The Neurofibromatosis (NF) Conference has been organized by the Children's Tumor Foundation (CTF) annually since 1985. With its roots in a small workshop-style gathering of a group called the """"""""NF Consortium"""""""" dedicated to cloning the genes underlying the Neurofibromatoses, the NF Conference has grown to a gathering of well over 350 NF researchers and clinicians from around the world. This meeting is recognized as the premier annual gathering of international NF researchers and physicians. Major contributors to this growth are the significant advances made in NF research in recent years and, particularly, recent advances into clinical trials and drug therapy development. In addition, state of-the-art genomic approaches have identified mutations in the NF genes as contributors to numerous types of sporadic cancers. Signaling pathways including the Hippo and Ras cascades are implicated in NF, and it is now recognized that mutations in genes along the Ras pathway result in a set of disorders, including NF1, called the """"""""Rasopathies"""""""". Traditionally a forum for research information exchange and consensus building, in recent years the NF Conference is also the principal international forum for reporting on the neurofibromatosis preclinical therapeutic pipeline and the expanding arena of neurofibromatosis clinical trials. Children's Tumor Foundation: 2013 NF Conference PI: Plotkin, Scott, M.D. 1

Public Health Relevance

The Children's Tumor Foundation NF Conference has a remarkable presence in the neurofibromatosis community. It plays a lynchpin role in accelerating neurofibromatosis research progress by serving as the premier annual gathering of researchers and clinicians in the neurofibromatosis community. The Conference provides a forum for research information exchange and discussion for neurofibromatosis researchers and clinicians from around the world. It fosters collaboration and consensus building, and has undoubtedly aided in the advances in translational research that have yielded the growing pipeline of NF clinical trials our community has today. Conference attendance has doubled from 120 attendees in 2005 to over 300 attendees in 2011. This has been driven by integration of new signaling pathways into NF research, the appreciation for NF pathways in sporadic diseases, and an expanding pre-clinical and clinical agenda component as the neurofibromatosis field progresses bench to bedside. About the NF Conference For over 25 years a neurofibromatosis research meeting has been organized and convened annually by the Children's Tumor Foundation, providing a forum to bring together basic and clinical NF investigators to share the latest research progress and focus on key issues in the NF research landscape. Until 2006 known as the NF Consortium, the meeting was re-branded and marketed in 2007 as The NF Conference to increase visibility and expand attendance. The meeting has always included high-profile keynote speakers from NF. Over the past few years, we have increased the presence of high-profile speakers from other areas of cancer research, neuroscience etc., to stimulate ideas and build connections between NF and other disorders. In addition we have elevated the presence at the NF Conference of young NF researchers, many supported by the Foundation's Young Investigator Award program, ensuring platform presentation slots for a proportion of these each year. The NF conference offers a unique opportunity for collaboration and, over the past couple of years has expanded from a largely basic research focus to also become the forum for sharing the latest in NF translational research and pre-clinical and clinical trials. However, even with this growth from 120 to over 300 attendees, the Foundation has strived to keep an intimate boutique feel to the NF Conference to ensure discussion and interaction. Abstract Books and summary reports from prior NF Conferences can be viewed online at About Neurofibromatosis and Emerging Therapeutics About Neurofibromatosis and Emerging Therapeutics Neurofibromatosis is a group of disorders - NF1, NF2, and schwannomatosis - collectively known as NF. . NF affects an estimated 100,000 persons in the US. NF1 is the most common, affecting 1:3,000 births;NF2 affects 1:25,000 births and schwannomatosis 1:40,000 births. The NFs are both tumor-suppressor syndromes and multisystem genetic disorders. NF causes tumors to grow anywhere in the nervous system;and though largely benign, 10-15% will become malignant. Even the histologically benign tumors of NF can cause significant morbidity and mortality because of their continuous growth and surgical inaccessibility. As a genetic disorder, NF can cause bone abnormalities, neuropathy, visual loss, pain, heart defects, and learning disabilitiesNF1 and NF2 are autosomal dominant genetic disorders: about fifty percent of new cases arise from spontaneous mutations with no family history of the disorder. The genetics and molecular biology of NF1 and NF2 are fairly well understood;in contrast, the understanding of schwannomatosis is less advanced. New therapies are being developed for NF1 and NF2 as our understanding of related signaling defects and molecular changes improves. Similarly, the identification of germ line SMARCB1 and somatic NF2 mutations in schwannomatosis has stimulated interest in the disorder. There are no approved drug treatments for NF yet, but over the past several years multiple clinical trials have been initiated. Over 30 open NF-specific trials are now listed on, funded by the DOD NF Clinical Trials Consortium, the Children's Tumor Foundation;and by additional sources including NIH funds. Children's Tumor Foundation: 2013 NF Conference PI: Plotkin, Scott M.D., PhD 1 NOTE: The critiques and criterion scores from individual reviewers are provided below in an essentially unedited form. The Resume and Summary of Discussion above summarizes the final outcome of the group discussion.

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National Institute of Neurological Disorders and Stroke (NINDS)
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National Institute of Neurological Disorders and Stroke Initial Review Group (NSD)
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Morris, Jill A
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Strowd 3rd, Roy E; Rodriguez, Fausto J; McLendon, Roger E et al. (2016) Histologically benign, clinically aggressive: Progressive non-optic pathway pilocytic astrocytomas in adults with NF1. Am J Med Genet A 170:1455-61
Plotkin, Scott R; Albers, Anne C; Babovic-Vuksanovic, Dusica et al. (2014) Update from the 2013 International Neurofibromatosis Conference. Am J Med Genet A 164A:2969-78