Scientists from the Florida campus of The Scripps Research Institute (TSRI) have identified a new drug candidate for an inherited form of cancer with no known cure.
The new study showed the drug candidate—known as FRAX97 (see structure) slowed the proliferation and progression of tumor cells in animal models of Neurofibromatosis type 2. This inherited type of cancer, caused by mutations in the anti-tumor gene NF2, leads to tumors of the auditory nerve that connects the inner ear to the brain.
The new compound, originally developed to treat neurodegenerative disease, targets a protein family known as p21-activated kinases or PAKs. These kinases (enzymes that add a phosphate group to other proteins and change their function) play a critical role in the development of Neurofibromatosis type 2. PAK1 has also been implicated in the growth of breast and lung cancers.
"Our study shows that if we inhibit these kinases we can counter the formation of tumors in this brain disease," said Joseph Kissil, a TSRI associate professor who led the study.