Fairly major research presentation on Friday:
PALO ALTO, Calif., April 11 /PRNewswire-FirstCall/ -- StemCells, Inc. (Nasdaq: STEM) will present preclinical data from a transplant study using the Company's proprietary human neural stem cells (CNS-SC) in Neuronal Ceroid Lipofuscinosis, a lysosomal storage disease commonly referred to as Batten Disease or NCL. Many lysosomal storage diseases, which include Tay-Sachs, Hunter, Krabbe, and Niemann-Pick diseases among others, are genetic disorders primarily affecting the central nervous system, in which enzyme deficiency causes the buildup of toxic waste materials. This results in the eventual destruction of nerve cells leading to the symptomatology of the disease, seizures, blindness, loss of motor skills and premature death.
Nobuko Uchida, Ph.D., Director of the Company's Neural Program will present the data today at the 9th International Congress on NCL in Chicago showing widespread engraftment in mice designed to model Batten Disease, and persistent production of the enzyme that is deficient in this disease. Significantly, the study showed a measurable reduction in the toxic waste material as well as preliminary indications that neuronal loss is also retarded. The study was done at StemCells, Inc. and at the laboratory of Dr. William Mobley at Stanford University.
"'Batten disease' refers to several closely related genetic disorders caused by deficiency of an enzyme required for normal cell metabolism. This results in an abnormal buildup of waste substances -- cellular debris, so to speak -- inside the cells of the brain, eye and other tissues, that eventually leads to cell death. Batten disease primarily affects infants and young children," explained Dr. Stan Tamaki, Director of Monoclonal Antibody Discovery and co-director of the program. The transgenic mice used in the study reported today were engineered to delete the gene involved in the infantile form of Batten disease, so that the toxic substances build up in their brain, as they do in human patients. StemCells' purified banked human CNS-SC were transplanted into the mice, migrated throughout their brains, and produced cells that made the missing enzyme.
"We observed a statistically significant decrease in the amount of stored toxic waste material in the transplanted mice," said Dr. Ann Tsukamoto, Vice President of Research and Development," and preliminary data also suggest that there is a larger number of surviving neurons in the brains of the transplanted transgenic mice compared to their non-transplanted littermates. We are delighted with these results. The improved neuronal survival needs to be confirmed with additional experiments, but these results give us hope that transplantation of our human CNS-SC may slow progression of or cure the disease. This has been a long time in coming. The mice had to be bred, tested for the mutation, and transplanted with the human cells, and then we had to wait an adequate time to insure long-term stability of the transplant. But it has been well worth the wait. Though good science in animal models takes a long time, it is the only way to insure true proof of concept for cell based therapies. Batten Disease is rare, but it is a debilitating condition that is fatal. There is no treatment currently available except palliative treatment to control some of the symptoms in the short run. It is our hope that our results will not only prove useful in Batten's, but can be extended to other lysosomal storage diseases that affect the central nervous system."
"There is a very large unmet need for new treatments for neurological disorders. StemCells, Inc. is successfully carrying out the proof of concept studies needed to develop treatments for the lysosomal storage disorders, diseases that disable and kill adults and children and for which current treatments are ineffective. There is genuine promise in the use of stem cells to treat and even cure these disorders," said Dr. William Mobley, Chairman Department of Neurology, Stanford University.
Phil Milto, President of the Nathan's Battle Foundation for Late Infantile NCL Therapy Development, stated "StemCells Inc.'s recent data is the most promising cell mediated data I have seen. These results are extremely encouraging for Batten's disease and potentially for all neurodegenerative disorders. The wide spread engraftment, large area of cell migration, and duration of enzyme production from their cells strongly demonstrates the current viability for the development of therapies for neurodegenerative disorders using StemCells' technology. This is the type of data that therapy development groups like ours have dreamed of seeing!"
StemCells continues its work using its proprietary human CNS-SC in other areas of disease and disability. The Company previously announced the promising results of a pilot study evaluating the use of these cells in a preclinical stroke model, which were presented at the Society for Neuroscience 32nd Annual Meeting in Orlando, Florida on November 6; that study was performed at the Departments of Neurosurgery and Neurology at Stanford University under the direction of Gary Steinberg, M.D., Ph.D., Chairman Department of Neurosurgery; Co-Director, Stanford Stroke Center. Preclinical studies using the human CNS-SC in spinal cord injury is also ongoing.
StemCells, Inc is a biotechnology company focused on the discovery, development and commercialization of stem cell-based therapies to treat diseases of the nervous system, liver, and pancreas. The Company's stem cell programs seek to repair or repopulate neural or other tissue that has been damaged or lost as a result of disease or injury. Further information about the Company is available on its web site, at www.stemcellsinc.com .
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