Treatment Of Lysosomal Storage Disorder Patients By Drug-enhanced Premature Stop Codon Read-through
Funder
National Health and Medical Research Council
Funding Amount
$431,764.00
Summary
Lysosomal storage disorders are a devastating set of genetic diseases with very severe clinical symptoms. In this project, we will investigate a new treatment strategy that is non-invasive and that will be applicable for a wide range of lysosomal storage disorder patients. The therapy will over-ride the molecular genetic lesion and will be preferentially targeted for patients who are at the severe end of the clinical spectrum, where treatment options are currently limited.
Understanding Parent Behaviours That Promote Language In Slow-to-talk Toddlers
Funder
National Health and Medical Research Council
Funding Amount
$95,144.00
Summary
Research suggests that parent-child interactions can have a positive effect on the language development of preschool age children. This community-based study aims to identify which specific parent behaviours promote language in a group of 200 slow-to-talk toddlers. If specific parent behaviours are found to result in better language outcomes in slow-to-talk toddlers, the results will have the potential to promote far more pro-active management of early language delay.
New Antimalarial Drug Leads Targeting Multiple Species And Life Cycle Stages
Funder
National Health and Medical Research Council
Funding Amount
$818,477.00
Summary
Malaria causes ~200 million clinical cases and >430,000 deaths annually. Prevention and treatment relies on drugs, however malaria parasite drug resistance is an enormous problem. To address this issue, and aim towards eliminating malaria, we need to develop new drugs. This project addresses this important health need by investigating the ability of new chemical compounds, developed at CSIRO, to kill human-infecting malaria parasites during different parts of their complicated lifecycles.
Molecular Determinants Of Therapeutic Response In Colorectal Cancer
Funder
National Health and Medical Research Council
Funding Amount
$87,646.00
Summary
Colorectal cancer (CRC) is the second most common cause of cancer death. We will examine whether treatments of advanced CRC can be optimized by (1) selection based on the patient's expression of biomarkers (ie. cancer proteins) and-or (2) appropriate combinations of chemotherapy and-or immunotherapy. Using samples from patients we aim to determine predictors of response to a single therapy (bevacizumab) or a novel combination of therapies (erlotonib and cetuximab) to enhance future treatments.
Targeting Complement C5a Receptor 2 As A Disease-modifying Treatment For Motor Neuron Disease
Funder
National Health and Medical Research Council
Funding Amount
$636,329.00
Summary
Motor neuron disease (MND) is a devasting terminal condition that has no effective treatment. We have identified a novel drug which inhibits an immune protein that can potentially treat MND. In this project we will test this drug in rodent models of MND, and validate its effectiveness in relevant MND immune cells. Ultimately, this project will identify a new potential drug for MND.
Development Of Specific Inhibitors Of Porphyromonas Gingivalis Gingipains Based On Their Cognate Propeptides
Funder
National Health and Medical Research Council
Funding Amount
$612,655.00
Summary
Gum disease (periodontitis) is an inflammatory disease caused by bacterial pathogens that is the major cause of tooth loss in adults. It is also associated with systemic diseases such as cardiovascular disease. In this study we will develop novel peptide-based therapeutics to inhibit the proteases that enable these bacteria to cause disease.
Peritoneal Metastases From Colorectal Carcinoma: Exploring The Potential Of Immunotherapy As A Treatment Adjunct
Funder
National Health and Medical Research Council
Funding Amount
$89,197.00
Summary
Twenty percent of patients with bowel cancer have disease involving the lining of the abdomen, called the peritoneum. These patients do poorly. The majority are inoperable, and chemotherapy has poor response in these patients. Therefore, there is a dire need to explore new treatments. Newer drugs that stimulate the immune cells to fight cancer have shown promise in other cancers. We aim to assess the potential of this treatment in peritoneal disease, with the aim of improving patient outcomes.
Identification Of Novel Targeted Therapies For JAK2-driven Leukemogenesis
Funder
National Health and Medical Research Council
Funding Amount
$392,717.00
Summary
Many leukemias are caused by particular signalling molecules becoming too active in blood cells. My research focusses on the molecules that are required by leukemic cells for their growth and survival. I will use mice that are prone to developing leukemia to study how these leukemias can be treated with drugs that block specific molecules. My goal is to discover new ways to treat leukemias that work better and have fewer side effects than current treatments.
Determining The Molecular Basis Of Therapy Resistance Conferred By Genetic Lesions In The Tumour Protein TP53 In Haematological Malignancies
Funder
National Health and Medical Research Council
Funding Amount
$885,183.00
Summary
Blood cancers that have genetic lesions in a tumour suppressor protein called TP53 respond poorly to therapy. Curing these patients is extremely challenging and new therapeutic strategies are desperately needed. Here, we aim to uncover the molecular mechanisms of drug resistance caused by loss of TP53 function and rationally design new therapies that may be curative. To do this, our team of leading scientists and clinicians will study patient samples and pre-clinical models of blood cancer.