StemCells, Inc. is sponsoring a Phase I clinical trial of its proprietary human neural stem cells (HuCNS-SC(tm)) at Oregon Health & Science University's (OHSU) Doernbecher Children's Hospital. This Phase I trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC as a treatment for infantile and late infantile Neuronal Ceroid Liposfuscinosis (NCL). NCL, often referred to as Batten disease, is a rare and fatal neurodegenerative disorder that afflicts infants and young children and is caused by a deficiency of a lysosomal enzyme. StemCells, Inc. believes this is the first FDA-approved clinical trial in which a purified composition of human neural stem cells will be used as the potential therapeutic agent.

Six patients will be enrolled in this open-label study, which will test two doses of HuCNS-SC. Children eligible for the study are those who have clinical symptoms of infantile or late infantile NCL, and loss-of-function mutations in either the CLN1 or CLN2 gene, respectively. Potential patients will be tested for eligibility and evaluated for baseline disease status prior to receiving HuCNS-SC. The primary objective of the trial is to evaluate the safety of HuCNS-SC. Trial participants will be monitored with standardized measures of development, cognition, behavior and language for one year following transplantation.

StemCells has demonstrated that HuCNS-SC produce the lysosomal enzymes that are missing in both infantile and late infantile NCL. Proof-of-concept studies in animal models have shown that transplanting HuCNS-SC leads to an increase in the amount of lysosomal enzyme in the brain, protection of neurons within the brain, and extended survival when compared to a non-transplanted control group. HuCNS-SC are isolated from normal fetal brain tissue, purified, expanded and then stored in frozen cell banks until they are transplanted. The cells are not genetically modified.

Robert Steiner, M.D., F.A.A.P., F.A.C.M.G., vice chairman for pediatric research, Doernbecher Children's Hospital; and professor of pediatrics, and molecular and medical genetics, Oregon Health & Science University School of Medicine, is principal investigator for the study. Nathan Selden, M.D., Ph.D., F.A.C.S., F.A.A.P., Head of the Division of Pediatric Neurological Surgery, Doernbecher Children's Hospital, and Campagna Associate Professor of Pediatric Neurological Surgery, OHSU School of Medicine, is co-principal investigator and will perform the surgical implantation procedures. Thomas K. Koch, M.D., F.A.A.P., F.A.A.N., director of pediatric neurology, professor of pediatrics and neurology, Doernbecher Children’s Hospital, Oregon Health & Science University School of Medicine, is co-investigator.

Stephen L. Huhn, M.D., F.A.C.S., F.A.A.P., chief of pediatric neurosurgery, and Gregory M. Enns, M.B., Ch.B., assistant professor and director, Biochemical Genetics Program, Division of Medical Genetics, both of Stanford Medical School, played a major role in the pre-clinical research and design of the protocol for this clinical trial.

Drs. Steiner, Selden and  Koch are representatives of a large team of health care professionals working on this research

Oregon Health & Science University
Oregon Health & Science University is Oregon's only health and research university and its only academic health center. As Portland's largest employer and the fourth largest employer in Oregon (excluding government), OHSU's size contributes to its ability to provide many services and community support activities not found anywhere else in the state. It serves more than 184,000 patients, and is a conduit for learning for more than 3,900 students and trainees. OHSU is the source of more than 200 community outreach programs that bring health and education services to each county in the state.  

As a leader in research, OHSU earns $274 million annually in research funding. The institution serves as a catalyst for the region's bioscience industry and is an incubator of discovery, averaging one new breakthrough or innovation every four days. OHSU disclosed 101 inventions in 2005 alone and has helped start 57 new spinoff companies, most of which are based in Oregon.

Doernbecher Children's Hospital
Doernbecher Children's Hospital, an integral part of Oregon Health & Science University, is a world-class academic health center that each year cares for more than 56,000 patients from across the United States. In the most patient- and family-centered environment, children from all corners of the country receive outstanding cancer treatment, specialized neurology care, highly sophisticated heart surgery, and care in many other pediatric specialties. In addition to multiple locations in the Portland metropolitan area, Doernbecher's pediatric experts travel around Oregon and southwest Washington providing pediatric specialty care through 13 outreach clinics.

StemCells, Inc.
StemCells, Inc. is a clinical stage 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 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 is the first company to directly identify and isolate human neural stem cells from normal brain tissue. These stem cells are expandable into cell banks for therapeutic use, which offers the potential of using normal, non-genetically modified cells as cell-based therapies.

StemCells, Inc. (NASDAQ: STEM) is a publicly traded company solely focused on stem cell research and development and has approximately 40 U.S. and 100 non-U.S. patents.

Robert Steiner, M.D.,
Principal Investigator
Robert Steiner, M.D., F.A.A.P., F.A.C.M.G., is vice chairman for pediatric research, Doernbecher Children's Hospital. He also is professor of pediatrics, and molecular and medical genetics, Oregon Health & Science University School of Medicine.

Steiner earned his medical degree at the University of Wisconsin Medical School. He completed his residency in pediatrics at the Children's Hospital Medical Center, University of Cincinnati, and then completed a fellowship in medical genetics at Seattle's Children's Hospital and Regional Medical Center, University of Washington.

Steiner specializes in the treatment of inborn errors of metabolism and osteogenesis imperfecta, a genetic disorder characterized by bones that break easily. He is involved in clinical and translational research supported by the National Institutes of Health.

Among his areas of expertise is Smith Lemli Opitz syndrome. The syndrome is caused by a defect in the final step in cholesterol synthesis and is characterized by distinctive facial features, small head size, mental retardation or learning disabilities, and behavioral problems.

Steiner is a fellow of the American College of Medical Genetics and of the American Academy of Pediatrics. He is a member of numerous professional organizations, including the American Society of Human Genetics, the American Association for the Advancement of Science and the American Board of Medical Genetics. He has published more than 70 papers in nationally and internationally respected peer-reviewed journals.

Nathan Selden, M.D., Ph.D.,
Co-Principal Investigator
Nathan Selden, M.D., Ph.D., F.A.C.S., F.A.A.P., is the Campagna Associate Professor of Pediatric Neurological Surgery and head of the Division of Pediatric Neurological Surgery at Doernbecher Children's Hospital, Oregon Health & Science University School of Medicine. Selden also serves as vice chairman for education and residency program director for the Department of Neurological Surgery at OHSU.

Selden earned his medical degree at Harvard Medical School and also completed a doctoral degree in neuroscience at Cambridge University, England, where he was a Marshall Scholar. He completed his residency in neurological surgery at the University of Michigan Medical Center and undertook postdoctoral fellowships in pediatric neurosurgery and neuroanatomy at Northwestern University Medical School.

His clinical research interests include pediatric brain tumors and epilepsy, congenital malformations of the spine and skull base, and pediatric neurotrauma.

Selden also has expertise in the treatment of hydrocephalus, spasticity, pediatric movement disorders, cranial deformities and synostosis, and pediatric vascular malformations.

His research includes a National Institutes of Health grant to study mechanisms of chronic pain transmission in the brain stem as well as funding from the Oregon Bioscience Innovation Fund.

Selden is a fellow of the American College of Surgeons and of the American Academy of Pediatrics. He is a member of numerous professional societies and serves on the Executive Committee of the Congress of Neurological Surgeons as well as on the editorial boards of Pediatric Neurosurgery and the Self-Assessment in Neurological Surgery, for which he is editor. Selden has been invited to present at dozens of national conferences and meetings and has published more than 30 scholarly articles on neuroscience, neurosurgery and surgical education.

Thomas K. Koch,
M.D., F.A.A.P., F.A.A.N.
Thomas K. Koch, M.D., F.A.A.P., F.A.A.N., is director of pediatric neurology, professor of pediatrics and neurology, Doernbecher Children’s Hospital, Oregon Health & Science University School of Medicine.

Koch earned his medical degree at the University of California, San Diego (UCSD).  He completed his residency in pediatrics at UCSD and residency and fellowship in neurology/child neurology at the University of California, San Francisco (UCSF). He also completed a fellowship in neurochemistry at UCSF.

He specializes in epilepsy, headache, cerebral malformations, genetic and metabolic diseases of the nervous system, and HIV and the nervous system. In addition to the stem cells clinical trial, Koch is engaged in clinical trials involving the use of new antiepileptic drugs for pediatric epilepsy and treatment trials for pediatric migraine. 

Koch is a member of many professional organizations, including the American Academy of Pediatrics, American Academy of Neurology, Child Neurology Society, American Epilepsy Society and American Neurologic Association, among others. He has given many lectures and presentations at prestigious professional meetings and conferences across the nation and has published numerous peer-reviewed articles. He is on the editorial board for the journal Pediatric Neurology.

Koch is the national chairman of the American Academy of Pediatrics’ Section on Neurology, secretary/treasurer of the Professors of Child Neurology, and an examiner for the American Board of Psychiatry and Neurology.

Amira Al-Uzri,
M.D., M.C.R.
Amira Al-Uzri, M.D., M.C.R., is an associate professor of pediatrics (pediatric nephrology and hypertension), and  director of the pediatric kidney transplant porgram at  Doernbecher Children's Hospital, OHSU School of Medicine.

Al-Uzri earned her medical degree from the University of Baghdad Medical College and a masters in clinical research (medical informatics) from OHSU. She completed her residency in pediatrics at Texas Tech and two fellowships -- pediatric nephrology and clinical pharmacy -- at the University o California, San Francisco.

Al-Uzri specializes in post transplantation anemia, kidney function measurement and body composition, and post-renal transplantation. She is a member of the American Society of Pediatric Nephrology and the American Society of Transplantation.

She has published numerous peer-reviewed articles and has been invited to speak at universities and research conferences nationwide.