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 in the peer-reviewed journal Cell Stem Cell, and will be featured in the September print edition, the company noted.
In a release, the Company noted that the study highlights its 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," said 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.
StemCells' 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. The Company's preclinical research has shown that HuCNS-SC cells can be directly transplanted; are able to engraft, migrate, and differentiate into neurons and glial cells; and possess the ability to survive for as long as one year with no sign of tumor formation or adverse effects. These findings show that HuCNS-SC cells, when transplanted, act like normal stem cells, suggesting the possibility of a continual replenishment of normal human neural cells.
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