StemCells’ novel neuroprotective approach for treating neurodegenerative diseases

StemCells, Inc. (NASDAQ:STEM) today announced the publication of preclinical data demonstrating for the first time that transplantation of its proprietary, purified human neural stem cells delays the loss of motor function in a mouse model of infantile neuronal ceroid lipofuscinosis (NCL). NCL, commonly referred to as Batten disease, is a fatal neurodegenerative disorder in children. This paper, “Neuroprotection of Host Cells by Human Central Nervous System Stem Cells in a Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis,” was published online today in the peer-reviewed journal Cell Stem Cell, and will be featured in the September 2009 print edition.

The study highlights StemCells’ novel neuroprotective approach to treating neurodegenerative diseases and the therapeutic potential of its neural stem cells. In this research, these cells were transplanted in a mouse model of infantile NCL and compared to a control (non-transplanted) group. The results demonstrate that the transplanted cells engraft, migrate throughout the brain and continuously secrete the missing lysosomal enzyme characteristic of NCL, which is needed to process cellular waste and keep neurons functioning and healthy. Compared with the control group, the mice that received the transplanted neural stem cells showed statistically significant reduction in cellular waste build-up, protection of critical host neurons and delayed loss of motor function.

“These exciting results suggest the prospect for improving the quality of life in patients suffering from NCL, and provide additional preclinical support for the development of our neural stem cells in this disease,” stated Stephen Huhn MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. “We are encouraged to continue our clinical development efforts with the hope of one day achieving a breakthrough in treating neurodegenerative diseases like NCL, which today have no cure.”

The study was conducted by researchers at StemCells, Inc., led by Nobuko Uchida, Ph.D., Vice President of the Company’s Stem Cell Biology Program, in collaboration with leading researchers in genetics, neurology and stem cell biology at King’s College London, the Stanford University School of Medicine and The Salk Institute for Biological Studies. The neural stem cells used in the study were supplied by StemCells, Inc.

StemCells is currently testing its neural stem cell product candidate, HuCNS-SC® cells, in clinical trials. The Company completed a Phase I trial in NCL in January 2009, the results of which demonstrated a favorable safety profile along with evidence of engraftment and long-term survival of the HuCNS-SC cells. StemCells has also received approval from the U.S. Food and Drug Administration (FDA) to initiate a Phase I trial of its HuCNS-SC cells to treat Pelizaeus-Merzbacher Disease (PMD), a fatal myelination disorder in the brain that primarily affects young children.


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