In a few years’ time, children born with a genetic blinding condition known as Familial Exudative Vitreoretinopathy (FEVR) may no longer have to fear losing their sight.
The condition is caused by a mutated gene that results in the improper development of veins and arteries in the retina.
The FEVR gene was first discovered by Dalhousie Medical School ophthalmologist, Dr Johane Robitaille, in 2002 – and the Nova Scotia-based researcher believes she may now have found a drug to stop the genetic effects from the mutated form.
Fellow Dalhousie University researcher, Dr Christopher McMaster, told OT that under normal circumstances, the gene acts to prevent the excessive development of blood vessels. However, in its FEVR form, this results in an incurable form of vision deterioration from retinal scarring.
The new drug works by giving blood vessel development a free rein, Dr McMaster said. It has so far proved successful in treating animals with a form of the disease.
After a $2m award from the Atlantic Canada Opportunities Agency, the team are now looking to start a clinical trial within four years.
Dr McMaster explained: “The drug candidate is being administered by intraocular injection in animal models of FEVR, so this is likely the route that it will initially be administered to patients enrolled into clinical trials as it enables precise delivery and dosing.”
Dr McMaster and Dr Robitaille have hopes for the drug beyond children diagnosed with the FEVR condition. They also plan to evaluate its potential for babies born with retinopathy of prematurity.
Dr McMaster highlighted that: “When a baby is born prematurely, they’re put into a high oxygen setting. That messes the signals that allow the retinal vessels to grow normally. So these babies can end up with the same problem as FEVR babies.”
Dr Robitaille added: “Theoretically, if the drug we have works for one condition, it should work for the other…And the treatment would be for a short period of time. Because once the retina’s vessels are developed, they’re developed.
“If you can get two months’ or so worth of treatments and be good for the rest of your life, that’s a huge step forward,” she emphasised.
Image credit: Axelle B