The news that Health Canada has approved gene therapy Luxturna to treat blindness couldn’t come at a better time, as October is Blindness Awareness Month. You don’t have to look far to find other high impact research on blindness happening right here in Toronto, Canada.
Dr. Molly Shoichet, University of Toronto (UofT), is a world renowned bioengineer with expertise in the areas of polymer synthesis, biomaterials design and drug delivery in the nervous system. Her research focuses on strategies to promote tissue repair after traumatic spinal cord injury, stroke and blindness. She enhances tumour targeting and drug screening via 3D cell culture by using hydrogels.
Recently, her lab developed a cell injection technique that could help reverse vision loss.
Two major causes of vision loss are age-related macular degeneration (AMD) and retinitis pigmentosa. Both conditions occur when cells die in the retina.
Dr. Shoichet and her team have figured out how to inject healthy photoreceptor and retinal pigmented epithelium (RPE) cells, at the same time, into damaged retinas in a mouse model. Read an article about her work written by Tyler Irving for Medicine by Design, one of the lead funders. You can also read her co-authored paper in the journal Biomaterials.
As you can appreciate, this work builds on earlier research conducted at UofT. Dr. Shoichet, along with Drs. Derek van der Kooy and Cindi Morshead, and a team of scientists and engineers that included Brian Ballios and Michael Cooke, made an important discovery for stem cell transplantation in the eye. (You can read about that earlier research here and a breakthrough paper by Dr. van der Kooy, “Retinal stem cells in the adult mammalian eye,” in Science.)
Dr. Andras Nagy, Mount Sinai Hospital, is another local scientist with a global reputation and significant discoveries to his credit. Since 2009, Dr. Nagy has been refining his non-viral method of creating stem cells from other cells of the body to identify possible cures for spinal cord injury, macular degeneration, diabetes, Parkinson’s disease and other devastating diseases.
He is working to treat wet-AMD using a combination of stem cell therapy and gene therapy. He and his team have figured out a way, using genetic engineering, to program stem cells to deliver proteins within the eye, removing the need to manually and regularly inject these sight-saving proteins into a patient’s eye. His research is funded by Fighting Blindness Canada. You can learn more about it here.
Fighting Blindness Canada (FBC) is a leader in supporting blindness research in Canada. Over the last 45 years, FBC has invested more than $40 million to educate Canadians about vision loss and fund research into treatments and cures. According to their site, FBC has “supported over 200 research grants that have led to over 600 new discoveries in areas such as stem cell research, neuroprotective therapies, technological developments, pharmaceuticals, and gene therapies.” FBC provides a wealth of information for patients and researchers, including descriptions of the research it funds across Canada.
Watch this video to learn about gene therapy for inherited retinal diseases, from the American Society of Gene and Cell Therapy.