Every field of research has its “rock stars,” specialists so revered for their knowledge, expertise and experience, they’re famous in certain circles worldwide. And when it comes to retinal-disease research, the stars are invariably linked to the Foundation Fighting Blindness, which either funds or has funded their vision-saving work. So, after interviewing a handful of them recently, we’ve put together a few research-oriented playlists on our revamped YouTube page.
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Archive for the Stem Cell Category
I am very excited about ProgSTAR, the Foundation’s new study monitoring and documenting the progress of vision loss and retinal changes in people with Stargardt disease. On the surface, the study might not sound very exciting, because it isn’t evaluating a potential cure. However, the information gleaned from ProgSTAR will be of enormous value in designing future clinical trials for Stargardt disease treatments.
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“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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Stem cells are a highly promising treatment approach for a wide range of conditions and diseases, because they can be used to replace virtually any type of cell or tissue in the body lost to disease or injury. In the not-too-distant future, in fact, researchers will be able to turn stem cells into new photoreceptors for people who have lost vision to a retinal disease.
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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Two days ago, as part of FFB’s “Light the Way to a Cure” fundraising campaign, I shared the story of Corey Haas, as an example of clinical trial participants benefitting from gene therapy studies the Foundation supports. In his case, the therapy’s for a retinal disease called Leber congenital amaurosis. Today, I’d like to share the story of Dick Coulson (pictured, left), who has age-related macular degeneration (AMD), the leading cause of blindness in people age 55 and older. It’s also the target of two treatments that are currently in clinical, or human, trials, both of which are derived from stem cells.
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At the Foundation Fighting Blindness, we are always looking forward. Our scientists are continually focused on achieving the next sight-saving breakthrough, and our donors and volunteers are always looking for new opportunities to raise more money to drive the research.
But for a moment, as we put the wraps on 2012, it is very inspiring to look back on the past year and reflect on the many exciting advancements that have been made in our quest for treatments and cures.
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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
Video Included:
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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