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First-line treatment of ONC 201 in newly diagnosed DIPG pediatric patients is now open.

Al's Comment:

 There are a few trials of this drug, as well as the compassionate use program, but a new trial opened that requires DIPG patients to start before radiation. It is hard to get the word out to those patients since things happen so fast when first diagnosed and most families are in shock.  So if you hear of a patient diagnosed with DIPG, mention this trial to them as an option.

Disclaimer:   The Musella Foundation gave a venture philanthropy grant to the company that makes Onc-201 - which means the foundation gets a return if the drug is successful.

Posted on: 10/10/2019

First-line treatment of ONC 201 in newly diagnosed DIPG pediatric patients is now open.

Onc201 is an experimental oral drug in clinical trials for DIPG.

This is a new first line treatment approach – which has to be started BEFORE radiation or any other treatments (except surgery) are started.  There are other trials, and a compassionate use program, for patients who already had radiation, but it makes the most sense to start it as early as possible and this trial will test that theory. No surgery or biopsy is required but is ok if they had it.

For details, go to and look at arm B. This trial adds the Onc-201 to standard radiation.

Please, direct families or caregivers interested in seeking help. 
For information about the compassionate use program, go to

For more details about Onc-201 go to


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