Seeing Disease at the Cellular Level: Could Adaptive Optics Imaging Transform the Future of Clinical Trials?
Treatments in clinical trials are creating new hope for people living with inherited retinal diseases, a group of conditions that are the leading cause of blindness among working-age Australians. While several promising therapies are now being developed, researchers and pharmaceutical companies face a significant challenge: proving whether these treatments are working. Many treatments are designed to slow the progression of disease rather than restore vision, yet conventional clinical trial measures often struggle to detect these subtle changes over practical timeframes.
A National Industry PhD project between The University of Melbourne, Centre for Eye Research Australia and a clinical stage biotechnology company is exploring whether an emerging imaging technology known as adaptive optics could help solve this problem. Adaptive optics compensates for the natural optical imperfections of the eye, allowing researchers to visualise individual retinal cells at an unprecedented level of detail. The project is investigating whether these cellular-level measurements could become new biomarkers for monitoring disease progression and evaluating the effectiveness of gene therapies in patients with inherited retinal diseases, particularly retinitis pigmentosa.
The importance of this challenge extends far beyond a single disease. Current clinical trials for inherited retinal diseases can be lengthy and expensive because existing outcome measures often require long observation periods before meaningful changes can be detected. Researchers and industry partners alike are seeking more sensitive ways to measure treatment effects. If adaptive optics imaging can detect changes at a cellular level, it may provide a more objective and responsive way to assess whether therapies are slowing degeneration, potentially enabling faster and more efficient clinical trials.
The research combines advanced retinal imaging with detailed clinical assessments and longitudinal patient monitoring. Participants undergo standard clinical testing alongside specialised imaging technologies, including adaptive optics imaging and other retinal imaging methods. By collecting and analysing data over time, the project aims to determine whether adaptive optics can reveal changes that are not visible using conventional imaging approaches and whether those changes could become meaningful clinical trial endpoints.
The potential beneficiaries are extensive. For patients, more sensitive outcome measures could help accelerate the development and approval of therapies targeting inherited retinal diseases and other vision-threatening conditions. For researchers and clinicians, improved imaging biomarkers could provide deeper insights into how retinal diseases progress and respond to treatment. For pharmaceutical companies, more reliable clinical trial endpoints may reduce uncertainty, shorten development timelines and improve the ability to demonstrate treatment efficacy to regulators.
The project also highlights the strengths of the Industry Researcher PhD model. PhD candidate Bhaj Grewal was already working within the clinical trials environment at Centre for Eye Research Australia before commencing the PhD. An optometrist and clinical trial coordinator, she was already leading inherited retinal disease clinical trials and brought extensive experience in participant recruitment, protocol delivery and data collection. The PhD is enabling her to combine this practical industry expertise with advanced research training, strengthening her capabilities in clinical research, data analysis and research translation.
Throughout the project, Bhaj has expanded her expertise in retinal imaging, scientific publishing, international collaboration and emerging technologies such as artificial intelligence. The research has included the development of advanced data curation pipelines and the co-development of AI-assisted tools for analysing adaptive optics imaging datasets. These experiences are helping build a unique skill set that spans clinical practice, research and industry innovation.
Bhaj’s Principal Supervisor, Professor Lauren Ayton, said that the opportunity for Bhaj to undertake a PhD whilst continuing to work in clinical trials has been game-changing for the team.
“Bhaj’s senior leadership of our clinical trials meant that it would have been very challenging for her to become a full-time PhD student, but it had always been a dream of hers to complete a higher research degree.
The Industry Researcher PhD model meant that the PhD project she undertook had direct relevance and translation to industry (both our clinical trials organisation, and the biotech company we worked with). Her research will get to patients faster and more effectively through this scheme, and also enabled her to continue to support the clinical trials whilst doing her research. A win-win for us all!”
The longer-term vision is also exciting. If adaptive optics imaging proves effective as a clinical trial endpoint, it could influence how inherited retinal disease clinical trials are designed globally. The project aims to generate evidence that could support discussions with regulatory bodies and contribute to the broader adoption of more sensitive clinical outcome measures. The technologies and methodologies developed may also have applications beyond inherited retinal diseases, potentially benefiting clinical trials for other retinal conditions where sensitive measures of disease progression remain a challenge.
Ultimately, this research is about making promising treatments easier to evaluate and potentially faster to reach the patients who need them. By combining world-leading imaging technology, industry expertise and clinical trial experience, Bhaj’s PhD research is helping to build new tools that may reshape how vision-saving therapies are developed, tested and delivered in the future.