The eyes have it: Elizabeth Rakoczy wins Florey Medal for wet AMD gene therapy

Molecular ophthalmologist Professor Elizabeth Rakoczy has been awarded the 2017 CSL Florey Medal for the creation of a new gene therapy for wet age-related macular degeneration (wet AMD) — the most common cause of blindness in the developed world.

Established by the Australian Institute of Policy and Science (AIPS) in 1998, the Florey Medal recognises significant lifetime achievement in biomedical science and/or human health advancement. Supported by CSL since 2007, the medal carries a $50,000 prize and has in the past been awarded to such esteemed names as Graeme Clark, Ian Frazer and Nobel Laureates Barry Marshall and Robin Warren.

This year, the award recognises Professor Rakoczy — the founding director of the Department of Molecular Ophthalmology at Lions Eye Institute (LEI), University of Western Australia (UWA) — for her contribution to the development of a new gene therapy for wet AMD. The disease causes central vision loss in more than 112,000 Australians and up to 8000 more commence treatment each year in the form of invasive eye injections. Each injection costs about $2000 and patients have six to eight per year.

The new gene therapy, which is proving to be safe and well tolerated in human trials, promises to replace monthly injections with a one-off treatment in which modified viruses take genes directly into cells. Professor Rakoczy first showed that they could carry a healthy replacement for a mutated gene that causes degeneration of the eye’s retina. She then showed they can deliver instructions for eye cells to produce their own treatment for wet AMD.

“It is our hope that in the next few years, millions of people suffering from wet AMD will be able to have single-injection therapy to control their condition,” Professor Rakoczy said.

The science behind the new treatment began more than 20 years ago when Professor Rakoczy was initially recruited to UWA and the LEI. It was the first research in Australia using gene therapy in ophthalmology or any other medical field and was named by the National Health and Medical Research Council in its 10 of the best national research projects in 2005.

Professor Rakoczy said her research demonstrates how a scientific discovery could make a fundamental difference, noting, “I have been fortunate to be around when recombinant gene technology became available so we could turn infectious viruses into useful delivery vehicles to develop localised ‘biofactories’ of a desired medication — in this case, in the back of the eye in the retina.”

Professor Elizabeth Rakoczy, winner of the 2017 CSL Florey Medal. Image credit: Lions Eye Institute, UWA.

Professor Rakoczy paid tribute to the more than 50 scientists, cell and molecular biologists, physicists, statisticians, virologists, veterinary scientists, ophthalmologists and students who worked together to bring the treatment to fruition. She additionally hopes to adapt her biofactory idea to other diseases to alleviate suffering.

“Professor Rakoczy is a quiet achiever, a world leader in gene therapy and a key contributor to advancing international eye research,” said CSL Chief Scientist Dr Andrew Cuthbertson. “CSL is proud to support this award, which recognises excellence in research as well as creating role models for the next generation of medical researchers. Gene and cell therapies hold the potential to significantly reduce vision loss over a patient’s lifetime, which is why work in this field is so important.”

Top image credit: ©iStockphoto.com/Brian Jackson