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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.

The Children's Tumor Foundation is delighted to announce that a Foundation-spearheaded paper outlining progress in NF2 clinical trials and strategies for further accelerating these trials has been accepted for publication in the American Journal of Medical Genetics.  This paper, "Consensus Recommendations for Current Treatments and Accelerating Clinical Trials for Patients with Neurofibromatosis Type 2" was authored by 17 international NF2 clinical and research experts as an outcome of a Children's Tumor Foundation-convened Workshop to accelerate NF2 clinical trials hosted in Las Vegas, NV last year.  Lead authors of the report are Dr. Jaishri Blakeley (Johns Hopkins University), Dr. D. Gareth Evans (University of Manchester) and Dr. Marco Giovannini (House Ear Institute). This is the second NF2 clinical trials consensus paper published under the auspices of the Children's Tumor Foundation. The first paper, published in Clinical Cancer Research and the result of a 2007 Foundation NF2 Workshop, helped initiate collaborations for the pioneering of NF2 clinical trials in the past few years. 

The new AJMG publication should appear in the near future.       
  

Dr. Chris Moertel, Director of the Children's Tumor Foundation Neurofibromatosis Clinic Network Affiliate Clinic at the University of Minnesota, is making progress in what has largely been an elusive area for clinical researchers - the development of vaccines to treat brain tumors. Dr. Moertel pioneered these studies by successfully halting brain tumor growth in a dog which had been injected with cell matter harvested from its own tumor.  He has since advanced the trials into seven patients with malignant glioma who have been treated with vaccine made from their own tumor matter. It is very early days but these studies look promising and will continue.  Vaccines have been challenging to develop but if successful would reduce the need for using chemotherapy drugs.  The target is currently malignant cancer but looking ahead there may be potential to apply a similar approach to neurofibromatosis tumors.  View a short TV interview with Dr. Moertel talking about the work here.