Dr Tom Edwards wants to use the infectious power of viruses to develop cures for blinding eye disease using gene therapy.
More than 16,000 Australians live with an inherited retinal disease (IRD). One type, retinitis pigmentosa, is the leading cause of blindness in the working-age population. Currently, there are no cures or treatments.
“The new gene therapy research gives me hope that in my lifetime there will be a treatment for retinitis pigmentosa,’’ says Kate Barrett (pictured right, with her sister).
She was diagnosed with the condition at the age of six and is now, aged 35, starting to experience some deterioration of her eyesight.
The National Stem Cell Foundation of Australia joined with the Centre for Eye Research Australia (CERA) on World Sight Day (14 October 2020) to raise money to fight genetic retinal diseases like retinitis pigmentosa, which cause progressive, irreversible vision loss.
That meant people could triple the impact of donations made on the day, with the two organisations pledging to match public donations dollar-for-dollar up to $50,000.
The $150,000 target was met and surpassed, totalling $165,000 by the end of the day, giving new hope for people like Kate and her family.
The donations will support the work of Dr Tom Edwards who is developing gene therapies to treat the diseases.
Tom uses stem cells derived from patient skin biopsies to create affected eye cells for his research.
“Thanks to the exciting progress being made in this field, for the first time we’re able to tell patients there’s hope,” he says.
Video: Kate and Nicole Barrett's story and Tom's research (credit: CERA)
Tom’s project is one of four selected by the Foundation in 2020 to receive funding to match donations from other sources, to a cap of $50,000 from the Foundation per project.
IRDs affect adults and children alike. For most young people, vision loss is due to a ‘spelling mistake’ in the genetic code that causes cells to malfunction.
Gene therapies work by correcting the disease-causing spelling mistakes in a person’s genetic code. Modified viruses are the delivery vehicles.
“When you get a common cold, that’s an example of a virus delivering its trouble-making genetic material into your body,” Tom explains.
“We are borrowing this ability to deliver a genetic payload of DNA to dysfunctional retinal cells.”
Tom’s project has met its target, but you can still donate to his research via the Foundation website, and we will pass it on.
“We’re particularly excited by the potential for Dr Tom Edwards’ gene therapy approach to be applied to many different inherited eye diseases, not just one,” says Foundation Chairman Dr Graeme Blackman AO.