ACT Receives Approval for First Human Embryonic Stem Cell Trial in Europe
Moorfields Eye Hospital in London is Site for Phase 1/2 Trial to Treat
Stargardt's Macular Dystrophy
MARLBOROUGH, Mass., Sep 22, 2011 (BUSINESS WIRE) -- Advanced Cell Technology,
Inc. ("ACT");(ACTC), a leader in the field of regenerative medicine, announced
today that it has received clearance from the U.K. Medicines and Healthcare
products Regulatory Agency (MHRA) to begin treating patients as part of a Phase
1/2 clinical trial for Stargardt's Macular Dystrophy (SMD) using retinal pigment
epithelium (RPE) derived from human embryonic stem cells (hESCs). ACT received
similar approval from the Gene Therapy Advisory Committee (GTAC), which has
responsibility for the ethical oversight of proposals to conduct clinical trials
involving gene or stem cell therapies in the U.K. The European Medicines Agency
(EMA) previously granted Orphan Drug designation for the company's RPE cell
product for use in treating SMD.
"This is another important milestone for ACT and for the field of regenerative
medicine," said Gary Rabin, chairman and CEO of ACT. "We are pleased that the
Moorfields Eye Hospital in London has agreed to participate as a site for this
study as we continue to assess the capabilities of hESC-derived RPE cells to
repair the retina and reduce the impact of these devastating eye diseases. We
recently announced the dosing of the first patients in our Phase 1/2 clinical
trials for Stargardt's macular dystrophy and dry age-related macular degeneration
(dry AMD) with hESC-derived RPE cells in the U.S., and both patients successfully
underwent the outpatient transplantation surgeries. Clearance from the MHRA to
begin an SMD trial in the U.K. is the first step in our European clinical trial
program. Europe not only represents the world's second-largest pharmaceutical
market, but it is also home to some of the best eye hospitals and surgeons in the
world. Building international relationships around our clinical programs, such as
with Professor James Bainbridge at Moorfields Eye Hospital is very important to
our strategy of developing new regenerative medicine therapies."
Stargardt's Macular Dystrophy affects an estimated 80,000 to 100,000 patients in
the U.S. and Europe, and causes progressive vision loss, usually starting in
people between the ages of 10 to 20. Eventually, blindness results from
photoreceptor loss associated with degeneration in the pigmented layer of the
retina, the retinal pigment epithelium. The first patient to be treated in the
U.S. with stem cell-derived RPE cells was a young woman who was already legally
blind as a consequence of this disease. This newly-approved clinical trial in
Europe will be a prospective, open-label study designed to determine the safety
and tolerability of RPE cells derived from hESCs following sub-retinal
transplantation to patients with advanced SMD, and it is similar in design to the
FDA-cleared U.S. trial initiated in July.
"This is the first time an embryonic stem cell trial has ever been approved
anywhere else in the world," said Robert Lanza, M.D., ACT's chief scientific
officer. "Stargardt's disease is currently untreatable, and is one of the leading
causes of juvenile blindness in the world. Collectively, degenerative eye
diseases afflict over 25 million people in the U.S. and Europe alone. These
diseases have a devastating impact on patients and their families, which has been
a strong motivating factor for developing this new treatment. In Stargardt's
disease, the loss of RPE cells in the patient's macula causes a loss of
photoreceptors -- the cones and rods with which we see -- leading to blindness.
We believe that transplanting new, healthy RPE cells may provide an effective
treatment for SMD and perhaps other macular degenerative diseases such as dry
AMD. We are excited to start these trials in Europe, and look forward to
analyzing the data we continue to collect in our ongoing trials to determine the
engraftment and function of the transplanted RPE cells."
The trial will be led by Professor James Bainbridge, consultant surgeon at
Moorfields Eye Hospital, and Chair of Retinal Studies at University College
London.
"Stargardt's disease is a form of macular degeneration that causes disabling loss
of sight in young people and is currently untreatable," said Professor
Bainbridge. "There is real potential that people with blinding disorders of the
retina including Stargardt's disease and age-related macular degeneration might
benefit in the future from transplantation of retinal cells. The ability to
generate retinal cells from stem cells in the laboratory has been a significant
advance and the opportunity to help translate such technology into new treatments
for patients is hugely exciting. Testing the safety of retinal cell
transplantation in this clinical trial will be an important step towards
achieving this aim."
About Macular Degeneration and SMD
Degenerative diseases of the retina are among the most common causes of
untreatable blindness in the world. As many as 30 million people in the U.S. and
Europe suffer from macular degeneration, which represents a $25-30 billion
worldwide market that has yet to be effectively addressed.
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. is a biotechnology company applying cellular
technology in the field of regenerative medicine. For more information, visit
advancedcell.com. |
