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Phase 1/2 trial of neural SCs for dry age-related macular degeneration.


Newark, Calif.-based StemCells, Inc. (STEM) on June 21 announced the start of a Phase 1/2 clinical trial of its purified human neural stem cells product in dry age-related macular degeneration (AMD,) referred to as geographic atrophy.

There are no approved treatments for dry AMD.

The trial of the company's proprietary HuCNS-SC product candidate is being conducted at the Retina Foundation of the Southwest's (RFSW) Anderson Vision Research Center in Dallas, Texas.

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," Birch said. "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 pre-clinical data."

Data from a recent preclinical study 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, M.D., of StemCells. "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."

The Phase 1/2 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 ophthalmologic^ 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.

HuCNS-SC is a highly purified composition of human neural stem cells that are expanded and stored as banks of cells.


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Title Annotation:In The Clinic ...
Publication:Stem Cell Business News
Date:Jul 2, 2012
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