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Leptin therapy gains FDA approval

Nature Biotechnology volume 32, pages 300–301 (2014)Cite this article

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For all the promise once heaped on this hormone, the approval is for a dramatically circumscribed population. Generalized lipodystrophy is a rare disorder characterized by absence of adipose tissue. With no adipose tissue to secrete the appetite-suppressant leptin, individuals with lipodystrophy eat voraciously. The consequences are catastrophic, says Stephen O'Rahilly, director of the University of Cambridge Metabolic Research Laboratories. Excess calories get stored as fat in liver and muscle cells leading to diabetes, high blood lipid levels and pancreatitis. “It's a totally appalling double whammy of being constantly hungry but of food being your greatest enemy,” says O'Rahilly. The disease manifests either as generalized or partial lipodystrophy. Both forms can be inherited or induced by medications or result from autoimmune disease or unknown causes.

The generalized form of lipodystrophy is extremely rare. There are fewer than 200 people in the US and 1,000 at most with partial lipodystrophy of varying severity, says Abhimanyu Garg, chief of the division of nutrition and metabolic diseases at the University of Texas (UT), Southwestern Medical Center. Currently there is no treatment other than managing complications. Myalept treatment yields striking results in people with generalized lipodystrophy. It markedly reduces food intake, improves blood glucose and triglyceride levels, in some cases normalizing levels of both.

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    Gunjan Sinha

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Sinha, G. Leptin therapy gains FDA approval. Nat Biotechnol 32, 300–301 (2014). https://doi.org/10.1038/nbt0414-300b

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