Jul 26, 2016

Breaking News: Six New FBC-Funded Research Projects Announced!

Research Data

We are thrilled to announce the six winning research projects from Fighting Blindness Canada’s annual grant competition! Keep reading to learn how these projects are fueling the development of new treatments for blinding eye diseases and watch this video to learn more about how they were selected.

Development of a Drug that Prevents Photoreceptor Death and Vision Loss
Dr. Philippe Monnier, Krembil Research Institute, University Health Network, Toronto ($240,000 over 3 years)
Before a new treatment can be tested in people, scientists need to show that it is both safe and effective in laboratory models of disease. During the earliest phases of research, scientists focus on discovering something truly novel and meaningful. More than a decade ago, Dr. Monnier made a ground-breaking discovery when he learned that a protein called neogenin plays a critical role in the life and death of neural (brain) cells. Since his discovery, Dr. Monnier has focused on developing neogenin-based treatments. With earlier funding from Fighting Blindness Canada (FBC), Dr. Monnier demonstrated that he could prevent vision loss in two different laboratory models of retinitis pigmentosa (RP) by manipulating neogenin. With renewed funding from FBC, Dr. Monnier is now focused on gathering essential pre-clinical data to enable a clinical trial testing a neogenin-based treatment for people living with RP.

Impact of Obesity on Age-Related Macular Degeneration
Dr. Przemyslaw (Mike) Sapieha, Hôpital Maisonneuve-Rosemont ($240,000 over 3 years)
Did you know that obesity is the second most important environmental risk factor for wet age-related macular degeneration (AMD)? Dr. Sapieha will lead a study to understand the relationship between high-fat diets and vision loss. To do this, Dr. Sapieha will explore how the microbes of our body (the microbiome) affect obesity, the body’s immune response, and ultimately the progression of AMD. By studying new wet AMD pathways, he aims to identify new therapeutic targets for the disease.

Defining the connections and survival mechanisms of retinal interneurons
Dr. David Picketts at Ottawa Hospital Research Institute ($240,000 over 3 years)
Many of the emerging treatments for blinding eye diseases are focused on replacing damaged photoreceptors or saving them from dying in the first place. The effectiveness of these treatments depends on restoring the function of photoreceptors, but they also depend on the eye’s other cells, which must survive and remain active. Some studies have shown that the eye’s inner neurons (the cells that connect the photoreceptors to the optic nerve) become damaged in degenerative diseases. Dr. Picketts will study how inner retinal neurons mature, what factors are important for their survival and how they integrate and modulate photoreceptor signals. This fundamental knowledge will increase the success of emerging therapies to treat retinal degeneration.

Why does the drug valproic acid (VPA) prevent some forms of blindness and make others worse?
Dr. Orson Moritz at the University of British Columbia ($240,000 over 3 years)
Dr. Moritz has shown that the drug valproic acid (VPA) prevents blindness in some genetic forms of RP but worsens it in other forms. To learn more, his research team performed similar tests with a group of drugs called histone deacetylase inhibitors, which are often used in cancer treatments, and discovered the same effects. In this study, Dr. Moritz will examine how and why these effects occur in different forms of RP. To do this, they will identify the exact enzymes (within the drugs) that are involved in the effects and identify the pathways that are impacting vision loss. They will test if VPA and histone deacetylase inhibitors function by increasing a cellular process called autophagy and examine if drugs that increase autophagy can be used to prevent blindness.

Whole genome sequencing of patients with early onset retinal dystrophies
Dr. Elise Héon, The Hospital for Sick Children (SickKids) ($240,000 over 3 years)
Retinal degenerations that affect young people are caused by changes in the genome (mutations). These errors can be identified with a technique called sequencing. Identifying mutations is important because mutation-specific treatments are currently being developed. At SickKids, Dr. Héon and her team are only able to identify mutations in approximately half of the people who are diagnosed with a retinal degenerative disease. In this project, Dr. Héon will use the latest sequencing technology and a targeted strategy to identify the most number of mutations possible. The team will also design experiments to gain a better understanding of why these mutations lead to retinal degeneration. This new knowledge will be important to patient counseling, to researchers and to those developing therapies.

Identifying New Drugs to Treat Retinitis Pigmentosa
Dr. Belinda Chang, University of Toronto ($40,000 over 1 year)
The blinding eye disease, retinitis pigmentosa (RP), is one of the most common forms of vision loss in young people and children, affecting approximately 1 in every 3,500 Canadians. Unfortunately, there are no treatments to stop the steady loss of vision. This genetic eye disease results from mutations to a variety of different genes, but the most common gene involved is called rhodopsin. Dr. Chang’s research is focused on identifying new sight-saving drugs for retinitis pigmentosa. To do this, the team has developed an innovative and highly-effective new test that will allow them to quickly screen a large volume of potential drug candidates. At the same time, this test will allow them to gain critical new knowledge about how and why changes to the rhodopsin gene cause this degenerative eye disease.

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How did the FFB select these 7 projects? (VIDEO)

https://youtu.be/fLE6sIY-ouY

[The visuals in the video feature footage from our 2016 Scientific Advisory Board meeting]

The main goal of FBC’s research program is to drive the development of new treatments for blinding eye diseases. Every single project in the research portfolio undergoes a rigorous selection process, which includes a process of “peer review” involving a team of vision research experts who make up FBC’s Scientific Advisory Board (SAB). These experts volunteer their time to carefully study each project that is submitted to the competition. As they review the project they ask the following questions: Is this project innovative? Will it lead to discoveries that are important for new treatments? Does the research team have the expertise to do the work? Do they have promising preliminary evidence? How does the project compare to others that have been submitted? What do we need to know about vision to drive the development of more treatments? The peer review process allows the SAB to identify the top projects with the greatest potential to drive FBC’s goal of developing new treatments. The SAB makes recommendations to FBC’s Board of Directors, who are responsible for selecting the projects with the greatest ability to achieve FBC’s vision of restoring hope and sight. Thank you to all of the generous FBC donors who make all of this research possible!

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