Research Plan Foundation for Prader-Willi Research
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  • Develop New Treatments for PWS

    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.

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Research Programs

  • Program 1: Clinical Care Research

    We all agree that our loved ones living with PWS need improved treatments NOW. Clinical care research aims to alleviate the symptoms (such as behavioral problems, scoliosis, seizures, GI motility, central adrenal insufficiency, hypotonia, etc.) of PWS to improve quality of life. Through this program, we will characterize common PWS clinical complications and evaluate existing medicines and treatments to determine if they can be used to optimize the health and well-being of our loved ones with PWS in the near term.

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  • Program 2: Creating Critical Knowledge for PWS

    Program 2A: Genotype to Phenotype

    To improve therapies for PWS, we need to better understand how the loss of the PWS genes leads to the characteristics of PWS and how genes outside the PWS region influence the severity of disease symptoms. Fundamental insights into the underlying basis of PWS symptoms will lead to breakthrough therapies and more personalized approaches to treating PWS.

    Example: PWS Genome Project. Genome sequencing is driving a more personalized understanding disease, and promises to revolutionize how clinical care is delivered. In this pilot phase, the PWS Genomes project will support the sequencing of 100 PWS genomes, with DNA sequence information linked to the Global PWS Registry data. Genome sequencing will identify gene variants that modify clinical aspects of PWS, modify response to medications and drugs in clinical trials, and impact drug safety. This up front investment will save dividends in the future as genome data will be stored and used for ongoing investigations as new research questions arise.

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  • Program 3: Therapeutic Approaches for PWS

    Program 3A Genetic Therapy for PWS

    Genetic therapy has the potential to address the root cause of PWS. Through our genetic therapy program, we will determine if genetic approaches can reverse the symptoms of PWS. For example, what will happen if we introduce a single activated PWS gene? Can we partially or completely reverse PWS symptoms through gene replacement or gene activation techniques?

    The goal of this program will be to focus on overall feasibility of gene activation and replacement as therapeutic strategies. This program requires a long-term investment, but has the potential to be life-changing for our loved ones with PWS.

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Grant Program

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.

  • Research Tools

    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.

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Science Policy

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.

How can you help ensure the plan is completed?

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.

Fundraise for PWS Research