PROGRAM & BOOK OF ABSTRACTS JUNE 13-16, 2009 • PORTLAND, OREGON New Frontiers Children’s Tumor FoundaTION 95 PINE STREET, 16TH FLOOR, NEW YORK, NY 10005 | WWW.CTF.ORG | 212.344.6633 Dear NF Conference Attendees: On behalf of the Children’s Tumor Foundation, welcome to the 2009 NF Conference: New Frontiers. The theme references the meeting content and also Portland itself, historically a gateway port of the Pacific North West. The urban setting offers a ‘new frontier’ in comparison to the mountain and beach locales of past NF Conferences, but one which we feel you will enjoy. Portland is an easy-going city offering history, beauty and relaxation – features encapsulated in our host hotel The Nines, itself a part of the tapestry of Portland history, renovated from the former landmark Meier & Frank department store. The last year has seen major NF research advances. The dovetailing of discovery, translation and the clinic can be seen throughout the meeting. We are firmly in the age of NF clinical trials and proud that the Children’s Tumor Foundation is part of this advance: in 2009 we funded our first two pilot Clinical Trial Awards. We continue to build a pipeline of candidate NF drug therapies through the Foundation’s multi-center NF Preclinical Consortium and the seed grant Drug Discovery Initiative (DDI) program. Through these translational initiatives we are cultivating NF collaborations with the biotechnology and pharmaceutical sector, a critical factor in moving NF research forward to the clinic. At the same time, basic research advances continue, such as in the unraveling of schwannomatosis. Through our Schwannomatosis Awards program, the Foundation has committed around $500,000 to schwannomatosis since INI-1 was identified as a candidate gene in 2007. This includes discovery research, genesis of schwannomatosis mouse models and now, establishing a Schwannomatosis Natural History Database. Underpinning the bench-to-bedside pathway is the Foundation’s NF Clinic Network, now 38 Affiliate Clinics in the United States and growing. NFCN strives to improve NF clinical care and to build bridges with the NF patient community. In planning is the addition of an NF BioBank and Patient Registry, to facilitate researcher access to NF tissue and clinical data, and empower NF patients to participate in accelerating NF research to the clinic. We still face many frontiers on the road to ending neurofibromatosis, but let’s celebrate the path we have carved so far. Enjoy the meeting! Sincerely, Kim Hunter-Schaedle, Ph.D. John W. Risner Chief Scientific Officer President CONTENTS Table of Contents INTRODUCTION Research Programs of the Children’s Tumor Foundation .....................................5 Foundation Staff at the 2009 NF Conference .......................................................6 Children’s Tumor Foundation National Programs .................................................7 CME Credits - NYU Welcome Letter and Course Description ................................10 INFORMATION Schedule At-A-Glance ........................................................................................16 Important Notes To Chairs, Speakers & Poster Presenters ...................................17 Vote For Your Favorite Posters ............................................................................18 Schedule ...........................................................................................................19 ABSTRACTS Keynote Presentations .......................................................................................27 Session 1: NF Clinical Trials Updates ..................................................................29 Session 2: Correlating NF Phenotype and Genotype ............................................33 Session 3A: Clinical Features of NF ....................................................................35 Session 3B: Molecular Basis of Tumors in NF .....................................................37 Session 4A: NF1 in Context: A View of Related Ras-MAPK Disorders ...................40 Session 4B: Basic - Unraveling the Signaling Pathways of NF ..............................43 Session 5B: Basic - Unraveling the Signaling Pathways of NF (cont.) ...................46 Session 6: Recent Advances in Understanding NF1 Cognitive Deficits ..................48 Session 7: NF2 and Schwannomatosis: What’s New? .........................................50 Session 8A: Novel Cellular and Animal Models of NF ...........................................53 Session 8B: Progress in Management of NF1 Bone Abnormalities .......................56 Session 9: NF Therapeutic Targets and Preclinical Drug Screening .......................60 POSTERS Poster Presentation: Session 1 (odd numbers) ...................................................63 Poster Presentation: Session 2 (even numbers) ..................................................83 APPENDIX Attendees List - 2009 NF Conference .................................................................103 Maps ................................................................................................................106 4 | Children’s Tumor Foundation • Ending Neurofibromatosis Through Research RESEARCH PROGRAMS Research Programs of the Children’s Tumor Foundation The Children’s Tumor Foundation is dedicated to ending neurofibromatosis (NF) through research. For over 25 years, Research Programs of the Children’s Tumor Foundation .....................................5 the Foundation has funded NF research with the goal of identifying NF drug therapies and improving the lives of those Foundation Staff at the 2009 NF Conference .......................................................6 living with the disorder. To date, the Foundation has committed over $30M to NF research initiatives. The Foundation also provides resources for NF patients and their families, and endeavors to increase public awareness of NF. Children’s Tumor Foundation National Programs .................................................7 CME Credits - NYU Welcome Letter and Course Description ................................10 The Foundation has been a long-time key advocate for federal support of NF research. The Foundation works closely with the National Institutes of Health and has been instrumental in the inception and continuation of the United States Schedule At-A-Glance ........................................................................................16 Army’s Congressionally Directed Medical Research Program. Important Notes To Chairs, Speakers & Poster Presenters ...................................17 Vote For Your Favorite Posters ............................................................................18 The research programs of the Children’s Tumor Foundation support discovery and translational research, clinical trials and clinical care. Schedule ...........................................................................................................19 Keynote Presentations .......................................................................................27 Open Programs: Call for Applications Session 1: NF Clinical Trials Updates ..................................................................29 • Young Investigator Awards: Our longest running research program provides two year grants for pre- and post- Session 2: Correlating NF Phenotype and Genotype ............................................33 doctoral researchers in NF research. YIA continues as a cornerstone program of the Foundation. Many of today’s Session 3A: Clinical Features of NF ....................................................................35 NF community leaders are former YIA recipients. Next deadline: early 2010. Session 3B: Molecular Basis of Tumors in NF .....................................................37 • Drug Discovery Initiative Awards: Grants of up to $50,000 for pilot preclinical drug screening. To date, this Session 4A: NF1 in Context: A View of Related Ras-MAPK Disorders ...................40 program has funded 25 drug screens. Multiple deadlines: see www.ctf.org. Session 4B: Basic - Unraveling the Signaling Pathways of NF ..............................43 • Clinical Trial Awards: Offer $125,000 to conduct small-scale pilot clinical trials of candidate NF therapeutics. Session 5B: Basic - Unraveling the Signaling Pathways of NF (cont.) ...................46 Deadline: Letter of Intent required. Contact the Foundation first. Session 6: Recent Advances in Understanding NF1 Cognitive Deficits ..................48 • NF Clinic Network (CTF-NFCN): Recognizes those clinics offering comprehensive NF clinical care. Session 7: NF2 and Schwannomatosis: What’s New? .........................................50 Now 38 Affiliate Clinics. Any clinic may apply for participation. U.S. Clinics eligible to receive seed funding to Session 8A: Novel Cellular and Animal Models of NF ...........................................53 expand clinic activities. Deadlines: February 28th and August 31st. Session 8B: Progress in Management of NF1 Bone Abnormalities .......................56 Session 9: NF Therapeutic Targets and Preclinical Drug Screening .......................60 Ongoing Programs (no current call for applications) • The NF Preclinical Consortium: part of a $5M effort to advance NF research in the clinics. Research teams at six Poster Presentation: Session 1 (odd numbers) ...................................................63 centers collaborating in screening the most promising candidate NF therapeutics in multiple