Full Press Release Details
Proceed with First Human Clinical Trial of Embryonic Stem Cell-Based
to Study GRNOPC1 in Patients with Acute Spinal Cord Injury
MENLO PARK, Calif.--(BUSINESS WIRE)--July 30, 2010--Geron Corporation
(Nasdaq:GERN) announced today that the U.S. Food and Drug Administration
(FDA) has notified the company that the clinical hold placed on Geron's
Investigational New Drug (IND) application has been lifted and the
company's Phase I clinical trial of GRNOPC1 in patients with acute
spinal cord injury may proceed.
The FDA notification enables Geron to move forward with the world's
first clinical trial of a human embryonic stem cell (hESC)-based therapy
in man. The Phase I multi-center trial is designed to establish the
safety of GRNOPC1 in patients with "complete" American Spinal Injury
Association (ASIA) Impairment Scale grade A subacute thoracic spinal
"We are pleased with the FDA's decision to allow our planned clinical
trial of GRNOPC1 in spinal cord injury to proceed," said Thomas B.
Okarma, Ph.D., M.D., Geron's president and CEO. "Our goals for the
application of GRNOPC1 in subacute spinal cord injury are unchanged - to
achieve restoration of spinal cord function by the injection of
hESC-derived oligodendrocyte progenitor cells directly into the lesion
site of the patient's injured spinal cord. Additionally, we are now
formally exploring the utility of GRNOPC1 in other degenerative CNS
disorders including Alzheimer's, multiple sclerosis and Canavan disease."
The clinical hold was placed following results from a single preclinical
animal study in which Geron observed a higher frequency of small cysts
within the injury site in the spinal cord of animals injected with
GRNOPC1 than had previously been noted in numerous foregoing studies. In
response to those results, Geron developed new markers and assays as
additional release specifications for GRNOPC1. The company completed an
additional confirmatory preclinical animal study to test the new markers
and assays, and subsequently submitted a request to the FDA for the
clinical hold to be lifted.
GRNOPC1, Geron's lead hESC-based therapeutic candidate, contains
hESC-derived oligodendrocyte progenitor cells that have demonstrated
remyelinating and nerve growth stimulating properties leading to
restoration of function in animal models of acute spinal cord injury (Journal
of Neuroscience, Vol. 25, 2005).
"The neurosurgical community is ready to begin the clinical testing of
this new approach to treating devastating spinal cord injury," said
Richard Fessler, M.D., Ph.D., professor of neurological surgery at the
Feinberg School of Medicine at Northwestern University. "We know that
demyelination is central to the pathology of the injury, and its
reversal by means of injecting oligodendrocyte progenitor cells would be
revolutionary for the field. If found to be safe and effective, the
therapy would provide a viable treatment option for thousands of
patients who suffer severe spinal cord injuries each year."
The GRNOPC1 Clinical Program
Patients eligible for the Phase I trial must have documented evidence of
functionally complete spinal cord injury with a neurological level of T3
to T10 spinal segments and agree to have GRNOPC1 injected
into the lesion sites between seven and 14 days after injury.
Although the primary endpoint of the trial is safety, the protocol
includes secondary endpoints to assess efficacy, such as improved
neuromuscular control or sensation in the trunk or lower extremities.
Once safety in this patient population has been established, Geron plans
to seek FDA approval to extend the study to increase the dose of
GRNOPC1, enroll subjects with complete cervical injuries and expand the
trial to include patients with severe incomplete (ASIA Impairment Scale
grade B or C) injuries to enable access to the therapy for as broad a
population of severe spinal cord-injured patients as is medically
Geron has selected up to seven U.S. medical centers as candidates to
participate in this study and in planned protocol extensions. The sites
will be identified as they come online and are ready to enroll subjects
Other Potential Neurological Indications for GRNOPC1
In addition to spinal cord injury, GRNOPC1 may have therapeutic utility
for other central nervous system indications. Geron has established a
number of collaborations with academic groups to test GRNOPC1 in
selected animal models of human disease for which there is a strong
rationale for the approach.
Alzheimer's Disease: Alzheimer's disease is a progressive,
fatal, degenerative disorder that attacks the neurons in the brain,
resulting in loss of memory, cognitive function such as reasoning and
language, and behavioral changes. According to the Alzheimer's
Association an estimated five million people in the United States have
Alzheimer's disease. GRNOPC1 is being evaluated in animal models of
Alzheimer's disease in collaboration with Professor Frank M. LaFerla,
Director of the Institute for Memory Impairments and Neurological
Disorders (UCI MIND) at the University of California, Irvine.
Multiple Sclerosis (MS): MS is an autoimmune disease that causes
demyelination of nerve axons in the brain and spinal cord often
progressing to physical and cognitive disability. There is currently no
known cure for the disease. According to the National Multiple Sclerosis
Society there are about 400,000 people in the United States with MS.
GRNOPC1 is being tested in a non-human primate model of MS in
collaboration with Professor Jeffery D. Kocsis of the Departments of
Neurology and Neurobiology at Yale University School of Medicine and the
Department of Veterans Affairs.
Canavan Disease: Canavan disease is a fatal neurological disorder
that belongs to a group of genetic disorders called leukodystrophies,
characterized by the abnormal development or degeneration of myelin.
Symptoms of Canavan disease present in the first six months of life and
death usually occurs at 3 - 10 years of age. GRNOPC1 is being tested in
a rodent model of Canavan disease in collaboration with Dr. Paola Leone,
Director of the Cell and Gene Therapy Center, at the University of
Medicine and Dentistry of New Jersey.
Background on GRNOPC1
Additional information on Geron's hESC programs and GRNOPC1 is available
at Geron's website www.geron.com.
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