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.
- 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
- 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.