Children's Hospital to participate in drug study to evaluate effects of metformin for treating type 2 diabetes

Children's Hospital Los Angeles will participate in a nationwide drug study evaluating the effects of metformin, a medication that has been shown to be safe and effective for treating type 2 diabetes. T1D Exchange, a nonprofit program dedicated to improving the lives of those living with type 1 diabetes by facilitating better care and accelerating research, was recently awarded a $2.8 million grant to conduct this study.

Normally diagnosed in children and young adults, type 1 diabetes is a chronic disease characterized by insufficient insulin production by the pancreas. Insulin is needed to allow glucose into the body's cells, where it can be converted into energy. Without insulin, glucose becomes trapped in the bloodstream and can cause complications such as cardiovascular disease, nerve damage, kidney disease and decreased vision. While there is no cure, type 1 diabetes can be managed through life-long insulin therapy and continuous monitoring of blood glucose levels.

Unlike type 2 diabetes, the development of type 1 diabetes is not influenced by weight or other environmental factors. However, "there is increasing evidence that individuals with type 1 diabetes have not escaped the general population trend toward obesity, despite the traditional phenotype of type 1 diabetics being of normal or underweight," says Jamie Wood, MD, physician in the Center for Endocrinology, Diabetes and Metabolism at Children's Hospital Los Angeles. "Overweight children with type 1 diabetes have trouble controlling their blood glucose levels because their bodies become resistant to insulin."

To combat this emerging issue, T1D Exchange's nationwide network of experienced type 1 diabetes researchers, patients and healthcare professionals, will conduct their first multi-site study to observe the effects of metformin. This drug has been used for the past 50 years by individuals with type 2 diabetes, but its safety and efficacy is largely unknown for treating type 1 diabetes. Metformin, which acts to reduce excessive glucose production, will be tested in combination with standard insulin therapy in overweight individuals with type 1 diabetes. Over a period of 6 months, the patients, aged 12 to 19 years, will be monitored for blood glucose control and insulin sensitivity with the goal of developing a successful treatment for this growing subset of patients.

"This study is our first opportunity in some time to test a new treatment for type 1 diabetes and accelerate the progress of therapies that will increase our patients' quality of life," says Mitchell Geffner, MD, division chief of the Center for Endocrinology, Diabetes and Metabolism. "We look forward to treating several study patients at Children's Hospital Los Angeles and seeing first-hand what this medication might do for the thousands of other patients with type 1 diabetes we see each year."

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