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StemCells, Inc. today provided an update on the Company's Phase I clinical trial of its proprietary HuCNS-SC(R) product candidate
PALO ALTO, CA, USA | January 18, 2008 | StemCells, Inc. (NASDAQ:STEM) today provided an update on the Company's Phase I clinical trial of its proprietary HuCNS-SC(R) product candidate (purified human neural stem cells). The trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC cells as a potential treatment for infantile and late infantile neuronal ceroid lipofuscinosis (NCL). NCL, which is often referred to as Batten disease, is a rare and fatal neurodegenerative condition afflicting infants and children. Enrollment and transplantation of all six patients planned for the trial has been completed.
"We are deeply saddened to learn that one of the patients in our trial has passed away," said Martin McGlynn, President and CEO of StemCells, Inc. "This is a stark and tragic reminder of the inevitable course of Batten disease and our deepest sympathies go out to the patient's family and friends."
The patient, a nine-year-old girl, was transplanted with HuCNS-SC cells in January 2007 and was due to return this month to the trial site, Oregon Health & Science University's (OHSU) Doernbecher Children's Hospital, for her 12 month follow-up. She was hospitalized nearly two weeks ago, suffering from an apparent viral infection, seizures and respiratory distress before succumbing earlier this week. Based upon the facts and information available at this time, it is the opinion of the principal investigators at OHSU, the independent Data Safety Monitoring Committee, and the Company's medical experts that this death was most likely due to the natural progression of the disease and not related to the HuCNS-SC cells or the transplantation procedure itself. StemCells, Inc. is continuing to investigate the death and has been in contact with the U.S. Food and Drug Administration (FDA) since first becoming aware of the patient's condition. The Company expects to provide additional updates about the trial in the future.
"Our research team was profoundly moved by the loss of one of our patients in the trial. Unfortunately, those of us involved in this trial have seen the devastating and deadly affects of NCL, a fatal degenerative disease, so her death was not unexpected. However, witnessing the death of a child is not something anyone can really be prepared for," said Robert D. Steiner, M.D., F.A.A.P., F.A.C.M.G., Professor of Pediatrics and Molecular and Medical Genetics, Vice Chair for Pediatric Research, Oregon Clinical and Translational Research Institute, Doernbecher Children's Hospital, OHSU School of Medicine and co-principal investigator of the trial. "She and her family as well as all of the other families in this trial were pioneers. All of those who have and will benefit from medical research owe courageous patients who participate in clinical trials a debt of gratitude."
"All of us at Doernbecher grieve for the loss of this precious child. She and her family have so generously given of themselves to take the first step toward what we all hope will someday be a cure for other children suffering from devastating diseases like this one," said Nathan Selden, M.D., Ph.D., F.A.C.S., F.A.A.P., Campagna Associate Professor of Pediatric Neurological Surgery and Head of the Division of Pediatric Neurological Surgery, Doernbecher Children's Hospital, OHSU School of Medicine and co-principal investigator.
About Neuronal Ceroid Lipofuscinosis (Batten Disease)
Neuronal ceroid lipofuscinosis is a fatal neurodegenerative disorder brought on by inherited genetic mutations. The disorder afflicts infants and young children, and the three most common forms of NCL--infantile, late infantile and juvenile onset--are often referred to as Batten disease. All forms have the same basic cause--lack of a lysosomal enzyme--and have similar progression and outcome. Children with NCL suffer seizures, progressive loss of motor skills, sight and mental capacity, eventually becoming blind, bedridden and unable to communicate.
Infantile or late infantile NCL is brought on by inherited mutations in the CLN1 gene, which codes for palmitoyl-protein thioesterase 1 (PPT1) or in the CLN2 gene, which codes for tripeptidyl peptidase I (TPP-I), respectively. The consequence of these gene mutations is either a defective or missing enzyme that leads to accumulation of lipofuscin-like fluorescent inclusions in various cell types. These non-degraded lysosomal inclusions accumulate to the point of interference with normal cellular function and ultimately lead to the pathological manifestations of the disease. One way to treat the disease is to provide the brain with a replacement source of functional enzyme that can be taken up by the enzyme-deficient cells.
About StemCells, Inc.
StemCells, Inc. is a clinical-stage biotechnology company focused on the discovery, development and commercialization of cell-based therapeutics to treat diseases of the nervous system, liver and pancreas. The Company's programs seek to repair or repopulate neural, liver or other tissue that has been damaged or lost as a result of disease or injury. StemCells has pioneered the discovery and development of HuCNS-SC(R) cells, its highly purified, expandable population of human neural stem cells. StemCells is currently conducting and has completed enrollment for a Phase I clinical trial of its proprietary HuCNS-SC product candidate as a treatment for neuronal ceroid lipofuscinosis (NCL). NCL, which is often referred to as Batten disease, is a rare and fatal neurodegenerative disease that affects infants and young children. StemCells owns or has exclusive rights to more than 53 issued or allowed U.S. patents and more than 150 granted or allowed non-U.S. patents. Further information about the Company is available on its web site at: www.stemcellsinc.com.
About OHSU and Doernbecher Children's Hospital
Oregon Health & Science University is the state's only health and research university and Oregon's only academic health center. OHSU is Portland's largest employer and the fourth largest employer in Oregon (excluding government), with more than 12,400 employees. OHSU provides many services and community support activities not found anywhere else in the state. It serves 189,000 patients annually and is a conduit for learning for more than 3,400 students and trainees. OHSU is the source of more than 200 community outreach programs that bring health and education services to every county in the state.
OHSU Doernbecher Children's Hospital is a world-class academic health center that each year cares for more than 60,000 patients from across the United States. In the most patient- and family-centered environment, children receive outstanding cancer treatment, specialized neurology care, highly sophisticated heart surgery, and care in many other pediatric specialties. In addition to several locations in the Portland metropolitan area, Doernbecher's pediatric experts travel throughout Oregon and southwest Washington providing pediatric specialty care at 13 outreach clinics.
Apart from statements of historical facts, the text of this press release constitutes forward-looking statements regarding, among other things, the Company's conduct of and enrollment in the clinical trial of its HuCNS-SC product candidate as a treatment for NCL. These forward-looking statements speak only as of the date of this news release. StemCells 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 the forward-looking statements due to risks and uncertainties to which the Company is subject, including uncertainty as to whether HuCNS-SC cells will prove safe in the current clinical trial; uncertainty as to whether the FDA or other applicable regulators or review boards will permit the Company to continue clinical testing in NCL despite the novel and unproven nature of the Company's technology; uncertainty whether results obtained in the animal models and in vitro studies of infantile NCL or other diseases and conditions will be able to be successfully translated into treatment for humans; uncertainties regarding the Company's ability to obtain the increased capital resources needed to continue such clinical trial and its other current research and development operations and to conduct the further research, preclinical development and clinical trials necessary for regulatory approvals; and other factors that are described under the heading "Risk Factors" in Item 1A of the Company's Quarterly Report on Form 10-Q. The clinical trial is not designed to establish efficacy and completion of the clinical trial by any patient in the clinical trial is not an indication of the efficacy of the Company's HuCNS-SC product; given the fragility of this patient population not all patients are expected to complete the clinical trial.
SOURCE: StemCells, Inc.
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