Physician-researchers at USC received a nearly $16 million grant from the California Institute for Regenerative Medicine to fund the development of a stem cell-based treatment for age-related macular degeneration, the leading cause of vision loss and blindness among the elderly.
Mark Humayun, professor of ophthalmology, cell and neurobiology, and biomedical engineering at the Keck School of Medicine of USC and the USC Viterbi School of Engineering, and David R. Hinton, the Gavin S. Herbert Professor of Retinal Research and professor of pathology and ophthalmology at the Keck School of Medicine, will lead the four-year study.
The California Institute and two international partners awarded more than $250 million to 14 multidisciplinary teams of researchers in California, the United Kingdom and Canada to develop stem cell-based therapies for 11 diseases. The Disease Team Research Awards mark the first institute funding explicitly expected to result in a filing with the U.S. Food and Drug Administration to begin a clinical trial.
The grants received formal approval on Oct. 29 from the Independent Citizens Oversight Committee, the 29-member governing board of the institute, and were announced at a press conference in Los Angeles.
USC faculty also will collaborate on grants awarded to other California institutions:
� Paula Cannon, associate professor of molecular microbiology and immunology at the Keck School of Medicine, is a co-investigator on a team that received $14 million to develop a novel therapy that may offer lifetime immunity to HIV infection
� Thomas Coates, professor of pediatrics and pathology at the Keck School of Medicine, is a co-investigator on a team that received $9 million to explore treating sickle cell disease using a gene therapy approach to modify patients’ blood-forming stem cell
� Michael Press, holder of the Harold E. Lee Chair in Cancer Research at the USC Norris Comprehensive Cancer Center and professor of pathology at the Keck School of Medicine, is the co-investigator on a nearly $20 million grant aimed at developing drugs that destroy cancer stem cells in solid tumors.
Age-related macular degeneration (AMD) is a progressive disease that causes distortion in central vision and eventually leads to blindness. It is estimated that by 2020, more than 450,000 Californians will suffer from vision loss or blindness due to degeneration. Effective treatment for the disease may be achieved by replacing damaged retinal pigment epithelium — the layer of cells at the back of the eye — and retinal cells with healthy ones derived from human embryonic stem cells, Humayun said.
“The funding will be tremendously helpful and will accelerate our research toward achieving a near-term stem cell-based therapy for AMD,” he said.
Humayun was elected to the Institute of Medicine for his groundbreaking work to restore sight to the blind. Election to the institute is considered one of the highest honors in the fields of health and medicine and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service.
Alan Trounson, president of the California Institute for Regenerative Medicine, said the pace of the disease team projects stands in contrast to the decade or more that’s usually required to reach clinical trials.
“Scientists have talked for years about the need to find ways to speed the pace of discovery,” Trounson said. “By encouraging applicants to form teams composed of the best researchers from around the world, we think CIRM will set a new standard for how translational research should be funded.”
For more information on USC’s stem cell programs, visit https://stemcell.usc.edu