Thursday, 26 January 2012


One of the hardest things about living with Prader-Willi Syndrome is that there is no cure.

Your child is not going to wake up one day and be free of the condition.

But research is going on, and every now and again a little chink of light shines through the clouds. 

At the moment, the light is emanating from a drug called oxytocin. An endochrinologist in France has been trying to discover if oxytocin can benefit people with PWS. Adult patients were injected (in the nose!) with the drug, (with others receiving placebos), and their behaviour then monitored. Researchers also tested their grasp of social interaction and ability to recognise emotions.

Over the next two days - in comparison with those given placebos - Prader-Willi patients given oxytocin were more trusting, less sad, less disruptive and had fewer conflicts with others, and scored more highly in tests which evaluated social understanding.

The research has also indicated that oxytocin neurons may play a physiological role in ingestive behaviour as “satiety neurons” in the human hypothalamus. Translated into plain English, this means they could help someone with Prader-Willi feel something they have never felt before: full up.* 

A trial is currently underway using oxytocin nasal spray. This news is not to be sniffed at. (Do you see what I did there?).

Meanwhile, there’s another new drug in PWS town, too. Researchers in Australia are looking at Exenatide, marketed as Byetta, which is used as a daily injectable treatment for diabetes, which increases insulin secretion by acting on the pancreas. It’s the side effects of this drug which could prove useful in the battle to control PWS: mild weight loss, and appetite suppression.** 

Of course, there is a caveat with all this. While the word ‘breakthrough’ is whizzing round my head, I know these two pieces of research are new, small scale, and need years and years of robust development and testing. They might, after all this, prove ineffective.

But they could, potentially, help my daughter. And that's a bit of hope to hang on to, goddammit.

* If you’re a clever so-and-so, you can read the French study online in full in the Orphanet Journal Of Rare Diseases.

** The Aussie lot’s scientific stuff is here in the Journal Of Endocrinology And Metabolism.

Both of these studies were brought to my attention (and neatly summarised) by those lovely people from The Prader-Willi Syndrome Association UK and The Prader-Willi Association USA.

Now I’m going to have to lie down. I just used a whole host of words with more than three syllables and provided links to scientific studies. My brain hasn’t hurt like this since I did poorly in my GCSE chemistry exam. 

EDIT: In the comments below, Fazackerly mentions this very interesting talk by scientist Paul Zak on oxytocin - which he calls 'the moral molecule': Paul Zak: Trust, Morality And Oxytocin. If you have a few minutes spare, it's fascinating stuff.

Video is Thomas Dolby - She Blinded Me With Science


  1. Let's keep 'em crossed Drakes

  2. Hi Drakeygirl

    You might be intereted in this TED talk which is also about the effects of oxytocin. It is not really about PWS but I still think you will find it interesting.

    1. Thanks a lot for that - you're right, it was interesting. I'm going to add it to the main body of the post.