Thank you, Mr. Chairman, for the opportunity to appear before you today to present testimony to the Subcommittee on the importance of continued funding for Neurofibromatosis (NF), a terrible genetic disorder directly associated with military purposes and closely linked to many common ailments widespread among the American population.
I am Karen Peluso, Executive Director of NF Inc.-New England, which is a participant in a national coalition of NF advocacy groups. I have been actively involved in creating awareness of NF and promoting scientific research in this area since 1982. I am here on behalf of the 100,000 Americans who suffer from NF, including my daughter, as well as approximately 150 million Americans who suffer from diseases linked to NF, including some of the most common forms of cancer, congenital heart disease, hypertension, and learning disabilities.
Mr. Chairman, I am requesting increased support, in the amount of $25 million, to continue the Army’s highly successful NF Research Program (NFRP). The program’s great success can be seen in the commencement of clinical trials only ten years since the discovery of the NF1 gene. Now, with NF in the expensive but critical era of clinical and translational research, scientists closely involved with the Army program have stated that the number of high-quality scientific applications justify a much larger program.
What is Neurofibromatosis (NF)?
NF is a genetic disorder involving the uncontrolled growth of tumors along the nervous system which can result in terrible disfigurement, deformity, deafness, blindness, brain tumors, cancer, and/or death. NF can also cause other abnormalities such as unsightly benign tumors across the entire body and bone deformities. In addition, approximately one-half of children with NF suffer from learning disabilities. It is the most common neurological disorder caused by a single gene. While not all NF patients suffer from the most severe symptoms, all NF patients and their families live with the uncertainty of not knowing whether they will be seriously affected one day because NF is a highly variable and progressive disease.
Approximately 100,000 Americans have NF. It appears in approximately one in every 3,500 births and strikes worldwide, without regard to gender, race or ethnicity. It is estimated that 50% of new cases result from a spontaneous mutation in an individual’s genes and 50% are inherited. There are two types of NF: NF1, which is more common, and NF2, which primarily involves acoustic neuromas and other tumors, causing deafness and balance problems.
Most strikingly, research has shown that NF is closely linked to cancer, brain tumors, learning disabilities, and heart disease, potentially affecting over 150 million Americans in this generation alone. NF’s Connection to the Military
NF research is directly linked to military purposes because it is closely linked to cancer, brain tumors, learning disabilities, brain tissue degeneration, nervous system degeneration, deafness, and balance. Because NF manifests itself in the nervous system, this Subcommittee, in past Report language, has stated that Army-supported research on NF includes important investigations into genetic mechanisms governing peripheral nerve regeneration after injury from such things as missile wounds and chemical toxins. For the same reason, this subcommittee also stated that NF may be relevant to understanding Gulf War Syndrome and to gaining a better understanding of wound healing. Today, NF research now includes important investigations into genetic mechanisms which involve not just the nervous system but also other cancers.
The Army’s Contribution to NF Research
Recognizing NF’s importance to both the military and to the general population, Congress has given the Army’s NF Research Program strong bipartisan support. After the initial three-year grants were successfully completed, Congress appropriated continued funding for the Army NF Research Program on an annual basis. From FY96 through FY03, this funding has amounted to $110.3 million, in addition to the original $8 million appropriation. Between FY96 and FY02, 299 proposals were received, of which 85 awards have been granted to researchers across the country and another 17 projects have been recommended for funding this year.
The Army program funds innovate, groundbreaking research which would not otherwise have been pursued, and it has produced major advances in NF research, such as the development of advanced animal models and clinical trials. The program has brought new researchers into the field of NF, as can be seen by the nearly 60% increase in applications in the past year along. Unfortunately, despite this increase, the number of awards has remained relatively constant over the past couple of years.
In order to ensure maximum efficiency, the Army collaborates closely with other federal agencies that are involved in NF research, such as NIH and the VA. Senior program staff from the National Cancer Institute (NCI) and the National Institute of Neurological Disorders and Stroke (NINDS), for example, have sat on the Army’s NF Research Program’s Integration Panel which sets the long-term vision and funding strategies for the program. This assures the highest scientific standard for research funding while ensuring that the Army program does not overlap with other research activities.
Because of the enormous advances that have been made as a result of the Army’s NF Research Program, research in NF has truly become one of the great success stories in the current revolution in molecular genetics, leading one major researcher to conclude that more is known about NF genetically than any other disease. Accordingly, many medical researchers believe that NF should serve as a model to study all diseases. Future Directions
The NF research community is now ready to embark on projects that translate the scientific discoveries from the lab to the clinic. This translational research holds incredible promise for NF patients, as well as for patients who suffer from many of the diseases linked to NF. This research is costly and will require an increased commitment on the federal level. Specifically, increased investment in the following areas would continue to advance NF research and are included in the Army’s NF research goals: $ Clinical trials $ Development of drug and genetic therapies $ Further development and maintenance of advanced animal models $ Expansion of biochemical research on the functions of the NF gene and discovery of new targets for drug therapy $ Natural History Studies and identification of modifier genes – such studies are already underway, and they will provide a baseline for testing potential therapies and differentiating among different phenotypes of NF $ Development of NF Centers, tissue banks, and patient registries
Fiscal Year 2004 Request
Mr. Chairman, the Army’s highly successful NF Research Program has shown tangible results and direct military application with broad implications for the general population as well. The program is now poised to fund translational and clinical research, which is the most promising yet the most expensive direction that NF research has taken. The program has succeeded in its mission to bring new researchers and new approaches to research into the field. Therefore, increased funding is now needed to take advantage of promising avenues of investigation, to continue to build on the successes of this program, and to fund this translational research thereby continuing the enormous return on the taxpayers’ investment.
In recent years, the program has granted its first two clinical trial awards but had to decline other clinical trial applications that scored in the “Excellent” range in the peer review process, solely because of limited funds. This is why scientists closely involved with Army program believe that the high quality of the scientific applications would justify a much larger program than is currently funded.
I am here today to respectfully request an appropriation of $25 million in your FY04 Department of Defense Appropriations bill for the Army Neurofibromatosis Research Program. This is a $5 million increase over the current level of funding as a step toward capitalizing on all of the research opportunities now available
Mr. Chairman, in addition to providing a clear military benefit, the DOD’s Neurofibromatosis Research Program also provides hope for the 100,000 Americans like my daughter who suffer from NF, as well as the tens of millions of Americans who suffer from NF’s related diseases such as cancer, learning disabilities, heart disease, and brain tumors. Leading researchers now believe that we are on the threshold of a treatment and a cure for this terrible disease. With this Subcommittee’s continued support, we will prevail.
Thank you for your support of this program and I appreciate the opportunity to submit this testimony to the Subcommittee.
