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First Stem Cell Transplant in Myelination Disorder Trial, StemCells, Inc (NASDAQ: STEM)

STEM announced its proprietary HuCNS-SC® human neural stem cells have been used to treat the first patient enrolled in its Phase I clinical trial in Pelizaeus-Merzbacher Disease (NASDAQ:PMD), a myelination disorder that afflicts male children. The stem cells were administered at the University of California (UCSF) Children’s Hospital by direct injection into the brain of a patient with connatal PMD and the most severe form of the disease. This marks the first time that neural stem cells have been transplanted as a potential treatment for a myelination disorder, and the second clinical trial involving the use of HuCNS-SC cells in a neurodegenerative disease.

Myelin is the substance that surrounds and insulates nerve cells’ communications fibers (also known as axons).  Without sufficient myelination, these fibers are unable to properly transmit nerve impulses, leading to a progressive loss of neurological function.  Multiple sclerosis, transverse myelitis and certain types of cerebral palsy are more commonly known myelination disorders that also affect the central nervous system.  Patients with PMD are born with a defective gene, which leads to insufficient myelin in the brain. Those with the most severe form of the disease, connatal PMD, lose the ability to walk and talk and eventually die, often before the age of 10. Currently, there are no effective treatments for PMD.

  • The Phase I trial is designed to assess the safety and preliminary effectiveness of HuCNS-SC cells as a potential treatment for PMD,
  • The trial is expected to enroll 4 patients with connatal PMD. All patients will be transplanted with HuCNS-SC cells, and will be immunosuppressed for 9 months,
  • Following transplantation, the patients will be evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC cells, the surgery, and the immunosuppression,
  • Preclinical studies performed by StemCells and its collaborators provide a rationale for potential therapeutic use of HuCNS-SC cells in myelination disorders,
  • 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,
  • StemCells has completed a Phase I clinical trial of its HuCNS-SC cells for the treatment of Neuronal Ceroid Lipofuscinosis (NYSE:NCL), a fatal brain disorder in children. Data from this trial demonstrated the clinical safety and tolerability of these cells,
  • STEM’s HuCNS-SC cells are also in preclinical development for other central nervous system disorders, including retinal degenerative diseases, such as age-related macular degeneration and retinitis pigmentosa, and spinal cord injury.