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Stem Cells and Macular Degeneration

Scheie Eye Institute, University of Pennsylvania
A female researcher looking at a test tube.
There is great potential for stem cell transplantation in age-related macular degeneration (AMD), and this article explores the latest research.

Patients with age-related macular degeneration (AMD) often ask whether stem cell therapy might improve their vision now, or at some point in the future.

The concept is that stem cells are “undifferentiated,” meaning that they have the potential to develop into many different cell types, including retinal cells, which can then replace cells that have died due to AMD. The two major cell types that die in AMD are retinal pigment epithelial (RPE) cells and photoreceptors.

It is much easier to replace RPE cells than photoreceptors. RPE cells normally sit on a membrane in a single layer, where they support the photoreceptors. Without RPE cells, photoreceptors die. This is the sequence of events in at least some patients with the “geographic atrophy” form of advanced, dry AMD. Stem cells can be induced to become RPE cells relatively easily, and then injected into their proper location. The challenge is that often these cells form clumps rather than a single layer.

Over the past few years, results of several small studies on transplantation of stem cell-derived RPE cells for AMD and the related disease, Stargardt’s were reported. The results show evidence that these transplanted cells can survive for at least a year, few adverse events occurred, and there is a hint that the vision may have improved slightly. Improved techniques including implantable scaffolds for the cells and use of medications to enhance outcomes are being investigated. While these results represent a promising start, more clinical trials and fine-tuning of techniques are needed.

It is more difficult to transplant photoreceptors, however. While stem cells can be induced to form photoreceptors, it has harder to “convince” them to become photoreceptors than RPE cells. Also, after photoreceptors are transplanted into the retina, they must connect with other retinal neurons and form synapses in order to conduct their signals. While this normally occurs in the developing retina, it is much harder to make this happen in an adult retina. Experiments in mice suggest that it can be accomplished, but so far this happens mainly when the donor cells and recipient retinas are at early developmental stages. Combinations of RPE cells with photoreceptors can also be generated from stem cells, and could also be considered for transplantation.

In summary, there is much excitement about the potential for stem cell transplantation in AMD, and this approach has a reasonable chance of helping patients with wet AMD or advanced dry (geographic atrophy) AMD at some point in the future. In the meantime, studies in mice as well as additional human clinical trials will continue to pave the way.

Important Clinical Trial Information

People who might be interested in enrolling in a cell transplantation clinical trial should ask their ophthalmologist about their eligibility and the current availability of trials. Under no circumstances should patients have cell transplantation or injection outside the protections of a clinical trial registered on clinicaltrials.gov or Antidote. Recently, stem cell injections into the eye, offered by a clinic but not part of a registered clinical trial, resulted in catastrophic vision loss due to the lack of proper regulatory approval. For more information on this issue, visit here.


  • Retinal Pigmented Epithelium (RPE) - a layer of cells that protects and nourishes the retina, removes waste products, prevents new blood vessel growth into the retinal layer and absorbs light not absorbed by the photoreceptor cells; these actions prevent the scattering of the light and enhance clarity of vision.
  • Photoreceptors - the light sensing nerve cells (rods and cones) located in the retina.


This content was first posted on: November 30, 2016

The information provided here is a public service of the BrightFocus Foundation and should not in any way substitute for personalized advice of a qualified healthcare professional; it is not intended to constitute medical advice. Please consult your physician for personalized medical advice. BrightFocus Foundation does not endorse any medical product, therapy, or resources mentioned or listed in this article. All medications and supplements should only be taken under medical supervision. Also, although we make every effort to keep the medical information on our website updated, we cannot guarantee that the posted information reflects the most up-to-date research.

These articles do not imply an endorsement of BrightFocus by the author or their institution, nor do they imply an endorsement of the institution or author by BrightFocus.

Some of the content may be adapted from other sources, which will be clearly identified within the article.

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