Can We Improve Cognition in People with Prader-Willi Syndrome?

Researchers are increasingly focusing on cognition in Prader-Willi syndrome (PWS), including attention, memory, planning, and flexible thinking. FPWR recently convened international experts to identify the best ways to measure cognitive improvements and prepare for future clinical trials of therapies designed to improve everyday functioning and independence.

Looking Beyond Hyperphagia: A New Focus on Cognition in Prader-Willi Syndrome

When most people think about Prader-Willi syndrome (PWS), they think about hyperphagia—the intense, persistent hunger that is one of the condition's hallmark features. While hyperphagia remains a major focus of research, families know that many of the daily challenges of PWS extend far beyond food.

For many individuals with PWS, difficulties with attention, memory, planning, decision-making, communication, and adapting to change can affect school, work, relationships, and everyday independence. These thinking skills—collectively known as cognition—are becoming an increasingly important area of research.

Recognizing this need, the Foundation for Prader-Willi Research (FPWR) recently convened an international group of experts to begin developing a roadmap for future clinical trials to improve cognition in people with PWS.

By preparing today, researchers will be better equipped to evaluate tomorrow's therapies.

What Is Cognition?

Cognition refers to the mental processes we use every day to learn, think, remember, solve problems, make decisions, communicate, and adapt to new situations.

Many individuals with PWS experience cognitive challenges.  Some individuals have mild learning differences, while others have more significant intellectual disabilities. Just as importantly, people with PWS often demonstrate meaningful cognitive strengths that can be built upon throughout life.

Understanding both strengths and challenges is essential as researchers work toward developing treatments that improve everyday functioning and quality of life.

Common Cognitive Challenges in PWS

Many individuals with PWS experience difficulties with:

• Maintaining attention and focus
• Working memory (holding and using information in the moment)
• Planning and organization
• Adapting to unexpected changes
• Processing information efficiently
• Understanding abstract concepts
• Social thinking and communication

Common Cognitive Strengths

Many individuals with PWS also demonstrate strengths in areas such as:

• Long-term memory
• Visual-spatial reasoning
• Reading ability
• Visual perception

While these patterns are common, it's important to remember that every individual with PWS has a unique cognitive profile.

Why Is Cognition Becoming a Research Priority?

Until recently, relatively few clinical trials in PWS have focused specifically on cognition. One reason is that researchers have not yet reached consensus on which thinking skills to target or how meaningful improvements should be measured.

As promising therapies begin emerging—including treatments that may improve brain function rather than addressing a single symptom—the PWS research community must be ready to evaluate whether these therapies truly make a difference.

That's why FPWR recently brought together approximately a dozen researchers and clinicians from the United States, Canada, and the United Kingdom with expertise in cognition, intellectual disability, and neurodevelopmental disorders.

Their goal was to develop a roadmap for future cognition research in PWS.

Preparing for Future Clinical Trials

During the workshop, experts discussed several important questions that will shape future research:

• Which aspects of cognition should new therapies target?
• Which assessment tools can best detect meaningful improvements in cognition in PWS?
• What challenges are unique to conducting cognition-focused clinical trials in PWS?
• What research needs to happen now to prepare for future clinical trials assessing cognitive function in PWS?

Answering these questions today will help ensure future clinical trials are designed to measure outcomes that matter most to individuals with PWS and their families.

How Do We Measure Cognition in PWS? 

One of the greatest challenges in cognition research is that no single test fully captures how someone functions in everyday life.

People with PWS have a wide range of intellectual abilities. Test performance may also vary from day to day depending on factors such as anxiety, rigidity, obsessive-compulsive behaviors, fatigue, motivation, or hyperphagia.

For that reason, researchers are looking beyond traditional IQ testing and focusing on assessments that reflect real-world functioning.

Families consistently identify improvements in everyday life as the outcomes that matter most, including the ability to:

• Adapt more easily to unexpected changes
• Stay focused on tasks
• Transition between activities or topics of conversations
• Solve everyday problems
• Manage daily routines more independently

These kinds of improvements have the potential to reduce stress for families while increasing independence and quality of life for individuals with PWS.

During the workshop, experts evaluated a variety of assessment tools to determine which best measure executive functioning, attention, processing speed, cognitive flexibility, and working and short-term memory—areas that are particularly important in PWS.

Learning From Other Neurodevelopmental Disorders

Researchers are also benefiting from advances being made across the broader field of neurodevelopmental disorders.

New medications and interventions are currently being studied in conditions that share some cognitive features with PWS. These include therapies that target brain signaling pathways, reduce neuroinflammation, or improve communication between brain cells. Researchers are also evaluating non-drug approaches such as neuromodulation, brain stimulation, and biofeedback.

Although these approaches are still under investigation, they may provide valuable insights that accelerate future research in PWS.

FPWR will continue monitoring these developments and identifying opportunities to bring the most promising scientific advances to the PWS community.

What's Next for Cognition Research in PWS?

This workshop represents the beginning of what FPWR expects will be a multi-year effort to advance cognition research in Prader-Willi syndrome.

By identifying meaningful treatment targets, selecting reliable outcome measures, and preparing researchers for future clinical trials, we are building the scientific foundation needed to evaluate the next generation of therapies.

Most importantly, this work keeps the priorities of individuals with PWS and their families at the center.

The ultimate goal isn't simply to improve performance on a cognitive test. It's to help someone with PWS better navigate unexpected changes, stay engaged in school or work, communicate more effectively, become more independent, and enjoy a fuller, more meaningful life.

As new therapies move closer to clinical trials, FPWR is helping ensure the research community is ready to measure what truly matters.

Frequently Asked Questions

What is cognition in Prader-Willi syndrome?

Cognition refers to the mental processes involved in learning, memory, attention, planning, problem-solving, communication, and adapting to new situations. Many individuals with PWS experience challenges in these areas.

Can cognition improve in people with PWS?

Researchers are actively working toward therapies that may improve aspects of cognition. While no treatments are currently approved specifically to improve cognitive function in PWS, FPWR and other researchers are laying the groundwork for future clinical trials.

Why is executive functioning important in PWS?

Executive functioning includes skills such as planning, organization, attention, working memory, flexible thinking, and self-control. Improvements in these areas could help individuals with PWS become more independent and better manage everyday challenges.

How could improving cognition affect daily life?

Even small improvements in cognitive function could have a meaningful impact on everyday life. Better attention, flexibility, planning, or problem-solving may help individuals with PWS manage daily routines, cope with changes, communicate more effectively, increase their independence, and reduce stress for both themselves and their families.