Source: Oregon Health & Science University
Date: June 8, 2009
Summary:
A groundbreaking clinical trial to test the safety and preliminary efficacy of StemCells, Inc.’s (NASDAQ: STEM) proprietary HuCNS-SC® product candidate in children with a rare neurodegenerative disease has yielded a favorable safety profile of the product and evidence of engraftment and long-term survival of the donor cells. The study results were presented at the 12th International Congress on Neuronal Ceroid Lipofuscinoses (NCL) held June 3 - 6, 2009, in Hamburg, Germany.
The Phase I trial of HuCNS-SC (purified human neural stem cells) conducted at OHSU Doernbecher Children's Hospital included six children with advanced stages of infantile and late-infantile neuronal ceroid lipofuscinosis (NCL), often referred to as Batten disease. The study participants were transplanted with HuCNS-SC cells and followed for 12 months.
Overall, the Phase I data demonstrated that high doses of HuCNS-SC cells transplanted directly into multiple sites within the brain followed by 12 months of immunosuppression were well tolerated by all six patients. The patients' medical, neurological and neuropsychological conditions following transplantation appeared consistent with the normal course of the disease.
