USC to develop stem cell-based treatment for age-related macular degeneration with CIRM grant

The award will speed research of stem cell-based treatment for age-related macular degeneration

Physician-researchers at the University of Southern California (USC) received a nearly $16 million grant from the California Institute of Regenerative Medicine (CIRM) 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, M.D., Ph.D., 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, M.D., 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.

CIRM and two international partners awarded more than $250 million to 14 multidisciplinary teams of researchers in California, the UK and Canada to develop stem cell-based therapies for 11 diseases. The Disease Team Research Awards mark the first CIRM funding explicitly expected to result in a filing with the FDA to begin a clinical trial.

The grants received formal approval today from the Independent Citizens Oversight Committee (ICOC), the 29-member governing board of the institute, and were announced at a press conference held in Los Angeles.

USC faculty will also collaborate on grants awarded to other California institutions:

• Paula Cannon, Ph.D., 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, M.D., 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, M.D., Ph.D., 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 AMD. 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 from CIRM will be tremendously helpful and will accelerate our research towards achieving a near-term stem cell based therapy for AMD," he said.

Humayun was elected this month to the prestigious 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.