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
“The OXT neurons seem to be good candidates for playing a
physiological role in ingestive behavior as "satiety neurons"
in the human hypothalamus.
study provides further evidence for hypothalamic and oxytocinergic
dysfunction in PWS. The associations between oxytocin, appetite
regulation, and obsessive compulsive symptomatology in PWS warrant
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
The same researchers from France, Tauber and Molinas, also did the
research on Modafinil (brand name Provigil) that we will
be reporting on in the upcoming Gathered View. Basically, it has
been shown to be safe and effective in treating excessive daytime
sleepiness in PWS.
I am also keeping in touch with the research on Exenatide which
is marketed as Byetta. It is an analogue of
the gut hormone GLP-1, that is normally released after a meal. It is
used as a novel daily injectable treatment for diabetes as it increases
insulin secretion by directly acting on the pancreas. GLP-1 is what is
called an ‘incretin’, i.e. increases insulin. It seems to have an
advantage over some other diabetes medicines in that (like metformin) it
does not cause weight gain and may even cause mild weight loss of under
It is unclear if this weight loss is just due to
delaying stomach emptying (and so increase ‘fullness’) or also by its
actions on nerve cells in the brainstem directly or indirectly via the
vagus nerve to reduce appetite. A concern would be that many people with
PWS already have delayed stomach emptying. So there is a theoretical
risk of this getting worse with Byetta treatment in PWS, which might
increase the risk of gastric necrosis associated with severe overeating.
An Australian group is doing a clinical trial on Exenatide (Byetta),
the UK reported a positive case study, and Children’s Hospital of Los
Angeles will be looking at the effects of different obesity markers
after six months of treatment with Byetta.