ARVO Delivered Good News from Several Retinal Treatment Clinical Trials

May 18, 2012

ARVO attendees
Promising reports from several human studies for emerging retinal disease treatments highlighted the 2012 meeting of the Association for Research in Vision and Ophthalmology (ARVO) held in Ft. Lauderdale, Florida, May 6-10. As the world’s largest eye-research event, the meeting convened more than 12,000 doctors, scientists and industry professionals, who attended thousands of presentations on the latest advancements being made in prominent ophthalmology clinics and laboratories around the globe.

Dr. Stephen Rose, chief research officer, Foundation Fighting Blindness, says that accelerating progress and expansion in retina-related clinical research over the past 18 months is unprecedented. As a result, the ARVO meeting has taken on a new atmosphere of excitement and relevance.

“The landscape began to change at the 2008 ARVO, when the first results were announced for the groundbreaking gene therapy clinical trials for Leber congenital amaurosis,” he recalls. “But this year, we heard updates from clinical researchers leading human studies of gene therapy for choroideremia, recessive retinitis pigmentosa, Stargardt disease, Usher syndrome and wet age-related macular degeneration. While these early studies are primarily evaluating safety, the progress is quite phenomenal. They are an important step toward getting treatments out to the people who need them.” He adds that virtually all of these trials were made possible through research funded by the Foundation.

The 2012 ARVO meeting also featured updates from the first-ever clinical trials of a stem-cell-derived treatment for retinal degenerations. Developed by Advanced Cell Technology (ACT), the therapy has been administered to six patients — three with dry age-related macular degeneration and three with Stargardt disease — in studies at the University of California, Los Angeles (UCLA) led by Dr. Steve Schwartz.

He reported that some of the patients think they are seeing better, and that the first patient participating in the Stargardt trial, a designer, can now see colors, details and even letters she couldn’t see before. He cautions that it is too early to draw conclusions about the treatment’s effects and safety profile, but his team is pleased with the results thus far. ACT’s clinical trial is being expanded to Bascom Palmer Eye Institute in Miami, Wills Eye Institute in Philadelphia, Massachusetts Eye and Ear Infirmary in Boston and Moorfields Eye Hospital in London, U.K.

In addition to potential treatments already in the clinic, a number of ARVO presentations featured updates on therapies poised to move into human studies, including a protein called rod-derived cone viability factor (RdCVF). Discovered by Foundation-funded investigators Drs. José Sahel and Thierry Léveillard of the Institut de la Vision in Paris, RdCVF has shown strong potential for preserving and restoring cones, the cells in the retina that provide central and color vision. The researchers have developed a gene therapy that provides sustained delivery of RdCVF, and which may be beneficial to people with a broad range of retinal diseases, including several forms of retinitis pigmentosa.

Dr. Rose says that, overall, more than 140 ARVO presentations and posters covered projects funded by the Foundation. “It‘s very heartening to attend a presentation on a cutting-edge research effort and see the Foundation’s logo on the researcher’s acknowledgment slide,” he says. “It’s affirmation that our investments are producing great returns and yielding clinical advances.”