USC researchers receive $6 million CIRM grant to advance novel dry AMD therapy research

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The California Institute for Regenerative Medicine (CIRM), the state’s stem cell agency, has awarded a 2-year, $6 million grant to researchers at the USC Dr. Allen and Charlotte Ginsburg Institute for Biomedical Therapeutics and the USC Roski Eye Institute.

The funding will support preclinical studies necessary to advance a novel treatment for dry age-related macular degeneration (AMD), a leading cause of blindness in older adults.

The investigators aim to accelerate progress against dry AMD, which affects approximately 16 million people in the United States. The disease is caused by damage to the retinal pigment epithelium (RPE), the cells that support the photoreceptors in the retina. These RPE cells play a critical role in protecting, nourishing, and regenerating the rods and cones that convert light into signals for the brain. Dry AMD, typically diagnosed in individuals aged 50 and older, is currently incurable and can lead to legal blindness.¹

Existing treatment options, including vitamin supplements and immune-modulating therapies, are limited to slowing disease progression. However, the USC team’s approach, supported by CIRM, involves injecting a mixture of restorative, anti-inflammatory, and antioxidant compounds secreted by stem cell-derived RPE cells.

“Nothing that’s currently out there halts or reverses the disease, and many patients end up progressing to blindness,” said Mark Humayun, MD, PhD, University Professor of Ophthalmology and Biomedical Engineering and holder of the Cornelius J. Pings Chair in Biomedical Sciences at the Keck School of Medicine of USC. “There’s a need for a new approach. If we can develop this injection and stop disease progression or actually improve vision, that would be amazing from a patient perspective.”

Building on previous success

The therapeutic potential of RPE secretions stems from prior research by Humayun and colleagues on an implantable patch of RPE cells grown from stem cells. This earlier therapy, which is entering mid-stage clinical trials and is being commercialized by Regenerative Patch Technologies, showed that the patch’s secreted fluid (the secretome) benefited retinal cells beyond its immediate area.

CIRM has supported the progression of this work since 2010, funding the RPE patch from preclinical development through clinical trials. The latest grant builds on this success, enabling the team to explore the therapeutic potential of the secretome and advance it toward early-stage human trials.¹

“CIRM has been incredibly important to this work,” said Humayun, who also is co-director of the USC Roski Eye Institute and director of the USC Ginsburg Institute for Biomedical Therapeutics. “All of this was made possible by CIRM support. California is really leading the way in stem cell research for a host of health challenges thanks to California public investment.”

Preparing for clinical trials

The team has already taken steps to fast-track the new therapy into clinical trials. They have held preliminary discussions with the FDA and identified certified partner facilities for manufacturing and testing under FDA-regulated good manufacturing practices.

The new therapy is designed for earlier stages of dry AMD, potentially complementing the RPE patch, which targets advanced disease. “Essentially, we get two shots on goal from the same product,” Humayun said. “If we’re successful, we can cover patients from A to Z in the continuum of their disease.”

Cross-disciplinary collaboration

Bioengineers from the USC Viterbi School of Engineering will play a critical role in optimizing the patch, and Humayun plans to collaborate with USC Viterbi artificial intelligence experts to enhance the secretome’s therapeutic potential.

“There are so many combinations and permutations for the genetic knobs and levers we can use, it would take forever if you just tried them sequentially,” he explained. “AI will help us better understand how to refine and improve this therapy. And as a ‘school of schools,’ USC provides a unique culture and environment for these types of interactions between disparate fields.”

A visionary legacy

Humayun, a recipient of the National Medal of Technology and Innovation, is renowned for his groundbreaking contributions to vision restoration, including the Argus II retinal prosthesis, also known as the "bionic eye." Since its FDA approval in 2013, the device has restored limited vision to hundreds of previously blind individuals.

The work is deeply personal for Humayun, whose grandmother, a central figure in his upbringing, ultimately lost her sight.

“There’s no other feeling quite like when you are able to help restore sight to somebody who is blind,” he said. “Professionally, you can’t ask for anything more. And on a personal note, it is deeply impactful for me given everything my grandmother meant to me.”

Reference:

1. sheilaro. $6 million grant drives potential treatment for common cause of vision loss toward the clinic. Newsroom. Published November 20, 2024. Accessed November 21, 2024. https://keck.usc.edu/news/6-million-grant-drives-potential-treatment-for-common-cause-of-vision-loss-toward-the-clinic/