Stem Cell Treatment Restores Sight to Patients in New Clinical Trial

A recently announced clinical trial using stem cell-derived ocular cells has shown preliminary results that are very promising. The first patients to receive the new treatment for people with wet age-related macular degeneration (AMD) have regained reading vision.
The study is a major milestone for the London Project to Cure Blindness, a partnership between Professor Pete Coffey from University College London and Professor Lyndon da Cruz, a retinal surgeon at Moorfields Eye Hospital NHS Foundation Trust. The Project has also been supported by the UCL Institute of Ophthalmology and the National Institute for Health Research (NIHR).

The results of this ground-breaking clinical study, published in Nature Biotechnology, described the implantation of a specially engineered patch of retinal pigment epithelium cells derived from stem cells to treat people with sudden severe sight loss from wet AMD.
The study investigated whether the diseased cells at the back of the patients’ affected eye could be replenished using the stem cell patch. A specially engineered surgical tool was used to insert the patch under the retina in the affected eye of each patient in an operation lasting one to two hours.
It is hoped that the treatment will also help treat dry AMD in the future. It’s the first description of a complete engineered tissue that has been successfully used in this way.
Developed in part by researchers at UC Santa Barbara, the team implanted the retinal eyepatch onto a subject at Moorfields Eye Hospital in London.
In the months before patient Douglas Waters’ surgery, his vision was poor and he couldn’t see anything out of his right eye at all. After the surgery, his eyesight improved so much that he could read the newspaper and help his wife with gardening.
“This study represents real progress in regenerative medicine and opens the door on new treatment options for people with age-related macular degeneration,” said the study co-author Coffey, a professor at the University of Santa Barbara’s Neuroscience Research Institute and co-director of the campus’s Center for Stem Cell Biology & Engineering.
“The results suggest that this new therapeutic approach is safe and provides good visual outcomes. The patients who received the treatment had very severe AMD, and their improved vision will go some way towards enhancing their quality of life," said Lyndon da Cruz, a consultant retinal surgeon at Moorfields Eye Hospital, "We recognize that this is a small group of patients, but we hope that what we have learned from this study will benefit many more in the future.”
Macular degeneration accounts for almost 50 percent of all visual impairment in the developed world and usually affects people over 50 years of age. AMD affects central vision, which is mainly used for reading, while leaving the surrounding vision normal. Wet AMD is generally caused by.abnormal blood vessels that leak fluid or blood into the region of the macula in the center of the retina, and it almost always begins as dry AMD.
In addition to Waters, a woman in her early 60s who also suffered from a severe form of wet AMD and declining vision underwent the procedure. She and Waters were monitored for 12 months and reported improvements to their vision. They went from not being able to read at all, even with glasses, to reading 60 to 80 words per minute with normal reading glasses.
“We hope this will lead to an affordable ‘off-the-shelf’ therapy that could be made available to NHS patients within the next five years,” said Coffey, who founded the London Project to Cure Blindness more than a decade ago.