Where Kids Volunteer
words by eve jacobs / photographs by john emerson
Sunanda Gaur, MD, professor of pediatrics, UMDNJ-Robert Wood Johnson Medical School, and director, Pediatric Clinical Research Center (PCRC),
own the hall from the office of Sunanda Gaur, MD, where we sit conversing, a young man lying in a hospital bed has just consented to be part of a Phase 1 clinical trial of a new systemic
antibiotic — which has been studied in adults, but not yet in kids.
The teen will be given one dose of the antibiotic, to be followed by six or seven blood draws over a period of 24 hours — not the most comfortable of prospects.
Approaching the parent and child to ask for their participation was sensitive, but Gaur is adept at this.
“I introduced myself and told them I’m an infectious disease specialist. I explained why it’s important to do clinical trials in children. I told them about this new trial, what the risks are and the benefits,” she says. (For this particular trial, the risk is low.)
“I explained to them that there may be no benefit to the child participating. I said, ‘We need data on this new medicine. This antibiotic covers some resistant bacteria, among them MRSA.’” Her exchange with the mother and son lasted about half an hour, and was a success.
“This mother said right away: ‘I understand. If the trial will help others, I would like my child to do this,’ and the teen agreed.” Children 7 and older must give assent, in addition to the parent or legal guardian, in order to be enrolled in a trial, the doctor explains.
But Gaur is very aware of the pitfalls of the process. “You have to be very careful how you present information,” she explains. “You must be very honest, very complete in how you describe the study. But it’s also important to appeal to the patient’s altruistic side if the study doesn’t directly benefit them. It’s always easier to enroll a patient with a chronic disease if you are offering a drug that has some promise for that disease.”
Just one year ago, the FDA issued a startling alert: “FDA strongly recommends that over-the-counter (OTC) cough and cold products should not be used for infants and children under 2 years of age because serious and potentially life-threatening side effects could occur…These include death, convulsions, rapid heart rates, and decreased levels of consciousness…”
Lisa Cerracchio, RNC, BSN research nurse coordinator, Pediatric CRC, with a
Why did babies become sick from cold medicines that anyone could purchase off the shelf in almost any drug store in this country? It’s because the old adage, “the proof is in the pudding,” has historically been ignored in regard to pharmaceuticals given to kids. It’s a well-known medical conundrum that many drugs given to children have never been tested in children. After conducting clinical trials on adults and gaining approval from the FDA, drugs can be prescribed for patients of any age.
“The doctor has had to extrapolate — break a pill or give a smaller dosage of a liquid medicine, maybe half the usual amount. But there are dangers in that approach,” says Gaur. “You can overdose or under-dose. You can cause harm. Kids have suffered because of this.”
A bill passed in 2002, called the Best Pharmaceuticals for Children Act, encourages drug testing in children by offering the companies a six-month patent extension. “Because of this legislation, more studies have been done. It has raised awareness of the concerns. But the incentive for testing drugs in children is financial.
Ideally, pharmaceutical companies should be federally mandated to test drugs in children,” states Gaur.
The pediatric CRC in New Brunswick was conceived seven years ago. “There was no dedicated clinical trials unit just for children in New Jersey,” she explains. With its own self-contained space, including four beds, a children’s playroom, laboratory, a waiting area and exam rooms, the unit also provides dedicated nursing staff, an administrative and budget coordinator, Cathy Whalen, who helps “get the trial through from a germ of an idea to actuality in the shortest possible time,” and an assistant director, Marc Sturgill, PharmD, who brings to the program an extensive background and expertise in pharmacology.
“Just two years ago, the unit was still in its infancy,” relates Gaur, an infectious disease specialist whose 20 plus years of experience include conducting more than 40 HIV/AIDS clinical trials. “Now we are doing 15 studies and there are more in the works. We help faculty with contracts and negotiations and provide support on the nuts and bolts of running these studies.” Pharmaceutical companies provide the majority of support for the trials, with the others being NIH-sponsored.
In fact, Gaur and Sturgill are discussing a soon-to-start diabetes trial that is truly state-of-the-art. “We are testing a new, very promising biologic that may have the potential to reverse Type 1 diabetes in those just diagnosed with the disease and this is the first time it’s being tested in children,” she says enthusiastically. “Some adults have actually come off insulin.” The study is intensive, requiring the child to come into the pediatric CRC every day for 14 days after first being diagnosed, but the benefits could be life-changing.
Johnson & Johnson, GlaxoSmithKline, Amgen, AstraZeneca, Macrogenics and Cubist are among the pharmaceutical companies testing their new therapies here on young children and teens for conditions as varied as cystic fibrosis, kidney disease, systemic infection, diabetes, migraine, digestive problems, HIV, and blood irregularities caused by cancer chemotherapy. Several NIH-supported trials are also in the works.
Lisa Cerracchio, RNC, BSN, research coordinator of the Center, is a strong advocate for pediatric research. “The information we get today will help other kids in the future — and sometimes it helps a child right now. These drugs have been proven safe in adults, the children are very closely monitored and everything is voluntary. I tell the parents and the child that anyone can call it quits at any time.”
The PCRC team (l to r): Sunanda Gaur, MD,
Lisa Cerracchio, BSN, research nurse coordinator Stephanie Reveron, RN Cathy Whalen,
program administrator Marc Sturgill, assistant director, and associate professor, Ernesto Mario School of Pharmacy, Rutgers
Sturgill’s interest in the effects of age, disease and xenobiotics (something foreign to the body such as a pesticide) on the activity of human drug metabolizing enzymes, as well as his experience working in the adult CRC at RWJMS from 1992 until 2005 — when he joined the team of the pediatric CRC — makes him a perfect fit for this Center. “We want this to become a place that pharmaceutical companies come to rely on,” he says. Not only does the pharmacologist work on protocol design, regulatory and IRB approval, and statistical analysis, but he sees teaching the next generation of pharmacologists for industry and health care delivery as a priority.
“Dr. Gaur and I want to develop a state-of-the-art training environment where future physicians, pharmacists and nurses can get hands-on experience in the safe conduct of clinical trials in children. We want them to understand the importance of including children in all phases of clinical research. Pediatricians still find themselves being forced to prescribe medications for children without adequate published dosing information,” he explains.
Although the majority of what the pediatric CRC currently does is industry sponsored dose-ranging Phase 1 and 2 studies, Sturgill, who is an associate professor at Rutger’s pharmacy school and an adjunct assistant professor at RWJMS, wants to promote more investigator initiated trials – “trials designed by our own faculty.”
“We are very busy,” he says, “but we would like to grow.”
In this child-focused, child-friendly environment, safe and healthy growth is an unarguable certainty.