New Drug Aims to Treat Hyperphagia in Those with Prader-Willi Syndrome


Hyperphagia is an extreme hunger and obsession with food, and it is a symptom of Prader-Willi syndrome. While many of the other effects of this condition have treatments, this symptom does not, and it can lead to excessive weight gain and childhood obesity. Millendo Therapeutics aims to create a drug that will successfully treat the hyperphagia of Prader-Willi syndrome. Livoletide is currently being tested in children, after being shown to reduce hunger-related behavior in adults. Millendo hopes that this treatment will be widely available soon.

About Prader-Willi Syndrome

Prader-Willi syndrome is a rare, genetic disorder that is characterized by a constant sense of hunger (hyperphagia). This hunger begins at age two. Physical, mental, and behavioral problems are all common in those with this syndrome. People may experience intellectual impairments and learning disabilities.

An error in one or more of the genes on chromosome 15 causes this condition. These errors disrupt the function of the hypothalamus, which is the part of the brain that is responsible for releasing hormones. It is typically a sporadic mutation that leads to the errors on chromosome 15.

Symptoms of Prader-Willi syndrome will change as people age. Right after birth, symptoms include poor muscle tone, distinctive facial features like a narrow forehead, almond-shaped eyes, and a triangular mouth; poor sucking reflex, poor responsiveness, and underdeveloped genitals. As a child reaches early childhood symptoms may change to hyperphagia, poor growth and physical development, cognitive impairment, delayed motor impairment, speech and behavioral problems, short stature, small hands and feet, and sleep disorders.

After doctors notice the characteristic signs of this disorder, they will order a blood test to confirm a diagnosis. Early diagnosis is helpful. Treatment is symptomatic, and it includes good nutrition, human growth hormones, weight management, sex hormone treatment, behavioral and physical therapy, and sleep disorder treatment.

About Livoletide

Millendo Therapeutics is based in Ann Arbor, Michigan. They are developing livoletide for the treatment of hyperphagia. It was evaluated in the ZEPHYR study in March of 2019. This study was double-blind, placebo-controlled, and enrolled 47 adult participants. For two weeks participants took three or four milligrams of livoletide. A significant decrease in food-related behaviors was reported.

The next step of this study is to evaluate the effects of livoletide in children. Recruitment of children from the ages of four to seven is currently happening, with four children already enrolled. Along with testing effectiveness, researchers want to learn more about the brain’s role in controlling hunger.

Find the source article here.

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