Dr. William Knowles presented the NF Cure Project concept to senior management of the National Neurofibromatosis Foundation (now the Children’s Tumor Foundation) in August 2002, two days after the NF1 diagnosis was confirmed for his twin sons. At that time, Dr. Knowles was Vice-President of Research at a privately held pharmaceutical company in Connecticut and was responsible for all aspects of the drug discovery process. One of the major drug discovery programs under his direction was the development of inhibitors of signaling pathways for the treatment of diabetes and metabolic disorders. This program was very much analogous to developing inhibitors of pathways that are thought to play a role in the NFs.
The mission of the NF Cure Project was then as it is today “To find therapies for the neurofibromatoses as soon as possible.”
The NF Cure Project, as originally conceived and as operated today, has two key features that play instrumental roles in helping to achieve the NF Cure Project mission: 1) focused efforts on obtaining from pharmaceutical companies drugs that are FDA approved, or are close to being FDA approved, to test for effectiveness in treating the NFs, and 2) operations run within a non-profit framework. Working together, these two features allow the NF Cure Project to conduct streamlined negotiations with pharmaceutical companies to quickly obtain drugs that have the highest likelihood of showing promise in treating NF, and moving into NF clinical testing in a speedy and cost-effective manner (see Project rationale).
Dr. Knowles was also invited to present the NF Cure Project concept to the National Institutes of Health (NIH) in Dec. 2003.
The Children’s Tumor Foundation Board voted to provide “seed money” for one year (July 2004 to June 2005) to allow the NF Cure Project to get off the ground.
The NF Cure Project currently operates as an independent entity in alliance with, and sponsored by, Neurofibromatosis, Inc.