FDA Approval To Initiate Clinical Trial Of HuCNS-SC Cells In A Myelin Disease

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StemCells, Inc. Receives FDA Approval To Initiate Clinical Trial Of

HuCNS-SC® Cells In A Myelin Disease

01 Jan 2009

http://www.medicalnewstoday.com/articles/134184.php

StemCells, Inc. (NASDAQ:STEM) today announced that it has received

approval from the U.S. Food and Drug Administration (FDA) to initiate

a clinical trial of the Company's proprietary HuCNS-SC product

candidate (purified human neural stem cells) to treat Pelizaeus-

Merzbacher Disease (PMD), a fatal brain disorder that mainly affects

young children. This Phase I trial is designed to evaluate the safety

and preliminary efficacy of HuCNS-SC cells as a treatment for PMD.

Currently, there are no approved treatments for this disease.

This is the Company's second FDA-approved clinical trial to evaluate

HuCNS-SC cells as a potential treatment for neurodegenerative

diseases. The first such study was the Company's Phase I clinical

trial of HuCNS-SC cells to treat neuronal ceroid lipofuscinoses

(NCL), or Batten disease. The Phase I NCL trial will be completed in

January 2009.

Patients with PMD are born with a gene mutation that results in

insufficient myelination of nerve fibers in the brain, neurological

impairment and eventually death. Myelin, which is produced by special

cells called oligodendrocytes, insulates nerve fibers to allow

electrical signals to be conducted normally. Other, more common,

myelination diseases include cerebral palsy, transverse myelitis and

multiple sclerosis.

Preclinical studies performed by the Company and its collaborators

provide a rationale for potential therapeutic use of HuCNS-SC cells

in myelination diseases. The Company has demonstrated that, when

transplanted into an animal model of hypomyelination (shiverer

mouse), HuCNS-SC cells engraft and differentiate into mature

oligodendrocytes and form myelin sheaths around host nerve fibers.

The initial myelination data in the shiverer mouse was published in

the Proceedings of the National Academy of Science (Cummings, et al.

2005) and the results of additional myelination studies were

presented by Nobuko Uchida, Ph.D., the Company's Vice President of

Stem Cell Biology, at the International Society of Stem Cell Research

annual meeting in Philadelphia earlier this year.

" PMD is one of the most severe myelin disorders known to man.

Children afflicted with this disease suffer progressive

neurodegeneration and an untimely death. Unfortunately, they have no

option for restorative treatment, " said Huhn MD, FACS, FAAP,

Vice President and Head of the CNS Program at StemCells, Inc. " We are

very pleased to be the first company to initiate a neural stem cell

trial for a myelination disease. Establishing safety and efficacy of

our cells in patients with PMD may pave the way for similar studies

in patients with other myelin diseases. " The Company has begun the

process of seeking approval by the Investigational Review Board (IRB)

of potential clinical trial sites in order to begin enrolling

patients.

About Pelizaeus-Merzbacher Disease (PMD)

PMD is a rare, degenerative, central nervous system disorder and is

one of a group of genetic disorders known as leukodystrophies.

Leukodystrophies involve abnormal growth of the myelin sheath which

is the fatty substance - or insulator - on nerve fibers in the brain

and spinal cord. PMD is most commonly caused by a genetic mutation

that affects an important protein found in myelin, proteolipid

protein (PLP). PMD is most frequently diagnosed in early childhood

and is associated with abnormal eye movements (nystagmus), abnormal

muscle function, and in some cases seizures. The disease form in

early infancy is referred to as connatal PMD and diagnosis in later

childhood is most typically associated with the classic form. The

neurological course of both forms is marked by progressive

deterioration resulting in premature death.

About HuCNS-SC® Cells

StemCells' lead product candidate, HuCNS-SC cells, is a purified

composition of normal 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; they engraft,

migrate, differentiate into neurons and glial cells; and they 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 a clinical-stage biotechnology company focused on

the discovery, development and commercialization of cell-based

therapeutics to treat diseases of the central nervous system and

liver. The Company's product development programs seek to repair or

repopulate CNS and liver tissue that has been damaged or lost as a

result of disease or injury. StemCells has pioneered the discovery

and development of HuCNS-SC cells, its highly purified, expandable

population of human neural stem cells. StemCells owns or has

exclusive rights to more than 50 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