Advanced Cell Technology Receives FDA Clearance For the First Clinical Trial
Using Embryonic Stem Cells to Treat Macular Degeneration
FDA Lifts Clinical Hold; Company to Commence a Phase I/II Clinical Trial at
Multiple Centers
MARLBOROUGH, Mass., Nov 22, 2010 (BUSINESS WIRE) -- Advanced Cell Technology,
Inc. ("ACT"; OTCBB:ACTC) announced today that the US Food and Drug Administration
(FDA) has cleared the Company's Investigational New Drug (IND) application to
immediately initiate a Phase I/II multicenter clinical trial using retinal cells
derived from human embryonic stem cells (hESCs) to treat patients with
Stargardt's Macular Dystrophy (SMD), one of the most common forms of juvenile
macular degeneration in the world. The decision removes the clinical hold that
the FDA had placed on the trial.
Stargardt's Macular Dystrophy causes progressive vision loss, usually starting in
children 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 (RPE). . "There is currently no
treatment for Stargardt's disease," said Dr. Robert Lanza, ACT's Chief Scientific
Officer. "Using stem cells, we can generate a virtually unlimited supply of
healthy RPE cells, which are the first cells to die off in SMD and other forms of
macular degeneration. We've tested these cells in animal models of eye disease.
In rats, we've seen 100% improvement in visual performance over untreated animals
without any adverse effects. Our studies showed that the cells were capable of
extensive rescue of photoreceptors in animals that otherwise would have gone
blind. Near-normal function was also achieved in a mouse model of Stargardt's
disease. We hope to see a similar benefit in patients with various forms of
macular degeneration."
The Phase I/II trial will be a prospective, open-label study that is designed to
determine the safety and tolerability of the RPE cells following sub-retinal
transplantation to patients with advanced SMD. A total of twelve patients will be
enrolled in the study at multiple clinical sites. The sites which are currently
under consideration are the Jules Stein Eye Institute at UCLA (headed by Dr.
Steven Schwartz); the Casey Eye Institute in Portland, Oregon (headed by Dr.
Peter Francis of the Oregon Health Sciences University); the University of
Massachusetts Memorial Medical Center in Worcester, Massachusetts (headed by Dr.
Shalesh Kaushal, Chair of the Department of Ophthalmology); the UMDNJ -- New
Jersey Medical School in Newark, New Jersey (headed by Dr. Marco Zarbin, Chair,
Institute of Ophthalmology and Visual Science); additional sites may be
considered.
Earlier this year the FDA also granted Orphan Drug designation for the company's
RPE cells. As a result, the company is eligible to receive a number of benefits,
including tax credits, access to grant funding for clinical trials such as this
one, accelerated FDA approval, and allowance for marketing exclusivity after drug
approval for a period of as long as seven years.
Raymond Lund, Ph.D., a scientific collaborator with ACT, and considered one of
the world's foremost experts in retinal cell physiology and vision restoration,
commented, "The study results of ACT's RPE cells implanted in the various animal
models of macular degeneration was phenomenal. If ACT observes even a fraction of
that benefit in humans, it will be nothing short of a home run."
"Initiating our macular degeneration clinical trial represents a significant
milestone in the progress of developing human embryonic stem cell-based therapies
aimed at large worldwide markets," said William M. Caldwell IV, ACT's Chairman
and CEO. "I think generations will look back at this time as one of the most
exciting in the history of medicine. With the initiation of this clinical trial,
and that of Geron's earlier this fall, the field of regenerative medicine is
poised to take embryonic stem cell therapies from the realm of nebulous potential
to that of tangible and real treatments that will make a significant difference
in the lives of millions of people worldwide. This is truly a 'game changer' for
the medical community."
Among the most common causes of untreatable blindness in the world are
degenerative diseases of the retina. As many as thirty 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 the "dry" form of AMD -- which is currently
untreatable. The prevalence increases to 30% in patients 75 to 85 years of age.
"These patient numbers are staggering in size," stated Edmund Mickunas, ACT's
Vice President of Regulatory, "and when the impact on health and quality-of-life
is considered in that context, macular degeneration represents one of the more
significant unmet medical needs in our society. With the momentum of this
Stargardt's trial, and the unique experience we have gained as one of the few
companies in the world having succeeded in taking an hESC program into the
clinic, we are preparing to extend our lead with an IND in the use for treating
age-related macular degeneration, as well as filing to begin clinical trials in
Europe, in the very near future."
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
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future growth in research and development programs, potential applications of our
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also be considered to be forward-looking statements. There are a number of
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from those indicated by such forward-looking statements, including: limited
operating history, need for future capital, risks inherent in the development and
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that could affect our results and other risks and uncertainties are detailed from
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SOURCE: Advanced Cell Technology, Inc.
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