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Posts tagged stargardt disease

FFB-Funded Scientists Report on Nine Promising Translational Research Efforts

Translational research — moving promising science out of laboratories and into clinical trials — is essential to getting vision-saving, retinal-disease treatments out to the millions who need them. With that said, translational research is also costly and high risk and requires extensive clinical development and regulatory knowledge.

The Foundation Fighting Blindness has taken the translational challenge head on by investing more than $75 million in therapy-development projects with strong clinical-trial potential through its Translational Research Acceleration Program (TRAP), which includes Gund-Harrington Scholar Awards.
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FFB Convenes Experts to Discuss Therapeutic Opportunities for Stargardt Disease

Meeting presenters (left to right): Philip Rosenfeld, MD, PhD; Ilyas Washington, PhD; Peter Charbel Issa, MD, PhD; Elias Traboulsi, MD, MEd; Ulrich Schraermeyer, PhD; Carel Hoyng, PhD; Paul Bernstein, MD, PhD; SriniVas Sadda, MD; Krzysztof Palczewski, PhD; Janet Sparrow, PhD; Artur Cideciyan, PhD; Hendrik Scholl, MD; Patricia Zilliox, PhD (not pictured)

Meeting presenters (left to right): Philip Rosenfeld, MD, PhD; Ilyas Washington, PhD; Peter Charbel Issa, MD, PhD; Elias Traboulsi, MD, MEd; Ulrich Schraermeyer, PhD; Carel Hoyng, PhD; Paul Bernstein, MD, PhD; SriniVas Sadda, MD; Krzysztof Palczewski, PhD; Janet Sparrow, PhD; Artur Cideciyan, PhD; Hendrik Scholl, MD; Patricia Zilliox, PhD (not pictured)

Stargardt disease is the world’s leading cause of inherited macular degeneration, affecting 30,000 people in the United States alone. It is also a challenging condition to understand and treat. While Stargardt disease often causes severe loss of central eyesight, its effect on vision and the retina can vary widely from patient to patient. The disease usually strikes in childhood or adolescence, but there are forms that cause significant vision loss much later in life. Also, a patient’s vision can remain stable for many years before a relatively sudden, steep decline occurs.

The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) convened 70 of the world’s top Stargardt disease research experts in Cleveland, Ohio, on February 17, 2017, to discuss these challenges and the current state of therapy development. Much of the information and data shared during the meeting came out of ProgSTAR, the FFB-CRI-funded natural history study of 365 Stargardt disease patients.
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FFB-CRI Leads Effort to Identify Outcome Measures for Therapies in Clinical Trials

Side view of a retina as captured by SD-OCT. The EZ Width is the yellow line extending between the arrows. The patient has advanced RP with significant loss of peripheral vision.

A key to gaining regulatory approval for an emerging retinal-disease therapy is quickly and accurately demonstrating that it saves or restores vision in a clinical trial. Though the goal sounds simple enough, proving that a potential treatment is working is actually difficult. That’s because commonly used measures of visual function — including visual acuity and visual fields — are not always reliable for evaluating vision changes in many people with inherited retinal conditions.

For example, visual acuity can remain stable for someone with retinitis pigmentosa (RP) for decades. While visual fields for people with RP contract over time, measuring the changes objectively is challenging; results for a given patient can vary significantly, even for the same patient on the same day.
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VISIONS 2016 – Dr. Richard Weleber Receives FFB’s Highest Research Honor, Recognized in Touching Video

Dr. Richard WeleberConsidering all that Richard Weleber, M.D., has accomplished over four decades —
including leadership and oversight of clinical trials for emerging retinal-disease therapies and innovations in retina imaging and functional evaluation at the world-renowned Casey Eye Institute, Oregon Health & Science University — it comes as no surprise that he’s been given FFB’s Llura Liggett Gund Award for career achievement. Dr. Weleber became the 10th recipient of the Foundation’s highest honor, named after FFB co-founder Lulie Gund, during the opening lunch of the VISIONS 2016 conference.
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VISIONS 2016 – Dr. Shomi Bhattacharya Wins FFB Award for Gaining an Understanding of Variations in Vision Loss

Dr. Shomi BhattacharyaInherited retinal conditions such as Stargardt disease and retinitis pigmentosa (RP) run in families. The diseases in some families span several generations with dozens of affected members. In other cases, a disease may only affect one or more siblings within a single generation. Researchers have understood the nature of these different inheritance patterns fairly well for several decades.
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Back in Baltimore – VISIONS 2016, FFB’s National Conference, Returns to Its Founding City

Baltimore's Inner HarborFor the second year in a row, the Foundation Fighting Blindness’ national conference — this year titled VISIONS 2016 — will take place in FFB’s founding city of Baltimore, Maryland, June 30-July 3. More than 500 people are expected to attend the event, which will offer dozens of research- and lifestyle-focused sessions designed for people who are visually impaired and sighted as well.
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Two Philanthropic Brothers – Selling Luxury Casual Wear for the Good of Retinal Research

Bryan and Bradford Manning in New York City.Bradford Manning and his brother, Bryan, have a great sense of humor. You can see it in the video for their new clothing line, Two Blind Brothers — which donates its proceeds to retinal research — and hear it when you talk to Bradford over the phone.
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ARVO 2016: Emerging Drug Targets Toxic Build-Up in Stargardt Disease

Dr. Hendrik Scholl at ARVO 2016.Like many diseases affecting the macula, the center of the retina, Stargardt disease is a waste-management problem. The “garbage” comes from the processing of vitamin A, which is an essential nutrient for vision. If you think of the retina as the engine for vision, vitamin A is the fuel; it enables the retina to convert light into the electrical signals that enable vision. And just like gasoline, which produces exhaust, vitamin A, when metabolized, leads to undesirable by-products.
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ARVO 2016: ProgSTAR, FFB-CRI’s Stargardt Disease Patient Study, Highlighted

Janet Cheetham, ProgSTAR's liaison to FFBOne of the hot topics at ARVO 2016 is ProgSTAR, the natural history study for people with Stargardt disease funded by the Foundation Fighting Blindness Clinical Research Institute (FFB-CRI). I caught up with Janet Cheetham, Pharm.D., the project’s liaison to FFB, to explain why the effort is important to therapy development. Having spent more than three decades in the development of retinal and ophthalmological treatments at Allergan, she brings a wealth of insight and knowledge to her role.
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For Rare Disease Day – The Many Benefits of Genetic Research

Rare Disease Day logoIt’s apropos that Rare Disease Day 2016 will be held on the rarest day on the calendar—Leap Day, February 29.

However, collectively, rare diseases are not uncommon. About 30 million Americans, nearly 10 percent of our population, are affected by one of 7,000 rare diseases. They’re an important public health issue, making it incumbent upon us to work hard to eradicate them.
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