Brian Ballios

Research Synopsis

Retinal degenerative diseases are disabling conditions affecting the vision of a significant number of Canadians.  Acquired conditions, such as age-related macular degeneration (AMD) or diabetic retinopathy, are the leading causes of irreversible blindness in senior and working-age adults, respectively.  The prevalence of these conditions is on the rise.  For example, AMD currently affects more than 2 million Canadians (6.5%) over the age of 50, and is the leading cause of irreversible blindness.  With our aging population, the prevalence of AMD is expected to increase by 97% in the next thirty years.  Treatments are aimed at slowing the progression of vision loss, but do not represent a regenerative approach to retinal repair.  While less prevalent, inherited retinal disorders (IRDs) affect an estimated 90,000 Canadians, with a total cost of disease – including healthcare costs, productivity and well-being – at upwards of $6.7 billion.  IRDs include conditions such as retinitis pigmentosa, Stargardt disease, Leber congenital amaurosis, choroideremia, cone (and cone-rod) dystrophy, and achromatopsia.  More than 270 genes have been identified for inherited monogeneic retinal disease.  With rare exception, treatments do not exists for inherited conditions.

New therapies for retinal degeneration are focused on the next generation of regenerative medicines.  These include gene and cell-based therapeutics, including stem cells.  Several of these approaching are already being applied in clinical trials and therapies.  While gene therapy has the potential to correct the underlying mechanism of disease in monogeneic disorders, it depends on the presence of viable light-sensitive cells.  Stem cell therapy has the potential to replace the light-sensitive photoreceptors lost in later-stage disease, when patients have suffered significant vision loss.  Cell-based therapies hold promise for both IRDs and acquired conditions such as AMD.

Our laboratory and clinical work is focused on:

[1] Understanding pathobiology of retinal disease, by establishing translational models of retinal degeneration;

[2] Discovering new therapeutics to treat retinal disease, through expertise in retinal and stem cell biology;

[3] Integrating new technologies, to enhance the performance of cell-based retinal therapies; and,

[4] Developing and applying preclinical technologies to execute first-in-human clinical studies

The overall goal of our work is to cure retinal blindness by discovering new therapies for inherited and acquired disease.