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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

Partnering Opportunities & Translational Funding for Neurofibromatosis Neurofibromatosis (NF): - A rare disease - 1:3,000 - Tumor suppressor genes – NF1,

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Page 1: Partnering Opportunities & Translational Funding for Neurofibromatosis Neurofibromatosis (NF): - A rare disease - 1:3,000 - Tumor suppressor genes – NF1,

 

 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

   

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Page 3: Partnering Opportunities & Translational Funding for Neurofibromatosis Neurofibromatosis (NF): - A rare disease - 1:3,000 - Tumor suppressor genes – NF1,

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Opportunities for Collaboration with CTF & NF ResearchFUNDING:Drug Discovery Initiative Awards $15K & $30K

- Deadlines Feb & August - 3 page app - fast turnaroundClinical 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

[email protected] www.ctf.org - http://www.partneringforcures.org/2010_innovators/29_neurofib.html