StemCells has launched its Alzheimer's disease program, supported by the California Institute for Regenerative Medicine (CIRM). The goal is to file, within four years, an IND application with the FDA to evaluate the company's proprietary HuCNS-SC product candidate (purified human neural stem cells) as a potential therapeutic in the treatment of Alzheimer's disease.

CIRM has agreed to provide approximately $19.3 million to the company in the form of a forgivable loan to help fund preclinical development and IND-enabling activities, and StemCells recently received an initial disbursement of $3.8 million. The funding was awarded under CIRM's Disease Team Therapy Development Award program (RFA 10-05) in September 2012. 

"With CIRM's support, we have taken the first steps toward the development of a novel cell-based therapeutic for use in the treatment of Alzheimer's disease," said Eliseo Salinas, M.D., executive vice president and head of R&D at StemCells. "There are no good treatment options for Alzheimer's disease; in particular, there are no approved drugs which alter the progression of the disease. Furthermore, in the past few years, several drugs have failed in late-stage clinical trials. These drugs, like most of the treatments currently in development, target a single modality in a complex disease believed to result from a biological cascade probably triggered by multiple genetic and environmental factors. Cell-based therapies have the potential to provide a therapeutic benefit by acting on several relevant biological targets under the regulation of the host."

"We know from the preclinical work that our proprietary HuCNS-SC cells survive in the toxic environment of the Alzheimer's disease brain and restore memory under the regulation of the host,” said Salinas. “So rather than targeting a single mechanism in this cascade, our strategy is to provide healthy, self-renewing cells that can halt or slow disease progression and therefore preserve or restore cognitive function. Even a modest slowing of disease progression could translate into substantial improvements in quality of life for patients and families, as well as significant economic savings for society."   

StemCells will evaluate its HuCNS-SC cells as a potential therapeutic in Alzheimer's disease in collaboration with researchers at the University of California, Irvine (UCI), led by Frank LaFerla, Ph.D., and Matthew Blurton-Jones, Ph.D.