What motivates people to participate in medical research? As a physician and researcher who has spent two decades conducting research to help children and families manage their diabetes, Linda DiMeglio, MD, MPH, poses that question often.
One teenager’s answer still resonates with DiMeglio, a professor of pediatrics at the Indiana University School of Medicine and physician at Riley at IU Health. “He said he wanted to be part of our study because he hoped in a few years, other kids who are diagnosed wouldn’t be managing their diabetes same the way he was,” she says.
Her research team in Pediatric Endocrinology/Diabetology at Riley at IU Health is chipping away at the problem, examining how new technologies such as cell phone glucometers, insulin pumps and glucose sensors can improve quality of life for children with Type 1 diabetes. They also play a leading role in multi-site studies that explore how certain drugs can promote insulin production and prevent Type 1 diabetes in at-risk people.
One of their latest projects takes a new approach to diabetes treatment and prevention. It uses a drug called DFMO, originally prescribed for conditions other than diabetes. In 2014 the Juvenile Diabetes Research Foundation funded DiMeglio as principal investigator for a clinical trial of the drug, which is currently enrolling participants in Indianapolis, Buffalo, New York and Milwaukee, Wisconsin.
While DFMO alone is not likely to cure diabetes, DiMeglio says it could prove beneficial for treatment of new onset diabetes. It’s an oral medication that can be used in combination with other drugs and, so far, it shows minimal side effects. “Since Type 1 diabetes is an autoimmune disease, most therapies to date are devoted to counteracting the immune system,” DiMeglio says. “This one aims to improve the health of cells that make insulin while they are under attack.”
The research originated through a partnership between DiMeglio and her colleague, Raghu Mirmira, MD, PhD, director of the Center for Diabetes and Metabolic Disease and professor of pediatrics at the IU School of Medicine (IUSM). Not long after Mirmira came to Indiana in 2008, the two began looking for ways to unite Mirmira’s laboratory
research with DiMeglio’s human research on diabetes and other diseases.
They found potential in a series of experiments Mirmira’s basic science group ran to see how various drugs could reverse the progression of diabetes in mice. One experiment involved DFMO, which seems to interfere with a process that can cause pancreatic beta cells to die. Beta cells produce, store and release insulin to the body. Keeping these cells healthy could be good for diabetic patients.
The three-site study aims to verify the safety and effectiveness of the drug given in varying doses. Researchers also study patient samples in a lab setting to see if patients who receive the drug share the same beta cell improvement previously seen in mice.
Their work shows why basic science, human volunteers and collaboration between researchers are so important to public health. “We could not have done this study without the basic science group, and the basic science group could not have done this without us,” DiMeglio says. “It’s a true partnership.”
As Indiana continues to build its reputation as a national leader in pediatric healthcare research, such collaboration often helps secure funding. “We have the expertise and the infrastructure at Riley at IU Health and IUSM to do these kinds of trials,” DiMeglio says. “We let a funding agency know that we were capable to do this study, got funding, took data from the lab about a drug that had already been used in humans
for other indications, showed that it worked in mice, procured a drug supply and got FDA and International Review Board approval. Now that we’re doing the trial, we’re giving human samples back to the lab to run the same assays.”
If you or a family member has Type 1 diabetes or the risk of having it, we invite you to learn more about this or other nearby studies that are open for enrollment. Contact us at firstname.lastname@example.org or call 317.278.8879.