The New York Times

Tuesday, October 14, 2014

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Study Backs Use of Stem Cells in Retinas

By ANDREW POLLACK OCT. 14, 2014

Dr. Steven D. Schwartz stands in his office alongside Snellen eye chart for testing vision.
Dr. Steven D. Schwartz said the stem cell treatment provided hope for regenerative medicine.
Credit: Monica Almeida/The New York Times

Since they were first isolated 16 years ago, human embryonic stem cells have been thought to have the potential to replace the body’s worn-out tissues and treat a wide variety of diseases.

The progress has been slow. But now researchers are reporting an encouraging step.

A therapy for eye diseases that was derived from stem cells appeared to be safe and might have improved the vision of some patients, according to a new study.

The results, published on Tuesday evening by the journal The Lancet, represent the most extensive human data yet on any treatment derived from such embryonic stem cells. The 18 patients in the study were followed for a median of 22 months, two of them for more than three years.

“This is a promising study, and it provides a lot of hope for regenerative medicine,” said Dr. Steven D. Schwartz, a retina specialist at the University of California, Los Angeles, and the lead author of the paper. “However, there’s a lot of work to be done.”

Human embryonic stem cells have been the focus of pitched ethical and political battles because they are typically created by destroying human embryos. Advanced Cell Technology, the company that sponsored this study, says it can create embryonic cells without destroying embryos, although at least one embryo was destroyed for the stem cells used in this study.

Stem cells, which can be turned into any type of cell in the body, could become a source of replacement tissue. But there has been concern that such cells, once put into the body, might turn into unwanted types of cells — bone cells in the eye, for instance — or form tumors.

In this study, that did not happen. If it had, it could have set the field back.

Advanced Cell Technology transformed embryonic stem cells into retinal pigment epithelium cells, which provide support for the light-sensing cells in the eye.

These pigmented cells were implanted in a short surgical procedure into one eye in each of the 18 patients. Half the patients had the dry form of age-related macular degeneration, and the others had another degenerative retinal disease called Stargardt’s macular dystrophy.

Vision improved by what is considered a significant amount in eight of the 18 treated eyes. By comparison, the untreated eyes of those same patients did not show an improvement.

Dr. Robert Lanza, the chief scientific officer of Advanced Cell Technology, said one rancher was able to ride his horse again. Another patient, a man, “cried and hugged me,” he said.

But outside experts said the improvements might have been caused not by the transplanted cells but by the procedure, the cataract surgeries some patients also had or a placebo effect, since patients knew which eye was treated.

“You stick a needle in a mouse eye, you get an effect,” said Dr. Martin Friedlander, a professor of cell and molecular biology at the Scripps Research Institute. “You can’t really say anything about efficacy at this point.”

The researchers said pigment levels increased in 13 eyes, a sign that the transplanted cells might have taken up residence, but increased pigment did not correlate with improved vision. And some other measures of retinal health did not improve.

“It’s not to the point where it’s going to make me jump up and down for joy,” Steve Rose, chief research officer at the Foundation Fighting Blindness, said of the data. He noted that while the cells themselves were apparently safe, there were side effects from the immune-suppressing drugs taken to prevent rejection of the cells as well as an infection from the surgical procedure.

Dr. Schwartz and Dr. Lanza were criticized by their colleagues for premature optimism when they published an earlier paper on this study in early 2012, after only two patients had been treated and followed for only a few months.

The trial is one of only a few involving human embryonic stem cells. Asterias Biotherapeutics is planning a trial in patients with spinal cord injury, following up on a trial that was halted in 2011 by a different company, Geron. ViaCyte will soon begin testing insulin-producing cells derived from embryonic stem cells as a treatment for Type 1 diabetes.

Attention in the field has shifted somewhat to so-called induced pluripotent stem cells, which can be made from skin rather than embryos. The first clinical trial of a therapy derived from such cells began recently in Japan — to treat a form of macular degeneration.

The eye is shaping up to be an early testing ground for various cell therapies, with several trials underway or planned.

One reason is that relatively few transplanted cells are needed. Another is that the eye is shielded to some extent from the immune system, making it less likely that transplanted cells will be rejected. Patients in the Lancet study used immune-suppressing drugs for only 13 weeks, while the recipient of a transplanted organ would most likely require such drugs for life.