One consequence of most retinal degenerations is that mitochondria, the power suppliers in cells, operate at reduced capacity. Ultimately, photoreceptors, the cells in the retina that provide vision, are lost. Thanks to earlier Foundation Fighting Blindness support, MitoChem has identified three compounds that appear to improve mitochondrial function and, thus, show potential for slowing vision loss caused by a variety of retinal degenerations.
“We are always excited by cross-cutting therapies that show promise for benefiting people with a range of conditions,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “With defects in more than 200 genes causing these retinal conditions, we are very attracted to potential treatments like MitoChem’s that can help a lot of people, including those for whom we can’t find the defective gene causing their disease.”
With the $2 million in funding from the Foundation Fighting Blindness, MitoChem’s goal is to determine which compound is the best drug candidate, and conduct the studies necessary to move it forward into a clinical trial.
The founders of MitoChem are Dr. Barb Rohrer, an expert in retinal degenerations, and Dr. Craig Beeson, a medicinal chemist. These scientists used high-throughput screening technology combining robotics, data processing systems and sensitive detectors to screen a library of 50,000 compounds to identify the three promising compounds, all of which have shown good results in preserving and even restoring vision in animal models of retinal degeneration.
About the Foundation Fighting Blindness
The Foundation Fighting Blindness is a national nonprofit driving research that will lead to preventions, treatments and cures for retinitis pigmentosa, macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases that affect more than 10 million Americans. In its 40-year history, the Foundation has raised more than $450 million as the leading non-governmental funder of retinal research. Breakthrough Foundation-funded studies using gene therapy have restored significant vision in children and young adults who were previously blind, paving the way for using this method to treat a wide variety of retinal degenerative diseases, and proving a cure is in sight. With a network of 50 chapters, the Foundation also provides support, education and resources to affected individuals and their families in communities across the country.
