Our research

There are very few treatments for neurodegenerative brain conditions, including Alzheimer’s disease, providing very limited symptomatic relief without changing the course of the diseases. Hence, there is an unmet need for new disease-modifying therapeutic approaches.

Through deciphering novel disease mechanisms, we have established a portfolio of innovative strategies and novel drug targets for:

• Alzheimer’s disease (AD)
• amyotrophic lateral sclerosis (ALS)
• epilepsy
• frontotemporal dementia (FTD)
• motor neuron disease (MND)
• stroke
• traumatic brain injury (TBI).

From our programs arose already two biotech spin-out biotech companies, Celosia Therapeutics (2023) and SynaptraBIO (2025), to bring novel treatments for motor neuron disease and Alzheimer’s disease to people living with the diseases.

To succeed in our mission, multidisciplinary team of the Macquarie University Dementia Research Centre works closely Australian and international biotech and pharmaceutical industry to accelerate translation.

Group leaders: Professor Lars Ittner, Dr Ole Tietz, Dr Janet van Eersel

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Underpinning capabilities of The Macquarie University Dementia Research Centre is uniquely positions for the rapid translation of discoveries into treatments through its integrated structures of specialised design and synthesis or production of high-quality:

• efficient adeno-associated virus (AAV) gene therapy vectors
• next-generation therapeutic peptides
• small molecule drug candidates
• target-specific antibodies.

Group leader: Dr Ole Tietz, Professor Yazi Ke

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To treat dementia, it is vital to first understand the processes that lead to neuronal dysfunction and brain atrophy. This forms the central pillar of the Macquarie University Dementia Research Centre programs.

Our team has made fundamental discoveries that shed new light on the causes of dementia, including novel disease mechanisms of the hallmark proteins tau and TDP-43.

The current discovery research program strives to identify novel disease mechanisms and reveal new avenues to treat these devastating conditions.

To achieve this aim, our different discovery research programs investigate a broad range of molecular processes in normal brain function and disease, including the roles of:

• aberrant cell signalling
• compromised cytoskeleton dynamics
• contributions of neuron subtypes
• molecular mechanisms of disease spreading
• neuronal network dysfunction
• RNA regulation.

Group leaders: Professor Thomas Fath, Professor Yazi Ke, Professor Lars Ittner, Dr Janet van Eersel, Dr Annika van Hummel

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Reliable and accurate disease models form the basis of understanding complex processes that lead neurodegeneration. Furthermore, these models are a critical step in developing and testing novel disease-modifying treatment. The Macquarie University Dementia Research Centre operated core units to provide:

• computation models of disease mechanisms (enable high throughput testing of compounds and mechanisms)
• genetically modified organisms
• induced human neurons (enable translatable testing of human disease mechanisms).

Together these models build a critical resource to enable complex testing of executive brain functions, translatable testing of human disease mechanisms and comprehensive testing of novel therapeutic approaches.

Group leaders: Professor Thomas Fath, Professor Yazi Ke, Dr Janet van Eersel

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Neuropathology provides the unique opportunity to visualise the changes during neurodegeneration in the human brains. Specifically, how disease-relevant protein abnormalities and the cell types and brain regions are affected that cause neurodegeneration.

Investigating the selective regional and cellular vulnerability in these disorders, and associated clinicopathological relationships, is a main research focus.

We examine clinically and pathologically well-characterised human brain tissue from sporadic and genetic neurodegenerative disorders – sourced from national and international Brain Banks and Research Institutions – and transgenic models of dementia.

Group leader: Dr Shelley Forrest

Clinical translation is a core pillar of the Macquarie University Dementia Research Centre’s ecosystem. Through its partnership with the KaRa Institute of Neurological Diseases, the centre’s affiliated clinical trials provider, we support the design and delivery of ethically rigorous, patient-centred clinical studies.

KaRa Institute of Neurological Diseases enables the progression of promising therapies from laboratory research into human trials, bridging discovery science with real-world clinical application.

Group Leader: Associate Professor Katheryn Goozee