Jun 4, 2019

Exploring the Possibilities of Stem Cell Therapy

With so many exciting possibilities in stem cell and gene therapy on the horizon, how does Fighting Blindness Canada choose which projects to fund?

The answer is simple: we fund the best, most promising research into treatments and cures for blinding eye diseases. That’s why we’re so proud to fund Dr. David Gamm’s research into a potential stem cell therapy for retinitis pigmentosa (RP) as part of our Restore Vision 20/20 research initiative!

Dr. Gamm’s research explores the possibility of using a stem-cell treatment to replace photoreceptors in people living with retinitis pigmentosa, which is a blinding eye disease that involves tunnel vision and night-blindness. While RP is recognized as a “rare disease,” it is one of the most common, yet rare, eye diseases, affecting between 1 in 3500 to 1 in 4000 Canadians. In RP, photoreceptors, which are the specialized rod and cone cells that respond to light in the retina, become damaged. By replacing defective and dying photoreceptors, Dr. Gamm’s research team hopes to develop a broad therapeutic approach to treat RP and other eye diseases that involve the loss of photoreceptor cells.

But what do you replace defective photoreceptors with? Dr. Gamm’s team believes that the answer lies with a special kind of stem cell called an induced pluripotent stem cell (iPSC). These cells can be coaxed to develop into photoreceptor cells, which can then be transplanted into the eye. Once transplanted, Dr. Gamm has shown that these new photoreceptors have the ability to respond to light.

It’s an exciting prospect – one which holds great potential for many of the inherited retinal diseases (IRDs) that cause photoreceptor death. Unlike gene therapies, which treat blinding eye diseases by targeting a specific gene that causes blindness, stem cell therapies aim to restore vision by replacing damaged cells with newly functioning cells. Because stem cell therapies are not “gene specific” the hope is that a photoreceptor replacement therapy might work for a variety of different diseases that involve the loss of photoreceptors. Furthermore, many gene therapies work to preserve, rather than restore, retinal function. This means that gene therapies can only work if a patient has surviving photoreceptors in their eye. In contrast, a stem cell therapy approach would replace dying photoreceptors regardless of the genetic defect in question, which means it could be applicable to a much broader patient group.

As with all of our Restore Vision 20/20 recipients, Fighting Blindness Canada chose to fund Dr. Gamm’s research because it holds tremendous potential for restoring sight for people living with blinding eye diseases. This world-class research is moving towards a first-in-human clinical trial, meaning that it is close to becoming an experimental treatment that will be ready to test in patients.

These translational research projects simply aren’t possible without the generosity of the incredible donors who support Fighting Blindness Canada. The Restore Vision 20/20 Initiative was made possible thanks to Donna Green and her mother, Goldie Feldman, as well as an anonymous donor. Together, they have contributed $2.5 million to drive the development of sight-restorative treatment for retinitis pigmentosa! Thanks to the incredible work of researchers like Dr. Gamm and donors like Donna and Goldie, treatments for blinding eye diseases truly are on the horizon.

Want to learn more about Dr. Gamm’s exciting FBC-funded research? Click here to watch a video of Dr. Gamm sharing his findings with the FBC community at a Vision Quest educational event in Vancouver! This page also includes a detailed written summary of his presentation.