Research Finds that Patients With Rare Disease Respond to Leptin Therapy

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Researchers at Michigan Medicine have found the livers of patients with a rare disease that affects metabolism have responded positively to leptin therapy.
The research team predicted the response of 23 patients with fatty liver to metreleptin, a man-made version of the naturally occurring hormone leptin, which regulates fat and glucose metabolism. They reported that patients with a lower baseline leptin level had a higher response rate after one year of treatment with metreleptin. They presented their findings at ENDO 2017, the annual meeting of the Endocrine Society.
Lipodystrophy is a group of rare diseases that share in common the selective loss of fat tissue from the body. Patients affected by the diseases generally have severe insulin resistance, high lipids in their blood, and fatty liver. The condition highlights how important fat cells are to regulating a person’s metabolism.
Elif Oral, MD, is associate professor of endocrinology at Michigan Medicine and principal investigator of the study, which was supported by MCRU services.
Read the full Michigan Medicine article here.