June 21, 2012
StemCells, Inc. Initiates Phase I/II Clinical Trial in Dry Age-Related Macular Degeneration
StemCells, Inc. (STEM) today announced initiation of a Phase I/II clinical trial of the Company’s proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD) referred to as Geographic Atrophy. There are no approved treatments for dry AMD.
The trial is being conducted at the Retina Foundation of the Southwest’s (RFSW) Anderson Vision Research Center in Dallas, Texas, one of the leading independent vision research centers in the United States. David G. Birch, Ph.D., Chief Scientific and Executive Officer of the RFSW and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, is the principal investigator of the study.
“Dry AMD is the most common form of macular degeneration, and has a very debilitating effect on quality of life,” said Dr. Birch. “Transplanting neural stem cells to protect photoreceptors in patients diagnosed with AMD is an innovative, but logical, approach, well supported by the Company’s recently published preclinical data. We are very excited to be conducting this trial at RFSW.”
A summary of the Company’s preclinical data was featured in the February 2012 issue of the international peer-reviewed European Journal of Neuroscience (available online 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 which has been used extensively to evaluate potential cell therapies. Transplantation of HuCNS-SC cells significantly protects 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.
“Unlike others in the field, our clinical strategy 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 provides a strong rationale for this approach in dry AMD and we hope to replicate these results in this clinical trial. We are very pleased to be working with Dr. Birch and the Retina Foundation of the Southwest, who have the expertise and referral base to undertake this important study. We anticipate that we will be able to accrue the requisite number of patients for this trial in relatively short order.”
About Age-Related Macular Degeneration
Age-related macular degeneration 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 50s or early 60s, and progresses painlessly, gradually destroying central vision. According to the RFSW website, there are approximately 1.75 million Americans age 40 years and older with some form of age-related macular degeneration, and the disease continues to be the number one cause of irreversible vision loss among senior citizens in the US with more than seven million at risk of developing AMD.
About the Trial
The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The trial will be 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 site 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 The Retina Foundation of the Southwest
The Retina Foundation of the Southwest is an independent, non-profit research organization located in Dallas, Texas. The Foundation opened its Anderson Vision Research Center in 1982, which has grown into one of the leading independent vision research centers in the United States, with a staff dedicated to finding the causes, treatments and potential cures for blinding eye diseases. Along the way, the Foundation has made a life changing difference in the lives of patients who suffer from retina related diseases. Patients are referred by ophthalmologists from around the world, and because the center is funded by philanthropic and federal grants, patient services are entirely free. The Foundation is a volunteer-driven organization dedicated to making a difference in people’s lives by improving their vision — no matter what their age or financial status. Scientists and clinicians at the Foundation use the power of modern technology to understand the intricacy of retinal function and conduct studies of hereditary and acquired blinding retinal disorders, including retinitis pigmentosa and age-related macular degeneration. Other studies address eye disorders affecting the sight of infants and children, including hereditary eye diseases, cataracts, prematurity and the consequences of eye muscle imbalance. Further information about the RFSW is available at http://www.retinafoundation.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 March 2012, the Company reported results from a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children. The trial results showed 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 interim safety data for the first patient cohort. The Company has also initiated a Phase I/II clinical trial in dry age-related macular degeneration (AMD). In addition, the Company is pursuing preclinical studies of its HuCNS-SC cells 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.
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