UC Irvine neuroscientists Frank LaFerla and Mathew Blurton-Jones today were awarded $3.6 million toward the development of an Alzheimer’s disease therapy involving human neural stem cells.

LaFerla, director of the UCI Institute for Brain Aging and Dementia, and Blurton-Jones will use the California Institute for Regenerative Medicine grant to test in mice whether human stem cells can reverse dementia caused by Alzheimer’s. The project builds on their previous work showing that mouse neural stem cells can restore memory in mice with brain damage.

UCI has been awarded about $59.8 million in grants from CIRM, ranking it fourth statewide for total CIRM funding and further establishing it as a hub for stem cell research in Southern California.

In all today, CIRM’s governing board approved $67.7 million in Early Translational awards for 15 projects at California institutions. The awards are intended to move promising basic stem cell research toward the clinic.

“We’re very excited about this research, which we hope will lay the groundwork for a stem cell-based therapy for Alzheimer’s disease,” LaFerla said. “It’s absolutely critical to discover whether stem cells can help people with Alzheimer’s. Though several promising drugs are in clinical trials, there is no cure for the disease.”

Alzheimer’s is the leading cause of dementia among the elderly. In the U.S., 5.3 million people have the disease, more than 500,000 of whom live in California. Every 71 seconds, an American develops Alzheimer’s.

LaFerla and Blurton-Jones will partner with Peter Donovan of UCI, along with scientists at other institutions throughout California and in Australia, on the project.

Recent experiments by Blurton-Jones and LaFerla showed that mouse neural stem cells partially reverse cognitive impairment in mice genetically altered to have key features of Alzheimer’s disease. The team believes the cells do this by secreting proteins called neurotrophins that stimulate brain connectivity and protect vulnerable cells from injury. The therapy does not reduce levels of proteins leading to the two signature Alzheimer’s lesions, plaques and tangles.

It will be trickier to test human neural stem cells in Alzheimer’s mice, LaFerla said. First, scientists must figure out how to suppress the mouse immune system so the cells are not rejected. Then they must select the human cells they believe will work best in the mice.

“The ultimate goal is to discover a therapy suitable for human clinical trials,” LaFerla said.

A CIRM working-group review of the project said the UCI-led team “is world-class and exemplary of CIRM’s goal to bring together the best and brightest in stem cell research.” It said reviewers “were enthusiastic about this proposal and felt that its impact could be profound.”

UCI’s stem cell scientists are pioneers in regeneration, large-scale production of specialized cells with very high purity, and methods for treating damaged tissue.

The university recently broke ground on a four-story building dedicated to stem cell research. When finished in 2010, it will house the Sue and Bill Gross Stem Cell Research Center, dozens of laboratory-based and clinical researchers, a stem cell training course, a master’s program in biotechnology with an emphasis on stem cell research, and programs and activities for patients and public education.

LaFerla also is seeking support and funds to construct a building for the Institute for Brain Aging and Dementia that would allow it to expand its clinical offerings.

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Jennifer Fitzenberger
949-824-3969
jfitzen@uci.edu