Stem Cells Seem Safe in Treating Eye Disease

A treatment based on embryonic stem cells clears a key safety hurdle, and might help restore vision.

Transplanted cells appear as a dark spot on the retina of a person with macular degeneration.

When stem cells were first culled from human embryos 16 years ago, scientists imagined they would soon be treating diabetes, heart disease, stroke, and many other diseases with cells manufactured in the lab.

Its all taken longer than they thought. But today, a Massachusetts biotech firm reported results from the largest, and longest, human test of a treatment based on embryonic stem cells, saying it appears safe and may have partly restored vision to patients going blind from degenerative diseases.

Results of the three-year study were described today in the Lancet by Advanced Cell Technology and collaborating eye specialists at the Jules Stein Eye Institute in Los Angeles, who transplanted lab-grown cells into the eyes of nine people with macular degeneration and nine with Stargardts macular dystrophy.

The idea behind Advanced Cells treatment is to replace retinal pigment epithelium cells, known as RPE cells, a type of caretaker tissue without which a persons photoreceptors also die, with supplies grown in laboratory. It uses embryonic stem cells as a starting point, coaxing them to generate millions of specialized retina cells. In the study, each patient received a transplant of between 50,000 and 150,000 of those cells into one eye.

The main objective of the study was to prove the cells were safe. Beyond seeing no worrisome side effects, the researchers also noted some improvements in the patients. According to the researchers, half of them improved enough to read two to three extra lines on an eye exam chart. Robert Lanza, chief scientific officer of Advanced Cell, called these results remarkable.

We have people saying things no one would make up, like Oh I can see the pattern on my furniture, or now I drive to the airport, he says. Clearly there is something going on here.

Lanza stresses the need for a larger study, which he says the company hopes to launch later this year in Stargardts patients. But if the vision results seen so far continue, Lanza says, this would be a therapy.

Some eye specialists said its too soon to say whether the vision improvements were real. The patients werent examined by independent specialists, they said, and eyesight in patients with low vision is notoriously difficult to measure. That leaves plenty of room for placebo effects or unconscious bias on the part of doctors.

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Stem Cells Seem Safe in Treating Eye Disease