New gene therapy trial helps people with genetic eye disease
Alberta Innovates – Health Solutions awards $5 million to Alberta Ocular Gene Therapy Team
Oct. 9, 2013
UPDATE, June 2015: Dr. Ian MacDonald and his AIHS-funded team are now doing clinical trials of this pioneering treatment. The treatment is intended to stop Choroideremia in its tracks with a single injection of what’s known as a viral vector—a small harmless virus that’s been modified to carry into the eye the ‘good’ gene needed to potentially prevent further loss of sight and to restore the vision of his patients—often with noticeable results in under a month.
“We’re using the natural properties of a virus, its ability to inject its DNA into human cells, to introduce a working copy of the needed gene into the retinal layers,” says Stephanie Chan, genetic counsellor and study co-ordinator. “The idea is that those cells will take up the good gene and use it to create the correct protein, replacing the defective gene that’s in the cells.” (From MedicalXpress.com)
Read more about the trial and latest developments in these recent news stories:
MedicalXpress.com – Pioneering gene therapy takes aim at inherited blindness
Edmonton Journal.com – Edmonton man first in Canada to receive retinal gene therapy
Edmonton Sun.com – Canada’s first human gene trial for eyes underway
Alberta Health Services news release June 29, 2015 – Pioneering gene therapy takes aim at inherited blindness
AIHS News Release: (Edmonton, Alberta) October 9, 2013… Mark Huyser-Wierenga, a crown prosecutor with the province of Alberta, cherishes his limited sight. Since he was fifteen years old, Mark’s vision has been slowly deteriorating. He can no longer ride his bike to work or live the active life he was used to living. Mark has Choroideremia, a genetic eye disease that he inherited from his mother. Researchers Ian MacDonald, MD and Tania Bubela, PhD are hoping to change things for Mark and others who have inherited genetic eye diseases. They have been awarded a Collaborative Research and Innovation Opportunity (CRIO) Team grant for up to $5 million for the next five years, from Alberta Innovates – Health Solutions (AIHS).
Drs. Ian MacDonald and Tania Bubela are leading a team of researchers, clinicians, and academics as part of the Alberta Ocular Gene Therapy team. They are starting to test a new treatment, utilizing gene therapy, for genetic eye diseases like Choroideremia. The first of its kind in Canada, the team hopes this clinical trial will lead to gene therapy that could improve or restore vision loss in those affected. They are optimistic that someday, those carrying the gene could be treated before any vision loss begins.
The Alberta Ocular Gene Therapy Team is working with ophthalmologists within Alberta and the Foundation Fighting Blindness Patient Registry to identify study participants who will enroll in this phase 1 trial. It is currently seeking patients who have Choroideremia. The trial will last for three years and will examine the safety protocol of the gene therapy method that the team is employing. If favorable results come from the phase 1 trial, this therapy will move into a larger phase 2 trial.
“Harnessing the potential of collaborations across the spectrum of health research activity to solve unmet health and health system needs is an approach AIHS is committed to through our programs of funding and support,” says Dr. Cy Frank, AIHS President and CEO. “Drs. MacDonald’s and Bubela’s teamwork demonstrates the caliber of activity that we are proud to support through our CRIO Teams and other programs.”
As a result of the investment from AIHS, the Foundation Fighting Blindness (FFB) also contributed to the Alberta Ocular Gene Therapy Team. An investment of $500 thousand will go to the team to help establish the clinical trial. “This clinical trial exemplifies the power of partnerships to move us closer to treatments and cures for devastating genetic eye diseases,” said Sharon Colle, President and CEO of the Foundation Fighting Blindness, one of three funding partners of this ground-breaking trial. “Our community is eagerly watching Dr. Ian MacDonald’s clinical trial – it holds so much hope, for people living with Choroideremia and over 1 million Canadian families affected by retinal eye diseases.”
One million Canadian families affected by retinal eye diseases.
Choroideremia is a condition characterized by progressive vision loss that mainly affects males. Choroideremia affects around 1 in 50,000 individuals. An ongoing loss of cells (atrophy) in the specialized light-sensitive tissue that lines the back of the eye (retina) and a nearby network of blood vessels (the choroid), cause vision to deteriorate. The vision impairment in choroideremia worsens over time and all individuals with this condition will develop blindness, usually in late adulthood.
Support for the Alberta Ocular Gene Therapy Team comes from a wide variety of sources, including a Canadian Institutes for Health Research (CIHR) Rare Diseases Emerging Team Grant, funding from Foundation Fighting Blindness, the Choroideremia Research Foundation Canada, Canada Foundation for Innovation (CFI), Alberta Enterprise and Advanced Education, and the University of Alberta, Department of Ophthalmology “Out of Sight” program.
The funding will create a Centre of Excellence for Ocular Gene Therapy at the University of Alberta. It will support a full translational platform to bring gene therapy research to the clinic. The platform will build on the “first” for Canada gene therapy clinical trial for Choroideremia.
Visit CHM Gene Therapy at University of Alberta for more information on this trial and to determine whether you are eligible.
The Foundation Fighting Blindness is Canada’s largest charity for sight-saving research and has contributed over $24 million to find the causes, treatments and cures for retinitis pigmentosa, macular degeneration and related blinding eye diseases. You’ll see: results thanks to Foundation-funded discoveries, made possible by its generous donors. The Foundation is also known by its signature events: Comic Vision, Cycle for Sight, Ride for Sight, and Vision Quest. Visit www.ffb.ca or call 1.800.461.3331 for the latest in vision research.
Alberta Innovates – Health Solutions supports top-quality, internationally competitive health research. AIHS research seeks to further our understanding of health and disease, and to produce results that will make a difference to the health, economy and societal wellbeing of Albertans and people around the world.