The first clinical trial of embryonic stem cells is on track to
start early next year on patients with spinal cord injury. Geron
(NASDAQ:GERN - news), the California-based Biotechnology company, will
carry out the study on accident victims in six trauma centres across
the US.
"The world's spotlight will be on this trial," Tom Okarma, Geron's
chief executive, told the Bio conference in Boston. To get it right,
the company has carried out several years of preparatory work in
collaboration with its academic partners at the University of
California, Irvine.
Geron's product will have been tested in 2,000 animals before it
goes into its first patient, Mr Okarma said. It consists of immature
oligodendrocytes - specialised nerve cells - grown from human embryonic
stem cells. The animal tests show that these can repair spinal cord
injuries in rats, by growing new nerves with the Myelin sheaths they
need to work properly. Paralysed rats can walk again.
Mr Okarma said the product was designed to repair recent spinal
damage and would need to be injected into patients within two weeks of
the accident. It could not help people with long-term paralysis such as
the late actor Christopher Reeve who did so much to champion stem cell
research for spinal injury.
Because the first application for a human embryonic stem cell trial
is bound to receive extraordinary scrutiny from the US Food and Drug
Administration, Geron is working closely with FDA officials to smooth
the path in advance of the submission. The embryonic stem cells are
already qualified for human therapeutic use, after exhaustive tests
showed that the cell cultures contain no contamination with animal
proteins or viruses.
Mr Okarma pointed out that human embryonic stem cell research was
less than 10 years old; the first such cells were produced at the
University of Wisconsin in 1998. "With breakthrough therapies such as
antibodies and genetic engineering there is usually a 10 to 20 year
period of government funding academic research that improves upon the
basic invention before industry can put its toes in," he said.
That has not happened with stem cells because of the Bush
Administration's federal funding restrictions. As a result, Mr Okarma
said, "we have to pay the bill for every academic collaboration - and
that is not sustainable."
Hans Kierstead, head of the embryonic stem cell team at UC Irvine,
was worried about the high level of public expectation. "I find it
unfair that people demand so much from such a young field," he said.
"Just look at the strides we have already made."
Bob Klein, architect of California's Proposition 71 authorising the
state to spend at least $3bn over 10 years on stem cell research, also
told the Bio conference of his concern about excessive expectations.
"In clinical trials there will inevitably be failures as well as
successes," he said.
"The ideological right is not asleep," said Mr Klein, who chairs
California Institute of Regenerative Medicine's governing board. "They
will be prepared to spin [any problems] to promote their opposition to
embryonic stem cell research, and we'll need a sophisticated
fast-response messaging team to put across our message."
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