US Approves First Trial Of Embryonic Stem Cells

January 23, 2009

The US regulators have given the go ahead for a biotech company to carry out 
the world's first trial of a treatment using embryonic stem cells, in this 
case to treat victims of spinal cord injury that leaves them unable to walk.

Although heralded as an early sign of new US president Obama's support for 
embryonic stem cell research, Dr Thomas Okarma, president and CEO of Geron 
Corp. of Menlo Park, California, the company that will be carrying out the 
trial, told Associated Press that strictly speaking it was not so in this 
case, since the next phase of the project would have been eligible for 
federal funds under Bush. It was the earlier development phases that had to 
be done with private funds because of funding restrictions on embryonic stem 
cell research brought in by Bush in 2001, said a report in Times Online.

Today's ruling by the US Food and Drug Administration (FDA) allows Geron to 
give 8 to 10 patients with spinal cord injury a single injection of cells 
made from embryonic stem cells. The testing will be done at several medical 
centres throughout the US and the patients will receive their injections 
within 2 weeks of their injury; unfortunately patients whose injuries are 
older than this are unlikely to benefit from such therapy.

According to a Geron statement, the Phase I trial is designed to establish 
the safety of the treatment, called GRNOPC1, in patients with "complete" 
American Spinal Injury Association (ASIA) grade A subacute thoracic spinal 
cord injuries.

Okarma told AP that they will also be looking for the possible return of 
feeling or movement in the legs. Animal studies have shown that injected 
embryonic stem cells can turn into specialist cells that replace damaged 
sheaths around nerves that have suffered "demyelination" and thereby restore 
their ability to send signals that control muscle and sense feelings.

Embryonic stem cells are like master cells, they can become virtually any 
cell of the body, which comprises about 200 types of tissue. There are other 
kinds of stem cells too, like adult stem cells, but they are more limited in 
what types of cell they can become and harder to "coax", so embryonic stem 
cells are considered the "gold standard" in stem cell research. They are 
however more controversial because harvesting them involves destroying 
embryos, whereas adult stem cells can be recovered without harming the 
donor.

If successful, the trial will lead to therapies with the potential to 
transform the lives of thousands of people who every year become paralysed 
from the chest down, and for whom few treatments exist. Experts predict that 
if successful, the therapy could be in general use within three to five 
years.

Okarma said in a press statement that:

"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 by the injection of healthy 
replacement cells."

He said that: "the ultimate goal is to achieve restoration of spinal cord 
function".

Although the FDA decision is independent of the White House, it is being 
received as a symbol of a new attitude to embryonic stem cell research and 
Obama is expected to start lifting funding restrictions next week, said a 
Times Online report.

Dr Richard Fessler, professor of neurological surgery at the Feinberg School 
of Medicine at Northwestern University, said that:

"The neurosurgical community is very excited by this new approach to 
treating devastating spinal cord injury."

"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 safe and effective, the therapy would 
provide a viable treatment option for thousands of patients who suffer 
severe spinal cord injuries each year," he added.

Polling figures suggest most Americans are in favour of stem cell research, 
but it was strong opposition lobbying that persuaded Bush to impose 
restrictions on federal funding.

Okarma said that the delay caused by the funding restriction meant there 
"are people out there who might have benefited, but who now cannot," he told 
the press.

Sources: Times Online, Associated Press, Geron.

Written by: Catharine Paddock, PhD
Copyright: Medical News Today
Not to be reproduced without permission of Medical News Today

Rayilyn Brown
Director AZNPF
Arizona Chapter National Parkinson Foundation
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