Without a doubt, as chief research officer at FFB, I get more questions about clinical trials – also known as human studies – than any other topic. So, I thought it would be useful to provide a list of the major clinical trials underway right now for retinal disease treatments.
All of the following, I might add, are being funded by the Foundation or were made possible by Foundation-funded preclinical research. I’ve categorized the trials by types of treatments and included links to articles or posts describing them in detail. Appended to each is the name of the company, facility or agency conducting and/or hosting the trial.
Gene Therapies
- Stargardt disease gene therapy — Oxford BioMedica
- Usher syndrome type 1B gene therapy — Oxford BioMedica
- Wet age related macular degeneration (AMD) gene therapy — Oxford BioMedica
- Wet AMD gene therapy — Genzyme
- Wet AMD gene therapy — Avalanche
- Choroideremia gene therapy — University of Oxford
- Retinitis pigmentosa (RP, MERTK) gene therapy — UCSD-King Khaled Eye Hospital
- Leber congenital amaurosis (LCA, RPE65) gene therapy — Children’s Hospital of Philadelphia
- Leber congenital amaurosis (LCA, RPE65) gene therapy — Universities of Pennsylvania and Florida
Cell-Based Therapies
- Stargardt disease treatment (RPE cells derived from stem cells) — Advanced Cell Technology
- Dry AMD treatment (RPE cells derived from stem cells) — Advanced Cell Technology
- RP and Usher syndrome (imaging study, encapsulated cell technology) — Neurotech
- Achromatopsia treatment (encapsulated cell technology) — National Eye Institute
Pharmaceuticals and Supplements
- Retinitis pigmentosa (autosomal dominant), valproic acid — FFB Clinical Research Institute
- LCA and RP (LRAT and RPE65), retinoid replacement — QLT
- X-Linked RP, DHA supplementation — Retina Foundation of the Southwest
Some Important Comments
This list doesn’t include every human study for a retinal degeneration. For example, there are four additional LCA-RPE65 gene therapy clinical trials that I didn’t include. There are several studies for age-related macular degeneration pharmaceuticals that are not on the list. I also didn’t include a few studies underway for artificial (a.k.a. “bionic”) retinas.
What is most impressive to me about the list is that nearly eight years ago, when I joined the Foundation, virtually none of these studies were underway. We’ve come a long way in that time. I expect this list to grow substantially in the next eight years, with some of the abovementioned emerging treatments gaining FDA approval.
Stay tuned and thanks for your interest in and/or support of sight-saving research.
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. 
Dr. Rose only listed regenerative medicine clinical trials either sponsored or financed by FFB. Anyone interested in learning about other stem cell and gene therapy clinical trials can obtain additional information by taking a look at two recent blog postings both of which include information on how to obtain my tables containing more information about the eleven stem cell clinical trials and the sixteen gene therapy clinical trials that I am tracking.
To learn more see: Menu 20: A List of Writeups on Stem Cells Used in Ophthalmology (link: http://tinyurl.com/blogmenu20)and,
Menu 21: A List of Writeups on Gene Therapy Used in Ophthalmology (link: http://tinyurl.com/blogmenu21).
Irv Arons
Irv Arons’ Journal (http://tinyurl.com/ijablog)
Thanks for sharing Irv!
Hi,
Is there a way someone from India can be a part of these clinical trials? My sister has Retinitis Pigmentosa (with <5% vision left) and we have been looking for positive developments in the cure of RP.
Thanks,
Priyanka
Priyanka, thank for your comment. You don’t necessarily have to live near a clinical trial site to participate in the study, but you do have to be able to travel to the site on a regular basis for exams and testing. That may be prohibitive for someone in India given that many of the sites right now are in Europe and the U.S. Keep in mind that your sister should not go into a clinical trial with the expectation of having vision saved or restored. While a positive effect on vision is a possibility, clinical trials are experiments with risks. People who choose to participate in them should do so for the sake of advancing science rather than saving their own vision.
I am a grandmother of two children with LCA that is caused bu the RDH12 gene. Is threre research being done for that specifically?
Keep up the good work!
Sincerely,
Patti Day
Hi Patti, thanks for posting. Dr. Debra Thompson at the University of Michigan, and Dr. Jean Bennett at the University of Pennsylvania are working on RDH12 gene therapies. They aren’t in clinical trials just yet, but the investigators are working toward that goal.
I sufered from retinitis pigmentosa and intrest to any treatment about this problem
Kazem, thanks for posting. As yet, there is no known cure for Retinitis Pigmentosa. However, intensive research is currently under way to discover the cause, prevention, and treatment of this disease. Please visit our website for more information on RP here (http://goo.gl/8F7hv). This page includes information on the potential treatments we are most hopeful of in the fight against RP. We put out new articles on the latest RP research as it comes in here: (http://goo.gl/OpfVl). If there is any more information we can help you with or if you have a particular question, please email us at info@fightblindness.org.
URGENT!! My best friend and cousin has an autoimmune disease that has attacked her retinas. They have tried many things to keep this disease at bay. She has developed Glaucoma and they have put in shunts and steroid discs, buckles and air bubbles when her retina detached. One morning she woke up and one of her eyes was squishy. The shunt had failed and her left eye is now completely useless and with much scar tissue, deformed. Now the shunts in her right eye has dislodged and sitting against her cornea in her right eye. She is going back to the same woman who did the procedure to both eyes. I took a picture of the shunt in her cornea this morning if you would like to see it. She goes back in Oct 3 to get the shunt removed and the Doc is going to put the shunt in the back of her eye? They say they won’t be able to see if it is working or get at it once they do this. I’m very scared because this is the first time this doc has done this and I’m afraid my closest friend is going to lose vision completely!! Please help if you can. I beg for any kind of info to help her. Or another doc to give a second opinion.
Hi Becca,
Thanks for messaging us. We are very sorry to hear about your friends vision loss and understand that this is a very hard time for the both of you. Our focus is on funding retinal disease research and providing resources for those suffering from these diseases. We do not have practicing physicians and so we cannot offer medical advice in cases of eye conditions. If you have not done so already, please visit the website of the National Eye Institute. This site and here in particular (http://www.nei.nih.gov/health/resourceSearch.asp?Disp=1&strKey=Autoimmune+Related+Diseases) can point you in the direction of other organizations and institutions that may be able to provide more resources for those suffering from autoimmune eye diseases and its symptoms. We wish you and your friend all the best during this difficult time.
My daughter is 25 years old and has been diagnosed the cone rod dystrophy. We have found very little information or a doctor that can help us with her disease. She has had testing done at the University of Wisconsin of Madison and is part of a study at the Eye Institute in Milwaukee, WI, but we have not heard anything from them since January 2012. We have also sent a blood sample to Carver Labs and her tests have come back negative for cone rod dystrophy. Please let us know where to go from here. Thank you
Dr. Rose and team at the FFB,
Thanks for the continued investments for more than four decades to get us to the point now where we now have so many great opportunities to treat and cure blindness. Having met a man at the recent Visions Conference who actually had vision restored with a prosthetic in a trial in Europe, and in learning of all the other progress with human patients, I’m very excited. Keep up the good work!
Scott
Thank you Scott! As always, its a pleasure to hear from you
I have two kids (ten and nine years old) both with Cone Rod Dystrophy.If We identify the gene that cost the problem. Can they be treated with any kind of gene terapy or some thing?Dr. John (Pei-Wen)ChiangPhd,FACMG Director of Csey Molecular Diagnostic Lab.Email chiangj@ohsu.edu Tel-503-494-5838 or 503-853-5644 can find the gene that cost the problem.The question is:Can you put me on the rigth direction to help my kids?.
Jesus,
Identifying the gene will help put you on the right track, so I encourage you to pursue that. It will help point you toward emerging treatments that may help your kids. For example, the StarGen gene therapy in clinical trials replaces the ABCA4 gene which can be a cause of cone-rod dystrophy. But cone-rod can be caused by other genes, as well. I think it is important that your kids see and stay in touch with a retinal specialist who can help guide you toward emerging treatments. Dr. Richard Weleber is at the same institution (OHSU) as Dr. Chiang. He is outstanding.
hello sir….my brother has RP problem….is there any tretment for this in india…plz sir..if there..kindly inform me….its abour his whole life…his age is 29 right now….
Hi Shreya,
We encourage you to take a look at the document linked to below. it provides the latest on research for RP. We are unaware of what resources are available in India, but it may be beneficial for you to reach out to this group (http://retinaindia.org/) for more information.
http://www.blindness.org/images/pdfs/disease%20information/research%20advancements/Advances_RP.pdf
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