Funding of Childhood Cancer, NF Research in Jeopardy

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Neurofibromatosis (NF) is a set of genetic disorders that can cause tumors to develop and grow along various types of nerves. The tumors may also affect the development of non-nervous system tissues such as skin and bone.

There are three types of NF tumors that result from mutation or loss of different tumor suppressor genes:

  • Neurofibromatosis type 1 (NF1) is the most frequent inherited cause of brain and nerve tumors. One in every 3,000 children is born with NF1, making it also one of the most common inherited human diseases worldwide. Enlargement and deformation of bones may also occur. Approximately 50% of people with NF1 also have learning disabilities. NF1 is caused by a mutation or loss of the tumor suppressor gene NF1.
  • Neurofibromatosis type 2 (NF2) is much rarer, occurring in one in 25,000 births. NF2 is characterized by the development of multiple tumors on the cranial and spinal nerves. The hallmark of NF2 is the formation of tumors that affect auditory nerves. Hearing loss beginning in the teens or early twenties is typically the first symptom of NF2. NF2 is caused by a mutation or loss of the tumor suppressor gene NF2.
  • Schwannomatosis is even rarer than NF2, affecting one in 40,000 individuals. SImilar to NF1 and NF2, Schwannomatosis tumors can develop on cranial, spinal and/or peripheral nerves. Although patients with Schwannomatosis do not have learning disabilities, they experience chronic pain and occasionally numbness, tingling and weakness. The candidate Schwannomatosis tumor suppressor gene is named INI1.

The National Institutes of Health (NIH) is the primary source of federal funding for biomedical research. However, other agencies also support research initiatives. In 1996, Congress added Neurofibromatosis to the Congressionally Directed Medical Research Program (CDMRP-NFRP). This program has been responsible for many advances in NF research, including NF mouse models, learning disabilities and nerve signaling pathways. In 2005, the Neurofibromatosis Research Program (NFRP) established the NF Clinical Trials Consortium, which is comprised of 10 major hospitals nationwide. The Consortium was established, not for drug discovery, but as a pipeline to test drugs repurposed to treat NF, including rapamycin (a relatively new immunosuppressant drug) and lovastatin (a statin used for lowering cholesterol). The Consortium will initially focus on NF1 for proof of concept. Once established, it will have the option of expanding to encompass NF2 and Schwannomatosis studies.

NF research program funding in jeopardy

The U.S. House and Senate included an $8 million appropriation for the CDMRP-NFRP in the FY2008 Defense Bill. This is a decrease of $2 million from 2007 and is over a 66% decrease from the high-water mark of $25 million in FY2005. Recently, I wrote about Flat Funding of Biomedical Research and The Threat to America’s Health. Separate from the NIH, the CDMRP is another funding source that supports research initiatives. The drastic funding cuts in the CDMRP-NFRP, specific to NF studies, endanger the research investment made to date, particularly with the NF Clinical Trials Consortium described above.

Childrens Tumor FoundationThe Children’s Tumor Foundation (CTF), a non-profit medical foundation dedicated to improving the health and well being of individuals and families affected by the neurofibromatoses, is the largest non-government funder of NF research in the world. In 1991, the CTF began a formal advocacy and lobbying program for federal funding of NF research. Recently, the CTF announced an advocacy campaign to increase federal funding of the CDMRP-NFRP [1]:

We are all aware of the budget pressures our country faces, and understand that the $25 million funded in 2005 is not realistic in the current environement. However, this small program has accomplished so much, and as we enter what we believe will be a period of rapidly increasing clinical trials, this is a particularly important time for continued support of this funding. We are asking all of you to contact your Congressman and Senators to seek their support. There is much discussion of earmark reform in Washington. It is important to note that this funding is not an earmark. It is not directed to any one institution, state or district. It is a long standing program that makes grants solely on a peer review basis. Further, this is not a partisan issue – this funding has benefited over the years from strong support from both Democrats and Republicans. The accomplishments and return on investment from the CDMRP are a shining example of what the federal government can achieve when legislators work with the scientific community and non-profit organizations.

Indeed, the CDMRP-NFRP is a small program. Congressional appropriations for NF from 1996 to 2008 totaled just $190.3 million. By comparison, CDMRP funding for breast cancer totaled $2222.7 million, for prostate cancer, $890 million [2]. Nevertheless, CDMRP funding for NF research in 2008 is critically important to address the needs of translational research (meaning to connect basic research to patient care), complications of NF with high morbidity and mortality, and refinement and standardization of imaging techniques and biomarkers for use in future clinical trials.

You can read more on the Children’s Tumor Foundation and Neurofibromatosis here at Highlight HEALTH. Additional non-profit organization resources are listed in the Highlight HEALTH Web Directory.

I’m actively involved in neuro-oncology, specifically NF research, and can attest to the importance of CDMRP-NFRP funding. I encourage you to take a moment and email your Senator and Representative and urge them to support increased Neurofibromatosis research funding through the CDMRP. You can find your legislators contact information by visiting the House and Senate websites. For the House website, simply enter your zip code in the box in the upper left corner; for the Senate website, select your state from the pulldown menu in the upper right corner. Use the contact information provided to email, fax or mail your request for support.

UPDATE: April 1st, 2008

Sample letters are now available (in MS Word format) for download, making it that much easier to email, fax or mail your Senator and Representative.


  1. The Children’s Tumor Foundation: Advocacy. Accessed 2008 Mar 30.
  2. Congressionally Directed Medical Research Programs: Funding History. Accessed 2008 Mar 30.
About the Author

Walter Jessen, Ph.D. is a Data Scientist, Digital Biologist, and Knowledge Engineer. His primary focus is to build and support expert systems, including AI (artificial intelligence) and user-generated platforms, and to identify and develop methods to capture, organize, integrate, and make accessible company knowledge. His research interests include disease biology modeling and biomarker identification. He is also a Principal at Highlight Health Media, which publishes Highlight HEALTH, and lead writer at Highlight HEALTH.