Hopes and Cures

Hopes and Cures spokes to John Risner, President of The Children’s Tumor Foundation about the group’s work, in particular about funding for research.  CTF is a non-profit 501(c) (3) medical foundation, dedicated to improving the health and well being of individuals and families affected by the neurofibromatosis (NF).

ABOUT NF

Neurofibromatosis (NF) is the term for three distinct genetic disorders, NF1, NF2 and Schwannomatosis.   The most common form, NF1 (1-3,000 births), has a wide range of severity, ranging from café au lait spots to learning disabilities, bone abnormalities, and brain and spinal tumors.  NF2 (1-25,000 births) is characterized by multiple brain and spinal tumors, commonly causing deafness, severe balance problems, decreased mobility and vision loss.  Schwannomatosis (1-40,000 births) causes nerve tumors associated with chronic pain.   NF affects both sexes and all races and ethnic groups equally.  NF is more prevalent than cystic fibrosis, Duchenne muscular dystrophy and Huntington’s disease combined. 

Ed. Note: The University of Utah describes possible symptoms of NF1

The Children’s Tumor Foundation focuses on four areas:

1) Research.  We fund, lobby for, and promote collaboration to increase research.  We are the largest non-government funder for this type of research.  We provide very early funding, what we call phase 0.5, and develop grant mechanisms to fill gaps in the research landscape where small amounts can have a large impact.  Projects we support in early stages often count on SBIR funding for phase 1 and 2 research.  

2) Public education.  We do a lot of advocacy work as well as family-oriented events to raise awareness of this illness.  Our website is a place that brings together patients, family members and scientists in a meaningful way. 

3) Clinical centers.   In an effort to increase the clinical centers for NF patients, CTF has followed the model developed by the Cystic Fibrosis Foundation.  We have developed a grant program to fund clinic coordinators in hospitals so that they can provide increased patient support, counseling and better track patient data.  Data is a key asset for clinical trials, and this step is important to enhance the appeal of NF to pharmaceutical companies to encourage more trials.  In addition, it provides the hospitals with assistance in supporting their physicians and coordinating the multidisciplinary needs of the NF community. 

4) Patient support.  The website itself is a great tool to help patients and parents of patients connect with each other, through chat rooms and discussion boards.  While the work done is focused nationally, it is not uncommon for international patients to also use the discussion board.  Also our Chapters and Affiliates nationwide provide a wealth of patient support group meetings and local services.

Drug Discovery Initiative

Currently there is no effective drug treatment for NF.  Because of this, we provide seed grants to encourage early-stage research for preclinical testing.  We launched this program in response to declining federal funding of research over the past few years.   The program has a simple 2-page application and a quick (3 month) turnaround.  These grants aren’t large (11-55k), but they can help get research for a treatment off the ground. 

As a requirement of funding, researchers post their models on our website.  It’s like Craigslist for research.  By posting the research to a public area, researchers are able to find each other easily and work can be a collaborative effort.  It’s a way to open up the drug pipeline.  This model is also similar to what other foundations have adopted, but on a smaller scale and at an earlier stage in the research.  One of the projects we funded last year at Ohio State was to test their small molecule compound, OSU03012 in NF mouse models.  Earlier this year, Arno Therapeutics, a biotech startup, licensed the compound from Ohio State.   This is the first one licensed, but we hope to see more. Because this program focuses on early stage research, we have not funded Phase I or II trials; we fund before SBIR can.   However, a lot of the work that we fund does end up being accomplished at the biotech level.  

The Children’s Tumor Foundation is very supportive of the House-passed SBIR bill.  We believe that the previous changes – to disallow VC-backed firms from competing for this Federal funding cut out the heart of the program.  Restoring funding for VC-backed firms makes sense, and will encourage continued investment in drug discovery and development.

The National Neurofibromatosis Foundation was founded in 1978 by Allan E. Rubenstein, M.D., Lynne Courtemanche Shapirio R.N., and Joel Hirschtritt, Esq.  In January 2005, the Foundation changed its name to the Children’s Tumor Foundation. The Foundation has a membership of over 50,000 constituents in all 50 of the United States, Washington, D.C., and 55 other countries.