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
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.
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
We received the following hopeful message from Cristel Nourissier,
General Secretary of
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:
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.
following is from my prior posting)
Exciting New Research for Prader-Willi Syndrome
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.
Director of Research & Medical Affairs
also been in touch with a researcher in Australia who are also doing a
study on oxytocin. The following is from the Australian researcher.
neurons seem to be good candidates for playing a physiological role in
ingestive behavior as "satiety neurons" in the human
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.
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