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The Drug Discovery Initiative Awards program is focused on seed funding preclinical drug testing studies on neurofibromatosis in cell or animal models, and is one of the most successful Children’s Tumor Foundation programs to date. Since this program began in 2006, we have funded over 45 DDI Awards focused on preclinical testing of drug therapies for all aspects of NF1, NF2 and schwannomatosis, from learning disabilities to tumor growth to pain. This has been an investment for the Foundation of $1 million, but we have seen our investment leveraged, as awardees have gone on to secure over $5 million in follow-on funding from federal and other sources.

We are delighted to announce the recipients of the Drug Discovery Initiative Awards from our fall 2011 round. Dr. Toshifumi Tomoda of the Beckman Research Institute receives a $15,000 in vitro Award to study autophagy-inducing compounds as candidate therapeutics for NF2. Dr. David Little of the University of Sydney receives a $30,000 in vivo Award to study MEK inhibition as a therapeutic approach in mouse models of NF1 related tibial pseudarthrosis.

We are also pleased to announce that for 2012 we are significantly increasing DDI Award levels! We now offer $25,000 for in vitro DDI Awards, $50,000 for in vivo DDI Awards, and $75,000 for Advanced DDI Awards. There are two DDI Award deadlines in 2012: February 28th and August 31st. No preliminary data is required for DDI Award applications, and cutting-edge ideas are encouraged. If you have any questions about the DDI program, please contact Min Wong at mwong@ctf.org.

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. 

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.       
  

Do you have a basic discovery that you think you can  translate to the clinic for the benefit of patients? The National Institutes of Health (NIH) Clinical Center has announced availability of funds for the 2012 Bench to Bedside Awards Program to support exactly this type of project. This unique program offers Awards of $135,000 over 2 years and supports clinical research - including in rare diseases such as neurofibromatosis. As part of its mission the program  faciliates access for researchers outside of the NIH to the institutes wealth of clinical research resources.  

A Letter of Intent is due on September 28, 2011.  More information about the program and the application process can be found at http://www.cc.nih.gov/ccc/btb/ or submit any questions by email to BenchtoBedside@mail.nih.gov.