Advanced Cell Technology reports positive early results from embryonic stem cell trial

By Chelsea Conaboy, Globe Staff

Researchers at the University of California, Los Angeles, and Advanced Cell Technology of Marlborough have become the first to publish a study involving the use of embryonic stem cells in humans.

The study, published online in the British medical journal The Lancet and involving just two patients, was designed to test the safety of injecting the cells into patients with degenerative eye conditions. In both patients, the cells behaved as expected after four months, with no safety concerns arising, the researchers reported today, and the patients reported improvement in their vision.

The study provides a boost for the beleaguered field of embryonic stem cell research but must be view cautiously, said Dr. George Q. Daley, director of the Stem Cell Transplantation Program at Children’s Hospital Boston and a faculty member at the Harvard Stem Cell Institute.

“We’re all enthusiastic to see actual trials of cells based on human embryonic stem cells, but it really is far too preliminary to conclude anything other than that more studies are warranted,” he said. “What we have to do is temper our hope with real skepticism.”

The researchers injected one eye of each patient with specialized eye cells derived from embryonic stem cells, which promote the health of photoreceptors in the eye. One, an adult woman, had Stargardt disease, a form of inherited juvenile macular degeneration. The other had age-related macular degeneration.

Dr. Robert Lanza, an author of the study and chief scientific officer at Advanced Cell Technology, a publicly traded company that funded the research, said the fact that the patients both reported improvements in their vision was “a bonus,” though he acknowledged that some of the change could be attributed to the placebo effect, or the patient’s own expectation for improvement as a result of the study.

The patient with Stargardt disease could detect hand motion before the injection. Within two weeks afterward, she could count fingers, the study said. She also reported improvements in her ability to detect color with the injected eye. There was no change in her other eye, the study said. The other patient showed improvement in reading a vision chart.

“In these advanced patients it would be hard to expect much improvement but we’re surprised,” Lanza said.

The patients were the first two in a set of trials that will study the use of the cells in a total of 24 people. The researchers injected the first patient in a separate trial in Europe on Friday.

The study authors said the goal of the research ultimately will be to treat patients with these conditions early on, with the hope of stopping or slowing the degenerative process. Lanza said their work could help to pave the way for the regenerative use of other kinds of cells, such as adult skin cells that are manipulated to behave like embryonic stem cells.