Study to Assess The Role of Leptin in Treating Type 1 Diabetes

To determine whether adding the hormone leptin to standard insulin therapy might help rein in the tumultuous blood-sugar levels of people with type 1 (insulin-dependent) diabetes, a clinical trial at UT Southwestern Medical Center is being carried out. This is the first type 1 diabetes treatment trial involving leptin, which is naturally produced by fat cells and involved in body-weight regulation. For this study, UT Southwestern researchers will be using metreleptin, a slightly modified form of the hormone that has been well-tolerated in other clinical trials.

"Leptin has been very effective in improving diabetes in patients with lipodystrophies who have extreme lack of body fat, and recently leptin therapy has helped improve blood sugar control in animal models of type 1 diabetes," said Dr. Abhimanyu Garg, professor of internal medicine and principal investigator of the trial.

"Although we have no assurances that this will work in humans, we hope that the addition of leptin will be beneficial to patients with type 1 diabetes."

The phase 1 study also is designed to evaluate the safety and tolerability of adding leptin to a diabetes treatment regimen.

In type 1 diabetes, formerly known as juvenile-onset diabetes, the pancreatic beta cells that produce insulin are destroyed by an autoimmune process. Type 1 diabetics must regiment their diets and take insulin multiple times a day to control blood-sugar levels and prevent diabetic coma. The autoimmune disease, for which there is no cure, affects about 1 million people in the U.S.

More recently, the laboratory of Dr. Roger Unger, professor of internal medicine at UT Southwestern, using mouse models of type 1 diabetes, found that administering leptin instead of insulin resulted in better management of blood-sugar variability and lipogenesis, the conversion of simple sugars into fatty acids.

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For the clinical study, 12 to 15 participants will add leptin twice a day to their standard insulin therapy over a five-month period. The trial will last a total of seven months and will include 11 visits - an initial screening, four inpatient visits and six outpatient evaluations - to UT Southwestern. The first inpatient visit will last a minimum of four days; the others will take two days each.

To be eligible for the initial screening, prospective trial participants must be between 18 and 50 years of age, have a body mass index (BMI) less than 25, and have been diagnosed with type 1 diabetes. BMI is a weight-to-height ratio commonly used in doctors' offices to gauge obesity. A normal BMI is between 18.5 and 25.

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Source-ANI