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Of course a single case report is not proof that a treatment works, but it is great to hear of individual miracles like this. I know of a few more that are just as miraculous with this drug. The journal it is published in is one of the most prestigious in the field - which means by publishing it they recognize how important an achievement this is.
As noted in the press release, our organization, the Musella Foundation (along with The Cure Starts Now Foundation, The Michael Mosier Defeat DIPG Foundation, Cancer Commons and xCures) has been a supporter of this treatment. We identified this as one of the highest priorities for us to support and we need help raising funds to help speed up this project (and others) so we can help other families have miracles too!
To recap: This experimental treatment, Onc-201, is for tumors that have the H3K27M mutation, which is also sometimes reported as a H3F3A mutation. It is mostly found in younger brain tumor patients, especially with tumors in the midline of the brain, brainstem, thalamus and DIPG. There is a clinical trial going on now for this treatment but if you do not qualify for the treatment, there is a compassionate use program that might be able to help. Contact us for details if you have this mutation!