Buffalo’s TrialNet Type 1 Diabetes Study Aims for Prevention

Children in Western New York and the Southern Tier are among more than 112,000 people around the globe participating in studies conducted by TrialNet, an international network funded primarily by the National Institutes of Health.

Since 2005, 1,425 children at high risk for the disease have been enrolled in Pathway to Prevention, the main TrialNet study conducted by the University at Buffalo and the Women and Children’s Hospital of Buffalo (WCHOB), says Quattrin, UB Distinguished Professor, A. Conger Goodyear Professor and chair of pediatrics.

Quattrin, who also is pediatrician-in-chief and chief of diabetes/endocrinology at WCHOB, oversees the local screening site, one of 200 around the world. The Buffalo-area site is affiliated with Columbia University, one of TrialNet’s 18 clinical centers.

The ongoing Pathway to Prevention study seeks ways to ward off the disease, in part by catching early warning signs.

This is significant because rates of Type 1 diabetes—an autoimmune disease in which the body destroys its ability to make insulin—are increasing by three to five percent annually.

Although the well-documented increase in Type 2 diabetes is attributed largely to obesity, reasons for the Type 1 increase are unknown.

Prior research in Buffalo and elsewhere has shown it can take years for the disease to progress to the point where patients experience symptoms such as thirst, extreme hunger, frequent urination, weight loss and blurred vision, says Quattrin, “Symptoms appear when there is only 10 to 15 percent of insulin left.”

A full five to 10 years before symptoms develop, however, patients may exhibit disease-heralding autoimmune biomarkers in the blood and certain genetic characteristics.

Pathway to Prevention builds upon earlier studies that identified disease predictors describing exactly how an individual at risk evolves 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 allows us to predict with extreme precision when and if an individual will come down with Type 1 diabetes.”

Through blood tests and glucose tolerance tests, Pathway to Prevention offers a way for families to know who is at high risk for developing diabetes.

Some children with specific biomarkers can enroll in a related TrialNet study testing the ability of oral insulin to delay the onset of the disease.

After Dawson Lockwood, 12, of Colden, N.Y., was diagnosed with Type 1 diabetes, his two siblings were tested through the Buffalo-area Pathway to Prevention study.

Jillian, 9, tested positive for biomarkers and was enrolled in the oral insulin study.

If she is among the participants actually taking insulin as opposed to a placebo “it is possible that it will help her body preserve more insulin, staving off diabetes for years,” says Quattrin.

“Preserving insulin, and therefore keeping blood sugars at a normal level, will greatly reduce the chance of complications that arise from poorly controlled blood sugars,” she says.

With treatment, Jillian could delay the disease until adulthood. “If the oral insulin helps Jillian not become diabetic until she’s 20 or 25, then we’ll take it,” says her mom, Kristine Lockwood.

Children who test negative—like Dawson’s brother, Connor—benefit from annual screenings.

“With Connor in Pathway to Prevention, the benefit is early diagnosis,” says Kristine Lockwood. “We won’t have to worry, ‘is he becoming diabetic?’ We will know if he is developing the autoantibodies.”

If participants develop biomarkers that show they will eventually develop diabetes, they can start insulin early and avoid the severe symptoms and hospitalization that newly diagnosed diabetics often experience.

The Lockwoods also are willing to contribute to the greater good of medical research. As Kristine Lockwood says, “If it finds a cure and helps somebody else, then it’s tenfold the reason for doing this.”

Children participating in Pathway to Prevention are at high risk for Type 1 diabetes, because they have a close relative with the disease.

Sisters, brothers, parents under age 45 and children of Type 1 diabetics are eligible to enroll in the study. More distant relatives, such as cousins, grandchildren, nieces or nephews, also may be able to participate.