Ghrelin may help reduce obsesity in children with genetic disorder

What if your child’s overeating was so uncontrollable that it led to eating out of the garbage, ransacking the kitchen and getting arrested for stealing food?

These scenarios are a daily reality for parents of children with Prader-Willi syndrome (PWS) – a complex and currently untreatable genetic disorder characterized by compulsive eating, food foraging, massive weight gain and delayed mental development.

In the first study of its kind, University of British Columbia clinical professor of pediatrics Dr. Jean-Pierre Chanoine will test the effectiveness of a long-acting form of octreotide, a drug similar to the hormone somatostatin, in reducing patients’ insatiable appetites. The drug is known to counteract ghrelin – a recently discovered hormone that stimulates appetite. In patients with PWS, ghrelin levels are three to five times that of normal eaters.

“This illness is a nightmare for families,” says Chanoine, who is head of the endocrinology and diabetes unit at BC Children’s Hospital (Children’s) and a member of the BC Research Institute for Children’s & Women’s Health. “It means 24-hour supervision to keep the child away from food and constant guidance to regulate behaviour. Most patients face a bleak prognosis of massive obesity and life-threatening related illnesses.”

In a year-long trial that starts this month at Children’s, Chanoine will enroll 10 subjects aged 10-17 years who have PWS. They will be given regular doses of octreotide or a placebo to determine if the drug can decrease ghrelin levels and reduce appetite.

A condition that affects an estimated 1 in approximately 15,000 children worldwide, PWS is caused by an abnormality in a chromosome that is normally contributed by the father. The syndrome affects males and females equally and is known in all ethnicities. More than one-third of patients with PWS weigh more than 200 per cent of their ideal body weight. Many people with PWS experience complications of obesity such as diabetes, respiratory problems and heart failure.

Genetic screening to confirm a diagnosis of PWS is available.