Success of Clinical Research Depends on the Altruism of Many Volunteers

Evidence suggests that having a close relative with type 1 diabetes puts individuals at greater risk of developing the disease, but one Yale researcher is investigating whether it's possible to prevent a genetic disposition from turning into a full-blown case of the disease.

Evidence suggests that having a close relative with type 1 diabetes puts individuals at greater risk of developing the disease, but one Yale researcher is investigating whether it’s possible to prevent a genetic disposition from turning into a full-blown case of the disease.

Dr. Kevan Herold, professor of immunobiology and internal medicine (endocrinology) at the School of Medicine, is participating in government-funded clinical research to study family histories and characteristics of those at risk for developing type 1 diabetes to determine whether early identification can help prevent the disease or limit its impact.

“The rate of diabetes in first-degree relatives can be as high as 90%,” says Herold, who is also involved in another study to determine whether oral insulin can delay or prevent the onset of the disease in relatives. “The disease starts years before it clinically presents.”

Just recently, Herold was approved by the Yale University Human Investigations Committee for yet another trial, to test whether a few injections with a protein (GAD-alum) can help people newly diagnosed with type 1 diabetes continue making some of their own insulin. Studies have shown that people who continue to make insulin have less trouble with low blood sugar and fewer complications from their diabetes than those who no longer make their own insulin.

Clinical trials, researchers say, are a necessary, critical step in the process of making sure that potential new treatments are safe and effective.

At Yale, there are hundreds of clinical trials underway at any given time, covering a wide variety of diseases and conditions. Guiding this critical element of the research process is the Yale Center for Clinical Investigation (YCCI), which was launched in early 2006 to help shepherd disease-related discoveries from the laboratory to patients in need. YCCI provides administrative, regulatory and grant-writing support for Yale’s scientific community.

That same year, the Yale School of Medicine won the largest grant in its history: a five-year, $57.3 million award from the National Institutes of Health to speed up the process of getting medical discoveries into the community. The grant is about to come up for renewal. Tesheia Johnson, chief operations officer of the YCCI, is hopeful that the federal government will continue its funding.

“Without it, some of our groundbreaking research might grind to a halt,” she says. “That would be a tragedy for people in need.”

Without clinical trials, Yale might never have successfully developed the insulin pump to treat diabetes, medications to treat HIV infection or the first therapy to create a cancer-fighting immune response in the body’s blood cells. All these discoveries involved years of research, and all depended upon volunteers who were willing to participate in clinical testing.

“Volunteers are the linchpin of the process,” says Johnson, noting that people choose to participate in clinical trials for various reasons: to explore new options that may provide personal therapeutic benefit, to help identify potential treatments for the market or simply to contribute to the advancement of medical knowledge.

Most clinical trials also need healthy people to volunteer as a “control group,” Johnson says. “They provide us with information we can use to compare outcomes with people who have a particular disease.”

Nevertheless, there are challenges to getting people to volunteer for clinical trials, according to Johnson. Some are reluctant to participate because of the time commitment, as volunteers must answer questionnaires and may need to make frequent visits to the study site. Others may be reluctant to participate due to the possibility of side effects. Such side effects are explained to the potential volunteer, as well as who is responsible for payment of such an injury, should one occur, during the informed consent process. The consent process must take place prior to the potential volunteer formally deciding whether or not to take part in the trial.

Johnson says that many people weigh these considerations and agree to participate because of the chance that they will help themselves or others.

“Even if they or a loved one never suffers from the particular disease, they understand that they are helping society at large,” Johnson says.

In Herold’s diabetes trial, researchers know that relatives of patients with type 1 diabetes have about a 3% to 4% chance of testing positive for the antibodies associated with the disease. Those antibodies are an early indication that damage to insulin-secreting cells in the pancreas may have already begun. Herold hopes his study will identify adults and children who have the antibodies in their blood so they can be monitored and treated in the earliest stages.

There are other experimental treatments that Herold is testing for patients who have already been discovered to have diabetes. For this and other studies, he needs people to volunteer.

“There is something to be gained in knowing you helped people avoid sickness, or recover faster,” says Johnson of the benefits for clinical trial participants. “You can’t put a price tag on that.”

To learn more about clinical trials at Yale, visit the YCCI website at www.yaletrials.org, which describes the various trials currently recruiting volunteers.

— By Helen Dodson

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Helen Dodson: helen.dodson@yale.edu, 203-436-3984