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    Testimony

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    Statement by
    Claude Lenfant, M.D.
    Director, National Heart, Lung, and Blood Institute
    on
    Review of NIH Programs With an Emphasis on Stroke, Heart Disease, and Blood Disease
    before the
    House Committee on Energy and Commerce Subcommittee on Health

    June 6, 2002

    Mr. Chairman and Members of the Subcommittee:

    I am pleased to have this opportunity to discuss the programs and activities of the National Heart, Lung, and Blood Institute (NHLBI). As requested, my comments will focus specifically on cardiovascular diseases—which include, among others, coronary heart disease, stroke, and congestive heart failure—and on blood diseases and resources.

    CARDIOVASCULAR DISEASES

    To begin with a historical perspective, let me mention that when the NHLBI was founded more than 50 years ago, this country was in the throes of an epidemic of heart disease. Beginning at the turn of the 20th century, and particular after the end of World War I, heart disease death rates increased quite precipitously among men and ominously among women. One could envision no end to this trend, as medical science was largely ignorant about the causes of heart disease and extremely limited in its ability to treat or prevent it. Now, thanks to decades of research, heart disease death rates among men have receded to the level of 100 years ago, and among women they are about 37 percent lower. Strokes death rates have plummeted, due in great measure to improvements in detection and treatment of high blood pressure. The average American can expect to live 5½ years longer today than was the case 30 years ago, and nearly 4 years of that gain in life expectancy can be attributed to our progress against cardiovascular diseases. I believe it is fair to say that medical science has made more advances in this area than in any other major disease.

    Nonetheless, many challenges remain. As the following chart illustrates, we in this country are far more likely to die of cardiovascular diseases than of any other cause.

    Graph

    Moreover, the societal burden of living with these diseases is considerable. Cardiovascular disease patients spend more than 30 million days each year in acute-care hospitals—far more than patients with other diagnoses. And, a recent study revealed that 13 of the top 22 prescription drugs taken in the United States address cardiovascular problems. Thus, beyond the suffering caused by these diseases, the health-care costs demand our attention.

    Heart failure accounts for a large and growing public health burden that has, in effect, become our next epidemic. Ironically, it is the cost of our success: having saved many people from dying of acute events, such as heart attack, we have created a large and vulnerable population with heart muscle damage. We now have at our disposal a number of palliative measures, ranging from drugs to instrumental interventions such as the left ventricular assist device. While they improve patients' quality of life by alleviating symptoms and reducing hospitalizations, they are by no means a cure. However, current research provides grounds for cautious optimism that a cure may ultimately be found. For example, we are stimulating research on cell-based therapy in the wake of astonishing discoveries that, contrary to everything we thought we knew before, cells of the heart and other organs are capable of regeneration. If we could find a way to harness and direct the body's ability to grow new cells, we would have an entirely new approach to therapy for diseases such as end-stage heart failure.

    The mapping of the human genome has provided an extraordinary opportunity to understand the genetic underpinnings of disease. We have initiated Programs of Genomic Applications, which seek to maximize the fruits of the new information in order to identify the causes of disease, determine who is susceptible to it, and tailor treatments and, possibly, cures to the individual. We have also launched a program to identify genetic modifiers of disease—genes that determine, for instance, why some people with high blood pressure suffer heart attacks, while others have strokes, still others experience kidney failure, and some escape with few ill effects. The ability to predict the course of disease in a given patient will open up a new era of therapeutic approaches.. Accumulating evidence suggests that inflammation—the body's normal, protective response to injury or infection—may be at the core of many chronic degenerative diseases such as atherosclerosis. This notion is supported by recent findings that blood levels of a substance called C-reactive protein, a marker of inflammatory activity, are correlated with risk of heart attack and stroke. Understanding the delicate balancing act of the immune system could pave the way for new preventive and therapeutic strategies. Related work from a number of laboratories has found that exposure to a variety of infectious agents is associated with development of vascular disease. We are vigorously pursuing basic research to elucidate the mechanisms underlying these phenomena in the expectation that it may ultimately lead to new approaches, perhaps even vaccines, to prevent cardiovascular disease.

    As we pursue these and other basic research avenues, we are working to strengthen clinical research to ensure that findings from the laboratory have a swift and effective impact on patient care. Our research centers program has been reconfigured as Specialized Centers of Clinically Oriented Research to sharpen its focus on the patient. We also conduct numerous clinical trials of promising approaches to treat or prevent disease. As you might expect, they include trials of medical and surgical interventions, but they also include trials that examine the value of lifestyle interventions such as the Dietary Approaches to Stop Hypertension (DASH) diet—an eating pattern that is rich in fruits, vegetables, and low-fat dairy products and low in fat and cholesterol—which has been shown to lower blood pressure. The DASH diet is now being tested in the context of an intensive behavioral intervention to promote other lifestyle changes to lower blood pressure (e.g., decreased salt and alcohol consumption, increased physical activity, and weight control). Two other trials focus on preventing excessive weight gain among teenaged African American girls—a population that is highly susceptible to weight-related problems such as high blood pressure and diabetes in adulthood—and on preventing the decline of physical activity that typically occurs among girls during the middle-school years.

    BLOOD DISEASES AND RESOURCES

    Turning to blood diseases and resources, we also have much progress to report. In sickle cell disease, which affects approximately 70,000 Americans, we have found that hydroxyurea, a chemotherapeutic drug that is taken by mouth, decreases the frequency of acute pain crises in adults and may actually prolong the life span. We are funding a study to determine whether benefits of this drug can be extended to very young children, thereby preventing primary damage to organs such as the spleen and kidneys. Clinical studies funded by the NHLBI also have proven the efficacy of transfusions in preventing the recurrence of stroke in young children with sickle cell disease.

    Clinical trials are also in progress to establish whether a cure is possible for Cooley's anemia and other hemoglobin disorders such as sickle cell disease through transplantation of hematopoietic (blood-forming) stem cells obtained from sibling donors. The cells can come from the circulating blood of the sibling or from umbilical cord blood, in cases where there is a newborn brother or sister. Also in this area, the NHLBI is funding studies on cord blood transplantation in children and adults to determine the most appropriate role for this source of stem cells in blood diseases such as acute leukemia. This approach may provide new hope for thousands of patients in need of a transplant, because cord blood is readily available, can be collected at no risk to the newborn donor, is less likely than bone marrow to transmit infection, and may work well despite less precise tissue matching,

    Gene therapy for the eventual cure of hemophilia is now under development by several companies. The original research leading to the actual commercial development of this approach came from funding provided by the NHLBI. Our own research in this area is gaining addition momentum with recent funding of Centers of Excellence in Gene Therapy, which are designed to move these studies rapidly into the clinical arena within the context of careful and appropriate safeguards for patient safety and welfare.

    In the early 1980s the Institute created a research program in transfusion medicine that has actively pursued methods to improve the safety of the U.S. blood supply. I am happy to report great success in this endeavor. For instance, the risk of contracting hepatitis C from a transfusion—a great public health concern—is now about 1 in 1.7 million units, whereas it was an estimated 1 in 25 units 2 decades ago. Taken as a whole, our investment in transfusion medicine research has given the United States a blood supply that is the safest in the world.

    EDUCATION AND OUTREACH

    To maximize the impact of research findings on the people whom we serve, the NHLBI is strongly committed to educating patients, health professionals, and the public about disease awareness, diagnosis, treatment, and prevention. Over the past 3 decades, we have conducted education programs in high blood pressure, cholesterol, blood resources, smoking, asthma, heart attack awareness, obesity, and sleep disorders. Two campaigns—one that has been under way for some time and one that is brand new—may be of particular interest to the Subcommittee.

    The NHLBI Stroke Belt Initiative had its origins in observations during the 1980s that a band of states located generally in the southeastern portion of the country (depicted in the graphic on the top of the page that follows) suffered an excessive death toll from stroke, and that extraordinary rates of high blood pressure were the culprit. In subsequent years, we worked with state health departments and other groups to address improvement of blood pressure control in these populations. The approaches taken are too numerous to mention, but they included church-based screenings ("High Blood Pressure Sunday," the first Sunday in May, is now established in many communities, and features sermons, gospel music, and cooking related to lowering blood pressure) and screening at baseball games (the "Strike out Stroke" campaign, which began with the Altanta Braves). As we look back on these efforts, it is clear that stroke is still a major problem in the Southeast. However, it is also apparent (see second graphic) that some of the greatest gains in reducing the number of stroke deaths per 100,000 population over the past 2 decades have occurred in the Stroke Belt states. Building on what has been learned about improving the health of high-risk communities, we are now working to extend our reach to other vulnerable subsets of the population. We have established what we call EDUCs (Enhanced Dissemination and Utilization Centers) in communities whose residents are at especially high risk of developing cardiovascular disease. These projects are mobilizing community resources—including health centers, churches, schools, businesses, and soup kitchens—to increase awareness and control of cardiovascular disease risk factors.

    Our very recent campaign, Act in Time to Heart Attack Signs, addresses a missed opportunity to save lives. More than 1 million Americans suffer heart attacks each year, and about 460,000 of these attacks are fatal. In many cases, the deaths occur because heart attack victims do not get to the hospital in time to benefit from the treatments we have to offer. Why? Often, patients fail to recognize the symptoms of heart attack, shrink from the notion of calling an ambulance, or worry that they will feel foolish if their distress turns out to be "indigestion." The new educational initiative seeks to counteract misconceptions about heart attack symptoms, alleviate patient fears, and emphasize the importance of getting treatment promptly. Materials have been developed—for the public and for doctors—to teach people the key messages: (1) recognize the symptoms and (2) call 9-1-1. Although the program is only in its 9th month, the Act in Time message is already an official course of the American Red Cross, and the National Council on the Aging is offering Act in Time in senior centers throughout the country.

    CONCLUSION

    We are confident that our approach, which is driven both by compelling public health needs and by extraordinary scientific opportunities, will continue to yield progress in the future. I would be pleased to answer any questions that the Subcommittee may have about the programs and plans of the NHLBI.


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Last revised: July 1, 2002