Stem Cell Research

Last medically reviewed: October 7, 2026

⚠ Important — educational information only, not medical advice

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Not an FDA-approved treatment unless explicitly stated. Except where a page names an FDA-approved product, the stem-cell, MSC, exosome, extracellular-vesicle, Wharton’s jelly and other biologic approaches described here are not FDA-approved for any eye condition. Research findings — especially laboratory and animal studies — are not proof that anything works or is safe in people.
Research-Use-Only (RUO): Research-Use-Only (RUO) materials referenced on EyeCyte are for laboratory research by qualified professionals only. They are not drugs, biologics or devices approved for human use, are not intended for administration to people, and are not intended to diagnose, treat, cure, mitigate or prevent any disease.
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"Stem cells" is a broad term. Researchers distinguish between pluripotent cells (embryonic stem cells and induced pluripotent stem cells), which can become almost any cell type, and adult or tissue-specific stem and stromal cells, which are more limited. In vision science these cell types are studied for very different purposes.

Research is not treatment. The studies summarized below are investigational. They are presented for education only, do not show that any approach is safe or effective in patients, and are not a recommendation. Unless stated as FDA-approved, none of these approaches is approved for this condition. RUO materials are not for human use.

The main cell types researchers study

Embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) are used to grow specialized retinal cells, such as retinal pigment epithelium (RPE), in the laboratory.

Mesenchymal stromal cells (MSCs) are studied mainly for the signals they release rather than for replacing eye cells.

Limbal stem cells maintain the corneal surface and are used in specialized corneal surgery.

Retinal progenitor cells are immature retinal cells studied in inherited retinal disease.

Cell replacement versus cell signaling

Some research aims to replace lost cells (for example, RPE in macular degeneration). Other research studies whether cells, or the molecules they secrete, could support or protect existing tissue. These are different hypotheses with different levels of evidence.

Where the evidence stands

Most stem-cell research in ophthalmology is preclinical or early-phase. Small human studies of stem-cell-derived RPE cells have primarily assessed safety. Regulatory approval for use in any eye disease is a separate, higher bar.

Regulatory status: to our knowledge as of our last review, no product in this category is FDA-approved to treat an eye disease. The FDA has warned the public about unapproved stem cell and exosome products.^{[1, 2, 3, 4]}

Why caution matters

Unapproved procedures offered outside regulated trials have caused serious harm, including blindness, in published cases.^[5]

Answers

Related conditions

Stargardt Disease Glaucoma

Frequently Asked Questions

Are stem cells an approved treatment for eye disease?

Not in general. Some specialized corneal procedures using limbal stem cells are established in certain jurisdictions; most other stem cell approaches remain investigational.

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References

Schwartz SD, et al. Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies. The Lancet, 2015. [Peer-reviewed study · citation details pending reviewer confirmation]
da Cruz L, et al. Phase 1 clinical study of an embryonic stem cell–derived retinal pigment epithelium patch in age-related macular degeneration. Nature Biotechnology, 2018. [Peer-reviewed study · citation details pending reviewer confirmation]
FDA Warns About Stem Cell Therapies (consumer update). U.S. Food and Drug Administration. [Regulator · citation details pending reviewer confirmation]
Approved Cellular and Gene Therapy Products. U.S. Food and Drug Administration. [Regulator · citation details pending reviewer confirmation]
Kuriyama AE, Albini TA, et al. Vision Loss after Intravitreal Injection of Autologous "Stem Cells" for AMD. New England Journal of Medicine, 2017. [Peer-reviewed study · citation details pending reviewer confirmation]