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Researchers participating in a small, multicenter study of sickle cell anemia

patients recently reported that fetal hemoglobin levels were raised to 15

percent or more through treatment with hydroxyurea, a common chemotherapy

drug.

If confirmed in larger studies, this approach may represent a

significant stride in reducing both the suffering and the high hospitalization

costs now incurred by sickle cell disease patients.

The identification and cloning of the defective gene in cystic fibrosis

(CF), which normally codes for a protein called cystic fibrosis transmembrane

conductance regulator (CFTR), have produced many research opportunities and

raised hopes for a cure through gene therapy.

NHLBI-supported researchers

recently demonstrated that the insertion of the normal CFTR gene Into cultured

human CF airway epithelial cells corrected the CF defect in vitro.

Further,

investigators in the NHLBI Intramural research program have reported success

in inserting the CF gene into the airway epithelial cells of living rats and

obtaining measurable indicators of gene expression in lung tissue.

These

advances have greatly improved the prospects for the possibility of gene therapy to cure the disease. The tools of molecular biology, have also

produced two advances that may have applicability to the short-term clinical

management of CF.

Administration of aerosolized alpha-1 antitrypsin into the

airways of individuals with CF has been shown to counteract the enzymes that

cause the tissue destruction associated with CF infections.

Related work has

demonstrated the effectiveness of DNase, an enzyme that digests DNA, in

reducing the thickness of CF mucus.

All together, these findings represent

significant advances toward better treatment and ultimate cure of CF.

Studies of the natural history of atherosclerosis and hypertension have

enhanced our understanding of the development of cardiovascular disease in

American Blacks.

An examination of autopsy material from young victims of

accidental death found that arterial fatty streaks were much more extensive in

Blacks than in whites of comparable age.

The appearance of these lesions was

s

significantly associated with elevated serum lipid levels and cigarette

smoking.

The association with smoking is a new observation that could provide

powerful impetus for controlling smoking in the young.

In other studies,

measurements of 24-hour blood pressure patterns revealed that blood pressure

is much less likely to drop at night in Blacks than in whites.

It is of

considerable interest that this phenomenon seems to be unique to American

Blacks and has not been observed in studies of Blacks born in other parts of

the world.

This so-called blunted nocturnal decline imposes additional

cardiovascular strain which may help explain the greater morbid consequences

of hypertension experienced by Blacks in the United States.

Women's health issues are being addressed in a number of new and ongoing

research programs of the Institute.

A pilot study to assess the efficacy of

various treatment interventions for asymptomatic myocardial ischemia is of

particular relevance to women in light of evidence that women experience

unrecognized myocardial infarction more frequently than do men.

As part of

this study, an assessment of the psychophysiologic characteristics of

myocardial ischemia will be conducted.

The Institute also has a strong

interest in how health-related behaviors become established and can be

modified.

This year, NHLBI-supported researchers presented evidence that

excess body weight is a strong, independent risk factor for coronary heart

disease in women.

A recent conference explored reasons for the greater

prevalence and more serious consequences of obesity in minority women.

Preliminary data from an NHLBI observational study comparing the development

of obesity in Black and white preadolescent girls indicate that differences

exist between the two races in height, body mass, and eating habits at ages

9-10 years.

The Institute also recently sponsored a conference on smoking and

body weight, a topic of considerable Interest in light of the observation that

smoking cessation results in more weight gain for women than for men.

Although the association between elevated cholesterol and heart disease

is well established, the implementation of a nationwide strategy to lower

cholesterol levels has been controversial.

This past year, the NHLBI

sponsored three conferences to reexamine the science base for the

recommendations of the National Cholesterol Education Program (NCEP).

The

conferences assessed data concerning the extent to which serum cholesterol

levels predict coronary heart disease in older persons and in women, reviewed

the evidence for associations between low serum cholesterol levels and certain

specific causes of morbidity and mortality, and addressed the costs and health

Implications of cholesterol lowering.

The conclusions reaffirmed the

Institute's current approach to cholesterol lowering, and recommended

additional research in a number of specific areas.

The topic of cost is being

addressed in a new NHLBI research program to identify alternative strategies

for cholesterol reduction.

A new NCEP expert panel report to be released this

spring will provide guidance about cholesterol assessment and intervention in

children and adolescents.

The NHLBI has also initiated a clinical trial of

cholesterol lowering in the elderly, which is expected to provide a firm

scientific basis for treatment recommendations in older

persons.

In the upcoming year, the NHLBI will continue support for its five

education programs in the areas of high blood pressure, cholesterol, smoking.

asthma, and blood resources, placing special emphasis on ways to reach

Americans at high risk, particularly minorities and those with low literacy

skills.

The Institute is also taking the first steps toward implementation of

a new national education program intended to reduce premature morbidity and

mortality from heart attack through early intervention.

The initial efforts

of this program will focus on educating health care professionals and their

patients about the need for immediate identification and treatment of patients

at the first symptoms and signs of a heart attack.

Such efforts are expected

not only to enhance survival, but also to contribute substantially to the

quality of life of heart attack patients and their familles.

Mr. Chairman, the FY 1992 budget request for the National Heart, Lung.

and Blood Institute is $1,209,924,000.

I would be pleased to answer any

questions that the committee may have.

BIOGRAPHICAL SKETCH OF DR. CLAUDE LENFANT

October 12, 1928, Paris, France

Education: B.S., University of Rennes, France, 1948.
Paris, France, 1956.

M.D., University of

Professional History: 1957-1958, Research Fellow, University of Buffalo, New York. 1958-1959, Research Fellow, Columbia University, New York, 1959-1960, Assistant Professor of Physiology, University of Lille, France. 1961-1965, clinical Instructor of Medicine and of Physiology and Biophysics, University of Washington, Seattle. 1966-1967, clinical Assistant Professor of Medicine and of Physiology and Biophysics, University of Washington, Seattle. 1968 1971, Associate Professor of Medicine and Physiology and Biophysics, University of Washington, Seattle. 1970-1972, Acting Associate Director, Collaborative R&D Program, National Heart and Lung Institute, NIH. 1970-1972, Associate Director for Lung Programs, National Heart and Lung Institute, NIH. 1971-1972, Professor of Medicine and Physiology and Biophysics, University of Washington, Seattle. 1972-1980, Director, Division of Lung Diseases, National Heart, Lung, and Blood Institute, NIH. 1981-1982. Associate Director for International Research, NIH. 1981-1982, Director, Fogarty International

Center for Advanced Study in the Health Sciences, NIH. 1982 - Present,
Director, National Heart, Lung. and Blood Institute, NIH.

Professional Organizations: Association of American Physicians, American
Society for Clinical Investigation, American Physiological Society, American
Federation for Clinical Research, French Physiological Society. International
Federation for Medical Electronics, American Society of Zoologists, Society
for Experimental Medicine and Biology. Undersea Medical Society. New York
Academy of Science.

Honors. Awards: Thesis Prize, University of Paris, France, 1956.
Commendation Tohoku Medical Society, Sendai, Japan, 1973. Superior Service
Honor Award, DHHS, 1974. Regents' Professor, University of California, Los
Angeles, 1975. Testimonial Dinner, American Thoracic Society, 1979. Honorary
Fellow, American College of Chest Physicians, 1979. Honorary Professor,
National Yang-Ming Medical College Taipei, Taiwan, 1980. Honorary Professor,
Universidad Peruana Cayetano Heredia, Lima, Peru, 1981. Senior Executive
Service Performance Award, 1982. Elected Institute of Medicine, National
Academy of Sciences, 1983. American Heart Association Scientific Councils'
"Distinguished Achievement Award," 1983. Senior Executive Service Performance
Award, 1983. Honorary Fellow Council on Clinical Cardiology • American Heart
Association, 1984. Senior Executive Service Performance Award, 1984. Forrest
M. Bird Contributory Award, American Respiratory Therapy Foundation, 1985.
Senior Executive Service Performance Award, 1985. Presidential Meritorious
Executive Rank Award, 1987. Elected Honorary member, Royal Society of
Medicine, England, 1988. llonorary Doctor of Science, State University of New
York, Buffalo, 1988. Elected member USSR Academy of Medical Sciences, 1988.
Cystic Fibrosis Foundation "Breath of Life" Award, 1988. Elected Honorary
Member - French Cardiology Society, 1989. Senior Executive Service
Performance Award, 1989. Brotherhood Award Association of Black
Cardiologists, 1990. Appointed, Board of Governors, US - Israel Binational
Science Foundation, 1990-1993, Senior Executive Service Performance Award,
1990.

Authorship. Editorship: Author or Co-Author of 192 scientific publications
Served on Editorial Boards of: Am. Journal of Physiology: Journal of Applied
Physiology: Respiratory Physiology; Am. Review of Respiratory Disease;
Undersea Biomedical Research; Proceedings of Society for Experimental Biology
and Medicine; Revue Francaise des Maladies Respiratories; Journal of Applied
Physiology: Respiration, Environmental and Exercise Physiology; Am. Journal of
Medicine; Continuing Education for Family Physicians; Executive Editor of 50
volume monograph series, Lung Biology in Health and Disease.

EDUCATION PROGRAMS
Senator HARKIN. Thank you very much, Dr. Lenfant.

Dr. Lenfant, I have been looking at your biomedical research spectrum which talks about taking

the research through the various steps from basic to clinical trials, demonstration, education, with the end product being knowledge dissemination. You have a very active series of education programs, including the national cholesterol education program, the national blood pressure edu. cation program, and the national asthma education program, and many others.

First of all, I want to congratulate you on this effort, and I believe the last step, education and knowledge dissemination, is critical. It ties in with my strong feelings on prevention. Before you can have prevention, people have to have knowledge and information, and once they are educated, then they can perhaps better take care of themselves.

My question to you is should we highlight these education programs more specifically in the budget

so that we can see in your Institute and perhaps all the other institutes what is being spent on knowledge dissemination or education.

Dr. LENFANT. Well, Mr. Chairman, this is a question that we are discussing extensively, and I have to tell you that as yet we have not come up with a definitive answer. If I may, let me tell you during the next 2 minutes about the pros and cons of having a line in our budget corresponding to all these programs.

The advantage is that clearly it would give them much greater visibility. Everybody would know they are here, and they would be recognized.

Also, administratively, it would clearly simplify greatly knowing the costs of these programs.

Now, there are some disadvantages which I also would like to mention. As you see from the spectrum that you referred to, we view our programs as a continuum going basically from basic research to Clinical research to demonstration projects and to prevention and dissemination. I think that the reason why our programs of dissemination are so successful is that they are based on up-todate research outcome. Now, if they were identified as a line in our budget, we feel that there would be a separation of sorts between the basic research, the clinical research, and this dissemination effort.

In addition, there would be also some problems with the tracking of some of the I hate to use the word indirect costs today-management costs, if you want, FTE's and other costs which are necessary to conduct these programs.

So, to summarize, there are some advantages, some disadvantages. We look at it continuously. We never came up with a definite answer. All I can say that if the Congress wants to have that as a special line in our budget, we could live with it, but we can live just as well if it is not a special line in the budget.

Senator HARKIN. Dr. Raub, do you have any feelings about that?

Mr. Hall just gave me this. Under each of the institutes, there is regular programs and research training, regular program, research training, regular program, research training. Should there be another line for prevention, education? That is really the question I had for Dr. Lenfant. Do you have any thoughts on this?

Dr. RAUB. From my view, sort of along the lines that Dr. Lenfant has suggested, I think it turns on communication, that it is in our interest and yours and everyone else's that our budget requests provide the information in a way that is useful to everybody involved, at the higher levels of the executive branch as well as with the Congress. And we certainly would be open to discussions with the staff elsewhere in the Department and with the committee staff here to look at formatting.

Senator HARKIN. Well, let's take a look at it.

Dr. RAUB. I would not want to see, nor do I think you are suggesting, that a formatting change in the budget display change the style of management within Dr. Lenfant's Institute, for example. But I think that we ought to be able to be sure that we are communicating in a way that you have the information you need readily at hand to make the judgments and oversight that is your responsibility, and we will be glad to cooperate on that.

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