MARLBOROUGH, Mass., June 16, 2011 /PRNewswire/ -- Advanced Cell Technology, Inc. (“ACT"; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today the enrollment of the first patients in its two Phase 1/2 clinical trials for Stargardt’s Macular Dystrophy (SMD) and Dry Age-Related Macular Degeneration (Dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patients were enrolled at the Jules Stein Eye Institute at the University of California, Los Angeles (UCLA).
The Phase 1/2 trials are prospective, open-label studies primarily designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with SMD and Dry AMD. Each study will enroll 12 patients with cohorts of three patients in an ascending dosage format. The primary endpoint of both studies is to determine the safety and tolerability of hESC-derived RPE cells at 12 months.
“The enrollment of the first patients in our two clinical trials marks an important step forward for the field of regenerative medicine,” said Gary Rabin, interim chairman and CEO of ACT. “We are very pleased with the progress that has been made toward bringing this ground-breaking technology to the patients who need it most. If these therapies work as we hope they will, particularly with small volumes of cells, then we should be in an excellent position to take advantage of our patented techniques for manufacturing large numbers of doses of RPE cells that can be conveniently stored and shipped to clinicians following the basic manufacturing and distribution systems already familiar to pharmaceutical and biotech companies.”
Principal investigator Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at the Jules Stein Eye Institute at UCLA, said, “These trials mark a significant step toward addressing what is one of the largest unmet medical needs of our time -- treatments for otherwise untreatable and common forms of legal blindness, Dry AMD, SMD and other forms of atrophic macular degeneration. Dry AMD is the most common form of macular degeneration. It is the leading cause of blindness in the developed world, and is the leading cause of blindness in people over the age of 55. The incidence of Dry AMD is expected to double over the next 20 years as the population ages. This trial will begin the process of understanding whether stem cell-derived RPE cells have the potential to be a safe and effective treatment for these debilitating diseases. We are looking forward to evaluating the safety and tolerability data of these Phase 1/2 trials, and hope that these early trials will also produce key information relating to engraftment and function of the transplanted RPE cells.”
The progress of disease in both SMD and Dry AMD includes atrophy or thinning of the layer of RPE cells in the patient’s 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 photoreceptors. Over time, the progressive loss of RPE cells and concomitant loss of photoreceptors can cause severe central visual deterioration and even blindness as the macula becomes less functional and central vision is gradually lost. ACT’s SMD and Dry AMD therapeutic programs utilize transplanted RPE cells to treat these conditions by replacing RPE cells in the patient’s eyes before all RPE function is lost.
“Initiating these two clinical trials represents an important milestone for embryonic stem cell research,” said Robert Lanza, M.D., chief scientific officer of ACT. “After a decade of extensive research and preclinical studies, it is very satisfying to finally be moving into the clinic. We hope that these cells will, in the future, provide a treatment not only for these two untreatable diseases Stargardt’s disease and macular degeneration but for patients suffering from a range other debilitating eye diseases.”
In addition to the Jules Stein Eye Institute at UCLA, the Casey Eye Institute (CEI) at Oregon Health & Science University (OHSU) in Portland, OR, is also open for enrollment of patients with SMD. As additional sites are ready to enroll patients with SMD and dry AMD, they will be listed on the Clinical Trials page on ACT’s Web site and on www.clinicaltrials.gov.
Further information about patient eligibility for the SMD and the Dry AMD studies are also available on www.clinicaltrials.gov.
About hESC-RPE Cells
The retinal pigment epithelium (RPE) is a highly specialized tissue that is located between the choroids and the neural retina. RPE cells support, protect and provide nutrition for the light sensitive photoreceptors. Human embryonic stem cells (hESCs) can differentiate into any cell type, including RPE cells. hESC-RPE cells 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. SMD 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 or RPE cell layer.
Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. 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.
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.
About the Jules Stein Eye Institute
Established in 1966, the Jules Stein Eye Institute represents the culmination of a dream shared by ophthalmologist, businessman and philanthropist Dr. Jules Stein and his wife, Doris, of creating a world-renowned center dedicated to the preservation of vision and the prevention of blindness. The Institute’s comprehensive programs for the care of patients with eye disorders, research in the vision sciences, education in the field of ophthalmology and outreach to the community, coupled with its state-of-the art facilities, have brought national and international recognition to UCLA and the Institute, as it continues its mission to advance ophthalmology worldwide.
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.
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SOURCE Advanced Cell Technology, Inc.
