into the Onc-201 compassionate use program!
Compassionate Use OUTSIDE of the USA. Right now it is not possible to get the drug outside of the USA on compassionate use (trials should open soon outside the USA) but people who really need the drug might be able to get it by coming to the USA. You would have to come the the USA frequently and have doctors visits and tests done here. The drug is free, but the doctors visits, tests, travel, and associated expenses are not. It can get very expensive. The last few patients who did this spent over $100,000 the first year. One spent $60,000 the first day. And that does not include hospitalization - if you have problems while here, hospitali costs can easily run another $250,000-$1 million, so it is not very practical.
We are trying to expand the USA compassionate use program outside the USA. The only thing holding us back is the cost. We do not want to charge patients so I came up with the idea of asking charities in other countries to chip in to pay for it. If you are interested, email or call us and I wil explain how it works and how close we are to getting it done!
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: