* Trial of patients with Stargardt's macular dystrophy
* Researchers can watch cells in eye in real time
(Adds details of potential macular degeneration market,
By Maggie Fox, Health and Science Editor
WASHINGTON, Nov 22 The U.S. Food and Drug
Administration has approved the second human trial of human
embryonic stem cells -- this one testing cells in people with a
progressive form of blindness, the company said on Monday.
Massachusetts-based Advanced Cell Technology ACTC.OB said
it would start testing its stem cell-based treatment on 12
patients with Stargardt's macular dystrophy.
It is the second trial of human embryonic stem cells to be
approved by the FDA this year. Last month Geron Corp (GERN.O)
enrolled the first patient in its study using the cells in
people whose spinal cords have been crushed. [ID:nN11175966]
"It is exciting -- a vindication. All this work really came
through," said Dr. Robert Lanza, chief medical officer of the
company, which has struggled to stay solvent as it gambled on
the controversial cells.
Stem cells are the body's master cells, the source of all
other cells. Proponents of using embryonic stem cells say the
field could transform medicine, providing treatments for
blindness, juvenile diabetes or severe injuries.
But opponents object because to get the cells, someone has
to take apart a human embryo.
Last year the Obama administration overturned the strictest
of the limitations on using federal funds for the research, but
this summer, two researchers challenged the policy.
A U.S. appeals court has ruled that funding could continue
while the government appeals, but grants from the National
Institutes of Health have been frozen and unfrozen as various
courts have weighed in. [ID:nN28118865]
Stargardt's disease causes progressive vision loss, usually
starting in children or young adults 10 to 20 years old, as eye
tissue called the retinal pigment epithelium, or RPE,
There is currently no treatment for the condition.
ACT has coaxed human embryonic stem cells into becoming RPE
cells, which will be infused into patients' eyes.
"We can generate a virtually unlimited supply of healthy
RPE cells," Lanza said.
If all goes well with the Stargardt's trial, Lanza says ACT
has its eyes on a much larger potential market -- macular
Up to 30 million people in the United States and Europe
have the blinding condition, which ACT sees as a potential
$25 billion-$30 billion worldwide market.
Lanza said getting FDA approval for the trial was
difficult. "They had us jumping through hoops," he said.
This was in part because the powerful embryonic cells have
the power to give rise to all cell types and can cause
teratomas -- strange tumors containing a mix of cells. Lanza
said the company can find even a single cell that might do this
and remove it from the treatment batch.
In addition, he said treating an eye disease offers the
opportunity to watch the treatment.
"We can look into the eye in real time and see what is
going on," he said.
Research centers at the Oregon Health and Sciences
University and University of Massachusetts will begin enrolling
patients after their internal review boards approve the trial.
"My guess is that we could start in as soon as two to three
months," Lanza said.
(Editing by Cynthia Osterman)