One year on from its launch in May 2024, the Queensland Children’s Hospital ACTION Centre has turned its bold vision into meaningful progress for children facing life-limiting conditions, from spinal muscular atrophy to blinding eye disease and epilepsy.
The Queensland Children’s Hospital ACTION (Advanced Therapies and Clinical Trials InnovatiON) Centre is a statewide hub working to give children living with rare diseases and complex illnesses safe and timely access to new and potentially life changing treatments.
In just 12 months, the team has already been selected for four advanced therapy trials, including one gene therapy trial, and is under consideration for five additional advanced therapy trials.
In what is a major step in translating research into real-world care, the trials offer a possibility of a future without rare disease and lay the groundwork for future advanced therapy trials for Queensland families.
“With these advanced therapies and clinical trials, we’re aiming to fix the underlying problem and be curative. We’re no longer just treating symptoms – we’re targeting the root cause of these diseases,” said Dr Andy Moore, Paediatric Oncologist and Director of Research at Children’s Health Queensland.
Shaping Queensland as a global leader in advanced clinical trials
Right now, approximately 2,200 children in Australia each year are born with a rare generic condition. 94% of conditions do not have an approved treatment.
The ACTION Centre represents a strategic effort to position Children’s Health Queensland within a highly competitive global and national landscape, currently dominated in Australia by paediatric institutions in Sydney and Melbourne.
Beyond the direct benefits to Queensland children, Children’s Hospital Foundation and our donors are supporting the broader clinical research community through strengthening Queensland’s capacity and reputation to deliver advanced therapy trials.
Children’s Health Queensland Chief Executive Frank Tracey said: “Children’s Health Queensland’s unwavering focus on achieving the best possible health outcomes for Queensland children demands a commitment to innovation and translational research.”
“By partnering with advanced therapies and clinical trial leads from across Australia and the world, we will not only improve access to novel therapies, but also drive the rapid translation of research into life-changing treatments and further enhance the clinical expertise within the Queensland,” he added.
Our ongoing support of the ACTION Centre
Continued investment into advanced therapy trials at the ACTION Centre requires ongoing support from our generous community.
Over the next few years, the ACTION Centre will continue to lay critical foundations to support future growth in advanced therapy trials. It will expand its trial portfolio and invest in modern infrastructure to support tomorrow’s children – sooner.
Children’s Hospital Foundation CEO, Lyndsey Rice is passionate about all Queensland children having access to world-class – when it is needed most.
“Until recently, most innovative therapies have been designed for adults. We believe every child diagnosed with a rare illness deserves the same access to quality care, and that starts with life saving research,” Ms Rice said.
“Research is only made possible through our generous community – the Sapphire Committee and the role their community played in our initial investment into the ACTION Centre is proof of what is possible when we work together to create real and transformational change,” she added.
With your support, we can invest in more than just research – we can help push the boundaries of medicine and bring life saving treatment to Queensland children, in Queensland.
To find out more about the Queensland Children’s Hospital ACTION Centre, head here
The difference that shapes their world
Through supporting the Children’s Hospital Foundation, you can improve childhoods and ensure all children and young people have access to treatment - sooner.
