The Centre For Research In Childhood Early Respiratory Disease
Funder
National Health and Medical Research Council
Funding Amount
$2,621,023.00
Summary
The Centre for Research in Childhood Early Respiratory Disease is dedicated to the prevention of lung disease in young children with chronic respiratory disease. We aim to better understand the process of lung disease to identify predictors of disease and to treat it better. We will investigate the psychosocial effects of early interventions to better manage families. We will facilitate collaborations to provide best practice guidance and will train the next generation of doctors and researchers
Epithelial Drivers Of Neutrophil Plasticity In Early Cystic Fibrosis Lung Disease
Funder
National Health and Medical Research Council
Funding Amount
$849,462.00
Summary
Why airway inflammation becomes chronic so early in life for people with cystic fibrosis (CF) is unclear. This project will use the latest techniques to characterise immune cells found in airways of infants with CF and model in the laboratory how immune cells react to the CF airway. We will challenge CF airway cells with different bugs that can infect the lung, then see if the responses by CF airway cells can change the normal response of immune cells, triggering chronic disease.
The Identification Of Thoracic Targets For Prevention And Intervention In Bronchopulmonary Dysplasia
Funder
National Health and Medical Research Council
Funding Amount
$316,449.00
Summary
The persistence of breathing problems from infancy to later life is a complication of premature birth with lifelong consequences. Breathing problems often occur together with lung disease, but prematurity can also affect heart and blood vessel development, and weakness of the main breathing muscle. We will find out how much the heart, lungs and diaphragm contribute to breathing problems in babies; helping us to better predict, diagnose and treat severe breathing problems in babies born preterm.
Lung, Heart And Respiratory Muscle Disease After Preterm Birth
Funder
National Health and Medical Research Council
Funding Amount
$1,328,858.00
Summary
Breathing problems persisting into infancy and later life is an important complication of premature birth with lifelong consequences. Breathing problems often occur together with lung disease, but prematurity can also affect heart and blood vessel development, and weakness of the main breathing muscle (the diaphragm). We will find out how much the heart and diaphragm contribute to breathing problems in babies, and will help us to better predict, diagnose and treat severe breathing problems.
Clinical Trial Of Minimally-invasive Surfactant Therapy
Funder
National Health and Medical Research Council
Funding Amount
$1,172,978.00
Summary
This proposal is for funding of a clinical trial investigating a new and less-invasive technique of giving surfactant to improve lung function in preterm babies born between 25 and 28 weeks gestation. The trial will examine whether giving surfactant using the less invasive method results in better outcomes, including a reduction in time on respiratory support, and higher survival without chronic oxygen dependency.
RCT Of Headbox Oxygen Vs CPAP For Neonatal Respiratory Distress In Non-tertiary Hospitals
Funder
National Health and Medical Research Council
Funding Amount
$225,500.00
Summary
Each year in NSW hundreds of babies are transferred from local general hospital nurseries to a hospital with Neonatal Intensive Care Unit (NICU) because of breathing difficulties. Frequently, mothers are unable to accompany their babies at the time of transfer, leading to stress. When a mother is eventually transferred she is often separated from her partner and local supports causing further anxiety. In addition, if a baby has to be transferred, invasive procedures may be needed to ensure safet ....Each year in NSW hundreds of babies are transferred from local general hospital nurseries to a hospital with Neonatal Intensive Care Unit (NICU) because of breathing difficulties. Frequently, mothers are unable to accompany their babies at the time of transfer, leading to stress. When a mother is eventually transferred she is often separated from her partner and local supports causing further anxiety. In addition, if a baby has to be transferred, invasive procedures may be needed to ensure safety. Currently, babies who need oxygen in a local hospital are placed in a crib with a clear plastic box around their head and oxygen is run into the box (headbox oxygen). There is an alternative method of providing oxygen called CPAP (Continuous Positive Airway Pressure). This involves giving the oxygen directly into the baby's nose via soft rubber prongs. CPAP has been used since the 1970's. It is the main form of respiratory support for infants in many NICUs in Australia and the world. There is some evidence that death and lung disease may be reduced. However, the babies in these studies are sicker and smaller than babies in local hospital nurseries. CPAP is used in some local nurseries in New Zealand, however no studies have been done to see if CPAP reduces the need for inter-hospital transfer. The study will involve hospitals that have been selected because of their level of on site medical and nursing staff. These hospitals will have support and advice from two NICUs that use CPAP as their main form of respiratory support. Babies who need oxygen will be randomly allocated to either have headbox oxygen or CPAP. If the baby becomes so unwell such that certain preset criteria are met, the baby will be transferred to a NICU in the usual way. If CPAP safely reduces the need for inter-hospital transfer, many parents will be saved the anxiety associated with transfer, and the separation it often causes.Read moreRead less
Mucopolysaccharidoses (MPS) are a related group of 11 debilitating genetic disorders affecting children. They result from a reduction or total deficiency of an enzyme required for the removal of carbohydrate structures called glycosaminoglycans (gags). Gag degradation occurs inside the cell in specific organelles termed lysosomes and in the absence of the appropriate enzyme, undegraded gag accumulates in the cell. This leads to a range of clinical symptoms and multiple tissue failure. Symptoms c ....Mucopolysaccharidoses (MPS) are a related group of 11 debilitating genetic disorders affecting children. They result from a reduction or total deficiency of an enzyme required for the removal of carbohydrate structures called glycosaminoglycans (gags). Gag degradation occurs inside the cell in specific organelles termed lysosomes and in the absence of the appropriate enzyme, undegraded gag accumulates in the cell. This leads to a range of clinical symptoms and multiple tissue failure. Symptoms common to more than one MPS type include mental deterioration, blindness, abdominal organ enlargement and bone growth problems leading to short stature and bone loss. My laboratory has had a long-term interest in developing treatment for MPS and our research led to the clinical implementation of enzyme replacement therapy (ERT) for MPS VI in 2005. While providing the first effective, multi-tissue treatment for MPS, our research showed that several tissues were not responsive to ERT. These are the brain, cartilage and cornea, thus children on ERT regimens will still suffer from mental retardation, arthritis and blindness. With the goal of treating these particular tissues we have developed a new approach to MPS therapy called substrate deprivation therapy (SDT). Instead of adding back the missing enzyme, SDT acts by decreasing gag production which in turn reduces the level of accumulated gag in cells. SDT results in the correction of MPS cells in culture and reduces several key clinical symptoms in the mouse model of MPS IIIA. In this proposal we will extend our research to evaluate the effect of SDT on brain and bone-joint pathology. Evaluation of efficacy will take place in the MPS VII mouse which exhibits both brain and bone disease and in a new model of MPS IVA developed specifically for this study which exhibits a joint pathology unique amongst the MPS disorders.Read moreRead less
Skeletal disease is a major problem for children with mucopolysaccharidoses (MPS). Patients suffer from early onset osteoporosis and osteoarthritis, severely affecting their quality of life. We will evaluate a lentiviral gene therapy vector developed in-house for its capacity to transduce bone, cartilage, synovial and ligament cells in a mouse model of MPS VI. Our goal is to generate high level, sustained expression of the deficient MPS enzyme and alter the course of skeletal disease in MPS.