Mobility Measures: Advancing Clinical Trials for Genetic Eye Diseases

Professor of Ophthalmology Gislin Dagnelie, Ph.D., a pioneer vision mobility researcher at the Johns Hopkins University School of Medicine, demonstrates the use of virtual reality scenes in a head-mounted display to assess the vision of patients with advanced genetic eye disease. With a new, dedicated mobility lab, Wilmer is well-positioned to expand services in the northeast region, attract new clinical trials and work with collaborative partners to provide new treatments for blinding eye diseases.
While clinical trials hold out hope for people with genetic eye diseases, researchers have been challenged in measuring whether — and how effectively — the latest gene and stem cell therapies are working. Since most patients with severe genetic eye diseases cannot clearly make out even the big “E” on an eye chart, Wilmer researchers needed new methods to assess improvement among clinical trial participants receiving active treatments versus placebos. Enter the Wilmer Eye Institute’s new Visual Mobility Lab, which is part of the Genetic Eye Disease (GEDi) Center at Wilmer. One of few such facilities in the world, the lab is proving key to Wilmer researchers’ efforts to develop protocols and outcomes measures for new drugs, therapies and interventions, says Mandeep Singh, M.D., Ph.D., the Andreas C. Dracopoulos Professor of Ophthalmology, who serves as co-director of GEDi with Jefferson Doyle, M.D., Ph.D., the Andreas C. Dracopoulos and Daniel Finkelstein, M.D., Rising Professor of Ophthalmology.
“The more we expand the number of clinical trials, the more shots on goal we get,” says Singh. “We’re hopeful that we'll soon be able to offer our patients new medications and treatments to slow down their progression, or even perhaps return their vision.”
In the mobility lab, Singh and his colleagues measure patient outcomes during and after treatment by observing patients as they navigate around obstacle courses that can be set up to simulate a busy street, crowded restaurant or interior of a home. For example, can the subject step over or around obstacles, walk up or down stairs, grasp the handle and enter a doorway?
Whether vision is tested using physical obstacles or via a virtual reality platform, the Wilmer researchers use objective measurements to assess how accurately patients maneuver and respond to the terrain. Such quantifiable measures are becoming “the new standard,” Singh says, and changing the way clinical trials at Wilmer are run for a host of genetic eye diseases, including retinitis pigmentosa, Usher syndrome, Stargardt disease and macular dystrophies.
Patients with genetic eye disease are passionate about participating in new clinical trials at Wilmer, Singh says.
“Our team salutes the volunteerism and brave spirit of patients and families who participate in trials to test a new drug,” he says. “It takes commitment and courage to receive the new medicines and undergo very comprehensive testing.” Typically, these patients need the support of their entire family, as well as friends, just to make it to these visits.
“We envision a future,” Singh says, “where treatments can be tailored to each individual patient’s specific needs.”