Personalised Therapeutics Group

The Personalised Therapeutics Group uses advanced stem cells and genomics technologies to model neurodegenerative diseases including amyotrophic lateral sclerosis (ALS), motor neurone disease (MND), Parkinson’s disease (PD) and dementia. Our research focuses on the ‘sporadic’ forms of disease that affect the vast majority of patients. These forms of disease have no known cause and we understand very little about why or how they occur.

Our laboratory uses stem cell (iPSC) technology to grow neurons in the laboratory genetically identical to the neurons inside the brain and spinal cord of the donor patient. This technology has allowed the study of sporadic forms of the disease for the first time, and provides the foundation for our large-scale modelling and drug screening efforts to identify new treatments for patients.

Research interests

Motor neurone disease
Amyotrophic lateral sclerosis
Parkinson’s disease
Dementia

Research programs

The FightMND Drug Screening and Precision Medicine Program (2017–2022) was an ambitious project in collaboration with the MND Group (Professor Bradley Turner) to develop a world-first platform to screen drugs directly on neurons grown from people with sporadic MND. We developed one of the world’s largest MND/ALS iPSC libraries (including over 100 sporadic donors) and established a dedicated state-of-the-art robotics platform for drug screening. Importantly, the program was the first to successfully recreate and validate sporadic MND/ALS in motor neurons grown in a laboratory. This breakthrough has enabled large-scale modelling and drug screening for MND for the first time.

With the unique ability to conduct large-scale modelling and drug screening for sporadic MND/ALS, our laboratory is leading multiple new initiatives to understand MND and identify new treatments.

MND iPSC clinical trials: Reassess all drugs previously tested in MND/ALS clinical trials for efficacy in sporadic MND patient motor neurons.
ALS functional genomics: Investigate the causes of sporadic MND/ALS.
Precision drug screening: Map the disease cascade in sporadic MND/ALS to identify and test new treatments.
Personalised medicine: Develop individualised testing for MND/ALS patients to predict optimal treatments.