Dr. Warren Pan from the University of Michigan Kellogg Eye Center was awarded a $70,000 grant to study: Pharmacologic activation of PKM2 for photoreceptor neuroprotection in inherited retinal diseases.

A leading cause of childhood blindness is due to a collection of over 280 inherited mutations that disrupt the photoreceptor cells of the eyes from converting light to vision. These photoreceptor cells are very active and require significant energy to perform their functions. They will examine PKM2, an enzyme important in energy production that is also linked to a gene mutation that results in childhood blindness.

The research goal is to pharmacologically increase the activity of PKM2 to improve photoreceptor health and subsequently vision. Dr. Pan will continually treat an animal model of inherited photoreceptor degeneration with our PKM2 activator drug and evaluate visual function, photoreceptor survival, and photoreceptor metabolism over the disease time-course. By studying these changes over time with and without the drug, we will begin to understand the cellular and metabolic mechanisms responsible for this devastating group of diseases that cause childhood blindness. These studies will move us closer to developing a novel drug capable of treating vision loss in this diverse set of inherited diseases regardless of gene mutation.