Our Research Strategy:
PWRFA has developed a directed and balanced research strategy to materially change life for those living with PWS. In the short-term we seek solutions to improve symptom management that lead to positive developmental and behavioural outcomes for those living with PWS. Longer-term, we are supporting cutting-edge research that is developing treatments to directly target the underlying cause of PWS.
We will achieve this by:
• Fostering deep medical expertise to improve the management of PWS
• Supporting clinical trials by Australian researchers, and promoting Australia as a high-quality PWS clinical trial site(s), thereby facilitating early access to new therapies
• Funding research into breakthrough medical treatments that offer genuine promise and potential.
We believe these breakthroughs are achievable in the near future, not only for future generations but for the current generation of people living with PWS.
“The will and expertise exist to develop a treatment – all that is needed is the funds” – Associate Professor Jeff Craig, Centre for Molecular and Medical Research, School of Medicine, Faculty of Health, Deakin University, and Honorary Senior Research Fellow, Murdoch Children’s Research Institute and member of the PWRFA Board and Scientific Advisory Council.
“I am convinced that targeted biomedical research coupled with disciplined research translation can deliver substantial improvements in quality of life and long-term prospects for people living with Prader-Willi syndrome” – Professor Ian Cooke, former CEO of the Cooperative Research Centre for Mental Health, Professorial Fellow Florey Institute for Neuroscience and Mental Health, PWRFA Acting Chair.
PWRFA’s strategy is to pursue a balanced portfolio of research that has been translated into a pipeline of projects, as per the diagram below:
Our Research Process:
PWRFA seeks to identify, nurture and enable novel world-class Australian research in PWS. Potential projects relevant to our mission are taken to our Scientific Advisory Council before being considered by our Board of directors.
Our research strategy is implemented by a team of dedicated individuals, including our Scientific Advisory Council, Board of directors, Research Director, CEO, and volunteers. This team of passionate individuals is driven by a desire to help people with PWS to lead independent, full and happy lives.
PWRFA acts as a central point for building connections across the research community; including scientists, clinicians, companies, government and other PWS organisations. We seek to increase awareness of PWS across the research sector, leading to new and improved programs of work that can grow into self-funding PWS research projects. We support researchers to take the discoveries made in these projects and translate them into real and effective outcomes for families.
Our current program of work includes:
• Developing a novel and innovative program of research to target the underlying genetic cause of PWS.
o Currently, we are co-funding an epigenetic research project run by Associate Professor Marnie Blewitt at the Walter and Eliza Hall Institute that is investigating the potential of targeting the epigenetic regulator SMCHD1 to treat PWS
• Supporting health care interventions that target symptoms of PWS.
• Working towards a PWS Registry that will best meet the needs of Australians with PWS.
• Engaging with leading local and international researchers, biotechnology and pharmaceutical companies to attract, support and promote clinical trials, thereby enabling early access to break-through treatments for Australians with PWS.
• Working with clinicians, hospitals and government to put research at the heart of patient care through the development of a health care model and plans to engage a PWS clinical fellow.
Collectively, these PWRFA research strategies are raising awareness of PWS across critical sectors, including government, pharmaceutical & biotechnology, academic research and in clinical settings.
Call for research projects:
If you are a researcher with a project which meets the aims of our foundation, please contact firstname.lastname@example.org. We give priority to Australian based researchers where possible.
How you can make a difference:
State-of-the-art research is expensive. There are no short cuts or quick fixes. But treatments that can make a difference to PWS are on the horizon.
To reach them we need support from individuals, charitable organisations and business.
Our research and our work isn’t possible without the generosity and goodwill of people from all walks of life, and from all corners of Australia.
Every donation takes us a step closer to identifying and using the best possible treatments to help families whose futures are turned upside-down by a Prader-Willi Syndrome diagnosis.