Development Of Therapeutic Copper Delivery Agents For Menkes Disease
Funder
National Health and Medical Research Council
Funding Amount
$651,467.00
Summary
Menkes disease does not currently have an effective treatment. The disease is caused by genetic defects that reduce copper transport into the brain and cause mental retardation and death. We have developed drugs that deliver copper into the brain and should cure Menkes disease. We aim to demonstrate that our drugs are effective in mice that have the same genetic defect as patients. Successful results will allow us to begin treating Menkes disease patients to determine if we can cure the disease.
Development Of Novel Gene Therapy Vectors For Thalassaemia
Funder
National Health and Medical Research Council
Funding Amount
$287,307.00
Summary
Thalassaemia, is a common inherited disorder affecting haemoglobin synthesis. Synthesis of ?/?-globin chain is balanced during normal red blood cell production. Any disruption in the ratio of ?/?-globin chain results in anaemia. In this study, we will explore gene therapy strategies to restore balanced ?:? globin expression and ultimately improve the severely anaemic phenotype in ?-thalassaemia patients.
Cachexia is a major side effect of cancer, resulting in significant muscle wasting, fat loss and organ failure. Up to 80% of cancer patients suffer and 25% succumb to this condition. This significantly affects the treatment regimens of cancer patients and affects their quality of life. We have developed monoclonal antibodies that block and reverse cachexia in preclinical mouse cancer models. Our aims are to humanise the antibody and manufacture it for the first clinical trial in humans.
We have discovered a single tumour factor which causes cancer cachexia, a wasting condition that is one of the worst complications of malignancy, for which there is no current effective treatment. We have developed antibodies which effectively block this condition in preclinical models and have produced human/humanised version of this. This application is to characterise these human antibodies to allow us proceed to clinical trials.
Peptide Therapeutics For The Treatment Of Autoimmune Diseases: Stability, Delivery And Disposition
Funder
National Health and Medical Research Council
Funding Amount
$368,467.00
Summary
Autoimmune diseases affect around 120 million people worldwide. This project will progress the development of a peptide that suppresses disease-causing autoantigen-specific immune responses without affecting protective responses. Different routes of delivery for this peptide will be evaluated, as well as slow-release formulations that will extend its in vivo lifetime. The outcome will be a patient-friendly form of this therapeutic lead that can be taken forward to preclinical evaluation.
A vitamin-sized capsule, containing gas sensors, is to be fully developed for assessing the state of health and diagnosing the diseases relevant to gastrointestinal tract. The capsule travels along the tract, transmitting information about the gas species generated by the microorganisms of the gut, which is closely associated with the health of the human under surveillance. The information will be invaluable for diagnostics and adjusting the diet to mitigate and cure the diseases of the guts.
Enhanced Deep Brain Stimulation Electrodes For Improved Efficacy In Parkinson’s Disease Therapy
Funder
National Health and Medical Research Council
Funding Amount
$809,940.00
Summary
We are developing an advanced Deep Brain Stimulation system to improve the treatment for Parkinson’s disease. Current devices benefit only half of the patients, due to imprecision in directing the electrical current to the ideal brain targets. Our system aims to deliver the current more selectively. Our implant will contain more and smaller electrodes. We will also use a state-of-the-art stimulator, implantable on the head, with capabilities far beyond the current technology.
Evaluation Of The Safety Of Lead Compounds For Allergic Asthma
Funder
National Health and Medical Research Council
Funding Amount
$310,568.00
Summary
Asthma is one of the most common chronic respiratory diseases in developed countries and is typically treated with corticosteroids which provide symptomatic relief and coarse non-specific treatment of the underlying disease. We are pursuing innovative therapies by targeting a different enzyme, HPGD2S, involved in the inflammatory mechanisms of asthma. We have developed potent, anti-inflammatory drug candidates and aim to profile the safety of these compounds before entering clinical studies.
Fighting Blindness With A Minimally Invasive Retinal Stimulator
Funder
National Health and Medical Research Council
Funding Amount
$998,194.00
Summary
Retinal degenerative conditions are the leading cause of blindness in developed nations, with over 200 million people afflicted worldwide. Our group has pioneered a minimally-invasive therapeutic stimulator that can arrest retinal degeneration without blocking vision. We are now ready to perform the prerequisite translational studies to develop and test a human-grade device. The ultimate goal is to be the first to develop a commercial therapeutic stimulator that protects against vision loss.
Development Of A Novel Endovascular Therapy For Occlusive Peripheral Vascular Disease
Funder
National Health and Medical Research Council
Funding Amount
$624,197.00
Summary
We aim to develop and assess a new medical device for the treatment of diseased arteries. The new device uses an angioplasty balloon coated with a new drug to open narrowed arteries and will enable the arteries to remain open for longer periods and with less likelihood of unwanted side effects from the procedure.