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Cancer therapy shows promise for some brain tumors

Al's Comment:

This is the experimental drug Onc-201.    When the article says that it was largely ineffective in a pilot study for GBM, they did not point out that that study included mostly GBM patients who did not have the H3 K27M mutation, and they have since realized that this mutation makes the tumor more sensitive to this drug.

 The current clinical trials (and compassionate use program) requires the H3 K27M mutation be present. (DIPGs do not require a biopsy – it is assumed that most will have the mutation).


The mutation H3 K27M is a marker of the worst of the worst brain tumors.  Most DIPGs have it, and it is found in the midline and spinal cord gliomas usually of younger adults and kids.  Until now, there really was no hope for these tumors.  This drug gives some hope. It is not a miracle cure for everyone but it is a huge step in the right direction.  It is an oral drug with minimal side effects.  Look at your pathology reports and see if you have this mutation. If you do, ask your doctor about Onc-201.

The Musella Foundation has been a supporter of it's development since they started.

Posted on: 12/05/2018


Cancer therapy shows promise for some brain tumors


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