Isabella Beukes, of Santa Rosa, Calif., has been legally blind for more than 40 years. An experimental treatment derived from embryonic stem cells seems to have enabled her now to see not just color but also some shapes. Tim Hussin for NPR hide caption

Isabella Beukes, of Santa Rosa, Calif., has been legally blind for more than 40 years. An experimental treatment derived from embryonic stem cells seems to have enabled her now to see not just color but also some shapes.

Scientists are reporting the first strong evidence that human embryonic stem cells may be helping patients.

The cells appear to have improved the vision in more than half of the 18 patients who had become legally blind because of two progressive, currently incurable eye diseases.

The researchers stress that the findings must be considered preliminary because the number of patients treated was relatively small and they have only been followed for an average of less than two years.

But the findings are quite promising. The patients had lost so much vision that there was no expectation that they could benefit, the researchers say.

"I'm astonished that this is working in the way that it is or seems to be working," says Steven Schwartz, a UCLA eye specialist who led the study, which was published Tuesday in the British medical journal The Lancet. "I'm very excited about it."

Other researchers agreed the work is preliminary, but also highly promising.

"It really does show for the very first time that patients can, in fact, benefit from the therapy," says Dr. Anthony Atala, a surgeon and director of the Wake Forest Institute for Regenerative Medicine at Wake Forest University.

What we did is put them into patients who have a disease where those particular cells are dying; and we replaced those dying tissues with new tissue that's derived from these stem cells. In a way it's a retinal transplant.

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Embryonic Stem Cells Restore Vision In Preliminary Human Test

LONDON (AP) - An experimental treatment for blindness that uses embryonic stem cells appears to be safe, and it improved vision in more than half of the patients who got it, two early studies show.

Researchers followed 18 patients for up to three years after treatment. The studies are the first to show safety of an embryonic stem cell treatment in humans for such a long period.

Its a wonderful first step but it doesnt prove that (stem cells) work, said Chris Mason, chair of regenerative medicine at University College London, who was not part of the research. He said it was encouraging the studies proved the treatment is safe and dispelled fears about stem cells promoting tumor growth.

Embryonic stem cells, which are recovered from embryos, can become any cell in the body. They are considered controversial by some because they involve destroying an embryo and some critics say adult stem cells, which are derived from tissue samples, should be used instead.

Scientists have long thought about transforming them into specific types of cells to help treat various diseases. In the new research, scientists turned stem cells into retinal cells to treat people with macular degeneration or Stargardts macular dystrophy, the leading causes of blindness in adults and children.

In each patient, the retinal cells were injected into the eye that had the worst vision. Ten of the 18 patients later reported they could see better with the treated eye than the other one. No safety problems were detected. The studies were paid for by the U.S. company that developed the treatment, Advanced Cell Technology, and were published online Tuesday in the journal, Lancet.

Dr. Robert Lanza, one of the study authors, said it was significant the stem cells survived years after the transplant and werent wiped out by the patients own immune systems. For some of the patients, Lanza noted their improved vision changed their lives, referring to a 75-year-old horse rancher who had been blind in one eye before the treatment.

One month after his treatment, his vision had improved (substantially) and he can even ride his horses again, Lanza said in an email. He said other patients have regained their independence with their newfound vision and said some people are now able to use their computers again, read their watches or travel on their own.

The next step will be to prove these (stem cell) treatments actually work, Mason said. Unless there is a sham group where you inject saline into (patients) eyes, we cant know for sure that it was the stem cells that were responsible.

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Stem cells to treat blindness appear to be safe

Published: 1:48PM Wednesday October 15, 2014 Source: AP

An experimental treatment for blindness that uses embryonic stem cells appears to be safe, and it improved vision in more than half of the patients who got it.

Researchers followed 18 patients for up to three years after treatment.

The studies are the first to show safety of an embryonic stem cell treatment in humans for such a long period.

"It's a wonderful first step but it doesn't prove that (stem cells) work," said Chris Mason, chair of regenerative medicine at University College London, who was not part of the research.

He said it was encouraging the studies proved the treatment is safe and dispelled fears about stem cells promoting tumour growth.

Embryonic stem cells, which are recovered from embryos, can become any cell in the body.

They are considered controversial by some because they involve destroying an embryo and some critics say adult stem cells, which are derived from tissue samples, should be used instead.

Scientists have long thought about transforming them into specific types of cells to help treat various diseases.

In the new research, scientists turned stem cells into retinal cells to treat people with macular degeneration or Stargardt's macular dystrophy, the leading causes of blindness in adults and children.

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Stem cells successfully treat blindness

TIME Health medicine Stem Cells Allow Nearly Blind Patients to See Stem cells could lead to new treatments for eye disorders Photography by Peter A. KemmerGetty Images/Flickr RF Embryonic stem cells can be turned into a therapy to help the sight of the nearly blind

In a report published in the journal Lancet, scientists led by Dr. Robert Lanza, chief scientific officer at Advanced Cell Technology, provide the first evidence that stem cells from human embryos can be a safe and effective source of therapies for two types of eye diseasesage-related macular degeneration, the most common cause of vision loss in people over age 60, and Stargardts macular dystrophy, a rarer, inherited condition that can leave patients legally blind and only able to sense hand motions.

In the study, 18 patients with either disorder received transplants of retinal epithelial cells (RPE) made from stem cells that came from human embryos. The embryos were from IVF procedures and donated for research. Lanza and his team devised a process of treating the stem cells so they could turn into the RPE cells. In patients with macular degeneration, these are the cells responsible for their vision loss; normally they help to keep the nerve cells that sense light in the retina healthy and functioning properly, but in those with macular degeneration or Stargardts, they start to deteriorate. Without RPE cells, the nerves then start to die, leading to gradual vision loss.

MORE: Stem Cell Miracle? New Therapies May Cure Chronic Conditions Like Alzheimers

The transplants of RPE cells were injected directly into the space in front of the retina of each patients most damaged eye. The new RPE cells cant force the formation of new nerve cells, but they can help the ones that are still there to keep functioning and doing their job to process light and help the patient to see. Only one RPE can maintain the health of a thousand photoreceptors, says Lanza.

The trial is the only one approved by the Food and Drug Administration involving human embryonic stem cells as a treatment. (Another, the first to gain the agencys approval, involved using human embryonic stem cells to treat spinal cord injury, but was stopped by the company.) Because the stem cells come from unrelated donors, and because they can grow into any of the bodys many cells types, experts have been concerned about their risks, including the possibility of tumors and immune rejection.

MORE: Early Success in a Human Embryonic Stem Cell Trial to Treat Blindness

But Lanza says the retinal space in the eye is the ideal place to test such cells, since the bodys immune cells dont enter this space. Even so, just to be safe, the patients were all given drugs to suppress their immune system for one week before the transplant and for 12 weeks following the surgery.

While the trial was only supposed to evaluate the safety of the therapy, it also provided valuable information about the technologys potential effectiveness. The patients have been followed for more than three years, and half of the 18 were able to read three more lines on the eye chart. That translated to critical improvements in their daily lives as wellsome were able to read their watch and use computers again.

Our goal was to prevent further progression of the disease, not reverse it and see visual improvement, says Lanza. But seeing the improvement in vision was frosting on the cake.

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Stem Cells Allow Nearly Blind Patients to See

No effective treatments exist for either (AMD) or Stargardt's macular dystrophy, both of which can result in complete blindness caused by the loss of light-receiving photoreceptor cells in the retina.

The new treatment uses stem cells to recreate a type of cell in the retina that supports those photoreceptors.

Stem cells derived from embryos that are only a few days old have the ability to develop into any kind of tissue in the body.

By bathing the stem cells in a specially formulated cocktail of chemicals the scientists were able to stimulate them into turning into fully mature retinal pigment epithelium cells. They were then transplanted directly into the eyes of patients suffering from blindness.

Tests showed substantial improvement in 10 of 18 treated eyes. Eight patients were able to read more than 15 additional letters on a sight chart in their first year after treatment.

"Embryonic stem cells have the potential to become any cell type in the body, but transplantation has been complicated by problems including the risk of tumour formation and immune rejection, said Professor Robert Lanza, chief scientific officer at the US company Advanced Cell Technology, which funded the research.

"As a result, sites that do not produce a strong immune response, such as the eye, have become the first parts of the human body to benefit from this technology."

The transplants have proved controversial because they use stem cells derived from spare human embryos left over from IVF treatment.

But experts said the announcement showed that such therapies could bring real benefits.

Anthony Atala, director of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, US, said: The work by Schwartz and colleagues is a major accomplishment, but the path to get to this point has not been smooth.

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Embryonic stem cells transplanted into eyes of blind restore sight

Embryonic stem cells transplanted into 18 patients with deteriorating eyesight restored some vision in more than half the volunteers, a study into the fledgling technology has found.

Stem cells derived from embryos "could provide a potentially safe new source of cells for the treatment of various unmet medical disorders requiring tissue repair or replacement", the report's authors said on Tuesday.

The study marks a new chapter in the long story of embryonic stem cells, which after their discovery in the 1990s were hailed as a miracle cure but then ran into problems.

Published in The Lancet, the paper looked at a US trial of stem cells among 18 patients suffering from two tragic, degenerative diseases of the retina.

Nine had a condition called Stargardt's macular dystrophy, a leading cause of juvenile blindness, and nine had dry atrophic age-related macular degeneration, which occurs among the middle-aged and elderly.

There is no conventional treatment for either condition, which eventually leads to complete blindness as the retina's light-receiving cells die out.

The participants were injected with one of three different doses of retinal cells derived from early-stage embryos - 50,000, 100,000 or 150,000 cells.

The transplants were placed in a space under the retina of the worst-affected eye.

The patients were monitored for up to 37 months, for an average of 22 months.

Out of the 18 treated eyes, 10 showed substantial improvements in vision, as measured by the ability to read letters on a board. Of these, eight patients were able to read 15 additional letters in the first year after transplant.

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Stem cells restore sight in new study