The Foundation for Prader-Willi Research (FPWR), with the input of community stakeholders, has developed a strategic research plan for Prader-Willi syndrome (PWS) to accelerate research and the development of therapies for PWS. Through the implementation of new translational research programs, the management of a world-class grants program, and the development of PWS research tools, FPWR will advance research in order to develop new treatments for PWS and improve the health and well being of those with PWS.
With the support of the PWS community, FPWR’s research program has grown from funding small pilot studies to encompassing a robust and diverse research portfolio that facilitates and guides the PWS research agenda. We have played an instrumental role in advancing the field of PWS research by supporting innovative research, encouraging collaboration, engaging pharmaceutical companies, and enabling a research-ready community.Read More
Over the next 5 years, FPWR will take an increasingly directed approach to accelerate the development of new therapies for PWS and make sure we have treatments for our loved ones as soon as possible. FPWR will add to its current world-class grant program an active translational research program aimed at streamlining the therapeutic development path. The grant program will continue to fund highly innovative, early stage studies, while the translational research program will focus on efficiently transforming the most promising approaches into new therapies. Shorter-term goals of evaluating existing approaches to improve health and quality of life will be balanced with support of longer-term, potentially transformative therapeutic development.
To address the challenges that limit our loved ones’ independence and quality of life, we will complement our grant program with three translational research programs and seven research tools:
Translational Research Programs
FPWR’s grant program allows FPWR to support cutting edge, innovative ideas from some of the world’s best scientists. A robust and consistent source of funding for this program is critical for the health of the PWS research community; supporting new investigators who are just starting on their research careers, established PWS researchers who want to explore new areas, and researchers outside the PWS field who want to apply their expertise to PWS. FPWR will continue to fund innovative projects each year that will feed the research and development pipeline, advance our understanding of PWS and help the discovery of new therapeutic strategies.
A well-thought-out set of tools and infrastructure is critical to support PWS research and accelerate therapeutic development. Such tools are typically not supported by other funding sources, so it is up to us, FPWR and the PWS community, to ensure these tools are developed. By providing critical tools that all researchers can use, we can leverage our funds and accelerate research at all stages of the therapeutic development path. FPWR is in a unique position to facilitate collaboration across stakeholders and provide research tools to the community, which will improve efficiency and accelerate the development of effective therapies for PWS.Read More
Pre-Clinical Animal Network. Drug development is long and costly. To protect our investments, we need to be able to efficiently predict the safety and efficacy of drug candidates for PWS prior to sending them into clinical trials. The pre-clinical animal network, composed of expert model laboratories, will improve the predictive value of our PWS models and improve our ability to accurately predict drug safety and efficacy.
New Animal Models. Disease models are critical for therapeutic development in order to test the safety and efficacy of candidate drugs. Based on the input of experts, we will develop the new animal models of PWS needed to advance therapeutic development.
PWS cellular network. Modeling PWS in a cell is critical for screening drugs in a highthroughput fashion. We will develop a network of investigators to develop assays and cellular models of PWS for identifying new drugs, reposition existing drugs or discover new targets and pathways that can be used for investigating disease mechanisms as well as drug discovery
PWS Clinical Trials Consortium. There are many opportunities and challenges for current and future clinical trials for treating hyperphagia in PWS. The PWS Clinical Trials Consortium is leveraging the expertise and perspective from industry, academia, governmental agencies, and patient organizations to address the scientific, technical, clinical, and regulatory needs for clinical trials.
Global PWS Registry. A comprehensive database of individuals with PWS will help us better understand the full spectrum of PWS characteristics, expedite the completion of clinical trials, and determine areas of needed research and treatments to improve the lives of those affected by PWS.
PWS Biobank. Biobanks are used to store biological samples, such as blood, urine, and cells, that can be made available to investigators as needed. We will evaluate the utility and feasibility of developing a PWS biobank and develop a standardized protocol for processing samples to improve data quality and reproducibility.
Data-Sharing Platform. A platform is needed to allow PWS investigators to readily share and analyze data and information. By pulling data from different sources, we will generate new information that will help us better understand the cause of PWS and develop new approaches to treat PWS.
Over the next 5 years, FPWR will take an increasingly directed approach to guide R&D in order to accelerate the development of new therapies for PWS and make sure we have treatments for our loved ones as soon as possible. FPWR is in a unique position to guide and synergize all research efforts. To accomplish this mission, FPWR will develop a set of policies that will ensure that all programs are moving in a timely-manner, that data is shared amongst the community to avoid duplication of efforts, and to help synergizing efforts from stakeholders across all disciplines to move along the therapeutic development pathway in an efficient manner.
The 5-Year PWS Research Plan will begin in 2017; however, we will need additional funds for full implementation. To accelerate PWS research and fully execute this plan, we will need $26M over the next 5 years. You can help change the future of our children by making a personal contribution or fundraising for FPWR! Get started by visiting our fundraising page.