Prader-Willi Syndrome Research
Energizing the Field!
FPWR has provided more than $3,000,000 in research support since 2003, with more than 60 research projects funded to date. We look forward to the findings of the new studies, just as we are beginning to see the payoffs from our investment of previous years.
FPWR support has contributed to more than thirty publications in the medical literature to date, advancing our understanding of the genetic alterations underlying PWS, the metabolic disturbances in PWS, learning strengths and differences, and how the brain regulates hunger in normal individuals and in those with PWS. Two new mouse models have been developed, which should allow therapeutic interventions to be quickly evaluated. Additional studies aimed at developing novel drugs for PWS, using stem cells to understand the PWS at the cellular level and applying cutting edge genetic technologies to examine the role of critical 'snoRNAs' in PWS are all underway.
To view summaries of FPWR funded projects, click here.
The 2013 Research Program
The 2013 Prader-Willi Syndrome (PWS) Research Program outlines research priorities and initiatives that will advance our mission to eliminate the challenges of PWS. This Program is based on the Prader-Willi Syndrome Research Strategy Workshop, which brought together leading clinical and basic scientists to consider the strengths, weakness, opportunities and unresolved research questions in PWS. Their recommendations are summarized in the document “Prader-Willi Syndrome Research Strategy Workshop: Report to the Community”. The 2013 PWS Research Program outlines new initiatives addressing priorities in the PWS field, and represents the next step towards our goal of helping those with PWS to lead healthier, more fulfilling lives.
Create a Global PWS Registry ($150K) Create a registry, integrated with the Global Rare Disease Registry, to gather clinical information on people with PWS from around the world. The Global PWS registry will enhance our understanding of PWS, facilitate clinical trials, and assist in the development of recommendations and standards of care.
Support PWS Working Groups ($50K) Provide funds to begin the implementation of the recommendations of working groups that have been created to bring together the expertise needed to guide the efficient advancement of critical research areas.
Support Investigator Initiated Projects ($400K) The investigator-initiated grant program allows researchers to propose their most innovative PWS research projects across a broad range of disciplines. A request for applications will be sent out in the Fall of 2013, we would like to spend a minimum of $400k in support of these grants.
Provide Resource Sharing Grants ($50K)
These small, rapid turnaround grants will allow scientists to share their important research tools with other scientists, promoting collaboration and eliminating duplication of effort.
Prepare for PWS Clinical Trials ($50K)
We have identified over 50 drugs/therapies that may have applicability to PWS. This project will prioritize potential therapeutics, provide funding to develop and validate measures of efficacy, and establish a plan to efficiently conduct PWS clinical trials.
PWS Clinical Trials ($400K)
As potential therapies are prioritized, the top priority drugs/therapies will need to be evaluated in clinical studies. Clinical trials can be very expensive. With additional funding, we can potentially fund multiple trials in 2013.