Casey Eye Institute Hosted International Meeting on Gene Therapy
Experts will build on momentum of current progress
Many of the world's leading vision scientists gathered at OHSU Casey Eye Institute on May 10 and 11, 2013 to advance the promising field of gene therapy for the treatment of serious retinal disorders. Gene therapy experts, some from such far-flung locations as China, Europe and Brazil, spent two days discussing the status of current clinical trials and sharing their knowledge in anticipation of the next stage of larger scale investigations.
"Gene therapy, which targets a specific biological function, has great potential in treating blinding eye diseases of the retina caused by a defective gene," said David Wilson, M.D., Thiele-Petti Chair of the Department of Ophthalmology and Director of Casey Eye Institute. The experimental treatment involves augmenting the function of the faulty gene with a healthy version of the gene introduced into eye cells by way of a non-active virus.
"There has been enough progress that we felt it was time to bring together everyone involved in gene therapy research to build on the momentum with our collective efforts," he said. "Because so many of these inherited diseases are rare, we need to do this on an international level in order to achieve the necessary number of participants for the next phase of clinical trials," he added. The group, which included nearly 70 participants from medical institutions, research centers and the biotech industry, also began the critical task of establishing common measures of success as scientists begin testing the effectiveness of these novel treatments. The ultimate goal is to create a formal study group for future collaborations, said Dr. Wilson.
Currently, Casey is conducting Phase I/II gene therapy trials for several rare inherited retinal disorders, including Usher syndrome, Leber Congenital Amaurosis and Stargardt disease. It also is one of two research sites in the world testing a gene therapy medication for the wet form of age-related macular degeneration.
The human eye is especially suitable for gene-based treatments because it is accessible yet separate from the rest of the body. It is also easier to introduce medications into the eye than in other organs and to objectively measure whether the experimental treatment is successful. Moreover, since only one eye is treated, the fellow untreated eye serves as a valuable control.
Casey has become an international leader in gene therapy research, developing technological capabilities essential to this new generation of eye research, such as an enhanced visual field test and genetic testing. “We look forward to this landmark collaborative effort and in playing a key role as we move to this next, exciting stage of research. Gene therapy truly is a revolutionary approach that offers tremendous hope for preserving vision,” said Dr. Wilson.
See what a participant in the UshStat clinical trial says about her involvement in the trial.