Stemcells, Inc. Announces First High-Dose Patient Transplanted in Phase I/II Clinical Trial in Dry Age-Related Macular …

NEWARK, Calif., Sept. 12, 2013 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced dosing of the first high-dose patient in the Company's Phase I/II clinical trial in dry age-related macular degeneration (AMD). The patient, the fifth overall in the 16-patient trial, was transplanted yesterday with one million HuCNS-SC(R) cells (purified human neural stem cells). The first four patients each received a dose of 200,000 cells. An independent Data Safety Monitoring Committee conducted a review of the trial data to date, and found no safety issues to preclude the trial from proceeding to the high dose.

"Advancing to the high dose, which is a five-fold increase from the low dose, is an important milestone in this trial," said Stephen Huhn, MD, FACS, FAAP, Vice President, CNS Clinical Research at StemCells. "Testing a cell dose of this magnitude in all the remaining patients planned for the trial will enhance our ability to assess the effect of the cells on visual acuity.

"Also, we are pleased to announce that we have received permission from the FDA to open three more U.S. trial sites in addition to the Retina Foundation of the Southwest and the Byers Eye Institute at Stanford, the two currently active centers. Expanding the number of sites will provide easier access to the trial for patients and help us achieve our goal of completing enrollment within the next nine months."

AMD afflicts approximately 30 million people worldwide and is the leading cause of vision loss in people over 55 years of age. Approximately 90 percent of AMD patients have the dry form of the disease, for which there are no approved treatments.

About the Clinical Trial

The Phase I/II trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry 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. Using both conventional and advanced state-of-the-art methods of ophthalmological assessment, patients will be evaluated 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.

The trial is currently enrolling at the Byers Eye Institute at Stanford in Palo Alto, California, and at the Retina Foundation of the Southwest in Dallas, Texas. 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.

A summary of the Company's preclinical data underlying the trial was featured in February 2012 in the peer-reviewed European Journal of Neuroscience (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 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.

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Stemcells, Inc. Announces First High-Dose Patient Transplanted in Phase I/II Clinical Trial in Dry Age-Related Macular ...