It’s fitting that as we enter 2020, the year of vision, we’re seeing science theory turn into life-changing treatments for people with vision loss. We’re seeing the promise of personalized medicine become a reality with lab discoveries reaching clinical trials for treatments in stem cell and gene therapy.
Gene therapy is a new kind of treatment that works by delivering a functioning copy of a gene directly into the eye. This newly-introduced, functioning gene acts as a treatment by replacing a gene that’s not working properly. After just a single treatment, gene therapy has the potential to restore sight, providing lifelong benefits.
The first targeted gene therapy was approved by the U.S. and European health authorities in 2018. It sets the precedent for the innovative new therapies on the way. It restores partial sight for individuals who have Leber congenital amaurosis (LCA) caused by mutations in the RPE65 gene.
From innovation to patient care
Jack McCormick was born with LCA and mutations in the RPE65 gene. He always had poor vision, but his condition worsened in his teens and he now uses a guide dog. He can see light and some shadows but can’t see a person’s face, even in good light. In poor light, he sees almost nothing.
“I’m so excited that research has delivered a potential treatment to restore my sight. I’m so hopeful that I may be able to see again, with the life skills of perseverance and determination that I learned growing up while losing my sight,” he says.
When this gene therapy treatment is submitted for approval in Canada, it will also be the test case for future gene therapies and personalized drugs. It’s clear that not only do we have to continue to support the research, from basic science through clinical trials, that have made this treatment a reality, but we have to ensure that Canadians have access to it — access that is equal, timely, and affordable. We know that science takes not years but decades to move an idea through to treatment. Our investments are starting to pay off and it’s vital that we work together to ensure that the hope and the cure can be a reality for Canadians.