Dr. Dominik Lewandowski from University of California Awarded $90,000 Knights Templar Eye Foundation Grant for Juvenile Macular Degeneration Treatment Research

Dr. Dominik Lewandowski from the University of California, Irvine School of Medicine was awarded a grant for $90,000 for his research entitled: Identifying new strategies for lowering ceramides in the retina as a potential approach in retinitis pigmentosa and juvenile macular degeneration treatment.

Retinitis pigmentosa (RP) and juvenile Stargardt macular degeneration (STGD) are inherited retina diseases that affect children and young adults, lead to visual impairment and can cause legal blindness. There are no approved therapies for these diseases. Even though pathophysiology differs between these conditions, some common mechanisms could be involved. Dr. Lewandowski’s findings and existing literature suggest that elevated ceramide, a cell death lipid messenger, could be a universal mechanism of retina degeneration in RP and STGD. He hypothesizes that reducing elevated ceramides can be used as a universal treatment approach to increase photoreceptor survival and improve vision in mouse models of RP and STGD.

He aims to test the effectiveness of his recently discovered ceramide-lowering formulation, composed of desipramine and L-cycloserine (DC), that improved vision in AdipoR1 KO – a mouse model of RP. Dr. Lewandowski wants to verify if the same approach can be used as a universal strategy for treating other models of retina degeneration in RP and STGD. Importantly, since the FDA approved both drugs over 30 years ago, they can be used safely in humans, particularly in pediatric patients. Additionally, he would like to explore the possibility of lowering ceramide in the retina by stimulating the activity of its ceramide-degrading enzyme, AdipoR1, with two agonists – AdipoRon and ALY688.

He hopes to get fresh insight into the therapeutic approaches for RP and STGD by targeting three different nodes of ceramide metabolism - inhibiting two key enzymes generating ceramide and stimulating AdipoR1 ceramide-degrading activity to lower pathologically increased ceramides.