StemCells, Inc. (STEM) revealed preliminary evidence of progressive and durable donor-cell derived myelination in all four patients who underwent transplantation with the company's proprietary HuCNS-SC cells in its clinical trial for Pelizaeus-Merzbacher disease, or PMD, a rare hypo-myelination disorder in children.
In addition, clinical assessment disclosed small but measureable gains in motor and/or cognitive function in three of the four patients; the fourth patient remained clinically stable.
The company noted that the open-label Phase I trial enrolled four patients with the connatal form of PMD, between the ages of 14 months and 5 years, and was designed to assess safety and preliminary efficacy of the intervention. The study used magnetic resonance, or MR, imaging, commonly employed in other neurological diseases, to explore signs of myelination related to the transplanted neural stem cells.
At the one-year interval, MR imaging showed changes compatible with increased myelination in the region of the transplantation.
Further, the company said that a separate four-year observational study will continue to monitor and report the future progress for all four patients.
"The finding of myelin formation in this first exploratory study is indeed very encouraging," said Stephen Huhn, Vice President and Head of the CNS Program at StemCells, Inc. "We believe that the results of this trial provide proof-of-concept and a compelling rationale for the Company to begin planning for a controlled Phase II study in PMD. These results may also have implications for other leukodystrophies, as well as more common myelin disorders including transverse myelitis, multiple sclerosis and periventricular white matter injury seen in Cerebral Palsy..."
by RTT Staff Writer
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