DEPARTMENT OF HEALTH AND HUMAN SERVICES
NATIONAL INSTITUTES OF HEALTH
Fiscal Year 2013 Budget Request
Statement for the Record for the House Subcommittee on Labor-HHS-Education Appropriations
Susan B. Shurin, M.D., Acting Director
National Heart, Lung, and Blood Institute
Mr. Chairman and Members of the Committee:
I am pleased to present the President’s budget request for the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH). The Fiscal Year (FY) 2013 NHLBI budget of $3,076,067,000 includes an increase of $709,000 over the comparable FY 2012 level of $3,075,358,000.
The NHLBI leads research and education programs to discover and apply knowledge to improve health by preventing and treating heart, lung, and blood diseases. I appreciate the opportunity to highlight just a few examples of our success in doing so and some of our most promising research programs that will enable further advances.
CHRONIC DISEASE RISK REDUCTION
Cardiovascular diseases (CVD) and pulmonary conditions are among the leading causes of disability and death around the world. Although their prevention and treatment have improved dramatically, without further progress they will continue to impose an increasing health burden as our population ages. A recent meta-analysis of lifetime risk for CVD underscored the availability of lifelong opportunities for CVD prevention. The Institute is funding a clinical trial to examine diet and exercise interventions to improve neurocognition in patients with CVD risk factors who have cognitive impairment. Effective ways to help people lose weight and sustain weight loss were identified in an NHLBI-supported study reported in November 2011; multiple ongoing projects are addressing ways to help children and adults in a wide range of circumstances improve their health through weight control and physical activity.
The NHLBI continues to focus upon understanding CVD risk in vulnerable populations. The Jackson Heart Study is addressing the biological, behavioral, and psychosocial factors that account for the high burden of CVD in African Americans. The Hispanic Community Health Study–Study of Latinos is addressing the factors involved in the prevalence and development of CVD in Hispanic populations in the U.S. Both studies are expected to be renewed in FY 2013. A new program planned for FY 2013 will foster development of effective and sustainable public health interventions to reduce CVD morbidity and mortality in high-risk rural populations.
INTERPRETING THE HUMAN GENOME IN HEALTH AND DISEASE
Data from the NHLBI’s substantial investment in whole exome sequencing of participants in its long-term cohort studies is paying off: data are now being deposited in dbGaP, the informatics resource at the National Library of Medicine, for use by investigators around the world. The return on this investment will provide valuable new diagnostics and treatments for the next decade.
The NHLBI has led multiple global consortia in sharing data and encouraging analysis of large genomic data sets linked to phenotype. One such consortium identified 16 genetic loci important for control of blood pressure that are now being explored by other NHLBI-supported investigators as new approaches to control blood pressure. Still other NHLBI-supported studies are revealing the genetic and environmental causes of chronic obstructive pulmonary disease (COPD), asthma, abnormalities of heart rhythm, and factors that affect the severity of hemoglobin disorders such as sickle cell disease.
NEW THERAPIES FOR HEART, LUNG, AND BLOOD DISORDERS
The NHLBI supports development of improved therapies for heart disease through resources such as the Cardiac Translational Research Implementation Program (C-TRIP) and their assessment in clinical trials through Institute-initiated programs such as the Pediatric Heart Network (now completing a trial in Marfan’s syndrome and multiple studies of genetics and clinical management of congenital heart disease), the Heart Failure Network (conducting studies of cellular and drug therapies of heart failure), and the Cardiothoracic Surgical Trials Network (conducting comparative studies of surgical approaches).
Several NHLBI programs are advancing translation of basic scientific knowledge into new therapies. The Centers for Advanced Diagnostics and Experimental Therapeutics in Lung Diseases (CADET) will accelerate the development of agents for diagnosing and treating lung diseases. Investigators are partnering with other NIH Programs such as Therapeutics for Rare and Neglected Diseases (TRND) to do early-stage translational work that will be followed by NHLBI-supported clinical trials.
GENE AND CELLULAR THERAPIES
NHLBI-supported scientists recently reported success in treating hemophilia B, an inherited bleeding disorder, in several patients with a single infusion of a gene therapy that durably boosted the production of the missing clotting factor. If confirmed in other patients, this approach may allow patients to minimize or discontinue expensive treatment with replacement clotting factor.
Encouraging results from studies that use gene therapies in animal models for other diseases offer promise for the treatment of human disease. For example, a unique genetic approach of replacing the single mutated amino acid in mice cured their sickle cell disease. A new form of gene therapy for heart failure improved heart function in pigs without apparent toxicity.
Bone marrow transplantation has been standard clinical therapy for certain diseases since the 1960s. The NHLBI is the primary institute supporting the Bone Marrow Transplant (BMT) Clinical Trials Network (CTN), with strong support from the NCI. A BMT CTN finding that use of mobilized peripheral blood stem cells rather than bone marrow substantially lowers the risk of graft-versus-host disease (an often fatal complication of BMT) has already affected practice and should lessen complications of BMT.
The NHLBI is supporting resources such as the Production Assistance for Cellular Therapies program to facilitate laboratory and clinical studies of cellular therapies to enhance healing after tissue damage caused by myocardial infarction and some forms of lung disease. Use of mesenchymal stem cells to repair tissue without scarring is being tested in early-stage human trials, with some very encouraging results.
The NHLBI supports infrastructures – registries, clinical trial networks, and biorepositories – to enable research on rare diseases and on risk factors for more common diseases. For example, both sporadic and Marfan-associated thoracic aortic disease may have a common pathway, and a genetic cause of aortic aneurysms may be more prevalent than previously thought. The NHLBI is a leader in conducting clinical trials in pulmonary hypertension and idiopathic pulmonary fibrosis. Linkage of genetic and clinical data with a biorepository is enabling identification of factors influencing the development of congenital heart disease.
Following promising studies in mice, the NHLBI is now completing a study of losartan, an FDA-approved antihypertensive drug, in Marfan syndrome. The NHLBI supported a clinical trial that showed rapamycin (Sirolimus) stabilized lung function, reduced symptoms, and improved quality of life in patients with lymphangioleiomyomatosis (LAM), a progressive cystic lung disease in women. NHLBI partnerships with patient advocacy organizations in the conduct of both trials facilitated their rapid enrollment and completion.
Sickle cell disease remains an area of intensive focus for the NHLBI. A trial recently demonstrated that hydroxyurea, known to be an effective treatment for adults, is also safe and effective in very young children. In FY 2013, the NHLBI plans to initiate Excellence in Hemoglobinopathy Research Awards to promote multidisciplinary basic and translational research and facilitate collaboration with clinical hematologists. The NHLBI has played a major role in an HHS-wide initiative to coordinate the research and health care delivery efforts of six HHS components to reduce the health burdens of hemoglobinopathies (sickle cell disease and thalassemia). The NHLBI is developing clinical practice guidelines to ensure that providers know the components of high quality, evidence-based care for sickle cell disease.
The NHLBI supports multiple studies, and works closely with the FDA, to ensure appropriate monitoring of the blood supply against potential threats. In 2010 and 2011, an NHLBI-led interagency group demonstrated that a xenotropic murine retrovirus (XMRV), which had been reported to be associated with chronic fatigue syndrome in some patients, did not pose a risk to the safety of the blood supply. NHLBI leadership ensured that this and other important health questions were quickly resolved.
Susan B. Shurin, M.D., Acting Director
National Heart, Lung, and Blood Institute
Susan B. Shurin, M.D., is Acting Director of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), where she oversees an extensive national research portfolio with an annual budget of approximately $3 billion. The NHLBI conducts and supports research to prevent, diagnose, and treat heart, lung, and blood diseases; fosters training of emerging investigators; and communicates research advances to the public. Through the support of research from bench to bedside and into the community, Dr. Shurin leads the NHLBI’s effort to transform new scientific knowledge into tangible improvements in health.
As acting director, Dr. Shurin represents the NHLBI in a wide variety of activities across the NIH and the Department of Health and Human Services. She is responsible for the basic, clinical and epidemiologic research supported and conducted by the Institute, translation of research findings to the public, and the intramural research program on the NIH campus. She has updated the NHLBI’s programs in research on hemoglobinopathies, including sickle cell disease, to recruit investigators from a variety of disciplines and ensure a broad focus on implementation of research results, especially for adult patients. She created a coordinating group of HHS agencies to work with the Office of the Secretary of HHS to enhance effectiveness of research and service programs for persons with hemoglobinopathies. Dr. Shurin created and oversees the NHLBI’s Global Health program, which includes a network of research projects in low- and middle-income countries building sustainable programs to combat chronic cardiovascular and lung diseases and is addressing the NHLBI’s strategic scientific goals through research in domestic and international settings. She chairs the Board of the Global Alliance for Chronic Disease, national funders of biomedical research from around the world addressing the pandemic of non-communicable diseases.
Dr. Shurin joined the NHLBI as Deputy Director in February 2006, coming from Case Western Reserve University in Cleveland, Ohio. In her role as deputy director, Dr. Shurin has been involved in multiple intramural and extramural activities of the NHLBI and responsible for oversight of the Institute’s clinical research portfolio. In October 2009 Dr. Shurin also assumed the role of Acting Director of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) while also serving as the deputy director of NHLBI.
Before joining the NHLBI, Dr. Shurin was professor of Pediatrics and Oncology at Case Western Reserve University in Cleveland, Ohio; Director of Pediatric Hematology-Oncology at Rainbow Babies and Children’s Hospital; Director of Pediatric Oncology at the Case Comprehensive Cancer Center; and Vice President and Secretary of the Corporation at Case Western Reserve University.
Dr. Shurin received her education and medical training at Harvard University and the Johns Hopkins University School of Medicine. Her laboratory research focused on the physiology of phagocyte function, recognition and killing of pathogens; mechanisms of hemolysis, red blood cell destruction; and iron overload, developing the first effective chelation therapy for chronically transfused patients with thalassemia major.
She has been active in clinical research in many aspects of pediatric hematology-oncology, including participation in the Children’s Cancer Group (CCG), now the Children’s Oncology Group, as well as multiple studies in sickle cell disease and hemostasis. She also served on the Executive Committee of the CCG and founded and chaired the CCG Bioethics Committee.