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 three hundred 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.
The Children's Tumor Foundation NF Conference has had a remarkable history since its inception in 1985, playing a lynchpin role as the premier annual gathering of researchers and clinicians in the neurofibromatosis community. The meeting is a forum for research information exchange and discussion for neurofibromatosis researchers and clinicians from around the world, and has traditionally fostered collaboration, consensus building, and advances in translational research culminating in clinical trials. Meeting attendance has doubled from 140 attendees in 2005 to 300 attendees in 2010, 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. For over 25 years the NF Conference 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. The Conference traditionally includes high- profile keynote speakers from other areas of cancer research, neuroscience etc., to stimulate ideas and build connections between NF and other disorders. In addition the NF Conference serves as an important showcase for the work of young NF researchers, many supported by the Foundation's Young Investigator Award program. The NF conference offers the opportunity for communication and 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 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 http://www.ctf.org/For-Scientists/nf-conference.html. About Neurofibromatosis and Emerging Therapeutics Neurofibromatosis is a group of genetic disorders - NF1, NF2, and schwannamatosis - collectively known as 'NF'. NF causes tumors to grow anywhere in the nervous system;and though largely benign, 10-15% will become malignant. Even the benign tumors of NF can cause significant morbidity and even mortality because of their continuous growth and often surgical inaccessibility. NF can also cause bone abnormalities, deafness, blindness, pain, heart defects, and in 60% of cases, learning disabilities. NF affects an estimated 100,000 persons in the US. NF1 is the most predominant affecting 1:3,000 births;NF2 affects 1:25,000 births. NF1 and NF2 are autosomal dominant: fifty percent of new cases arise from spontaneous mutations with no family history of the disorder. Schwannomatosis affects an estimated 1:40,000 births and is not well understood. The genetics and molecular biology of NF1 and NF2 are fairly well understood. Using understanding of signaling defects and molecular changes therapies are being proposed. Understanding of Schwannomatosis is progressing since a candidate gene INI1 was identified. This disorder may also be linked to NF2 and other genes. In 2008, the NF clinical landscape was further expanded with the identification of another NF1-like disorder, Legius Syndrome, associated with a SPRED1 gene mutation on Chr. 15, and with a clinical presentation of 'mild'NF1. There are no effective drug treatments for any form of NF yet, but over the past several years multiple clinical trials have been initiated. The NF Clinical Trials Consortium, formed in 2005 and funded by Congressionally Directed Medical Research Program for NF (CDMRP NFRP), launched the first multi-site Phase II clinical trial for NF1 - a Phase II Rapamycin clinical trial on plexiform neurofibromas - in 2008, and a Phase II trial of Lovastatin for NF1 learning disabilities in 2009. Additional Phase stage 'pilot'trials have been or are ongoing outside of the Consortium. These include Phase 0, I and IIa trials for NF1 (lapatinib, Avastin and PTC-299). The Children's Tumor Foundation has helped fill this pilot trial pipeline by offering Clinical Trial Awards of $125,000;to date two have been funded - the lapatanib and sorafenib trials mentioned above. To help fill the preclinical pipeline of drug candidates, CTF offers Drug Discovery Initiative seed grants for NF preclinical drug screening and also supports a $4M/six-site multiyear NF Preclinical Consortium.
