Molecular Profiling PX-478

PX-478

This new oral drug in a Phase I clinical trial is an inhibitor of hypoxia-inducible factor-1 alpha (HIF-1a).  It is under development by Pro-1X Pharmaceuticals, now part of Biomira Pharmaceuticals.  For information at the site of the manufacturer, click here. 

HIF-1a promotes angiogenesis.  Of note with respect to GIST, one activation trigger for HIF-1a is the "pseudohypoxia" resulting from succinate dehydrogenase deficiencies, which have been found in some adult GIST patients, some Carney Triad patients, and almost all Carney-Stratakis syndrome patients.

For trial information link to clinicaltrials.gov