A summary of the trial results will be presented on Saturday, 31-Mar, at the 2012 European Leukodystrophy Association (ELA) Families/Scientists Meeting in Paris:
- Webcast: Monday, 2-Apr, at 11:00ET
- http://investor.stemcellsinc.com/phoenix.zhtml?c=86230&p=irol-irhome
BackGround: What is Pelizaeus-Merzbacher Disease (PMD) ?
PMD is a myelination disorder that primarily affects young children. It is a rare central nervous system 
disorder in which coordination, motor abilities, and intellectual function are delayed to variable extents. In November of 2009 STEM began a trial at the University of California, San Francisco (UCSF) Benioff Children's Hospital. In February 2010 the cells were used to treat the first patient enrolled in the trial, marking the first time that neural stem cells have been transplanted as a potential treatment for a myelination disorder. In February 2011, the fourth and final patient was enrolled and dosed. Results of the trial will be reported on Monday (see link above).
This Phase I trial has been designed to assess the safety and preliminary efficacy of STEM's HuCNS-SC cells as a potential treatment for PMD. While the primary focus in this first trial is safety, the company is looking for evidence of new myelin formation in the patients’ brains following the transplantation of the cells, as well as any signs of improved neurological function.
The four enrolled patients have connatal PMD, the most severe form of the disease. All patients were transplanted with the HuCNS-SC cells and were 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. In addition, MRI examination of the brain post-transplant should enable the measurement of new myelin formation. The company is hoping to follow the effects of this therapy long-term, so,we expect a four-year observational study to be reviewed.
Daily Dose Conclusion: This is cutting edge science. Safety come first but STEM is moving in areas that represent totally unmet medical needs. The tough financing environment as hurt the whole stem cell sector but the science is marching forward. STEM remains a pioneer with their HuCNS-SC technology.
Myelination Disorders / PMD (continued)
The gene mutations responsible for PMD result in improperly produced or too much proteolipid protein (PLP), which proves toxic to oligodendrocytes, the CNS cells that produce myelin. Myelin, comprised of fats, cholesterol and protein, is critical to healthy functioning of the central nervous system because it provides the insulation needed for proper transmission of nerve impulses.
In myelination disorders, the deficient myelin sheath does not properly insulate the axon, so transmission of nerve impulses is impeded.
The StemCells Approach: Myelin Production to Protect Nerve Cells
When StemCells human neural stem cells are transplanted in animals, they migrate to the sites where myelin is deficient. They differentiate into oligodendrocytes, which form healthy myelin sheaths to protect axons, helping nerve cells communicate with each other. They do this by developing myelin appendages that wrap around the axons of nearby neurons to provide the insulation (myelin) needed for proper transmission of nerve impulses.
