2016 PAHMR recipients
The Recipient of the 2016 Premier’s Award for Health & Medical Research
Dr Stephanie Simonds - Monash University
Cardiovascular diseases are the leading cause of death in Australia and globally. Excess body weight significantly increases the risk of cardiovascular disease development and it has been estimated that as much as 70 per cent of cardiovascular diseases can be explained by an increase in body fat.
Dr Stephanie Simonds’ research at the Biomedical Discovery Institute, Monash University, examined why cardiovascular diseases developed in obesity. Her research demonstrated that leptin, a hormone secreted into plasma by fat, is the cause of elevated blood pressure in obesity. Her research also demonstrated that obese children, deficient of either leptin or the leptin receptor, had lower systolic blood pressure compared to aged and Body Mass Index matched controls.
Leptin regulates body weight through communicating the degree of body fat to the brain, producing strong physiological changes in food intake and energy expenditure.
Dr Simonds’ research also demonstrated that increased blood pressure could be reduced through blockage of the leptin receptor. She was also able to identify a population of neurons that expressed the leptin receptors in the Dorso Medial Hypothalamic region of the brain, demonstrating that leptin activates these neurons and this was responsible for increasing hypertension in obesity.
Dr Simonds’ findings have furthered our understanding of how cardiovascular diseases develop in obesity and potential treatments.
Commendations were awarded to
In no particular order:
Dr Brian Liddicoat - St Vincent's Institute of Research
Aicardi-Goutieres Syndrome (AGS) is a rare genetic autoimmunity disorder that affects newborns and young children. This an extremely debilitating disorder for which there is currently no treatment. AGS sufferers have profound intellectual disability, painful skin lesions and an unfortunately short lifespan. Mutations in some patients with AGS cause a defect in an enzyme called ADAR1, which modifies the messenger molecules produced by our genes. In his doctoral research at the St Vincent’s Institute of Medical Research, Dr Brian Liddicoat discovered how faulty ADAR1 causes autoimmunity in normal cells in the same way it does in AGS.
He also found genes that we can target with drugs to dampen autoimmune responses in AGS, which has the potential to lead to the first treatment for AGS.
After this discovery, Dr Liddicoat was able to show that cancer cells also require normal ADAR1 activity to survive. This means that new drugs may be developed to block ADAR1 activity.
Dr Liddicoat’s research has also identified a therapeutic window to treat leukaemia and potentially other cancers.
Dr Julia Marchingo - Walter and Eliza Hall Institute of Medical Research
The immune response provides powerful protection against harmful infections. Recent trials have harnessed the power of the immune system to treat cancer with stunning success. Similarly, therapies that target immune cells have improved the health of patients with a range of autoimmune diseases.
These treatments function by manipulating the strength and combination of the positive and negative signals immune cells receive to reinvigorate the anticancer response or suppress the autoimmune response. Signal combinations are currently chosen using a largely empirical ‘trial and error’ approach. Therefore, strategies to rationally design immune therapies are needed for faster development of better treatments.
During her doctoral studies at the Walter and Eliza Hall Institute of Medical Research, Dr Julia Marchingo analysed how different stimulatory signals control the size of the immune response. She discovered that each individual signal could be added together and by using a surprisingly simple mathematical formula predicted the size of the immune response when signal strength or combinations were altered.
With further development this predictive framework has great promise to facilitate the rational design of immune therapies to improve treatments for patients with viral infections, cancer and autoimmune diseases.
Dr Thomas Oxley - The University of Melbourne
Brain machine interfaces are a new technology that enables brain activity to be recorded, and utilised for direct robotic control by thought. This technology promises to allow patients with previously untreatable conditions including spinal cord injury, the potential to manipulate mobility assist devices, including exoskeletons, with direct thought control. Dr Thomas Oxley’s doctoral research at the University of Melbourne and the Royal Melbourne Hospital culminated in the first scientific report of a brain machine interface that can be implanted into the brain without the requirement for open brain surgery. The technology, called a stentrode, is an electrode array fabricated onto a selfexpanding stent that is implanted via a minimally invasive procedure.
Dr Oxley’s doctoral research involved the invention of the device, it’s validation against more invasive devices, and demonstration of its recording sensitivity and bandwidth over a 6 month period of implantation. Over the course of the PhD candidature over $3.8 million dollars in funding was secured, a research laboratory established in the University of Melbourne, two patents were filed and a start-up company was formed. This technology may represent a paradigm-shift in the field of brain machine interfacing.
Dr Oxley and his colleagues are now working towards a first in-human clinical trial of this pioneering, minimally invasive brain machine interface technology to enable thought control of exoskeletons for patients with paralysis from spinal cord injury.