Cystic Fibrosis affects 1 in 2,500 babies and mainly affects the lungs, but liver disease can add to poor health, with about a 1/3 of children developing serious liver disease. Current blood tests to identify them are unreliable and only work when liver disease is well advanced. Sampling a piece of liver, via a biopsy, ...
Harry Perkins Institute of Medical Research
Detecting liver disease early in children with cystic fibrosis
GOAL
$14,891
Australia > WA > Metro
08/01/2024 > 08/01/2026
Field of Interest
- Health/wellbeing and medical research
Target Population
- People with a disability, illness or disease
- Early years (0-5)
Harry Perkins Institute of Medical Research
The Harry Perkins Institute is a leading medical research institute in WA, pioneering scientific breakthroughs and collaborating with other world leading institutes to deliver improved health outcomes for the global community. Our key focus areas include cancer, cardiovascular disease, diabetes, and rare genetic diseases. By consolidating and expanding our fundamental discovery research in these fields, we aim to understand these complex conditions, find innovative solutions, and kinder treatments to defeat disease.
We have a bold vision to improve community health for Australian families. To give families in WA, and beyond, the opportunity to live longer, healthier, and better lives. We carry out life saving research, run clinical trials via our subsidiary, Linear, collaborate with institutions around the world to share our discoveries, and foster an environment for the brightest minds in WA to flourish.
Our world-class teams accelerate the delivery of life-saving breakthroughs to improve the health of all Australians both today and for our future generations, to help families stay together for longer.
Project Summary
Cystic Fibrosis affects 1 in 2,500 babies and mainly affects the lungs, but liver disease can add to poor health, with about a 1/3 of children developing serious liver disease. Current blood tests to identify them are unreliable and only work when liver disease is well advanced. Sampling a piece of liver, via a biopsy, is the best way to tell, but this is an invasive and complicated procedure, and is also expensive and not possible to do repeatedly.
This project aims to develop a new blood test that tells us whether liver stem cells are present. Stem cells grow in diseased liver to repair it. In other liver diseases we found that the amount of liver stem cells can tell us about the condition of the liver. If there are many, the liver is sick. A blood test that can identify what is known as a ‘biomarker’, would enable us to measure the amount of stem cells in the blood of children with cystic fibrosis and inform us of the conditions of the liver.
Laboratory equipment, including the ThermoMixer for heating and mixing samples, the Centrifuge for separating liquid mixtures with different densities to analyse samples, a rotor for the Centrifuge (which are the spinning parts that hold the samples) and key consumables used with the equipment, are vital to progress this project.
This blood test would be able to inform doctors which children are developing early liver disease and should undergo treatment, and which have a serious condition and need special care to avoid liver failure.
Project Outcomes
We aim to develop a blood test that correlates liver disease in children with cystic fibrosis (CF) in the early stages before irreversible cirrhosis is established.
CF-related liver disease is established usually in a person’s late-teens or 20’s.
Currently, it is only when cirrhosis or portal hypertension is established that clinicians can diagnose that a patient with CF has developed liver disease, however by that stage the cirrhosis is irreversible.
This fundamental deficiency in the ability to monitor for the initial stages of liver disease in CF patients has severely impacted the study of the progress of, and possible interventions for CF-related liver disease.
Children with CF are a sick and vulnerable population. Australian data in 2023 also confirmed CF patients have significantly lower tertiary education levels due to the frequency of hospitalisations, as well as a higher number of both respiratory and non-respiratory hospitalisations according to Australian data from 2020.
These children also suffer from a greater disease burden compared to CF patients without CF-related liver disease, increased nutritional interventions such as placement of a gastronomy tube and are at a higher risk of CF-related bone disease and diabetes.
Being able to identify early liver disease through a blood test in children with CF, before they develop irreversible cirrhosis, will allow us to evaluate the effectiveness of interventions to alleviate their disease burden, hospitalisation, and improve their quality of life, morbidity, and mortality for these already vulnerable children.
Budget Breakdown
TOTAL BUDGET: $14,891
FUNDING
| Funding source | Amount |
|---|---|
| Funding Gap (unconfirmed) | $14,891 |
EXPENSES
| Expense item | Amount |
|---|---|
| Eppendorf ThermoMixer® C for heating and mixing 96-well microplates Catalog No. 5382000066 (for Luminex assay development) | $5,066 |
| Centrifuge 5425 R refrigerated Catalog No. 5406000283 | $7,444 |
| Eppendorf™ Rotor for Centrifuge 5425 / 5425 R Catalog No.13-864-458 | $1,361 |
| 16-Tube SureBeads™ Magnetic Rack Catalog No. 1614916 Bio-Rad (for Luminex assay development) | $470 |
| Streptavidin–HRP High sensitivity ThermoFisher Catalog No. 21130 (for ELISA assay development) | $550 |
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