Oxytocin vs. placebo for the treatment of hyperphagia in Prader-Willi syndrome

Prader-Willi Syndrome (PWS) is a neurodevelopmental disorder caused by lack of inherited genes from fathers on chromosome 15. PWS is characterized by intellectual disabilities, repetitive and compulsive behaviors, social cognition deficits, increased eating and obesity. These individuals typically consume up to three times the normal caloric intake and hoard food, due to decreased satiation rather than increased hunger. Oxytocin (OXT) is a hormone proposed to contribute to the development of behaviors observed in ASD, including repetitive behaviors and social information processing. We have demonstrated improvement in these areas with OXT treatment in ASD individuals. As persons with PWS have comparable repetitive behaviors and social deficits, OXT has also been implicated in this syndrome. The number and size of OXT neurons in the brain is reduced in persons with PWS. Furthermore, mice with reduced OXT neurons overeat and are obese, and when given OXT, they decreased food intake and had loss of body weight. OXT treatment could prove beneficial to PWS individuals as overeating, repetitive behaviors and social cognition deficits are problems associated with this syndrome, and these people have decreased OXT neurons. We propose to conduct a treatment study of intranasal OXT vs. placebo in children and adolescents with PWS and symptoms of ASD. OXT has already been proven safe and effective in a treatment study of socialization and disruptive behavior in adults with PWS, and we hypothesize that OXT will be superior to placebo and have a positive effect on child and adolescent PWS eating and repetitive behaviors, and social cognition. If proven superior, this data will add to the current knowledge that OXT is a beneficial treatment with minimal side effects for PWS symptoms. Additional knowledge of OXT’s ability to reduce overeating could lead to improvement of patient’s quality of life and reduction in familial stress.

(Dr. Hollander's website).

This "Best Idea Grant" was funded by One Small Step funds, raised in partnership with the Prader-Willi Syndrome Association (USA), and with additional support from Kids Need Cures.