Personal message from Bill Knowles, Ph.D.
Dear friends,
I, like many of you, have loved ones with neurofibromatosis. My twin boys, who just celebrated their fourth birthday, were born with NF1. Through our experiences over the past few years, my family and I know personally what it is like to deal with numerous NF1-related medical issues and what it is like to worry about the medical uncertainties of the future.
I am also a pharmaceutical scientist who previously led research teams focused on finding new drug therapies to treat diabetes and metabolic disorders, and who now has turned my full attention to the neurofibromatoses (NFs). The discoveries of the NF1 (1990) and NF2 (1993) genes, as well as the discoveries of cell signaling abnormalities that result from the mutations in these genes, point to the very real possibility that NF1 and NF2 may be treated by the very same drugs that pharmaceutical companies are now testing or marketing to treat other diseases. The NF Project will initially focus on drugs that treat cancer.
From my perspective, and from the perspective of other pharmaceutical scientists, the most rational strategy for quickly finding effective therapies for the neurofibromatoses is clear. Now is the time to leverage our understanding of the NF1 and NF2 pathologies, and combine it with the success of the pharmaceutical industry in developing new cancer drugs, to implement translational research programs designed to determine whether these drugs could effectively treat tumors characteristic of NF1 and NF2. Now is the time to translate our knowledge into something that is meaningful to individuals that suffer today from the consequences of these diseases. I want therapies available in the very near future, not 15-20 years from now.
To achieve our mission, the NF Cure Project is working in partnership with pharmaceutical companies that have drugs of interest and with well-established NF scientists who can conduct NF translational research using these drugs. To date, the response from the pharmaceutical industry has been overwhelmingly positive and many drugs have been made available for our research (see Progress). I and my NF scientist partners intend to continue to develop and expand these partnerships with pharmaceutical companies until all drugs of therapeutic potential have been tested in NF models and those that are effective are progressed into NF clinical trials.
The NF Cure Project will seek funds from the NIH, from the Congressionally Directed Medical Research Program of the Dept. of Defense, from the pharmaceutical industry, from other foundations and from individuals who believe in the NF Project mission. My hope is to sustain and expand this program to achieve our mission as quickly as possible, so that individuals affected by the NFs no longer suffer from the consequence of having these diseases.
Sincerely,
Bill
