Friday, January 23, 2009

[StemCells] FDA approves Genron embryonic SC spinal trials

First Embryonic Stem-Cell Trial Gets Approval From the FDA Article
By RON WINSLOW and ALICIA MUNDY
Jan 23, 2009

In a watershed moment for one of the most contentious areas of
science and American politics, the U.S. Food and Drug Administration
cleared the way for the first-ever human trial of a medical treatment
derived from embryonic stem cells.

Geron Corp., a Menlo Park, Calif., biotechnology company, is expected
to announce Friday that it received a green light from the agency to
mount a study of its stem-cell treatment for spinal cord injuries in
up to 10 patients. The announcement caps more than a decade of
advances in the company's labs and comes on the cusp of a widely
expected shift in U.S. policy toward support of embryonic stem-cell
research after years of official opposition.

"This is the dawn of a new era in medical therapeutics," said Thomas
B. Okarma, Geron's president and chief executive officer. The hope
that stem-cell therapy will repair and regenerate diseased organs and
tissue "goes beyond what pills and scalpels can ever do."

Limits on stem-cell research, which prevented federal funding and
were imposed by Congress and former President George W. Bush for
ethical and religious reasons, have had a chilling effect on both
academic and corporate research involving such cells. Proponents of
stem-cell research say restrictions have delayed development of
promising new treatments, while critics contend that harvesting stem
cells from embryos destroys human life.

President Barack Obama said during his campaign that overturning
research limits would be a top priority in his administration.

Both Geron and the FDA said the timing of the decision to approve the
study was coincidental. "The FDA looks to the science on these types
of issues, and we approve [such applications] based on a showing of
safety," said Karen Riley, an FDA spokeswoman. "Political
considerations have no role in this process."

Approval of the study is far from a guarantee that stem-cell
treatments will work or make it to the market, but it is likely to be
seen as an indication that opportunities for stem-cell research are
poised to open and will fuel enthusiasm among academic and corporate
researchers.

Mr. Obama's plans for acting on the current research restrictions
haven't been finalized. Shortly after the election, Obama advisers
thrilled biotech companies and investors when they suggested that the
new president could use his executive authority to undo the Bush
administration ban. But in a Jan. 18 interview on CNN, Mr. Obama said
he might let Congress take the lead. "I like the idea of the American
people's representatives expressing their views on an issue like
this," he said.

Regulating stem-cell therapy is new turf for both industry and the
FDA, a major reason why it took the agency nearly a year to review
Geron's 21,000-page application for the trial, which it filed last
March. Approval came in a phone call Wednesday afternoon, Dr. Okarma
said.

The study will focus on the safety of the treatment. At an FDA
hearing in April, several firms' executives and researchers
complained that they were at a loss about what the FDA wanted in
terms of clinical trials involving stem cells because the FDA itself
wasn't sure.

Embryonic stem cells are the building-block cells that help drive
prenatal development. Geron has developed banks of embryonic stem
cells and found a way to coax them into differentiating as they do in
nature into progenitors of specific cells that make spinal-cord
tissue, heart muscle, cartilage and other organs and tissues.

Spinal-cord injury is one of medicine's most debilitating conditions,
typically causing paralysis and other issues for which there are few,
if any, effective treatments. The Geron study will enroll paralyzed
patients who can be treated within 14 days of their injury. Patients
will be evaluated for at least one year, after which, if the
treatment proves safe, the company hopes to increase the dose and
expand the potential candidates for the therapy.

In addition to safety, researchers will look for signs that the
treatment is effective.

Write to Ron Winslow at ron.winslow@wsj.com and Alicia Mundy at
alicia.mundy@wsj.com

Printed in The Wall Street Journal, page A12
http://online.wsj.com/article/SB123268485825709415.html

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StemCells subscribers may also be interested in these sites:

Children's Neurobiological Solutions
http://www.CNSfoundation.org/

Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123

The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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