Kids Advanced Therapeutics

Despite significant strides in paediatric medical research, a substantial burden persists in NSW due to various paediatric diseases. Rare cancers, neurodevelopmental disorders, and early-onset rare diseases present particularly challenging scenarios with limited treatment options. For instance, in Australia, about 20% of children with cancer succumb to the disease weekly, while those with neurodevelopmental disorders face lifelong challenges and reduced life expectancy. Moreover, over half of the 7000 known rare genetic diseases affect children, with 95% lacking any available treatments. There is a pressing need therefore to develop new treatment strategies and medicines to improve outcomes for affected children.
As the national leader in paediatric disease research and clinical trials networks, NSW is uniquely positioned to address these challenges. By uniting talented clinicians and researchers, the Kids Advanced Therapeutics (KAT) program has been established to facilitate the development and delivery of advanced therapeutics to children in NSW and beyond.
This initiative promises numerous benefits, including improved quality of life through cures for genetic conditions, support for the burgeoning field of advanced therapeutics, and the elevation of Sydney Children’s Hospitals Network as a national leader in this domain. Key objectives include:

• Providing specialised and focused support for the development and delivery of pharma-sponsored and investigator-initiated early phase and advanced therapeutics clinical trials at Sydney Children’s Hospitals Network.
• Increasing the number of trials delivered and reducing the time for those therapies demonstrating clinical benefit to be translated into clinical care
• Providing expert assessment of the regulatory and sponsor risks for investigator-initiated trials

Rare genetic diseases collectively affect approximately 5% of children. For the first time, advanced therapeutics have the potential to transform the lives of children by offering a cure. This has been witnessed in the lives of children with Spinal Muscular Atrophy (SMA) being transformed with a single injection of gene replacement therapy and novel gene therapies for other genetic disorders are about to be used.

There is however an urgent need to continue building capacity and capability for Sydney Children’s Hospitals Network to lead nationally and support the exponential growth in advanced therapeutics over the next five years.

Through the unique collaboration of clinicians and researchers and the ongoing funding support of Luminesce Alliance, Sydney Children’s Hospitals Network has the essential elements to establish a world leading advanced therapeutics program.

By also integrating the THerapeutic INnovations for Kids (THINK) drug discovery program, also supported via the Luminesce Alliance Drug Discovery Enabling Platform , we broaden our efforts to facilitate groundbreaking Phase I/II clinical trials targeting paediatric diseases.

Lead Investigators

• Dr Michelle Lorentzos
  Clinical Trials Medical Lead, Children’s Hospital Westmead, Sydney Children’s Hospitals Network
• Ms Lani Attwood
  Associate Director Research Operations, Kids Research, Sydney Children’s Hospitals Network

Research Team

• Adjunct Professor Paula Bray, Director Research, Sydney Children’s Hospitals Network
• Dr Laura Fawcett, SCH Clinical Trials Lead, Sydney Children’s Hospitals Network
• Asra Gholami, Program Manager

• Children’s Cancer Institute
• Sydney Children’s Hospitals Network
• University of NSW
• University of Sydney

The Kids Advanced Therapeutics educational webinar series features world-class experts in their respective fields, sharing the current and future work in the advanced therapeutics space. We invite you to join us as we explore the way advanced therapeutics is driving clinicians and researchers to prepare for a new wave of therapies that will revolutionise the future of paediatric health care.

Visit the Kids Advanced Therapeutics webinar series here.

World first trial for kids with Duchenne muscular dystrophy | SCHN Kids Research (nsw.gov.au)

Gene therapy research a breakthrough for children with SMA