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34 Robert WoodJohnson
could be truly groundbreaking. "We've never had drugs like
this--take two pills a day, for example, and see dramatic
improvement for cystic fibrosis," says Dr. Scanlin.
Usually airway clearance, which involves a rigorous daily
routine, is the protocol for patients with CF. "These new drugs
are an improvement on top of the daily regimen," says Dr.
Scanlin. The cystic fibrosis study is approved for patients as
young as 12 years old, and the therapeutic development net-
work is getting geared up to do research with children who are
6 or older. "It's exciting if in the future, after a newborn screen-
ing, we can start the child on medications before scarring
begins on his or her lungs," the doctor adds.
Record Enrollment in Studies for Sickle Cell
he PCRC is also conducting a study for Mast Therapeutics
that looks at a new drug to treat sickle cell crisis. The
drug, MST-188, would enable kids to leave the hospital sooner
and enhance their quality of care by decreasing the time they
are in crisis. The study is a placebo trial that involves a 48-hour
infusion. Researchers look at pain medications used and the
last dose in comparison to patients not getting the drug. The
PCRC sickle cell study has among the highest enrollment num-
bers in the country. "We have a relationship with these patients,
many of whom we've known since they were babies," says
Richard Drachtman, MD, professor of pediatrics and chief,
division of pediatric hematology and oncology. "They are will-
ing and excited to participate in this research."
According to Dr. Drachtman, the PCRC will soon conduct a
study for another sickle cell drug, from Pfizer, called Rivipansel.
The study, which has been put on the fast track, will look at
treating sickle cell crisis by attacking the inflammatory compo-
nent. A number of patients are already enrolled. Dr.
Drachtman, like Dr. Gaur, emphasizes the importance of pedi-
atric research. "It's very important to get these trials done," he
says. "These kids are the generation of the future. We want
them to have a better life--and to be advocates for research."
Studying Low-Birth-Weight Babies
n neonatal intensive care units (NICUs), bronchopulmonary
dysplasia (BPD) is a threat to babies who were born weigh-
ing less than 1,500 grams. It's the most common chronic prob-
lem in neonates who are on prolonged mechanical ventilation
due to respiratory distress syndrome. "These babies often go
home on oxygen with saturation monitors and are prone to
infection," says Maya Ramagopal, MD, assistant professor of
pediatrics. Dr. Ramagopal, who is spearheading research on
BPD through the PCRC, says the infants often undergo tra-
cheal aspiration--an invasive method to suction the airway--
when they develop signs of a lung infection. "We asked our-
selves, how could we make things better for these babies?
"We thought about how children with asthma are monitored
and applied that thinking to the BPD study," says Dr.
Ramagopal. Older children with asthma can breathe into an
RTube to obtain exhaled breath condensate (EBC), in which indi-
cators of airway inflammation can be measured. Dr. Ramagopal
and her colleagues Elizabeth Yen, MD '10, neonatology fellow,
Department of Pediatrics, and Robert J. Laumbach, MD '97,
MPH, associate professor of environmental and occupational
health, Rutgers School of Public Health, collected EBC and tra-
cheal aspirates from neonates and measured cytokines and nitrite
levels--indicators of inflammation and oxidative stress. If the
levels in EBC and tracheal aspirators are correlated, EBC might
eliminate the need for the invasive method.
To date, the researchers have enrolled 40 infants, at or less
n neonatal intensive care units, bronchopulmonary
dysplasia (BPD) is the most common chronic problem in
neonates who are on prolonged mechanical ventilation due
to respiratory distress syndrome. "We asked ourselves, how
could we make things better for these babies?" says Maya
Ramagopal, MD, assistant professor of pediatrics, who is
spearheading research on BPD through the Pediatric Clinical
Research Center at Robert Wood Johnson Medical School.