NEWARK, Calif., April 18, 2013 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced the addition of the Byers Eye Institute at Stanford, located in Palo Alto, Calif., as a second site for the Company's Phase I/II clinical trial of its proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD). AMD is the leading cause of vision loss and blindness in people over 55 years of age, and approximately 30 million people worldwide are afflicted with the disease. Approximately 90 percent of AMD patients have the dry form of the disease and there are no approved treatments for dry AMD.
The Byers Eye Institute at Stanford, which is part of Stanford Hospital & Clinics, is dedicated to combating blindness and preserving sight. The Institute leverages the research and teaching strengths of Stanford and integrates all vision care services at Stanford into one state-of-the-art facility. Theodore Leng, MD, FACS, clinical assistant professor in ophthalmology at the Stanford University School of Medicine, is the lead investigator at the site. Stanford's Department of Ophthalmology is a nationally acclaimed leader for treatment of retinal diseases, refractive disorders, neuro-ophthalmic disorders and diseases of the vitrea.
"We are excited to be a part of this groundbreaking clinical trial for macular degeneration," said Dr. Leng. "The Company's preclinical data indicates that transplanting neural stem cells to protect photoreceptors may prove to be a viable approach to this debilitating disease. That data provides a very strong rationale for this innovative cell therapy trial."
"The clinical strategy with our neural stem cells is to preserve visual function before it is lost," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Our published preclinical data supports this approach in dry AMD and we hope to replicate those results in this clinical trial. We are very happy to be adding the Byers Eye Institute at Stanford and their participation is expected to accelerate patient enrollment for this trial."
The Company initiated the Phase I/II clinical trial last year at the Retina Foundation of the Southwest (RFSW) in Dallas, Texas. A summary of the Company's preclinical data underlying the trial was featured in the February 2012 issue of the international peer-reviewed European Journal of Neuroscience (available at http://onlinelibrary.wiley.com/doi/10.1111/j.1460-9568.2011.07970.x/abstract). The data demonstrated that HuCNS-SC cells protect host photoreceptors and preserve vision in the Royal College of Surgeons (RCS) rat, a well-established animal model of retinal disease that has been used extensively to evaluate potential cellular therapies. Transplantation of HuCNS-SC cells into RCS rats significantly protected photoreceptors from degeneration. Moreover, the number of cone photoreceptors, which are responsible for central vision, remained constant over an extended period, consistent with the sustained visual acuity and light sensitivity observed in the study. In humans, degeneration of the cone photoreceptors accounts for the unique pattern of vision loss in dry AMD.
About Age-Related Macular Degeneration (AMD)
Age-related macular degeneration (AMD) refers to a loss of photoreceptors (rods and cones) from the macula, the central part of the retina. AMD is a degenerative retinal disease that typically strikes adults in their 50's or 60's, and progresses until central vision is lost. There are approximately 1.75 million Americans age 40 years and older with some form of AMD, and the disease continues to be the number one cause of irreversible vision loss among senior citizens in the United States with more than seven million at risk of developing AMD.
About the Clinical Trial
The Phase I/II trial is evaluating the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry age-related macular degeneration (AMD). The trial is an open-label, dose-escalation study, and is expected to enroll a total of 16 patients. The HuCNS-SC cells will be administered by a single injection into the space beneath the retina in the most affected eye. Patients' vision will be evaluated using both conventional and advanced state-of-the-art methods of ophthalmological assessment. Evaluations will be performed at predetermined intervals over a one-year period to assess safety and signs of visual benefit. Patients will then be followed for an additional four years in a separate observational study. Patients interested in participating in the clinical trial should contact the Byers Eye Institute at Stanford at (650) 498-4486 or the Retina Foundation of the Southwest at (214) 363-3911.
About HuCNS-SC Cells
StemCells' lead product candidate, HuCNS-SC cells, is a highly purified composition of human neural stem cells that are expanded and stored as banks of cells. The Company's preclinical research has shown that HuCNS-SC cells can be directly transplanted in the central nervous system (CNS) with no sign of tumor formation or adverse effects. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, this suggests the possibility of a durable clinical effect following a single transplantation. StemCells believes that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS, and to date has demonstrated human safety data from completed and ongoing clinical studies.
About Stanford Hospital & Clinics
Stanford Hospital & Clinics is dedicated to providing leading edge and coordinated care to each and every patient. It is internationally renowned for expertise in areas such as cancer treatment, neuroscience, surgery, cardiovascular medicine and organ transplant, as well as for translating medical breakthroughs into patient care. Throughout its history, Stanford has been at the forefront of discovery and innovation, as researchers and clinicians work together to improve health on a global level. Stanford Hospital & Clinics: Healing humanity through science and compassion, one patient at a time. For more information, visit www.stanfordhospital.org.
About StemCells, Inc.
StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. The Company's lead therapeutic product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in development as a potential treatment for a broad range of central nervous system disorders. In a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, the Company has shown preliminary evidence of progressive and durable donor-derived myelination in all four patients transplanted with HuCNS-SC cells. The Company is also conducting a Phase I/II clinical trial in chronic spinal cord injury in Switzerland and recently reported positive data for the first patient cohort. The Company is also conducting a Phase I/II clinical trial in dry age-related macular degeneration (AMD), and is pursuing preclinical studies in Alzheimer's disease. StemCells also markets stem cell research products, including media and reagents, under the SC Proven(R) brand. Further information about StemCells is available at http://www.stemcellsinc.com.
Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the Securities Act of 1933, as amended, and the Securities Exchange Act of 1934, as amended, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the prospect of the Company's HuCNS-SC cells to preserve vision; the prospect and timing of patient enrollment in the Company's clinical trial in dry AMD; the potential of the Company's HuCNS-SC cells to treat a broad range of central nervous system disorders; and the future business operations of the Company, including its ability to conduct clinical trials as well as its other research and product development efforts. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including the fact that additional trials will be required to demonstrate the safety and efficacy of the Company's HuCNS-SC cells for the treatment of any disease or disorder; uncertainty as to whether the results of the Company's preclinical studies in retinal disease will be replicated in humans; uncertainty as to whether the FDA or other applicable regulatory agencies or review boards will permit the Company to continue clinical testing in spinal cord injury, PMD, AMD, or in future clinical trials of proposed therapies for other diseases or conditions given the novel and unproven nature of the Company's technologies; uncertainties regarding the timing and duration of any clinical trials; uncertainties regarding the Company's ability to recruit the patients required to conduct its clinical trials or to obtain meaningful results; uncertainties regarding the Company's ability to obtain the increased capital resources needed to continue its current and planned research and development operations; uncertainty as to whether HuCNS-SC cells and any products that may be generated in the future in the Company's cell-based programs will prove safe and clinically effective and not cause tumors or other adverse side effects; uncertainties regarding the Company's ability to commercialize a therapeutic product and its ability to successfully compete with other products on the market; and other factors that are described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2012, and in its subsequent reports on Forms 10-Q and 8-K.
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