StemCells, Inc. announced the addition of the Byers Eye Institute at Stanford, located in Palo Alto, Calif., as a second site for the company's phase 1/2 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% 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 https://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.