Message #29 From:
Stock News Bot Date: December 6, 2006 04:30:00 AM
STEM News Investigator in Phase I Clinical Trial for Batten Disease to Present at Lysosomal Disease Network World Symposium
PALO ALTO, Calif.--(BUSINESS WIRE)--Robert D. Steiner, M.D., F.A.A.P., F.A.C.M.G., will present at the Third
Annual Lysosomal Disease Network World Symposium at 1:30 p.m. EST
on Friday, December 8, 2006. This year’s World
Symposium will be held December 7-9, 2006 at Walt Disney World in
Orlando, Florida.
Dr. Steiner is a lead investigator in the Phase I clinical trial
sponsored by StemCells, Inc., (NASDAQ: STEM) which is designed to
evaluate the safety and preliminary efficacy of HuCNS-SC™
as a treatment for infantile and late infantile neuronal ceroid
lipofuscinosis (NCL). NCL, often referred to as Batten disease, is a
rare and fatal neurodegenerative condition afflicting infants and
children. Dr. Steiner’s presentation, entitled “A
Phase 1 Clinical Study of Human CNS Stem Cells (HuCNS-SC™)
in Patients with Neuronal Ceroid Lipofuscinosis”,
will highlight the rationale and pre-clinical data that suggest
transplanting human neural stem cells may lead to a possible treatment
for NCL. The first transplantation of HuCNS-SC into a patient in the
trial took place November 14, 2006 at Oregon Health & Science University’s
(OHSU) Doernbecher Children’s Hospital.
Dr. Steiner is vice chairman of pediatric research and head of the
Division of Metabolism at Doernbecher Children’s
Hospital, and professor of Pediatrics and Molecular & Medical Genetics
at OHSU School of Medicine. In addition to Dr. Steiner, the Phase I
clinical trial is being led by 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
and OHSU School of Medicine and Thomas K. Koch, M.D., F.A.A.P.,
F.A.A.N., director of Pediatric Neurology and professor of Pediatrics
and Neurology at Doernbecher and OHSU School of Medicine.
About Neuronal Ceroid Lipofuscinosis (Batten Disease)
Neuronal ceroid lipofuscinosis is a rare and 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.
In infantile and late infantile NCL, the disorder 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
substrates accumulate to the point of interference with normal cellular
and tissue 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 HuCNS-SC™
StemCells’ human neural stem cells (HuCNS-SC)
are a cell-based therapeutic prepared under controlled conditions. When
HuCNS-SC are transplanted into the brain of a mouse model developed to
mimic the human form of infantile NCL, the cells spread throughout the
brain and produce the missing lysosomal enzyme. The enzyme level
increases and continues to do so over time after the transplant. Thus,
placement of HuCNS-SC in appropriate places in the brain provides the
prospect of long-term delivery of the missing lysosomal enzyme. In
laboratory studies, HuCNS-SC also produces the lysosomal enzyme missing
in late infantile NCL, the other subtype being studied in the clinical
trial. The production of both enzymes by HuCNS-SC provides a scientific
rationale for enzyme replacement and cellular rescue in these two
subtypes of NCL.
About the Clinical Trial
The Phase I trial is designed to evaluate the safety and preliminary
efficacy of HuCNS-SC as a treatment of infantile and late infantile NCL.
The trial is open label with two dose cohorts, and is expected to enroll
six patients. Potential patients will be tested for eligibility and then
evaluated for baseline disease status prior to transplantation of
HuCNS-SC. In addition to measuring the safety of HuCNS-SC, patients
enrolled in the study will be evaluated with standardized measures of
development, cognition, behavior and language for one year following
transplantation.
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 is
the first company to directly identify and isolate human neural stem
cells from normal brain tissue. These 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 has
approximately 40 U.S. and 100 non-U.S. patents. Further information
about the Company is available on its Web site at www.stemcellsinc.com.
About OHSU
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 spin-off companies,
most of which are based in Oregon.
About 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.
Apart from statements of historical facts, the text of this press
release constitutes forward-looking statements regarding, among other
things, the future business operations of StemCells, Inc. (the “Company”)
and its ability to conduct clinical trials as well as its research and
product development efforts. The 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 whether results obtained in the animal models and in vitro
studies of infantile NCL or other diseases and conditions will be able
to be translated into treatment for humans; uncertainty as to whether
HuCNS-SC will prove safe in the current clinical trial; uncertainty as
to whether the current trial will provide any information about the
possible efficacy of HuCNS-SC in treating NCL; uncertainty as to whether
the FDA or other applicable regulators or review boards will permit the
Company to continue clinical testing in NCL or in future clinical trials
of proposed therapies for other diseases or conditions despite the novel
and unproven nature of the Company’s
technology; uncertainties regarding the timing and duration of any
clinical trials; uncertainties regarding the Company’s
ability to obtain the increased capital resources needed to continue its
current research and development operations and to conduct the research,
preclinical development and clinical trials necessary for regulatory
approvals; uncertainty regarding the validity and enforceability of the
Company’s patents; uncertainty as to whether
HuCNS-SC and any products that may be generated in the future in the
Company’s research and development programs
will prove safe and clinically effective and not cause tumors or other
side effects; uncertainty as to whether the Company will achieve
revenues from product sales or become profitable; and other factors that
are described under the heading “Risk Factors”
in Item 1A of the Company’s Annual Report on
Form 10-K.
