Researchers are developing a number of promising treatment approaches for blinding retinal diseases, which include: correcting genetic defects; replacing lost retinal cells with new ones; and implanting electronic chips, like the recently FDA-approved Argus II. But, sometimes, saving vision simply comes down to keeping retinal cells alive, or at least slowing their degeneration. Known as “neuroprotection,” this approach isn’t just for the retina — it has the potential to preserve and protect all kinds of neural cells, including brain tissue and cells of the central nervous system.
What’s signficant about many emerging neuroprotective therapies for the retina is that they have the potential to treat a wide range of diseases, regardless of the genetic mutation causing vision loss. With about 200 genes linked to retinal diseases, this is a huge plus. They might also be used in conjunction with a genetic therapy to enhance its vision-saving effects.
Though neuroprotection usually isn’t addressing the root cause of the disease, it may be nearly as beneficial as a permanent fix, if useful vision can be saved for a person’s lifetime.
Neuroprotective treatments are being developed in many forms — proteins, drugs (small molecules) and nutrients. And there are several ways to get a neuroprotective therapy to the retina, including eye drops and oral medications.
Imagine having a therapy factory in the retina — in the forms of transplanted cells or genes — that provides continual release of neuroprotective proteins. Well, researchers are working on those, too.
Here are three examples of emerging neuroprotective therapies for the retina:
Protecting Mitochondria — MitoChem Therapeutics, a Foundation-funded start-up company, has identified two compounds, which show potential for saving vision for people affected by several retinal diseases, including retinitis pigmentosa (RP), cone-rod dystrophy, Bardet-Biedl syndrome, Usher syndrome, Stargardt disease and age-related macular degeneration (AMD).
The compounds work by protecting mitochondria, the power supplies for all cells in the body, including those in the retina. Investigators have refined the compounds to the point where they save virtually all of the photoreceptors in a mouse model of retinal degeneration. They’ve also demonstrated that eye drops can effectively deliver large amounts of one compound to retinas in eyes comparable in size to those in humans. Eye drops are beneficial, because they minimize potential systemic side effects.
Docosahexaenoic Acid (DHA) — DHA is an important structural component of cells in the brain and the retina. It appears to have many neuroprotective properties and is prescribed for a variety of conditions, including heart disease. Perhaps most relevant to retinal degenerative diseases, DHA can reduce the destructive effects of inflammation and oxidative stress. It also helps maintain the structure and metabolism of photoreceptors.
The Retina Foundation of the Southwest is completing a Foundation-funded clinical study of DHA for males with X-linked RP. Previous clinical studies conducted by Dr. Eliot Berson show that vitamin A combined with DHA can slow vision loss in people with RP. In 2014, the National Eye Institute will complete a clinical study of DHA and other nutrients for people at risk of advanced AMD. DHA can be obtained by eating salmon, tuna and other coldwater fish, or by taking fish-oil or algae supplements.
Rod-Derived Cone Viability Factor (RdCVF) — Drs. José Sahel and Thierry Léveillard, of the Institut de la Vision in Paris, received the Foundation’s Trustee Award for their discovery of Rod-derived Cone Viability Factor (RdCVF), a protein that preserves and rescues cones, the cells in the retina that provide central and color vision. They are now developing a gene therapy that provides sustained delivery of RdCVF after a single treatment. The protein could be beneficial to people affected by a broad range of retinal diseases, including several forms of retinitis pigmentosa. With Foundation funding, the researchers are working to move their RdCVF gene therapy into a clinical trial.
There are several other emerging neuroprotective therapies in the Foundation’s research portfolio. Stay tuned to Eye on the Cure and the Foundation’s website for updates on these emerging cross-cutting treatments.
Pictured, above: Some neuroprotective therapies may be delivered by eye drops. (Photo courtesy of the National Eye Institute.)
Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases. 
As the Foundation's senior science writer, Ben writes science and research articles for the Foundation’s website, newsletters and Eye on the Cure blog. 
As the Foundation's senior writer, Rich writes and edits content for all of the Foundation’s print and online publications, including its blog, Eye on the Cure. 
I have retina detachment, and wonder if this research would benifits me or not. I really want to keep my vision alive. Thank you
Sarah Davenport
Thanks for your comment Sarah,
This is a good question. Research has shown that neuroprotection can be used for retinal detachment. However there are some issues to keep in mind. We don’t know if neuroprotection for a disease like retinitis pigmentosa will work for a retinal detachment. Also, I presume that neuroprotection will work best if administered soon after the detachment. However, we at the Foundation Fighting Blindness are not experts in detachments so I suggest you talk with a doctor who is familiar with retinal detachments to get a better sense of what might or might not work.
Hi there, I desire to subscribe for this webpage to obtain most up-to-date updates,
so where can i do it please help.
Hi Darren, are you able to use RSS? Please copy our RSS feed link into an RSS reader, such as your email, and it should allow you to receive our updates. If you need additional help with this, please let us know.
Interested in hearing about research developments in RP and how to preserve cells
Thanks for you interest Marjory!
I am 29 and have cone rod dystrophy. I am interested in any more info available about preserving cells or any trials currently being done for this disease. Thank you.
Sabrina, the vision-preserving examples (MitoChem and RdCVF) listed in the article have the greatest near-term potential for treatment in humans with cone-rod dystrophy. Stayed tuned to our blog and Web site for new announcements on these and other emerging therapies as they move closer to clinical trials.
It may be helpful to try and determine which gene is linked to your disease, if you haven’t been genetically tested. That can help you better understand which emerging therapies might be best for you.
Here is a link to genetic testing resources on our Web site:
http://www.blindness.org/index.php?option=com_content&view=category&id=105&Itemid=132
I have had rod and cone dystrophy for20 years. Can any of this really help?
Hi Jim, good question. The earlier these can be administered in the course of disease, the better (when they are available as treatments). However, even if you don’t have many photoreceptors left, they may be able to help preserve the ones that remain. It is difficult for us to comment on their applicability to you, personally, without knowing your specific case. But again, if you have some vision left, they may be beneficial.
I’m interested in any information about research developement for RP and how to preserve cells and prolong vision.
Hi June, thanks for your comment. Below is a link to our website that provides more information on RP. You may find the information in the treatments section most helpful.
http://www.blindness.org/index.php?option=com_content&view=article&id=50&Itemid=67
Dear Sir,
My son suffers from Ushers Syndrome. I was wondering if we could get access to the eye drops mentioned in the article headed ” Staying Alive: Saving Retinal Cells to Preserve Vision”
Thank You
Hi Serge, thanks for commenting.
The treatments mentioned in the article are not available just yet. They are moving toward clinical trials. DHA is in a clinical trial and we expect to hear results on that study very soon.
There is also a gene therapy study underway for Usher syndrome type 1B. Here is a report on the study from last fall:
http://www.blindness.org/index.php?view=article&catid=67%3Ausher-syndrome&id=3390%3Ausher-syndrome-gene-therapy-safe-thus-far-in-three-patients&option=com_content&Itemid=123
I think it is important for you to try and determine what type of Usher syndrome your son has – that will help you determine which emerging treatments may be best for him. Contact the Foundation at info@fightblindness.org – let us know where you live – and we can try to recommend a doctor who can order a genetic test, if your current doctor is not familiar with the process.
Also, here is a link to genetic testing resources on our Web site:
http://www.blindness.org/index.php?option=com_content&view=category&id=105&Itemid=132
My wife has RP and believe the “Recessive ” type and she is 41 with 3 young kids to care for. We have been a member of FFB since 1997. We appreciated all the articles from the hard copy days of the quaterly reports to the new aged on line blogs, sites and etc. You folks are doing a great service and i sure hope as many RP and other eye related issues hope for a cure.
We wanted to know which of these promising treatment is for sure soon becomes available to the general public? We have been going to well known, two of the well known research universities since 1997, did all sorts of testing, screening blood work to find the mutated gene, in which still has not been located, and as of 8 years ago, she has developed CME which is actually more urgent they tell us to treat and one of the treatments gave her Catracts which we have to get both of her lens removed and replaced and much more. Basically we have seen the best of the best in this filed in CA and MI state but the common issue we have been hearning is funding or the lack of.
The research doctors tells us that “we have the staff, the know how, the equipements even at some facility, but no funding to do the RP or other types of eye disorder genetic related issues. So what that means, we know how to fix people but no funds. I guess this is the issue all over. Truly is sad. We have been told the cure is underway, every five years, and nothing has happened. I wonder if within next few years, these emerging new therapies will actually save visions.
Any comments or perhaps a facility that can better help my wife with her RP eye care until the cure is availble is appreciated. Again, thank you
My daughter has Tap Dystrophy
Is there any new development in healt posibility
Thank you in advance
John Aagaard
John, thanks for your comment.
We are not sure what “Tap Dystrophy” is. Perhaps you misspelled the name of the condition. Can you please tell us more about the condition and if there is another name for it?
My wife has RP since 1995 and she is 59 now. She has been on VitA Palmitate 15,000 IU, 1 pill a day since 1995. She is showing progressive loss of field since early 1912. You folks are doing a great service and i sure hope as many RP and other eye related issues hope for a cure.
Shall appreciate receiving valuable cure-info and guidance for her to sail through immediate and distant future.
Thanks.
Thanks for your support Tarun!