Current Research

2019 Research Projects

Prof Roger Chung Macquarie University $183,488 over 1 year
Preclinical evaluation of novel therapies for clearance of TDP-43 in amyotrophic lateral sclerosis

Professor Chung’s team recently identified mutations in a specific gene (CCNF) as the cause of amyotrophic lateral sclerosis (ALS) in a large Australian family. A number of different mutations in the CCNF gene were identified by their international collaborators, and more recently by other international research groups. CCNF encodes a component of the protein that is a central regulator of protein degradation within cells. Because abnormal accumulation and aggregation of a protein, called TDP-43, inside motor neurons is the key pathological hallmark of the disease, it is possible that defective CCNF might contribute to a common convergent mechanism that leads to the abnormal protein aggregation that causes ALS.

To explore this further, Professor Chung’s team have successfully undertaken further experiments and screening. The data generated from these experiments and screenings has provided compelling evidence.

NFMRI funding would be used towards a study that will provide strong pre-clinical evidence of efficacy for a proposed gene therapy.  This is essential data for advancing this innovation through commercial development.  This discovery is currently protected through a PCT that is due for conversion to National Phase in 2019, and potential commercial investors (pharma etc) that they have approached have indicated that positive indications in a pre-clinical mouse study are required before they can consider the innovation for investment.

Dr Clare Stirzaker
Garvan Institute of Medical Research
$141,834 over one year
Liquid biopsy monitoring for triple negative breast cancer: a novel epigenetic test

Dr Stirzaker and her team have performed the FIRST genome-wide profiling study on DNA methylation (epigenetics) in Triple Negative Breast Cancer (TNBC). Funding from NFMRI would be used to develop this TNBC-specific blood-based biomarker test, by providing access to the sensitive methylation assay that has been developed in the laboratory of Prof Trau and Dr Korbie at the University of Queensland. This assay is particularly important as it allows, for the first time, up to 50 methylation signatures to be tested on the same clinical sample in one test. In addition, the test employs next-generation sequencing which allows unprecedented sensitivity to be achieved, critical to accurately detect tumour methylation in a blood sample when circulating tumour DNA may comprise only 1% of the total circulating free DNA.

This project is supported in partnership with the generous funding from the NSW Community Foundation, the NSW Community Foundation – Nicholas and Phyllis Pinter Trust (both are managed by Equity Trustees) and NFMRI.

Dr Adam Taylor
Griffith University
$50,000 over 1.5 years
Liposome delivery of a chikungunya virus vaccine candidate: a solution to vaccine production bottlenecks

Dr Taylor has had several partnering discussions with industry around licensing or co-development of their live-attenuated chikungunya virus (CHIKV) vaccine candidate. This highlighted a single barrier for investment: production limits. The modifications that make the virus safe and effective for use as a vaccine, prevent rapid, large-scale production of the virus. It simply doesn’t replicate fast enough. In response to this feedback, they have developed an alternative vaccine delivery vehicle that removes the need for in vitro scale up, and therefore, removes the production limit. NFMRI funding will enable conduct of efficacy testing on the new formulation to confirm immune response and storage efficacy. This type of late pre-clinical research activity is not typically funded through NHMRC, but is critical to obtaining the required data to entice an industry partner, and consequently, bridge the ‘valley of death’. CHIKV is transmissible between animals and humans via a mosquito vector. As global temperatures are rising, the mosquito populations in South-East Asia and Queensland are migrating south and their prevalence in New South Wales is increasing.

This project is supported in partnership with the generous funding from the NSW Department of Primary industries and NFMRI.

 

Dr Steven Wise
Heart Research Institute
$95,022 over one year
Durable treatment of peripheral artery disease

Dr Wise is seeking support for an injectable treatment for peripheral artery disease. Peripheral artery disease has a significant impact on the health of humans, affecting over 2.3 million Australians and 200 million people globally. There is currently no lasting effective treatment, and thousands of cases result in amputations each year. This intervention has the potential to provide an urgently needed improved treatment option.

NFMRI funding provides support for the one-year research plan incorporating a rat model and rabbit model study that will demonstrate safety and efficacy in two established animal models of vessel injury and healing – key criteria for attracting future investment.

These two models will complete the optimisation and proof-of-concept stages for the technology (rat model), before going head-to-head with current clinical practice in arteries of increasing anatomical similarity to humans (rabbit). Together these studies will provide the necessary data package to enable investors to confidently drive the technology to the next stage of development and toward clinical translation.

 

A/Prof Joanne Macdonald
University of Sunshine Coast
$140,550 over 1.25 years
A rapid, sensitive and portable molecular genetic test for diagnosis of Malaria in blood

In a project previously funded by the Bill and Melinda Gates Foundation, A/Prof Macdonald and her team developed rapid assay technology for the detection of Malaria (Plasmodium falciparum) in mosquitoes. Support is now required to validate the rapid and sensitive Malaria test for detecting subclinical infection levels at a collaborating institute by testing it on human samples containing low levels of infection. These samples are uniquely available via a collaborator already performing human clinical trials for treatment of Malaria infections. If it can be demonstrated that the test has higher sensitivity and can detect subclinical parasite levels, then the test will be well positioned to attract funding and investment for development into both the clinical detection market, as well as the market for tests that can assist with community screening for eradication programs.

NFMRI funding will also help to determine the optimal manufacturing reagents to achieve the best possible sensitivity, specificity and reliability of testing kits, to provide further confidence for potential investors that our test can be reliably manufactured. The team will also expand the assay to detect other malaria strains such as P. vivax, which will extend the number of countries the tests can be employed in, as the relative prevalence of Plasmodium strains differs between countries.

This project is supported in partnership with the generous funding from the NSW Department of Primary industries and NFMRI.

2018 Research Projects

A/Prof Janet Davies Queensland University of Technology $99,953 from 2018 to 2019
Point of care diagnosis for hay fever and asthma; development and validation of rapid subtropical specific IgE tests

This project proposes to use allergen molecules of subtropical grass pollen for more specific tests and treatments to assist people allergic to grasses in subtropical regions. Approximately 15% of the Australian population suffers from allergic diseases and the devastating effect of such allergies was felt in the recent thunderstorm asthma event in Melbourne where over 12,000 people were affected and 9 reportedly died. After identifying and characterising all the key allergens of two major subtropical grass pollens and making headway in subtropical grass pollens research, A/Prof Davies is proposing to partner with Abionic SA, a Swiss company that has developed an instrument that quickly measures levels of sensitivity to allergens in doctors’ rooms, to investigate whether recombinant version of their pollen allergens are effective as a more specific and rapid point of care diagnostic test for grass pollen allergy in warmer regions of the world. NFMRI funding would help support optimal generation and purification of two quality assessed recombinant allergen components, as well as trialling these component on a new point of care diagnostic platform. The pre-commercial research will advance the innovations quickly for commercial uptake.

 

Prof Michael Good AO Griffith University $200,000 during 2018
Manufacture and evaluation of a chemically attenuated Plasmodium falciparum whole parasite blood-stage malaria vaccine

Prof Good’s team has developed and patented a novel approach to a malaria vaccine that in their published pre-clinical studies has shown long-lasting protection against different strains and species of the malaria parasite. This approach is based on the use of the entire parasite which is made non-infectious by treatment with a chemical agent. For the human malaria parasite (Plasmodium falciparum) vaccine manufacture involves in vitro culture of the malaria parasite followed by chemical treatment. This has since been administered as a single dose to eight volunteers who all developed strong cellular immune responses. Since the vaccine has been shown to be safe and well-tolerated, Prof Good’s team now plans to undertake a Phase Ib trial, which will involve 36 volunteers and test the efficacy of the vaccine. NFMRI funding would help support the vaccine manufacture for this trial. The associated clinical trial and activities are already funded via other sources, including Rotary.

 

A/Prof Philip Sutton Murdoch Childrens Research Institute $150,000 during 2018
Vaccinating against Helicobacter pylori-induced gastric cancer

A/Prof Sutton has invented a vaccine that can prevent gastritis in mice. His vaccine targets the enzyme produced by H.pylori, which opens up gaps in the normally tight, impermeable barrier of the stomach lining. A/Prof Sutton believes that by preventing disruption of this barrier, either before or after H.pylori infection, that they may completely prevent the development of gastritis. A/Prof Sutton wishes to test this vaccine in clinical trials, but to do so he needs to optimise the manufacturing process of the vaccine antigen in order to be able to produce the antigen in sufficient quantity and quality for taking into clinical trials. NFMRI funding will help support optimisation of the manufacturing process for the vaccine antigen. Luinabio, one of the most experienced company in Australia for producing recombinant vaccine antigens used in clinical trials would be contracted by A/Prof Sutton to carry out this work.

 

Dr Joanna Woodcock University of South Australia $50,002 during 2018
Preclinical evaluation of 14-3-3 protein inhibitors for lung cancer therapy

The cellular protein, 14-3-3, is abundant in many cancers including lung cancer and the increased amount of 14-3-3 protein above normal levels strongly equates to the severity of the cancer and poor patient survival. Importantly, in experimental systems, reduction in levels of 14-3-3 protein in lung cancer cells has been shown to block cancer cell growth and cause cell death. Therefore, 14-3-3 protein represents a promising ‘molecular target’ for the development of new anti-cancer treatment for lung cancer. Several other laboratories in the world have attempted to generate drugs to interfere with14-3-3, but without much success. Through Dr Woodcock’s research, they have found a novel way to inactivate 14-3-3 protein which already shows greater promise. Based on their knowledge of 14-3-3 protein structure and function, they have identified chemical compounds that selectively inactivate 14-3-3 and have shown that these compounds kill lung cancer cells and reduce lung cancer tumour growth in an animal model. They are currently evaluating these compounds in more relevant models of human lung cancer to assess the potential of our 14-3-3-targeting compounds as anti-cancer drugs for lung cancer. This project is supported thanks to generous funding from the NSW Community Foundation, the NSW Community Foundation – Nicholas and Phyllis Pinter Trust (both are managed by Equity Trustees) and NFMRI. Our combined funding will help support the assessment of pharmacokinetic properties of the drug compounds by scientists at CDCO and analysis of those compounds’ properties using RPPA. These studies will enable Dr Woodwock to fully assess the drug-like properties of those compounds and their potential efficacy for lung cancer.

 

Prof Eric Gowans The University of Adelaide $293,880 from 2016 to 2018
A DNA vaccine for Zika virus

The Gowans laboratory has developed a novel DNA vaccine that is more effective than canonical DNA vaccines and elicits robust immune responses in small (mice) and large (pigs) animals.  A vaccine for the Zika virus is urgently required because there is no therapy, and the link with microcephaly in children born to mothers who were infected during pregnancy demands that women of child-bearing potential be immunised.  As canonical vaccines (eg. live attenuated vaccines) require a considerable period of development, a DNA vaccine that can be generated in a matter of weeks represents an attractive alternative.

In this proposal, Prof. Gowans’ team examine the efficacy of novel DNA vaccines designed to elicit cell-mediated immunity to the Zika virus non-structural proteins or Zika neutralizing antibody in mice.  Thus, a major component of the project is to examine the immune responses in mice and pigs, and the protective efficacy of the vaccines in mice, with a view to identifying the most appropriate strategy to further develop for follow up studies in human clinical trials.

This project is supported thanks to generous support from the NSW Department of Primary Industries.

 

Prof Des Richardson The University of Sydney $105,500 (2017-2018)
‘Commercial translation of innovative null hepcidin analogues that prevent the anaemia of chronic disease (ACD)’

The anaemia of chronic disease (ACD) is a severe cause of morbidity and mortality in many millions of patients with cancer or inflammatory diseases and is due to excessive levels of the hormone hepcidin. These diseases induce excessive levels of hepcidin, which in turn promotes iron storage, thus preventing its release into the blood leading to severe and debilitating anaemia.

Prof Richardson has discovered that hepcidin is bound in the blood by a specific protein and has since developed an analogue that leads to urinary excretions of excessive hepcidin. NFMRI support will enable commercialisation of this optimal analogue.

 

Dr Nicholas Opie The University of Melbourne $390,000 (2017-2018)
‘Safety validation of the stent rode: a biomedical device for paralysis that converts thoughts into computer commands”

Dr Opie has developed a minimally invasive brain machine interface that has the potential to return mobility and independence to people with paralysis. His technology can record brain signals and convert them into useful commands that can be used to control computers, wheelchairs, exoskeletons and/or prosthetic limbs. Translation of existing brain machines is hampered by invasive surgical procedures, which require access the brain and lead to immune reactions that are causing device failure within months. Dr Opie and his team have already done enormous progress since receiving a $1.3m seed funding grant from the Defence Advanced Research Projects Agency – having already developed an implantable stent-electrode array that can record neural information from within a blood vessel, mitigating risks associated with open brain surgery.

NFMRI support will help them translate this research into clinical translation via the conduction of a world-first human trial in 2018. To meet this milestone, they must first manufacture the technology in an FDA approved and ISO certified facility and conduct the necessary preclinical experiments to demonstrate reliability, efficacy and safety.

 

A/Prof Michelle Hill QIMR Berghofer $169,204 (2017)
‘Blood glycoprotein panel for early detection of oesophageal cancer’

A/Prof Hill’s research aims to transform the detection and management of oesophageal adenocarcinoma (OAC) by developing a blood test. OAC is increasingly common due to growth of the major risk factors: chronic reflux and obesity. Although effective treatments are available for early OAC, outcomes remain poor because most cases are diagnosed at advanced stages due to the lack of practical and effective screening tools. A/Prof Hill has identified and patented a panel of readily translatable glycoprotein biomarkers, which can differentiate OAC from benign conditions and healthy controls. Her research program is in the process of evaluating these markers in large patient samples, as a step toward development of a diagnostic test that can be introduced into clinical practice.

NFMRI support will help develop one embodiment of the innovation, the clinical immunoassay. Working with industry partner Precision Antibody, we aim to generate monoclonal antibodies that recognise the three best biomarkers for use to generate immuno-assays. Successful completion will enable the development of a practical diagnostic test based on our biomarkers.

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Looking beyond the research and considering translational needs when funding research. How well are your expectations, application and review processes, measures of success and funding strategy aligned with the next steps for translation?

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