Retinal disease, including retinitis pigmentosa, age-related macular degeneration, and Stargardt Disease, is one of the leading causes of vision loss in the country. Patients can inherit these retinal diseases, which usually result in the loss of photoreceptor cells or retinal epithelium cells, the cells that line the back of the retina. These diseases have become the most common cause of blindness in the working-age population in certain countries, prompting scientists and clinicians to seek solutions. (1)
One of the emerging treatments being studied is the use of stem cells. Stem cell therapy for vision loss aims to replace damaged retinal cells with new stem cells, potentially slowing vision loss and restoring essential retinal function.
Dr. John C. Haasis III is the medical director and founder of the Daisy Institute. Dr. Haasis offers more than 25 years of experience and has treated thousands of patients with cutting-edge treatments and technologies. When he is not seeing patients, Dr. Haasis participates in research and furthers the field of regenerative medicine.
Please note: The Daisy Institute does not currently offer stem cell treatments for vision loss. This article is intended to provide general information about ongoing research in stem cell therapies for eye diseases and vision restoration.
If you would like to learn more about stem cells, please contact us online or call (864) 775-5682 to reach the office closest to you:
Contents
About Stem Cells
Stem cells are special types of cells that can self-renew and differentiate into cells found in other tissues and organs in the body. These cells have been heavily researched for their potential therapeutic effects, such as regenerating damaged cells, stimulating the production of new red blood cells, and reducing cell death. (2) While these trials are still in their investigational and early phases, they have shown promising developments, suggesting that stem cells may help with vision loss and eye diseases.
Types of Stem Cells
There are numerous types of stem cells found within the body, each possessing its own unique functions. However, researchers have not investigated every type of stem cell for degenerative retinal applications. Instead, they have identified specific cells for their potential during testing, as well as their function within the body.
Pluripotent Stem Cells
Pluripotent stem cells are a specific type of stem cell that can differentiate into various cell types with extensive self-renewal potential. (3)
- Induced Pluripotent Stem Cells (iPSCs): Induced pluripotent stem cells are a type of pluripotent stem cell often derived from adult skin and blood. These cells can reprogram into an embryonic-like pluripotent state, allowing them to proliferate almost indefinitely. (4)
- Human Embryonic Stem Cells (hESCs): Embryonic stem cells are specific stem cells found in the inner cell mass of a human blastocyst. These cells can differentiate into the endoderm, ectoderm, and mesoderm, the three fundamental germ layers that form each organ within the body.
Multipotent Stem Cells
Multipotent stem cells are stem cells that can evolve into cells that perform specific, specialized functions. These cells are often found within the bone marrow. Primary multipotent stem cell research currently focuses on:
- Mesenchymal Stem Cells (MSCs): Mesenchymal stem cells have a multipotential capability and derive from bone marrow, adipose tissue, and umbilical cord blood. These cells can differentiate into diverse types of cells, including retinal cells.
- Retinal Progenitor Cells (hRPCs): Human retinal progenitor cells derive from fetal retinas. These cells can differentiate into all retinal cell types under certain conditions, and researchers are currently studying how they slow the rate of regional degeneration. (5)
About Vision Loss and Eye Diseases
Vision loss is a widespread concern that affects millions of individuals around the world. Vision loss and impairment can stem from age-related concerns, genetic disorders, and more. While numerous concerns can lead to blindness and poor vision, stem cell researchers have primarily focused on addressing diseases and disorders such as age-related macular degeneration, retinitis pigmentosa, and Stargardt Disease.
Age-related macular degeneration (AMD) is a degenerative eye disease and the leading cause of severe vision impairment in elderly patients. This accumulation of drusen—tiny fat deposits—near the outer retina characterizes this disorder. This buildup can damage the photoreceptors and lead to central vision loss. AMD affects 196 million people worldwide, with approximately 20 million of those patients residing within the United States. (6)
Retinitis Pigmentosa (RP)
Retinitis pigmentosa is a group of genetic disorders that gradually degenerate the photoreceptors and light-processing cells in the retina. RP typically presents itself as night blindness or a progressive loss of peripheral vision, leading to severe visual impairment and reductions in one’s quality of life. (7)
Stargardt Disease (STGD)
Stargardt Disease is an inherited eye disease that develops in adolescents. This disease causes the central part of the retina to progressively stop functioning, leading to gradual vision loss. Stargardt Disease causes the metabolic waste material to accumulate within the retinal pigment epithelium, leading to central vision loss. (8)
Macular degeneration stands as a dominant cause of severe visual impairment, which has prompted research for effective treatments and therapies. Clinical trials and research have been focusing on transplanting stem cells into areas of concern to replace or repair damaged retinal cells and photoreceptors. These trials focused on using human embryonic stem cells, human induced pluripotent stem cells, and mesenchymal stem cells.
Trials Using Human Embryonic Stem Cells
Clinical trials have been conducted to study the effects of potential stem cell therapies on retinal disorders and concerns. These studies consisted of taking retinal pigment epithelium cells derived from human embryonic stem cells and administering them into the space beneath the retina. The patients were also given immunosuppressive drugs to prevent potential rejection, as the cells came from donors. Most patients in the study showed signs that the transplanted cells were able to survive and integrate within the eye; however, several side effects were experienced across multiple patients. Researchers performed another follow-up study years later that reconfirmed potentially positive results, with no abnormal tumor growth or tissue formations. (9)
Trials Using Human Induced Pluripotent Stem Cells
In Japan, researchers performed a study using autologous hiPSCs to treat a patient with macular degeneration. This treatment usually features a scaffold, but the autologous nature of the treatment eliminated the need for scaffolding. There were disruptions to this trial due to regulation changes, but the treated patient’s condition stayed stable with no noted improvements or declines. (9)
Stem Cells for Retinitis Pigmentosa
Trials Using Induced Pluripotent Stem Cells
In 2025, a pharmaceutical company started a prospective, observational study to study the efficacy of allogeneic iPS cell-derived retinal sheets. These stem cells are taken from a donor and structured into a 3D retina using specialized lab techniques. The 3D retina is then turned into a sheet, which contains multiple layers like a natural retina. The sheets contain photoreceptor precursor cells, which are immature cells that may mature once transplanted. These cells are vital for vision.
This trial is ongoing, but it demonstrates great potential for providing a widespread treatment for retinitis pigmentosa. The involved parties in the study have expressed interest in commercial application and hope to offer a new treatment to patients as soon as possible. (10)
Trials Using Mesenchymal Stem Cells
Studies found that infusing the vein with human umbilical cord mesenchymal stem cells (UCMSCs) may improve clarity of vision. Researchers conducted a clinical trial where they injected UCMSCs into the fatty tissue between the choroid and sclera layers of the eyes. There were no serious reactions observed, demonstrating the safety of the cell infusion. (7)
Stem Cells for Stargardt Disease
Trials Using Human Embryonic Stem Cells
One investigation studied the safety of human embryonic stem cells for early-stage Stargardt Disease. Throughout the course of the treatment, patients were given clinical-grade human embryonic stem cell-derived retinal pigment epithelial (hESC-RPE) cells. They were followed over the course of 5 years, and while there were no serious adverse effects, there were no significant improvements in vision. The most serious risk for this treatment was tumor formation, but no tumors were found in any patients, making this treatment deemed well-tolerated and safe for early-stage Stargardt Disease. (11)
References
- Chen X, Xu N, Li J, Zhao M, Huang L. Stem cell therapy for inherited retinal diseases: a systematic review and meta-analysis. Stem Cell Research & Therapy. 2023;14(1). doi:https://doi.org/10.1186/s13287-023-03526-x
- Ghasroldasht MM, Seok J, Park HS, Liakath Ali FB, Al-Hendy A. Stem Cell Therapy: From Idea to Clinical Practice. International Journal of Molecular Sciences. 2022;23(5):2850. doi:https://doi.org/10.3390/ijms23052850
- Abu-Dawud R, Graffmann N, Ferber S, Wruck W, Adjaye J. Pluripotent stem cells: induction and self-renewal. Philosophical Transactions of the Royal Society B: Biological Sciences. 2018;373(1750):20170213. doi:https://doi.org/10.1098/rstb.2017.0213
- Cerneckis J, Cai H, Shi Y. Induced Pluripotent Stem Cells (iPSCs): Molecular Mechanisms of Induction and Applications. Signal Transduction and Targeted Therapy. 2024;9(1). doi:https://doi.org/10.1038/s41392-024-01809-0
- Wang Z, Gao F, Zhang M, et al. Intravitreal Injection of Human Retinal Progenitor Cells for Treatment of Retinal Degeneration. Medical Science Monitor. 2020;26. doi:https://doi.org/10.12659/msm.921184
- Fleckenstein M, Steffen Schmitz-Valckenberg, Chakravarthy U. Age-Related Macular Degeneration: A Review. JAMA. 2024;331(2):147-157. doi:https://doi.org/10.1001/jama.2023.26074
- Qi XY, Cao DR, Chen XQ, Zhang P. Retinitis pigmentosa and stem cell therapy. International Journal of Ophthalmology. 2024;17(7):1363-1369. doi:https://doi.org/10.18240/ijo.2024.07.22
- Kohli P, Kaur K. Stargardt Disease. PubMed. Published 2022. https://www.ncbi.nlm.nih.gov/books/NBK587351/
- Radu M, Daniel Constantin Brănișteanu, Ruxandra Angela Pirvulescu, Otilia Maria Dumitrescu, Mihai Alexandru Ionescu, Zemba M. Exploring Stem-Cell-Based Therapies for Retinal Regeneration. Life. 2024;14(6):668-668. doi:https://doi.org/10.3390/life14060668
- Initiation of Phase1/2 Study on Allogeneic iPS Cell-derived Retinal Sheet for Retinitis Pigmentosa in the United States | Sumitomo Pharma. Sumitomo-pharma.com. Published 2024. Accessed October 31, 2025. https://www.sumitomo-pharma.com/news/20241129.html
- Li SY, Liu Y, Wang L, et al. A phase I clinical trial of human embryonic stem cell-derived retinal pigment epithelial cells for early-stage Stargardt macular degeneration: 5-year follow-up. Cell Proliferation. 2021;54(9):e13100. doi:https://doi.org/10.1111/cpr.13100