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February 13, 2004
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For Further Information:
Bess Andrews
617-355-6420
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New Federal Program Speeds Application of Basic Research Advances to Clinical Care
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The National Heart, Lung, and Blood Institute (NHLBI) has awarded a major new grant to Children's Hospital Boston to accelerate research aimed at understanding heart development and treating pediatric heart disease. The grant is part of a new initiative, the Specialized Centers of Clinically Oriented Research (SCCOR) program, sponsored by the National Institutes of Health (NIH) to foster multidisciplinary collaborations so that basic research advances are rapidly translated to clinical care. Researchers at Children's Hospital Boston and affiliated Harvard institutions, led by Dr. Jane W. Newburger, Associate Chief of Cardiology, will receive approximately $20 million over 5 years. Children's Hospital Boston is one of four pediatric heart centers to share a total of $68 million in NHLBI SCCOR funding; the other centers are Children's Hospital of Philadelphia, Children's Hospital of Pittsburgh, and Cincinnati Children's Hospital Medical Center.
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Newburger's group will focus on improving the treatment and outcomes of children with tetralogy of Fallot (TOF), a common congenital heart condition characterized by a ventricular septal defect (a hole in the wall separating the right and left ventricles, the heart's pumping chambers) and pulmonary stenosis (obstructed blood flow to the lungs). TOF, which is named for the French physician who first described it in 1888, comprises approximately 8 to10 percent of congenital heart disease in the United States and is the most common form of cyanotic (impeding oxygenation of the blood) congenital heart disease. In approaches ranging from laboratory-based investigations to clinical human studies, Newburger and her colleagues will explore TOF's genetic causes; the developmental, neurologic, and behavioral outcomes in adolescent survivors of TOF; surgical methods to optimize long-term health of the right ventricle; and molecular pathways and cellular strategies for improving right ventricular function, including heart muscle regeneration using zebrafish models. Newburger's colleagues on the SCCOR grant include clinical and basic scientists from Children's Hospital, Harvard Medical School, the Beth Israel Deaconess Medical Center, and Harvard University.
''Over the past two decades, advances in surgical and medical care have reduced mortality rates for virtually all forms of congenital heart disease. With improved survival has come the critical need to understand the basis of congenital heart disease in children and to improve their quality of life,'' says Newburger.
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Since the first surgical repair of TOF nearly 50 years ago, survival of patients with TOF has steadily increased, with early mortality currently less than 2 percent. Late survival has also improved, with recent reports showing a 20-year survival rate nearing 90 percent. A conservative estimate projects that the number of survivors of TOF repair in the United States exceeds 100,000 and increases by as many as 4,000 annually. With improving survival of children born with TOF has come increasing recognition of its long-term consequences, including an enlarged right ventricle (the heart chamber pumping to the lungs), and cardiac arrhythmias (problems with the electrical system of the heart), which increase with age and sometimes require medical therapy, radiofrequency ablation, or implantation of automatic cardiac defibrillators. Poor function of the right ventricle, symptoms of right heart failure, and arrhythmias affect a substantial proportion of individuals with TOF as they grow into adulthood. Moreover, some patients with TOF can have learning problems that may be congenital or genetic, or may result from the therapies, such as cardiac surgery, necessary to treat their heart disease. Newburger and her colleagues hope to prevent these consequences and ultimately prevent the condition altogether.
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Cardiovascular malformations, which are present in approximately one percent of live births, are the largest contributor to deaths from birth defects. Patient advocacy groups have advocated to make February 14th Congenital Heart Defects Awareness Day. Sponsored by the Congenital Heart Information Network, this national awareness day is dedicated to help reduce illness and death due to congenital heart defects, and to honor affected families. Despite enormous strides in understanding basic mechanisms of heart development, the underlying causes of most congenital cardiovascular defects remain unknown. And while clinical advances have made medical and surgical treatment of complex heart defects possible in the tiniest of infants, the survivors still face numerous challenges.
The NHLBI-supported SCCORs encompass priorities of the NIH Roadmap for Medical Research outlined in autumn 2003 by NIH Director Dr. Elias A. Zerhouni. The projects highlight the strategy of encouraging a team approach that combines skills and disciplines to accelerate movement of scientific discoveries from the bench to the bedside. The use of multidisciplinary teams is a key feature of the pediatric SCCORs, which will enlist a cadre of experts, including pediatric cardiologists and cardiovascular surgeons, immunologists, geneticists, and other pediatric clinicians, as well as molecular biologists, cell biologists, and biostatisticians, who will pool their talents to conduct state-of-the-art research.
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Children's Hospital Boston is home to the world's largest research enterprise based at a pediatric medical center, where its discoveries have benefited both children and adults for over 100 years. More than 500 scientists, including seven members of the National Academy of Sciences, nine members of the Institute of Medicine and nine members of the Howard Hughes Medical Institute comprise Children's research community. Founded in 1869 as a 20-bed hospital for children, Children's Hospital Boston today is a 300-bed comprehensive center for pediatric and adolescent health care grounded in the values of excellence in patient care and sensitivity to the complex needs and diversity of children and families. It is also the primary pediatric teaching affiliate of Harvard Medical School. For more information about the hospital visit: www.childrenshospital.org.
NHLBI is part of the National Institutes of Health (NIH), the Federal Government's primary agency for biomedical and behavioral research. NIH is a component of the U.S. Department of Health and Human Services. NHLBI press releases and other materials, including a report of the Task Force on Research in Pediatric Cardiovascular Disease, are available online at: http://www.nhlbi.nih.gov/resources/docs/pediatric_cvd.pdf.
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