USC Launches Phase 2b Trial of Stem Cell Retinal Implant for Advanced Dry Age-Related Macular Degeneration

Researchers at the USC Roski Eye Institute (search)Search term have launched a phase 2b clinical trial testing a novel stem cell-based retinal implant that could potentially restore vision in patients with advanced dry age-related macular degeneration (search)Search disease. The trial represents a significant advancement in treating the most common form of the leading cause of vision loss among Americans aged 65 and older.

Breakthrough Stem Cell Technology

The investigational treatment utilizes embryonic stem cells that have been bioengineered in laboratory conditions to develop into retinal pigment epithelium (RPE) cells. These lab-grown cells are attached to an ultra-thin patch implant, described as thinner than a strand of hair, which holds the cells in place once surgically inserted into the retina.

"We are hoping to determine if the stem-cell based retinal implant can not only stop the progression of dry age-related macular degeneration (search)Search disease, but actually improve patients' vision," said Sun Young Lee, MD, PhD, a retinal surgeon with Keck Medicine (search)Search term and principal investigator of the study site. "The findings could be groundbreaking because while there are a few treatments available that delay the progress of macular degeneration, there are none able to reverse the damage already done."

Promising Early Results Drive Phase 2b Trial

The current trial builds upon encouraging results from earlier research conducted by USC Roski Eye Institute (search)Search term specialists. The initial study involving a small patient population demonstrated that the implant was well-tolerated, remained securely positioned in the eye, and was successfully absorbed into retinal tissue. Notably, 27% of patients experienced some level of vision improvement.

"The earlier phase of the clinical trial showed the treatment to be safe with the potential to benefit patients' vision; this next phase will investigate whether the therapy can achieve clinically significant improvements in vision," explained Lee, who also serves as an associate professor of ophthalmology and physiology & neuroscience at the Keck School of Medicine of USC.

Addressing Critical Unmet Medical Need

Age-related macular degeneration (search)Search disease affects approximately 20 million Americans, with the dry form being the most widespread variant. The disease targets the macula, a small area at the center of the retina essential for sharp, detailed vision. In advanced cases, RPE cells that line the macula and play a critical role in maintaining healthy vision become damaged or destroyed, leading directly to visual decline and the development of geographic atrophy (search)Search disease.

The progressive nature of the disease causes faces, book pages, and objects directly in front of patients to become obscured by blurry, dark, or blind spots, significantly impacting quality of life.

Surgical Procedure and Mechanism of Action

During an outpatient surgical procedure, Keck Medicine (search)Search term eye surgeons will implant a thin layer of the lab-produced RPE cells directly into the retina. The bioengineered cells are designed to take over the function of damaged native cells and operate as normal RPE cells would.

"The study will explore if the lab-engineered implant will take over for the damaged cells, function as normal RPE cells would, and improve vision for patients who may currently have no other options for improvement," said Rodrigo Antonio Brant Fernandes, MD, PhD, an ophthalmologist with Keck Medicine (search)Search term and the study surgeon.

Trial Design and Patient Eligibility

The phase 2b trial is a masked study, meaning some enrolled participants will receive the actual implant while others undergo a simulated procedure. Keck Medicine (search)Search term serves as one of five locations nationwide participating in the clinical trial, with plans to enroll 24 patients total.

Eligible patients must be between ages 55-90 with advanced dry age-related macular degeneration (search)Search disease and a diagnosis of geographic atrophy (search)Search disease, indicating their RPE cells are damaged or not functioning properly. Participants will be monitored for at least one year to evaluate implant tolerance and track any changes in vision.

Industry Collaboration and Future Outlook

The bioengineered RPE retinal implant is produced by Regenerative Patch Technologies LLC (search)Search term, a clinical-stage company focused on developing stem cell-based implants for retinal diseases. Mark S. Humayun, MD, PhD, co-director of the USC Roski Eye Institute (search)Search term and co-inventor of the implant, is also a co-founder of the company.

"The USC Roski Eye Institute (search)Search term is dedicated to furthering innovative treatments to help improve lives by restoring vision," said Humayun, who also serves as director of the USC Ginsberg Institute for Biomedical Therapeutics and holds the Dennis and Michele Slivinski Chair in Macular Degeneration Research at the Keck School. "Stem cell-derived retinal implants may offer one of the greatest possibilities for helping patients with dry age-related macular degeneration (search)Search disease and one day, may offer a cure."

The technology used to manufacture the implant is exclusively licensed to Regenerative Patch Technologies by the University of Southern California, the California Institute of Technology, and the University of California Santa Barbara. Funding for the clinical trial comes in part from the California Institute for Regenerative Medicine (search)Search company, the Marcus Foundation (search)Search term, and USC.