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Robert Wood Johnson
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MEDICINE 31
housed in a dedicated 5,000 square feet of space within the
Child Health Institute of New Jersey. The staff includes
physicians, research nurses, nurse coordinators, a pediatric
psychologist, and a research pharmacist who coordinate the
business of conducting clinical trials. The challenges are
huge: protecting children from exposure to undue risks
while using effective methods to test promising drugs.
Access to Drugs Motivates Families
J
ulianne Wilson, RN, research coordinator, says the stud-
ies give kids the chance to receive medications they might
not otherwise have access to. "Most drugs are FDA-
approved for adults, but not approved for children," she
says. There's another reason families decide to participate.
"We tell our patients and families, even if the study doesn't
help them, it will help others in their situation in the future,"
says Wilson. "No matter the outcome, we learn from every
study, helping other children who have these diseases."
Research coordinator Lisa Cerracchio, RN, recalls one
feel-good moment that affected the entire team. "A young
boy suffered from idiopathic thrombocytopenic purpura
(ITP)--a disorder that can lead to easy or excessive bruising
and bleeding. He couldn't participate in gym or play contact
sports," Cerracchio says. "He came in for injections for an
ITP trial. Just as a result of the drugs to treat his condition
that he received in the study, he found out he could sign up
for Pop Warner football. He got the biggest smile on his face.
We walked away feeling fantastic because he was so happy."
The center has expanded in scope since its inception in
2007. "Because of the quality research and data produced
by the studies at the PCRC, there has been tremendous
growth," says Dr. Gaur. Now the center is part of national
and international networks doing research to look for new
treatments. There are about 25 studies under way at any one
time. Some may have one or two patients in them; in others,
as many as 15 are enrolled. Researchers are currently doing
work that could have far-reaching consequences.
"One of the primary missions of the PCRC is the training
of tomorrow's pediatric clinical investigators," says Marc
Sturgill, PharmD, associate professor and chair, Department
of Pharmacy Practice and Administration, Rutgers Ernest
Mario School of Pharmacy, and associate director of educa-
tion, PCRC. "We're actively involved in the training and men-
toring of medical and pharmacy students and residents as well
as Rutgers University pharmaceutical industry fellows."
Breaking Ground on Drugs
for Cystic Fibrosis
T
he cystic fibrosis (CF) research collaboration is rela-
tively new. Thomas F. Scanlin, MD, professor and sen-
ior vice chair, Department of Pediatrics, and chief, division
of pulmonary medicine and the Cystic Fibrosis Center,
explains, "We've partnered with the PCRC to become a
therapeutic development network." The first trial potential-
ly affects only 4 percent of cystic fibrosis patients, but there
was spectacular improvement in members of that group.
Then a breakthrough drug was tested that could affect more
than half of all patients with CF. The trial produced a result
that was less than expected--but a newer, more advanced
version of that drug is being readied for research. Results