Stem cells have the potential to treat a broad range of human disorders. While approaches based on embryonic stem (ES) cells and induced pluripotent stem (iPS) cells have attracted the most media attention, they are complicated by specific challenges. The use of tissue-derived (non-embryonic or “adult”) stem cells in medical therapy offers an equally promising approach and avoids many of the challenges associated with ES or iPS cell therapies.

StemCells, Inc.’s lead product candidate, HuCNS-SC cells, is a highly purified composition of tissue-derived human neural stem cells that are expanded and stored as banks of cells. When transplanted into animal models, HuCNS-SC cells engraft, migrate, differentiate into neurons and glial cells, and survive long-term with no sign of tumor formation or adverse effects. Because of this demonstrated ability to engraft and survive long-term, a single transplantation of HuCNS-SC cells offers the chance for a durable clinical benefit.

StemCells is the only company authorized by the FDA to conduct clinical trials involving transplantation of neural stem cells into the brain. StemCells’ first trial, which was completed in January 2009, was a Phase 1 study in neuronal ceroid lipofuscinosis, a fatal neurodegenerative disorder in children. The trial data demonstrated a positive safety profile of the HuCNS-SC cells. StemCells continues to follow patients who completed the trial who are all 2 to 3 years post-transplant, and to date, no safety concerns have been identified. StemCells is now preparing to initiate a second NCL clinical trial, which will focus more specifically on measuring the effect of HuCNS-SC cells on disease progression.

StemCells is also conducting a Phase 1 trial in Pelizaeus-Merzbacher Disease, a fatal myelination disorder. The primary endpoint is safety,  but researchers will also look for evidence of new myelin formation. The outcome may reveal important information for the treatment of more common myelination disorders including multiple sclerosis and cerebral palsy.

StemCells believes that the data being accumulated for these initial indications will facilitate future clinical testing of HuCNS-SC cells in a broad range of other neurodegenerative disorders, as well as in spinal cord injury and retinal degenerative diseases, such as age-related macular degeneration and retinitis pigmentosa.

The company hopes to initiate clinical studies in spinal cord injury in 2011 and retinal diseases in 2012.