Pitolisant the new medication for PWS ?

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We have just got this unbelievable news about the new medication called Pitolisant (Wakix) what was approved by the European Medicines Agency in fall 2015. The drug will be available in Europe in fall of 2016 and PWS communities are working hard on the intention to bring the drug to Canada and United States. 

It is expected that pitolisant will be uniquely able to meet the unmet needs of the

Prader- Willi Syndrome (PWS) community!

Pitolisant the new medication for Prader-Willi Syndrome?
Pitolisant targets the histamine 3 receptor (H3R). The H3R is found primarily in the nervous system and gastrointestinal tract. It has many functions throughout the body and its role in health and disease is under active investigation. Thus far, scientists know that the H3R regulates many neurotransmitters (brain chemicals) and plays a role in the regulation of sleep and hunger. Scientists learn and publish more about the H3R every month.

 Recently Holger Stark, PhD of the Heinrich Heine University of Dusseldorf in Germany presented his research on pitolisant and the H3R at the International Prader-Willi Syndrome Organization meeting in Toronto. Dr. Stark is an international leader in research on the H3R and his presentation raised the possibility that pitolisant may benefit individuals with PWS.

While improved wakefulness alone would be a major improvement in the quality of life of individuals with PWS, there are many reasons to believe that the neurologic benefits of pitolisant in the PWS population will extend beyond improving wakefulness. For example, many individuals with PWS struggle with mental health issues and learning disabilities which may be improved by pitolisant. Moreover, research suggests that the cognitive enhancing activities of drugs such as pitolisant may help protect against schizophrenia, attention deficit disorder, and other cognitive disorders. Lastly, children with PWS are often plagued with severe gastrointestinal problems that remain difficult to treat. Pitolisant may affect multiple cells in the gastrointestinal tract and could theoretically improve these symptoms. For these reasons, we propose that it is biologically plausible that pitolisant would be useful for the PWS patient population, not only in the treatment of excessive daytime sleepiness, but that it would also improve the ability of individuals with the syndrome to respond to environmental stressors such as changes in temperature, high glucose loads, and complex social interactions.

 Chion Foundation hypothesize that pitolisant may be uniquely positioned to meet the needs of patients with PWS. They propose that it will normalize sleep, behavior, and cognition. We are also eager to explore whether or not it will relieve other burdens of the complex syndrome.

There are already some families who tried Pitolisant for their PWS child and they say it is a life-changer for them.

Please read these articles below for more information: