Alberta Diabetes Institute to Study Use of Anti-Obesity Drug in Alström and Bardet-Biedl Patients

According to a publication from EurekAlert, a clinical trial of setmelanotide, an experimental anti-obesity drug, is set to begin soon at the Alberta Diabetes Institute (of the University of Alberta). Setmelanotide, owned and developed by Rhythm Pharmaceuticals (Boston, Mass.), is believed to stimulate a biological pathway associated with obesity — especially childhood obesity.

About MC4R and Genetic Obesity

Melanocortin-4 receptors (MC4R) are proteins chiefly found in the brain that, until somewhat recently, had no known function. In recent decades, researchers have discovered that MC4R forms part of a complex biological chain that, when activated, helps maintain bodyweight by regulating appetite and increasing energy expenditure.

In 1998, genetic studies of humans found that mutations to the gene responsible for coding MC4R proteins, also called MC4R (in medical literature, the names of genes are written in italics — the names of proteins are not), were associated with higher rates of of obesity. Further research proved even more condemning — researchers now consider MC4R mutations to be the root cause of most monogenic forms of obesity. Over 150 distinct mutations have been identified in MC4R to date.

The Alberta Diabetes Institute study of setmelanotide will recruit individuals with Alström and Bardet-Biedl syndromes. Though these conditions are genetically rooted and frequently include obesity as a symptom, they have diverse body-wide affects including cardiomyopathy, vision, and hearing loss.

About Setmelanotide and the Canadian Study

Setmelanotide “is thought” to work by stimulating the MC4R pathway, helping regulate patients’ hunger. Since mutations to MC4R are associated with both obesity and dysfunction of the MC4R pathway, researchers believe that using setmelanotide as a substitute pathway agonist could make weight management easier for genetically obese individuals.

The study, set to begin by the end of the month, will follow a number of participants with Alström and Bardet-Biedl syndromes, which frequently cause obesity. For 14 weeks, participants will receive either setmelanotide or a placebo in a double-blind phase of the study. Following that, all participants will receive setmelanotide for an additional 38 weeks. After the 52-week period is over, participants will be weighed in.

The clinical study team, led by Dr. Andrea Haqq (a clinical scientist and associate professor at the University of Alberta), will consider setmelanotide a success in patients who experience a 10% or greater reduction to their body mass indices.

As the trial expands, other clinical sites may be opened around Canada. Dr. Haqq has also expressed intentions to coordinate her team’s findings with colleagues in the United States and United Kingdom. The team research is sponsored by setmelanotide’s principal developer, Rhythm Pharmaceuticals.


Genetics could be responsible for “60 to 70 per cent of what sets our body mass index.” How do you think research on the genetic causes of obesity will affect obesity rates worldwide? Share your thoughts with Patient Worthy!

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