Al's Comment:

 The tumor type is Papillary craniopharyngiomas. Although this is a small study of 16 patients, the amazing thing is that no patient’s tumor progressed while on vemurafenib/cobimetinib, and none have died.
The treatment is a combination of a BRAF/MEK inhibitor (vemurafenib/cobimetinib). 
   If you have any tumor (not just brain - but pancreatic, ovarian, colon, melanoma, etc) with alterations in BRAF / MEK, it may be worth it to show your doctor this article.  Both drugs are FDA approved so this should be easy to get - although expensive at about $13,000 per month.   If you can not get these drugs, xCures (our partner) is running an expanded access program for another MAPK inhibitor for tumors with any of these alterations: KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations.

This shows the importance of getting genomic testing for your tumor.  It has actionable results in a minority of cases but when it find the right markers, it opens the door to using targeted drugs that may help a lot.

Posted on: 07/14/2023

Rare Brain Tumor Responds to Targeted Tumor Treatment with ‘Unprecedented’ Success


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