By Leslie Ridgeway
New research to slow vision loss for macular degeneration patients has been funded at Keck Medicine of USC as part of the third round of the California Institute of Regenerative Medicine’s (CIRM) Disease Team awards.
The nearly $19 million study is led by principal investigators Mark Humayun, MD, PhD, professor of ophthalmology and biomedical engineering and professor of cell and neurobiology at the Keck School of Medicine of USC, and David Hinton, MD, professor of pathology, neurological surgery and ophthalmology at the Keck School. The study is one of six projects approved by the program. Funding from CIRM for all six projects totals $61 million.
Humayun said, “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. CIRM has previously funded the early stages of this research, and with the much-needed funding they provide 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 age-related macular degeneration (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, MD, MBA, 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 Drs. 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 also includes scientists from University of California at Santa Barbara and City of Hope.
“The goal of the Disease Team award is to help accelerate the development of new therapies,” said Alan Trounson, PhD, 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.”
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.