NHLBI awards $68 million to fund clinical centers in pediatric heart disease

02/13/04

New program speeds application of basic research advances to clinical care

The National Heart, Lung, and Blood Institute (NHLBI), one of the National Institutes of Health (NIH), has awarded grants to four centers to accelerate research aimed at understanding heart development and treating pediatric heart disease.

The research is part of a new program, the Specialized Centers of Clinically Oriented Research (SCCOR) program, which is designed to foster multidisciplinary collaborations so that basic research advances are rapidly translated to clinical care. This research also supports the NIH roadmap initiative unveiled last fall by NIH Director Elias A. Zerhouni, M.D.

"These centers address a critical public health need for research that examines the basis of congenital and acquired heart disease in children," said Acting NHLBI Director Barbara Alving, M.D. "By understanding the choreography of molecular events that creates a four-chambered organ with valves, vessels, and electrical wiring, we can hope to better prevent, diagnose and treat pediatric heart disorders," she added.

Birth defects are a leading cause of infant mortality. Cardiovascular malformations, which are present in approximately one percent of live births, are the largest contributor to deaths from birth defects. Despite the enormous strides researchers have made in understanding heart development at the cellular level, in many cases the underlying cause of the cardiovascular defect is 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.

"If we can improve the odds of these children surviving-and increase their quality of life-then this program will have been a success," said Gail Pearson, M.D., Sc.D., leader of the NHLBI's Heart Development, Function and Failure Scientific Research Group.

Pearson noted that February 14 is Congenital Heart Defects Awareness Day. This national awareness day, sponsored by the Congenital Heart Information Network, is dedicated to helping reduce illness and death due to congenital heart defects, and to honor affected families.

According to Pearson, the use of multidisciplinary teams is a key feature of the pediatric SCCORs. The Centers 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 addressing a central pediatric cardiovascular theme.

The research themes, the centers awarded the 5-year grants, and the principal investigators of the SCCORS are:

  • Boston Children's Hospital (Dr. Jane Newburger). This group will aim to improve the treatment and outcome 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) and pulmonary stenosis (obstructed blood flow to the lungs). Projects will explore aspects of TOF, including its genetic causes; the developmental, neurologic, and behavioral outcomes in adolescents; 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.

  • The Children's Hospital of Philadelphia (Dr. Robert Levy). This team will investigate the genetic mechanisms that impact on the causes, treatment, and outcomes of congenital heart defects. One of the projects will explore novel biomaterials to improve surgical approaches for the repair of these defects in children. Other projects will focus on the genetic contributions to the etiology of congenital cardiac defects. The impact of specific genetic alterations on the health status of children with congenital heart defects will also be studied.

  • University of Pittsburgh and Children's Hospital of Pittsburgh (Dr. Steven Webber). This center will focus on improving long-term survival in pediatric heart transplantation. Some of the cutting-edge issues to be examined include decreasing the need for immunosuppression by improving the body's acceptance of a transplanted heart, understanding post-transplant tumors (lymphomas) and developing novel treatments for them, and identifying genetic markers that may predict disparities -- including racial, gender, and ethnic disparities -- in transplant outcomes.

  • Cincinnati Children's Hospital Medical Center (Dr. D. Woodrow Benson). This center will focus on the genetic basis of valve disease and identification of the molecular cause of valve defects so that risk can be assessed. By utilizing state-of-the-art molecular genetic analyses, researchers hope to define the pathogenesis of valve disease, facilitating the development of molecular-based therapy.

    Two of the centers (Boston and Pittsburgh) also will have Clinical Research Skills Development Cores. The Cores are designed to train fellows and junior faculty in the art and science of clinical research. New clinical investigators will gain experience in areas such as grant writing, ethical conduct of research, and clinical trial design.

    Source: Eurekalert & others

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