A Novel Treatment for Non-Tuberculous Mycobacteria Lung Infections in People with Cystic Fibrosis.
Project Duration – 2 Years
NTM lung disease is a serious infection caused by bacteria that are common in the environment and can cause lung damage. These types of infections are particularly devastating for people with cystic fibrosis as their prevalence is rapidly rising (up to 30% in adult patients in the US).
To address this problem and evaluate the research undertaken, partnership is key. This project will see researchers utilise Australia’s first cystic fibrosis rat model, the CF-Rat which was established by the Cystic Fibrosis Airway Research Group (CFARG) and funded by Cure4 Cystic Fibrosis.
Led by Associate Professor Sarah Vreugde, her team intends to develop a novel treatment for Non-Tuberculous Mycobacteria Lung Infections in People with Cystic Fibrosis.
Sarah is the Chief Research Scientist for the Department of Otolaryngology working together with Professor PJ Wormald at The Queen Elizabeth Hospital and The University of Adelaide since July 2011.
Her main research interest is chronic relapsing infections and chronic inflammation of the upper airways and wound healing. Sarah graduated with High Distinction as a Medical Doctor from the Brussels University (VUB) in Belgium (1994). She subsequently specialized in Otolaryngology, Head and Neck surgery at the same university.
She performed doctoral studies and obtained her PhD degree in molecular biology (Tel Aviv University, Israel, Laboratory of Prof. K. Avraham) in 2003.
From 2008 to 2011, she was employed in the European Patent Office (The Hague, The Netherlands), where she was analysing the patentability of National, European and International Patent applications in the Department of Biotechnology.
Through this grant Sarah’s research project will target Non-Tuberculous Mycobacteria (NTM) lung infections in people with cystic fibrosis, which has unequivocally been defined an urgent unmet medical need.
NTM lung disease is a serious infection caused by bacteria that are common in the environment and can cause lung damage. They’re aerosolized, which means that the bacteria can exist in water and soil particles that are in the air.
They’re associated with a more rapid decline in lung function and with lung transplant failure. Importantly NTM are naturally resistant to antibiotics and even disinfectants and they’re challenging to treat. They require long-term (more than 12 months duration) systemic and topical treatments with up to five concurrent antibiotics. This can cause significant side effects requiring cessation of therapy.
Sarah’s technology is unique because it is the only treatment in pre-clinical development that targets the bacterial iron metabolism, critical for microbial virulence and survival. It also has potent antioxidant, iron chelating and anti-inflammatory action.
These combined effects are thought to be particularly beneficial in the cystic fibrosis airways where a mucus iron overload is present in association with increased microbial virulence and inflammation, and topical iron chelating treatments are already trialled in the cystic fibrosis population.
Sarah says, ‘our pilot data supports a strong and fast antimicrobial action of the treatment with a complete eradication of NTM from within infected macrophages.’
The project will determine the mode of action of the technology and provide proof-of-concept of the technology in a preclinical animal model of cystic fibrosis. These studies are needed to advance the technology towards clinical translation.
The aims and milestones of this project are:
- Define formulation, MOA, dosing regime (expected completion October 2020)
- Ethics approval for CF rat model (NTM model, DEF-GAPP Poc study) (expected completion May 2020)
- Optimise the delivery method of medication into the lungs (expected completion December 2020)
- Establish NTM CF rat model (expected completion December 2020)
- Complete proof of concept studies in NTM CF rat model (expected completion June 2021)
Associate Professor Sarah Vreugde – University of Adelaide
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