This year marks the fortieth anniversary of the National Eye Institute (NEI), during which we celebrate 4 decades of leadership in basic and clinical science of the eye and vision. The NEI emerged from the National Institute of Neurological Diseases and Blindness in 1968 as the first government organization solely dedicated to research on human visual diseases and disorders. Its establishment was the result of both a Congressional interest and urging by prominent ophthalmologists.
Dr Jules Stein was one of these ophthalmologists. He founded Research to Prevent Blindness, Inc, in 1960, and soon after, he championed the cause for creating a national eye institute. A Gallup poll at the time demonstrated a perceived need for such an organization, revealing that blindness was second only to cancer as the most feared ailment among Americans.
In a separate campaign, Dr Alfred Edward Maumenee, Jr., chairman of the Ophthalmology Department at Johns Hopkins University, enlisted help from his colleagues to form the Association of University Professors of Ophthalmology in 1966. One of their first acts was to advocate for the creation of a government eye institute.
When the bill to establish an eye institute was introduced in Congress in 1967, Lions Club member Dr Ralph W. Ryan, former head of the National Institute of Neurological Diseases and Blindness section on clinical ophthalmology, organized the Lions, who sent more than 100 000 telegrams and letters to urge bringing the legislation to the floor of the House for a vote.
President Lyndon B. Johnson signed the law to found the NEI on August 16, 1968. He emphasized his commitment to improving the vision of Americans in his signing statement: “Nearly every family in America has at least one member suffering some form of vision problem or eye disease … . These tragedies need not occur … . There is much that remains to be learned.”
Pioneering Randomized Controlled Trials
Dr Carl Kupfer became the first director of the NEI in 1970, and within a year, he had established an Office of Biometry and Epidemiology, an Office of the Director of Intramural Research, a Laboratory of Vision Research, and a Clinical Branch. Dr Kupfer pioneered the use of clinical trials at the National Institutes of Health (NIH), setting the stage for the randomized controlled trials and comparative effectiveness studies that are now critically important to translational research opportunities. His insistence on using the scientific method and analysis of the placebo effect set the standard for current clinical trials. Today, the vision science community continues to uphold the value of well-designed, well-conducted clinical trials as an “indispensable tool of clinical research.”
Results obtained from the first 3 randomized, masked, controlled clinical trials sponsored by the NEI were extremely valuable for demonstrating the validity of vision loss treatments. The Diabetic Retinopathy Study, initiated in 1971, the Diabetic Retinopathy Vitrectomy Study (1976 through 1983), and the Early Treatment Diabetic Retinopathy Study (1979 through 1985) all clearly demonstrated the success of photocoagulation and of vitrectomy in preventing vision loss in patients who would otherwise go blind.
These research successes launched the NEI into several decades of extensive clinical trials for conditions of glaucoma through the Collaborative Initial Glaucoma Treatment Study and the Ocular Hypertension Treatment Study and for age-related macular degeneration, which included the Age-Related Eye Disease Study, among others.
The NEI also recognized the value of investigator collaborations by promoting networks to facilitate clinical research. The Pediatric Eye Disease Investigator Group is a consortium of academic and community clinicians who work to understand childhood eye disorders, and the Diabetic Retinopathy Clinical Research Network uses its collaborative power to advance the study of retinopathies. These networks currently are active with NEI support and continue Dr Kupfer’s legacy.
Ground-Breaking Basic Research
The formation of the NEI provided opportunities for basic science of vision to blossom on the NIH campus. In a series of elegant experiments in the mid 1980s, the alphaA-crystallin promoter was identified and characterized, and through mouse models, intramural researchers demonstrated the oncogenic potential of the lens. In addition to furthering the study of the lens itself, this groundbreaking work in molecular biology had far-reaching consequences for visual science and more broadly for science as a whole.
Basic vision research outside of NIH also flourished because of the strong support of investigator-initiated research, of which the R01 grant mechanism remains the hallmark. In 1986, NEI-supported research established a novel mechanism for ocular cancer in identifying that loss of the function of the Rb tumor suppressor gene can lead to retinoblastoma eye cancer.
In 1993 the first structural description of a G-protein coupled receptor, the light sensitive and very stable rhodopsin, was reported. This finding increased our understanding of retinal biology and opened new avenues for drug discovery. Several years later, NEI grantee Dr Peter Agre received the 2003 Nobel Prize in Chemistry for his 1991 discovery of aquaporin, the first known channel that facilitates and regulates water transport across cell membranes, hugely significant for the highly fluid lens. Scientists have now begun to identify the role of aquaporin in a number of ocular diseases, including Sjögren syndrome and congenital cataracts, and other organs and tissues, including the kidney, brain, respiratory tract, and salivary gland.
During the 1980s and 1990s, NEI-supported investigators also took advantage of new molecular biology techniques to clone a number of eye disease genes and to develop animal models to understand their roles in cataract, retinopathy, and myopia. Based on these efforts, the vision community has access to more than 500 genes that cause visual loss in a Mendelian inheritance fashion. These genes represent more than 20% of all human disease genes currently cloned.
Ground-Breaking Basic Research
The formation of the NEI provided opportunities for basic science of vision to blossom on the NIH campus. In a series of elegant experiments in the mid 1980s, the alphaA-crystallin promoter was identified and characterized, and through mouse models, intramural researchers demonstrated the oncogenic potential of the lens. In addition to furthering the study of the lens itself, this groundbreaking work in molecular biology had far-reaching consequences for visual science and more broadly for science as a whole.
Basic vision research outside of NIH also flourished because of the strong support of investigator-initiated research, of which the R01 grant mechanism remains the hallmark. In 1986, NEI-supported research established a novel mechanism for ocular cancer in identifying that loss of the function of the Rb tumor suppressor gene can lead to retinoblastoma eye cancer.
In 1993 the first structural description of a G-protein coupled receptor, the light sensitive and very stable rhodopsin, was reported. This finding increased our understanding of retinal biology and opened new avenues for drug discovery. Several years later, NEI grantee Dr Peter Agre received the 2003 Nobel Prize in Chemistry for his 1991 discovery of aquaporin, the first known channel that facilitates and regulates water transport across cell membranes, hugely significant for the highly fluid lens. Scientists have now begun to identify the role of aquaporin in a number of ocular diseases, including Sjögren syndrome and congenital cataracts, and other organs and tissues, including the kidney, brain, respiratory tract, and salivary gland.
During the 1980s and 1990s, NEI-supported investigators also took advantage of new molecular biology techniques to clone a number of eye disease genes and to develop animal models to understand their roles in cataract, retinopathy, and myopia. Based on these efforts, the vision community has access to more than 500 genes that cause visual loss in a Mendelian inheritance fashion. These genes represent more than 20% of all human disease genes currently cloned.
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