Why We Are HopeFULL for the Future of People with Prader-Willi Syndrome

Prader-Willi syndrome (PWS) is a complex genetic disorder with a range of challenging symptoms, including hyperphagia (excessive hunger), obesity, and behavioral issues. Despite these challenges, recent advancements in medical research and therapeutic developments offer substantial hope for individuals with PWS and their families.

Here are some of the key reasons why the future looks promising for those affected by PWS.

Gene therapies are becoming a reality.

In recent years, there has been a dramatic increase in gene therapy approvals. 19% of FDA-approved gene therapies (7 of 36) were approved last year, and analysts are predicting 10–20 more will be approved by 2025; seven years ago there were zero. This is good news for any genetic disorder, as it demonstrates that both regulatory agencies, drug developers, and the general population are becoming more familiar and comfortable with the process. There are still difficult questions we need to answer as we develop gene therapy for PWS, but the approvals for other diseases will reduce the risk involved with developing gene therapy for PWS, paving the way for PWS researchers. Learn more about genetic therapies for PWS.

There is a tremendous amount of activity in the obesity field.

After years and years of failures, there are finally new classes of drugs that are showing impressive effects in triggering weight loss in typical individuals. Drugs like Wegovy and Mounjaro are ‘game changers’ for the general obese population. It remains to be seen if these drugs are safe and will have similar efficacy in PWS, but a slew of additional anti-obesity drugs are in development with different'mechanisms of action’, so there will likely be new options to evaluate in our loved ones with PWS. Even if these turn out not to be the blockbusters they are in the general population, they may be important ‘tools in the toolbox’ for managing PWS. As well, insights into whether or not these drugs do or don’t work in PWS will help us find the pathways that are most important to target for PWS-associated hyperphagia. 

The results of the 'PATH for PWS' study will support the development of all new treatments for PWS and guide clinical care. 

We are enormously grateful to the 600+ families who participated in the 4-year Registry-based “PATH for PWS” study. Thanks to the efforts of these families, we were able to document the serious medical events that occurred in PATH participants and measure changes in PWS-associated behaviors over time. Findings from the PATH study have shown that individuals receiving Soleno’s drug (DCCR) have positive changes in hyperphagia and other PWS-associated behaviors compared to those not taking the drug. We expect PATH data to continue to inform clinical practice and clinical trials for PWS for years to come.

A new tool will allow for efficient screening of 4,000 potential new drugs.

Ongoing research has identified several differences between PWS and typical brain cells. FPWR has commissioned the development of large-scale tests that will screen thousands of potential treatments to identify potential drugs that fix these brain cell deficits. We expect to be ready by mid-2025 to screen 4,000 approved drugs. Results from these screens will have a measurable impact on the future of PWS research.

We have a robust pipeline of PWS clinical trials.

Currently, 8 PWS clinical trials targeting hyperphagia, behavior, and daytime sleepiness are enrolling people with PWS, and additional trials are expected to begin in the next 12 months. New companies continue to enter the PWS space, and new discoveries from our research programs will continue to feed the pipeline. Learn more about PWS clinical trials. 

We are close to having our first treatment for hyperphagia.

A New Drug Application for DCCR is currently under review with the FDA with a target completion date of December 27, 2024. In clinical trials, DCCR has improved hyperphagia and decreased PWS-related behaviors. If approved, DCCR will be the first approved treatment for hyperphagia.  Learn more about DCCR.

How can you help advance research faster?

Progress requires participation from our PWS community. We need your action to ensure treatments for PWS are found! How can you advance PWS research?

1. Join the Global PWS Registry. When you complete surveys in the Global PWS Registry, you are helping us identify trends that can be used to improve how we care for our loved ones with PWS, direct where we should place more resources, and help support the advancement of drugs from clinical trials through the FDA approval process. Learn more about the Global PWS Registry.
   
   
2. Participate in Clinical Trials. Clinical trials are a critical step in finding treatments for PWS. Hundreds of volunteers are required every year to support PWS clinical trials. Please consider participating in a trial! Learn more about PWS clinical trials.
   
   
3. Fundraise or Donate for Research. Fundraising is an empowering way to accelerate cutting-edge research. FPWR is working night and day to find treatments for our loved ones with PWS. With your help, we can get there faster! Learn more and get started fundraising today.