THINK Drug Discovery

Despite significant advances in paediatric medical research, a substantial portion of paediatric diseases in NSW still pose significant health burdens. Rare and high-risk paediatric cancers, neurodevelopmental disorders, and early-onset rare diseases exhibit particularly challenging outlooks, with limited and ineffective treatment options. For instance, approximately 20% of children with cancer in Australia succumb to the disease each week, while those with neurodevelopmental disorders face lifelong health challenges and reduced life expectancy. Moreover, of the 7000 known rare genetic diseases, over half affect children, with 95% lacking any available treatments. It is imperative to develop new treatment strategies and medicines to enhance outcomes for children afflicted with these illnesses.

The primary barriers to developing new drugs for childhood diseases include reluctance from large pharmaceutical companies due to smaller markets and perceived risks, as well as a lack of centralised and integrated pipeline of technologies, capabilities and expertise that includes all stages of the drug discovery and development pathway in NSW and nationwide.

To overcome this challenge, Luminesce Alliance is championing the THINK initiative, a paediatric-focused drug discovery endeavor. As the sole paediatric-centered drug discovery and development capability in the nation, THINK holds immense promise for improving children’s health outcomes in NSW. By targeting childhood diseases with the most pressing unmet needs, THINK stands to revolutionise paediatric patient care, potentially accelerating the delivery of new medicines to clinical practice by 7-10 years compared to the standard adult clinical trial pathway. Leveraging regulatory reforms both domestically and internationally, THINK aims to expedite the progression of developed drugs into clinical trials and eventual commercialisation.

Luminesce Alliance’s support is broadening the scope of the THINK Enabling Platform to encompass the discovery of new drugs for both cancer and non-cancer-related childhood diseases. Collaborating with experts in cancer, rare diseases, and neurodevelopmental disorders, THINK will identify crucial drug discovery projects with the potential to address rare paediatric illnesses. By delivering novel drug candidates primed for clinical trials, THINK will not only catalyze industry interest but also foster the expansion and retention of expertise within NSW. This initiative will unlock new opportunities for commercialisation in the region.

The THINK Enabling Platform is integrated into the Kids Advanced Therapeutics (KAT) Enabling Platform, enhancing Sydney Children’s Hospitals Network’s capacity to lead nationally and support the burgeoning field of advanced therapeutics. By integrating THINK into the Kids Advanced Therapeutics (KAT) Enabling Platform, we broaden our efforts to facilitate groundbreaking Phase I/II clinical trials for paediatric diseases.

Lead Investigator

• Professor Ian Street, Director, THerapeutic INnovations for Kids (THINK), Children’s Cancer Institute (CCI), UNSW

Research Team 

• Associate Professor Greg Arndt, Head of Drug Discovery, THINK; Head, Drug Discovery Centre, CCI
• Associate Professor Mark Cowley, Deputy Director Enabling Platforms and Collaboration, CCI and the Project Lead for Advanced Genome and Transcriptome Analytics
• Associate Professor Antoine de Weck, Group Leader, Computational Drug Discovery Biology, CCI
• Associate Professor Paul Ekert, Deputy Director Research Themes, CCI
• Ms Vanessa Tyrrell, Program Leader, Zero Childhood Cancer, Co-theme Head Personalised Medicine, CCI
• Associate Professor David Ziegler, Clinical Trials Unit Head and Staff Specialist, Kids Cancer Centre at Sydney Children’s Hospital, Group Leader, Brain Tumours, CCI
• Professor Robyn Jamieson, Head of Eye Genetics Research Unit at CMRI Head of Eye Genetics Clinics at The Children’s Hospital at Westmead and Westmead Adult Hospital
• Professor Russell Dale, Head of Kids Neuroscience Centre, Children’s Hospital at Westmead, Professor of Paediatric Neurology, University of Sydney
• Dr Tim Failes, Senior Drug Discovery Officer, THINK High Throughput Screening Facility Manager, Drug Discovery Centre, CCI

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

Paediatric precision medicine researchers and clinicians are invited to submit EOI’s to access the resources of the THINK Advanced Therapeutics Pipeline Program to identify new therapeutics.

Find out more here.