Science Supplement: Stem Cell Therapy Can Significantly Improve Vision

Release date: 2018-04-11

Age related macular degeneration (AMD) can be said to be the visual "killer" of middle-aged and elderly people. In the United States, it is the number one cause of blindness among elderly people over the age of 65. As the world's aging society continues to rise, its incidence is also rising. AMD is divided into two types: dry and wet. Dry AMD is caused by the deterioration and death of retinal pigment epithelium (RPE), which leads to dysfunction and death of photoreceptor cells. Photoreceptor cells at the macular spot in the center of the retina are particularly sensitive, which causes the central vision of the elderly to gradually decrease, making it difficult to read, write, drive, and recognize faces. Currently, this disease is thought to cause irreversible damage to vision.

â–² This study was published on the cover of Science Translational Medicine (Source: Science Translational Medicine)

But a new study published last week in Science Translational Medicine brought hope to patients. In this study, researchers at the University of Southern California teamed up with other California institutes, companies, and hospitals to develop an RPE implant made from stem cells. Clinical 1/2a trial results show that this graft not only prevents further deterioration of the disease, but also significantly improves the vision of some patients.

Because the underlying cause of visual deterioration is the death of RPE cells, how to replace dead RPE cells becomes a key issue in the treatment of dry AMD. In this regard, human embryonic stem cell-derived RPE (hESC-RPE) seems to be the ideal choice. The cell culture process for differentiating hESC cells into RPE has become increasingly mature, and experiments have shown that injection of hESC-RPE cells into the subretinal can restore part of the RPE structure in patients with dry AMD.

â–² RPE cells differentiated from stem cells (Source: USC Roski Eye Institute)

However, this strategy also has its own drawbacks, because free hESC-RPE cells cannot be integrated into the designated location of the retina, which may result in the loss of hESC-RPE cells in areas of RPE loss in the retina, while other otherwise normal areas are hESC-RPE grows and the amount of pigment is extremely high.

The RPE implant designed by researchers at the University of Southern California solves this problem. This graft allows hESC-RPE cells to grow on a synthetic backbone into a single cell layer with the same structure as the RPE in the retina. And the artificial skeleton can help maintain the structure of the single cell layer so that they do not curl or collapse into a mass. The implant can be placed in the area of ​​the retina where RPE cells disappear by surgery, helping to restore vision in a particular area.

â–² RPE implants designed by researchers (Source: Science Translational Medicine)

In the clinical 1/2a trial, four patients with advanced dry AMD underwent a transplant. The first thing that needs to be confirmed is the safety of the surgery and the implant. A one-year follow-up examination of the patient showed that all patients who underwent surgery did not experience serious side effects. The graft was not implanted in the retina and there was evidence that it was integrated into the retina, which is a prerequisite for the transplant to improve vision. So does it improve the patient's visual ability?

Examination of the patient's visual ability showed that one of the four recipients with the transplant had a significant improvement in central vision, while two of the patients with previously missing RPE cells had visual function restored after the graft was obtained. The visual acuity of all treated patients did not deteriorate further.

"Our goal is to rejuvenate residual photoreceptor cells in the retina by transplanting healthy RPE cells and prevent further loss of these cells," Biomedical at the University of California, Santa Barbara. Professor Dennis Clegg said: "Ultimately, we hope to be able to perform transplants at an earlier stage of the disease, thus preventing patients from losing photoreceptor cells from the outset."

Reference materials:

[1] Researchers test stem cell-based retinal implant for common cause of vision loss

[2] Treating vision loss

[3] A bioengineered retinal pigment epithelial monolayer for advanced, dry age-related macular degeneration

Source: Academic Jingwei

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