As 2022 begins, Centre for Eye Research Australia (CERA) Managing Director Professor Keith Martin looks forward to forging new partnerships with eye care professionals and putting patients at the forefront of research.
As an eye doctor, the most frustrating part of my job is having to tell a patient that there is nothing more we can do to save their vision.
From the bench to bedside, our end-game is the same: we want to stop people from going blind and restore sight for people who have lost their vision
Informing a patient that the available treatments that work for most people don’t appear to be working for them, or that there is no treatment available for their disease, is very hard news to deliver.
The desire to change that story, and offer hope to patients, inspired me to take up a research career more than two decades ago.
And it’s what motivates the team here at the Centre for Eye Research Australia, whether working in the lab on pre-clinical research, observing patients in natural history studies, or testing new therapies in clinical trials.
From the bench to bedside, our end-game is the same: we want to stop people from going blind and restore sight for people who have lost their vision.
One of the factors that attracted me to move to CERA, from the University of Cambridge three years ago, was the unique combination of basic and clinicianscientists working together to develop new treatments for patients.
Although it is small, CERA punches well above its weight – together with the University of Melbourne we are consistently ranking in the top five for academic output in ophthalmology in the World University rankings, alongside much bigger organisations such as Harvard University, Johns Hopkins and University College London.
Combined with its location in the world-leading Royal Victorian Eye and Ear Hospital and Melbourne’s thriving biotech precinct, I could see the real opportunity to do transformational research that would make a real difference for patients.
Despite the unpredictability of the past two years, caused by the COVID-19 pandemic, we have seen significant advances in our research.
After an initial dip in 2020, clinical trials activity has rebounded. We have embarked on a number of new clinical trials that are giving Australian patients early access to cutting edge treatments, and have expanded our research capabilities.
In 2021, we established three new research groups at CERA and appointed two new Principal Investigators from our existing team.
We welcomed Dr Luis Alarcon-Martinez who will establish Australia’s first vascular neuroscience research team.
Dr Alarcon-Martinez joins CERA from the University of Montreal where he was part of a team which demonstrated how cells in the retina communicate with each other and regulate blood supply to keep vision intact.
We hope his expertise in using cutting-edge new microscopy techniques to identify ‘nanotubes’, which help control the delivery of blood to the areas of the eye where it is most needed, will revolutionise our understanding of the causes of diseases that affect the eye and brain, such as glaucoma, Alzheimer’s disease and strokes.
As the only one of a handful of scientists in the world with this expertise, Dr Alarcon-Martinez will make a valuable addition to our work to develop new treatments for glaucoma and identify new biomarkers for eye and brain diseases.
We also welcomed Associate Professor Rick Liu, who has moved from the University of Tasmania to establish a new genetic engineering team at CERA.
Associate Professor Liu’s team will further add to our research capabilities in gene therapies.
His work in Tasmania has been very impressive and he brings many skills that will be useful to other research teams at CERA, including my own which is developing gene therapies for glaucoma.
His group will focus on using gene editing technology to treat inherited retinal diseases, diabetic retinopathy, age-related macular degeneration (AMD) and glaucoma.
They are also looking to the next generation of gene therapy – ‘switchable’ gene therapy – where a therapeutic gene can be turned on and off to develop better gene therapies for the long-term management of eye disease.
We also welcomed experienced ocular oncologist Dr Rod O’Day to our team as an Honorary Principal Investigator to establish an ocular oncology research team.
Dr O’Day has passion for research, and we are excited to see how his work will help CERA deliver new understandings and treatments that make a real difference to people with cancers that impact the eye.
We are also excited by the promotion of two existing team members.
Dr Jennifer Fan-Gaskin is now Principal Investigator of Ocular Fibrosis, where she will continue her important research to prevent scarring after glaucoma surgery.
Scarring after glaucoma surgery can sometimes cause the surgery to fail, causing eye pressure to go back up and the disease to progress. Current drugs to prevent scarring can sometimes lead to side effects and damage to surrounding eye tissue. So Dr Fan Gaskin’s team is investigating the use of several possible drugs, including an anti-oxidant compound, to reduce the risk of side effects.
Dr Zhichao Wu – who this year received a prestigious NHMRC Investigator Grant to further his research – has also been promoted to Principal Investigator and will establish a new clinical biomarkers unit.
One of the major barriers in the discovery of new treatments for conditions like AMD and glaucoma is the need for large, lengthy and costly clinical trials.
Dr Wu’s research aims to fast-track the discovery of sight-saving treatments by identifying new biomarkers that will help assess their effectiveness in a shorter time.
GENE THERAPY PROMISE
The treatment last year, of two young patients with Leber’s Congenital Amaurosis in Sydney with the world’s first approved ocular gene therapy, Luxturna, shows the enormous potential of gene therapy.
Until recently, patients with inherited retinal diseases and conditions with more complex genetic risk factors like dry AMD had no hope of a cure. Now gene therapies are offering real prospects for saving and restoring sight.
There are about 40 clinical trials for various conditions under way globally to develop more treatments like Luxturna, which will be approved for human use.
It’s an exciting time for patients, clinicians and scientists. At CERA, a number of our teams, including my own, are involved in pre-clinical work to develop gene therapies for eye diseases including glaucoma, AMD and Stargardt’s disease.
Our clinical researchers are also partnering with industry sponsors to trial potential new treatments.
In May last year, we joined forces with the team from the Royal Victorian Eye and Ear Hospital to deliver Australia’s first clinical trial of an investigational gene therapy for dry AMD.
The therapy, being developed by Gyroscope Therapeutics Limited, is being assessed in two studies evaluating its safety and effectiveness for the treatment of geographic atrophy (GA) secondary to AMD.
So far, seven patients have received the treatment and we look forward to working with more participants in 2022.
We are also one of five sites in Australia trialling the use of an antioxidant to prevent vision loss from retinitis pigmentosa in people with Usher Syndrome.
These are just two examples of trials which are giving patients with untreatable eye diseases early access to potential new treatments. Both demonstrate just how far research into treatments for inherited retinal diseases has progressed.
Glaucoma continues to be a key focus area for CERA. There is much interest in the next phase of our TAMING (Targeting Metabolic Insufficiency in Glaucoma) trial, which will further investigate the role of nicotinamide, a form of vitamin B3, in preventing vision loss from glaucoma.
After promising results from an earlier trial, I will be working with my colleague Dr Flora Hui on a trial which will involve more than 150 patients in Melbourne, along with participants from interstate and overseas.
Our Macular Research Unit, led by Professor Robyn Guymer, is continuing with an ambitious clinical research program, which will involve hundreds of new participants over the next year.
Natural history studies play a critical role in helping researchers better understand eye disease.
When researchers can observe people with eye disease over a number of years, they get a better idea of how the disease progresses and the impact it can have on a person’s life.
This enables us to give patients a more accurate prognosis – but equally as important, it enables us to identify people who may be suitable for, and interested in, taking part in future trials.
One of the great challenges of trialling many of the potential new gene therapies for inherited retinal diseases (IRDs) is finding patients with the right genetic code to take part in the treatment trial.
In 2022, our Retinal Gene Therapy Unit will continue to build on its collaboration with the University of Melbourne in a natural history study which aims to build a patient database of people with IRDs.
The project, which now involves 150 patients, will give us valuable information about the clinical progress of IRDs.
It will also play a critical role in matching patients with trials as potential new therapies emerge – and we hope it will encourage more trial sponsors to bring their studies to Australia to benefit patients.
FOCUS ON PATIENTS
One of the keys to successful clinical research is the relationship between researchers and participants.
We simply could not do our research without the altruism of people who generously give us their time and trust by agreeing to take part in clinical trials and natural history studies.
This means we need to fully understand what is important to them and their families, ensure that they are well informed about the role of research, and kept up-to-date about potential new treatments on the horizon.
Just one example of this is the work of Honorary Researcher Associate Professor Heather Mack, who is conducting a survey which asked Australians with IRDs about their views on potential gene therapies.
The survey attracted over 700 responses and we are looking forward to the release of the survey results early in 2022.
Knowing how well people with IRDs understand the potential of gene therapy, their expectations, and the support and information they need, will be critical in helping them make informed decisions about participating in research or treatment in the future.
Survey findings will also inform clinician training – ensuring that they are providing accurate and up-to-date information for patients.
And it will be a valuable resource for regulatory agencies and others providing information about gene therapy.
IMPROVING EYE CARE
One of the real positives of clinical research is the impact it has across the health system as more clinicians are exposed to innovative new practices.
At CERA, our work is founded on partnerships across the eye care sector – with ophthalmologists, optometrists, orthoptists and primary health care workers in diverse areas like diabetes care and Indigenous eye health.
We want to support eye health professionals to make a difference for patients. Just one example of this is our founding work on the critical eye care initiative, KeepSight, which is providing an eye check reminder to more than 150,000 Australians with diabetes.
Another is our early role in Oculo – the platform which allows optometrists and ophthalmologists to share patient images to improve clinical outcomes.
In 2022, we will continue to forge new partnerships with eye care practitioners through our health services and clinical research.
We are looking forward to further increasing access to clinical research by utilising telehealth and other new technologies.
Our Clinical Trials Research Centre is also partnering with more suburban ophthalmology clinics to enable their patients to take part in our trials.
Our macular researchers are also expanding their network into optometry, providing vital education on AMD, and providing optometrists with the chance to involve their patients in new studies.
Research is all about collaboration – and in 2022 we look forward to deepening our partnerships and working more closely with eye health professionals to make a real difference for patients.
Professor Keith Martin is Managing Director of the Centre for Eye Research Australia. He is a clinician scientist ophthalmologist and Professor and Head of Ophthalmology at the University of Melbourne. His research focus is on glaucoma, particularly investigating new strategies to protect and regenerate the optic nerve. He was previously Professor and Head of Ophthalmology and Deputy Director of the Centre for Brain Repair at the University of Cambridge.