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Archive for the Stem Cell Category

Several New Stem Cell Clinical Trials Poised to Begin in Two to Three Years

Microscopic Stem CellsOne of the more exciting aspects of FFB’s recent annual conference, VISIONS 2013, was the news on the clinical development of several emerging stem cell therapies. The field has picked up a lot of steam, with many new human studies of stem-cell treatments anticipated to begin in 2014 and 2015.
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Patient Registries Help Advance Research for Rare Diseases

Image of Keyboard and StethiscopeRare disease research is challenging, because patient information about the conditions is inherently limited. Patient recruitment for clinical trials can be especially difficult, because so few people are affected.

To address these issues, a number of foundations, patient advocacy groups and governmental institutions have launched online registries to collect patient information for use by researchers, doctors and public health experts.
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FFB’s New Research Playlists on Youtube

Image of Eye and YouTube IconEvery 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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Proving a Vision-Saving Treatment Works

Dr. Hendrik Scholl conducts an electroretinogram, or ERG, with a patient at the Wilmer Eye Institute.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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Moving Vision-Saving Treatments Out to the People Who Need Them

A lab technician“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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Safely Harnessing the Power of Stem Cells

Image of a Stem CellStem 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.

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Orphan Drugs Get Special Treatment

Image of lab equipmentYou 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.

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Found in Translation: Advancing Treatments Into Human Studies

A clinical trial patient is examined

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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Curing Blindness, Part 2: Dick’s Story

Image of Dick CoulsonTwo 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

Image of lab beakerAt 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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