Original investigators continue on path of discovery, innovation

San Francisco–The first Catalyst for a Cure research team has been working together for 11 years to elucidate the molecular mechanisms of neurodegeneration in glaucoma and new therapeutic targets.

At the annual Catalyst for a Cure Benefit and Gala Dinner on Thursday evening, the four principal investigators discussed their discoveries and how they are applying this new knowledge to impact the future of patient care.

Philip Horner, PhD, of University of Washington, Seattle, said that based on an early determination that death of retinal ganglion cells in glaucoma follows a protracted course, his group aimed to characterize the upstream events as a means of identifying novel targets for interventions to slow or prevent the disease. Their subsequent investigations have yielded some surprising findings about early changes in gene function of the ganglion cells and activation of the glial cells and microglia, Dr. Horner said.

Nicholas Marsh-Armstrong, PhD, said that he and his coworkers at Johns Hopkins School of Medicine, Baltimore, are extremely excited about the work being done in their laboratory that stems from a series of surprising findings about the optic nerve head.

“The future looks quite promising,” said Dr. Marsh-Armstrong. “I can’t say when we will have a very significant treatment to halt the disease, but I think there are some very basic findings here that may herald entirely new approaches to therapy.”

David Calkins, PhD, of Vanderbilt Eye Institute, Nashville, TN, noted that the identification of several molecular cascades that are believed to translate stress in the eye to the earliest neuronal responses occurring in glaucoma has led his laboratory to identify agents able to quench the stress response and abate degeneration and progression.

“We are very excited now because we have had some very promising results with some of the new drugs we have tested in preclinical models,” Dr. Calkins added.

Monica L. Vetter, PhD, of University of Utah, Salt Lake City, said that the group’s focus on describing the early pathogenic mechanisms of retinal ganglion cell death rather than on ways to manage IOP has been transformational.

“It has opened up a whole new world of thinking about the pathways controlling the decline of neurons and provides new opportunities for intervening at much earlier stages,” Dr. Vetter explained. “Critical pathways and players are being identified and there are now strategies in the pipeline that are targeting specific components we know are involved in the course of the disease. They will take some time to develop and test, but we think we are well on the way to having approaches in hand that will have a significant impact.”

Catalyst for a Cure research team provides an update about their progress in finding a cure for glaucoma.

Catalyst for a Cure Research Progress 2013 from Glaucoma Research on Vimeo.

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