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Posts tagged Dr. Hendrik Scholl

FFB-CRI Leads Effort to Identify Outcome Measures for Therapies in Clinical Trials

Side view of a retina as captured by SD-OCT. The EZ Width is the yellow line extending between the arrows. The patient has advanced RP with significant loss of peripheral vision.

A key to gaining regulatory approval for an emerging retinal-disease therapy is quickly and accurately demonstrating that it saves or restores vision in a clinical trial. Though the goal sounds simple enough, proving that a potential treatment is working is actually difficult. That’s because commonly used measures of visual function — including visual acuity and visual fields — are not always reliable for evaluating vision changes in many people with inherited retinal conditions.

For example, visual acuity can remain stable for someone with retinitis pigmentosa (RP) for decades. While visual fields for people with RP contract over time, measuring the changes objectively is challenging; results for a given patient can vary significantly, even for the same patient on the same day.
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ARVO 2016: Emerging Drug Targets Toxic Build-Up in Stargardt Disease

Dr. Hendrik Scholl at ARVO 2016.Like many diseases affecting the macula, the center of the retina, Stargardt disease is a waste-management problem. The “garbage” comes from the processing of vitamin A, which is an essential nutrient for vision. If you think of the retina as the engine for vision, vitamin A is the fuel; it enables the retina to convert light into the electrical signals that enable vision. And just like gasoline, which produces exhaust, vitamin A, when metabolized, leads to undesirable by-products.
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