Stem cells tested as dry AMD treatment

Newark, CA-StemCells Inc. has begun a phase I/II clinical trial of the company’s proprietary purified human neural stem cells product candidate (HuCNS-SC) for dry age-related macular degeneration (AMD), referred to as geographic atrophy, for which no treatments are approved.

“Dry AMD . . . has a very debilitating effect on quality of life,” said David G. Birch, PhD, chief scientific and executive officer of the Retina Foundation of the Southwest (RFSW), where the trial is being conducted. The principal investigator of the study, he also directs the Rose-Silverthorne Retinal Degenerations Laboratory.

“Transplanting neural stem cells to protect photoreceptors in patients [in whom AMD is] diagnosed . . . is an innovative, but logical approach, well supported by the company’s recently published preclinical data,” Dr. Birch added. “We are very excited to be conducting this trial at RFSW.”

A summary of the company’s preclinical data was published in the February issue of the European Journal of Neuroscience (http://onlinelibrary.wiley.com/doi/10.1111/j.1460-9568.2011.07970.x/abstract). The cells were found to protect host photoreceptors and preserve vision in the Royal College of Surgeons rat, an established animal model of retinal disease that has been used extensively to evaluate potential cell therapies. Transplantation of the cells 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.

“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 central nervous system program at StemCells Inc. “Our published preclinical data provide a strong rationale for this approach in dry AMD, and we hope to replicate these results in this clinical trial.”

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