December 27, 2011 – The year 2011 was a big one not only for the Foundation Fighting Blindness, but for the entire retinal research field. The progress FFB made in the drive to the clinic was unprecedented; it launched or made possible six new clinical trials this year, and its past funding continued to bolster efforts by other parties. The successes include several firsts as well as new treatment approaches, such as optogenetics, that weren’t even on the Foundation’s radar just a couple of years ago.
Here, in no particular order, are the Top 11 Retinal Research Advances in 2011:
•The Foundation launched a clinical trial of valproic acid, an FDA-approved seizure drug, for potentially slowing vision loss in people with autosomal dominant retinitis pigmentosa.
•The first-ever human studies of a stem-cell-derived treatment for retinal disease (Stargardt disease and dry age-related macular degeneration) were launched by Advanced Cell Technology.
•Second Sight’s “bionic retina” became the first commercially available device of its kind. It can be purchased in Europe, and the company is working to bring it to market in the United States.
•The Foundation’s U.K. partner Oxford BioMedica began the first-ever clinical trial of gene therapy for Stargardt disease.
•Using next-generation technology, researchers at the University of Iowa identified the gene MAK as the cause of about one-third of all cases of autosomal recessive retinitis pigmentosa in people with Ashkenazi Jewish descent.
•The Foundation invested more than $8 million in six gene therapy projects, all of which are scheduled to be ready for clinical trials within three years.
•The FDA approved Eylea for the treatment of wet age-related macular degeneration. The therapy requires fewer injections than other available treatments.
•The FDA authorized Oxford BioMedica’s launch of the first-ever gene therapy clinical trial for Usher syndrome (type 1B).
•Several research teams used innovative optogenetic techniques to enable a variety of cell types in the retina — including cone, ganglion and bipolar cells — to respond to light, restoring vision in preclinical models of advanced disease.
•The first-ever clinical trial of gene therapy for choroideremia began in Oxford, U.K.
•The first-ever clinical trial of gene therapy for an autosomal recessive form of retinitis pigmentosa (MERTK mutations) was launched at the King Khaled Eye Specialist Hospital in Saudi Arabia. |
