DEPARTMENT OF HEALTH AND HUMAN SERVICES
NATIONAL INSTITUTES OF HEALTH
Fiscal Year 2016 Budget Request
Statement for the Record
Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies
Gary H. Gibbons, M.D.
Director, National Heart, Lung, and Blood Institute
Mr. Chairman and Members of the Subcommittee:
I am pleased to present the President’s Fiscal Year (FY) 2016 budget request for the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH). The FY 2016 budget request includes $2,987,685,000 for NHLBI, which is $4,948,000 over the FY 2015 enacted level of $2,982,737,000.
NHLBI investments in biomedical research on heart, lung, blood, and sleep (HLBS) disorders have contributed to dramatic improvements in longevity, quality of life, and the economy of the nation. For example, long term research investments have helped to reduce deaths from cardiovascular disease (CVD) by over 70 percent in the past 40 years. Such investments also pay economic dividends. A recent analysis of data from the Women’s Health Initiative (WHI), which found that postmenopausal hormone therapy failed to protect women from coronary heart disease and increased the risk of developing breast cancer, revealed a $140 return on each dollar invested in the trial. Yet heart and lung diseases remain the leading causes of death, disability and rising health care costs from non-communicable diseases in the United States and around the world.
Today’s discovery science presents opportunities to pursue questions that could not be explored a decade ago. We hope to understand the intricate and dynamic biological systems that sustain health and identify what goes awry earlier in disease processes. NHLBI is advancing precision medicine to prevent and preempt chronic disease and ensure that each person receives the right treatment at the right time.
TRANSFORMING CLINICAL PRACTICE WITH PRECISION MEDICINE
Fundamental discovery science and its translation into therapeutics and clinical innovation are the foundation of our success in altering the trajectory of disease. Pioneering work in genomics allows us to build upon that success. For example, in 1989, the identification of the gene that causes cystic fibrosis (CF)—the CFTR gene—led to earlier diagnosis and improved symptom management. Additional NIH-funded research revealed the various CFTR mutations that led to impaired lung function. The most common mutation, F508del, alters the proper folding of the CFTR protein and blocks its transport to the lung cell surface. These fundamental insights into the cellular actions of CFTR served as an invaluable guide for drug development in the private sector yielding two new drugs that hold promise for improving lung function in most patients with CF. These exciting findings exemplify the potential for precision medicine to tailor the most effective therapy based on an individual’s characteristics.
To accelerate the discovery of the molecular basis of disease and to identify new targets for therapeutic development, NHLBI is exploring new directions in precision medicine. One new initiative, Trans-Omics for Precision Medicine, will begin by conducting whole genome sequencing of 20,000 individuals from diverse groups to study a variety of HLBS disorders. Combining this data with molecular, imaging, environmental, and clinical data will lead to better characterization of factors that predispose to or protect against disease, greater understanding of different subtypes of disease, and ultimately to developing more targeted, personalized treatments.
PREEMPTING AND PREVENTING CHRONIC DISEASE
Precision medicine and advances in stem cell biology offer new opportunities to preempt and prevent chronic illnesses such as Sickle Cell Disease (SCD), a genetic disorder that once severely limited children’s lifespans. Today, as a result of NHLBI-funded research which improved symptom management and reduced risk of stroke, most individuals with SCD can live into their fifties. However, SCD remains a chronic disease with life-long complications. New gene-editing tools, which replace defective DNA with healthy DNA, provide an exciting approach that could preempt disease manifestation. In a recent proof-of-concept study, researchers generated induced pluripotent stem cells from SCD patients, replaced the sickle gene with a healthy one, and then successfully converted the stem cells into mature normal red blood cells. If proven safe, these gene-corrected red blood cells could be used for transfusions to reduce complications from repeated donor transfusions. Even more promising, this technology could lead to the ability to correct the SCD gene in a patient’s own bone marrow, providing hope for a widely available cure of SCD in the future.
Similarly, significant progress continues to be made in the diagnosis and management of chronic lung diseases. Chronic Obstructive Pulmonary Disease (COPD) is the third leading cause of death and a significant contributor to disability in the United States. NHLBI-supported research has shown that certain treatments and lifestyle changes—such as wearing oxygen masks at night and quitting smoking—can improve quality of life for people with COPD. NHLBI is now using new technologies to study the complex structure of the lung in ways not possible before. For example, NHLBI’s COPDGene study has demonstrated that COPD is not one disorder, rather, there are several subtypes and some clusters are associated with known genetic variants. NHLBI’s LungMAP project is examining the complex interplay among different cell types that result in the development of healthy lungs. These technologies will allow us to better understand pre-symptomatic stages of lung disease, differentiate subclasses of disease early, and identify factors that contribute to resilience, which could lead to more effective treatments to prevent lung disease progression before symptoms begin and irreversible damage has occurred.
REDUCING HEALTH INEQUITIES
NHLBI has a long history of studying how HLBS diseases impact subgroups of the population differently and that the inclusion of these subgroups in research can identify ways to tailor treatments. For example, the NHLBI-funded Women’s Ischemia Syndrome Evaluation (WISE) study showed that the location of blockages in the coronary arteries that cause heart attacks differs in women compared to men. NHLBI-supported clinical trials also demonstrated that low-dose aspirin was effective in reducing the risk of stroke in women but not heart attack, unlike what was seen in men. These results gave rise to clinical guidelines for the differential use of aspirin in CVD prevention for men and women. Ongoing basic research in animal models is exploring the role of sex as a biological variable underlying this difference.
Similarly, NHLBI is invested in better understanding factors that increase risk of CVD among African Americans. For example, a recent study of participants from the Jackson Heart Study and WHI showed that two risk variants in the APOL1 gene, which are known to contribute to kidney disease, also increase risk of CVD in African Americans. Studies such as these identify ways to reduce health inequities and point to new lines of research that can provide a foundation for improving health of all citizens.
NHLBI remains committed to continuing its legacy of success in supporting research that addresses the major causes of death. The ability to arrest disease before symptoms begin and treatment costs are incurred can generate an unprecedented return-on-investment in biomedical research and a healthier and wealthier nation.
Gary H. Gibbons, M.D.
Director, National Heart, Lung and Blood Institute
Gary H. Gibbons, M.D., is Director of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), where he oversees the third largest institute at the NIH, with an annual budget of approximately $3 billion and a staff of nearly 1,000 federal employees.
NHLBI provides global leadership for research, training, and education programs to promote the prevention and treatment of heart, lung, and blood diseases and enhance the health of all individuals so that they can live longer and more fulfilling lives.
Prior to being named director of the NHLBI, Dr. Gibbons served as a member of the National Heart, Lung, and Blood Advisory Council (NHLBAC) from 2009-2012. He was also a member of the NHLBI Board of Extramural Experts (BEE), a working group of the NHLBAC.
Before joining NHLBI, Dr. Gibbons served as the founding director of the Cardiovascular Research Institute, chairperson of the Department of Physiology, and professor of physiology and medicine at the Morehouse School of Medicine, in Atlanta.
Under his leadership of the Cardiovascular Research Institute, Dr. Gibbons directed NIH-funded research in the fields of vascular biology, genomic medicine, and the pathogenesis of vascular diseases. During his tenure, the Cardiovascular Research Institute emerged as a center of excellence, leading the way in discoveries related to the cardiovascular health of minority populations. Dr. Gibbons received several patents for innovations derived from his research in the fields of vascular biology and the pathogenesis of vascular diseases.
Dr. Gibbons earned his undergraduate degree from Princeton University in Princeton, N.J., and graduated magna cum laude from Harvard Medical School in Boston. He completed his residency and cardiology fellowship at the Harvard-affiliated Brigham and Women's Hospital in Boston. Prior to joining the Morehouse School of Medicine in 1999, Dr. Gibbons was a member of the faculty at Stanford University in Stanford, Calif., from 1990-1996, and at Harvard Medical School from 1996-1999.
Throughout his career, Dr. Gibbons has received numerous honors, including election to the Institute of Medicine of the National Academies of Sciences; selection as a Robert Wood Johnson Foundation Minority Faculty Development Awardee; selection as a Pew Foundation Biomedical Scholar; and recognition as an Established Investigator of the American Heart Association (AHA).