Retinal Disorders / AMD: Pre-clinical Studies
Published pre-clinical data demonstrates the therapeutic potential of the StemCells HuCNS-SC® (purified human neural stem cells) platform technology to treat retinal degenerative diseases such as age-related macular degeneration (AMD).
StemCells human neural stem cells preserve visual acuity in RCS rats as shown by optokinetic tests measuring visual function over time.
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Extensive pre-clinical studies performed by StemCells, Inc. in collaboration with the Casey Eye Institute at Oregon Health & Science University (OHSU) show that, when transplanted into the sub-retinal space of the RCS (Royal College of Surgeons) rat, a well-established animal model of retinal degeneration, the Company’s human neural stem cells protect the retina from progressive degeneration. The observation that a single HuCNS-SC transplant preserves rod and cone photoreceptors and provides long-term functional benefit strongly supports human testing in patients suffering from vision loss due to photoreceptor degeneration.
The transplanted cells exhibit robust, long-term protection of both rod and cone photoreceptors, as well as the preservation of visual function long term, as measured by two separate visual tests. The ability to protect cones, in particular, is significant in regard to AMD, since it is the progressive deterioration of these specific cells that ultimately results in the devastating vision loss caused by this disease. The protection of both rods and cones is important in considering the potential of using human neural stem cells as a treatment for retinitis pigmentosa and other retinal degenerative disorders.
Data indicates that the neuroprotective transplantation of HuCNS-SC cells results in the stabilization of photoreceptor degeneration and slowing of progressive vision loss. This is relevant particularly in the setting of the slow rate of changes observed in the natural history of dry AMD compared to other diseases.
A more recent study, the results of which were published in September 2013, showed not only that HuCNS-SC cells preserve the number of photoreceptors that would otherwise be lost, but also that the surviving photoreceptors appear healthy and normal, and maintain their synaptic connection to other important cells necessary for visual function.
These effects potentially could translate into rescue of visual acuity and improvement of visual function in patients, such as peripheral, night, and color vision, as well as enhanced reading speed, facial recognition, mobility and overall quality of life.
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