02-28-2014, 01:42 AM
The US Food and Drug Administration (FDA) has just approved the drug metreleptin as a therapy for the rare metabolic disease lipodystrophy. Patients with lipodystrophy are deficient in the hormone leptin; meterleptin is a drug form of this hormone. Lipodystrophy is extremely rare, having been reported in 300-500 people internationally. It can be inherited or acquired and is characterised by almost complete absence of fat tissue either from birth or developing during childhood. Despite their extremely lean and muscular body shape, patients paradoxically suffer from diseases such as severe diabetes and high blood pressure due to elevations of triglyceride levels. This leaves them extremely susceptible to cardiovascular disease and stroke.
Standard treatment for lipodystrophy has up until now focused on high-dose insulin to tackle diabetes plus triglyceride- or lipid-lowering medications. The newly approved drug, metreleptin, works by curbing appetite and normalising metabolism. Dr. Abhimanyu Garg, Chief of the Division of Nutrition and Metabolic Diseases at University of Texas (UT) Southwestern initiated the first metreleptin trial in collaboration with the National Institutes of Health (NIH). He says: “Many lipodystrophy patients have benefited from leptin therapy. While it is not a cure, leptin does help manage complications that can include diabetes, high blood lipids, and accumulation of fat in the liver.” Dr Garg’s research first led to identification of mutations in the gene that lipodystrophy.
However, the potential therapeutic potential of leptin was discovered almost by accident due to serendipitous creation of a leptin-deficient mouse model of generalized lipodystrophy by Nobel Laureates Dr. Michael Brown and Dr. Joseph Goldstein while researching cholesterol metabolism. When these mice were injected with leptin, it was discovered that their appetite was suppressed and that their insulin resistance, diabetes, and fatty liver improved. This led to the suggestion that Dr Garg try leptin as a therapeutic in lipodystrophy in humans. The first clinical trial was carried out by UT Southwestern in collaboration with the NIH’s National Institute of Diabetes and Digestive and Kidney Diseases and the pharmaceutical company Amgen. In 2002, the researchers reported that leptin therapy indeed controlled insulin resistance and triglyceride levels in lipodystrophy patients as well as decreasing build-up of fat in the liver. When used in human lipodystrophy patients, an appropriate dose can remove the need for insulin or at least for high-dose insulin treatment.
Dr. Garg, in collaboration with the NIH and Amgen, holds both U.S. and European patents in collaboration for use of leptin for lipodystrophy treatment. Bristpl-Myers Squibb acquired rights to the leptin molecular franchise in 2006 via their subsidiary, Amylin Pharmaceuticals. Dr Gard is currently a consultant for Bristol-Myers Squibb, who make metreleptin in partnership with AstraZeneca.
Source:
UT Southwestern Medical Centre press release; available at http://www.eurekalert.org/pub_releases/2...022514.php
Standard treatment for lipodystrophy has up until now focused on high-dose insulin to tackle diabetes plus triglyceride- or lipid-lowering medications. The newly approved drug, metreleptin, works by curbing appetite and normalising metabolism. Dr. Abhimanyu Garg, Chief of the Division of Nutrition and Metabolic Diseases at University of Texas (UT) Southwestern initiated the first metreleptin trial in collaboration with the National Institutes of Health (NIH). He says: “Many lipodystrophy patients have benefited from leptin therapy. While it is not a cure, leptin does help manage complications that can include diabetes, high blood lipids, and accumulation of fat in the liver.” Dr Garg’s research first led to identification of mutations in the gene that lipodystrophy.
However, the potential therapeutic potential of leptin was discovered almost by accident due to serendipitous creation of a leptin-deficient mouse model of generalized lipodystrophy by Nobel Laureates Dr. Michael Brown and Dr. Joseph Goldstein while researching cholesterol metabolism. When these mice were injected with leptin, it was discovered that their appetite was suppressed and that their insulin resistance, diabetes, and fatty liver improved. This led to the suggestion that Dr Garg try leptin as a therapeutic in lipodystrophy in humans. The first clinical trial was carried out by UT Southwestern in collaboration with the NIH’s National Institute of Diabetes and Digestive and Kidney Diseases and the pharmaceutical company Amgen. In 2002, the researchers reported that leptin therapy indeed controlled insulin resistance and triglyceride levels in lipodystrophy patients as well as decreasing build-up of fat in the liver. When used in human lipodystrophy patients, an appropriate dose can remove the need for insulin or at least for high-dose insulin treatment.
Dr. Garg, in collaboration with the NIH and Amgen, holds both U.S. and European patents in collaboration for use of leptin for lipodystrophy treatment. Bristpl-Myers Squibb acquired rights to the leptin molecular franchise in 2006 via their subsidiary, Amylin Pharmaceuticals. Dr Gard is currently a consultant for Bristol-Myers Squibb, who make metreleptin in partnership with AstraZeneca.
Source:
UT Southwestern Medical Centre press release; available at http://www.eurekalert.org/pub_releases/2...022514.php
![[+]](https://www.biotechnologyforums.com/images/netpen-pro/collapse_collapsed.png)