Source: BUSINESS WIRE
Thursday, November 12, 2009
Data Provide Preclinical Proof-of-Concept to Support Clinical
Development of GRNOPC1 in Patients With Cervical Spinal Cord Injuries
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MENLO PARK, Calif.--(BUSINESS WIRE)--Geron Corporation (Nasdaq:GERN) today announced the publication of data
showing that oligodendrocyte progenitor cells (OPCs) derived from human
embryonic stem cells (hESCs), when transplanted into a rodent model of
cervical spinal cord injury, reduced tissue damage within the lesion and
improved recovery of locomotor function. These data provide preclinical
proof-of-concept for the use of GRNOPC1, Geron’s hESC-derived
oligodendrocyte progenitor product, in patients with cervical spinal
cord injuries. Over half of the 11,000 human spinal cord injuries that
are sustained in the U.S. annually are in the cervical region.
The study was authored by Geron collaborator Dr. Hans S. Keirstead and
colleagues at the Reeve-Irvine Research Center and the Sue & Bill Gross
Stem Cell Research Center at the University of California at Irvine. The
paper was published online in advance of print in the journal Stem
Cells. The abstract of the publication is available at http://www3.interscience.wiley.com/journal/122666108/abstract.
Oligodendrocytes have two main functions in the spinal cord; they
produce the myelin that wraps around nerve fibers to enable electrical
impulse conduction and they produce other molecules (neurotrophic
factors) that help to maintain nerve cells. In spinal cord injury
oligodendrocytes are lost, resulting in the loss of myelin and death of
nerve cells that can cause paralysis below the injury. The present
study, conducted in a cervical model of spinal cord injury, adds to
previous work in a thoracic model, which has demonstrated that injection
of hESC-derived OPCs into the site of injury improved locomotor function
with evidence of remyelination of nerve fibers.
The cervical injury model used in this study induced widespread tissue
loss resulting in a cavity in the spinal cord. In contrast, there was no
cavity in the spinal cord of the rodents that had been injected with
hESC-derived OPCs seven days after injury, and the transplant area
contained human oligodendrocytes. Further analysis of the injury sites
revealed there were significantly more normally myelinated neurons,
fewer demyelinated neurons, and importantly, a greater number of
preserved motor neurons compared to controls. These data provide in
vivo evidence that hESC-derived OPCs may protect the spinal cord
from tissue damage induced by injury in addition to having a
remyelinating function. Along with these observations was noted a
decrease in genes associated with tissue damage and inflammation
suggestive of a mechanism in which hESC-OPCs are exerting their
tissue-sparing effect.
Critically, the preservation of motor neurons within the spinal cord was
shown to correlate with functional recovery. In the cervical injury
model forelimb function was compromised. The animals that had received
hESC-OPCs showed significantly greater improvement in forelimb stride
length and range of motion compared to untreated controls.
“We are excited by Dr. Keirstead’s study in the cervical injury model,”
said Thomas B. Okarma, Ph.D., M.D., Geron's president and chief
executive officer. “These preclinical studies demonstrate that
transplantation of hESC-derived OPCs resulted in sparing of spinal cord
tissue in the cervical lesion site. This sparing starts very soon after
injection and importantly, results in the preservation of motor neurons
which is correlated to recovery of forelimb movement. Our own
IND-enabling safety and efficacy studies with GRNOPC1 in a cervical
injury model are ongoing and will be submitted to the FDA upon
completion.”
About Geron
Geron is developing first-in-class biopharmaceuticals for the treatment
of cancer and chronic degenerative diseases, including spinal cord
injury, heart failure and diabetes. The company is advancing an
anti-cancer drug and a cancer vaccine that target the enzyme telomerase
through multiple clinical trials in different cancers. For more
information, visit www.geron.com.
This news release may contain forward-looking statements made pursuant
to the “safe harbor” provisions of the Private Securities Litigation
Reform Act of 1995. Investors are cautioned that statements in this
press release regarding potential applications of Geron’s human
embryonic stem cell technology constitute forward-looking statements
that involve risks and uncertainties, including, without limitation,
risks inherent in the development and commercialization of potential
products, uncertainty of clinical trial results or regulatory approvals
or clearances, need for future capital, dependence upon collaborators
and maintenance of our intellectual property rights. Actual results may
differ materially from the results anticipated in these forward-looking
statements. Additional information on potential factors that could
affect our results and other risks and uncertainties are detailed from
time to time in Geron’s periodic reports, including the quarterly report
on Form 10-Q for the quarter ended September 30, 2009.