ACTION Centre to provide greater access to life-saving new therapies

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Ready for ACTION: Children’s Hospital Foundation Chief Executive Officer Lyndsey Rice, the Pearce family Steve, John, Carolyn and Josiah, and Children’s Health Queensland’s Director of Research Assoc. Professor Andy Moore and Chief Executive Frank Tracey at the launch of the Queensland Children’s Hospital ACTION Centre.

Children living with rare diseases and complex illnesses will have better access to cutting-edge treatments thanks to a dedicated clinical trials centre of excellence at the Queensland Children’s Hospital.

Premier Steven Miles officially launched the Queensland Children’s Hospital ACTION (Advanced therapies and Clinical Trials InnovatiON) Centre today, wrapping up Clinical Trials Week at the hospital.

The ACTION Centre, established with a $400,000 investment by the Children’s Hospital Foundation, will give children living with rare diseases and complex illnesses safe and timely access to new and potentially life-changing therapies.

About 400,000 Australian children currently live with a rare disease. One-third of these children will not live to see their fifth birthday.

Rapidly evolving 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.

These therapies can correct or compensate for the underlying genetic abnormality causing a disease, or regenerate, repair or replace damaged cells or tissues in diseases such as cancer, spinal muscular atrophy (SMA) and inherited retinal blindness.

A new gene therapy has spared the Pearce family of Brisbane the heartache of losing a second child to SMA, a rare degenerative genetic disease affecting the central nervous system.

Steve and Carolyn Pearce lost their first child Hannah at eight months to SMA in 2014, before treatment options existed.

Eight years later, the couple’s second son John was born with the same condition but he is thriving today thanks to the gene therapy Zolgensma he received at 5 days old.

The therapy replaced the missing or nonworking SMN1 gene, which helps motor neurons work properly.

“It’s meant the world to us to have the support and this gene therapy available for John,” Mrs Pearce said.

“We have a beautiful healthy boy - if you look at him you wouldn’t know he was missing a gene at birth. He’s literally a walking miracle.

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

Clinical trials of advanced therapies are vital for translating research into new standards of care.

Some advanced therapy clinical trials are currently not available in Queensland, with families having to travel interstate or overseas to access these potentially life-saving treatments for their children.

John received his gene therapy infusion in Sydney as the Queensland Children’s Hospital was not set up for gene therapy treatment at the time. The Queensland Children’s Hospital has been administering gene therapy (Zolgensma) since March 2023 and 3 patients have received the life-saving treatment to date.

Children’s Health Queensland Chief Executive Frank Tracey said stage one of the ACTION Centre would build capacity and capability at the Queensland Children’s Hospital to deliver more advanced therapies and help attract global research partnerships to support the centre into the future.

“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," Mr Tracey said.

“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 Children’s Hospital.”

Children’s Hospital Foundation Chief Executive Officer Lyndsey Rice said: “We are incredibly proud to be a founding partner of the Queensland Children’s Hospital ACTION Centre and to play a role in bringing advanced therapy clinical trials to the bedsides of Queensland children."

“Through our initial investment into the ACTION Centre, we can help fast-track potentially life-saving scientific discoveries within our home state and bring hope to families of children who currently live with a rare disease or complex illness.”

Read the media statement: ACTION Centre to boost access to life-saving new therapies for children.

Learn more about the Queensland Children’s Hospital ACTION Centre.