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Treatments for Retinal Diseases are Leading to Therapies for the Brain

An image of the brainThe retina—the thin, fragile layer of tissue lining the back of the eye—gives us the invaluable gift of vision. It works like film or digital sensors in a camera by converting the light that enters our eye into electrical signals. Those signals are sent over the optic nerve to the back of the brain, where they are used to create the images we see.

The retina is an extension of the brain. Like the brain tissue inside our skulls, the retina is comprised of neural cells. This fact is highly significant because it means that a drug or biological therapy that helps save retinal cells in a person with a retinal degenerative disease like retinitis pigmentosa (RP) or macular degeneration might also preserve cells in the brain of someone with a neurodegenerative condition such as Parkinson’s, Alzheimer’s or multiple sclerosis.

For researchers developing treatments for neurodegenerations, the retina is often a better target for testing and evaluating potential therapies than tissue inside the skull would be. First and foremost, the retina is much more accessible than the brain, because it’s a small piece of tissue and is located outside of the skull. Also, because people have two retinas, one in each eye, scientists can treat one retina and compare the effect of a therapy—for example, a change in vision—to the untreated retina. Last, because the retina is relatively isolated, there is less chance of collateral damage if an emerging therapy has unwanted effects.

Retinal research is moving at an incredible pace. Just 10 years ago, there were few therapies in clinical trials. But, today, dozens of drugs, gene therapies and stem-cell treatments for retinal degenerative diseases are now moving into and through human studies. Some are even restoring vision to people who are blind.

The retinal-disease gene therapy for RPE65 gene mutations being developed by Spark Therapeutics—and made possible by decades of Foundation-funded research—is in good position to be approved by the U.S. Food and Drug Administration (FDA). Researchers studying brain and neural diseases closely monitor the success of retinal researchers, because it gives them clear, attractive targets for therapy development.

Optogenetics is a prime example of a promising, emerging therapeutic technique that shows great potential for conditions that affect the retina, brain and central nervous system. It works by bestowing light sensitivity to neural cells. In the retina, the approach shows promise for restoring vision to someone who is otherwise completely blind. By bestowing light sensitivity to the brain, researchers hope to use light to activate neural cells to restore function to someone with a neurodegenerative disease (e.g., Parkinson’s), depression or even a sleep disorder.

RetroSense, a start-up biopharmaceutical company in Michigan that’s been supported by the Foundation, recently received FDA authorization to launch a clinical trial of its optogenetic therapy for people who are blind from RP. If the study is successful, the company hopes to use it for people with macular degeneration as well.

Stay tuned to Eye on the Cure and the Foundation’s website for updates on emerging retinal-disease therapies, including those that may bolster development of treatments for the brain.

Brain image, above, courtesy of the National Institute of General Medical Sciences.


15 Responses to 'Treatments for Retinal Diseases are Leading to Therapies for the Brain'

  1. Yvonne says:

    Will there be testing on those who still have their sight but are seeing a decline?

    • EyeOnTheCure says:

      Absolutely! Individuals that still have sight that is declining should visit their retinal specialist at least once per year to document. In Phase 2 and Phase 3 trials, it is important to show efficacy. This can best be accomplished by evaluating individuals who have declining sight in both eyes. If one eye is treated, the hope is that it should be possible to detect a treatment effect between the treated and untreated eye.

    • Lorena Higgins says:

      Hello, my father who is 83 years old and live in Ecuador South America, was diagnostics with lost of his retina, he can see but only shadows. I would like to know if there is any possibility I can bring him to the USA to be treated? or to be a part of a research for blind people.

  2. Ingrid England says:

    Will there be help for someone who had cystic macular edema surgery and now has a problem with the cornea?

    I. England

  3. Tammy Burnett says:

    My girlfriend as had RP for 37 years and is there any cure? She has some vision. She sees shadows. Also sees dark and light colors.Is there a place in the US for her to get treatment to improve her vision? I will try my best to get her there to that place.That will help her get some of her vision back. Thank You for your time.

    • EyeOnTheCure says:

      Your girlfriend should consider genetic testing to try and identify the mutant gene responsible for causing her retinal disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. Furthermore, with a molecular diagnosis, she may qualify for one of the gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf

      Whether her disease gene is identified or not, your girlfriend should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      It may also be helpful for your girlfriend to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  4. Christopher-Dean says:

    Hi my fiance has RP and,Well she has been diagnosed with RP since she was a child she is 20 years old now and we just had a baby born on January the 29th. sometimes i sit at night till the sun comes up and get so scared for her not going to get to see Jane grow up..my fiance has it bad already loosing her sight and hearing….pleeaase tell me what i can do to help her she is all i’ve got…

    • EyeOnTheCure says:

      Dear Christopher, Your fiance should consider genetic testing to identify the mutant gene responsible for causing her RP. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, she may qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf

      Whether her disease gene is identified or not, she should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  5. south africa
    george joubert i have rp from 8jaar im now 47 jaar old i lost my sunior posision because i cant see at night and i lost my horisontical and furtikel sigtwhit 10%
    is there eney thing that cane save my sigt

  6. Our Doctor Dr. Vimal Parmar , best oplthalmlogist in India , & Lasik Surgery specialist Provides You The Best Eye Care treatment . You can ask Your Question to him Here Or Can Visit http://mahaveereyehospital.com/

  7. GSs says:

    There are amy more people living whith RP around the world. The bad thing is that if you have RP there are more chances that your child may could have same. There is no cure for the disease in the world even may Researches are on the way but there isn’t any evidance of their success. For finding solution or cure of these types of diseaises there is need of money and donatinsa. So donate for RP Research as much as posible .

  8. A Mohan says:

    My wife Dr S Sheela is 54 years having RP with macular degenaration any treatment pl give information and details for taking treatment

    • EyeOnTheCure says:

      Your wife should know that there are three inherited forms of RP: recessive, dominant and X-linked. If she is not sure which type she has, she should ask her ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance:
      http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf

      Your wife should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, your wife should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

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