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Clinical Trials on Oxytocin for PWS Are Moving Forward

If you will recall, I wrote an article for our newsletter and have on our home page information (see below) on oxytocin (carbetocin is a long-acting oxytocin analog) .as a potential promising drug for Prader-Willi syndrome. I received the notice below from our IPWSO board member, Jackie Waters in the UK: Note that this drug has not been approved for development for PWS yet, but the potential for approval for a clinical trial looks encouraging. I have been in contact with Ferring Pharmaceuticals, the company mentioned that is working to get approval to develop carbetocin for PWS. We will do what we can to support the efforts of all interesting in pursuing carbetocin and/or oxytocin for PWS

Note: The first drug approved under the Orphan Drug Act in the USA was the growth hormone, Genotropin in 2000, and another growth hormone drug, Omnitrope was approved in 2010.  

Janalee Heinemann, M.S.W., PWSA (USA) Director of Research & Medical Affairs

 (The following was posted by the PWS UK Association Instant News) 

Designation of an orphan drug for Prader Willi syndrome by the European Medicine Agency 

We received the following hopeful message from Cristel Nourissier, General Secretary of Eurordis, and parent of someone with PWS

Please let me share with you a great message of hope for people living with Prader Willi syndrome and their families, and maybe also for other rare diseases associated with food, autistic, and some psychiatric behaviour problems in the future.

The Committee for Orphan Medicinal Products of the European Medicine Agency recommended last week the granting of an orphan designation for carbetocin. This is the first orphan designation opinion for the treatment of Prader-Willi syndrome.

Orphan designation will give the sponsor of carbetocin, Ferring Pharmaceuticals, access to incentives that encourage and support the development of this medicine. You can have a look at EMA website: http://www.ema.europa.eu "Committee for Orphan Medicinal Products (COMP) January 2012 highlights "

This very important breakthrough is the result of our common commitment for the many years for people living with Prader Willi syndrome. Lifetime dedication of a few researchers and clinicians, plenty of volunteer work in our associations made this possible.

This information is now public and can be given to families. However, it is important to explain that the development of a new drug may take between 6 to 12 years.

 

 

(The following is from my prior posting)

Exciting New Research for Prader-Willi Syndrome

Janalee Heinemann, MSW
Director of Research & Medical Affairs 

There has been a lot of interest and excitement about three pharmacological products being researched that may have an impact on PWS.  One getting a lot of publicity is oxytocin. I know the researchers, Professor Maithé Tauber and Catherine Molinas from France, who recently published their results.  They sent me a copy of their full study, which will be reported on in the PWSA (USA) newsletter, The Gathered View.  Professor Tauber states, “Oxytocin is a key hormone in building social interactions and empathy.” and “Two days after administration of oxytocin, we noticed that our patients had increased trust, decreased sadness and showed less disruptive behavior.”  Because of space, we are holding on the report of the oxytocin study until the next edition.   

I have also been in touch with a researcher in Australia who are also doing a study on oxytocin.  The following is from the Australian researcher. 

“The OXT neurons seem to be good candidates for playing a physiological role in ingestive behavior as "satiety neurons" in the human hypothalamus.

§   One study provides further evidence for hypothalamic and oxytocinergic dysfunction in PWS. The associations between oxytocin, appetite regulation, and obsessive compulsive symptomatology in PWS warrant further investigation.

§  We are currently conducting a trial of oxytocin nasal spray in PWS. We are still in the trial phase. Hopefully we will be able to form some conclusions by the end of the year.”
            ~Stewart L. Einfeld, Chair of Mental Health
            Senior Scientist, Brain and Mind Research Institute
            University of Sydney

 

edited: 02/09/2012

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