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We are delighted to announce that the report from the Children's Tumor Foundation 2011 Neurofibromatosis Conference has been published in the November 16th issue of the scientific journal Acta Neuropathologica. 
Held in Jackson Hole, WY in June, the 2011 NF Conference was our biggest to date attracting over 300 international NF researchers and clinicians.  We thank the Conference Co-Chairs Dr. Nancy Ratner (Cincinnati Children's Hospital Medical Center) and Dr. Michel Kalamarides (Hopital Beaujon, Paris) for their leadership in both the Conference and the publication.
This marks the third year in a row for publishing the NF Conference report in a scientific journal. The 2009 and 2010 NF Conference reports were published in the American Journal of Medical Genetics. The goal of these publications is to inform and update the broader research and clinical community on the exciting progress being made in NF research and to encourage more persons to focus on NF research.
The 2012 NF Conference takes place June 9-12, 2012 in New Orleans, LA. Look for more announcements soon!

The neurofibromatosis clinical team at the Cincinnati Children's Hospital Medical Center is conducting a research survey to find out if people with neurofibromatosis type 1 (NF1) break their bones more often and have different types of broken bones (fractures) than those who do not have NF1. Adults 40-70 years old with NF1 and their spouses and/or siblings who do not have NF1 may be eligible to participate. The information learned from this research study may indirectly benefit other patients with NF1 in the future by providing a better understanding of NF1-associated bone disease.  

This study consists of completing a survey that is estimated to take a total of 20 minutes and contains questions about participants’ fracture history, physical activity, and diet.

For more information on the study, click here.  

The Children's Tumor Foundation is delighted to announce our co-sponsorship of the third "State of the Art" International NF2 Conference to be held at the Manchester Conference Center, United Kingdom, May 21-22, 2012. The meeting will be hosted by the Manchester University NF2 Multidisciplinary Team.

The "State of the Art" meeting is a key event for the NF2 clinical and research community.  Past "State of the Art" meetings were held in Paris (2006) and Las Vegas (2010) and attracted attendance by the world leadership of NF2 clinical care, scientific and translational research, and clinical trials.  The 2012 meeting will focus on Epidemiology, Genetics and Natural History of NF2, NF2 Surgery and Radiosurgery, Auditory Rehabilitation and Animal Models and preclinical and clinical trials. 

Given the tremendous progress made in NF2 research and clinical trials in recent years we anticipate a very exciting meeting in 2012!  As with previous "State of the Art" meetings, the aim is to encourage discussion in a relaxed setting, and Manchester is sure to provide a unique venue.

For more information: http://www.nf2international2012.co.uk/

                As neurofibromatosis clinical trials increase in number, the clinicians leading them are keen to design the trials to be as effective and meaningful as possible. A key part of this effort is developing the right trial endpoints - measures and metrics that can be used to determine if a drug or intervention is effective or not.  To tackle this area, a team of neurofibromatosis clinicians and researchers has formed  a working group called Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS). Spearheaded by Dr. Scott Plotkin (MGH) and Dr. Brigitte Widemann (NCI) and first convened in June at the Children’s Tumor Foundation 2011 NF Conference, around 30 leading NF clinicans and researchers participating in the REiNS met in Boston to continue their planning and discussions.

                Past endpoints for neurofibromatosis clinical trials have included changes in maximum tumor dimension on MRI scans or changes in cognitive function on neuropsychological assessment (for learning disabilities).  Looking ahead, more advanced endpoints under discussion (and already being piloted in some cases) include volumetric tumor analysis and whole body MRIs; and the use of biomarkers – biological indicators in the blood or other body fluids to determine if a drug is working. REiNS members have organized into groups to focus on different measures and plan to meet every few months to continue advancing this project.

                The Children’s Tumor Foundation is delighted to be investing in endpoint development through our Clinical Research Awards program.  Currently our funded projects include developing a computerized test for more accurate assessment of learning disabilities trials; identifying a blood biomarker of NF1 status; and developing measures of response for optic pathway glioma trials. We will be announcing more funded awards in early 2012. 

                Clinicians or researchers interested in participating in REiNS can contact Vanessa Merker at MGH: vmerker@partners.org. 

Clinical trials are showing that drugs don’t always work on every person with a specific medical condition. This is most likely due to genetic differences that make some patients less responsive to specific drugs. Understanding an individual’s genetics is likely to be a key driver in future decision making on drug selection for individual patients. However, pharmaceutical companies have not yet fully embraced the practice of integrating genetic information into clinical trials, perhaps due to increased cost or the risk of introducing yet another variable into a trial. So if the pharma companies are not advancing this research, who will do this?

The answer may well rest with medical foundations like ours. Last week, the Multiple Myeloma Research Foundation (MMRF) launched a “Personalized Medicine Initiative” – a 1,000-patient study that will track patients from multiple myeloma diagnosis through treatment, over a minimum of five years. Sequential tissue sampling will identify how a person’s molecular profile may affect his or her clinical progression and response to treatment. This is a major undertaking for a foundation, but MMRF is addressing this need as no one else is doing this type of study.  Read more in this article.

Per our 2011 NF Strategic Plan, the Children’s Tumor Foundation will launch major new neurofibromatosis initiatives in genetics/genomics in 2012 to better understand how NF can be targeted by drug therapies. The Foundation has also recently began to fund research to identify biomarkers (e.g. blood components) that can serve as a surrogate measure and early predictor of whether or not a drug is being effective in treating NF in any one person. We will be expanding our biomarkers research in 2012.