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Fiscal Year 2004 Budget Request
Statement to the Senate Subcommittee on Labor-DHHS-Education Appropriations (April 8, 2003)
Stephen E. Straus, M.D., Director
National Center for Complementary and Alternative Medicine
Mr. Chairman and Members of the Committee:
I am pleased to present the President's Fiscal Year (FY) 2004 budget request for the National Center for Complementary and Alternative Medicine (NCCAM). The FY 2004 budget includes $116.2 million, an increase of $2.9 million over the FY 2003 enacted level of $113.3 million comparable for transfers proposed in the President's request.
Introduction
Arthritis, depression, menopause, cancer...for millions of Americans, these and other health concerns are not being adequately addressed through conventional medicine. Many are turning outside the medical mainstream to approaches that embrace the whole person--mind, body, and spirit. From acupuncture to dietary supplements, complementary and alternative medicine (CAM) approaches are affordable and accessible, but largely untested. Under NCCAM’s leadership, researchers are applying the tools of modern science to discover which CAM practices work, why and how they work, and whether they are truly safe. Exploring CAM through rigorous science will lead to the integration of proven CAM practices with conventional medicine, thus improving the lives of all Americans.
Standardization and Characterization of Dietary Supplements
Dietary supplements, one of the most popular categories of CAM practices, are used by 10 percent of American adults.¹ Many consumers use dietary supplements with the expectation that they are effective in the self-treatment and prevention of disease and the promotion of wellness and, further, with the assumption that they are safe. Under the law, supplements are classified as foods and not held to the same rigorous standards as drugs.
Research supported by NCCAM indicates that Americans who take ginseng cannot rely on the label to accurately reflect the product’s contents. After examining 25 commercial ginseng products, one NCCAM grantee reported that the concentrations of ginseng differed by as much as ten-fold from the label. The lack of standardized dietary supplements is not only an issue of consumer safety, it is also an issue for researchers who need well-characterized and standardized products to study their purported benefits.
NCCAM’s recent experience with PC SPES, a patented mixture of eight herbs, is an example of another problem with some dietary supplements--contamination. In 2001, thousands of men with advanced prostate cancer in America were taking PC SPES. Based on encouraging early clinical results, NCCAM was supporting four research studies, including a clinical trial, to determine the safety, efficacy, and mechanism of action (i.e., how it works) of PC SPES. In February 2002, the California Department of Health Services and the Food and Drug Administration reported that PC SPES was contaminated with undeclared prescription drug ingredients. This finding led the manufacturer to recall the product and subsequently cease its operations. NCCAM immediately put its studies on hold and convened meetings with scientists, prostate cancer specialists, patients, and industry representatives to determine if an uncontaminated product could be made available to the public, allowing the research to resume. As a result of these meetings, NCCAM resumed its laboratory studies of the cellular and molecular biology of PC SPES and declared its interest in resuming clinical trials once an unadulterated, fully characterized, and standardized product is available.
NCCAM is taking several steps to address the critical issue of product standardization and quality. Among the top-selling products in the dietary supplement industry are products like echinacea (Echinacea purpurea), taken to prevent and treat colds; milk thistle (Silybum marianum), taken to treat chronic hepatitis and cirrhosis; and feverfew (Tanacetum parthenium), taken to lower fevers. All of these products have shown promise in small uncontrolled studies; however, each has problems with standardization, precluding their full and objective study. NCCAM is using the Small Business Innovative Research (SBIR) program to obtain well-characterized and standardized clinical-trial-grade materials of these supplements. This investment in high-quality products will be followed by studies to define the optimal dose of each product. To implement this second step, in 2004, NCCAM plans to establish a Dietary Supplement Standardization and Characterization Center (DSSCC) to serve as a resource for the analysis of dietary supplements, especially botanical products, before they are used in clinical trials.
¹Hanyu Ni, Catherine Simile, and Ann M. Hardy, "Utilization of Complementary and Alternative Medicine by United States Adults: Results From the 1999 National Health Interview Survey," Medical Care, Vol. 40, No. 4, pp. 353-358.
Determining the Mechanisms of Action of CAM Interventions
While pursuing innovative approaches to ensuring the safety of its clinical trial products, NCCAM continues to support basic and clinical studies. The central objective of many of these studies is to examine the mechanisms of action underlying various CAM therapies. In 2002, for example, NCCAM-supported researchers conducted an important body of research on alternatives to conventional hormone therapy--an area of obvious interest for millions of women who are seeking safe and effective alternatives to conventional hormone therapy for relief of menopausal symptoms and related conditions. Specifically, scientists are using in vitro systems to examine how some popular dietary supplements act on biochemical pathways responsive to estrogen. Others are examining the estrogenic activity and specific mechanisms of estrogen receptor regulation of a Chinese herbal extract; identifying the active compounds of black cohosh (Cimifuga racemosa) and red clover (Trifolium pratense); and investigating the range and mechanisms of action of two plant-based estrogens, genistein and diadzein, and extracts of soy on immune function. These studies will clarify what biochemical effects supplements might have on women and indicate which, if any, are worthy of testing in a clinical trial.
Building on the results of a detailed scientific review that NCCAM conducted with the Agency for Healthcare Research and Quality on the popular dietary supplement, S-Adenosyl-L-Methionine (SAMe), the Center is also supporting projects on the mechanisms of action of SAMe that are consistent with the findings of the report. One grantee is using cultured cells to better characterize the biochemistry of liver injury and what role SAMe may play in preventing liver damage. Another investigator is using a mouse model of hepatitis and liver cancer to study the role of SAMe in regulating liver cell growth and death.
A trio of studies indicate that Ginkgo biloba may provide multiple levels of protection to neural tissues and contribute to the body of evidence explaining how Ginkgo may be beneficial in preventing the onset of dementia. NCCAM-supported investigators reported that a standardized Ginkgo extract protects cells from oxidative stress and apoptosis (programmed cell death). Using model systems to study the factors that regulate cell death, the investigators showed that the Ginkgo extracts increase the lifespan of the worm, Caenorhabditis elegans, protect cultured neural cells from undergoing programmed death, and hinder an early step in the biochemical processes leading to neurodegeneration.
In FY 2003, NCCAM made several awards as part of the initiatives it launched with NIH partners to elucidate the underlying biological pathways of the placebo effect and to reveal factors important for eliciting the placebo effect in clinical practice setting. The Center designated mind-body medicine as a priority research area in FY 2003, recognizing the potential contributions to prevention and treatment of chronic diseases that could be made by interventions based on evidence from innovative psychophysiological research. Most recently, NCCAM joined other NIH partners to solicit applications from institutions poised to advance research on mind-body interactions and health.
Evaluating CAM Therapies in Rigorous Clinical Trials
NCCAM supports basic and preclinical research to test therapies for eventual use in clinical trials with the ultimate objective being to translate safe and effective therapies into widespread practice. In addition, NCCAM-supported clinical trials test CAM products already being widely used by the public. Ultimately, NCCAM wants to answer the central question: "Does it work?"
In 2002, NCCAM announced the results of its first large-scale clinical trial. The trial evaluated a St. John’s wort (Hypericum perforatum) product, a popular herb for depression, as a treatment for major depression of moderate severity and found it to be ineffective as compared to placebo. Although the results of this trial showed that St. John’s wort is not effective for this type of depression, the outcome provided practitioners and patients alike with valuable data. NCCAM is following-up on this finding by co-funding a new trial to test St. John’s wort as a treatment for minor depression, a less severe but very common type of depressive illness. The trial begins this year and will enroll 300 patients at three sites nationwide.
Because CAM products and practices are already used by millions of Americans, NCCAM supports a higher percentage of clinical research than the other NIH Institutes and Centers. As part of its clinical research portfolio, NCCAM is supporting 12 ongoing large-scale clinical trials with other NIH Institutes and Centers. These trials include the largest ever study of Ginkgo biloba for the prevention of dementia–-a critical study given the aforementioned body of evidence that exists regarding Ginkgo’s potential protective effects. The list also includes the largest ever study of dietary supplements (selenium and vitamin E), involving 30,000 men, for the prevention of prostate cancer. In Fiscal Year 2002, NCCAM cosponsored the first large clinical trial to test chelation therapy as a treatment for coronary artery disease. Also in FY 2002, the NCCAM Intramural Research Program initiated its first clinical trial, which is evaluating electroacupuncture in reducing the severe nausea experienced by many children following intensive cancer chemotherapy. NCCAM is taking action to ensure the quality and safety of its clinical trials.
In 2002, the Center established the Office of Clinical and Regulatory Affairs to help plan, coordinate, and monitor NCCAM-supported clinical trials. All of these activities reflect NCCAM’s investment in and commitment to clinical research.
Building Research Infrastructure and Intellectual Capital
The success of NCCAM’s future research endeavors depends on the availability of skilled investigators in both the conventional and CAM research communities. Toward this end, NCCAM is supporting dozens of mentored and independent trainees, from the pre-doctoral level through mid-career and senior faculty members. In 2002, NCCAM made institutional training and clinical research career awards to CAM institutions and joined the new NIH-wide loan repayment program with awards to two junior practitioner-investigators, marking a series of "firsts" for NCCAM.
In addition to its investment in training programs, NCCAM continues to support a robust research centers program, providing a critical CAM research infrastructure. In 2002, NCCAM sought to strengthen its centers program by convening an expert panel to evaluate the program’s current structure and objectives. The panel recommended a more flexible approach to supporting future centers research. This new approach, which employs a mix of funding and research mechanisms, will ideally expand the participation among investigators with varying degrees of research expertise at both CAM and conventional institutions in a multi-disciplinary fashion. Implementation of this strategy began in Fiscal Year 2003 and will continue through Fiscal Year 2005.
Conclusion
NCCAM has made significant progress in its first 4 years. Between FY 2000 and FY 2001, the number of people enrolled in NCCAM-supported clinical research projects doubled. The Center, in a partnership with other NIH Institutes, launched some of the largest clinical studies of CAM therapies ever conducted. NCCAM took steps to improve the safety and efficacy of its clinical research studies and the quality of the information disseminated to the public about CAM therapies. Finally, the Center increased its level of support to researchers who are applying cutting-edge scientific tools to study the most promising CAM approaches to the most important public health challenges facing our nation. I look forward to keeping you and the American public apprised of NCCAM’s future activities and accomplishments.
