The Power of Volunteering for Clinical Research

By Samuel Dagogo-Jack, MD, FACE

A volunteer is someone who chooses to do something freely. Volunteers give freely of themselves, their time, and their resources. Many of you have donated your time, energy, money, and other resources to one cause or another, for the greater good. However, there is another aspect of volunteering you should know about.

Volunteers in clinical research have allowed modern medicine to advance. You may be surprised to know that until very recently, doctors had very little scientific facts or proven remedies to offer their patients. What moved the practice of medicine from the “dark ages” to the modern medicine of today are the advances in medical research and the people who volunteered for those studies.

Generally speaking, four elements come together to make for successful clinical research and discovery of new scientific breakthroughs.

First is the scientist who comes up with an idea. The idea must be unique and able to be tested.

Second is where the research takes place. That environment (including the groups overseeing the research and the group conducting the research, material support, laboratory, physical location, etc.) must support the growth of new ideas and must allow the proposed research study to be conducted.

Third is adequate funding. This usually comes in the form of a research grant. However, many great discoveries and breakthroughs came from a cash-strapped genius who worked alone in a crowded shack. For instance, in the 1920s Frederick Banting led a research team at the University of Toronto that discovered insulin, which radically improved the fate of millions of people around the world. His research was not funded by any major grant. He got loans from his father and brother so that he could keep working on his research.

Other noteworthy people who did not get large grants to fund their research were the Wright brothers and Einstein. However, clinical studies these days are critically dependent on large grants because research is extremely expensive and complex.

Fourth and the most important engine of clinical research are the selfless men and women who volunteer to serve as research participants. For example, after furious and demanding hard work through most of 1921, Dr. Banting’s group finally produced a crude “insulin” extract from animal pancreas and needed to test whether it would work. In January 1922, Leonard Thompson, a teenager with diabetes, became the first human being to receive an injection of the crude insulin extract. The result was astounding. For the first time ever it was clear that an extract from animal pancreas had an “anti-diabetic” effect on a human. After that finding, researchers perfected the crude insulin extract and mass produced it for use in patients around the world. Leonard Thompson was the first volunteer in human research regarding insulin treatment.

In 1948, the Framingham Heart Study was launched. Its goal was to identify “the common factors that contribute to cardiovascular disease by following its development over a long period of time in a large group of participants.” A total of 5,209 adult residents of Framingham, Massachusetts, signed up. That group of volunteers made endless visits to the research center, gave countless specimens of blood and body fluids, underwent repeated clinical exams, and passed on the baton to their children and grandchildren. The Framingham Heart Study now follows the descendants of the original volunteers! The study has given us great insight into risk factors for heart disease. Much of what we know today regarding the effects of high blood cholesterol, blood pressure, obesity, diabetes, and other factors on the development of heart disease can be traced to discoveries made in the Framingham Heart Study. Another example, the Nurses Health Study, was launched in 1976. Approximately 122,000 nurses volunteered to respond to questions about diseases and health-related topics, including smoking, hormone use, and menopausal status. The volunteers answered these questions repeatedly for the study over many, many years. The results have been published in multiple scientific journal articles and have led to a greater understanding of women’s health. The Physicians Health Study, started in 1982, provided information regarding the benefits of aspirin in prevention of cardiovascular disease. The study showed that low-dose aspirin decreased the risk of a first heart attack by 44%. More than 25,000 doctors volunteered for that study.

The power of volunteers is clear in several clinical trials in diabetes. The Diabetes Control and Complications Trial (DCCT), started in 1983, enrolled 1,441 patients. These volunteers have been attending research clinics for nearly 30 years! After the initial 10 years, the name of the study was changed to EDIC (Epidemiology of Diabetes Interventions and Complications) and the original volunteers were asked to stay in the study. I recently saw one man in his early 40s at a follow-up visit at our University of Tennessee site of the DCCT. He joined the study at age 15, and he has been coming ever since! DCCT volunteers had to follow a strict regimen of frequent home blood sugar (glucose) testing, multiple daily insulin injections, and frequent contacts with the research teams. Because of the volunteers, researchers in the DCCT study made the important discovery that good blood sugar control results in A1C levels of 7% or lower, and helps prevent the development of diabetic complications in the eyes, kidneys, and nerves. The continued participation by the volunteers has enabled the study to make additional discoveries on the impact of blood sugar control on heart disease, bladder function, and other conditions.

Together, diabetes and pre-diabetes affect about 80 million Americans. Of these, 54 million persons have pre-diabetes, a condition in which the blood sugar is abnormal but not severe enough to qualify as diabetes. Today, diabetes is treated with a wide variety of medications, all of which were discovered through research involving volunteers! Used properly with healthy eating and an active lifestyle, these medications help control high blood sugar and prevent tissue damage that can lead to diabetic complications (such as blindness and kidney failure). However, researchers have decided that it is smarter to prevent diabetes from occurring in the first place. The critical discovery on how best to prevent type 2 diabetes owes its very existence to more than 3,000 study volunteers for the Diabetes Prevention Program (DPP). The DPP study, sponsored by the National Institutes of Health, discovered that people with pre-diabetes develop diabetes at a rate of about 10% per year. However, with modest changes in lifestyle (walking 30 minutes, five times weekly, and decreasing calories), the rate of progression from pre-diabetes to diabetes was cut nearly 60%.

The DPP inspired our ongoing Pathobiology of Prediabetes in A Bi-racial Cohort (POP-ABC) Study at the University of Tennessee Health Science Center. In this study, we’re focusing on the early stages before even pre-diabetes develops. This way we can better understand the triggers that create abnormal blood glucose levels. Nearly 400 African American and white volunteers have joined the POP-ABC study. Knowing the triggers would enable future development of better targeted interventions. This will allow us to prevent early abnormal blood sugar levels. The cure for this epidemic of our time—diabetes—can only come from well-directed research to find the earliest root causes of the problem. Clearly, taking new scientific ideas and making them clinically relevant breakthroughs cannot occur without volunteers for medical studies. Without research volunteers, the best ideas and projects would be “dead in the water.” The volunteers in the DPP who helped us make the important discoveries and other research volunteers in other projects represent the brightest hope of humanity. They share their time generously with society. They follow study protocols, and in the process generate results. Although volunteers tend to join studies because they are concerned about their own health, they help further research to help others in the future. In that regard, our research volunteers are indeed worthy ambassadors of science. In the effort to find an answer for the diabetes epidemic, we salute the contributions and sacrifice of all those who have served as VOLUNTEERS for research projects.

Dr. Samuel Dagogo-Jack takes care of patients with diabetes and endocrine/metabolic disorders at the University of Tennessee Health Science Center, Memphis, Tennessee. He is also Professor of Medicine and Chief of the Division of Endocrinology at UTHSC, and Director of the Clinical Research Center. Dr. Dagogo-Jack’s research focuses on the prediction and prevention of diabetes and prediabetes. He is an Editor for the Journal of Clinical Endocrinology and Metabolism and Associate Editor for Diabetes Care. Dr. Dagogo-Jack has published 16 book chapters and more than 200 scientific papers and abstracts.