Janalee Heinemann, MSW
PWSA (USA) Director of Research & Medical Affairs
Hopefully by now you have all read the exciting news either in the New York Times article or in the press release by the pharmaceutical company Zafgen about the exciting clinical trial results on the phase 2 study of Beloranib in patients with PWS. The drug effectively reduces body fat content while allowing patients with PWS to eat more food without gaining weight and hunger related behaviors are improved.
This is just one example of how we impact research on PWS besides direct funding to research. In 2012 I helped the president and CEO of Zafgen to coordinate a meeting during our Hyperphagia Conference in Baton Rouge at Pennington. The goal was to put together a collaborative team that could move a clinical trial forward. We had that meeting in November 2012. For those of you that don’t know much about research, to get approval from the FDA, work with the appropriate clinical and research sites, get IRB approval, recruit appropriate candidates for the research, deal with the complexity of a placebo-controlled, randomized double-blind study, and to calculate the results, is a HUGE project that often takes several years. To go from our 1st meeting in November 2012 and have the results publicized less than 14 months later is an incredible victory for Zafgen, the researchers (principal investigator was Dr. Jennifer Miller who is on our PWSA (USA) clinical advisory board), and for our families with PWS.
Just today I talked to another pharmaceutical company that wants to start clinical trial in a couple months on another drug that holds a lot of promise for impacting the hyperphagia. I have already worked with them over the last 2 years on getting FDA approval, finding out what sites might be viable for this research, and how we can promote recruiting candidates, etc. This is just a few of the exciting types of support we give to help move research forward.
We have put together a great collaborative PWS Therapeutic Development Team including myself, Rob Lutz, PWSA (USA) board member, Jim Kane, PWSA (USA) research advocacy chair, Theresa Strong, FPWR scientific chair, Dr. Steve Heymsfield, with Pennington Biomedical Research Center, and other members from FPWR and PWSA (USA). We all want what’s best for our children with PWS and are committed to working together to improve the quality of their lives.
In this same spirit of collaboration, we would like to announce that FPWR is having a Webinar on what you need to know about PWS Clinical Trials by Theresa Strong and Dr. Jennifer Miller. I think this webinar would be of benefit for many of our families, so please consider attending.
This is an exciting time for Prader-Willi syndrome research. Together we are moving mountains!