Discovery of new treatments for brain development disorders

Aim

To improve the quality of life for children and their families through the identification of new treatments for patients with a wider spectrum of genetic causes of brain developmental disorders.

Collaborators

The Royal Children’s Hospital, the Walter and Eliza Hall Institute of Medical Research and the University of Melbourne.

About the program

We anticipate that through this program of research, more children with intellectual disability will receive targeted treatment to enhance their brain development. By building capacity in registries, data science, clinical trials and implementation science, we will be in a position to generate new evidence about the cause, prevention and best treatment for neurodevelopmental disability. Such developments will not only improve the conditions of children with brain developmental disorders, but will enhance their general health, their future prospects and will impact the health and wellbeing of their families also. These benefits will potentially alleviate the economics of disability support on society.

Professor Ken Smith
The Lorenzo and Pamela Galli Chair in Medical Biology

Ken Smith

Professor Ken Smith is Director of the Walter and Eliza Hall Institute and Head of the Department of Medical Biology at the University of Melbourne.

He was appointed as Lorenzo and Pamela Galli Chair in Medical Biology at WEHI and the University of Melbourne in 2024, on his return to Australia from the United Kingdom, where he was Head of the Department of Medicine at Cambridge University from 2010 to 2024.

Professor Smith's research focuses on immune deficiency and autoimmune disease. His research sees him collaborating with precinct partners the University of Melbourne, Peter MacCallum Cancer Centre and the Royal Children’s Hospital and Murdoch Children’s Research Institute.

Professor Doug Hilton

Photo of Doug Hilton

Professor Doug Hilton is former Director of the Walter and Eliza Hall Institute, former Head of the Department of Medical Biology at the University of Melbourne, and a member of the Australian Medical Research Advisory Board (AMRAB).

In 2017, Professor Hilton was named the inaugural Lorenzo and Pamela Galli Chair in Medical Biology at WEHI and the University of Melbourne, a position he held until his departure to take up the role of Chief Executive of CSIRO in 2023.

He is best known for his discoveries in the area of cytokine signalling, his advocacy for health and medical research, and for gender equity in science. As head of the Molecular Medicine division at WEHI, Professor Hilton’s lab aims to understand which of the 30,000 genes are important in the production and function of blood cells, and how this information can be used to better prevent, diagnose and treat blood cell diseases such as leukaemia, arthritis and asthma.

Professor Hilton has been awarded numerous prizes for his research into how blood cells communicate and has led major collaborations with industry to translate his discoveries from the bench to the bedside. He is an inventor of more than 20 patent families, most of which have been licensed, and is a co-founder of the biotechnology company MuriGen.