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Research – how do we feel about it?

Thanks to everyone who contributed to our post on social media which asked ‘how people feel about research’. It’s great to hear everyone’s perspective on research. As a charity we recognise research can be an emotive subject. On one hand there are the frustrating limitations of science (in the here and now) and painfully long drug approval processes; On the other, there is the prospect of new treatments which could make a positive and perhaps transformational difference to the lives of our loved ones and those around them.

Unfortunately, research takes time! To get a drug to approval takes years as it follows along a path of initial research toward phase three drug trials. At each step there is always the prospect of failure and disappointment – even at the final hurdle, as we painfully saw with Carbetocin back in 2022 (albeit thankfully the drug is to undergo a new phase 3 trial with a new company). All the while we have to wait whilst our loved ones go without treatments which could potentially alleviate hyperphagia, sleep issues or assist with behaviours and body tone.

Despite these frustrations we believe there is reason to be optimistic and that we will see new treatments in the not-too-distant future. This belief is on the basis that there are currently seven trials underway in locations across the world (PWS Clinical Trials Alert ( and six trials at phase two or three. There is also the prospect that DCCR will be approved by the FDA in the USA later this year. If it were, it would mark the first drug approval since Growth Hormone. Speaking of Growth Hormone, it is worth reflecting on how far the PWS community have come since that drug was approved. Not only has Growth Hormone made a significantly positive impact ‘altering the natural history’ of PWS, but we have also saw significant advances in collective understanding about diet and standards of care. Meanwhile understanding of PWS at a cellular level has grown exponentially. These are all good things which have come from Research.

What does the future hold? Going forward it is entirely possible that we will see treatments for the significant unmet needs of the condition (hyperphagia, narcolepsy and difficult behaviours). Whilst drug trials have been frustratingly absent from the UK there is the real prospect that could change and FPWR UK are currently in conversation with drug companies about this.  As we look further into the future, advances in areas like Gene Therapies and Artificial Intelligence could open up new possibilities for further treatments and there has been a lot of exciting preliminary work in these areas.

Ultimately as a charity we believe in the power of science and research. We know there are some truly talented individuals working hard to help our loved ones. Our challenge is that the road is long, and we have to keep as many people on that road with us because our work is particularly crucial for early stage research. Because our charity is almost exclusively made up of parents of those with PWS, we completely share the frustration that there isn’t more RIGHT NOW! But we believe in a brighter future none the less and in the meantime, we also believe it’s important to share the brilliant uniqueness of all of our loved ones with PWS.