Follow-up to Dr. Rob Nicholls webinar
Dr. Nicholls did a wonderful job on describing the genetics of PWS and his involvement with some of the major breakthroughs in this area. We are very fortunate to have him on our PWSA (USA) Scientific Advisory Board. For more information on Dr. Nicholls webinar and his ride for research you can visit his website www.robsrideforresearch.com . I would just like to follow up on a couple of the questions asked during the webinar.
Janalee Heinemann – PWSA (USA) Director of Research & Medical Affairs
- Q — Amy: Many people ask “Why isn’t there a medicine for them to stop eating?” Many pharmaceutical companies have developed several products to improve appetite control, is this an area of research that is also being explored with PWS
A — The good news is that this is an area where there is currently more interest in using PWS for research than ever before! Several pharmaceutical companies wisely see PWS as the “window of opportunity” for finding breakthroughs in the area of appetite and obesity — and as we all know, there is a worldwide interest in coming up with answers to this complex and expanding problem. There are so many companies either looking at the potential of using PWS, or are in the early stages of doing a drug study on PWS, that we’ve established a collaborative PWS Therapeutic Development Team to track, encourage, and assist the progress of drug development for PWS. This team consists of those of us with a special interest and expertise from PWSA (USA), FPWR, and Pennington. An example of some of the pharmaceutical agents already in the planning stages are: a belornib, belviq, qsymia, and oxytocin. There are several others that we are tracking and working with companies to encourage further development. I would forewarn parents though that there many products touting appetite and weight control that are not effective for PWS. We will keep you informed of the progress of the research studies that appear the most promising.
- Q — There were several questions during the webinar on growth hormone that would take too much space to answer here, but it reminds me that I hope you are all getting our newsletter, The Gathered View. Over the last several years, we have had many articles on growth hormone in the newsletter. A small example is that in the most recent newsletter, I wrote a report from our international conference in Cambridge. The following are excerpts from the scientific portion of that conference that dealt specifically with growth hormone.
- An eight year study on growth hormone treatment (GHT) demonstrated that GHT is a potent force for counteracting the clinical course of obesity in children with PWS. (Nienke Bakker, The Netherlands)
- The conclusion of an Italian study further confirms that long-term growth hormone treatment in adults with PWS significantly improves body composition, muscle size and quality, and increases muscle strength and exercise tolerance. (Graziano Grugni, Italy)
- In a Scandinavian study looking at growth hormone treatment (GHT) in adults with PWS to see if there was an increased risk of sleep apnea due to GHT, they compared those treated with GHT or a placebo for 12 months. They did a baseline and a follow-up sleep study. It was found that GHT in adults with PWS is not associated with an increased risk of sleep apnea.(S. Farholt, Sweden)
If you do not get our PWSA (USA) newsletter, to assure that you are kept updated on the latest information regarding Prader-Willi syndrome, please click here to sign up for membership or contact our Communications Specialist, Ben Karp at email@example.com . Signing up as a member, you will also have access to all the past newsletters that have a wealth of information. Also, remember that for specific medical questions or problems, you can contact me, and for non- medically related issues, you can contact our other two support counselors, Kate Beaver or Evan Farrar. Just call 800-926-4797.