The Argus II Retinal Prosthesis System was recently granted market approval by the U.S. Food and Drug Administration (FDA), which marks a significant milestone in the development of a therapy that has spanned more than 20 years. It was at Johns Hopkins University, in the early 1990s, that I, along with Drs. de Juan, Humayun and Dagnelie, with some early support from FFB, carried out the very first short-term (one hour or so) experiments with human volunteers. We demonstrated that electrical stimulation of the retina could, indeed, produce spots of light perceived by blind patients.
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.
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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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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With the elections now behind us, the U.S. Congress needs to address an issue that could delay by years, perhaps even decades, the ability of those with serious diseases to access treatments that will at the very least change their lives for the better, if not save them. The looming “fiscal cliff” – Congress’ current means of tackling a substantial budget deficit – is a combination of tax increases and spending cuts that will kick in January 1, 2013, if no further legislative action is taken. And it’s a huge mistake.
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Rachel Weeks, who has Usher syndrome – a disease affecting both vision and hearing – is a dedicated triathlete. With guidance from her sister, Rebecca, she runs, swims and bikes her way through triathlons with only a 10-degree field of vision and 30 percent of her hearing. But once, as she prepared to plunge into the water with hundreds of competitors, a woman insisted on congratulating her for her efforts. At that particular moment, it served as a disorienting reminder of the challenges she faces.
I was very excited to learn that another clinical trial using stem cells for the potential treatment of a retinal disease — in this case, dry age-related macular degeneration (AMD) — is now underway. StemCells, Inc. (got to love the company’s name) announced last week that its first patient was treated at the Retina Foundation of the Southwest in Dallas.
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Three weeks ago, in recounting my participation in Charity Day at Cantor Fitzgerald, I mentioned that FFB had established October as Save Your Vision Month (SYVM). Well, now it’s here, so I wanted to reiterate what SYVM is all about and how the Foundation has been preparing for it the past couple of weeks.
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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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