A U.S.-based company has received permission to start Europe's
first clinical trial involving human embryonic stem (hES) cells.
Advanced Cell
Technology (ACT), based in Marlborough, Massachusetts, received
approval today from the U.K. Medicines and Healthcare products
Regulatory Agency (MHRA)
to begin a trial that will treat 12 patients with Stargardt's
macular dystrophy. The disease strikes people between the ages of 10 and
20, causing
progressive vision loss. There is currently no treatment.
Eye surgeon James Bainbridge of Moorfields Eye Hospital and
University College London will lead the U.K. trial. He and his
colleagues will inject into
patients' eyes retinal pigment epithelial cells that ACT derived
from hES cells. In animal models, the cells have been able to stave off
or even
reverse disease progression. The phase I/II trial will primarily
examine the treatment's safety, however.
Robert Lanza, ACT's chief scientific officer, says the trial has
essentially the same design as the company's U.S. trial with
Stargardt's patients,
which received approval last year.
The fact
that the U.S. Food and Drug Administration had approved the
trial, however, did not make it a shoo-in with U.K. authorities, Lanza
says. "I thought it
was going to sail right through. I was wrong; they were very
rigorous."
The new trial is the first one using hES cells to receive
approval outside the United States, Lanza says. The company plans to
apply for approval for
a second U.K. trial for age-related macular degeneration, he
adds, which would complement its U.S.-based trial for the same
condition. They then plan
to wait until the first results are in before starting any
additional trials, he says. The first two U.S. patients received the
company's cells in July—one patient with Stargardt's disease and one
with age-related macular degeneration. The first indications are
positive, Lanza says. "We're very
pleased with what we see so far."
