Meet Dr. Steve Rose
Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases.
Archive for the ‘Stem Cell’ Category
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.
Found in Translation: Advancing Treatments Into Human Studies
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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Top 12 Research Advancements of 2012
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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