ja_mageia

As many of you know, the primary federal support for NF research comes from the Congressionally Directed Medical Research Program (CDMRP) at the Department of Defense.  NF has been supported through this program since 1996, and this funding has been critical to the great strides we have made in NF research. With CDMRP funds, researchers have been able to:

- Identify the major functions of the NF genes in both their normal and mutated states.

- Develop sophisticated animal models which are now used for pre-clinical drug testing. 

- Create the NF Clinical Trials Consortium, the first forum for NF clinical researchers to truly collaborate on clinical trials.

- BEGIN TESTING DRUGS!  

All of this work over the years has allowed the CDMRP to begin focusing on clinical trials.  The Clinical Trials Consortium, which was established by this program in 2006, is the most important part of this program.

Increasing pressures on the defense budget are resulting in cuts to various programs, and we need your help in sending the message to Congress that federal funding for NF research should continue at a robust level. This is more important now than ever before.  The FY2012 Defense Appropriations Bill passed by the House of Representatives earlier this year included $12.8 million for NF research. The Senate did not provide this level of funding in its version of the bill.  We need your help in requesting that Congress retain the House-passed funding level for the NF CDMRP when House and Senate leaders meet to negotiate the differences in their two bills. Time is of the essence - please write your Member of Congress today!  A template letter of support is included on this page to help make this easy. 

We are at a critical stage for NF research.  CDMRP funding has brought NF research to the commencement of clinical trials, and continued funding will allow new drugs to be tested through the Clinical Trials Consortium, helping us move closer to the goal of therapies for NF and conditions related to this debilitating disorder.  Thank you for your continued support and for reaching out to Congress in support of federal NF research funding!

The National Institutes of Health (NIH) has a program called the Common Fund that supports exceptionally innovative and potentially high impact programs that are inherently high-risk but have the potential for high-payoff by catalyzing research across all of NIH and in the biomedical research community. These programs are managed by the NIH Office of the Director in partnership with the various NIH Institutes, Centers and Offices. Additional information about the NIH Common Fund can be found at http://commonfund.nih.gov.
New ideas for Common Fund programs are identified annually, and public comment is encouraged. NIH is now asking for YOUR input for 2013 programs.  Which ideas you think have the potential to fundamentally change how we think about, support, or do research in a specific field, or to create a new field all together?
Provide your input today:  http://commonfund.nih.gov/strategicplanning
The feedback period is open until Wednesday, September 14, 2011.

This weekend the Children's Tumor Foundation participated with around 100 researchers,  clinicians and foundation representatives in the Second International Rasopathies Network Conference in Chicago.  The Rasopathies Network was conceived through a collaboration between physicians focused on, and families affected by, 'Rasopathies' - or rare diseases that affect signaling in the Ras pathway. Rasopathies include neurofibromatosis, Noonan's Syndrome, Costello Syndrome, Cardio-Facio-Cutaneous (CFC) Syndrome and Leopard Syndrome. These rare conditions share many common features, including learning disabilities, and skin and bone manifestations, with impact on quality and span of life. The goal of the Rasopathies Network is to drive collaboration between scientists working on the different rare diseases to accelerate research. The  2011 Rasopathies Network Conference was organized to occur at the end of a six-day schedule of family conferences and clinics for Noonan's, Costello and CFC families, which made for a busy and informative time.  Overall neurofibromatosis research has made significant advances compared to the other rare diseases and we  got some good words for our NF Clinic Network and preclinical programs.  But we can definitely learn from some of the approaches the other groups are taking, for example to develop patient health records, and to improve clinical care guidelines. As candidate drugs emerge, it is likely that they will have applicability across many of these rasopathies therefore ongoing communication is vital as the research moves forward.  At the close of the weekend CTF and the NF Network (formerly NF Inc) jointly participated in a small NF Symposium for local attendees where Alcino Silva (UCLA) and Maria Acosta (Children's National)  gave updates on learning disabilities research and trials; I presented some highlights from the recent 2011 NF Conference;  and Kim Bischoff (NF Network) gave a presentation on the importance of advocacy for NGF research funding. 
We looks forward to the 2013 meeting of this group, and to future collaborations with the Rasopathies Network and to both sharing our knowledge and learning a lot!

An article in this week's Time magazine 'Check your Charity' highlights parameters you should look for if considering supporting a medical research charity or foundation. Issues highlighted include - what percentage of the funds raised actually go to medical research programs?  Is the charity or foundation monitoring outcome metrics to demonstrate progress and success? Children's Tumor Foundation has already demonstrated fiscal success through our maintained 4 Star Charity Navigator rating and we received a mention in the New York Times last December in reference to wise charitable investments.  Now further recognition has come from our metrics of success. In December 2010 the Children's Tumor Foundation was invited by to join Mr. Mike Milken's  FasterCures group TRAIN (The Research Acceleration and Innovation Network). The FasterCures initiative is focused on bringing business acumen to medical foundations, having them set and live up to metrics, and to place foundations as the central pivot of research progress in the foundation/government/industry triangle.  The FasterCures TRAIN is a select group of foundations such as the Multiple Myeloma Research Foundation and Michael J Fox Foundation that have demonstrated innovative approaches to advancing research progress as well as developing successful approaches to monitoring the efficacy of our research programs.  This week's Time article highlights the value of TRAIN, in the words of FasterCures Executive Director Margaret Anderson, "those that are willing to change want to learn from other groups ... and believe in 'Let's make it as efficient as possible.'"
The Children's Tumor Foundation is delighted to be among the select foundations in TRAIN and will continue to strive to make our neurofibromatosis research programs as effective and accountable as possible.

The Neurofibromatosis Research Program (NFRP) has announced the funding mechanisms for the FY11 appropriation of $16M. For full info visit: http://cdmrp.army.mil/funding/nfrp.shtml

Clinical Consortium Award - Maximum funding $9M for direct costs

Clinical Trial Award - Maximum funding $900K for direct costs

Exploration-Hypothesis Development Award - Maximum funding $100K for direct costs

Investigator-Initiated Research Award - Maximum funding $525K or $675K for Optional Qualified Collaborator for direct costs

Investigator-Initiated Focused Research Award - Maximum funding $525K or $675K for Optional Qualified Collaborator for direct costs

New Investigator Award - Maximum funding $400K in direct costs

Postdoctoral Traineeship Award - Maximum funding $100K in direct costs