1. Partnering Opportunities & Translational Funding for Neurofibromatosis Neurofibromatosis (NF): - A rare disease - 1:3,000 - Tumor suppressor genes – NF1, NF2 -Brain/nerve tumors; benign & malignant -Learning disabilities, bone dysplasia, pain NF Research Today: -Candidate drug targets -Preclinical models - GEMs -Trial pipeline -44 site NF Clinic Network -NF BioBank & Patient Registry Why Should You Look at NF? -Cancer crossover - Ras – broad relevance -Growing commercial interest in rare diseases 1

2. 2 CTF NF Translational Pipeline Opportunities engage industry Drug Discovery Initiative Clinical Research Award $15K, $30K vitro/vivo screen candidate NF Rx $100K – pilot clinical trial or biomarker study

3. 3 Opportunities for Collaboration with CTF & NF Research FUNDING: Drug Discovery Initiative Awards $15K & $30K - Deadlines Feb & August - 3 page app - fast turnaround Clinical Research Awards $100K - LOI deadline Spring 2011 INDUSTRY/DRUGS: - Growing NF interest – CTF at BIO, FasterCures, etc. - Industry advisors on RAB – AstraZeneca, Lilly, Plexxikon... CELLS, ANIMAL MODELS, TISSUES: - Online DDI Toolbox; NF BioBank - CTF MA funded sites e.g. Harvard, MIT, Whitehead, Children’s, Tufts CLINICAL TRIALS: Clinical Trials Consortium, Patient Registry NETWORKING: 2011 NF Conference, Jackson Hole, WY June 11-14. ____________________________________________________ Kim Hunter-Schaedle, Ph.D. CSO, Children’s Tumor Foundation khs@ctf.orgwww.ctf.org - http://www.partneringforcures.org/2010_innovators/29_neurofib.htmlhttp://www.partneringforcures.org/2010_innovators/29_neurofib.html