From fatal childhood cancers to debilitating neurological disorders, the rapidly emerging field of advanced therapeutics is transforming how we treat devasting complex and rare diseases in children and young people.

Leading the way in delivering these novel therapies is The Sydney Children’s Hospitals Network in collaboration with its valued partners across Australia and around the world.

Recently, the Network showcased the power of collaboration at the Kids Advanced Therapeutics Symposium, bringing together healthcare providers, researchers, industry leaders, community partners and families, to foster lively discussions around the biggest challenges and opportunities in the emerging field.

The day opened with a keynote address by Dr Mimi Lee MD, an internationally renowned genetic medicine expert and Chief of Precision Genetic Medicines at American genetic testing company, GeneDx.

While her address touched on the economic and technological challenges facing researchers and healthcare providers in advanced therapeutic translational research, she also encouraged bravery and resilience.

“Today’s model simply was not designed to accommodate this type of revolutionary paradigm shift to genetic technology and advanced computing utilities,” Dr Mimi Lee said.

“Let’s put our fears aside and lean on each other’s inspirations. Rare disease kids and families are counting on us. We need to rapidly embrace new and emerging technologies with courage.”

The day continued with insights from people with lived experience and from patient advocacy groups, who highlighted their role in shaping how healthcare providers care for complex and rare diseases, such as Angelman syndrome, genetic epilepsy or childhood dementia.

Dr Boris Waldman, Parent and Patient Advocate, Foundation for Angelman Syndrome Therapeutics Australia, called out to the clinicians in the room.

“Let us—the parents, the ones who live with rare disease everyday—help you, the clinicians,” said Dr Waldman.

Collaborating with these groups can provide better patient-centred care, either through co-designing clinical trials or improving the rare disease diagnosis journey.

It was also made clear that even the smallest considerations, like an early phone call explaining an upcoming hospital appointment, go a long way in supporting the children and families, as most are already burdened by a complex healthcare journey.

Industry and government leaders also engaged with researchers, discussing the technological and regulatory infrastructure challenges they face day to day.

“We have all the ingredients, it's all about mobilising them,” said Dr Julia Warning, Principal Policy Officer, Advanced Therapeutics, Office for Health and Medical Research, NSW Ministry of Health.

Experts unanimously agreed that Australia’s clinical trial capacity is growing. More than seven  years ago, The Sydney Children’s Hospitals Network dosed  its first patient with a gene therapy for spinal muscular atrophy. Today, the Network is supporting gene and cell therapy trials for neuromuscular and neurodevelopmental conditions, inherited blinding eye disease and deafness, childhood cancer and phage therapy targeting antimicrobial resistance.

Dr Marguerite Evans-Galea, Deputy Director, Strategy and Planning, Australian Regenerative Medicine Institute, commented on the importance of horizon scanning, understanding the upcoming therapies in the research pipeline.

“This is such an exciting time. We have a growing ecosystem to tackle these new therapies coming,” said Dr Evans-Galea.

Stephen Thompson, Chief Executive Officer of the recently launched Viral Vector Manufacturing Facility, highlighted the progress made by bringing local viral vector manufacturing capabilities to Australia.

“The opportunity here is to make that translation pathway here in Australia and that starts with clinical research organisations,” said Mr Thompson.

Keeping up with this momentum are the several translation research hubs across the country embedding research into paediatric hospitals, including The Sydney Children’s Hospitals Network, the Royal Children’s Hospital, Queensland Children's Hospital, Perth Children's Hospital and Monash Children's Hospital all of which had representatives at the symposium.

Prof Andrew Davidson, Director, Melbourne Children's Trial Centre, Murdoch Children's Research Institute, explained their biggest strength as paediatric health research leaders.

“Our paediatric hospitals want to work together, and that is a big strength of ours,” said Prof Davidson.

Dr Michelle Lorentzos, Advanced Therapeutics Medical Lead and Paediatric Neurologist at The Sydney Children’s Hospitals Network, agreed, further explaining the unique position Australia holds in the paediatric rare disease trials setting.

“We are leading the way in clinical care pathways for ultra rare conditions, and Australia is positioned well with our few rare disease numbers in a spread-out population, putting us in a great place to teach the world how to do it in the right way,” said Dr Lorentzos.

The discussions also touched on equity, research data ethics and effective processes that foster collaborations between states, all in the name of helping sick children and young people across Australia.

Overall, by bringing together community leaders, researchers, government and industry experts in one place to tackle the barriers and opportunities facing advanced therapeutics, the day represented the driving force behind Australia in bringing these novel treatments to those who need them most.

The symposium was kindly supported by Sydney Children’s Hospitals Foundation and their volunteers, as well as the Luminesce Alliance. The event was organised by the Kids Advanced Therapeutics program based at Kids Research, part of The Sydney Children’s Hospitals Network.