Washington, D.C., Nov. 5 – Will gene therapy that restored sight in dogs born blind prove successful in human clinical trials? This and other similar questions are being explored by leaders from a number of research disciplines, corporations, foundations, and government institutions at an international symposium being held in Washington this weekend. The National Neurovision Research Institute (NNRI), in conjunction with six of the National Institutes of Health, the FDA, academic institutions, and private concerns have marshaled over 170 of the world’s best and brightest to pursue what Dr. Elias Zerhouni, Director of the National Institutes of Health, dubbed “New Pathways to Discovery” in his “Road Map” launched last year.
The First International Symposium on Translational Clinical Research for Inherited and Orphan Retinal Diseases is targeting both gene and drug therapies that are on the verge of moving from basic laboratory research into human clinical trials. The RPE65 gene that has been found to restore vision in dogs born with Leber Congenital Amaurosis (LCA) is ready to “take the next step,” says Dr. William Hauswirth, a scientist from Florida who has led the project team, with funding from both the National Eye Institute and the Foundation Fighting Blindness (FFB), NNRI’s parent organization. Although children, who are the most susceptible to LCA, will not be used for these studies, the genes’ receptivity and effectiveness in adult patients will serve as a valid test of its efficacy in fighting this blinding disease. With “leading experts from academic and health care institutions, pharmaceutical and biotech companies, foundations, government research institutes and investment communities from around the world joining us, we are confident of going beyond the verge of breakthroughs,” explained Dr. Morton Goldberg, the NNRI’s Board Chairman, and recently retired head of the Johns Hopkins Wilmer Eye Institute.
This first Symposium is historic in nature, providing an opportunity for representatives of science and business to initiate collaborations and to discover new ways to expedite clinical trials and commercialization for retinal degenerative disease therapies. Dr. Paul Sieving, Director of the National Eye Institute, believes that research success in one discipline will provide insight for other disciplines. “We are delighted to have the opportunity to interact with authorities from our sister Institutes, as well as the Office of Rare Disease, and the Office of Orphan Products Development.”
The National Institutes of Health’s Office of Rare Diseases is fully supportive of the NNRI. “We have embraced the initiative demonstrated by this symposium,” confirmed Dr. Stephen Groft, the office’s director. “Research on ‘orphan’ diseases, in which there are fewer than 200,000 patients nationwide, has been stymied frequently due to the cost of developing treatments for which there are too few users to make them financially feasible in the conventional commercial sense. A sizable number of these diseases cause blindness, and it is imperative that efforts aimed at their eradication and treatment continue to be advanced.
The NNRI’s First International Symposium on Translational Clinical Research for Inherited and Orphan Retinal Diseases was sponsored by the National Eye Institute (NEI) ; Office of Rare Diseases – Department of Health and Human Services, (NIH), National Institute on Aging, (NIH); National Institute of Neurological Disorders and Stroke, (NIH); National Heart, Lung, and Blood Institute, (NIH); National Institute on Deafness and Other Communication Disorders. (NIH); Office of Orphan Products Development, (FDA); Alcon; and the W.K. Kellogg Foundation.
NNRI’s Mission The mission of the National Neurovision Research Institute (NNRI) is to accelerate the translation of laboratory-based research into clinical trials aimed at developing treatments and cures for retinal degenerative diseases. Many of these retinal diseases are considered rare in the United States; yet ensuring that adequate and effective drugs are created and delivered to those in need is of paramount importance nonetheless. Unfortunately, given the rare nature of these diseases and the high cost of treatment development, insufficient resources are currently being provided in the clinical trials arena.
NNRI realizes the strong need for more intense therapeutic development in this area. To succeed in its mission, NNRI seeks to bridge the gap between scientific, clinical, governmental, pharmaceutical and financial communities, thereby ensuring a more rapid delivery of pharmaceutical and genetic therapies for “orphan” retinal degenerative diseases (RDD). The institute also aggressively pursues needed funding to accomplish this important objective.
As a medical research institute, NNRI searches for potential therapies and pathways to enhance the drug discovery process for retinal diseases. To advance the translation of clinical trials to the marketing of safe and effective drug therapies, NNRI works to implement Phase I and Phase II clinical trials of potential therapeutic remedies for RDD patients.
NNRI’s Background NNRI is a newly established non-profit organization that was created to support the Foundation Fighting Blindness (FFB). FFB, which was created in 1971, seeks to find treatments and cures for macular degeneration, retinitis pigmentosa (RP), Usher syndrome and all other retinal degenerative diseases. With over nine million Americans of various age groups and races suffering from vision loss, FFB’s mission is to invest as many resources as possible into research and related programs. To that end, NNRI supports FFB in its mission to ensure that promising research is translated into clinical trials more rapidly.