ALS is a devastating disease marked by motor neuron degeneration, leading to extreme muscle weakness, paralysis and death. Previous research supports the transplant of glial (cells surrounding the motor neurons) as a possible treatment for ALS, and studies of precursor cells that develop into a type of glial cell called astrocytes have shown promise in animal studies. The researchers hope to develop an ALS therapy based on using human embryonic stem cells to create astrocyte precursor cells for transplant into patients, where the hope is the astrocytes would mature into new and healthy glial cells.

Webster Cavenee, PhD, director LICR and professor of medicine at UC San Diego School of Medicine, is co-principal investigator of a $19.2 million grant to UC San Francisco for a six-institution study of the effectiveness of using neural stem cell-based gene therapy to fight brain tumors. The disease team, led by principle investigator Mitchel Berger of UCSF, also includes researchers from UCLA, the Salk Research Institute, LICR, and the Burnham Institute.

Brain tumors are incurable, and current therapies are limited by the inability to reach widely spread tumor cells throughout the brain. But neural stem cells have an affinity for tumor cells, and the researchers are planning to attach payloads of "killer genes" to the stem cells, hoping that the stem cells will be drawn to the tumors, killing them. The team will test the effectiveness of human neural stem cells and two different types of genes in various mouse models of a type of deadly brain tumor, glioblastoma.

The Disease Team Research Awards include approximately $8 million from the Medical Research Council, UK, and $35 million from the Cancer Stem Cell Consortium, Canada, to fund the international portions of the collaborations.

CIRM President Alan Trounson 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. 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," he said.

Other diseases being targeted by the teams include HIV/AIDS, type1 diabetes, macular degeneration, heart attack and stroke, sickle cell anemia and skin disease.

The disease team grant to UC San Diego brings its total CIRM funding to nearly $65.6 million since the first funding was awarded in 2006.

Source: Moores UCSD Cancer Center