Stem-Cell Blindness Treatment Shows Promise

An experimental first-of-its-kind stem cell therapy is one step closer to becoming a viable treatment option to help restore vision to people with advanced dry age-related macular degeneration — a leading cause of blindness.

A team of doctors and researchers at the University of Southern California Roski Eye Institute and other California institutions reported the results of a preliminary study of the treatment, a stem cell-based retinal implant, in the journal Science Translational Medicine.

The research, funded in part by the California Institute for Regenerative Medicine, found the treatment to be safe, caused no serious side effects, and was well tolerated by patients involved in the work, Medical Xpress reports.

"Our study shows that this unique stem cell-based retinal implant thus far is well-tolerated, and preliminary results suggest it may help people with advanced dry age-related macular degeneration," said lead research and implant inventor Dr. Mark S. Humayun, co-director of the Roski Institute.

The new treatment involved implanting a layer of stem cell-derived cells in the retinas of four patients by a USC Roski Eye Institute surgeon. In people with macular degeneration, retinal cells degenerate, leading to visions problems.

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The patients were followed for up to one year after surgery to assess the safety of the implants — a primary goal of early clinical trials; follow-up studies gauge effectiveness of new treatments.

This week, the researchers reported the implants were found to be safe for all of the patients and also integrated with the patients' retinal tissue, which is essential for the treatment to be able to improve visual function.

The research team also said a preliminary assessment of the therapy's efficacy was positive. One patient had improvement in visual acuity, which was measured by how many letters could read on an eye chart. Two patients had gains in visual function, measured by how well they could use the area of the retina treated by the implant.

None of the patients showed evidence of progression in vision loss

"This is the first human trial of this novel stem cell-based implant, which is designed to replace a single-cell layer that degenerates in patients with dry age-related macular degeneration," said Dr. Amir H. Kashani, MD, a surgeon and assistant professor of clinical ophthalmology at the Keck School of Medicine of USC. "This implant has the potential to stop the progression of the disease or even improve patients' vision. Proving its safety in humans is the first step in accomplishing that goal."

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Dry age-related macular degeneration is the most common type of the disease. Over time, it can lead to loss of central vision, effecting people's ability to perform daily tasks like reading, writing, driving, and seeing faces.

The condition affects 1.7 million Americans — a figure is projected to rise to nearly double by 2020. It is a leading cause of severe visual impairment in adults older than 65.

The USC research is the latest in a series of recent studies to suggest stem-cell therapies hold great promise in treating vision problems

Last month, British doctors reported a different type of stem cell therapy restored partial vision to two patients with age-related macular degeneration.

Embyronic stem cells were converted into patches of eye cells and grown in the lab, then inserted into the back of one eye in each of the patients.

A year after the transplants were performed, at Moorfields Eye Hospital in London, both patients report improved vision in the treated eye.

Another eight more patients are scheduled to take part in a follow-up clinical trial of the procedure, reported in the journal Nature Biotechnology.

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