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Last updated on April 18, 2014 at 17:24 EDT

StemCells Reports Promising Results From Human Neural Stem Cells Study

October 31, 2008

StemCells, a clinical-stage biotechnology company, has reported that its proprietary HuCNS-SC product candidate, when transplanted into a well-established animal model, can protect the retina from progressive degeneration.

This promising study was conducted by Raymond Lund, a researcher and professor at the Casey Eye Institute at Oregon Health & Science University (OHSU) and his research team.

In this preclinical study, Dr Lund and his co-investigator at OHSU, Peter Francis, transplanted HuCNS-SC cells (purified human neural stem cells) into the Royal College of Surgeons (RCS) rat, a well established animal model of retinal degeneration. In the RCS model, a genetic mutation causes dysfunction of the retinal pigmented cells.

Dysfunction in these cells, whose normal function is to support photoreceptors in the eye, causes progressive loss of the photoreceptors and degeneration of the retina, and ultimately, loss of visual function. Photoreceptor loss in the RCS rat begins as early as three weeks of age and by 24 weeks all photoreceptors are typically lost.

In the study, the researchers transplanted HuCNS-SC cells into one eye of 21-day-old RCS rats while keeping the opposite eye as the control. Animals were evaluated starting at day 40 (19 days post transplant) and then at routine intervals up to 150 days post transplant. The evaluations showed that the HuCNS-SC cells survived the transplants and engrafted, and the eyes transplanted with the cells showed preservation of the photoreceptors and stabilization of visual function.

Stephen Huhn, vice president and head of the CNS program at StemCells, said: “This study confirms the results of previously published academic studies evaluating neural stem cell transplantation into the retina and provides us with the rationale to pursue clinical testing of HuCNS-SC cells for retinal disorders.

“We are already conducting additional preclinical studies and a pre- investigational new drug (IND) meeting has been scheduled with the FDA in December 2008 to determine the pathway to a successful IND filing.”