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Treatment for Lipodystrophy Reveals New Workings of Leptin


The hormone leptin plays an essential role in energy balance. It reduces the amount of food one eats and increases the amount of energy one burns. Because of these effects, the hormone has been the center of much research in the area of obesity reduction. Today, scientists are looking more broadly at leptin’s role in the body.

To get a clearer understanding of the workings of this crucial hormone, researchers are studying conditions that result in very low levels of leptin. Lipodystrophy is one such condition.

Lipodystrophy results in the destruction or disappearance of fat cells from all or part of the body. People with lipodystrophy have a deficiency of leptin. They also experience high levels of triglycerides, insulin resistance, and fatty liver (since the normal storage location for body fat—fat cells—are absent in people with lipodystrophy, fat gets stored in the liver and a fatty, enlarged liver results).

Researchers at the Diabetes Branch of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and the University of Texas Southwestern Medical Center in Dallas treated nine lipodystrophy patients with leptin for 4 months to see if their conditions would improve.

After 4 months, all patients had lower triglyceride levels, improved control of blood sugar, and smaller livers. Patients also ate less food. All but one patient lost weight. Eight of nine patients had diabetes, and all eight were able to decrease or discontinue their diabetes therapy with leptin treatment.

Lowered triglyceride levels mean these patients now have a lower risk for heart disease and stroke. With improved blood sugar, they have a lower risk for diabetes-related conditions including blindness and kidney disease. With less fat in their livers, they have a lower risk for cirrhosis. All of the patients continue to receive leptin-replacement therapy because of their positive health changes.

Previous research brought to light the critical role of leptin in energy balance. This study, through improvements to lipodystrophy-related metabolic abnormalities, reveals novel roles for the hormone: regulation of triglyceride levels and of the body’s sensitivity to insulin levels. Researchers will continue to investigate all this multi-faceted hormone does in the body.

The full report appears in the February 21, 2002 New England Journal of Medicine and can be accessed at s


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