Scientists have succeeded in restoring vision in blind people using stem cells for the first time

Three patients with severe visual impairment who received stem cell transplants saw significant improvements in their vision. The results lasted for more than a year. A fourth person with severe visual impairment also experienced improvements in their vision, but they did not last as long. The four were the first to receive reprogrammed stem cell transplants to treat damaged corneas, the publication reports. Nature.

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The results, described in the medical journal The Lancet, are impressive, said Kapil Bharti, a translational stem cell researcher at the US National Eye Institute.

"The results merit more patients trying this approach," says colleague Gene Loring of Scripps Research in La Jolla, California.

reprogrammed cells

What is the treatment? The outer layer of the cornea is supported by a reservoir of stem cells located in the limbal ring (the dark ring around the iris). When this important source of rejuvenation is depleted (a condition known as limbal stem cell deficiency, LSCD), scar tissue covers the cornea, eventually leading to blindness. This can result from eye injury or autoimmune and genetic diseases.

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Treatment for LSCD typically involves transplanting corneal cells derived from stem cells from a healthy human eye, an invasive procedure with uncertain results. When both eyes are affected, corneal transplants from deceased donors are possible, but sometimes the transplanted tissue is rejected by the recipient's immune system.

Koji Nishida, an ophthalmologist at Osaka University in Japan, and his colleagues used an alternative cell source, induced pluripotent stem (iPS) cells, to perform corneal transplants. They took blood cells from a healthy donor and first reprogrammed them to an embryonic state, then transformed them into a thin, transparent layer of corneal epithelial cells.

Between June 2019 and November 2020, the team enrolled two women and two men, aged 39 to 72, with LSCD in both eyes. During the surgery, the team scraped away the layer of scar tissue covering the damaged cornea in only one eye, then sewed in donor-derived epithelial sheets and placed a soft, protective contact lens on top.

Vision test

Two years after the transplant, none of the recipients had experienced any serious side effects. The grafts did not form tumors (a known risk of iPS cell growth) and showed no obvious signs of attack by the recipients’ immune systems, even in those who were not receiving immunosuppressants. But Bharti says more transplants are needed to ensure the procedure is safe.

Following transplantation, all four recipients experienced immediate improvement in vision and a reduction in the corneal area affected by LSCD. Improvements were maintained in all but one recipient, who experienced slight regression during the first year of follow-up.

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It’s not yet clear what exactly caused the improvement in vision, Bharti said. It’s possible that the transplanted cells themselves multiplied in the recipient’s cornea. It could also be because scar tissue was removed before the transplant, or because the transplant caused the recipient’s own cells to migrate from other areas of the eye and rejuvenate the cornea.

Nishida said they plan to begin clinical trials in March to evaluate the treatment's effectiveness. Several other iPS cell-based trials for eye diseases are currently underway around the world, Bharti said.

“These success stories show that we are moving in the right direction,” he concluded.

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