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Retinal Regeneration is Major Focus of NEI’s Audacious Goal

Paul SieivingThe National Eye Institute (NEI) just announced that fighting blindness — retinal degenerations, in particular — has gotten audacious. It’s potentially life-changing news for people with these difficult conditions.

For more than a year, the NEI has focused significant resources on establishing what it calls an “audacious goal” to fundamentally change the game in vision research and eye care. With so many great breakthroughs occurring in a variety of eye research areas, the NEI recognized it was the ideal time to consolidate energies of the vision science community and present a challenging goal that NEI Director Dr. Paul Sieving said is “big, important and inspiring.” The development of the initiative included review of more than 500 suggested research goals, which were read by more than 80 experts.

I am delighted to report that the NEI’s unveiling of its audacious goal at the annual meeting of the Association for Research in Vision and Ophthalmology is an enormous boost and affirmation for those of us on the front lines of fighting retinal degenerative diseases. The goal, “to regenerate the neurons and neural connections in the eye and visual system,” is exactly what people with retinal diseases need to save and restore their vision. And most of the cutting-edge technologies that will get us to the goal — including gene therapies and stem cells — are exactly what the Foundation Fighting Blindness has taken a leadership role in supporting for the last four decades.

When you look back at the biggest breakthroughs made in retinal research — the identification of the first retinitis pigmentosa gene, the proof that gene therapy can restore vision in humans, and making new photoreceptors from stem cells — most were made by visionary researchers funded by the Foundation. We empowered those scientists to take the bold risks that held the potential to save and restore vision. Many times, those risks led to big advances and dramatically changed the retinal research landscape.

It took audacity for Gordon Gund and the late Ben Berman to establish the Foundation in 1971. But they knew no one would do it for them. Everyone who has joined the Foundation’s family since — researchers, donors and volunteers alike — is audacious in their own way. Whether you are forming a VisionWalk team, organizing a chapter meeting or donating money, you are stepping out of your comfort zone to make a difference. You know the work isn’t always easy, but you also know that no one will do it for you.

We at the Foundation applaud the NEI for its audacious goal and look forward to hearing more about the details to achieve it. The initiative holds great potential for helping people with retinal degenerations. The Foundation stands ready to help the vision community achieve this goal by continuing to drive the cutting-edge research that will deliver the preventions, treatments and cures available to the millions who need it.

In the meantime, don’t forget to be audacious. After all, you are part of the Foundation Fighting Blindness.

Pictured Above: Paul A. Sieving, M.D., Ph.D., Director of the National Eye Institute

16 Responses to 'Retinal Regeneration is Major Focus of NEI’s Audacious Goal'

  1. Irv Arons says:

    To read about one of the “audacious” techniques that researchers are working on, please take a look at “A New Technique for Restoring Normal Vision to the Blind”.

    A scientist and professor at Cornell and a professor of ophthalmology at the Univ. of Florida have teamed up to develop a technique to bypass the damaged photoreceptors causing blindness in the diseases noted above, and restore near normal vision, first in animals and soon in humans.

    Dr. Sheila Nirenberg at Cornell and Dr. William Hauswirth of the Univ. of Florida will soon be setting up a human clinical trial to try their new technique that sends enhanced visual signals to the brain using a combination of a mathematical code and a technology called optogenetics.

    To learn more about this discovery, please follow this link:

  2. Hopefully, a cure is in sight, especially in our ‘generation’. Every newsletter both via paper/mail and email/internet raises the hopes of literally hundreds of thousands seriously affected by RP, AMD and a host of other retinal ‘challenges’. Those who are ‘sighted’ say that “seeing is believing”. Those with retinal disorders, RP, AMD etc. are also saying ” we are watching “. A cure is in sight. Cantor Stephen Chaiet

  3. Harvey Cohen says:

    Fantastic goal! I applaud Dr. sieving and the NEI for the audacious objective…..all success,

  4. Darlene Vlazny says:

    Please forward info or contact information.

    Our son, Ryan, has Ushers Syndrome (retinitis pigmentosa, etc.) – 26 years old.

    He is deaf and uses Manual sign language (done in his hand).

    He is legally blind; however, his vision in his right eye is suddenly deteriorating at an accelerated pace.

    His specialist who has been working on gene-based therapies for at least 12 years, has recently informed us that it is Not an option & he will start studies on stem cell therapies.

    Our son does NOT have another 12 years. He is employed (IT/computer related) and could lost his job and independence soon.

    We are desperate for help.

    Can you please forward info on who we may contact who has been involved in stem cell research and may be further along in the therapy and possible clinical trials?
    In the US? In another country? (England, Israel, or …?).

    We would sincerely appreciate ANY assistance or redirection. We certainly do not want to go somewhere controversial, but are quickly running out of time.

    Thank you for your consideration and dedication.

    Darlene Vlazny, 954-295-0754,

    • EyeOnTheCure says:


      Sorry to hear that your son is losing his vision so rapidly. Currently, there are stem cell clinical trials underway for age-related macular degeneration and Stargardt disease. A stem cell clinical trial for retinitis pigmentosa is probably a year or two away from beginning. With that said, you need to keep in mind that clinical trials are experiments and have risks. Also, the inclusion and exclusions criteria can significantly restrict who is eligible to participate in the studies. Please keep in mind that many/most overseas stem cell trials and therapies are not legitimate.

      As you may know, there is also a gene therapy clinical trial underway for Usher syndrome type 1B. To be eligible for that, your son would need to be genetically tested to determine which gene is linked to his condition. Even if he does not have much vision remaining, gene therapy might still provide some benefit.

      You can learn more about the genetic testing process at:

      I understand your concern for your son. Foundation-funded researchers are working as hard and as quickly as they can to come up with answers.

  5. While the news contain in Dr. Rose’s post is generally encouraging, I continue to be frustrated by the FFB’s apparent decision to essentially ignore the fact the FDA approved trial by Advanced Cell Technology to inject embroyic stem cells into AMD and SMD patients is (1) ongoing at clinics at UCLA, Miami, Philedephia, and London (2) nearly two years have passed since the first injection without any safety concerns,(3) that the oversight committee has approved and allowed the injection of a greater dose into patients in the second cohort—indicating at the very least safety after two years since the first patient was injected, and (4) with respect to SMD, the oversight committee has allowed the creation of a sub-cohort of young patients with vision as good as 20-100–which can only be a highly positive step and decision by the oversight committee. FFB ought to get the word out and get behind this research for the benefit of my SMB afflicted daughter and thousands of others who are “waiting.”

  6. When you need a human guinea pig, I am available. (Greensboro, N.C.)

    • EyeOnTheCure says:

      Leonard, thanks for your support. As always, we’ll be reporting on all these promising areas of research as they continue to develop – please stay tuned.

  7. Paul Fibraio says:

    This is very interesting and I hope you reach your goal. My son has /congenital Liebers, if I am spelling it correctly, would this procedure also apply to this disorder?

    • EyeOnTheCure says:

      Hi Paul, thanks for your comment. This research is still at an early stage (several years form human study), so it is somewhat difficult to determine who can benefit from it. With that said, it might someday benefit people with LCA. Stay tuned.

  8. Farkhanda sheikh says:

    My son is 27 years old and has RP he is loosing his sigt right now he is legally blind and cannot drive, he also looking for jobs and could not find any job as yet, planning to work on his master degree program in Social work. Would you please tell me to whome shall I contact for new treatments here at USA or any other country.

  9. Michelle Maretich says:

    I have RP (A-Typical Pars Planitis w retinal degeneration). I am wiling to be a guinea pig for any of your trials.
    Michelle Maretich (814) 942-0957

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