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NIAMS coalition this year will send a powerful message and challenge to the research community.
Last year the needs of the patients and the patient advocate groups were heard and the chronic underfunding of NIAMS was partially alleviated by the increases you provided. Seventy percent of those additional funds will go into funding 72 more individual research grants during fiscal year 1991 than in fiscal year 1990. NIAMS funding rates will be able to increase from 16.3 percent to 22 percent, still below the average funding percentile for NIH as a whole. With these added funds, more grants were funded in important areas, so that the role of growth factors in scleroderma, the importance of the immune system in psoriasis, and the mechanism of blistering diseases in the elderly could be studied in more detail. However, there were still many research opportunities which could not be investigated further because of the restricted funding environment.
RECENT SKIN RESEARCH SUCCESSES
Prevention of disease and the maintenance of health is the ultimate aim of medicine. Prevention is also a key element in containing the cost of medical care in our country, and can only be based on sound and detailed knowledge of normal and altered biology. Prevention is also a process in which the individual who may be susceptible to a disease is empowered and is an active participant in the process of protecting health. Health is, therefore, a concern and a partnership of the patients, physicians, nurses, and other health personnel. Prevention is a long-term effort to which dermatology is committed. I will now discuss our efforts in the prevention and understanding of skin cancer.
Skin cancer is a growing epidemic in all parts of our nation; in the sunbelt as well as in the northern climes. Many factors are related to this epidemic including increased sun exposure related to changes in leisure activities and life style, recently identified genetic factors, and probably unidentified factors. All types of skin cancers, including basal cell, squamous cell carcinomas and malignant melanoma have increased over the past two decades.
Dermatology has responded to this epidemic in several ways. The American Academy of Dermatology has created a massive public information campaign on the dangers of excessive sun exposure, including an education program cosponsored by the American Cancer Society, emphasizing sunburn prevention in the lower grades of elementary school. This is extremely important since individuals receive more than one-half of their total sun exposure before the age of 18.
In addition, the American Academy of Dermatology has, for the past seven years promoted a national melanoma and skin cancer screening program each May. Onethird of the dermatologists in the nation volunteer to provide free skin cancer screening. Last year, almost 100,000 people were screened and almost 1,000 lesions suspected to be melanoma, the most potentially fatal form of skin cancer, were found.
There is significant research funded by NIAMS which is directly aimed at understanding the mechanisms of skin cancer; this research is necessary for a proper long-term program in prevention. These studies include defining the factors controlling melanocyte growth, the cells which produce the pigment in the skin and which change in melanoma. Some forms of melanoma are genetic, and efforts to map and eventually to characterize the genes which make individuals susceptible to melanoma are in progress.
The keratinocyte, the cell which forms the bulk of the epidermis, the outer layer of our skin, is the cell which is causing the most common skin tumors, basal cell and squamous cell carcinomas. Extensive studies funded by the institute have allowed characterization of the proteins which make up the constituents of these cells. Studies of the growth control of these cells and the role of growth factors and oncogenes in relation to the keratinocyte are in progress. The role of sunlight on the immune system in skin is very important and no doubt plays a major role in the maintenance and progression of skin cancer. Studies of how the local immune effects are translated into the generalized immune efforts which are seen after sun exposure are also under study. A small molecule, IL-1, is released from the outer layers of the skin after sunburn and is the cause of the fever many have experienced after severe sunburn. Vitamin A has profound effects on the skin, and the use of systemic vitamin A analogues, such as Accutane (the same agents used to treat severe acne), is able to prevent skin cancers in individuals with two genetic disorders giving abnormal susceptibility to ultraviolet radiation, xeroderma pigmentosum and the basal cell nevus syndrome.
This year two of the genes defective in xeroderma pigmentosum have been identified. Understanding how vitamin A controls and regulates the genes of keratinocytes
and other cells in the skin is a major research interest of the institute. In addition, several proteins which are receptors and the carriers of vitamin A to the genes of the cells have been identified, and the exact sites on the genes which interact with the vitamin A receptors have been characterized in the past year.
What remains to be done and what are the opportunities? Skin cancer begins slowly in one or a few cells in any site. There is a long lag-time during which there are small numbers of malignant cells still confined to the skin. These are currently treated by the best way possible today: early diagnosis by the dermatologist and then surgical removal. We need earlier diagnostic techniques and medical treatments to prevent or reverse the early changes in the skin even before the abnormal lesions are visible on the skin.
The medical costs of skin cancer to the American population are very large. At least 600-00 new cases of skin cancer will be diagnosed and treated this year. The estimated annual cost of skin cancer, including melanoma treatment, is at least $400,000,000 per year in direct medical costs, not including loss of income. There are each year 6,300 deaths from melanoma and 2,000 deaths from non-melanoma skin cancers. If the cost for one year of treatment was invested in research over & ten-year period, it could have tremendous monetary savings in addition to the nonmonetary benefit of saving lives. There is precedent from other biomedical advances that the annual saving realized from research can be up to 13 times the total cost of that research. Research in understanding the process of skin cancer and preventing it is a prudent investment in the health of the people of this country, as well as an excellent financial investment.
I am very optimistic that we are positioned to make more research advances than current funding allows because researchers now have the scientific tools and methodologies to study important dermatological health problems such as skin cancers, psoriasis, atopic dermatitis, acne, lupus, and ichthyosis, to name a few. You, and we, have built a magnificent research structure capable of performing important research; however, funding for the research performed at NIAMS is at a level which prevents us from progressing at full speed. While we work at this lower funding level, there is unnecessary suffering and there are patients who are not getting full benefits of the research which could be done today.
Due to the generosity and support of this committee, the study section at the National Institutes of Health, which I chair, in which most of the skin research grants are reviewed, will be able to fund 22 percent of the grants which we consider scientifically acceptable. However, I also know that the grants between 22 and 40 percent are excellent and failure to fund them frustrates able young investigators. Those grants propose important research which will lead to important answers. They contain ideas and hypotheses which, if they could be studied, could have a large payoff for health, and savings in medical costs.
CURRENT RESEARCH NEEDS
NIAMS and other institutes recently sponsored a conference on gene mapping, the principle of reverse genetics which can be used to elucidate the basic defect in several important skin diseases, including psoriasis, atopic dermatitis and other genetically determined diseases of the skin. This strategy has already defined the basic defect in neurofibromatosis and cystic fibrosis. Thus, the scientific methodology exists; and now all these labor intensive projects require new research funds to create major advances. These are the kinds of studies which could be barely envisioned when the NIAMS was founded five years ago.
A very successful conference was held at NIH in October which was cosponsored by NIAMS and the Alopecia Areata Foundation. There was great excitement over the advances in understanding the biology of the hair follicle. NIAMS-sponsored research revealed a new source for the cells which cause the hair to regrow. A rat model for alopecia areata suggests several new avenues of research which can benefit the 1 percent of the population which is afflicted by this disorder, and which may profoundly affect the self-image, self-confidence and the spirit of the affected patient.
We strongly support the recommendations of the Coalition for the National Institute of Arthritis and Musculoskeletal and Skin Diseases which requests $47 million to be added to the fiscal year 1992 budget over the President's budget. This will permit the NIAMS overall to:
-Achieve true parity with other NIH institutes;
-Increase RO-1 funding from 27 percent to 33 percent of approved grants;
-Train additional researchers to a total of 328;
-Increase the intramural research to $21.2 million making it more on par with the funding of the intramural programs of other institutes; and
-Give the research community, especially researchers at the beginning of their careers, the funding to continue developing and refining the knowledge which will lead to the understanding and eventually the prevention of chronic disease which limit the body and the spirits of those afflicted with them.
We also support the Coalition for Health Funding's request of $9.77 billion for the NIH overall, which would provide increases for the other institutes which support skin disease-related research, such as the National Cancer Institute, the National Institute on Aging, the National Institute for Occupational Safety and Health, and the National Institute of Allergy and Infectious Diseases.
Senator BURDICK. Thank you. Can you tell us if there are any new therapies for the treatment of skin cancer?
Dr. GOLDSMITH. Yes; skin cancer has usually been treated with a variety of surgical therapies, and people have been very concerned about undergoing the knife. Many people are shy of undergoing the knife.
There have been recent trials with interferon, which is an injection which is effective in some forms of skin cancer, and there are forms of vitamin A, which can be given orally, which are able to prevent skin cancer. So I think that with those at least two new forms of therapy we have great hope that there can be other therapies and other preventive therapies.
For instance, we probably need an oral sunburn pill. All of us have to slather ourselves with sunscreen, which is really a pain for anyone who has really tried to do this. And these are the kind of things we need research for.
Thank you for your attention.
STATEMENT OF BARBARA BUTLER, CHAIRPERSON, COALITION OF PATIENT ADVOCATES FOR SKIN DISEASE RESEARCH
Senator BURDICK. Our next witness will be Barbara Butler, Coalition of Patient Advocates for Skin Disease Research.
Ms. BUTLER. Mr. Chairman, my name is Barbara Butler, and I am the chairperson for the Coalition of Patient Advocates for Skin Disease Research.
We wish to thank the committee for the support they gave the National Institute of Arthritis, Musculoskeletal and Skin Diseases last year. It has given so much hope to so very many, hope not only for those of us that deal with skin disease daily but also hope that our children and future generations can look to a brighter and healthier future.
Our coalition is comprised of 17 national lay skin organizations. We represent diseases that are frequently disfiguring, disabling, and potentially fatal. We estimate that 60 million Americans are treated annually for skin disease, skin disease which is the most common cause of chronic illness in the United States, thus translating into lost work time, as well as staggering economic costs.
We therefore respectfully urge the Congress the NIAMS budget for fiscal year 1992 by $47 million. We support this increase, as
skin disease has already proven in our own lives to represent a tremendous financial burden, lost work time and/or permanent disability, and tremendous personal embarrassment.
We would hope to see the last four skin centers funded, as was mandated in the creation of NIAMS. These center grants provide funds for integrating, coordinating and fostering the interdisciplinary cooperation of a group of established investigators, who are conducting the highest quality of skin research. They are indeed a wise and cost-effective investment.
The future of skin research holds great promise. Discoveries right now are around the corner. Recently, research at NIAMS has uncovered two basic, molecular mechanisms for two very serious skin diseases, EB, and pemphigus. NIAMS-funded research has also discovered new laboratory markers for brain involvement in lupus. Thanks to the support of medical research, progress is being made, but to meet the continuing challenges of ongoing research and the technological transfer of this information to patients, it is essential that adequate resources be available to fund.
Thank you for giving me this opportunity to testify. I am in fact here today due to your past support of biomedical research.
Senator BURDICK. I have no questions. Thank you for your testi
STATEMENT OF BARBARA BUTLER, COCHAIRman, CoalitioN OF PATIENT ADVOCATES FOR SKIN DISEASE RESEARCH
Mr. Chairman and members of the subcommittee, my name is Barbara Butler, Chairman of the Coalition of Patient Advocates for Skin Disease Research (CPA/ SDR). The Coalition wishes to express its sincere thanks to the Chairman of the Subcommittee for this opportunity to testify regarding the budget of the National Institute of Arthritis, Musculoskeletal and Skin Diseases.
The Coalition is comprised of 17 national lay skin disease organizations. The member organizations of the Coalition are supported by patients and their families who live with skin disease daily. For the majority of patients served by these organizations, treatment options continue to remain limited or, in some cases, nonexistent. The skin diseases represented by this Coalition are frequently disabling, disfiguring and/or fatal. We estimate that 60 million Americans are treated annually for skin disease. Skin disease is one of the leading causes of lost time from work and the economic cost, including work loss, due to skin disease is staggering. Skin disease is the most common cause of chronic illness in the United States.
Many skin patients have received significant benefits from Federal funds being directed through the National Institutes of Health. The majority of research funding for skin disease comes from the National Institute of Arthritis, Musculoskeletal and Skin Diseases (NIAMS).
In 1985, Congress established the National Institute of Arthritis-and Musculoskeletal and Skin Diseases as a separate Institute for the purpose of intensifying and coordinating the national effort to combat arthritis and musculoskeletal and skin diseases. The primary reasons for establishing this Institute were to increase the visibility of programs of research in arthritis and musculoskeletal and skin diseases and to increase opportunities to develop and expand these programs further. Steady progress has been made toward a better understanding of these diseases. However, NIAMS has not experienced the growth envisioned in the establishment of the Institute, and opportunities for significant research advances in arthritis and musculoskeletal and skin diseases have passed unfulfilled.
Recent research related to the causes and treatment of skin diseases has resulted in substantial progress in the development and application of new knowledge. In particular, advances in molecular biology, improved knowledge of the immune system and the structure and function of connective tissue promise new breakthroughs
in the diagnosis, treatment, and, ultimately, prevention of theses diseases. Support for basic research has led to improved understanding of skin disease and this knowledge is being applied to reduce the enormous human and economic burdens imposed by chronic, debilitating diseases.
To support diversified research thrusts, NIAMS funds research centers through the Skin Diseases Research Core Centers. Two skin disease research core centers were established in 1988; one at Case Western University, the other at New York University.
A skin disease research center grant provides funds for integrating, coordinating and fostering the interdisciplinary cooperation of a group of established investigators conducting-research programs of high quality that relate to a common theme in skin disease. These centers bring together related facilities within a grantee institution to pursue interdisciplinary research on skin diseases. By providing more accessible resources, these special grants are expected to ensure greater productivity at a given grantee institution. This Coalition encourages expansion of the skin centers program and, in particular, recommends that the full complement of six skin diseases research core centers be funded.
We appreciate the funding of the two core centers and the initial funding for the new intramural program for skin. These programs are an excellent beginning to the end of skin disease but continued funding is needed to maintain the high quality of science currently underway.
We strongly feel that skin disease research holds the key to unlocking many of the mysteries surrounding chronic, disfiguring and life-threatening skin diseases. The future of skin disease research holds great promise and discoveries are right around the corner due to improved technology and application of knowledge gleaned from other research disciplines. Research is certain to lead to further breakthroughs in the diagnosis and treatment of these diseases and offers hope for even better treatments. NIAMS research is making a difference. I would like to review some of the recent advancements:
NIAMS research uncovered the basic molecular mechanisms for two very serious skin diseases, epidermolysis bullosa and-pemphigus. NIAMS research has discovered the mechanism by which estrogen prevents bone loss and the discovery of new laboratory markers for disease of the brain in patients with Lupus (SLE). NIH research is a healthy investment for the future. Recently, NIAMS intramural scientists conducted a long-term clinical trial which convincingly demonstrated that combined drug treatments will sustain life-supporting kidney function in systemic lupus patients. This single health care advance has been translated to an estimated potential one year savings of $69.81 million. Along with the significant cost savings, this one advance has had a considerable impact on improvement in quality of life and reduction in premature mortality.
The NIAMS Coalition, representing 43 organizations, including the Coalition of Patient Advocates for Skin Disease Research, respectfully urges Congress to increase the fiscal year 1992 NIAMS budget by $47 million. This would effectively increase funding from 27 percent to 33 percent of approved grants, train additional researchers, for a total of 328, support urgently needed clinical trials, fund 4 additional skin research centers, for a total of 6, and increase the intramural research program to $21.2 million.
Progress is being made, but to meet the continuing challenge of ongoing research and technology transfer to patients and their families, it is essential that adequate resources and well-trained scientists be available to focus on skin disorders.
We, as a Coalition, support this proposed increase of $47 million as we believe the increase will, in the long-term, have significant, positive health benefits for the millions of Americans who are affected by skin diseases.
As you will hear today from representatives of the Coalition, the NIAMS is essential to the future of the hundreds of thousands of patients who are afflicted with severe skin diseases.
Here with us today are 2 representatives of the member organizations to tell you their concerns about the future of NIAMS and what it means to them. The following organizations are represented here today: National Congenital Port Wine Stain Foundation and Eczema Association for Science & Education.
All of the member organizations of the Coalition join me in thanking this Committee and Congress for the support of skin disease research. Thank you.
STATEMENT OF LADONNA G. WILLIAMS, SPECIAL REPRESENTATIVE OF THE ECZEMA ASSOCIATION
Senator BURDICK. The next witness will be Ms. LaDonna Williams of the Eczema Association for Science and Education.