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Jul 14, 2015

Three-year FBC Grant Ends With New Discovery

Turning research into treatments takes a long time. Some studies have estimated that approximately 14% of new discoveries make it into the clinic, and that this journey from the laboratory into the clinic takes an average of seventeen years (Pozen and Kline 2011). At Fighting Blindness Canada (FBC), we are working every day to beat these odds because WE KNOW IT IS POSSIBLE! For inspiration, just look to FBC-funded scientist, Dr. Ian MacDonald, who recently embarked on a new clinical trial to treat a childhood form of blindness, choroideremia.

Creating new sight-saving therapies requires a tremendous investment of resources from a variety of stakeholders, including researchers, doctors, corporations, governments and patients. Drs. Robert Gendron and Hélène Paradis, at Memorial University in Newfoundland, are in it for the long haul. Along with a team of collaborators, Drs. Gendron and Paradis are searching for better treatments for age-related macular degeneration (AMD) and diabetic retinopathy. In both of these diseases, vision loss is associated with leaky blood vessels in the eye.

With funding from Fighting Blindness Canada, the team discovered a new protein, Tubedown (that is present in the normal eye), which helps to prevent retinal blood vessels from leaking in experimental models. Along with their collaborators, the team studied what happened when Tubedown signalling was not working properly. Importantly, they discovered that people with wet age-related macular degeneration and/or people living with diabetic retinopathy have a loss of Tubedown expression in blood vessels of the retina.

Although Drs. Gendron and Paradis’s team most recent 3-year operating grant recently came to a close, their research never stops. There is still much more to learn about how manipulating the Tubedown pathway might lead to new sight-saving therapies. Fortunately, the completion of the grant coincided with a wonderful discovery! After more than a decade of work, Drs. Gendron and Paradis’s team have discovered important details about how Tubedown works—namely, Tubedown works with other proteins (the c-Src/Cortactin pathway) to maintain the health of blood vessels in the eye. These findings now provide the rationale and tools for designing alternative prevention and therapy for neovascular retinopathy.

These findings that link the experimental work in the laboratory to different patient communities are key because they illustrate how new targets for drug development for retinal disease are discovered and how they might be ultimately used to help people suffering from retinal diseases which share common regulatory pathways.

At Vision Quest, our annual education conference, one of our goals is build stronger, more meaningful connections between all of the different people who are committed to turning research into new therapies, including researchers like Drs. Gendron and Paradis, as well as doctors, pharmaceutical and biotechnology companies, regulators, government officials, and people living with vision loss and their families.

About the Study

The study was published in Biology Open and is titled “Tubedown regulation of retinal endothelial permeability signaling pathways.” Research was conducted collaboratively by Nhu Ho, Robert L. Gendron, Kindra Grozinger, Maria A. Whelan, Emily Anne Hicks, Bimal Tennakoon, Danielle Gardiner, and Hélène Paradis all from Memorial University and William V. Good from the Smith-Kettlewell Eye Research Institute.

Cited Works:

Pozen, R. and Kline, H. 2011. Defining Success for Translational Research Organization. Science Translational Medicine, vol 3, issue 94.

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