“Translational research” is the mantra for many of the retinal scientists funded by the Foundation Fighting Blindness. In a nutshell, the phrase refers to the advancement of vision-saving therapies from laboratories into clinical trials and out to the people who need them.
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Dr. Steve Rose
As the Foundation Fighting Blindness’ chief research officer, Dr. Stephen Rose – who prefers to be called Steve – is highly respected for his expertise, intelligence and tireless commitment to finding treatments and cures for vision-robbing retinal diseases. But it’s his accessibility and down-to-earth nature that stand out for many of his peers and Foundation members.
“With Steve, the door is always open. You can walk into his office at almost any time with a problem or an issue, and he is ready and willing to help,” says Dr. Brian Mansfield, the Foundation’s deputy chief research officer. “He also has a phenomenal memory and knowledge base to draw from. And in the event he doesn’t have an immediate answer for you, he’ll pick up the phone and reach out to his many friends and collaborators throughout the science community. The man is incredibly well-connected.”
Steve, who has a Ph.D. in microbiology and joined the Foundation in 2004, supervises its research efforts, which include awarding grants and facilitating clinical trials. He also manages the Science department and works closely with the Foundation’s Scientific Advisory Board, Board of Directors and Science Liaison Committee. Before joining the Foundation, he served more than 14 years with the National Institutes of Health, where he led several divisions, including genetics, clinical research and transplantation studies.
He’s also a member of many prominent scientific and research organizations, including: the Health Research Alliance, the American Society of Gene and Cell Therapy, the Association for Research in Vision and Ophthalmology and the American Association of Immunologists.
Despite his background, Steve has a knack for explaining scientific concepts and retinal research in terms that everyone understands, making him a popular speaker at Foundation and retinal-field events. Bill Schmidt, the Foundation’s chief executive officer, appreciates Steve for, among other things, his enthusiasm.
“There’s no one more passionate about retinal research,” Bill says, “and he does all he can to ensure that the Foundation supports only the best projects — those with strong sight-saving potential. There’s no better advocate for those who’ve lost or are losing their sight, and our recent success in driving research into the clinic is great evidence of that.”
One of the first lessons I learned in my career was that research study results often raise as many questions as they answer. Such is the case with new results from the gene therapy clinical trial for children and young adults with Leber congenital amaurosis (LCA, RPE65 mutations) at the Universities of Pennsylvania and Florida.
Photo has been altered to show how someone with AMD might see the Mona Lisa.
Diseases such as age-related macular degeneration, cone-rod dystrophy and Stargardt disease cause scotomas, or blind spots, which often have devastating effects on central vision. They cause gaps in a person’s visual field, making it difficult to see words in a book, images on a computer monitor or TV and the features of someone’s face.
But as clinical researchers are learning, central vision loss also causes people to have problems with fixation, the ability to maintain one’s gaze in a single direction, which is an added challenge in recognizing faces.
You wouldn’t think that being called an “orphan” is a good thing. But for developers of treatments for rare diseases — including inherited retinal conditions — “orphan” status provides valuable benefits, such as tax incentives, access to special research grants and assistance with clinical trial design. The orphan designation also gives seven years of market exclusivity to the developer of a treatment.
Photo courtesy of the National Eye Institute
I am always excited when a new research paper comes across my desk reporting on an emerging treatment that has saved or restored vision in an animal or cell-based model of retinal disease. The advancement provides meaningful hope for a therapy that can benefit people. But it raises a big question for the Foundation Fighting Blindness: What will it take to move the treatment into and through human studies?
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It’s hard to miss the media hubbub about the potential health benefits of the omega-3 fatty acid called docosahexaenoic acid (DHA). Research has suggested that DHA in your diet is good for preventing and treating cardiovascular diseases, neurological conditions, depression and a slew of other conditions and maladies, including those that affect the retina.
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With a population of about 17,000, Beaver Dam, Wisconsin, is a typical Midwestern community and, according to its website, “a thriving city and wonderful place to call home.” With attractions such as the beautiful 6,000-acre Beaver Dam Lake and the Midwest Cream Cheese Competition, who am I to argue? But most important, at least in the fight against blindness, Beaver Dam plays a big role in the search for knowledge about age-related macular degeneration (AMD).
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Occasionally, a person losing his or her vision to a retinal degeneration or a family member will ask me about complete eye transplantation. “Why can’t someone just get a new eye?”
Well, we can transplant corneas, the eye’s outer, protective covering. And we can replace lenses clouded by cataracts. (Remarkably, there are about 40,000 corneal transplants and 3 million cataract surgeries in the U.S. every year.)
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It’s a mystery that has confounded scientists for many years: Why don’t mice with Usher syndrome type 1 — one of three types of combined blindness and deafness in humans — lose vision? It is an important question, because mouse models help us understand how vision is lost and how effective treatments might be. But if the mouse isn’t losing vision, how can we tell if a potential vision-saving therapy is working?
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Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases. 
As the Foundation's senior science writer, Ben writes science and research articles for the Foundation’s website, newsletters and Eye on the Cure blog. 
As the Foundation's senior writer, Rich writes and edits content for all of the Foundation’s print and online publications, including its blog, Eye on the Cure. 
Nobel Prize Winners Have Big Impact on Emerging Retinal Treatments
By Dr. Steve Rose on October 15, 2012 | 0 Comments
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For science geeks like me, the announcement of Nobel Prize for Medicine winners is always exciting news. But this year’s two recipients are especially near and dear to my heart, because their groundbreaking work is having an enormous impact on sight-saving retinal research.
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