While much of FPWR’s work starts with discovery research, our mission extends across the full drug development pathway, supporting progress from foundational science all the way to potential treatments for Prader-Willi syndrome (PWS).
Discovery research is where scientists ask the core questions that define PWS: What is happening in the body and brain? Which biological systems are disrupted? And how do those disruptions lead to symptoms like hyperphagia, anxiety, or challenges with cognition?
Thanks to dramatic advances in scientific tools, researchers can now read DNA in extraordinary detail, observe how neurons communicate, and map specific brain regions such as the hypothalamus—an area critical to hunger, metabolism, and hormones. Using these tools, FPWR has helped world-class scientists identify the biological pathways most affected in PWS.
This growing understanding brings us to the next critical step: turning knowledge into treatments.
Scientific discovery only changes lives when it moves beyond the lab—toward real solutions for real families.
From Discover to Action: The Therapeutic Accelerator Program (TAP)
Every disrupted biological process represents a possible opportunity for intervention—something that could be corrected, modified, or bypassed. But promising ideas don’t automatically become therapies. They must be carefully tested to determine whether they are truly viable.
To meet this challenge, FPWR created the Therapeutic Accelerator Program (TAP).
TAP advances the most promising therapeutic ideas for PWS by providing targeted funding and hands-on project management to rigorously test their viability. Rather than funding open-ended research, TAP supports focused studies designed to answer a clear, practical question:
Is this approach strong enough to move closer to a human clinical trial—or not?
Through close collaboration with each research team, FPWR engages specialized scientific and clinical expertise to complete these studies efficiently and strategically. This structure allows the program to prioritize the most promising ideas, accelerate their progress, and confidently discontinue approaches that do not show sufficient potential—ensuring that time, energy, and resources stay focused on what matters most.
TAP’s Impact So Far
Since launching in 2022, TAP has supported 14 therapeutic interventions targeting some of the most challenging symptoms of PWS, including hyperphagia, inflammation, cognition, metabolism, and anxiety. These projects span a wide range of treatment strategies—from small molecules and peptides to dietary approaches, gene therapies, and novel delivery systems.
To date, 78% of TAP projects have generated results strong enough to justify further study, bringing them one critical go/no-go decision closer to entering human clinical trials.
Just as importantly, TAP has helped clarify which paths not to pursue. Three of the 14 tested interventions were shown not to be viable in their existing form, allowing them to be confidently removed from further consideration. This kind of clarity is essential—it sharpens the field’s focus and strengthens future decision-making.
Why This Matters to Families
Behind every TAP project is a community of families waiting for better options. Each experiment, each data point, brings us closer to treatments that could meaningfully improve daily life for people with PWS—whether that means easing constant hunger, reducing anxiety, or supporting cognition and independence.
The success of TAP underscores the importance of dedicated funding for innovative, early-stage therapeutic development. With sufficient support, FPWR aims to fund up to additional new TAP projects each year, continuing to move the strongest ideas toward clinical trials.
We are deeply grateful to the donors and supporters who make this work possible, and we look forward to sharing more as results are published and progress continues.
