Overview of the NF Cure Project The NF Cure Project operates within a non-profit framework to arrange for the implementation of “translational research” projects that test selected approved drugs and drugs in the late stages of clinical development for efficacy in preventing the pathologies associated with the neurofibromatoses (NFs).  The drugs of initial focus are cancer drugs. The underlying rationale that drives the NF Cure Project involves taking advantage of the successes of NF researchers and clinicians in elucidating the molecular basis of certain pathologies associated with the NFs and also taking advantage of the recent successes of the pharmaceutical industry in discovering effective cancer drugs. As it turns out, the molecular bases of NF pathologies may be corrected by these relatively new drugs that the pharmaceutical industry has already developed, or is in the process of developing, to treat various cancers.   Thanks to the efforts of NF scientists and clinicians, all the components of a superb NF drug discovery program are in place for use in the testing of drugs.  In addition, the knowledge of the clinical progression of NF1 and NF2 is increasing and the methods and protocols for testing drugs for efficacy are either in place or are being developed at several clinical research centers.  Everything known about the molecular basis of NF1, and possibly NF2, suggests that these exisitng cancer drugs could be effective therapies for the NFs.   In leading the NF Cure Project, Dr. Knowles will partner with NF scientists and pharmaceutical companies to investigate whether the pharmaceutical companies’ drugs will be effective in treating the NFs.  Selected drugs will be tested in various laboratory experiments and those that are effective will be targeted for progression into human clinical trials.  Although much work remains to be done to determine whether these drug compounds are indeed effective in treating NF-related pathologies, by partnering with the pharmaceutical industry and capitalizing on their investment, hard work, and research successes, the NF Cure Project bypasses much of the time and cost associated with developing new drug therapies from ground zero (see Project Rationale).