StemCells, Inc. has announced initiation of a Phase I/II clinical trial of the Company's proprietary HuCNS-SC® 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."