Foundation Commits $2 Million to Development of a Cross-Cutting Drug Treatment

A key to survival for any organism, plant or animal, is energy. And, in humans, every cell gets its energy from a tiny, organ-like structure called a mitochondrion. It operates like a power plant, providing the energy needed to stay alive and functioning. Among their many functions, mitochondria combine sugar and oxygen, which serve as the cells’ supply of fuel.

One consequence of most retinal degenerations, including retinitis pigmentosa and macular degeneration, is that mitochondria operate at reduced capacity, because of disease-related stress. Ultimately, photoreceptors, the cells in the retina that provide vision, are lost.

The Foundation Fighting Blindness is giving $2 million to MitoChem Therapeutics, a start-up company which, thanks to prior Foundation support, has identified three compounds that appear to boost mitochondrial function and, thus, show potential for slowing vision loss caused by a variety of retinal degenerations. The goal is to determine which one will work best in people and move it into a clinical trial.

Dr. Stephen Rose, chief research officer of the Foundation, says that finding treatments which benefit people with a range of conditions is an important part of the Foundation’s mission. “We are always excited by cross-cutting therapies. 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 gene causing their disease.”

In 2009, the founders of MitoChem – Dr. Barb Rohrer, an expert in retinal degenerations, and Dr. Craig Beeson, a medicinal chemist – screened a library of 50,000 compounds to identify those which enhanced mitochondrial function. They used a high-throughput screening technology combining robotics, data processing systems and sensitive detectors to determine, both quickly and efficiently, which compounds have vision-saving properties. Three emerged from that process and subsequently showed good results in preserving and even restoring vision in animal models of retinal degeneration.

With the goal of moving a cross-cutting treatment into a clinical trial, MitoChem will now: determine which compound is the best drug candidate; develop a drug formulation that will work as an eye drop; and conduct further toxicity and efficacy studies to ensure the drug is ready for human studies.

An important focus of the project is meeting FDA requirements to gain authorization to launch the study. “The FDA will look closely at everything from animal model study results, to manufacturing and production of the compound, to the actual design of the trial before giving MitoChem the OK to begin the study in humans,” says Dr. Rose. “We will be working closely with Drs. Rohrer and Beeson to help ensure that those requirements are met. It’s challenging and expensive, but it’s what we have to do to get the treatment into and through the trial and out to the people who need it.”