Gleevec NF1 Trial


New NF1 Clinical Trial for the Treatment of Plexiform Neurofibromas
The NF Cure Project in association with colleagues at Indiana University / Riley's Children Hospital would like to announce a new experimental clinical trial for the treatment of NF1 plexiform neurofibromas.

The drug that is being tested, Gleevec, is a targeted therapy that inhibits several key enzymes that stimulate cell proliferation, angiogenesis and mast cell activity.  Gleevec was developed for the treatment of chronic myelogenous leukemia (CML) and was approved by the FDA in 2000.  Distinguished NF scientists have shown that these same enzymes are hyperactive in cells taken from NF1 plexiform neurofibromas and that Gleevec can restore the normal behavior of these cells.  It is therefore hypothesized that if these enzymes are indeed responsible, solely or in part, for the growth of plexiforms; then Gleevec should suppress plexiform growth and possibly decrease plexiform size.

On November 16, 2006 the Knowles family entered one of their sons in the Gleevec trial.

Details and contact information regarding this trial can be found at Gleevec NF1 trial.