Annette Bakker, Salvatore La Rosa, Marco Nievo. Children’s Tumor Foundation.

The Children’s Tumor Foundation is the worlds leading non-government funder of NF research to find effective treatments for neurofibromatosis. Foundation-funded research was critical in isolating the genes that cause NF and is now focused on finding treatments to improve the lives of those living with the disorder. This includes the foundation’s active participation in drug discovery research and clinical trials. We sponsor a network of 44 NF Clinics throughout the country to improve access to quality care for those with NF, and ensure best practices in treating the disorder.

The key research goals for the Foundation are to attract pharmaceutical industry and biotech to the NF field via a collaborative business model, actively bridging academic science to industry to patients. Weoffer an industry-level testing platform and the necessary tools to allow preclinical proof-of- concept testing of both existingmolecules (that are in development or on the market for other disease indications) as well as innovative still undisclosed molecular mechanisms. The Children’s TumorFoundation continues its active scouting for compounds that may benefit NF patients, and expands the necessary services that allow clinical trials, such as the NF PatientRegistry, a biobank, and the development of consented efficacy outcome measures (REINS).

This new business model, allowing CTF to be a partner/collaborator with academia and industry, rather than only a funder, will be presented. Multiple pilots along these lines are currently ongoing.