Children’s On Cutting Edge Of Research With GCRC
from The Children's Hospital (TCH) News, March 2005
The Children’s Hospital’s General Clinical Research
Center – one of just six GCRCs devoted to pediatrics in
the nation – currently is conducting 140 different
studies with more than 150 principal investigators and
co-investigators.
There’s a lot we don’t know about childhood diseases.
And the only way to find out more about those diseases and develop better treatments is to study them, said Ronald J. Sokol, MD, program director for The Children’s Hospital’s Pediatric General Clinical Research Center (GCRC).
In the 43 years since Children’s GCRC – one of just six GCRCs devoted solely to pediatrics in the nation – was established, Children’s has pioneered numerous protocols for treating childhood diseases, Dr. Sokol said.
“The current way preemies are treated is based on discoveries made here,” he said. “Newborn screening for cystic fibrosis (CF) was first tested here in 1982 and has proliferated across the country.”
Children’s GCRC also developed numerous treatments for children with HIV, diabetes, celiac disease and childhood liver diseases.
None of those advances would be possible without the National Institutes of Health-funded GCRC, which provides an optimal setting for medical investigators to conduct safe, controlled, state-of-the-art, inpatient and outpatient studies.
The University of Colorado at Denver Health Sciences Center (UCDHSC) and Children’s GCRC receive approximately $3.4 million a year, which funds the salaries of its 42 dedicated staff members, the core lab – used solely for GCRC research, six of 10 beds in an inpatient unit, an outpatient unit, equipment and supplies. Staff members include research nurses, biostatisticians, bionutritionists, bioinformatics personnel and research subject advocates.
But new and innovative treatments don’t start that way: they first begin as a doctor’s idea for a new way to treat a disease, Dr. Sokol said. The doctor, known as the principal investigator, submits a proposal to the GCRC, whose Scientific Advisory Committee (SAC) reviews the proposal and decides whether to support it. The proposal also must be submitted to the Colorado Multiple Institutional Review Board (COMIRB) for approval.
For example, Jennifer Hagman, MD, recently submitted a proposal to treat anorexia nervosa with a drug. The GCRC’s biostatisticians helped design a study, and bioinformatics personnel helped Dr. Hagman develop a confidential database of patients enrolled. The patients enrolled in Dr. Hagman’s study will only see nurses from the GCRC, who are trained in research protocols. If the patients are admitted to the hospital, they will sleep in the GCRC’s beds.
Once Dr. Hagman collects the data, which can take anywhere from six months to years based on the study, GCRC staff will help analyze the data.“Our biostatisticians are highly trained to assist and analyze data,” Dr. Sokol said.
The principal investigator of the study decides how long the study will be, which questions to answer, and how many patients to enroll.
“We’ve funded a study with one patient (a non-anemic child with an iron dependency) and now we’re doing a study on 1,400 patients with asthma in the ED,” said Theresa O’Lonergan, who ensures protocols are conducted ethically and according to federal regulations in her job as a research subject advocate.
The SAC receives about five proposals a month, said Puff Stevens, administrative director of the pediatric GCRC. The GCRC currently has 140 different studies underway, with more than 150 principal investigators and co-investigators, Dr. Sokol said. “We want to better understand the effects of diseases,” he said.
Approximately 16 of those studies are for different aspects of treatment of Cystic Fibrosis, Dr. Sokol said. One of the studies is on polycystic kidney disease (PKD) – one of the largest such studies in the nation, he said. Another study is on ways to treat type 2 diabetes. Another is testing a new type of insulin to treat type 1 diabetes. Ten to 20 of the studies relate to HIV disease. Yet another is testing inhaled nitric oxide’s effects on neonatal lung diseases.
The pediatric GCRC also performs some studies on adults when related to childhood health, Dr. Sokol said, including one study on a new treatment to prevent the transmission of HIV from a pregnant mother to her child.
The opportunity to do such important, government-funded research is a big draw for Children’s, Dr. Sokol said.
“We are one of the larger pediatric GCRCs in the country,” he said. “It’s very instrumental in attracting the best and brightest caretakers. It’s an opportunity for a doctor to participate in and perform top-notch clinical research.”
Stevens said, “It’s one of the easier ways for younger investigators to obtain NIH funding. The support and staff make it possible to do research they couldn’t do otherwise.”
The pediatric GCRC recruits patients for studies through pediatricians, family medicine physicians and PCPs in the community, through newspaper ads or by word of mouth, Dr. Sokol said.
Each of the 82 GCRCs across the country, including the one at Children’s, receives funding in five cycles, Stevens said. The pediatric GCRC completed an application for a grant in October that would fund it until 2011. The GCRC will find out if they will receive the funding in March of 2006, said Stevens, who coordinates the grant application process. The grant the pediatric GCRC submitted in October was 2,500 pages and required the full-time efforts of six to eight staff members for six months, she said.
The grant is awarded to UCDHSC, Stevens said – the site of the GCRC until 1990 – and a subcontract is written to Children’s. Some of Children’s GCRC staff see patients at UCDHSC as well, Dr. Sokol said.
The GCRC at The Children’s Hospital – one of the largest pediatric GCRCs in the country – is a real “gem,” Dr. Sokol said. “Only an elite group of children’s hospitals have pediatric GCRCs,” he said. “It allows us to be on the cutting edge of research and therapies and attract the best and the brightest.”
