Clinical trial to study loss of vision

Research on age-related macular degeneration conducted by Mark Humayun, left, and David Hinton has been funded by the California Institute for Regenerative Medicine.
Research on age-related macular degeneration conducted by Mark Humayun, left, and David Hinton has been funded by the California Institute for Regenerative Medicine.

Keck Medicine of USC research to slow vision loss for macular degeneration patients has been funded as part of the third round of the California Institute for Regenerative Medicine’s (CIRM) Disease Team awards.

The nearly $19 million study is led by principal investigators Mark Humayun, professor of ophthalmology and biomedical engineering and cell and neurobiology at the Keck School of Medicine of USC and David Hinton, professor of pathology, neurological surgery and ophthalmology at the 
Keck School. Both Humayun and Hinton are principal investigators with USC Stem Cell.

The study is one of six projects approved by the program. Funding from CIRM for all six projects totals $61 million.

“We believe this research will help us restore native photoreceptors so we can slow vision loss and even restore eyesight in people who suffer from advanced dry age-related macular degeneration [AMD],” Humayun said. “CIRM has previously funded the early stages of this research, and with the much-needed funding they are providing through this Disease Team grant, we will take our research to the clinical trial stage.”

The team proposes to use embryonic stem cells to produce the support cells, or retina pigment epithelium (RPE) cells, needed to replace cells lost in AMD. Research has shown that the loss of RPE cells, located in a thin sheet at the back of the eye, leads to AMD. To restore the RPE cells, the USC team proposes growing thin sheets of stem cell-derived cells to be surgically implanted into the eye, replacing diseased sheets and restoring the photoreceptors, the light sensitive cells of the retina.

“This research could be a game-changer in AMD,” said Keck School Dean Carmen A. Puliafito, an ophthalmologist specializing in retinal diseases. “Estimates indicate that by 2020, more than 450,000 people in California alone will suffer vision loss or blindness because of this disease, and the innovative approach by doctors Humayun and Hinton shows distinct promise.”

With the CIRM funding, the team will proceed to test the surgically implanted cell sheets in a Phase I clinical trial.

The USC team of researchers includes scientists from the University of California at Santa Barbara and City of Hope.

AMD is a progressive disease affecting part of the retina, called the macula, which enables people to read, see faces and drive. Victims initially experience distorted vision at the center of their vision, eventually progressing to legal blindness. AMD is the leading cause of vision loss and blindness among the elderly, and it is estimated that more than 1.75 million people in the United States suffer from AMD.

The CIRM Disease Team awards are designed to encourage multidisciplinary teams of researchers from academic institutions, medical centers and industry to work together and to develop new treatments for a broad range of therapies. Recipients were selected from 14 applications, all of which were reviewed by an independent group of internationally renowned scientists.

CIRM approved awards to teams from the University of California, San Diego, Stanford University, the University of California, Los Angeles and the University of California, Davis, covering diseases including leukemia and other solid tumor cancers, such as breast and prostate, that have not responded to conventional treatment. Other diseases targeted include sickle cell disease.

“The goal of the Disease Team award is to help accelerate the development of new therapies,” said Alan Trounson, president of CIRM.

“I think this is the sharp end of the CIRM program — we need to get therapies into clinical trials. The scientists are working together as teams to demonstrate the safety and efficacy of their products that have evolved from discoveries in the laboratory,” he added. “What’s impressive about this series of awards is that five of the six successful applications are for the continuation of work we had previously funded. It’s a reflection of the importance of continuity of funding, enabling scientists to keep their teams together and move their work forward as quickly as possible.”

The funding was approved by the stem cell agency’s governing board, the Independent Citizen’s Oversight Committee, at a two-day meeting in Los Angeles.

Mentioned in this article: Mark Humayun, MD, PhD