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We’re proud to announce the launch of the Queensland Children’s Hospital ACTION (Advanced therapies and Clinical Trials InnovatiON) Centre – a new statewide hub promising to bring new hope and advanced treatments to families with life-limiting conditions. 

Launched by the Premier of Queensland, the Honourable Steven Miles today, the ACTION Centre will give children living with rare diseases and complex illnesses safe and timely access to new and potentially life-changing treatments.


A new approach to treatment

Advanced therapies – such as gene therapy, cell therapy, and tissue engineering – have the potential to provide cures or significantly improve the health of children with rare diseases and complex illnesses.

Through bringing new advanced therapies and clinical trials to Queensland, the Queensland Children’s Hospital ACTION Centre offers new hope for our children.

Using recent technological advancements, this statewide hub aims to push the boundaries of what is possible in paediatric medicine – significantly increasing the number and range of clinical trials offered in Queensland.

Exterior of Centre for Children's Health Research

The urgent need for specialised care

In Australia, around 400,000 children are living with rare or complex conditions. Conditions like spinal muscular atrophy, blinding eye disease, childhood dementia, epilepsy, metabolic disorders, and childhood cancers.

These diseases are life-altering and often debilitating. One-third of children with rare or complex conditions will not live to see their fifth birthday.

Until now, most innovative therapies have been designed for adults – leaving a gap in the care and treatment of children with life-limiting illnesses who require specialised treatments.

Families across Queensland often faced the tough choice between missing out on treatment or traveling interstate or overseas to access a trial.

The ACTION Centre is here to change that – offering kids like John world-class care, right here in Queensland.


Closing the gap

Diagnosed antenatally with spinal muscular atrophy (Type 1), John’s future looks different from that of his sister, Hannah, who died of the same condition in 2014.

At just five days old, John received a life-saving gene therapy infusion known as Zolgensma.

This treatment, which was not available in Queensland at the time, delivers a new working copy of the human SMN gene.

This one-time infusion means that John can now live a normal life without any day-to-day impacts of his condition.

“It’s almost impossible to put into words what it means because, for us, the difference has been life and death.”

– Carolyn, John’s Mum

Now 18 months old, John is one of the first patients at Queensland Children’s Hospital to benefit from gene therapy, which will now be accessible to even more Queensland kids through the ACTION Centre.


A new era of paediatric healthcare

Children’s Hospital Foundation is proud to provide the initial investment to make the ACTION Centre a reality – an investment only made possible by the generosity and support of our community.

In particular, we are deeply grateful to the Sapphire Committee and their community for raising these life-changing funds at last year’s inaugural Sapphire Gala.

“By opening the ACTION Centre, we are starting a new chapter for our children, that is the first of many in ensuring advanced treatment is accessible to each and every Queensland child – when it’s needed most,” said Lyndsey Rice, Children’s Hospital Foundation CEO.

More than just a research centre, the ACTION Centre is the first step in Queensland Children’s Research Collaborative Council’s plan to bring together top experts, leading academics, and international health industry partners – quickly turning scientific discoveries into life-changing treatments.

By investing in advanced and innovative therapies, we are one step closer to a future where every child has the chance to lead a healthy, fulfilling life.