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The Catalyst in Vision Science Research to Prevent Blindness The Catalyst in Vision Science RPB ANNUAL REPORT 2011 Research to Prevent Blindness 645 Madison Avenue New York, NY 10022-1010 Jules Stein, MD, Founder (1896-1981) David F. Weeks, RPB Chairman Emeritus OFFICERS DIanE S. SWIFT Chairman JOHN I. BLOOmbERG Vice President RICHARD E. BAKER Treasurer & Assistant Secretary BOARD OF TRUSTEES WILLIAM H. AHmansON Jean Bennett, MD, PhD; Luk Vandenberghe, PhD; James Wilson, MD, PhD RICHARD E. BAKER* JOHN I. BLOOmbERG On the cover: In order to people’s faces—all JOHN KUSMIERSKY deliver a corrective gene things they couldn’t do SIdnEY J. SHEInbERG to a site within the eye before,” according to the where it can take the place researchers. DIANE S. SWIFT* of a defective gene and KaTRIna VandEN HEUVEL function properly, scientists “However, to broaden our WILLIam J. VandEN HEUVEL must have an appropriate ability to treat inherited eye delivery agent, or vector. diseases, we will need a TOM WERTHEIMER As we reported in 2009, larger vector toolkit,” says * Members of the Executive Committee RPB-supported researchers Jean Bennett, MD, PhD, a successfully packaged a lead member of the team JamES V. ROmanO normal version of a gene and recipient of RPB support Chief Operating Officer & Secretary missing in Leber’s congenital for 15 years. amaurosis (LCA) inside a MATTHEW LEVINE genetically engineered Pictured on the cover is Director of Communications and Marketing vector, called an adeno- evidence that they have COndON O’MEARA McGINTY associated virus (AAV). The taken a major stride in & DOnnELLY LLP AUDITORS vector delivered the gene to that direction. Using a cells in the retina, where the second-generation AAV gene produces an enzyme technology, they delivered that restores light receptors. a green fluorescent protein transgene to retinal Recently, the researchers pigment epithelial cells further improved eyesight and photoreceptor cells, in three of those treated the problem area for other patients by applying retinal diseases such as therapy in their other eye, retinitis pigmentosa. In without adverse effects. the image, cell nuclei The patients were “able to are labeled blue and walk around at night, shop cone photoreceptors are for groceries and recognize labeled red. A Tradition of Innovation Innovation is and always has been at the heart of RPB’s activities. From day one, our broad mission has been to serve as a catalyst for vision science, with a far-reaching strategy designed to evolve to meet academic research needs. Our first innovation was to place unrestricted funds in the hands of a department chair to fill financial gaps, equip labs and pursue original ideas. Many more have followed, all with the intent of freeing the investigator to break new scientific ground. In 2011, we carried that tradition forward with the launch of a new grant category, specifically created to facilitate out-of-the- box research, and named, aptly, the RPB Innovative Ophthalmic Research Award. With this award category, RPB will provide the means for departments of ophthalmology to develop collaborations with researchers working in other basic scientific disciplines, as part of an effort to bring new technologies and cutting-edge translational science into ophthalmology. We are already excited by the innovative proposals from our first awardees in this category (see pages 10 - 11). 2011 was also the beginning of a transitional period for RPB. David F. Weeks, who served as RPB’s Chairman and/or senior executive for 50 years, retired. David was tireless in his pursuit of the mission he inherited from RPB’s founder, Dr. Jules Stein: to develop, sustain and enhance a national community of visionary vision scientists. Throughout the coming transition, RPB will continue to award grants to qualifying departments of ophthalmology and promising vision researchers at all stages of their careers. We will listen to the needs of the vision research community and find new ways to address them. By staying true to our mission and harnessing the passion of the researchers whom we support, we can ensure that we—the greater We—are always moving closer to the prevention of blindness and the restoration of sight. Diane S. Swift Chairman www.rpbusa.org | 3 Christopher Girkin, MD, University of Alabama at Birmingham School of Medicine, is using a CAVE immersive virtual reality system to visualize 3D models Advances In of the lamina cribrosa, a thin, mesh-like portion of sclera (the white of the eye) at the base of the optic disc through which retinal nerve fibers leave the eye Eye Research to form the optic nerve. This structure plays a critical 2011 role in the early development of glaucoma. There is a flexibility that we encourage in the application of RPB funds that generates the plethora of advances in eye research that we report yearly. “The power of an RPB grant is that it provides a department with unrestricted funds to support various research projects at the chair’s discretion,” says Nicholas J. Volpe, MD, chairman of the Department Macular of dry AMD), an RPB- of Ophthalmology, Northwestern Degeneration and supported laboratory University Feinberg School of Retinal Disorders has developed two Medicine. “With an RPB grant a A collaborative promising therapies for investigation at the prevention of the department is able to provide start three RPB-supported condition. This study also up for small projects, initiate bridge institutions revealed that elaborates, for the first time, a disease-causing funding for ongoing research, facilitate steroids attached to nanoparticles formed role for a large section collaborations and invest strategically an injectable, sustained- of the human genome in research areas that are novel and not release drug delivery once regarded as non- system that may preserve coding “junk DNA.” easily funded.” vision for those with dry age-related macular A recent clinical trial, degeneration (AMD) and For a complete bibliography of the on a single patient retinitis pigmentosa. 1,322 peer-reviewed, published studies with Stargardt macular supported by RPB in 2011, visit dystrophy, using a gene In another promising, vector that is novel www.rpbusa.org/rpb/ research/search/. sustained-treatment in its application in approach to treating ophthalmology, may AMD, scientists report that pave the way for further gene therapy using a use of gene therapy for protein called protectin conditions of the eye as significantly reduced well the rest of the body. uncontrolled blood vessel growth and cell death. Following successful pre- clinical, long-term safety In a discovery that may studies involving the use prevent geographic of adult, bone marrow- atrophy (the end stage derived stem cells as 4 | Research to Prevent Blindness Annual Report 2011 Infectious Eye with those conditions. The Diseases enhanced information An RPB-supported will guide the timing and report links antibiotic extent of treatments. eye drops, which are routinely prescribed after RPB investigators intraocular injections to are exploring the treat AMD, to resistant development of a strains of ocular bacteria novel technology that as well as multiple- would use light pulses drug resistance. The to deliver drugs across researchers suggest “the the tough surface of need for more judicious the back of the eye, use of ophthalmic non-invasively, offering antibiotics.” many advantages over intravitreal injections. An RPB study suggests that Ganciclovir The first images of the rod ophthalmic gel may be photoreceptor mosaic an effective treatment were obtained by RPB for human adenovirus scientists, representing conjunctivitis, the most a major step forward common cause of viral in using retinal imaging “pink eye,” for which tools to study the living there currently is no human retina. topical treatment. A device that uses the therapy for patients with Ocular Cancer Advances in tongue to transmit visual retinal disorders, the The same RPB scientists Technology signals to the brain, first clinical trial will be who previously called the BrainPort, initiated in 2012. This developed a screening could become a approach may treat test to predict whether lower-cost, non-invasive patients with previously the cancer, uveal alternative to retinal irreversible blindness melanoma, would implants. Wearing a from age-related spread to the liver and tiny video camera macular degeneration, other parts of the body mounted to eyeglasses retinal vein occlusion, have identified a drug, and connected by wire commonly used to treat Szilárd Kiss, MD to an electrode sensor diabetic retinopathy or A 3D image reveals retinitis pigmentosa. seizures, which may held in contact with their that several cysts, which tongue, participants make eye tumors less appeared separate with likely to grow. in the study were able An RPB-supported conventional imaging, to identify, sense and are actually connected. study cites the possible avoid objects in front of influence of awareness- RPB investigators them and improve their driven changes in have identified the As potential treatments walking speed. behavior—stopping mechanism that makes for dry AMD and other smoking, improving retinoblastoma so retinal disorders move aggressive, as well as diet, increasing closer to availability, a new treatment target scientists are using physical activity, and possible therapy. This advanced imaging controlling blood childhood eye cancer technology to learn pressure—as causes can cause death if not more about the behind a decline in the addressed early, and development of
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