Vision loss from retinal diseases like age-related macular degeneration (AMD) has always been permanent and irreversible. But now, thanks to advancements in stem cell therapy for retinal disease, that may change.
Though still in their clinical trial stages, studies are showing immense promise in the ability of stem cell therapy (and gene therapy) to not just treat retinal disease but actually reverse vision loss that happens as a result. That’s a huge potential breakthrough in retinal disease management and a game-changer for the more than two million Americans suffering from advanced AMD.
Keep in mind that when it comes to regenerative treatments like stem cell and gene therapies, vigilance is key. These treatments are still in development and not yet available at advanced retinal clinics. Avoid scams by steering clear of “cell-therapy centers” and other unregulated clinics offering regenerative therapy for vision loss at this time.
Now that we’ve got the disclaimer out of the way, let’s look at just how groundbreaking this new advancement is. Read on for a quick overview of stem cell and gene therapy for retinal illness, including how they work and how to (possibly) get early access.
Retinal stem cell therapy works by using healthy stem cells to repair and/or replace damaged tissue in the retina.
In early-stage clinical trials and studies, scientists are transplanting these healthy cells—which have different functions based on the type of stem cell they are—into the eyes of individuals with retinal degenerative disorders (RDDs) and seeing promising results. This includes the potential to treat conditions like age-related macular degeneration (AMD) and retinitis Pigmentosa, among other retinal diseases, and reverse symptoms like progressive vision loss through targeted stem cell injections.
This treatment is still experimental. However, stem cell therapy is currently in use for the treatment of other, non-eye-related degenerative disorders, such as Parkinson’s Disease and Multiple Sclerosis (MS).
In the context of retinal disease, there are two main types of stem cells that scientists are focused on:
During stem cell therapy, healthy stem cells are carefully injected into the eyes where they get to work on the tissue damaged by AMD or other retinal diseases. Multiple types of injections are being studied for their safety and efficacy, with at least 10 clinical trials already at advanced stages.
Both pluripotent and multipotent stem cells are self-renewing, meaning they divide and replicate on their own once established in the body. Currently, research is looking at the use of pluripotent stem cells for retinal tissue replacement and multipotent stem cells for damaged tissue repair.
Age-related macular degeneration is among the leading causes of vision loss among older adults. Fortunately, much of the focus of stem cell therapy for retinal disease is placed on AMD, suggesting that an effective treatment for AMD-related vision loss may be just around the corner.
At the moment, multiple types of stem cells are being evaluated for their ability to treat AMD, particularly pluripotent iPSC and RPE cells. Head to our clinical trials page to see which AMD studies Mid Atlantic Retina is currently participating in.
Other conditions stem cell therapy may be successful at treating in the future include retinal detachment and retinal tears.
Stem cell therapy isn’t the only thing on the horizon for retinal disease management. Scientists and ophthalmology researchers are also exploring gene therapy, which involves replacing faulty genes with healthy ones, as well as the direct modification of retinal cell DNA through gene editing innovations like CRISPR-Cas9.
In terms of timing, we may see widespread use of ocular gene therapy earlier than stem cell therapy. The FDA, for example, approved Luxturna, a one-time injection that delivers healthy genes into the retina and can improve vision for those with inherited retinitis pigmentosa, specifically those with RPE65 mutations.
In the future, gene therapy may be used as a complement to stem cell treatments for retinal disease or on its own, where appropriate.
It’s important to keep in mind that, with very limited exceptions, stem cell and gene therapies are not currently approved for the treatment of retinal disease. That being said, research is moving fast, and we may see things progress sooner rather than later.
In clinical trials, the biggest benefits of these therapies seem to include vision restoration and disease stabilization for a number of retinal conditions. But there are limitations too, such as challenges with accurate cell delivery and immune rejection, plus a lot of unknowns around long-term safety and efficacy.
Hopefully, we will have more concrete answers soon. In the meantime, check back regularly for more updates on clinical trials and what treatments are available for AMD and other types of retinal disease.
Stem cell therapy for retinal disease is not yet FDA approved or available in eye clinics, but you may be a good candidate for a clinical trial or early access.
Potential clinical trial candidates include those with certain retinal conditions, such as AMD and retinitis pigmentosa. Most trials are looking for individuals with early-stage illnesses, which presumably have some healthy retinal cells still available.
Regenerative stem cell and gene therapies represent a new frontier in retinal disease management. If you’re interested in learning more, inquire about our clinical trials or schedule a consultation with a Mid Atlantic Retina specialist. We can help you stay informed on the progress of these and other promising clinical trials, as well as make sure you get the regular retina checkups you need for long-term eye and vision health.
Dr Brett Foxman, Dr Scott Foxman, Dr Thomas Margolis and Dr Daniel Connors will continue to treat patients at the Northfield, Vineland and Cape May Court House locations.
