Children’s Research Institute Gaining Momentum
By Holly Auer, The Post and Courier, Charleston, S.C.
Feb. 20–Inside the Medical University of South Carolina’s Charles P. Darby Children’s Research Institute, in laboratories tasked with investigating mysteries from childhood cancers to insulin resistance, secrets are hiding.
And bit by bit, the scientists who work there are teasing those secrets out into view.
A year after it opened, the facility is gaining speed in its drive toward a still-novel concept in the medical research world: Seamlessly linking basic science findings with the patients who need them to get well.
Emboldened by a flood of research on the tiniest pieces of the human body — its genes, proteins and stem cells — and worried about increasingly complex health problems, the National Institutes of Health is calling for better integration of laboratory science and clinical investigation.
The CRI is designed to answer that challenge. Though the groundwork has been laid, Bernard Maria, a pediatric neurologist who serves as the CRI’s executive director, said the process often remains slow going.
Medical schools teach very little about how to conduct research, Maria said, and the financial pressures of managed-care systems demand that doctors devote their whole day to caring for patients. Even if they’re inclined to do research, few have time to compete for grant money.
And then there are the egos and the stereotypes about who takes what path in medicine.
Basic scientists tend to perceive clinical researchers as second class citizens, Maria said, who don’t understand or respect the science that allows them to heal patients. The clinicians, on the other hand, see the scientists as anti-social lab rats who spend their days fiddling with tiny animals.
“It’s as if you took people that speak two totally different languages and sat them at a table,” Maria said. “What’s going to come of that?”
Plus, there’s the fickle nature of science itself — hypotheses can fall to pieces, and experiments don’t always go as planned. It all requires a degree of patience many people don’t have in a world driven by instant gratification.
“Science doesn’t work like ‘Eureka!’ ” said Rob Gourdie, a professor of cell biology whose CRI lab team created a new wound-healing technology from a naturally occurring protein in the body that regulates communication between cells. “It’s a group of people, with constant discussion going on. Over time, the picture starts to arrive.”
After a year at the CRI, that picture is varied and potentially ground-breaking. Among the array of first-year findings from basic science and clinical investigators:
–One of the same statin drugs that lowers dangerously high cholesterol in adults appears to help prevent Type 1 diabetes, also known as juvenile diabetes. Armed with positive results in animals, investigators now believe trials on humans may be warranted.
–A multi-disciplinary approach to treating children in the emergency room cuts down on overcrowding during wait times, costs per patient and the number of patients who leave the hospital without getting treatment. At the same time, patient satisfaction increases.
–Children with the devastating bone disease osteoporosis can benefit from a regimen of Fosamax, the same drug used widely in adults with bone density problems, plus vitamin D and calcium. During the yearlong study, child participants showed increased bone mineral density and had no new fractures.
Other research in the new facility is on its way to having commercial applications. Gourdie and his team have formed a biotechnology company and applied for a patent for their wound-healing technology. Experiments are underway now in animals, and Gourdie is hopeful the results will be used in fields from cosmetic surgery to diabetic wound care.
Sometimes, just having the new research facilities on hand has spurred new knowledge in the hospital setting — almost instantaneously.
Maria, for instance, recently cared for an infant who was found to have a brain tumor and multiple fluid-filled cysts in her head. Perplexed by the strange presentation, he sent samples of the fluid to the CRI’s proteomics lab, which studies the ever-changing role of tiny proteins in the body.
The results of their investigation came back in just a few days — a protein normally produced in the liver had showed up in the fluid. To Maria, that finding opened up doors for alternative treatments no one had considered.
“With this tumor, people were throwing their hands up in the air,” he said. “That should never be good enough.”
It’s hoped that in time the proteomics investigators will reveal more information about how different proteins act on tumors, which could be an important aspect of MUSC’s push to gain a designation from the National Cancer Institute, said Dr. Daniel Knapp, who oversees the proteomics program. Other protein findings may come to bear on psychiatric treatment.
In all these projects, there will likely be stumbles. But it’s all part of the game when it comes to beating down what ails the human body.
“Nature’s a hard teacher,” Gourdie said. “It doesn’t give up its secrets easily.”
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