Prader-Willi Syndrome (PWS) is a condition characterized by growth hormone deficiency, hypogonadism, various behavioral disturbances, an insatiable hunger drive (hyperphagia) and excessive eating leading to life-threatening obesity. The specific causes of the disturbed eating behavior in persons with PWS remain unknown. More importantly, effective therapies for control of their excess appetite drive and management of their obesity are still lacking. One plausible explanation that has been put forward for their overeating is the massively high level of a circulating stomach-derived hormone called ghrelin. Recently, an approved medication licensed for the treatment of patients with diabetes called exenatide, has been shown to cause a reduction in appetite and food intake associated with significant and progressive weight loss. The active substance of this drug has also been shown in animal experimental studies to cause a significant reduction in the levels of the hormone ghrelin. We therefore postulate that exenatide may exert similar appetite-suppressing and weight-losing effects in patients with PWS. In order to explore this hypothesis further we propose to undertake a pilot clinical trial of exenatide in patients with PWS. Our proposed study will help further the goals of the Foundation for Prader-Willi Research by potentially opening up new avenues in the pharmacological treatment of this disabling condition.
*Project to be initiated when Human Subject review is complete and approved.
Christina Daousi, MD
University Hosptial Aintree