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Save the Date! The Children's Tumor Foundation's annual NF Forum will occur June 8-10, 2012 in New Orleans, LA. This year, for the first time, the Forum will be held adjacent to the NF Conference which is the world's largest gathering of NF researchers, doctors, scientists, and clinicians.

Here is a video from last year's NF Forum:

Click here for photos from 2011's NF Forum.

For more information please contact Traceann Adams at 212-344-6633, ext. 236, or tadams.com. 

Please mark your calendar and plan to attend!

The Children's Tumor Foundation is pleased to announce that it has been honored with a 4-star rating from Charity Navigator, America's largest and most-utilized independent evaluator of philanthropies. This is the 3rd year in a row that Charity Navigator has given the Foundation its highest ranking. Less than 10% of charities rated have received 4-stars for three consecutive years, which places the Foundation in prestigious company.

"Receiving four out of a possible four stars indicates that your organization adheres to good governance and other best practices that minimize the chance of unethical activities, and consistently executes its mission in a fiscally responsible way," wrote Ken Berger, president and chief executive officer of Charity Navigator, in a letter to the Foundation, "This 'exceptional' designation differentiates the Children's Tumor Foundation from its peers and demonstrates to the public that it is worthy of their trust and their donors' hard-earned dollars."

George Orfanakos, president of the Children's Tumor Foundation, stated, "Being recognized by an organization as highly regarded as Charity Navigator is extremely validating. We expect the highest standards from ourselves and it is encouraging to know that we are succeeding. This is a testament to the hard work, dedication, and efficiency of our scientific community, our generous donor base, our Board of Directors, our volunteers, and our staff, all of whom are united in the common goal of ending NF. At the Children's Tumor Foundation, we are committed to investing our precious resources efficiently and effectively into neurofibromatosis (NF) research, public education, and patient support. This 4-star rating for the third consecutive year affirms our practice of financial transparency and elevates us into the realm of the nation's most respected philanthropies. This is a community that we are proud to be a part of, and a status that we plan to sustain."

Forbes, Business Week, and Kiplinger's Financial Magazine, among others, have profiled and celebrated Charity Navigator's unique method of applying data-driven analysis to the charitable sector. Statistics show that users of Charity Navigator's site gave more than they planned to after viewing Charity Navigator's findings, and in fact, it is estimated that last year Charity Navigator influenced approximately $10 billion in charitable gifts.

To view the Children's Tumor Foundation's Charity Navigator page please click here.

To view the letter acknowledging the Foundation's rating please click here (pdf).

The story below, from the Detroit Free Press, highlights not only true grit and determination, but a community of compassion and spirit.   Special thanks to Dan Gilbert and the Quicken Family of Companies for their role in this holiday triumph. 

http://www.freep.com/article/20111224/NEWS01/112240368/Wayne-State-grad-s-dream-of-medical-school-becomes-a-reality?odyssey=mod|newswell|text|FRONTPAGE|s

House and Senate negotiators have agreed to include $12.8 million for NF research in
the 2012 Department of Defense budget. While this represents a cut from last year’s
funding level of $16 million, even more drastic reductions to NF research had been
feared.

George Orfanakos, President of the Children’s Tumor Foundation, praised NF
advocates for making their voices heard in Washington, particularly during the past two
months of budget negotiations.

“Only a full-court press from the NF community prevented further erosion of government
funding for a program that is critical to developing viable treatments for those with NF,”
he said.

The NF research funding has been included in the broader appropriations bill that is
being sent to President Obama for signature.

The Children’s Tumor Foundation has appointed Annette Bakker, Ph.D., to the newly-created position of Senior Vice President of Research and Drug Development.  The Foundation is dedicated to improving the health of those affected by neurofibromatosis, more commonly referred to as NF, and is the largest non-governmental funder of NF research nationally.

Dr. Bakker is originally from Belgium and holds a Ph.D. in cell biology from the University of Antwerp.  After more than a decade practicing basic research at University of Antwerp, Yale Medical School, and the Salpetriere Hospital in Paris, she was appointed assistant professor at University of Paris. Since 1998, Dr. Bakker has applied her basic research skills in drug discovery. Most recently she created and headed the oncology research and development unit at Siena Biotech in Italy, where she focused on drug discovery and development. Dr. Bakker’s research was internationally recognized by more than 30 peer-reviewed papers, patents, and innovation awards.
Dr. Bakker was hired after an exhaustive, international search and her appointment is part of the Foundation’s ongoing effort to expand its NF-related medical and research programs, notably in preclinical drug testing and clinical trials.  As well as having a broad background in neuroscience and cancer research, Dr. Bakker brings her research and pharmaceutical industry expertise of liaising and negotiating with biotechnology and pharmaceutical companies.
“Annette has proven experience building relationships with pharmaceutical companies and scientists across the globe, which can accelerate the development of promising treatments for patients with NF,” said George Orfanakos, President of the Children’s Tumor Foundation.

Since 2005, the Children’s Tumor Foundation’s research efforts have been led by Dr. Kim Hunter-Schaedle, Chief Scientific Officer. Earlier this month, Dr. Hunter-Schaedle resigned from her position as of yearend.  During her tenure, Dr. Hunter-Schaedle directed a major expansion of the funding and scope of Foundation-sponsored scientific and medical activities, and coordinated activities with 44 partnering treatment facilities nationwide that are part of the NF Clinic Network.

Bruce Korf, M.D., Ph.D., a respected NF researcher at the University of Alabama Birmingham, and the Chairperson of the Children's Tumor Foundation's Medical Advisory Committee, said, "As the Foundation's first Chief Scientific Officer, Kim was the architect of the CTF research program, which is now recognized as the leading privately-funded NF research program in the world."

In addition to her drug development responsibilities, Dr. Bakker will ensure a seamless transition until a new scientific officer can be hired.

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 National Institutes of Health has announced a funding opportunity for the NIH Director’s Early Independence Awards for junior investigators wishing to “skip the post-doc” and immediately begin independent research. Budgets may be up to $250,000 in direct costs per year for up to five years.

Eligible candidates must be within one year of receipt of a terminal research degree (Ph.D., etc.) or completion of clinical residency.

All areas of research relevant to the mission of NIH welcome. These are highly competitive awards but I encourage young neurofibromatosis investigators to apply given the NIH's growing interest in, and support of, rare diseases research.
The deadline for submitting Early Independence Award applications is January 30, 2012 with Letters of Intent due by December 30, 2011. See the instructions in RFA-RM-11-007. Frequently Asked Questions about the Early Independence Award Program are answered at http://commonfund.nih.gov/earlyindependence/.

Schwannomatosis is the rarest form of neurofibromatosis (NF), affecting an estimated 1:40,000 people, and it causes tumors to grow on peripheral nerves as well as severe and unmanageable pain.  It has also been the most mysterious form of NF, both in terms of accurate clinical diagnosis and in terms of understanding its biology and genetics.

In 2005, the Children's Tumor Foundation spearheaded the publication of the first diagnostic criteria for schwannomatosis.  In those initial critieria, it was stated that if a person had one or two vestibular schwannomas  (tumors on the 8th cranial nerve in the brain) then this would exclude a diagnosis of schwannomatosis and indicate a diagnosis of NF2.  In 2007,  the first candidate gene for schwannomatosis - INI1/SmarcB1/Snf5 - was identified, and though this gene does not appear to be universally involved in all cases of schwannomatosis, it has aided in the diagnosis of the disorder and in researching its molecular basis. 

Now a team led by Dr. D. Gareth Evans (University of Manchester) reports in the American Journal of Medical Genetics on two people each presenting with a unilateral (single) vestibular tumor as well as peripheral nerve tumors but for whom a diagnosis of NF2 has been eliminated through clinical or genetic evaluation.  This is an important finding, and suggests that there may be other cases elsewhere that have previously been diagnosed as NF2 that are in fact schwannomatosis.

The Children's Tumor Foundation has made significant contributions to advancing schwannomatosis research through grant funding, including establishing the first Schwannomatosis International Database, as well as organizing a series of International Schwannomatosis Workshops to spur collaboration.  In December, we'll be announcing some newly funded schwannomatosis research projects and unveiling future plans for our continued commitment to advancing this research area.

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.