Hormone Overcomes Genetic Cause of Morbid Obesity

MedicalResearch.com Interview with:
Dr. Peter Kühnen MD
Institute for Experimental Pediatric Endocrinology
Charité–Universitätsmedizin Berlin
Berlin, Germany

MedicalResearch.com: What is the background for this study? What are the main findings?

Dr. Kühnen: The patients, which were included in this study, are suffering from a genetic defect in a gene called POMC. This gene is cleaved into different hormones as e.g. MSH (melanocyte stimulating hormone). MSH is very important for the regulation of satiety by activation of the MC-4 receptor. For this reason these patients are persistent hyperphagic due to the lack of MSH and they gain weight very fast in the first months of their life.
Setmelanotide activates the MC-4 receptor, which is important for the activation of satiety. By restoring the lost function Setmelanotide leads to a reduction of hyperphagia and to a reduction of body weight in this POMC deficient patients.

MedicalResearch.com: What should readers take away from your report?

Dr. Kühnen: This study used a targeted approach to treat obesity in patients with a specific genetic defect in the POMC gene. By restoring the lost function in the pathway, hyperphagia can be reduced and this leads to severe weight loss, which of importance for the patients.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Dr. Kühnen: We targeted people with a specific genetic defect that causes extreme obesity with a drug that restores lost function, which leads to severe improvement of the body weight. In future studies we will concentrate our efforts to analyze the potential to treat other genetic defects within this pathway that are rare orphan disorders and cause life threatening obesity.

MedicalResearch.com: Is there anything else you would like to add?

Dr. Kühnen: This study provides great insights into how genetics behind obesity works. This MC-4 receptor agonist activates the pathway below acting as replacement therapy for POMC deficient patients and leading to transformational changes of weight and appetite.


Proopiomelanocortin Deficiency Treated with a Melanocortin-4 Receptor Agonist
Peter Kühnen, M.D., Karine Clément, M.D., Ph.D., Susanna Wiegand, M.D., Oliver Blankenstein, M.D., Keith Gottesdiener, M.D., Lea L. Martini, M.D., Knut Mai, M.D., Ulrike Blume-Peytavi, M.D., Annette Grüters, M.D., and Heiko Krude, M.D.
N Engl J Med 2016; 375:240-246July 21, 2016DOI: 10.1056/NEJMoa1512693

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Last Updated on July 21, 2016 by Marie Benz MD FAAD