Updating, StemCells (STEM)
STEM’s preclinical research has shown that HuCNS-SC cells can be directly transplanted in the central nervous system.
- The transplanted cells are able to engraft, migrate, differentiate into neurons and glial cells, and possess the ability to survive long term with no sign of tumor formation or adverse effects;
- Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, this suggests the possibility of a durable clinical effect following a single transplantation;
- Preclinical studies performed by STEM and its collaborators provide a rationale for potential therapeutic use of HuCNS-SC cells in myelination disorders.
STEM has completed a Phase I clinical trial of its HuCNS-SC cells for the treatment of Neuronal Ceroid Lipofuscinosis (NCL, also often referred to as Batten disease), a fatal brain disorder in children.
- Data from this trial demonstrated the clinical safety and tolerability of these cells;
- STEM plans to initiate a 2nd clinical trial in NCL during Q4/10;
- HuCNS-SC cells are also in preclinical development for other central nervous system disorders, including spinal cord injury and age-related macular degeneration.
STEM has demonstrated that, when transplanted into an animal model of hypomyelination (shiverer mouse), HuCNS-SC cells engraft and differentiate into mature, specialized cells called oligodendrocytes, and form myelin sheaths around host nerve fibers.