- According to a 2016 study by the NIH, visual impairment and blindness cases in the U.S. are expected to double by 2050.
- Three of the most common causes for vision loss are:
- Age-Related Macular Degeneration (AMD) — The number of Americans living with AMD is estimated at 3 million. AMD causes the layer of cells that support the photoreceptors to be destroyed, resulting in loss of central vision and gradual total vision loss.
- Retinitis Pigmentosa (RP) — Is an inherited and progressive disease that causes degeneration of photoreceptors, resulting in loss of peripheral vision and eventually leading to complete blindness.
- Limbal Stem Cell Deficiency — Is an inherited disease caused by the loss of stem cells that refresh the outer surface of the eye, called the cornea, resulting in blurred vision and gradual total vision loss.
How Stem Cells Can be Used to Treat Vision Loss
- Age-Related Macular Degeneration — The retina’s layer of support cells called retinal pigment epithelial cells (RPE cells), can be replaced and/or protected using stem cells.
- Retinitis Pigmentosa — Stem cells can be used to prevent the photoreceptors from dying, and potentially replacing already-destroyed cells with new ones.
- Limbal Stem Cell Deficiency — Limbal stem cells can be derived, expanded, and delivered to the eye to rebuild the cornea.
CIRM’s Progress: Selected Research Highlights
- CIRM has funded six clinical trials in the area of eye disease and vision loss across five California institutions. Some examples include:
- In a Phase 1 clinical trial, USC and UC Santa Barbara scientists used stem cells to develop a scaffold that could be inserted into the back of the eyes of patients with AMD to either replace the dying neurons or protect the existing ones. This trial was shown to be safe and the results were promising, leading to preparation for a Phase 2 trial. Read more about one patient’s experience with AMD and her involvement in this CIRM-funded trial here.
- In a Phase 2b clinical trial, UC Irvine researchers, in collaboration with the company, jCyte, are using stem cells to generate retinal progenitor cells — the supporting cells for the photoreceptors — and deliver those to patients with Retinitis Pigmentosa. In this way, still-healthy photoreceptors may be protected from damage by the disease or new photoreceptors may replace those already lost. At the optimal dose, all the patients in this trial showed an average improvement of 2 complete lines on the eye exam. These data were further corroborated in 12-month analyses where the functional benefit of high dose delivery of stem cell-derived retinal progenitors was demonstrated. Read more about one patient’s experience with Retinitis Pigmentosa and her encouraging involvement in this CIRM-funded trial here.
- In a Phase 1 clinical trial, UCLA researchers are testing the safety of taking the remaining limbal stem cells from LSCD patients, expanding them in the laboratory and returning them to the patient’s eye.