NHLBI Awards $68 Million to Fund Clinical Centers in
Pediatric Heart Disease
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.
The NHLBI-supported SCCORs encompass priorities of
the NIH Roadmap for Medical
Research. The projects highlight the strategy of encouraging
a team approach that
combines skills and disciplines in order to accelerate
movement of scientific discoveries from the bench
to the bedside.
To arrange an interview with Dr. Gail Pearson, contact
the NHLBI Communications Office at 301-496-4236 or
nhlbinews@nhlbi.nih.gov. Contact information for the
principal investigators follows:
To interview Dr. Woodrow Benson of Cincinnati Children's
Hospital Medical Center, contact Jim Feuer at 513-636-4656;
to interview Dr. Robert Levy of Children’s Hospital
of Philadelphia, contact John Ascenzi at 267-426-6055;
to interview Dr. Jane Newburger of Boston Children’s
Hospital, contact Bess Andrews at (617) 355-6420;
to interview Dr. Steven Webber, University of Pittsburgh,
contact Lisa Rossi at (412) 647-3555.
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
are available on the NHLBI
Web Site. See below for direct links to a report
and summary of the Task Force on Research in Pediatric
Cardiovascular Disease:
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