StemCells, Inc. Announces Filing of IND for Human Neural Stem Cell Transplant Treatment for Batten Disease
Phase I Clinical Trial at Stanford Would Be First-Ever Using
Transplantation of Human Neural (Brain) Stem Cells
PALO ALTO, Calif.--(BUSINESS WIRE)--Jan. 4, 2005-- StemCells, Inc. (NASDAQ: STEM) today announced the filing of its
first Investigational New Drug (IND) application to the U.S. Food and
Drug Administration (FDA). Subject to approval, the Company plans to
begin its first clinical investigation of its human neural (brain)
stem cells (hCNS-SC - StemCells' proprietary neural cell therapy
product) in Batten disease. Batten disease is a rare, fatal genetic
disorder that affects the central nervous system of children. If
approved by the FDA, this would mark the first-ever FDA-approved
clinical trial to use a purified composition of human neural stem
cells as the potential therapeutic agent.
On approval of the IND by the FDA, the study will be conducted at
Lucile Packard Children's Hospital/Stanford University Medical Center
(LPCH/SUMC) under the direction of Stephen L. Huhn, MD, FACS, FAAP,
Chief of Pediatric Neurosurgery and Gregory M. Enns, MB, ChB,
Assistant Professor and Director, Biochemical Genetics Program,
Division of Medical Genetics.
"This is truly a significant milestone, not only for StemCells,
Inc., but also for all scientists who have been seeking to evaluate
possible therapies for neural and neurodegenerative diseases," said
Martin McGlynn, chief executive officer of StemCells, Inc. "Our
pre-clinical research has shown great promise and this filing is an
essential step in discovering how to translate those pre-clinical
results into treatment of human victims of terrible disorders like
Batten disease and other neurodegenerative lysosomal storage diseases.
It is only a first step, though - this is a Phase I, or safety, trial,
from which we hope to learn about the behavior of the cells when they
are transplanted into a human recipient. There will be many other
steps to take before we arrive. But it is our hope that
transplantation of human neural stem cells could prove to be a
platform technology for a wide range of conditions for which there is
now no reliable and effective treatment."
"We are looking forward to working with the scientists at
StemCells in this historic clinical trial," said Dr. Huhn, at Stanford
University School of Medicine. "We are exploring new territory, which
dictates that we proceed with due caution. I believe, however, that
our path has been determined by rigorous research and a well-designed
protocol. Physicians have been essentially helpless to assist children
suffering from Batten's, and all of us involved in this trial are
hoping it will lead to a future in which we will have an efficient
treatment, or even a cure. As a pediatric neurosurgeon, I am
particularly excited about this avenue of research."
About the Clinical Trial
The proposed Phase I trial is designed to investigate the safety
of hCNS-SC in the treatment of infantile and late-infantile neuronal
ceroid lipofuscinosis (NCL), the most severe forms of a group of
disorders commonly referred to as Batten disease. The trial will be an
open label study of two dose levels involving three subjects in each
of two cohorts. The primary objective of the trial will be to measure
the safety of hCNS-SC. However, the trial will also evaluate
hCNS-SC's ability to affect the progression of the disease. The
patient/subject evaluation will be up to one year post hCNS-SC
transplantation. Candidates from anywhere in the world will be
referred by their primary physicians to the Co-principal Investigators
at LPCH/SUMC. Potential patients will be tested for eligibility and
then evaluated for baseline disease status prior to transplantation.
The Company is committed to following the effects of this therapy
long-term, so potential trial patients will also be asked to commit to
a four year follow-up study.
About Batten Disease
Batten disease is named after the British pediatrician who first
described the juvenile form of NCL in 1903. It is also known as
Spielmeyer-Vogt-Sjogren-Batten disease. The name is now commonly used
to encompass all three forms of NCL. The forms of NCL are classified
by age of onset (infantile, late infantile and juvenile) but are more
precisely classifiable in terms of the specific enzyme causing the
disease. They all have the same basic cause - lack of a lysosomal
enzyme - and similar progression and outcome, but are all genetically
different. Children with Batten disease suffer seizures, progressive
loss of motor skills, sight and mental capacity, eventually becoming
blind, bedridden and unable to communicate. Today, Batten disease is
always fatal.
In two sub-types of the NCLs - infantile and late infantile or,
more technically, CLN1 and CLN2 - normally secreted housekeeping
lysosomal enzymes are either defective or missing altogether, as a
result of gene mutations. Lack of either enzyme causes a buildup of
lipofuscin (aggregates of lipids and proteins) primarily in the brain
and leads to neuronal cell loss.
In the proposed clinical trial, hCNS-SC will be transplanted in
the CLN1 and CLN2 patients in part to determine if the transplanted
cells secrete the missing lysosomal enzymes in the brains of affected
individuals. hCNS-SC have been shown to produce both PPT1 and TPP-I
enzymes, providing a scientific justification for enzyme replacement
and cellular rescue in this indication. In preclinical models of PPT1
deficiency, the corresponding enzyme activity increases with time
after transplantation.
About hCNS-SC and the Clinical Trial
StemCell's human central nervous system stem cells (hCNS-SC) are
a somatic cell therapy product consisting of neural cells prepared
under controlled conditions. Neural stem cells, a rare subset of brain
cells, are isolated from the human fetal brain, purified, propagated,
and tested; they are then frozen in cell banks from which hCNS-SC
doses can be prepared.
NCLs are lysosomal storage disorders brought on by inherited
genetic mutations in CLN1 gene, which codes for palmitoyl-protein
thioesterase 1 (PPT1) and in the CLN2 gene, which codes for
tripeptidyl peptidase I (TPP-I). The consequence of these mutations is
the accumulation of lipofuscin-like fluorescent inclusions in various
cell types that eventually lead to cell degeneration. Presumably,
non-degraded lysosomal substrates accumulate to the point where they
interfere with normal cellular and tissue function and lead to the
pathological manifestations of the related disease. To correct the
major defect in these subjects, enzyme would have to be available in
the brain where it can be taken up by the enzyme deficient cells. A
property of hCNS-SC is production of both PPT1 and TPP-I enzymes.
Thus, placement of hCNS-SC in appropriate places in the brain has the
prospect of replacing the missing enzyme.
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CONTACT: StemCells, Inc.
Judi Lum, 650-475-3100
or
Schwartz Communications, Inc.
Media:
781-684-0770 or 415-512-0770
stemcells@schwartz-pr.com
SOURCE: StemCells, Inc.
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