ACT Announces Europe's First Human Embryonic Stem Cell Transplant in Patient with
Stargardt's Disease
Moorfields Eye Hospital in London Initiates Clinical Trial Using hESC-derived RPE
cells
MARLBOROUGH, Mass., Jan 23, 2012 (BUSINESS WIRE) -- Advanced Cell Technology,
Inc. ("ACT"; OTCBB: ACTC), a leader in the field of regenerative medicine, today
announced treatment of the first patient in its Phase 1/2 clinical trial for
Stargardt's macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells
derived from human embryonic stem cells (hESCs). The surgery was performed on
Friday, Jan. 20, at the Moorfields Eye Hospital in London by a team of surgeons
led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of
Retinal Studies at University College London. The patient successfully underwent
the procedure without any complications. ACT and Moorfields Eye Hospital received
clearance in September from the U.K.'s Medicines and Healthcare products
Regulatory Agency (MHRA) to begin this trial in Europe.
"Our clinical trial program for hESC-derived RPE cells has taken another critical
step as we move forward with treating patients at Moorfields Eye Hospital," said
Gary Rabin, chairman and chief executive officer of ACT. "The treatment of the
first patient in Europe is tangible evidence that stem cell research and
development of cell therapies is making progress. It is a milestone for
scientists, stem cell advocates and patients hoping for cures as well as much as
it is one for ACT. Stargardt's macular dystrophy affects up to 100,000 patients
in Europe and North America, and causes progressive vision loss often ending with
blindness. We are honored to be working with Professor Bainbridge at Moorfields
Eye Hospital, and are very pleased with the smooth progress of the trial thus
far."
Professor James Bainbridge, the study's principal investigator, said, "The
patient, who is severely sight-impaired, underwent transplantation of fully
differentiated retinal pigment epithelial (RPE) cells derived from human
embryonic stem cells. There were no complications, and the patient has tolerated
the surgical procedure well. We will be regularly monitoring the safety and
tolerability of the transplanted cells. While this is primarily a safety trial,
we will have the opportunity to image engraftment of RPE cells non-invasively and
to assess any changes in sight. We are very excited to be working with ACT on the
first human embryonic stem cell trial in Europe. Stargardt's macular dystrophy is
a serious and progressive disease that usually starts between the age of 10 and
20 years. It includes degeneration of RPE cells in the macula at the center of
the retina, the region specialized for high acuity vision. With the loss of RPE
cells in the macula comes the eventual loss of light-sensitive photoreceptor
cells, leading to blindness at the prime of life. We hope that transplantation of
healthy RPE cells might also help in other significant degenerative eye diseases
affecting the retina for which there are no effective treatment options --
particularly dry age-related macular degeneration which is the leading cause of
blindness in Europe."
The Phase 1/2 trial will involve 12 patients, with cohorts of 3 patients each in
an ascending dosage format and is similar in design to the FDA-approved U.S.
trial that was initiated in July 2011. The open-label study is designed to
determine the safety and tolerability of hESC-derived RPE cells following
sub-retinal transplantation in patients with Stargardt's macular dystrophy at 12
months, the study's primary endpoint.
"Stem cells provide the possibility of providing new treatment strategies for
currently incurable retinal degenerative diseases," said Robert Lanza, M.D.,
chief scientific officer of ACT. "Although these initial studies are designed to
determine the safety and tolerability of hESC-derived RPE, we eventually hope to
treat patients earlier in the course of the disease, further increasing the
likelihood the new cells will rescue photoreceptors and prevent visual loss. We
recently initiated two clinical trials in the U.S. and are very pleased to be
starting the first clinical trial in Europe. To-date, our preclinical and
clinical data relating to the safety and effectiveness of this approach is very
encouraging. We look forward to working with Professor Bainbridge and Moorfields
Eye Hospital to obtain additional clinical data and to enroll further patients in
this trial. As a scientist, it is satisfying to see years of benchside research
finally moving into the clinic. We believe RPE is just the first of many
different vital differentiated cell types that may help patients suffering from a
wide spectrum of eye disorders caused by conditions ranging from diabetes to
vascular and autoimmune diseases."
In August 2011, the National Institute of Health Research (NIHR) announced the
award of Biomedical Research Centre (BRC) for Ophthalmology at Moorfields Eye
Hospital NHS Foundation Trust and UCL Institute of Ophthalmology. The award
provides 26.5 million pounds Sterling over five years to translate lab-based
discoveries into new cutting edge treatments, technologies, diagnostics and other
interventions in clinical settings.
About hESC-RPE Cells
The retinal pigment epithelium (RPE) is a highly specialized tissue located
between the choroids and the neural retina. RPE cells support, protect and
provide nutrition for the light-sensitive photoreceptors. Human embryonic stem
cells differentiate into any cell type, including RPE cells, -- and have a
similar expression of RPE-specific genes compared to human RPE cells and
demonstrate the full transition from the hESC state.
About SMD, Dry AMD and Degenerative Diseases of the Retina
Stargardt's macular dystrophy (SMD) is one of the most common forms of macular
degeneration in the world. Stargardt's causes progressive vision loss, usually
starting between 10 to 20 years of age. Eventually, blindness results from
photoreceptor loss associated with degeneration in the pigmented layer of the
retina, called the retinal pigment epithelium or RPE cell layer.
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 United
States and Europe suffer from macular degeneration, which represents a $25-30
billion worldwide market that has yet to be effectively addressed. Approximately
10% of people ages 66 to 74 will have symptoms of macular degeneration, the vast
majority suffering from the "dry" form of AMD -- which is currently untreatable.
The prevalence increases to 30% in patients 75 to 85 years of age.
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.
Forward-Looking Statements
Statements in this news release regarding future financial and operating results,
future growth in research and development programs, potential applications of our
technology, opportunities for the company and any other statements about the
future expectations, beliefs, goals, plans, or prospects expressed by management
constitute forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. Any statements that are not statements
of historical fact (including statements containing the words "will," "believes,"
"plans," "anticipates," "expects," "estimates," and similar expressions) should
also be considered to be forward-looking statements. There are a number of
important factors that could cause actual results or events to differ materially
from those indicated by such forward-looking statements, including: limited
operating history, need for future capital, risks inherent in the development and
commercialization of potential products, protection of our intellectual property,
and economic conditions generally. Additional information on potential factors
that could affect our results and other risks and uncertainties are detailed from
time to time in the company's periodic reports, including the report on Form 10-K
for the year ended December 31, 2010. Forward-looking statements are based on the
beliefs, opinions, and expectations of the company's management at the time they
are made, and the company does not assume any obligation to update its
forward-looking statements if those beliefs, opinions, expectations, or other
circumstances should change. Forward-looking statements are based on the beliefs,
opinions, and expectations of the company's management at the time they are made,
and the company does not assume any obligation to update its forward-looking
statements if those beliefs, opinions, expectations, or other circumstances
should change. There can be no assurance that the Company's clinical trials will
be successful.
SOURCE: Advanced Cell Technology, Inc.
Investors:
CEOcast, Inc.
James Young, 212-732-4300
or
Press:
Russo Partners
Martina Schwarzkopf, Ph.D., 212-845-4292 or 347-591-8785
martina.schwarzkopf@russopartnersllc.com
or
Tony Russo, Ph.D., 212-845-4251
tony.russo@russopartnersllc.com
or
ACT Corporate Communications
Bill Douglass, 646-450-3615
or
Business Development:
Advanced Cell Technology
Matthew Vincent, Ph.D., 508-756-1212 x324
mvincent@advancedcell.com |
