New clinical trial offers hope for patients with advanced dry” age-related macular degeneration

New clinical trial offers hope for patients with advanced dry” age-related macular degeneration

Age-related macular degeneration, the leading cause of vision loss and blindness in Americans age 65 and older, is a progressive disease that affects central vision. Over time, faces, book pages, and anything directly in front of a person become obscured by blurry, dark, or blind spots.

Now a novel clinical study offers hope for patients with advanced “dry” age-related macular degeneration. Dry age-related macular degeneration is the most common form of the disease.

Researchers at the USC Roski Eye Institute, part of Keck Medicine of USC, are launching a Phase 2b clinical trial studying whether bioengineered stem cells designed to replace cells in the retina damaged by macular degeneration could restore vision. The cells are attached to an implant – an ultra-thin patch thinner than a strand of hair – that holds the cells in place.

We hope to find out whether the stem cell-based retinal implant can not only stop the progression of dry age-related macular degeneration but actually improve patients' vision. The results could be groundbreaking because although there are some treatment options that slow the progression of macular degeneration, there are none that are able to reverse the damage that has already been done.”

Sun Young Lee, MD, PhD, retinal surgeon at Keck Medicine and principal investigator of the Keck Medicine Study Center

The clinical trial follows early research conducted by experts at the USC Roski Eye Institute on a small group of patients that showed the implant was well tolerated, remained in the eye and was successfully absorbed into retinal tissue. In addition, 27% of patients had some improvement in vision.

"The earlier phase of the clinical trial demonstrated that the treatment is safe and has the potential to improve patients' vision. The next phase will examine whether the therapy can produce clinically significant improvements in vision," said Lee, who is also an associate professor of ophthalmology and physiology and neuroscience at the Keck School of Medicine of USC.

How the retinal implant works

Approximately 20 million Americans live with age-related macular degeneration. This number also includes cases of wet macular degeneration, a rarer but more serious form of the disease.

Age-related macular degeneration affects the macula of the eye, which is located in the center of the retina and is responsible for central vision. In advanced cases, the retinal pigment epithelium (RPE) cells, which line the macula and are critical for producing clear vision in the retina, become damaged or destroyed, resulting in vision loss.

The retinal implant used in the clinical trial is derived from embryonic stem cells that are grown into RPE cells in a laboratory. During an outpatient surgical procedure, Keck Medicine eye surgeons implant a tiny layer of the lab-made RPE cells into the retina.

“The study will examine whether the lab-made implant will replace the damaged cells, function like normal RPE cells, and improve vision in patients who may not currently have other options for improvement,” said Rodrigo Antonio Brant Fernandes, MD, PhD, an ophthalmologist at Keck Medicine and study surgeon.

Clinical trial details

Keck Medicine is one of five sites in the country enrolling patients in the clinical trial. The study is masked – some enrolled participants will receive the implant while others will receive a simulated implant.

Eligible patients must be between 55 and 90 years old and have advanced dry age-related macular degeneration and have been diagnosed with geographic atrophy, meaning their RPE cells are damaged or not functioning.

Patients will be monitored for at least a year to determine how the implant is tolerated and whether there are any changes in vision. 24 patients will take part in the study.

Anyone interested in learning more about the process can contact Mariana Edwards at [email protected] or Kimberly Rodriguez at [email protected].

“The USC Roski Eye Institute is dedicated to advancing innovative treatments to improve lives by restoring vision,” said Dr. “Stem cell-derived retinal implants may offer one of the greatest opportunities to help patients with dry age-related macular degeneration and potentially one day provide a cure.”

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