Johns
Hopkins Children's Center has received a nearly $5
million grant from the National Heart,
Lung and Blood Institute to establish a basic and
translational research center for sickle cell disease
that will consolidate research, treatment and care of adult
and pediatric patients under one roof and
speed up the translation of scientific discovery from bench
to bedside. In addition, the center will
offer counseling and education services to patients and
their families.
Sickle cell disease is an inherited blood disorder
marked by a mutation in the hemoglobin genes.
The defect causes oxygen-starved, abnormal crescent-shaped
red blood cells that give the disorder
its name. The sickle-shaped cells get stuck in blood
vessels, leading to excruciatingly painful strokes
and organ damage. Sickle cell anemia affects nearly 72,000
Americans, primarily African-Americans.
"This center will be a marriage of all aspects of
science and treatment, from basic science and
clinical research to patient care and public health
research, all part of the quest to treat and
ultimately cure sickle cell disease," said lead
investigator James F. Casella, Rainey Professor and chief
of Pediatric Hematology at Johns Hopkins.
By drawing on the expertise of researchers from
diverse areas, the center's faculty and staff
expect to advance promising therapies more rapidly. For
example, a basic-science project led by Allen
Everett, a pediatric cardiologist, will use the science of
proteomics, the study of proteins, to look for
biomarkers involved in silent strokes, which are a leading
cause of neurologic complications in sickle
cell patients. Discoveries in this area will ultimately
lead to better understanding, earlier diagnosis,
treatment and prevention of neurovascular problems in
sickle cell patients.
On the public health end of the spectrum, researchers
from the Johns Hopkins Bloomberg
School of Public Health, led by Cynthia Minkovitz, will
examine how local public health services affect
disease course and survival, and then pinpoint public
health measures that will help eliminate state-to-
state disparities in patient outcomes.
The translational arm of the center, led by urologist
Arthur Burnett, will help take lab
discoveries to the patient's bedside. For example,
scientists will study the role of nitric oxide in sickle
cell priapism, long-lasting painful erections that are a
common complication of the disease.
Researchers will then examine whether certain medications
that affect nitric oxide levels might
reduce and prevent this dreaded complication, which often
requires treatment at the emergency room
or hospitalization.
The center will also support a faculty scholar in
sickle cell disease and a summer program in
sickle cell disease research for high school students.
Other partners on the grant include the University of
Alabama, which, like Johns Hopkins, has a
long history of sickle cell disease research and care.
In February, The Johns Hopkins Hospital opened an
urgent care center that specializes in
treating sickle cell patients experiencing acute pain. In
2000, Hopkins opened an adult sickle cell
disease center that focuses on chronic care.
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