According to a story from labblog.uofmhealth.org, one of the defining features of Prader-Willi syndrome, a rare disorder, is hyperphagia, which is extreme overeating and obsession with food. While this is just one of the problems that the disorder can inflict, it is one of the most serious, and often leads to obesity at a very early age. This undoubtedly takes a toll on the health of the patient. Millendo Therapeutics, a biopharmaceutical company that calls Ann Arbor, MI its home, is aiming to address hyperphagia in this genetic illness.
About Prader-Willi Syndrome
Prader-Willi syndrome is a genetic disorder which is most characterized by childhood obesity that results from an abnormal, insatiable appetite. This obesity often continues into adulthood. In most cases, the syndrome is caused by the deletion of a certain section of chromosome 15. In about a quarter of cases, the patient receives two copies of chromosome 15 from the mother but gets none from the father. This syndrome is not considered heritable, as the genetic changes occur during gestation. Symptoms of Prader-Willi syndrome include slow development, poor feeding, muscle weakness, obesity, over-eating, abnormal flexibility, scoliosis, sleeping excessively, speech delays, intellectual disability, poor muscle tone, delayed puberty, and infertility. Excessive eating also leads to elevated risk of diabetes. Management may include physical, occupational, and speech therapy, limiting access to food, and injections of growth hormone (in child patients only). To learn more about Prader-Willi syndrome, click here.
Signs of Progress
The company’s solution to hyperphagia is a drug called livelotide, which has shown some potential in clinical studies so far. A sample of 47 adult patients took either a three or four mg dose of the drug once per day during a two week period, and study investigators reported a substantial drop in hyperphagia behaviors. There is no doubt that these findings are promising, but at the end of the day, these patients are affected from a very early age. As a result, testing livelotide in kids with Prader-Willi syndrome is going to be important.
Child patients between the ages of four and seven years are currently being recruited for another study. While the main goal of the study is to test the drug’s impact, the researchers also hope to glean important data about how the brain controls hunger and food consumption.
