AMD Stem Cell Therapy Shows Potential for Vision Gain

Stem cells could possibly be used to treat dry macular degeneration, according to research from 2 early clinical trials.

The studies, presented at the 121st Annual Meeting of the American Academy of Ophthalmology in New Orleans, LA, showed that ocular injection stem cell therapy may be capable of replacing damaged cells, and even improving vision, without serious side effects.

Initial researchers from Hadassah-Hebrew University Medical Center in Jerusalem, Israel, turned embryonic stem cells into retinal pigment epithelial (RPE) cells — designed to support the retinas — and injected them into the eyes of age-related macular generation (AMD) patients.

RPE cells provide healthy retinas nutrition while managing waste for photoreceptors — the cells that process light and allow sight. AMD causes dysfunction and degeneration of RPE cells.

With no therapies designed to fully treat AMD — the leading cause of new blindness in people aged 55 years or older — stem cells could make a major difference in in the 30 million-plus patients worldwide.

The Jerusalem-based researchers injected a suspension of either 50,000 or 200,000 stem-derived RPE cells underneath patients’ retinas. The patients reported well-tolerance, reportings signs of retina healing within a few weeks. Images taken of the back of the eye indiciated to researchers that the transplanted cells may have survived.

Eyal Banin, MD, PhD, lead investigator and one of the stem cell technology developers, said the team was “encouraged by the results thus far.”

“But this is just a first step in the long road towards making regenerative cell therapy a reality in macular and retinal degeneration,” Banin said.

The second study of the stem cell therapy came from a researchers at the Bascom Palmer Eye Institute in Miami, FL. The team conducted 2 studies of the stem-derived cells on dry AMD patients, and patients with Stargardt disease. The disease, the most common form of inherited juvenile AMD, is a different form of retinal degeneration with no available treatment.

Researchers found the implanted stem cells survived for up to 3 years, with no serious side effects. Some patients even gained vision.

Lead researcher Ninel Z. Gregori, MD, noted that the RPE cells appeared to be well-tolerated.

“There were no serious adverse events attributable to the transplanted RPE cells, including no tumor formation,” Gregori said. “This study supports further development of human embryonic stem cell-derived RPE for degenerative diseases of the macula.”

With reported efficacy and tolerance in short-term and long-term treatment of AMD, an additional trial for the investigatory stem cells is expected to be opened in the US soon.