This is a milestone in the Foundation Fighting Blindness’s 34 year history. It involves five research teams, four at prominent universities in Canada and one in the U.S. It’s also the first time that the CIHR is partnering with a health charity on a grant to develop therapies for degenerative eye diseases. The partnership will provide a much needed boost to eye research, which still receives significantly less funding in Canada than other major disease areas such as diabetes or cancer.
“This is a huge milestone for the Foundation Fighting Blindness and represents a historic shift in the type of vision research being funded,” said Sharon Colle, President and CEO, FFB. “We’re taking the knowledge generated through decades of research into causes of these diseases, and are now applying it to the development of long awaited therapies.”
The five research teams involved will provide various specialized skills required for the success in this research project. The project will be led by Dr. Robert Molday, a cell biologist from the University of British Columbia. The team’s other experts in gene therapy are Dr. Jim Hu from the University of Toronto, and Dr. Bill Hauswirth from the University of Florida. Dr. Marinko Sarunic of Simon Fraser University will be responsible for the retinal imaging component of the project. As the team’s clinician-scientist, Dr. Robert Koenekoop will oversee the visual function testing and the gene analyses, first in animals and then in humans with a variety of retinal degenerations.
The strategy is to replace the defective gene with a “new healthy gene” in specific animal models for retinal degenerative diseases with the aim of slowing photoreceptor loss and partially restoring vision. Success in these animal models would lead to future human clinical trials.
"The application of gene therapy for three retinal degenerative diseases will be investigated: Stargardt macular dystrophy, Leber congenital amaurosis (LCA), and retinitis pigmentosa," Dr. Molday explains. “The recent success in gene therapy for RPE65 has been highly conclusive for LCA; we believe that we can learn from this and advance even more quickly this time.”
RPE65 mutations are one cause of Leber congenital amaurosis. Three independent research teams have very recently shown that injecting a healthy version of that gene to young adults can partially restore their vision.
Canadian Institutes of Health Research (CIHR)
CIHR is the Government of Canada's agency responsible for funding health research in Canada. Nine projects were awarded grants through the CIHR program entitled “Regenerative Medicine and Nanomedicine – Emerging Team Grants – July 2008.” All of these projects hold great hope for medical applications in the fields of nanotechnology, stem cells, tissue engineering and rehabilitative sciences. To receive a grant, a team must show a multidisciplinary commitment to addressing problems in regenerative medicine. The long-term goal of this program is to develop innovative treatments that are scientifically based and socially validated.
Foundation Fighting Blindness (FFB)
The Foundation Fighting Blindness (FFB) is Canada's largest private contributor of vision research, thanks to its generous donors and long-time annual fundraisers, Comic Vision & Ride for Sight. Since its inception in 1974 the FFB has funded dozens of research discoveries to identify the causes of genetic forms of blindness at universities and hospitals across Canada. Today, these discoveries have helped bring scientists to this very exciting time in vision research, translating knowledge into treatments to restore the gift of sight.