Irvine, Calif. – April 2020 – Center of Translational Vision postdoctoral scholar Henri Leinonen, PhD, in the Department of Ophthalmology at the UCI School of Medicine, was awarded a $70,000 pediatric ophthalmology career-starter research grant from the Knights Templar Foundation, Inc. to study “Development of a disease-modifying therapy for inherited retinal dystrophies by a drug repurposing strategy.”
The Knights Templar Eye Foundation is committed to support research that can help launch the careers of clinical or basic researchers committed to the understanding, prevention and cure of vision threatening diseases in infants and children. They support clinical or basic research on conditions that can advance treatment or prevention.
Inherited retinal degenerative (IRDs) diseases lead to visual decline and many of them can cause blindness by adolescence. IRDs are a devastating burden for affected individuals and their families. The combined prevalence of IRDs is relatively high in Western countries as roughly one in 2000 people can be affected. Unfortunately, preventive therapies do not exist for these diseases.
A treatment that would decelerate retinal degeneration regardless of the underlying pathology could bring about remedies expeditiously for the treatment of IRDs, especially if drugs that already have been accepted for clinical use in other indications could be repurposed. We have previously shown that a combination of repurposed drugs can significantly slow the disease process and improve vision in animal models of human blinding diseases. The successful application of these drugs for the treatment of retinal degeneration would however greatly benefit from mechanistic studies to decipher how they are protective for retinal cells. The further development of novel treatments based on the concept of repurposed drugs is unlikely without this knowledge. To address the problem, the experiments proposed in the funded project aims to uncover the cellular mechanisms that support retinal protection using dopamine-agonist drugs, the most crucial part of the previously identified drug combination.
The results can lead to prompt clinical application, as the proposed experiments will use existing drugs approved for human use by the FDA. Mechanistic insights into the protective effect of dopamine agonists will provide the basis for new, innovative drug development pipelines to treat human IRDs.