Florey Medal awarded to prominent ophthalmologist

The Florey Medal, established by the Australian Institute of Policy and Science, recognises significant lifetime achievent in biomedical science and/or human health advancent. Rakoczy, a researcher at the Centre for Ophthalmology at the University of Western Australia (UWA) and also the Lions Eye Institute (LEI), was presented with the award and a $50,000 prize in Canberra last week.However, while thrilled with the award, Rakoczy was quick to also highlight the work of her team and the significance of the research they had been conducting.{{quote-a:r-w:350-I:2-Q:“The technological approach that we have been using will be the standard of 21st century medicine.”-WHO:Elizabeth Rakoczy, molecular ophthalmologist}}“I was really delighted, not just for myself, but my group – I accepted it in the name of all the people that I worked with on this project. More importantly, this is the first time that this significant award went to a brand new technology,” she explained.“The technological approach that we have been using will be the standard of 21st century medicine, so in this sense I think it is very important.”LEI managing director Professor David Mackey said the Florey Medal recognised that Rakoczy’s body of work was of international importance and an example of a basic research project that could be translated into a revolutionary treatment for patients.Rakoczy’s groundbreaking research was the first in Australia to use gene therapy for ophthalmology and was also recognised as one of the 10 best national research projects by the National Health and Medical Research Council (NHMRC) in 2005. Phase 1/2A human trials of the therapy have so far been conducted on 40 people with wet AMD and proved safe and well tolerated, despite the fact that some of the subjects were over 90-years-old and gene therapy had never been used on such an advanced age group.While the trials were only focused on testing the safety of the therapy, anecdotally Rakoczy said there had been positive signs in terms of preventing vision loss as well. If successful, the therapy has the potential to replace expensive monthly anti-VEGF injections with a one-off treatment.“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,” Rakoczy said.“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 the desired medication. It’s a nice approach because, particularly with subretinal injection, we don’t have to wory about immune rejection because the protein is naturally there.”Bearing any unforeseen obstacles, it will become the first gene therapy ever approved for treating a very common disease. The technology has already been licensed to a start up company in the US and Rakoczy expects it should be available in the next 3–5 years.