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Testimony on National Heart, Lung, and Blood Institute's FY 1998 Budget by Dr. Claude Lenfant
Director, National Heart, Lung, and Blood Institute
National Institutes of Health
Accompanied by
Dr. Carl Roth, Associate Director for Scientific Program Operations, NHLBI
Mr. James Wehling, Financial Management Officer, NHLBI
Ms. Sheila Merritt, Executive Officer, NHLBI
and
Dr. Harold Varmus, Director, NIH
Mr. Dennis P. Williams, Deputy Assistant Secretary, Budget, DHHS

U.S. Department of Health and Human Services

Before the House Appropriations Committee, Subcommittee on Labor, Health and Human Services, Education and Related Agencies
March 4, 1997

I am pleased to address this Committee once again on behalf of the National Heart, Lung, and Blood Institute (NHLBI).

We are now on the threshold of the Institute's golden anniversary, an opportunity to reflect with pride and a certain sense of awe on the remarkable achievements of medical research that have occurred since the National Heart Institute was founded in 1948. Undoubtedly, more than a few people in this room had grandparents who suffered heart attacks 50 years ago. The grim reality was that many of them died on the spot; and for the few who made it to the hospital alive, we had little to offer but bed rest, painkillers, and the gloomy prognosis of life as a "cardiac cripple" and death before one's time. Worse, we had very little idea of how such devastating events could be prevented.

Progress made during the intervening years has been truly outstanding. Consider the array of approaches that we now call upon to combat heart disease: diagnostic tests to assess heart and blood vessel anatomy and function; CPR, defibrillation, coronary care units, and emergency care to address life-threatening events; "clotbuster" drugs and such other treatments as aspirin and beta-blockers to protect the heart in the hours after an attack; and high-technology approaches, such as pacemakers, coronary bypass surgery, "balloon" angioplasty, and heart transplantation to extend life and enhance its quality. Moreover, we have gained valuable insight into the characteristics and habits that predispose people to develop cardiovascular disease hypertension, high blood cholesterol, smoking, obesity, diabetes, lack of exercise and have raised public awareness of the importance of modifying these risks.

The public health benefits that have accrued from our investment in research have been of such magnitude that we can justifiably point to the Institute's first half century as the "golden age" of cardiovascular, lung, and blood investigation. Nonetheless, we have much reason to believe that the next half century indeed, the next 5, 10, or 20 years may eclipse the accomplishments of the past. Let me share with you our vision of the future.

Fixing the failing heart. Heart failure is one of the most vexing illnesses of the 1990s because its relentless downhill course is overtaking ever greater numbers of Americans. A central problem of heart failure is that adult heart muscle cells, once lost to disease, are irreplaceable. Thus, treatments to date have focused on optimizing the function of the cells that remain or replacing the heart in its entirety, through transplantation of a human donor heart or provision of an artificial heart. Research advances now offer clues to innovative approaches for rejuvenating the failing heart. For example, it may be possible to "turn on" the capacity that existed in fetal life for heart muscle cells to reproduce themselves, thereby replacing old or damaged cells. Investigators are also exploring the possibility that healthy cells from donor muscle can be grafted into a failing heart to enhance its pumping ability. Innovative preliminary work in rats indicates that grafted cells taken from skeletal muscle may be able to "heal" areas of heart muscle that have been damaged by heart attack. A third area of investigation involves examination of the genetic factors that regulate apoptosis, the "programmed death" that normally occurs in cells. This work offers much promise not only for treating, but also for preventing, heart failure.

Opportunities for asthma prevention. Asthma is an increasingly prevalent condition that diminishes the quality of life for millions of children and adults in the United States. While it has long been known that asthma tends to "run in families" and to be triggered by allergens, infections, pollutants and other exposures found in day-to-day life, real progress is now being made in unraveling the genetic and environmental factors that influence this disease. Scientists have uncovered evidence of genes that affect allergy and bronchial hyperresponsiveness, two key aspects of asthma, and the quest to pinpoint specific genes and determine their function is now under way. This information should lead to better understanding of the primary defects in asthma, and to development of effective preventive strategies focused on individuals who are vulnerable but have not yet acquired the disease. Equally exciting work is capitalizing on recent observations that respiratory infections may actually protect some children from developing asthma, while exacerbating asthma in others. Using modern techniques of cell and molecular biology, scientists now have the opportunity to understand how infectious agents interact with both the lungs and the immune system to induce the airway inflammation and hyperresponsiveness that characterize asthma.

New hope for hereditary blood disorders. Exciting developments are taking place in research and treatment of hemophilia, the scourge of generations of families worldwide. It was only in 1987 that NHLBI-supported basic research led to identification of the genes for clotting factors VIII and IX, whose lack causes hemophilia A and B, respectively. Rapid subsequent advances led to development of recombinant factor VIII, created with minimal use of blood-derived products for a much lower risk of infection; quite recently, recombinant factor IX, the first hemophilia treatment that is totally free of blood products, was shown to be effective in clinical trials not only for bleeding episodes, but also for use in surgery. Further genetic engineering techniques are being used to create new "combination" clotting factors that have high activity and can be given in low doses, thereby reducing today's high treatment costs. As work proceeds on improved treatments, scientists are closing in on a cure. Although much remains to be done before gene therapy can be offered to patients, a number of studies have shown that hemophilic mice and dogs given the appropriate factor VIII or IX gene can produce normal, healthy clotting factors. The fact that only a small increase in production of active clotting factor is needed to correct severe hemophilia indicates that gene therapy, when it comes, has the potential to halt the frequent, painful, and dangerous bleeding episodes that result in so many factor infusions and hospitalizations.

Targeted preventive strategies. Disease prevention has always been our ultimate goal, and our efforts in the area of hypertension detection and control, for example, have paid off handsomely in preventing stroke deaths. Nonetheless, we have to treat a large number of hypertensives to prevent a smaller number of deaths, because we are as yet unable to identify the subset of patients who are most susceptible to stroke. Similarly, we advocate lifestyle modifications to prevent hypertension, such as limitation of sodium intake, on a population-wide basis because we lack the knowledge to target this advice to the people most likely to benefit from it. Our vision of the future includes a vastly increased ability to understand and detect individual susceptibility to disease and provide targeted interventions on the basis of that information. First, our accelerated search for the genetic causes of such complex diseases as hypertension promises to reveal the reasons for individual variations in susceptibility and lead to tailored prevention and treatment strategies. Second, our increasing ability to detect subclinical disease may enable us to halt pathological processes before conventional signs and symptoms appear. These new approaches offer enormous potential for cost-effective improvements in the health of the nation.

I recognize that much of the bright promise I have outlined for you here may seem speculative and futuristic, because I am relying on very basic studies using scientific approaches that were unheard of even a decade ago. The pathway between basic science investigations and real-life applications may not be straightforward, but it has met the test of time and it is growing ever shorter. For example, a scant 6 years ago I reported findings about a class of inflammatory mediators, leukotrienes, that are involved in asthma attacks, and suggested that this discovery might someday inspire development of new asthma drugs that block leukotriene activity. Flip through a major medical journal today, and you will find a full-page advertisement for such a drug under the headline "Leukotrienes This asthma troublemaker is in for a surprise." Such is the pace of current heart, lung, and blood research.

During the past half century, life expectancy has soared due, in no small measure, to the fruits of biomedical research. Advances against cardiovascular, lung, and blood diseases have added years to the lives of many Americans. Our goal and our expectation are that the next decades of research will enable us to "add life to years," so that the long life we have earned is also a comfortable and fulfilling one.