MedicalResearch.com: What is the background for this study? What are the main findings?
Response: We are focusing our interest on rare monogenic forms of obesity.
The hormones leptin and MSH are playing a pivotal role for the regulation of satiety and body weight. Mutations within this pathway, e.g. in the leptin receptor gene, are leading to severe hyperphagia and early onset obesity. Although tremendous effort it is extremely difficult for the affected patients to stabilize their body weight for a longer period of time. For this reason it has been analyzed within this investigated initiated trial whether patients with a leptin receptor mutation benefit from a treatment with the MC4R agonist setmelanotide. The treatment led to a reduction of the initially increased hunger feeling and to a reduction of body weight. Additionally, we identified molecular evidences that a specific signaling cascade of the MSH receptor (MC4R) is of importance for the regulation of body weight.
MedicalResearch.com: What should readers take away from your report?
Response: Monogenic forms of obesity are rare. Severe hyperphagia due to missing satiety signals within in a specific pathway are leading to severe hyperphagia and early onset obesity. Now there is evidence that at least patients with a lack of the satiety signal MSH (POMC gene mutation) and patients with an impaired function of the leptin receptor might benefit from a pharmacological treatment option with the MC4R agonist.
MedicalResearch.com: What recommendations do you have for future research as a result of this work?
Response: Additional studies are needed to gain further information about the efficacy/ safety of this MC4R agonist in patients with an impaired function of the so-called leptin-melanocortin signaling cascade. Furthermore it will be analyzed to which extend the different signaling capacities of the MC4 receptor are of importance for the regulation of body weight.
Citations: Karine Clément, Heike Biebermann, I. Sadaf Farooqi, Lex Van der Ploeg, Barbara Wolters, Christine Poitou, Lia Puder, Fred Fiedorek, Keith Gottesdiener, Gunnar Kleinau, Nicolas Heyder, Patrick Scheerer, Ulrike Blume-Peytavi, Irina Jahnke, Shubh Sharma, Jacek Mokrosinski, Susanna Wiegand, Anne Müller, Katja Weiß, Knut Mai, Joachim Spranger, Annette Grüters, Oliver Blankenstein, Heiko Krude, Peter Kühnen. MC4R agonism promotes durable weight loss in patients with leptin receptor deficiency. Nature Medicine, 2018; DOI: 10.1038/s41591-018-0015-9
The information on MedicalResearch.com is provided for educational purposes only, and is in no way intended to diagnose, cure, or treat any medical or other condition. Always seek the advice of your physician or other qualified health and ask your doctor any questions you may have regarding a medical condition. In addition to all other limitations and disclaimers in this agreement, service provider and its third party providers disclaim any liability or loss in connection with the content provided on this website.