Teresa Quattrin and a TrialNet family in Western New York discuss the benefits of the study.
Published May 2, 2013
More than 1,000 Western New York children who are at risk of developing Type 1 diabetes are helping scientists at UB and Women and Children’s Hospital of Buffalo (WCHOB) find ways to prevent, delay or reverse the disease.
They are participants in TrialNet, an international network of researchers funded by the National Institutes of Health and dedicated to finding ways to prevent, delay and reverse the progression of Type 1 diabetes. The children are at high risk because they have a close relative, often a sibling, with the disease.
“Out of the more than 112,000 participants worldwide, since 2005, we have enrolled 1,425 in our main TrialNet study ‘Pathway to Prevention,’” explains Teresa Quattrin, UB Distinguished Professor and A. Conger Goodyear Professor and chair, Department of Pediatrics, School of Medicine and Biomedical Sciences, and pediatrician-in-chief and chief of the Division of Diabetes/Endocrinology at WCHOB.
Quattrin is principal investigator on the grant at the UB and WCHOB center, one of 200 TrialNet screening sites worldwide; it is affiliated with Columbia University, one of TrialNet’s 18 clinical centers.
The increased incidence of Type 2 diabetes—caused largely by obesity—has been well-documented.
But rates of Type 1 diabetes, an autoimmune disease in which the body destroys its ability to make insulin and requires insulin injections for survival, also are increasing by 3 to 5 percent annually. The reasons are as yet unknown, but TrialNet is giving families at risk new options and new hope.
According to Quattrin, previous research in Buffalo and elsewhere has revealed that it can take years for Type 1 diabetes to progress to the point where the patient exhibits symptoms, such as thirst, extreme hunger, frequent urination, weight loss and blurred vision. A patient may be completely healthy for years, but may exhibit biomarkers in the blood and genetic characteristics that reveal that within five or 10 years, it is likely they will experience symptoms and develop Type 1 diabetes.
“In individuals who are going to become Type 1 diabetics, their ability to make insulin declines very slowly over a period of years, up to the point where there is only 10-15 percent insulin left and that’s when symptoms appear,” Quattrin says. “TrialNet focuses on that window that precedes the onset of diabetes.”
TrialNet builds upon earlier studies that identified autoimmune biomarkers, known as autoantibodies, and other factors that describe exactly how an individual who is at risk evolves over time into a Type 1 diabetic.
“These sophisticated studies were able to determine the factors, such as an individual’s genetic makeup, as well as autoimmune conditions, that influence why someone develops Type 1 diabetes faster than someone else,” Quattrin says. “These studies have provided a kind of road map that now allow us to predict with extreme precision when and if an individual will come down with Type 1 diabetes.”
Sisters, brothers, parents under age 45 and children of Type 1 diabetics are eligible to enroll. More distant relatives also may be able to enroll. Through blood tests and glucose tolerance tests, TrialNet offers a way for families to know who is at high risk for developing diabetes. Some children at risk with specific biomarkers can be enrolled in a study in which they take oral insulin that may be able to delay the onset of the disease.
That’s what the Lockwood family of Colden, N.Y., hopes for 9-year-old Jillian Lockwood. She got involved in TrialNet when her brother, 12-year-old Dawson, was diagnosed with Type 1 diabetes at WCHOB in 2009. Not long after the diagnosis, Jillian and her older brother, Connor, were tested for biomarkers as part of TrialNet; Jillian tested positive.
Jillian is taking either oral insulin or a placebo; if she is taking the insulin, it is possible, Quattrin explains, that it will help her body preserve more insulin, staving off diabetes for years.
“Preserving insulin for more years, and therefore keeping blood sugars at a normal level, will greatly reduce the chance of complications that arise from poorly controlled blood sugars,” says Quattrin.
Children, like Connor Lockwood, who test negative, are screened annually so that if they do develop biomarkers that show they eventually will develop diabetes, they can start insulin early and not experience the severe symptoms and hospitalization that newly diagnosed diabetics undergo.
“With Connor in TrialNet, the benefit is early diagnosis,” says Mrs. Lockwood. “We won’t have to worry, is he becoming diabetic? We will know if he is developing the autoantibodies. And if the oral insulin means that could help Jillian not become diabetic until she’s 20 or 25, then we’ll take it,” she says. “And if it finds a cure and helps somebody else, then it’s tenfold the reason for doing this.”
Family members of Type 1 diabetics—either siblings, parents, children, cousins, grandchildren, nieces or nephews—who are interested in participating in TrialNet in Buffalo should contact Amanda House, project coordinator for TrialNet, at WCHOB at 878-7268.More than 1,000 Western New York children who are at risk of developing Type 1 diabetes are helping scientists at UB and Women and Children’s Hospital of Buffalo (WCHOB) find ways to prevent, delay or reverse the disease.
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