Geron, a California-based
cell-therapy company that has been working with embryonic stem cells for the
last decade,
finally received clearance from the U.S. Food and Drug Administration to begin
clinical trials of its cell-based therapy for spinal-cord injury.
The trial is
limited to eight patients with newly acquired spinal-cord injuries who will
receive injections of the cell therapy, called GRNOPC1, within two weeks of
their accident. GRNOPC1 is made by
transforming embryonic stem cells into oligodendrocytes–a type of brain cell
that wraps itself around neurons, forming a fatty insulation layer that allows
electrical messages to be conducted throughout the nervous system. In many spinal-cord injuries, these cells are damaged, but the underlying nerve cells remain
intact. These cells are then injected into the site of the injury, coating exposed
nerves and restoring communication to the nervous system.
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Scientists
published the results of a successful study testing the therapy in animals in
2005, showing that paralyzed rats injected with the cells were able to walk
again. Since then, Geron has been conducting numerous studies intended to
show the safety of the cell-based therapy, as well as developing production
methods that would make the cells as easy to use as more traditional
treatments. Geron researchers have also developed a way to reliably freeze and
thaw brain cells, so that they can be manufactured in a central location,
and then shipped to the hospitals where they will be used.
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Because
embryonic-stem-cell-based therapies are so new, the FDA has had trouble deciding
how to evaluate new drug applications. (Geron’s president and CEO, Thomas
Okarma, thought approval was imminent
when I spoke with him in 2006. See “Human Tests of
Embryonic Stem Cell Treatments Planned.”) The initial trial is designed to
assess safety, but doctors will also measure its effectiveness, such as
improved neuromuscular control or sensation in the trunk or lower extremities.
The announcement
comes amid general excitement in the stem-cell field, thanks to Barack Obama’s
promise to lift funding restrictions for embryonic-stem-cell research. (See “Braving Medicine’s
Frontier.”)
Geron’s Thomas
Okarma said in a statement, “This
marks the beginning of what is potentially a new chapter in medical therapeutics–one that reaches beyond pills to a new level of healing: the restoration of
organ and tissue function achieved by the injection of healthy replacement
cells. The ultimate goal for the use of GRNOPC1 is 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.”
This video illustrates how Geron’s cell therapy works in rats.