Nov. 9, 2009 - The first human embryonic stem cell
treatment approved by the FDA for human testing has been shown to
restore limb function in rats with neck spinal cord injuries setting
the stage for the first embryonic stem cell clinical trial to include
people with cervical damage.
In January, the U.S. Food & Drug Administration
gave Geron Corp. of Menlo Park, Calif., permission to test the UC Irvine
treatment in individuals with thoracic spinal cord injuries, which occur
below the neck. (See release below news story.)
However, trying it in those with cervical damage
wasn't approved because preclinical testing with rats hadn't been
completed.
Stem cells have some kind of guidance system and
migrate to the area of injury and, although they're not making new brain
cells, they may be enhancing the repair processes. April 16, 2009
Results of the cervical study currently appear
online in the journal Stem Cells. UCI scientist Hans Keirstead
hopes the data will prompt the FDA to authorize clinical testing of the
treatment in people with both types of spinal cord damage. About 52
percent of spinal cord injuries are cervical and 48 percent thoracic.
"People with cervical damage often have lost or
impaired limb movement and bowel, bladder or sexual function, and
currently there's no effective treatment. It's a challenging existence,"
said Keirstead, a primary author of the study. "What our therapy did to
injured rodents is phenomenal. If we see even a fraction of that benefit
in humans, it will be nothing short of a home run."
A week after test rats with 100 percent walking
ability suffered neck spinal cord injuries, some received the stem cell
treatment. The walking ability of those that didn't degraded to 38
percent. Treated rats' ability, however, was restored to 97 percent.
UCI's therapy utilizes human embryonic stem cells
destined to become spinal cord cells called oligodendrocytes. These are
the building blocks of myelin, the biological insulation for nerve
fibers that's critical to proper functioning of the central nervous
system. When myelin is stripped away through injury or disease,
paralysis can occur.
Lead author and doctoral student Jason Sharp,
Keirstead and colleagues discovered that the stem cells not only rebuilt
myelin but prevented tissue death and triggered nerve fiber regrowth.
They also suppressed the immune response, causing an increase in
anti-inflammatory molecules.
"The transplant created a healing environment in
the spinal cord," said Keirstead, who is co-director of the Sue & Bill
Gross Stem Cell Research Center and on the faculty of the Reeve-Irvine
Research Center - named for late actor Christopher Reeve, who became a
quadriplegic after a cervical spinal cord injury.
In addition to Keirstead and Sharp, Jennifer Frame,
Monica Siegenthaler and Dr. Gabriel Nistor of UCI worked on the study,
which was supported by Geron Corp., a University of California Discovery
Grant, the Roman Reed Spinal Cord Injury Research Fund of California,
Research for Cure, and individual donations to the Reeve-Irvine Research
Center.
About the University of California, Irvine:
Founded in 1965, UCI is a top-ranked university dedicated to research,
scholarship and community service. Led by Chancellor Michael Drake since
2005, UCI is among the fastest-growing University of California
campuses, with more than 27,000 undergraduate and graduate students,
1,100 faculty and 9,200 staff. The top employer in dynamic Orange
County, UCI contributes an annual economic impact of $4.2 billion. For
more UCI news, visit
www.today.uci.edu.
UCI behind world's first embryonic stem
cell study in humans
FDA approves Geron Corp. clinical trial
for spinal cord injury treatment
Irvine, Calif., January 23, 2009
- A therapy developed at UC Irvine that made paralyzed rats
walk again will become the worlds first embryonic stem cell
treatment tested in humans.
The U.S. Food and Drug Administration
has approved the therapy, based on work by a research team
led by Hans Keirstead, co-director of the UCI Sue and Bill
Gross Stem Cell Research Center, for a clinical trial in
patients with acute spinal cord injury.
Geron Corp. of Menlo Park, Calif., will
conduct the clinical trial.
This trial was approved only after
rigorous safety testing and consultation of countless
experts in the field, Keirstead said. Any benefit to the
patient, even an incremental one, would be a resounding
victory.
The therapy contains human embryonic
stem cells destined to become spinal cord cells called
oligodendrocytes. These are the building blocks of myelin,
the biological insulation for nerve fibers that is critical
for maintaining electrical conduction in the central nervous
system. When myelin is stripped away, through injury or
disease, paralysis can occur.
In laboratory tests, Keirstead and his
colleague, Dr. Gabriel Nistor, developed a technique for
prompting human embryonic stem cells to develop into
oligodendrocyte cells.
Injected into rats with spinal cord
injuries, the precursor cells turned into oligodendrocytes
and migrated to the injured area of the spinal cord. As the
cells wrapped around damaged neurons, new myelin tissue
formed, allowing electrical conduction to resume and the
rats to walk again.
This success, published in the
Journal of Neuroscience in 2005, was the subject of
dozens of media stories, including a 60 Minutes segment.
According to Geron, patients eligible
for the phase-one trial must have a certain type of spinal
cord damage and be willing to receive injections 7-14 days
after injury. Geron has selected up to seven U.S. medical
centers that may participate in the study.
UCI has a robust stem cell research
program that has received more than $52 million from the
California Institute for Regenerative Medicine. UCIs
scientists are pioneers in regeneration, large-scale
production of specialized cells with very high purity, and
methods for treating damaged tissues.
UCI recently broke ground for a
four-story building dedicated to stem cell research. When
finished in 2010, the building will house the stem cell
center, dozens of laboratory-based and clinical researchers,
a stem cell techniques course, a masters program in
biotechnology with an emphasis on stem cell research, and
programs and activities for patients and public education.
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