Prof David Amor secures NHMRC grant to accelerate precision therapies

Murdoch Children’s Research Institute (MCRI) Professor David Amor has secured funding to lead a research team to accelerate precision treatments for children with neurodevelopmental disorders. 

MCRI Neurodisability and Rehabilitation Group Leader Professor David Amor was awarded a $5 million National Health and Medical Research Council (NHMRC) Synergy Grant to bridge the gap between gene discovery and effective therapies for neurodevelopmental disorders.

Neurodevelopmental disorders affect up to 15 per cent of children around the world. In Australia,one in 10 children live with a neurodevelopmental disorder.

While advances in genomic medicine have meant about half of neurodevelopmental disorders have an identifiable genetic cause across more than 1,000 genes, targeted treatments for children are still unavailable and care is limited to supportive, symptom-based interventions.

Professor Amorand his team will establish an integrated precision medicine pipelineand use genetically defined exemplar conditions such as neurofibromatosis type 1, KAT6A syndrome and KMT2A-related disorder to investigate the biological processes driving neurodevelopmental disorders, identify new treatments and prepare promising therapies for clinical trials.

Image: Professor David Amor 

Professor Amor will combine detailed patient data with patient-derived stem cells, cerebral organoid models and animal studies to better understand what different neurodevelopmental disorders have in common and what makes each condition unique.

He will also develop biomarkers and clinically meaningful outcome measures to track disease progression and assess potential treatments. By leveraging insights from common and ultra-rare genetic conditions, the project aims to improve the design of future clinical trials and accelerate the development of precision therapies for children.

Professor Amor said the research will bring together genomics, clinical care and advanced disease models under a single unified framework to streamline the path from scientific discovery to new effectivetreatments.

“We want to ensure that advances in genetic discovery are matched by advances in treatment to give children and their families more options,” Dr Amor said.

"I am excited to lead an expert team to tackle some of the biggest barriers to developing precision therapies for children with neurodevelopmental disorders.” 

NHMRC’s Synergy Grants aims to support outstanding multidisciplinary teams of investigators to work together to answer questions that cannot be answered by a single investigator.

Read more about Professor Amor’s work, and MCRI’s Neurodisability and Rehabilitation research.