SEPTEMBER 10, 2014 (Lisbon, Portugal) Six Harvard Medical School (HMS) researchers were among the recipients of the 2014 António Champalimaud Vision Award, the highest distinction in ophthalmology and visual science.
The award was given for the development of anti-angiogenic therapy for retinal disease. The researchers include Joan Whitten Miller, M.D., Evangelos S. Gragoudas, M.D., and Patricia A. D'Amore, Ph.D., MBA, of Massachusetts Eye and Ear; Lloyd Paul Aiello, M.D., Ph.D., of Mass. Eye and Ear and Joslin Diabetes Center; George L. King, M.D., of Joslin Diabetes Center; and Anthony P. Adamis, M.D., of Genentech, who is also affiliated with HMS Ophthalmology and Mass. Eye and Ear. Napoleone Ferrara, M.D., of University of California, San Diego School of Medicine and Moores Cancer Center, also received the award.
The 2014 António Champalimaud Vision Laureates were honored on Sept. 10, 2014 during a ceremony held at the Champalimaud Centre for the Unknown in Lisbon, Portugal. Presiding at the ceremony was His Excellency Aníbal António Cavaco Silva, President of the Portuguese Republic.
Established by The Champalimaud Foundation in 2006, the António Champalimaud Vision Award honors outstanding contributions to the preservation and understanding of sight. In even-numbered years, the award is given for vision research, and in alternate years it recognizes efforts to alleviate visual problems in developing countries or through humanitarian endeavors.
Award recipients are selected by an international jury panel that includes two Nobel Laureates and other prominent figures. The Champalimaud Vision Award is often referred to as the "Nobel Prize for Vision" and with its €1 million ($1.3 million USD) purse, it is among the world's largest scientific and humanitarian prizes.
In the 1990s, the 2014 Champalimaud Award Laureates worked in parallel and in collaboration to identify vascular endothelial growth factor (VEGF) as the major trigger for angiogenesis in the eye. Angiogenesis, or blood vessel growth, underlies the pathology of various blinding retinal disorders, including age-related macular degeneration (AMD) and diabetic retinopathy. Abnormal vascular growth ―a process called neovascularization―above or below the retina allows fluid to leak into the central retina, causing vision loss.
The researchers then demonstrated that blocking VEGF could suppress ocular angiogenesis. This biomedical breakthrough led to a new class of ophthalmic anti-VEGF drugs, which first became available in the United States December 2004 with the introduction of pegaptanib (Macugen®) for the neovascular or "wet" form of AMD. Multiple ophthalmic drugs targeting VEGF activity have since followed, including the widely used ranibizumab (Lucentis®), introduced June 2006, and aflibercept (Eylea®), introduced November 2011. Bevacizumab (Avastin®), an anti-VEGF drug originally developed for cancer and introduced February 2004, is also widely used for treating retinal disease.
The development of anti-VEGF therapy for retinal disease is considered one of the top biomedical advances of the past decade. In 2006, the development of ranibizumab for neovascular AMD was featured in Breakthrough of the Year, a list of the 10 most significant scientific developments compiled annually by the journal Science.
The global impact of the 2014 Champalimaud Laureate's work is significant. According to the World Health Organization, AMD is the leading cause of vision loss in industrialized countries and is estimated to cause blindness or severe visual impairment in nearly 9 million people worldwide. Diabetic retinopathy is the leading cause of blindness among working-age adults in industrialized nations and causes an estimated 5 million cases of blindness worldwide.
With the aging population and global diabetes rates on the rise, AMD and diabetic retinopathy are becoming significant worldwide socioeconomic concerns.
Results of Phase III trials of Lucentis®, first published in 2006, showed remarkable results: two years of monthly eye injections preserved vision for nearly 95% of treated patients, and visual acuity improved for nearly one-third.
Based on these trials, it is estimated that two years of Lucentis® treatment reduces visual impairment in neovascular AMD by 37% and legal blindness by 72%. By 2010, global annual use of Lucentis® reached more than 3 million injections, translating to approximately 500,000 patients treated per year worldwide.
Eylea® has since become the predominant approved therapy for neovascular AMD, treating 26% of patients (surpassing patient share of 21% for Lucentis® or ranibizumab), while Avastin® continues to be the most widely used off-label treatment for wet AMD.
Extrapolating from these data and December 2013 estimates of U.S. sales, one can conclude that over 500,000 ophthalmic patients in the United States and more 1 million worldwide are treated annually with all anti-VEGF agents combined.
Anti-VEGF therapy is now a mainstay of patient care for neovascular AMD, diabetic macular edema (swelling of the central retina, a complication of diabetic retinopathy) and macular edema following retinal vein occlusion. VEGF inhibitors hold potential for a growing list of indications, including neovascular glaucoma and retinopathy of prematurity. Moreover, VEGF inhibitors have been used experimentally to treat over 50 ocular diseases.
"The work of the nominees has had a tremendous impact on the work of my faculty colleagues—both in the clinic and in the laboratory," says Paul Lichter, M.D., Chair of Ophthalmology and Visual Sciences from 1978 to 2012 and Founding Director of the Kellogg Eye Center at University of Michigan. "Anti-VEGF therapy ranks at the top of the list of pharmacological treatments that have been developed for retinal disease, and have helped masses of people in my long career as an ophthalmologist."
"To one degree or another, AMD affects nearly 70% of people over age 75 in Europe and the United States. This group's body of work has fundamentally changed the world for millions of citizens worldwide," says David W. Parke II, MD, Executive Vice President and CEO of the American Academy of Ophthalmology. "I am a retina subspecialist, and for the first twenty years of my clinical career, I had nothing to offer my elderly patients who were robbed of vision, of independence, and of enjoyment of life by neovascular AMD. I can now tell them that, with newer medications spawned by the work of the Champalimaud Award Laureates, ophthalmologists have a very high likelihood of arresting disease progression and a reasonable chance of recovering some lost sight."
Jeffrey S. Flier, M.D., Dean of the Faculty of Medicine at Harvard Medical School, praised the collaborative nature of the research that spanned academia and industry to translate scientific discovery to clinical reality. "These scientists' triumphs show what can happen when fertile minds meet, brainstorm and combine ideas in alliances that cross institutional and academic boundaries," said Dr. Flier. "Their discoveries remind us why medicine must begin with exploring science at the basic level—shedding light on pathological mechanisms and pinpointing the root causes of human disease."
The researchers will use the funds from the award to further their research efforts to cure blindness.
"It is a great honor to be recognized as a group for our translational work in the field of angiogenesis," said Dr. Miller. "We are thrilled to receive this wonderful award, and remain inspired to continue our investigations, pursuing our passion to improve the lives of patients around the world, so that children born today may see throughout their lives."
About Massachusetts Eye and Ear
Mass. Eye and Ear clinicians and scientists are driven by a mission to find cures for blindness, deafness and diseases of the head and neck. Led by the Eaton-Peabody Laboratory in Otology, the Howe Laboratory in Ophthalmology, the Berman-Gund Laboratories for Retinal Degenerations, and Schepens Eye Research Institute, Mass. Eye and Ear in Boston is the world's largest vision and hearing research center, offering hope and healing to patients everywhere through discovery and innovation. Mass. Eye and Ear is a Harvard Medical School teaching hospital and trains future medical leaders in ophthalmology and otolaryngology, through residency as well as clinical and research fellowships. Internationally acclaimed since its founding in 1824, Mass. Eye and Ear employs full-time, board-certified physicians who offer high-quality and affordable specialty care that ranges from the routine to the very complex. U.S. News & World Report's "Best Hospitals Survey" has consistently ranked the Mass. Eye and Ear Departments of Otolaryngology and Ophthalmology as among the top hospitals in the nation.
About Harvard Medical School
Harvard Medical School has more than 7,500 full-time faculty working in 11 academic departments located at the School's Boston campus or in one of 47 hospital-based clinical departments at 16 Harvard-affiliated teaching hospitals and research institutes. Those affiliates include Beth Israel Deaconess Medical Center, Brigham and Women's Hospital, Cambridge Health Alliance, Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Pilgrim Health Care, Hebrew Senior Life, Joslin Diabetes Center, Judge Baker Children's Center, Massachusetts Eye and Ear Infirmary, Massachusetts General Hospital, McLean Hospital, Mount Auburn Hospital, Schepens Eye Research Institute, Spaulding Rehabilitation Hospital and VA Boston Healthcare System.