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Cystic Fibrosis Research Limited (CFRL) Innovation Grant 2022

Project Title: Improving detection and assessment of lung disease in young children with cystic fibrosis

Name: Tamara Blake

Institution: University of Queensland

Value:  $80,000

Duration: 1 year

Summary:  In Australia, one in 2,500 babies are born with cystic fibrosis (CF). lrreversible damage to their lungs begins very early in life and often without any symptoms. A significant limitation to early disease detection in patients with CF has been the lack of safe and sensitive measures suitable for use in infants and toddlers. Currently, the only methods available to monitor lung changes is performed under sedation in specialised laboratories. This is often a traumatic experience for patients and their families. We propose to assess the appropriateness of a new, innovative technique that can easily and reliably measure lung function in very young children. Oscillometry can be performed during quiet sleep and is very sensitive to early changes in the CF lung. Results from this pilot study will inform future study designs that will aim to better understand the mechanisms of early CF lung disease and better define the role of new sensitive measures that can monitor disease progression. Use of this technique in this population will also provide parents and physicians with detailed lung health information much sooner than current methods.

Reports: None due
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Abbie Fennessy Memorial Fellowship 2022 (sponsored by Mediplast)

Project Title: Investigations into the associations between exercise, physical activity and sedentary behaviour with glycaemic control in adults with cystic fibrosis after initiation of Trikafta TM.

Name: Tiffany Dwyer

Institution: University of Sydney

Value:  $5,000

Duration: 1 year

Summary:  The aim of the grant is to perform continuous glucose monitoring (CGM) during supervised moderate intensity exercise and unsupervised habitual physical activity, to determine the prevalence of hypoglycaemia and glucose variability in adults with CF after they are established on TrikaftaTM. Comparisons will be made between participants with normal glucose tolerance (NGT), impaired glucose tolerance (IGT) and CFRD. 

Reports: None due
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Cystic Fibrosis Lung Health Grant 2022

Project Title: Does the MetaNeb®, a new airway clearance device, change measure of lung oxygenation, secretion clearance and other measure of lung function in adults with CF when they are well?

Name: Naomi Chapman

Institution: Curtin University, WA

Value:  $5,000

Duration: 1 year

Summary:  The aim of this study is to determine whether in adults with stable CF, a single treatment session using the MetaNeb® produces greater change in measures of secretion clearance and lung oxygenation using fMRI and other measures of lung function compared to normal huff and cough.  We hypothesise that the treatment session that includes the use of the MetaNeb® device will produce greater changes in these measures, when compared with the treatment session that comprises huff and cough alone. 

Reports: None due
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The Dorothy Nell Marzol Innovation Grant 2022

Project Title: Australian-wide surveillance for fungal infection: A national metagenomic analysis across 19 CF centres

Name: Steven Taylor

Institution: South Australian Health & Medical Research Institute

Value:  $80,000

Duration: 1 year

Summary:  Lung infections associated with cystic fibrosis (CF) are devastating. They contribute to both everyday breathlessness as well as the acute lung events. The microbes that cause these lung infections are under constant pressure to adapt and change to the treatments that doctors prescribe to try and eradicate them. Over the last few years, several revolutionary changes have been made to how people with CF are treated. We are already seeing that the main bacteria associated with CF lung infections are declining because of these therapies. However, previously when we see changes to the microbes that cause lung infections, we see emergence of new microbes. This poses great risks as treatment plans for these new microbes are less established. My research will investigate the lung “microbiome” of over 900 samples already collected from people with CF across Australia. This will include looking at the fungi in the lungs, which are often overlooked, but which are known to become more common following changes to treatment. By looking at samples collected before and after starting new treatments, we can identify key microbes to monitor, giving us the best chance to protect people with CF from new infections. 

Reports: None due
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ACFRT Top Up Scholarship 2021

Project Title: The impact of helpful and harmful immune responses in the CF lung

Name: Emma Ledger

Institution: University of Queensland Diamantina Institute

Value:  $15,000

Duration: 3 years

Summary:  Pseudomonas aeruginosa is a highly multi-drug resistant bacteria which causes chronic lung infections in individuals with cystic fibrosis (CF). This bacterium is extremely difficult to eradicate and is a major cause of morbidity and mortality in CF. Our research investigates specific antibodies produced by patients that enhance, rather than clear these bacterial infections resulting in further disease burden.  This project aims to better understand the prevalence, impact, and mechanisms of these ‘bad antibodies’ in individuals with CF by characterising them for the first time within patient sputum and towards the patient’s infecting strain. Additionally, we aim to investigate the impact the latest CF transmembrane conductance regulator (CFTR) modulator therapies have on P. aeruginosa infection and these dysregulated antibody responses. This research is critical for the development of targeted therapies to neutralise these ‘bad antibodies’, clear persistent multi-drug resistant lung infections and improve patient outcomes further in this post-modulator era. 

Reports: None due
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CFWA Golf Classic Grant 2021

Project Title: Neutrophils in CF

Name: Luke Garratt

Institution: Telethon Kids Institute/University of Western Australia

Value:  $28,000

Duration: 1 year

Summary:  In cystic fibrosis (CF), neutrophils accumulate in areas of the lung as mucus builds up and germs infect the airways. My research has found that in CF airway inflammation, neutrophils choose to actively release their harmful products rather than trying to eat and kill the germs causing infection. In a laboratory model of CF airway inflammation, we have found that only certain germs induce this behaviour. We are now studying how the gene expression of neutrophils determines their function, with the aim to understand whether we can prevent certain functions by inducing or repressing genes within the neutrophil. This CFWA funded project is exploring the ability of new nanoparticle technologies to modify gene expression in neutrophils, as neutrophils are short-lived cells that do not respond to traditional methods. The hope is that this technology could inform a new class of anti-inflammatories for controlling neutrophil behaviour in CF airways.

Reports: None due
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The Cystic Fibrosis Metabolic Monitoring Grant 2021

(sponsored by COSMED Asia-Pacific Pty Ltd)

Project Title: The road map to personalised diet management in children with cystic fibrosis

Name: Hiran Selvadurai and Andrea Kench

Institution: The Children's Hospital, Westmead

Value:  $5,000

Duration: 1 year

Summary:  We  know that people with cystic fibrosis (CF) need more energy from food to grow and fight infections than healthy people. However, there is very little information available on specific macronutrient (carbohydrate, protein and fat) requirements for children with CF. In particular, it is not known how macronutrients contribute towards fueling the body during periods of rest and during exercise. This information is needed so that we can implement personalised nutritional goals. This is particularly important in the context of new CF treatments (modulator medications) which have led to a significant improvement in lung function, weight and quality of life. Further, significant infections such as NTM is a growing problem in patients with CF but it is not clear how these infections impact on macronutrient use and energy expenditure in children with CF. Exciting new technologies now enable us to quantify nutritional macronutrient requirements for children. The purpose of this research study is to ascertain if we can determine macronutrient requirements for children with CF.  

Reports: None due
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Ann Maree Bosch Career Fellowship 2021

Project Title: Progressing novel treatment options to improve management of infections in people with cystic fibrosis

Name: Stefanie Bader

Institution: Walter & Eliza Hall Institute of Medical Research

Value:  $10,000

Duration: 1 year

Summary: Bacterial infection is the most common cause of death in people with cystic fibrosis (CF). The immune cells responsible for combating bacteria are often defective in CF patients and can damage the lungs and gut while trying to fight infection. This reaction worsens disease and fails to kill the pathogen. We propose to use a new class of drugs that can tackle this problem in two ways: by killing infected host cells, reducing the number of bacteria and by removing defective immune cells. This approach has the potential to improve quality and length of life in people with CF by addressing disease in the lung and the gut.  The Fellowship would provide an invaluable opportunity for me to travel to a lab in the Netherlands which specialises in CF and the gut. There, I would be able to test our drugs both in in-vivo and organoid models of CF gut disease to gain substantial knowledge on how to apply their techniques to our research at WEHI. This would allow my lab to test a greater array of drugs in Australia. I will share the techniques I learn, not just with my lab at WEHI, but with other CF researchers in Australia. 

Reports: Six month report

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Conquer CF (Respiratory) Innovation Grant 2021

Project Title: Establishing a pipeline for repurposing anti-inflammatory drugs to tackle viral-induced exacerbations in children with cystic fibrois.

Name: Samuel Montgomery

Institution: Curtin University/Telethon Kids Institute

Value:  $50,000

Duration: 1 year

Summary:  This study aims to improve the lung health of young children with cystic fibrosis (CF) following a viral infection. Rhinovirus infection (the “cold” virus) is common in young children, often resulting in presentation to hospital emergency departments. This is worsened in children with chronic airway disease such as the inheritable disease cystic fibrosis (CF) where infection with rhinovirus results in deteriorating lung health, sadly often permanently, leading to chronic bacterial infection and increased hospitalisation. Rhinovirus infects the cells lining the airway (the “epithelium”) provoking a large inflammatory response, which can lead to lung damage. We have previously identified a specific type of inflammation which is associated with decreasing lung health in children without bacterial infection. We propose to block this inflammation using a drug, interleukin-1 receptor antagonist, as a new anti-inflammatory treatment for the cold virus in children with CF. We will use a model of the airway in the laboratory to test the safety and effectiveness of this drug to reduce this inflammation. This study aims to provide evidence to progress to a clinical trial, as this drug is already approved for use in other diseases and could be rapidly translated into clinical use for children with CF. 

Reports: None due
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CFWA Golf Committee Contribution to CF Research 2021

Project Title: Does a diagnosis of Tracheobronchomalacia affect health outcomes in children with cystic fibrosis?

Name: Julie Depiazzi and Crystal Bourke

Institution: Perth Children's Hospital

Value:  $5,000

Duration: 1 year

Summary:  Around 41% of young children with cystic fibrosis (CF) in Western Australia have been reported to have tracheobronchomalacia (TBM) seen during bronchoscopy – a condition where parts of the windpipe or airways are floppier than would be considered normal. In the short-term this TBM finding can lead to a change of the child’s daily physiotherapy and airway clearance program, however despite its prevalence, little is known about the long-term impact of TBM. 

The aim of this study is to compare health outcomes of children with CF, both with and without TBM, up to the age of 4 years, and will include imaging, inflammation markers and infection comparisons. We hope our findings will provide clinicians and families with more insight into, if any, long-term impacts of this condition.

Reports: None due
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Conquer CF (GI) Innovation Grant 2021 

Project Title: Short Chain Fatty Acids and Gastrointestinal complications in cystic fibrosis

Name: Josie van Dorst

Institution: University of New South Wales

Value:  $50,000

Duration: 1 year

Summary: Cystic fibrosis (CF) is a life-shortening genetic condition, which causes thick mucus, chronic infection, and inflammation not only in the lungs but also the gut. People with CF also have an imbalance between beneficial and harmful gut bacteria, which are linked to poor growth, gut symptoms, chronic gut inflammation and increased cancer risk. Short chain fatty acids (SCFA) are produced by beneficial bacteria in the gut, mainly from prebiotic dietary fibres. SCFAs fuel the activities of beneficial bacteria and have many important roles in the gut and wider health, including combating inflammation and cancer activity, and regulating the communication with the lungs (gut-lung axis) and the immune system. Despite this, there is limited knowledge regarding SCFA in individuals with CF.

Reports: None due
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Abbie Fennessy Memorial Fellowship 2021 (sponsored by Mediplast)

Project Title: The Early Childhood Project: Circle of Security-Parenting in cystic fibrosis (CF)

Name: Stella Li

Institution: The Children's Hospital, Westmead

Value: $5,000

Duration: 1 year

Summary:  This project focuses on supporting parents of children with CF during the early childhood period (under 5). This is a period that often coincides with CF diagnosis and can be filled with stress, worry, and guilt. We are offering the Circle of Security parenting program which aims to help parents reflect on and meet their child's medical and emotional needs and ultimately strengthening the parent-child relationship.

Reports:  none due
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CFWA Top Up Scholarship 2021

Project Title: Studying how pseudomonias aeruginosa becomes resistant to phage therapy to identify how to prevent it occuring

Name: Andrew Vaitekenas

Institution: Curtin University

Value:  $37,500

Duration: 3 years

Summary:   Psuedomonas aeruginosa causes harmful lung infections in CF lungs and becomes resistant to existing antibiotic treatments. There is a lack of antibiotics being developed and alternative treatments are needed. A therapy using viruses that specifically kill bacteria is effective, but P. aeruginosa can become resistant to this too. My project aims to understand how P. aeruginosa becomes resistant to phages and what this means for the bacteria. I will use this knowledge to develop and test strategies that increase the effectiveness of phage therapy and prevent resistance occurring. These strategies can aid our group in making phage therapy a regularly used therapy, preventing further resistance and treating those with infections that are currently untreatable.

Reports: None due
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Special CF Research Seeding Grant 2021 (sponsored by CFACT)

Project Title: Development of new mucus-thinning drugs for CF patients

Name: Shiyi Xi

Institution: The Walter & Eliza Hall Institute of Medical Research

Value:  $5,000

Duration: 1 year

Summary:   Mucus-based airway obstructions and the complications they cause are a significant source of morbidity and mortality in CF patients. The current leading mucolytic drug Pulmozyme® only works on 70% of patients, and many people would greatly benefit from more effective alternatives. This project will focus on a promising new target for novel mucolytic drugs - a mucus-thickening protein called trefoil factor-3 (TFF3). My lab has generated and identified 15 candidate antibodies that antagonize TFF3 activity and will be evaluated on patient sputum samples. We aim to benchmark the activity of these potential mucolytics against Pulmozyme® and to assess if they have additive of synergistic activities with the current drugs.

Reports: 
Six month report
Final report


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ACFRT Studentship Grant 2021 (sponsored by CFRL)

Project Title: Investigation of factors influencing glucose control in cystic fibrosis 

Name: Rebecca Keating

Institution: University of Queensland

Value:  $15,000

Duration: 3 years

Summary:   This research project will aim to determine whether the use of DPP4 inhibitors assist in removing blood glucose levels for the entire day in individuals with CF impaired glucose tolerance. It will assess its effects on gut hormoneas, gut microbacteria and inflammation. It will also determine whether using a mixed meal tolerance test may be more useful in determining CF impaired glucose tolerance.

Reports: None due
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Lung Health Grant 2021 (sponsored by FBM)

Project Title: Telehealth to promote mucociliiary clearance in adolescents with cystic fibrosis

Name: Anna Middleton

Institution: The Children's Hospital, Westmead NSW

Value:  $5,000

Duration: 1 year

Summary:   CF adolescents with poor adherence to airway-clearance and exercise will be randomised to a 4-week block of either stationary airway-clearance or the same routine interspersed with short intervals of exercise. Sessions will be supervised by a physiotherapist over telehealth twice a week. After a 2-week wash-out period participants will cross-over to the alternative intervention. Adherence, ease of expectoration, spirometry and exercise perceptions will be measured after each block of therapy. We hope to evaluate whether telehealth in CF adolescents improves airway-clearance technique and adherence and whether the addition of short intervals of exercise interspersed during airway-clearance improves mucociliary-clearance.

Reports: None due
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Innovation Grant 2021 

Project Title: Safety and effectiveness of triple-caftor combinations in cystic fibrosis during pregnancy

Name: Elena Schneider-Futschik

Institution: University of Melbourne

Value:  $80,000

Duration: 1 year

Summary:   The recent approval of the novel CFTR modulator triple combination Trikafta targets the majority of cystic fibrosis (CF) patients, but potentially excludes pregnant women as the clinical efficacy of these important drugs is limited by our lack of understanding what short and long-term effects these drugs could have on developing babies. This project will investigate the entry of the triple combination across the placenta in an animal CF model that replicates a common CF mutation. The project will also assess the potential of maternal administration of these drugs to reduce the amount of CF damage occurring before birth. This innovative approach has significant potential in improving clinical practice worldwide

Reports: None due
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Innovation Grant 2021 (sponsored by CFSA) 

Project Title: Sphingosine-1 phosphate and zinc - novel mediators of vascular dysfunction in children with CF

Name: Andrew Tai

Institution: The University of Adelaide

Value:  $50,000

Duration: 1 year

Summary:   We continue to collect lung and blood samples from children with CF and controls. There was a delay with bronchoscopy procedures for a few months during the recent COVID surge. Samples will be tested in parallel when sufficient numbers are obtained. We have optimised all techniques, completed testing of markers of vascular dysfunction in blood from adult CF patients (as comparators for the paediatric bloods), and are undertaking mechanistic in vitro studies focusing on changes in the sphingosine signalling pathway and markers of endothelial functions in cell lines. Significant changes to date have included an activation of the ET-1/TGFb/pSmad axis in adult CF patients; data that will advise our investigations using the paediatric CF samples. In a mechanistic approach the effects of the CFTR inhibitor CFTRinh172, and CFTR correctors (Ivacaftor and Elexacaftor) are used for testing whether these changes are caused intrinsically by CFTR impairment in the patients cells.  

Reports:   None due

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Conquer CF (Respiratory) Innovation Grant 2020

Project Title: Designing optimal phage cocktails for kids with cystic fibrosis (DOCK-CF)

Name: Ameneh Khatami

Institution: University of Sydney

Value:  $50,000

Duration: 1 year

Summary:   To manage chest infections, children with cystic fibrosis (CF) are exposed to broad-spectrum, toxic antibiotics several times per year. This is only moderately effective, with infections often becoming increasingly difficult to treat due to evolving antimicrobial resistance. Bacteriophages (phages) are viruses that replicate within bacteria, causing highly selective bacterial killing. Since phages do not attack human cells they are safe for clinical use, and their targeted bacterial killing reduces their impact on our healthy microbiome (commensal bacteria). Thus, phage therapy offers a safer and more precise solution than antibiotics.We hypothesise that Pseudomonas aeruginosa and Staphylococcus aureus strains that colonise children with CF can be eradicated effectively by combinations of phages (“phage cocktails”). This would result in reduced lung damage and allow recovery of normal lung growth, improving the life expectancy of children with CF. In addition, reduced toxicity and microbiome effects compared to antibiotics would improve quality of life for patients and their families. This proposal aims to identify phages with ‘killing’ activity against the main strains of these bacteria isolated from a large cohort of children with CF.  Research outcomes will feed into a translational pipeline to investigate this promising therapy in children with CF within 5 years.

Reports: Interim Report

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Golf PhD Top Up Scholarship 2020 (sponsored by CFWA)

Project Title: Me, myself and I: An exploratory study of self-concept in adults with Cystic Fibrosis (CF) in an evolving era of care.

Name: Maggie Harrigan

Institution: University of Western Australia

Value:  $37,500

Duration: 3  years

Summary:   The PhD study explores self-concept (sometimes called self-identity) e.g. how you view yourself, a sense of who you are as a person in the various aspects of life. Outside CF, self-concept has been widely associated with improved health, however there is currently a lack of self-concept research within CF. I will be examining levels of self-concept within the adult CF community and associations between self-concept, mental, social and physical health. The study will also explore the potential impact of evolving CFTR modulator treatments on self-concept.  As CF treatments evolve, what is means to have CF continues to change, potentially bringing new opportunities and challenges.  Amidst this change it is pertinent to develop our understanding of the social and emotional needs of those living with CF.  

Living with cystic fibrosis during the COVID-19 pandemic: a social connectedness perspective

 

Reports:  none due

Golf PhD Top Up Scholarship 2020 (sponsored by CFWA)

Project Title: Investigating an alternative therapeutic agent for the treatment for bacterial infections in kids with cystic fibrosis

Name: Joshua Iszatt

Institution: Curtin University

Value:  $37,500

Duration: 3  years

Summary:   This project is focused around the development and investigation of a therapeutic agent that could be used to treat multi-drug resistant bacteria that frequently cause persistent lung infections in children with cystic fibrosis.

Reports:  none due

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Ann Maree Bosch Fellowship 2020

Project Title: The Gut-Lung Axis in early cystic fibrosis (GLAX-CF)

Name: Katherine Frayman

Institution: Murdoch Children's Research Institute

Value:  $9,500

Duration: 1 year

Summary:  My research explores the development of the gastrointestinal and lower airway microbiota during the first two years of life in newly diagnosed infants with cystic fibrosis, and their relationship to each other, to lower airway inflammation and to clinical manifestations of disease. It will examine the impact of continuous antibiotic prophylaxis on the microbial milieu in both niches, and its relationship to clinically relevant outcomes. Ultimately, my research aims to highlight opportunities for intervention, for example, modification of the gastrointestinal microbiota with pro-, pre- or antibiotics, in order to influence respiratory phenotype. The Ann Maree Bosch Career Fellowship will enable me to travel to the United States to establish key collaborations with the University of Washington and Seattle Children’s Hospital, Washington and attend the North American Cystic Fibrosis Conference.

Reports:  None due 

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Conquer CF (GI) Innovation Grant 2020

Project Title: Towards understanding gut host-microbe interactions and personalised probiotic therapy in cystic fibrosis using organoid-derived 2D intestinal models

Name: Keith Ooi

Institution: University of New South Wales

Value:  $50,000

Duration: 1 year

Summary: Cystic fibrosis (CF) is a life-shortening genetic condition, which causes thick mucus, chronic infection, and inflammation in the lungs and gastrointestinal (GI) tract. People with CF have an imbalance in their GI bacterial population (microbiome) known as “dysbiosis”, and chronic GI inflammation, both of which are linked to poor growth, GI symptoms and increased cancer risk. A survey of CF patients and clinicians globally has ranked GI symptoms 2nd in priority, ahead of respiratory issues, yet no GI-specific therapies currently exist. Stem cells obtained from gut biopsies from people with CF can be used to grow 2-dimensional and 3-dimensional organoid culture systems which replicate real life (“mini guts”). We will investigate the host-microbe interactions involved in GI dysbiosis, inflammation and malignancy, using CF patient-specific intestinal organoids. We believe this study will provide a crucial step towards personalised therapies based on host-microbe interactions with the aim of reducing GI complications in CF. Successful personalised therapies would revolutionise CF treatments and other conditions subject to host-microbe interactions. 

Reports: 6 month report

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Abbie Fennessy Memorial Fellowship 2020

 (supported by Technipro-Pulmomed Pty Ltd & Cystic Fibrosis Australia)

Project Title: CF Physio.com:  development of interactive and education videos of inhalation therapy and airway clearance

Name: Jen Hauser & Jenny Bishop

Institution: Royal Hobart Hospital & Westmead Hospital

Value: $5,000

Duration: 1 year

Summary:  CFPhysio.com is an educational website which currently translates the TSANZ clinical practice guidelines “Physiotherapy for CF”, and evidence based educational literature into a practical, self-paced, educational resource for physiotherapists. The website currently provides a platform for education and learning of evidence-based physiotherapy management in cystic fibrosis, for physiotherapists. Since its official launch in August 2019, over 400 physiotherapists have registered and used the site. A second stage of the project is proposed, including the provision of educational resources for individuals with CF, carers, family, friends, employers, teachers etc, including video content of inhalation therapy and physiotherapy treatment techniques. 

Reports:  Final Report

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Innovation Grant 2020

Project Title: Enhancing the innate ability of CF macrophages to kill and clear MABS

Name: Abdullah Tarique

Institution: University of Queensland

Value: $80,000

Duration: 1 Year

Summary: Currently 13-20% of patients with CF are infected with Mycobacterium abscessus (MABS) and there is growing concern that increasing incidence of MABS infection is leading to accelerated pulmonary decline, and to reduced survival. MABS are notoriously difficult to treat, in part because of widespread antibiotic resistance, thus necessitating the use of toxic drug regimens. We herein propose to evaluate an alternative approach to clear MABS by boosting the anti-bacterial responses of CF patients using novel therapeutic agents. Macrophages are the professional killer cells that kill bacteria including MABS from the lungs. Sputum and venous blood are the two main sources for macrophage researchers. Due to inadequate volume of patient’s specimen available for research, macrophage researchers struggle in getting enough macrophages from patients’ specimen which thereby limit macrophage research in CF. We herein propose to develop an immortalized CF macrophage cell line that will offer indefinite supply of CF macrophages to the researchers to investigate macrophage defect in CF as well as finding out ways to improve their anti-microbial responses in CF.

Reports: none due

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Ann Maree Bosch Career Fellowship 2019

Project Title: Targeting a forgotten cell to prevent cystic fibrosis lung disease

Name: Shivanthan Shanthikumar 

Institution: Murdoch Children's Research Institute

Value: $9,000

Duration: 1 year

Summary:  The life expectancy for someone born with cystic fibrosis (CF) today is only 44 years. In CF the major cause of shortened life expectancy, as well as reduced quality of life, is lung disease. A major cause of lung disease in CF is excessive inflammation which causes to irreversible lung damage. Currently a number of treatments are used in CF such as antibiotics and chest physiotherapy, however no treatments target inflammation. There are new treatments which address the malfunctioning ion channel which causes CF, however these treatments only target specific certain genetic mutations, and so not all patients benefit from them. Any treatments aimed at inflammation in CF would benefit all patients, and could be used together with existing and new treatments. There are a number of different cell types that contribute to inflammation, however one that has not been investigated is the alveolar macrophage. These cells only exist in the lung and are one of the initial cells that trigger an inflammatory response. There are multiple types of alveolar macrophages in CF lungs, and thus far they have not been studied. The overall aim of my research is to identify targets for treatment relating to alveolar macrophages.

Reports: Final Report

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David Millar Giles Innovation Grant 2019

Project Title:  An Australian Alliance of personalised lab grown mini-organs to save the rarest of them all

Name: Shafagh Waters

Institution: University of New South Wales

Value: $45,000

Duration: 1 Year

Summary: Some individuals with rare CFTR mutations have been shown to benefit from the available modulator therapies. Sadly, most will have no opportunity to access these breakthrough treatments. Currently no clinical test exists to predict patient’s response to a modulator drug. We are using a new technology in the field of personalised medicine. Stem cell derived mini-organs are generated from small biopsies serving as a personal CF model or an AVATAR. They are tested in the lab to predict an individual CF patients’ responses to therapeutic agents. Should one or more therapies prove effective in the lab, these can be recommended for use as targeted therapies for the patient.

In the last 2 years we have created AVATARs from children with CF that visit the Sydney Children’s Hospital and have predicted outcome of modulator therapies. In this application we propose to extend our platform to create an Australian-wide alliance for people with rare CF by extending to 11 CF clinics (6 paediatric and 5 adult). This project will provide a novel therapeutic opportunity, ultimately enabling ‘managed’ off-label access to the CFTR modulator therapies for individuals with rare CFTR mutations who show response to the therapy in a prospective mini-organ test.

Reports: Final Report

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Innovation Grant 2019

Project Title: Optimising patient-derived stem cell technology in cystic fibrosis to predict CFTR modulator response

Name: Gerard Kaiko

Institution: University of Newcastle and Hunter Medical Research Institute (Newcastle and Northern NSW)

Value: $80,000

Duration: 1 Year

Summary: CFTR modulator therapy offers great hope to improve the quality of life of cystic fibrosis (CF) patients with an array of different mutations. However, the high-cost, limitations of efficacy, and restrictions of therapies to clinical trial targeted mutations has created a powerful need for a lab test to better predict which CFTR mutations and which individual patients will respond to a given therapy. This type of precision-medicine is already critical but will only become more vital in the future as CFTR modulator therapy options increase. Stem cell technology has enabled patient cells to be readily grown in the laboratory. The use of ‘mini-guts’, grown from a CF patient intestinal biopsy, is being used in the Netherlands to suggest whether a given CFTR mutation may respond to a given therapy. To enable this type of testing to be put into practice in Australia there are two major questions that we aim to address in this project. Firstly, what is the optimal stem cell technology and functional test to apply to patient cells to determine whether they will respond to therapy. Secondly, can we predict a patient’s response to CFTR therapy in the clinic by testing the drugs on their stem cells.

Reports: 6 month report, Final Report

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Innovation Grant 2019 CFCC Victoria

Project Title: Colorectal Cancer

Name: Keith Ooi

Institution: University of New South Wales

Value: $50,000

Duration: 1 Year

Summary: Adults with cystic fibrosis (CF) now face new complications such as colorectal cancer (CRC). The cause is unknown but gut inflammation and gut bacteria imbalances are possible causes. The risk for CRC in CF is high enough that new CRC screening guidelines recommend colonoscopy for patients >40 years and for post-transplant patients >30 years old. Performing colonoscopies in adult CF patients is complex due to comorbid diseases including poor lung function and diabetes. Non-invasive screening tests are needed, similar to faecal occult blood testing (FOBT) used for general population screening. While FOBT utility in CF remains unclear, other stool tests for gut inflammation (calprotectin and M2-PK) have shown promise in detection of CRC and polyps in the general population. They are elevated in children with CF and may be potential early markers of future CRC in adults with CF. To investigate this further, we will invite patients who meet criteria for CRC screening to provide stool samples prior to their scheduled colonoscopies. Markers of gut inflammation including calprotectin and M2-PK, FOBT and imbalances in gut bacteria will be assessed and compared with incidence of CRC and number of adenomas. Predictors for colorectal cancer and precancerous lesions will also be assessed.

Reports: 6 month report

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ACFRT Top-up Scholarship 2019

Project Title: Adverse Early Risk Factors for Adult Cardiovascular Disease in Paediatric Cystic Fibrosis

Name: Thomas Saunders

Institution: Murdoch Children's Research Institute

Duration: 3 years

Value: $5,000

Summary: Life with cystic fibrosis (CF) has undergone dramatic changes over the past decades, thanks to early interventions throughout childhood, including a high fat diet. Unlike previous generations, current CF children are living beyond their twenties, and now into their fifth or sixth decades. However the new medical challenges they will face are largely unknown. In particular, the impact of the leading causes of death worldwide, atherosclerosis and cardiovascular disease (CVD- heart attack and stroke), are unknown. Lung diseases similar to CF have much higher risks of CVD than the general population and there are increasing case reports of CF individuals developing CVD.

Atherosclerosis (hardening of the arteries) is thought to be largely preventable, especially when intervention starts in childhood. Risk factors such as inflammation, metabolic stress, and abnormal fat metabolism are all found in people with CF; but they are also prescribed high-fat diets, necessary to maintain good weight gain and growth. As CF life expectancy improves, and there are more and more instances of CVD in CF adults, novel studies aiming to detect it will be crucial.

This study investigates if CF children have early changes to blood vessels, biomarkers and immune system that may lead to atherosclerosis and CVD in adulthood. We will perform non-invasive ultrasounds looking at arteries, assess smaller vessels with retinal photographs, and measure specific biomarkers and immune cells in the blood.

This study is expected to show that CF children do have changes that suggest they may be at higher risk of atherosclerosis and CVD in the future. This will indicate if our current nutritional goals are sufficient, and guide changes to minimise the impact of CVD in CF adults. As people with CF live longer, it is critical that their increased survival also has improved quality of life with any treatment side-effects minimised. As such, this study will have a direct impact on how we treat people with CF in the future.

Reports:  None due

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ACFRT Top-up Scholarship 2019

Project Title: Computer Modelling of the Root Cause of Cystic Fibrosis

Name: Miro Astore

Institution: The University of Sydney

Duration: 3 years

Value: $5,000

Summary: We will use physics based atomic models to develop our understanding of cystic fibrosis and find treatments for it. With a focus on those who cannot currently access existing treatments.

Reports:  None due

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CFWA Golf Classic Scholarship - WA Top Up Grant 2019

Project Title: Exploring the therapeutic potential of phage therapy to treat Pseudomonas aeruginosa infection in people with cystic fibrosis

Name: Renee Ng

Institution: University of Western Australia

Value: $37,500 

Duration: 3 years

Summary: This study aims to explore a new treatment option for patients with bacterial lung infections using bacteriophages (‘phages’), a virus that targets and “eats” bacteria, eradicating them. Phage therapy is cheaper causes no side effects which is seen with currently used antibiotics. This project aims to understand if phage therapy can kill the bacteria and reduce the inflammation caused by infection.

Reports:  None Due

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CFWA Golf Classic Scholarship - WA Top Up Grant 2019

Project Title:  Does the MetaNeb®, a new airway clearance device, change lung function in adults with cystic fibrosis when they are well and when they are hospitalised for a lung infection?

Name: Naomi Chapman

Institution: Curtin University

Value:  $37,500

Duration: 3 years

Summary:  Cystic fibrosis (CF) is an inherited lung disease that causes excessive airway secretions, damaging airways and leading to chronic infection. Clearing the airways is a vital component of their care and is believed to reduce lung infections and slow disease progression. Although there are a range of techniques used to clear airway secretions, there is no evidence to support one over others. Therefore, the choice of technique is based largely on patient/therapist preference. This project will look at the effects of a new airway clearance device called the MetaNeb®, on lung function, secretion clearance and CF related symptoms in adults with CF who are well and those hospitalised with a respiratory exacerbation or chest infection. If this device is shown to be more effective than the commonly used techniques, this would be of great clinical significance as it will assist in guiding clinical use of the device throughout CF centres in Australia and internationally.

Reports:  None due

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Abbie Fennessy Memorial Fellowship 2019

 (supported by Technipro-Pulmomed Pty Ltd & Cystic Fibrosis Australia)

Project Title: Infection Control and prevention with non-invasive ventilation use

Name: Olivia McGuiness

Institution: Royal Prince Alfred Hospital

Value: $5,000

Duration: 1 year

Summary: Infection control and prevention is of paramount importance in the health care setting. The National Health and Medical Research Council in collaboration with the Australian Commission on Safety and Quality in Healthcare emphasize the importance of standard and transmission-based precautions (NHMRC, 2010). Recommendations for infection control and prevention are outlined for several commonly used medical devices including central venous catheters and mechanical ventilators used in the intensive care. There are no such guidelines for non-invasive ventilator (NIV) devices. Due to its high costs, patients usually obtain these devices from a hospital or government equipment loan program. NIV devices are used or the management of patients with acute and chronic respiratory failure including those with chronic obstructive lung disease (COPD), sleep disordered breathing and suppurative lung diseases, like Cystic Fibrosis (CF).

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Innovation Grant 2018

Project Title: Detailed characterisation of structure-function relationships in mild cystic fibrosis lung disease and validation of an ultra low dose high resolution CT (HRCT) scanning protocol

Name: Paul Robinson

Institution: Children's Hospital Westmead, NSW

Value: $80,000

Duration: 1 year 

Summary:  Cystic Fibrosis (CF) is a life limiting condition causing mucus build up and infection within the lungs, ultimately resulting in death due to respiratory failure.

One approach to improve survival focuses on the accurate detection of early CF lung disease to facilitate earlier treatment intervention. Traditional tools have limitations in this setting, but newer techniques exist which are more sensitive, such as Multiple Breath Washout (MBW) and CT scanning.

However, the optimal way to use them remains unclear and the radiation associated with CT scanning remains a concern for CF physicians. This work uses advanced analysis approaches to improve our understanding of the changes that occur in early CF lung disease during childhood.

We will describe how the detailed information gained from these tests can be better used to detect early disease, and investigate the utility of ultra low dose CT scanning approaches in this setting to alleviate radiation concerns. Our assembled research team, with international expertise across these techniques is ideally positioned to carry out this work.

The results of study will provide vital help in early detection of CF lung disease, enhancing our ability for early treatment intervention and improved outcomes.

Reports:  Six month report and Final Report

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Innovation Grant 2018

Project Title: Novel Multi-Omic Insight into Evolution of Antibiotic Resistance in Psudomonas aeruginosa (PA) in Cystic Fibrosis (CF) and Relationship to Clinical Outcomes

Name: Dr Ama-Tawiah Essilfie

Institution: Lung Infection and Inflammation Group at QIMR - Berghofer Institute of Medical Research, QLD

Value: $80,000

Duration: 1 year 

Summary:   Multi-antibiotic resistant (PA) is commonly found in CF patients. Persistent infection with PA is associated with worsening lung disease and  more frequent hospitalisations. Resistance may be naturally occurring or be acquired following years of exposure to antibiotic therapy, making PA very difficult to treat.

Many people with CF are infected with epidemic, antibiotic multi-resistant strains of bacteria. Despite progress in understanding antibiotic resistant mechanisms, little has changed in how antibiotics are prescribed. Using state of the art gene sequencing techniques, this project will investigate why antibiotic resistance is so common in epidemic PA compared to non-epidemic strains and whether this increased resistance causes more severe disease.

Additionally, the project will investigate the potential of a new technique for quantifying antibiotic resistance in sputum as an improved method of antibiotic selection compared to conventional methods. If successful, this could lead to improved clinical outcomes and shorten hospital stays.

Reports:
6 month Report
Final Report

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Ann Maree Bosch Career Fellowship 2018

Project Title: Cystic fibrosis airway gene therapy: towards first-in-human clinical trials

Name: Alexandra McCarron

Institution: Cystic Fibrosis Airway Research Group (CFARG); Children’s Hospital, Adelaide SA

Value: $10,000

Duration: 1 Year

Summary: Cystic fibrosis (CF) affects many organs, but the lung disease is the major cause of morbidity and mortality. Our primary focus at the Cystic Fibrosis Airway Research Group (CFARG) is to develop a gene-addition therapy, to prevent, halt, or improve treatment of CF lung disease – for people with any CF mutation type. By introducing a correctly-functioning CFTR (cystic fibrosis transmembrane conductance regulator) gene into the airway cells, and creating functioning CFTR protein, lung health should be dramatically improved.

The CFTR gene is delivered to the airway cells using a specialised gene delivery vehicle known as vector. As pre-clinical studies progress, an important translational step is developing a way to produce the large volumes of gene vector required for human clinical trials and eventual commercialisation.

Reports: Final Report

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ACFRT Top-up Scholarship 2018

Project Title: Understanding the Mechanism of Interations between Anti-Inflammatory and Antibiotics for Optimal Cystic Fibrosis Treatment

Name: Zara Sheikh

Institution: Discipline of Pharmacology, School of Medicine, University of Sydney

Duration: 3 years

Value: $ 5,000

Summary: The mainstay of current Cystic Fibrosis (CF) treatment is based on chronic antibiotics therapy delivered by inhalation, as adjunct to either oral/inhaled anti-inflammatory drugs, and/or oral/intravenous antibiotics to treat pulmonary exacerbations.

This project will investigate the different combinations of anti-inflammatory and antibiotics used in CF treatment to unravel their interactions, and understand if their combined use could potentially impact on their efficacy and treatment outcomes.

This project could potentially improve the day-to-day lives of CF patients, reducing the treatment burden, shortening treatments times and decreasing treatment cost, ultimately improving quality of life.

Reports: Final Report

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Abbie Fennessy Memorial Fellowship 2018

 (supported by Technipro-Pulmomed Pty Ltd & Cystic Fibrosis Australia)

Project Title: High intensity interval training using telehealth for adolescents with Cystic Fibrosis

Name: Anna Middleton

Institution: The Children's Hospital at Westmead

Value: $ 5,000

Duration: 1 year

Summary: Improvements in the management of pulmonary and gastrointestinal manifestations of cystic fibrosis (CF) have seen life expectancy increase dramatically. The emergence of secondary complications that adversely affect function and quality of life are commonly seen in adolescents and adults with CF. One of these complications is skeletal muscle dysfunction [1]. Skeletal muscle dysfunction in CF manifests with reductions in muscle strength, endurance and skeletal muscle mass [1]. Skeletal muscle mass and strength are strongly correlated with peak oxygen uptake [2-4], prognosis and survival [5-8]. Compromised skeletal muscle strength, endurance and function are correlated with reduced anaerobic performance [9-11] and reduced aerobic capacity [4, 12, 13]. Aerobic capacity is a strong predictor of mortality [6-8, 14], requirement for hospitalisation [15] and quality of life [16, 17]. 

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ACFRT in conjunction with CFSA Special Grant 2018

Project Title: A novel treatment strategy for cystic fibrosis S. aureus-associated chronic rhinosinusitis

Name: Professor P-J Wormald

iInstitution: Adelaide and Flinders Universities 

Duration: 1 year

Summary: Chronic Rhinosinusitis (CRS) is one of the most common manifestations in patients with Cystic Fibrosis (CF) contributing to CF lung disease. The frequent and often long-term use of antibiotics to treat relapsing airway infections significantly contributes to the threat of Multi Drug Resistant (MDR) pathogens. There is an urgent need for the development of new treatments that are effective at eliminating infections with MDR pathogens.

Bacteriophage (phage) is a virus that targets and kills one specific bacterial species, leaving the human mucosa and commensal species unaffected. In collaboration with Ampliphi Biosciences, our team has recently successfully completed a human clinical trial to test the safety and preliminary efficacy of a phage cocktail to treat S. aureus CRS. We have further improved this treatment to develop a novel phage-based formulation that is now able to eradicate rather than reduce S. aureus including MDR S. aureus (MRSA).

This project brings together our Adelaide-based Rhinology team (Prof PJ Wormald and A/Prof S Vreugde) with CF centres located in Adelaide and at the Royal Brompton Hospital (RBH) in London. The team aims to optimise the delivery of the new formulation to make it suitable for topical delivery to the sinus region in CF patients.

Reports: None Due

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ACFRT in conjunction with CFSA Special Grant 2018

Project Title: Lentiviral Mediated Airway Gene Therapy: Is lysophosphatidylcholine required for safe and effective gene transfer in a cystic fibrosis lung?

Name: Dr Chantelle Lee Carpentieri

Institution: University of Adelaide & Women's and Children's Hospital 

Value: $30,000

Duration: 3 years

Summary: The current treatments for CF airway disease are not preventative or curative. The addition of a properly-functioning CFTR gene into affected CF airway cells is recognised as the only rational method likely to prevent, halt or treat CF airway disease, regardless of mutation type.

To facilitate gene transfer using our Adelaide-designed lentiviral CFTR gene vector, the CF airway research group has developed a unique treatment protocol, successfully tested in several animal models. This unique treatment protocol first prepares the airway to be receptive to the gene vector using an airway-conditioning compound called lysophosphatidylcholine (LPC).  

Using this two-step dosing method (gene vector and LPC), a single dose of gene vector has produced functional CFTR correction in mouse nasal airways. Additionally, we can use this approach to introduce various test genes into cells of the airways of rats, sheep, ferrets and other animals. Despite the absence of safety issues due to LPC in these species, the safety of LPC in a CF lung has not yet been tested due to lack of an appropriate CF animal model. Now that our research group has established a CF rat colony in Adelaide, the aim of my project will be to test the safety and efficacy of our gene transfer methods for potential future use in the treatment of CF lung disease.

Reports: None Due

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Antimicrobial Resistance In Cystic Fibrosis 2018

International Task force

The prevalence of antimicrobial resistance (AMR) among microbial pathogens is increasing and is a high priority worldwide for interventions from antibiotic stewardship to new antibiotic development (http://www.who.int/antimicrobial-resistance/en/).  Increasing AMR prevalence is driven by the widespread use of antibiotics.

AMR is commonly encountered in bacteria and fungi isolated from the airways of people with cystic fibrosis (CF).  This is an expected finding given the high use of antibiotics in CF patients. The increasing survival of people with CF will result in greater lifetime exposure to antibiotics.

There is a lack of full understanding for stakeholders including people with CF, clinicians, the pharmaceutical industry, and (drug approving) regulatory bodies, about the relevance of AMR and how this should influence drug development and usage in the treatment of CF lung disease.

The Taskforce will develop guidelines on the interpretation of AMR for clinical care in CF. The Taskforce will also provide guidance on the role of AMR in clinical trials with new antimicrobial agents. The Taskforce that will have international and multidisciplinary representation will address the following objectives:

  • Understand how chronic infections differ from acute infections with respect to the microbiological assumptions regarding AMR.
  • Describe current and developing methodologies for determining antimicrobial resistance.
  • Assess the value of current susceptibility testing including the frequency and timing of testing.
  • Offer guidance for the use of antimicrobial resistance testing in the conduct of clinical trials by the pharmaceutical industry and regulatory agencies.
  • Set key research priorities for the development of appropriate future application of AMR diagnostics to improve patient outcomes.
  • Explore how monitoring antibiotic usage impacts on AMR in people with CF.

Australia/New Zealand Representatives

  • Cass Byrnes, CF pediatrician
  • Jason Roberts, pharmacist
  • Tim Kidd, microbiologist
  • Scott Bell, adult CF physician

Update February 2018

Thanks again to CFA for their support of this international working group. The working group met at the North American CF Conference and had approximately 50 to 60 participants.

Five groups have been established:

  1. Antimicrobial resistance definitions 
  2. Literature review and development of current evidence 
  3. Antimicrobial stewardship 
  4. Education for clinicians, patients, families and participants 

Since that meeting, the Steering Committee Group have had a teleconference to report the update. All four groups are demonstrating rapid progression. The aim is to meet again in Artimino in September 2018 in order to finalise the recommendations of the working group for antimicrobial resistance in cystic fibrosis.

Findings of the groups are anticipated to be presented in a symposium of the North American CF Conference in Denver in October 2018 and again in the European CF Conference in Liverpool in June 2019. 

At least three publications are likely to be generated by the working group but importantly will provide the clinician, research and patient community with an update on AMR in cystic fibrosis.

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Innovation Grant in Partnership with Cystic Fibrosis Research Limited (QLD) 2017

Title: RNA therapeutics: novel paradigm in mutation independent CF therapy

Name: Shafagh Waters

Institution: University of NSW (UNSW)

Value: $ 70,000

Duration: 1 year

Summary: Cystic fibrosis (CF) is a genetic disease, most common among people of European ancestry, afflicting ~70,000 individuals worldwide. It is caused by defects in a single gene that codes for the CF Transmembrane Regulator (CFTR) protein. Historically, therapies were restricted to treatment of symptoms, but new molecular therapies that restore function to the defective CFTR protein have been developed. Unfortunately, these therapies are only effective for 5% of people with CF.

This project examines how a gene (called BGas) can be genetically manipulated for CF therapy. When BGas is removed from the cell, CFTR function is restored. This therapy will be beneficial to majority of CF patients around the globe – not just 5%.

Miniaturized versions of CF patient gut (mini-gut), using their own cells as starting material are created in the lab. followed by BGas-removing compounds being added to the “mini-gut” to identify those responsive to BGas therapy.

The invaluable results from this project could drive future trials, delivering BGas-removing compounds directly to the CF patients. A similar approach has had promising outcomes for children with spinal muscular atrophy. The discovery of BGas-removing compounds may finally offer light at the end of the tunnel for CF therapy.

Reports:

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Innovation Grant 2017

Project Title: Investigating the role and therapeutic targeting of iron in cystic fibrosis

Name: Jay Horvat

Institution: University of Newcastle, NSW

Value: $80,000

Duration: 1 year 

Summary: Whilst improved therapeutic strategies have become available for CF, many patients still frequently experience exacerbations of disease that result in progressively increasing lung pathology and deteriorating lung function.

Infections play an important role in this progression of CF with persistent Pseudomonas infections a major contributor to CF morbidity and mortality. More effective strategies are desperately needed to improve efficacy for the treatment of Pseudomonas infection in CF in order to improve patient outcomes.

Studies have shown that iron levels are increased in the airways of CF patients and increased iron stimulates Pseudomonas biofilm formation, aggregation, adhesion and invasion, allowing the pathogen to avoid host removal. Indeed, in clinically stable CF patients, there is a strong positive correlation between sputum iron and Pseudomonas levels. These findings suggest that increased iron in CF patients is a potential causal factor of Pseudomonas persistence.

In this new study, we will use a novel combination of experimental models of increased iron loading in the lung and Pseudomonas infection and clinical investigations to better understand the role of iron in the pathogenesis of CF. We will also determine whether therapeutically suppressing increased iron levels in the airways improves the clearance of Pseudomonas infection from the lungs

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Ann Maree Bosch Career Fellowship 2017

Project Title: RNA-based therapeutics for mutation-independent CF therapy

Name: Sharon Wong

Institution: School of Women’s and Children’s Health & University of NSW (UNSW)

Value: $10,000

Duration: 1 Year

Summary: Cystic fibrosis (CF) is caused by mutations of the CFTR gene which results in dysregulated salt and fluid transport in the body. There has been significant progress in CF therapy in the last 25 years; the average life expectancy of CF patients are now at ~38 years compared to 12 years when the gene was first discovered. Indeed, medications currently approved for CF patients, Kalydeco® and Orkambi® significantly improve symptoms and quality of life of CF patients, although they are only effective for patients with certain mutations eg. G551D and heterozygous DF508. Treatment option for patients with other CFTR mutations or perhaps one that works for all CF patients indiscriminately is still lacking.

The goal of this project is to develop an effective treatment for the general CF population, irrespective of their mutation types. We first establish a personalized and predictive platform for testing potential drugs in our lab by using patient-derived tissue specimens. Miniaturized versions of each CF patient are created using their own stem cells from lung and gut tissue, called the “CF AVATAR”, which swells up when a potential new drug restores the defective function of the CFTR protein. Notably, a recent study showed the reduction or absence of a novel human gene called BGas was shown to boost CFTR levels independent of CFTR mutation types. We will thus explore the therapeutic potential of a few small molecules targeting BGas in the patient-derived “CF AVATAR”. If successful, gene targeting will herald a new dawn in CF therapy, with one drug that works wonders across the entire CF population.

Reports: None to date (not due)

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Hardie Foundation Scholarship - WA Top Up Grant 2017

Project Title: The role of Interleukin and necrosis leading to neutrophilic inflammation in children with cystic fibrosis.

Name: Samuel Montgomery

Institution: Telethon Kids Institute WA

Value: $ 37,500

Duration: 3 years

Summary: This study aims to determine whether inflammation resulting from cell death in the airway is increased in early lung disease in Cystic Fibrosis (CF). It also aims to identify differences in specific gene pathways during inflammation resulting from cell death in the CF airway.

Reports: Final Report

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CFWA Golf Classic Scholarship - WA Top Up Grant 2017

Project Title: Personalised antisense oligonucleotide therapy to correct CFTR function in Cystic Fibrosis patients.

Name: Kelly Martinovich

Institution: School of Paediatrics and Child Health, Princess Margaret Hospital WA

Value: $ 37,500

Duration: 3 years

Summary: Current on the market small molecule therapies do not help all patients with CF, since it is caused by over 2000 different mutations. Here, we aim to develop therapies that target the more rare CF mutations. Many mutations change the message that is passed on from the CF gene. This faulty message results in a poorly functioning CFTR channel. The CFTR channel maintains the airway surface liquid in the lungs that helps clears infections.

The therapies that we will aim to develop are another type of small molecule called antisense oligonucleotides (AOs).  AOs can change the faulty message transferred from gene to protein and improve the function of the CFTR protein. Furthermore, AOs can be designed to target a specific site in the CF gene depending on the location and effect of the CF causing mutation that the specific person holds. In predicted cases, AOs in theory could reduce the severity of CF. It is hoped that this study will initiate a personalised medicine pathway generating molecules that lessen the disease severity caused by specific CF-causing mutations.

Reports: Final Report

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ACFRT Top-up Scholarship 2017

Project Title: Does high intensity interval training improve fitness in people with cystic fibrosis?

Name: Abbey Sawyer

Institution: School of Physiotherapy and Exercise Science, Curtin University WA             

Duration: 3 years

Value: $ 5,000

Summary: People with cystic fibrosis (CF) who are fitter have improved quality of life and may live longer.

Currently, people with CF are encourage to exercise for 30 to 60 minutes a day, in line with recommendations provided for people who are healthy. This is often difficult to achieve due to the high daily treatment burden and competing demands in life such as work, study and family.

This project will investigate whether 10 minutes of high intensity interval training over an 8-week period improves fitness and other important outcomes such as quality of life, mood, confidence to complete exercise and enjoyment in people with CF.

Reports: Final Report

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ACFRT Top-up Scholarship 2017

Project Title: Are altered carbohydrates associated with compromised immune cells in cystic fibrosis?

Name: Harry Tjondro

Institution: Department of Chemistry & Biomolecular Science – Macquarie University

Value: $ 5,000

Duration: 3 years

Summary: This project will for the first time use biochemistry to explore the aberrant carbohydrate signatures and their functional significance on CF neutrophils relative to healthy neutrophils, an important type of innate immune cells in CF.

Advancing our understanding of the structural and functional alterations of carbohydrates in CF neutrophils will be valuable in order to unravel the underlying disease mechanisms.

This study will generate valuable, fundamental biochemical knowledge to support the generation of new immune-based therapeutics to alleviate the disease burden of affected individuals.

Reports: Final Report

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ACFRT Top-up Scholarship 2017

Project Title: Pseudomonas aeruginosa behavior in cystic fibrosis: new insights using novel super-resolution microscopy.

Name: Simone Visser

Institution: Department of Respiratory Medicine – RPAH, NSW

Value: $ 5,000

Duration: 3 years

Summary:  Pseudomonas aeruginosa and other emerging bacterial pathogens (multi-resistant Staphylococcus aureus, Achromobacter xylosoxidans, Stenotrophomonas maltophilia) establish chronic lung infections in patients with cystic fibrosis by forming biofilms in the airways. 

Biofilms are adherent, slimy layers of bacteria, which are highly impermeable and resistant to antibiotic therapy, making treatment very challenging. It is well recognised that after development of chronic bacterial lung infections, patients experience deterioration in lung function, increased flare-ups and poorer survival.  There is an urgent need to develop new effective treatments against bacteria and their biofilms.

Our group has found that innovative triple combination therapy (glutathione/DNase/antibiotic) is effective in reducing pseudomonas biofilms (Ashish Kumar et al, Frontiers in Microbiology 2017; 8:1-18).  I am now expanding that work into the emerging CF pathogens, which are typically under-studied and include achromobacter, stenotrophomonas, and methicillin-resistant staphylococcus aureus. According to recent registry data, these bacterial species are increasingly common in CF patients. Further, chronic infection with these bacteria is linked with poorer lung health. I am investigating whether similar triple combination therapy is effective against the biofilms of these bacteria.

The ACFRT Grant has allowed me to begin investigating innovative combination antibacterial therapy to treat chronic lung infections in cystic fibrosis.  The combination treatment targets bacterial biofilms (slimy layer), which is usually impenetrable and resistant to typical antibiotic therapies.

Reports: Final Report

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Abbie Fennessy Memorial Fellowship 2017

 (supported by Technipro-Pulmomed Pty Ltd & Cystic Fibrosis Australia)

Project Title: Airway Clearance Adherence Monitoring System (ACAMS) – development and initial benchtop trial.

Name: Nathan Ward

Institution: Royal Adelaide Hospital (RAH)

Value: $ 5,000

Duration: 1 year

Summary: As part of his PhD candidature at La Trobe University, Nathan developed an electronic device that can potentially be used for both clinical and research assessment of adherence to positive pressure-based airway clearance techniques. The device has been called the Airway Clearance Adherence Monitoring System (ACAMS).

Using the funds provided through the Abbie Fennessy Memorial Fellowship, five ACAMS units will undergo independent pressure accuracy testing against Australian standards. Following this testing, the five units will undergo an initial benchtop trial to prove their utility with PariPEP S, PariOPEP and Acapella DH devices for a one week period for each device.

If this initial benchtop study demonstrates that the ACAMS device can accurately record performance of these techniques and provide analysable data on the quality of the technique performance, these data will be used to support applications for an initial pilot clinical trial. It is envisaged that this trial would recruit adults with cystic fibrosis attending the Royal Adelaide Hospital Adult Cystic Fibrosis Service.

The Abbie Fennessy Memorial Fellowship will also be used to cover the costs for Nathan to attend the North American Cystic Fibrosis Conference (NACFC) in October 2018, where he will I will be submitting an abstract on the development and benchtop validation of the ACAMS device along with two other abstracts for presentation at the NACFC.

Reports - none due

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Special Grant 2016

Project Title: Multi-action antibiotics to treat chronic biofilm infections

Name: Michael Kelso

Institution: University of Wollongong

Duration: 3 years

Summary: Biofilms often build up in the lungs of people with CF. They contain large populations of bacterial cells and are encapsulated within gum-like materials which protect bacteria against the action of antibiotics and against the action of cells in the patient’s immune system.

Antibiotic resistance can be increased up to 1000-fold in biofilms. Associate Professor Michael Kelso from UOW together with members of his research team were the first to discover that low concentrations of nitric oxide (NO) act as a signal that triggers bacteria in biofilms to disperse. When this happens the bacteria become more sensitive to antibiotics and to the body’s immune system. This project investigates the effects of combinations of NO-releasing compounds with antibiotics (cephalosporins) to develop a new way of targeting delivery of NO to biofilms.

Reports:

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Special Grant 2016

Project Title: Determining factors that influence the acceptance and adoption of a gene therapy for cystic fibrosis airway disease

Name: David Parsons, Martin Donnelly & Ivanka Pritchard

Institution: University School of Health Sciences, Flinders University & The Cystic Fibrosis Airway Research Group at the Women’s and Children’s Hospital

Duration: 1 year

Summary: This grant is designed to develop an ongoing relationship between the School of Health Sciences, Flinders University (CI Prichard), The Cystic Fibrosis Airway Research Group at the Women’s and Children’s Hospital (WCH; CIs Donnelley and Parsons), and Cystic Fibrosis Australia.

The primary focus of the WCH group is on developing a gene therapy treatment for cystic fibrosis (CF), and they have expertise in molecular analyses and animal models. Flinders University provides expertise in health psychology and the understanding of attitudes and beliefs in relation to treatments and health outcomes. CF Australia is a philanthropic organisation set up for the purpose of providing support and services to people with CF, their carers and families, and funding high quality research.

The aim of this partnership is to use the combined expertise and resources of the group to assess the perceived acceptability of, and public support for, gene therapy for CF. We will use this information to obtain future grant funding to develop education programs to better inform the CF community about the risks and benefits of this type of treatment to improve the success of clinical trials.

Findings from this project will contribute to the effectiveness of our future applications for funding as we will be able to include our assessments of the feasibility of the approach from both the scientific/biological and the CF community acceptance-spheres.

Reports: to follow

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