Prader-Willi syndrome is complex and presents a unique set of challenges reflecting a false state of starvation. This includes anxiety, compulsivity, life-threatening hyperphagia, mild to moderate levels of intellectual disability, growth hormone deficiency, and a high risk of obesity beginning in childhood.
The Science Behind the Treatment of Prader-Willi Syndrome
Chromosome 15 Abnormalities
Abnormalities in a small section of chromosome 15 lead to dysfunction in a part of the brain called the hypothalamus. This area of the brain helps regulate if someone is hungry and whether they are satisfied after eating.
Research has shown that oxytocin may play a key role in Prader-Willi syndrome. Oxytocin is a neurotransmitter produced in the hypothalamus and released into the blood via the pituitary gland. Oxytocin regulates both social-emotional and feeding behaviors – enhancing the feeling of satiety. Those affected by Prader-Willi syndrome have fewer oxytocin producing neurons in a part of the hypothalamus known as the paraventricular nucleus.
Data suggests that our investigational therapy, LV-101 (intranasal carbetocin) may help to replace some of the oxytocin that is not present in those with Prader-Willi syndrome. LV-101 is unique because it is highly selective for the oxytocin receptor. This improved selectivity is believed to reduce the “off-target” vasopressin effects seen when oxytocin is administered on its own.