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).

Pre-clinical Results

StemCells human neural stem cells preserve visual acuity in RCS rats as shown by optokinetic tests measuring visual function over time.

Download PDF of published research; see page 3, Figure A…

Spacial Frequency (cycles per degree) 0.6  NCL Chart Data
  Age (days) p‹0.001
  • ■  Non-diseased (normal)
  • ■  Diseased: With neural stem cells transplanted
  • ■  Diseased: With cell medium (only) transplanted
  • ■  Diseased: No transplantation

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.

Learn about our AMD clinical trials…

View our publications on retinal disorders…

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About AMD

AMD is the number one cause of legal blindness for those over age 55, and the leading cause of vision loss in developed countries. There is currently no cure for AMD.

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Clinical Program

Based upon the strength of both our pre-clinical studies and the interim data from our Phase I/II clinical trial in geographic atrophy of age-related macular degeneration (GA-AMD), StemCells, Inc. is initiating a Phase II proof of concept clinical trial using our proprietary HuCNS-SC® human neural stem cells for the treatment of GA-AMD.

Learn about our AMD clinical trials…


Findings from studies performed by StemCells, Inc. and its collaborators on the use of human neural stem cells for the treatment of retinal disorders have been published.

View our publications on retinal disorders…