Using viruses to restore sight by turning eye cells into biofactories
Perth researcher Elizabeth Rakoczy led the world’s first human gene therapy trial for wet age-related macular degeneration (AMD), a leading cause of blindness in the elderly, which affects 112,000 Australians.
Elizabeth has developed a process to turn eye cells into bio-factories, making their own medication on the spot. This gene therapy uses a modified virus to carry a gene into cells in the eye, replacing the need for frequent, painful and costly eye injections (~$2,000 each; six to eight per year).
She will receive a $50K prize and the CSL Florey Medal for lifetime achievement at the annual medical research dinner at Parliament House in Canberra on Wednesday.
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Using viruses to restore sight:
Researcher restoring sight wins $50,000 CSL Florey Medal for lifetime achievement
Award presentation: 9 pm (Canberra time), 6 December in the Great Hall, Parliament House
Full profile, photos, and HD footage available at: www.scienceinpublic.com.au/floreymedal
Professor Elizabeth Rakoczy has developed a process to turn eye cells into bio-factories, making their own medication on the spot.
This gene therapy uses a modified virus to carry a gene into cells in the eye, replacing the need for frequent, painful and costly eye injections.·Elizabeth led the world’s first human gene therapy trial for wet age-related macular degeneration (AMD), a leading cause of blindness in the elderly.
Past CSL Florey Medallists include Graeme Clarke, Ian Frazer, and Nobel Laureates Barry Marshall and Robin Warren.Elizabeth Rakoczy is modifying viruses to use their powers for good. She’s created a new gene therapy for wet AMD that is reversing vision loss in clinical trial patients. Her treatment means one injection instead of several per year.
Modified viruses are gene therapy’s delivery vehicles, taking genes directly into cells. Elizabeth 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, a complex eye disease.
More than 112,000 Australians have wet AMD—the most devastating form of AMD—and up to 8,000 more commence treatment for it each year. Each injection of the current treatment costs about $2,000, and patients have six to eight per year. Costs will rise with Australia’s ageing population. Gene therapy offers an alternative.
Elizabeth hopes to adapt her bio-factory idea to other diseases to alleviate suffering.
The CSL Florey Medal has been presented every two years since 1998 by the Australian Institute of Policy and Science (AIPS). The award recognises a lifetime of achievement in biomedical science and human health advancement. It carries a cash prize of $50,000 and has been supported by CSL since 2007.
“Professor Rakoczy is a quiet achiever, a world leader in gene therapy, and a key contributor to advancing international eye research,” says CSL’s 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.”
“In winning the CSL Florey Medal, Professor Rakoczy joins an elite group of Australian medical researchers who have followed in the footsteps of Howard Florey,” says AIPS director Camille Thomson.
“To quote Sir Robert Menzies, ‘In terms of world wellbeing, Florey was the most important man ever born in Australia’.”
Professor Elizabeth Rakoczy is the founding Director of the Department of Molecular Ophthalmology at Lions Eye Institute, University of Western Australia.