Respirion offers hope for people living with cystic fibrosis
May 13, 2024
- An international clinical trial is underway on a Western Australian-developed inhaled antibiotic combination for people with cystic fibrosis
- A preliminary clinical study had shown the treatment is effective in reducing infection and improving lung function
- Potential for the drug to be used to treat other lung infections in children and adults
A promising new treatment pioneered in Western Australia for people with cystic fibrosis has commenced testing in a clinical trial in the United States and Australia.
Developed by researchers at Telethon Kids Institute and Perth Children’s Hospital, the treatment, known as RSP-1502, uses an antibiotic booster to improve the effectiveness of treatment for respiratory infections in people with cystic fibrosis.
An early WA-based trial using RSP-1502 to treat chronically infected CF patients demonstrated an 800-times greater reduction in bacterial load, and an average 16 per cent improvement in lung function compared to 5 per cent for those patients using a standard of care treatment. Enhanced lung function is vital for patients, enabling them to engage more in normal activities such as moving around, participating in sports, enjoying improved quality of life and ultimately, increasing their overall life expectancy.
The promising results from the previous trial led to the new inhaled drug being developed by Respirion Pharmaceuticals Pty Ltd, a Telethon Kids spin-out company, supported by funding from Brandon Capital through the Federal Government’s Biomedical Translation Fund, and the US Cystic Fibrosis Foundation.
Telethon Kids Executive Director Professor Jonathan Carapetis said that following on from the successful first study in WA patients, the next phase of clinical trials was an exciting step for the new drug.
“With the threat of increasing resistance to the limited antibiotics that we have available, new options are urgently needed for kids with cystic fibrosis,” Professor Carapetis said. “This new combination represents a long-awaited breakthrough for this vulnerable population with the potential to deliver better anti-infective performance and greater improvement in lung function for CF patients at any age or stage of their disease.”
The Phase 1 component of the current trial is already underway and will determine the safety and tolerability of RSP-1502. Respirion is actively finalising options to expand the study to include a Phase 2 component which include children and allow determination of efficacy.
In doing so, researchers led by WA paediatric respiratory clinician, Professor Barry Clements from the Wal-yan Respiratory Research Centre, are hopeful this clinical trial will bring the new drug closer to receiving international regulatory approval, thereby providing people with CF access to a more effective treatment which, if started early in childhood could slow disease progression.
“Persistent lung infections and the inevitable decline in lung function remain urgent unmet needs in cystic fibrosis patients,” Professor Clements said.
Cystic fibrosis is a genetic disorder that occurs in about one in 2500 births where the defect predisposes patients as young as under one year of age to lung infections with bacteria such as Pseudomonas aeruginosa. Once established, these infections are difficult to clear with existing treatment options, and lead to progressive lung damage, decreased lung function, and eventually respiratory failure. With few effective options for the treatment of chronic lung infections in people with cystic fibrosis, RSP-1502 represents a potential breakthrough for this vulnerable population.
ENDS
Media contact:
Kirrily Davis, Head of Brand, Communications and Engagement – Brandon Capital kdavis@brandoncapital.com.au
Simone Knox, Media and Content Manager – Telethon Kids Institute
simone.knox@telethonkids.org.au
About Telethon Kids Institute
Telethon Kids Institute is one of the largest and most successful medical research institutes in Australia. We’ve created a bold blueprint that brings together community, researchers, practitioners, policy makers and funders, who share our vision to improve the health and wellbeing of children through excellence in research. The Institute is headed by leading paediatrician and infectious diseases expert Professor Jonathan Carapetis, with Founding Director Professor Fiona Stanley now Patron. Telethon Kids is independent and not-for-profit. The majority of funding comes from our success in winning national and international competitive research grants. We also receive significant philanthropic support from corporate Australia and the community.
About the Wal-yan Respiratory Research Centre
The Wal-yan Respiratory Research Centre is a global epicentre for paediatric respiratory research informing clinical practice and driving a new research agenda for childhood lung health. A powerhouse partnership between the Telethon Kids Institute, Perth Children’s Hospital Foundation and Perth Children’s Hospital, the Centre aims to prevent and cure respiratory illness in children and ensure that all children have healthy lungs for life. The Centre has a 30-year history of globally competitive research from diverse groups of internationally acclaimed researchers in Western Australia, working in paediatric research. For more information, visit: walyanrespiratory.org.au
