Our Pre-clinical Studies

The work of StemCells, Inc. scientists has already generated the means to supply human neural stem cells (HuCNS-SC® cells), which, upon transplantation, can differentiate into healthy new cells or tissues, and which may thereby be capable of alleviating or potentially even curing a broad array of intractable conditions of the central nervous system. These cells have been studied extensively in our pre-clinical programs as prospective treatments for indications in all three components of the central nervous system:

The Eye — The introduction of healthy neural stem cells preserves vision, as shown in our pre-clinical studies in an animal model that replicates important aspects of AMD.

The Spinal Cord — When transplanted into the spinal cord of mice with lower spinal cord injury (thoracic cord trauma that results in hind limb paralysis), our human neural stem cells have been shown to form new myelin sheaths around damaged nerve axons, as well as new neurons, restoring lost motor function long-term.

The Brain — Transplanted neural stem cells appear to be able to replace dysfunctional cells, such as the oligodendrocytes needed to alleviate myelination disorders, such as PMD. Where impaired cellular function is associated with the progressive decline commonly seen in degenerative diseases like NCL, stem cells can deliver specialized cells that secrete, metabolize or regulate essential substances. Promising data in a study on Alzheimer’s disease indicates that when human neural stem cells are transplanted into the hippocampus of affected mice, the cells show consistent and robust engraftment, as well as evidence of a significant increase in the pre-and post-synaptic spines that provide an anatomical substrate for memory storage and synaptic transmission. We have also demonstrated that our human neural stem cells enhance functional recovery after stroke in rats.

In Vivo Veritas

A key enabling tool for advancing our cell-based therapeutics towards the clinic has been the establishment of representative in vivo models of disease with which to study the engraftment, migration, differentiation and function of human cells transplanted into rodents. We have amassed a comprehensive catalog of tissue samples from these rodents.


The Company's pre-clinical studies have shown that our HuCNS‑SC cells can:

  • Protect host neurons by providing a missing enzyme in models of lysosomal storage disease
  • Produce myelin in models of dysmyelination
  • Restore motor function in a spinal cord injury model
  • Protect photoreceptors and preserve visual function in models of retinal degeneration