Scientia Senior lecturer, UNSW
What is the problem you are trying to address?
Cystic Fibrosis (CF) remains an incurable life-limiting inherited condition. Despite development of therapeutics that directly repair the CFTR protein dysfunction, the clinical heterogeneity in therapeutic response in patients, has necessitated development of a clinical tool that predicts treatment response for individual patients.
What is your research doing to solve this problem?
My team and I have developed a national alliance of CF clinics and have created an Australian biobank of 200 CF patient stem-cell-derived airway and gut organoid. We have built a high throughput drug discovery and screening platform for implementing CF personalised medicine.
Please list your top 5 public outreach or science communication outputs:
- My work was given as evidence by Rare Voices Australia at the 2021 Parliamentary Inquiry into the approval process for new drugs and novel technologies.
- The resultant Zimmerman report recommended the creation of a Centre for Precision Medicine and Rare Disease to be embedded within the Department of Health.
- Similarly, my work has influenced the development of the critical policy White Paper “Cell and Gene Therapies: risking to the challenge”, 2021
- My work is being used to help advocate for equitable access to new drugs for those 10% patients who cannot access drug treatment and will be a major focus by CF Australia’s advocacy plan in 2022.
- My community engagement to promote CF research, is evident by interviews on Channel 9 News, ABC Radio National’s Health Report, speaking at 14 different educational and fundraising events including Sydney Children’s Hospital Cystic Fibrosis Community and Parents Education Night, Rotary SOLAS Charity Regatta 2020, The Bathers’ Gold Lunch 2020
- A national survey to assess the acceptance of organoids to guide treatment decisionsERJ2021.