Future path4 min read
Induced pluripotent stem cells
iPSC
Induced pluripotent stem cells are mature adult cells that scientists have reprogrammed in the laboratory. Their use to replace damaged cells is being tested, including an early trial in eye disease, but they are not an approved treatment.
In 2019 the NIH launched the first clinical trial in the United States of a therapy using stem cells derived from the patient themselves to replace dying cells in the retina in people with geographic atrophy, the advanced "dry" form of age-related macular degeneration. The trial is phase I/IIa in 12 patients, and the first American patient received the treatment in 2022.
The primary outcome the trial measures is the safety of implanting the patch, and it is not expected to improve participants' vision enough to help with reading. The NIH says major technical obstacles remain and will probably be overcome only after years of intensive research. The FDA warns against unapproved stem cell products that may carry serious safety problems; the only approved stem cell products it has are blood-forming cells derived from cord blood.
- In widespread clinical use
- Approved for specific uses
- In clinical trials
- Preclinical
- Concept
A phase I/IIa trial of the first American therapy from patient-derived iPS cells in eye disease; and the only stem cell products approved by the FDA are blood-forming cells from cord blood.
- Retinal cell patch for patients with geographic atrophy (advanced dry macular degeneration)In clinical trials
A first-in-human phase I/IIa trial, with safety as its primary aim.21
- "Stem cell" products marketed without approval
The FDA warns that they may carry serious safety problems that put patients at risk.3
Status last checked: · What the maturity levels mean
What is this path?
In 2006 researchers identified conditions that allow some mature adult human cells to be reprogrammed, and reprogrammed stem cells are called "induced pluripotent stem cells" (iPSCs); see Stem cells.
They can be derived from the patient themselves, as in the NIH trial that uses replacement tissue from patient-derived iPS cells.
Where does the research stand?
In December 2019 the NIH launched the first American clinical trial of a therapy using patient-derived stem cells to replace dying cells in the retina, under a phase I/IIa protocol involving 12 patients with advanced geographic atrophy.
In August 2022 the first patient in the United States received replacement tissue made from iPS cells derived from them.
Geographic atrophy is the advanced "dry" form of age-related macular degeneration, and the trial is a single-arm "first-in-human" type; see Ophthalmology.
What separates it from clinical use?
The primary outcome the trial measures is the safety of implanting the patch, and it is not expected to improve participants' vision enough to help with reading.
Major technical obstacles remain, and will probably be overcome only after years of intensive research.
The only stem cell products approved by the FDA in the United States are blood-forming cells derived from cord blood, and unapproved products may carry serious safety problems.
Common questions
I was offered stem cell treatment at a private clinic; is it safe?
The FDA warns that unapproved stem cell products may carry serious safety problems that put patients at risk, and the only approved stem cell products it has are blood-forming cells from cord blood. Discuss any such offer with your doctor before any decision.3
Questions for your doctor
- Is this treatment approved by a regulator, or is it offered within a registered clinical trial?
- What proven treatments are available for my condition now?
References
- 1NIH. NIH launches first U.S. clinical trial of patient-derived stem cell therapy to replace dying cells in retina. www.nih.gov/news-events/news-releases/nih-launches-first-us-clinical-trial-patient-derived-stem-cell-therapy-replace-dying-cells-retinaHealth agencies & guidelines · Accessed 2026-10-08
- 2National Eye Institute (NIH). Clinical Trial Highlight: Stem Cell Transplants for Dry AMD. www.nei.nih.gov/eye-health-information/eye-conditions-and-diseases/age-related-macular-degeneration/clinical-trial-highlight-stem-cell-transplants-dry-amdHealth agencies & guidelines · Accessed 2026-10-08
- 3U.S. FDA. Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes. www.fda.gov/vaccines-blood-biologics/consumers-biologics/consumer-alert-regenerative-medicine-products-including-stem-cells-and-exosomesHealth agencies & guidelines · Accessed 2026-10-08
- 4NIH Stem Cell Information. Stem Cell Basics. stemcells.nih.gov/info/basics/stc-basicsHealth agencies & guidelines · Accessed 2026-10-08
- 5NIH. First U.S. patient receives autologous stem cell therapy to treat dry AMD. www.nih.gov/news-events/news-releases/first-us-patient-receives-autologous-stem-cell-therapy-treat-dry-amdHealth agencies & guidelines · Accessed 2026-10-08
Review status: Edited content · Last updated:
Change log
- — Page created.
Educational content only — no diagnosis, and no substitute for a clinician.