Advanced Cell Technology Receives FDA Clearance For the First Clinical Trial Using Embryonic Stem Cells to Treat Macular Dege...
November 22 2010 - 8:00AM
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 http://www.advancedcell.com.
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