Research into childhood diseases help children today and tomorrow
Over the years, medical care has seen major advancements, many times because of clinical research studies that look at new drugs, new treatments and new devices. At the Atlantic Center for Research, pediatric clinical trials “have uncovered very important basic science as well as clinical findings for a variety of diseases,” says pediatric gastroenterologist Joel Rosh, MD, vice chairman, Pediatric Clinical Development and Research Affairs.
“There is a lot of research happening at Goryeb Children’s Hospital, and our experience has been that we have a very engaged patient population,” says Dr. Rosh. “There are safeguards in place to make sure that subjects in human trials are protected. In pediatrics, we not only get consent from the parents but assent from the child as well.”
The program has been involved in research trials for pediatric diabetes, cystic fibrosis, cancer, neuromuscular diseases, and inflammatory bowel disease, to name a few. “We’ve had close to 500 children whom we’ve studied with inflammatory bowel disease, and this has led to important and clinically impactful findings,” says Dr. Rosh. “There are other medical conditions, such as pediatric neuromuscular disease, where there were never any treatments and, because of research, there are now therapies that are actually approved and available to our patients and the children in our community.”
Key members of the team include clinical research nurses who work with families to make sure they understand everything involved in a study. “Our research nurses are critical to the success of every research project,” says Dr. Rosh.
Last year, more than 100 children treated at Goryeb were enrolled in research protocols and more than two dozen entered into drug trials. Such trials provided access to treatments that would otherwise not have been available to them. Some of the protocols are treatment-based, so many patients have been helped as a result of participating in a trial.
“In addition, many of our research projects are investigating the actual causes of disease and we make sure to give feedback to the family as we make discoveries and report findings. In performing this type of research on chronic diseases, we may not get answers applicable to every child participating in the study today, but their participation may help others so that when today’s research participants have children, they will not have to worry about the same disease.”