StemCells' Milestone Clinical Trial in Pelizaeus-Merzbacher Disease Shows Evidence of Myelination Following Human Neural Stem Cell Transplantation

StemCells, Inc. STEM today announced preliminary evidence of progressive and durable donor-cell derived myelination in all four patients who underwent transplantation with the Company's proprietary HuCNS-SC® cells (purified human neural stem cells) in its clinical trial for Pelizaeus-Merzbacher disease, a rare hypo-myelination disorder in children. In addition, clinical assessment revealed small but measureable gains in motor and/or cognitive function in three of the four patients; the fourth patient remained clinically stable. The study was conducted by researchers at the University of California, San Francisco. A summary of the trial results were presented Saturday, March 31, at the 2012 European Leukodystrophy Association (ELA) Families/Scientists Meeting in Paris. The findings are being submitted for publication in a peer-reviewed scientific journal. "The results from this Phase I study are meaningful and important," said study investigator Nalin Gupta, MD, PhD, UCSF associate professor of neurological surgery and pediatrics and chief of pediatric neurological surgery at UCSF Benioff Children's Hospital. "The safety and clinical outcomes a year after transplantation in this Phase I study, combined with durable radiological signals of myelin formation, provide objective evidence of a biological effect of HuCNS-SC transplantation that addresses the fundamental basis of the pathology in the brain of PMD patients. We also wish to recognize the families' contribution to this study. These advances would not be possible without their willingness to participate in this clinical research."