A groundbreaking clinical trial to test the safety and preliminary efficacy of StemCells, Inc.’s 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 OHSU Doernbecher research team worked very hard to carry out this highly complex research and is heartened to see that this approach appears to be safe. We are delighted that this first trial of human neural stem cells was successful and offers hope for effective treatment of NCL and other neurodegenerative disorders," said Robert Steiner, M.D., F.A.A.P., F.A.C.M.G., co-principal investigator, professor of pediatrics and molecular and medical genetics, and vice chairman for pediatric research at Oregon Health & Science University Doernbecher Children's Hospital.
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. Read more in the OHSU media release.