StemCells, Inc. Demonstrates Protection of Neurological Function in Batten Mouse.Preclinical Proof-of-Concept Data for the Company's Neural Stem Cells stem cells, unspecialized human or animal cells that can produce mature specialized body cells and at the same time replicate themselves. Embryonic stem cells are derived from a blastocyst (the blastula typical of placental mammals; see embryo), which is very young Published in Cell Stem Cell stem cell
In living organisms, an undifferentiated cell that can produce other cells that eventually make up specialized tissues and organs. There are two major types of stem cells, embryonic and adult.
PALO ALTO, Calif. -- StemCells, Inc. (NASDAQ NASDAQ
in full National Association of Securities Dealers Automated Quotations
U.S. market for over-the-counter securities. Established in 1971 by the National Association of Securities Dealers (NASD), NASDAQ is an automated quotation system that reports on :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 Neuronal Ceroid Lipofuscinoses (NCL, also known as Batten Disease) is the general name for a family of at least eight genetically separate neurodegenerative disorders that result from excessive accumlation of lipopigments (lipofuscin) in the body's tissues. (NCL NCL Norwegian Cruise Line
NCL New Caledonia (ISO Country code)
NCL National Consumers League (Washington, DC)
NCL Neuronal Ceroid Lipofuscinosis (adult type) ). NCL, commonly referred to as Batten disease Batten disease
see ceroid lipofuscinosis. , is a fatal neurodegenerative disorder neurodegenerative disorder Neurology A chronic progressive neuropathy characterized by selective and generally symmetrical loss of neurons in motor, sensory, or cognitive systems Types by area Cerebral cortex–Alzheimer's disease, Pick's disease, Lewy body 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 en·graft
tr.v. en·graft·ed, en·graft·ing, en·grafts
1. To graft (a scion) onto or into another plant.
2. To plant firmly; establish. , migrate throughout the brain and continuously secrete the missing lysosomal lysosomal
pertaining to or emanating from lysosomes.
enzymes located in the lysosomes.
lysosomal phospholipidosis 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 FACS Fellow of the American College of Surgeons.
Fellow of the American College of Surgeons
fluorescence-activated cell sorter. , FAAP FAAP Fundação Armando Álvares Penteado (University from São Paulo - Brazil)
FAAP Fellow of the American Academy of Pediatrics
FAAP Framework for African Agricultural Productivity
FAAP Food Allergy Action Plan
FAAP Federal-Aid Airport Program , Vice President and Head of the CNS See Continuous net settlement.
See continuous net settlement (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 Stanford University School of Medicine is affiliated with Stanford University and is located at Stanford University Medical Center in Stanford, California, adjacent to Palo Alto and Menlo Park. and The Salk Institute for Biological Studies The Salk Institute for Biological Studies is an independent, non-profit, scientific research laboratory located in La Jolla, California. It was founded in 1960 by Jonas Salk, M.D., the developer of the polio vaccine. . 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([R]) 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 engraftment /en·graft·ment/ (en-graft´ment) incorporation of grafted tissue into the body of the host.
The process of transplanted stem cells reproducing new cells. and long-term survival of the HuCNS-SC cells. StemCells has also received approval from the U.S. Food and Drug Administration (FDA FDA
Food and Drug Administration
n.pr See Food and Drug Administration.
n.pr the abbreviation for the Food and Drug Administration. ) to initiate a Phase I trial of its HuCNS-SC cells to treat Pelizaeus-Merzbacher Disease (PMD (Polarization Mode Dispersion) The type of dispersion that occurs in singlemode fiber due to a lack of perfect symmetry in the fiber and from external pressures on the cable. Light travels over singlemode fiber in two polarization states. ), a fatal myelination myelination /my·elin·a·tion/ (mi?e-lin-a´shun) myelinization.
my·e·li·na·tion or my·e·li·ni·za·tion
The acquisition, development, or formation of a myelin sheath around a nerve fiber. disorder in the brain that primarily affects young children.
About Neuronal Ceroid Lipofuscinosis (Batten Disease)
Neuronal ceroid lipofuscinosis (NCL) is a fatal neurodegenerative disorder that afflicts infants and young children. The disorder, often referred to as Batten disease, is caused by genetic mutations, and children who inherit the defective gene are unable to produce enough of an enzyme that processes cellular waste substances that accumulate in a part of cells known as the lysosome lysosome
Membrane-enclosed organelle found in all eukaryotic cells (see eukaryote) that is responsible for the cell's digestion of macromolecules, old cell parts, and microorganisms. . Without the enzyme, the cellular waste builds up, and eventually the cells cannot function and die. Children with NCL appear healthy when born, but as their brain cells die, they begin to suffer seizures and progressively lose motor skills, sight and mental capacity. Eventually, they become blind, bedridden bed·rid·den or bed·rid
Confined to bed because of illness or infirmity. and unable to communicate or function independently. There currently is no cure for the disease. The infantile and late infantile forms of NCL are caused by different genetic mutations. As the names imply, the two forms begin to afflict patients at different stages of infancy, but both have similar disease progression and outcomes.
About HuCNS-SC([R]) Cells
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.
About StemCells, Inc.
StemCells, Inc. is focused on the development and commercialization of cell-based technologies. In its therapeutic product development programs, StemCells is targeting diseases of the central nervous system and liver. StemCells' lead product candidate, HuCNS-SC([R]) cells (purified human neural stem cells), is in clinical development for the treatment of two fatal neurodegenerative disorders that primarily affect young children. StemCells also markets specialty cell culture media products under the brand SC Proven([R]), and is developing its cell-based technologies for use in drug discovery and development. The Company has exclusive rights to approximately 55 issued or allowed U.S. patents and approximately 200 granted or allowed non-U.S. patents. Further information about StemCells is available on its web site at www.stemcellsinc.com.
Apart from statements of historical fact, the text of this press release constitutes forward-looking statements regarding, among other things, the therapeutic potential of the Company's human neural stem cells and the prospects for future commercialization of a product to treat neurodegenerative diseases. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including: uncertainties with respect to the fact that additional trials will be required to confirm the safety and demonstrate the efficacy of the Company's HuCNS-SC cells for the treatment of NCL or any other disease; risks whether the FDA or other applicable regulatory agencies will permit the Company to continue clinical testing in NCL, PMD or in future clinical trials of proposed therapies for other diseases or conditions despite the novel and unproven nature of the Company's technologies; uncertainties about the design of future clinical trials and whether the Company will receive the necessary support of a clinical trial site and its institutional review board to pursue future clinical trials in NCL, PMD or in proposed therapies for other diseases or conditions; uncertainties regarding the Company's ability to obtain the increased capital resources needed to continue its current and planned research and development operations, including such operations of the Company for non-therapeutic applications, and to conduct the research, preclinical development and clinical trials necessary for regulatory approvals; uncertainty as to whether HuCNS-SC cells and any products that may be generated in the future in the Company's cell-based programs will prove safe and clinically effective and not cause tumors or other adverse side effects; uncertainties regarding whether results in preclinical research in animals will be indicative of future clinical results in humans; uncertainties regarding the Company's manufacturing capabilities given its increasing preclinical and clinical commitments; and other factors that are described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2008 and in its subsequent reports on Form 10-Q and Form 8-K.