Tuesday, February 8, 2011

Prader-Willi Syndrome Question 4: Is there a cure for Prader-Willi Syndrome?

Q. Is there a cure for Prader-Willi Syndrome?

A. No, there currently is no cure for PWS.  Since this syndrome is genetically-based, any kind of "cure" will involve altering the pathways of many different signals sent from the hypothalamus to the rest of the body.  Finding a cure for PWS involves lots and lots and lots and lots of research!  The tough part about that, is that because the PWS community itself is not large, pharmaceutical companies (who would stand to make the most money from any drug-based therapies seen as a 'cure' for PWS) would not initially be making a ton of money - and so they aren't chomping at the bit to fund PWS research, unfortunately.  That is why fundraising efforts involving the PWS community are SO important, because compared to the amount of funding given to research efforts and quality-of-life supports for something like cancer, PWS is woefully UNDER-FUNDED.  This is very frustrating to parents and caregivers of individuals who struggle with PWS - we'd like a cure too.  Quality-of-life is just as much an issue for my child, even though she's not undergoing chemo or radiation or losing all her hair!

Sometimes people have asked whether appetite suppressants, or surgeries like bariatric surgery, are of any help for people with PWS - as these would seem to address the extreme, constant, deadly hunger sensations which are such a threat to those with PWS.  Unfortunately, appetite suppressants and surgeries have proven unsuccessful with, and actually very dangerous for, people with PWS.  You see, PWS is not really about appetite, or the size of the stomach.  It involves an erroneous signal being consistently sent from the hypothalamus to the hunger centers in the brain, and the signal is constantly giving the "you're hungry, you're hungry" message.  The message keeps coming even when the person's stomach is full to the brim with food.  Appetite suppressants don't turn off that signal; and surgeries which alter the stomach's size definitely do not turn off that signal. 

How do I personally feel, as a parent of a child with PWS, about there someday being a "cure"?  Well....of course I would love for there to be some kind of drug that S could take, or safe surgical procedure that could be done, which could relieve her of even half of what she goes through every day.  I would especially love it if some smart researcher could formulate a drug which could turn off that hunger signal...that, for S, is the hardest part of this.  No surprise there - of course it is hard for her to think about much else, when her brain is telling her she is starving, every second of every day!  But I am also realistic about the "cure" idea.  PWS is genetic, which means it is embedded in every single cell of her body.  Whatever might be found to help the PWS community will involve some pretty complex research and, at this rate of funding, is still some years down the road.  So I do have hope for some kind of cure, but I know that we have to wait...hopefully something will be discovered in S's lifetime. 

While much, much more research is needed, I am encouraged to know that there is research currently being done on PWS, how it happens, how the genes in question work exactly, what role the hypothalamus plays in satiety and obesity, and what might be done to address the challenges caused by PWS.  The general population should be excited by PWS research as well, because if some of these questions about the "mechanics" of PWS can be answered, then questions about obesity itself will hopefully get answered as well.  If we can better understand why we eat what we eat, I think we may all be better off.

For more information about Prader-Willi Syndrome, and to donate towards research and quality-of-life resources, visit http://www.pwsausa.org/ OR http://www.pwsaohio.org/.



Rachel said...

Wow thanks for all that information. I have struggled with my weight for my whole life, I cannot imagine being desperately hungry all of the time. How do you structure your daughter's meals and snacks, do you keep her on a schedule? Is possible obesity the greatest risk or is there other major effects of a lifetime of PWS? Sorry to rapid fire more questions at you, but I don't think I could get the answers to these questions from anyone who has not lived with it.

Rachel said...

sorry to clog your comment section! I just wanted to say I love the new blog design!