On February 9, 2010, Children’s Hospital at the University of California – San Francisco (UCSF) began Phase I safety studies on a potential treatment for Pelizaeus-Merzbacher Disease (PMD). This study is sponsored by StemCells, Inc., the company providing proprietary HuCNS-SC® human neural stem cells. The initial trials on young males with the connatal form of PMD will determine the safety of using the HuCNS-SC® cells on patients with a myelin disorder while monitoring their potential effectiveness as a treatment for PMD. Candidates were male, between 6 months and five years of age, with genetic confirmation of connatal PMD. These trials are currently in progress, and all four candidates have been chosen and initiated.
On April 2, 2012, there was a News Release from Stem Cells Inc. regarding the initial results of their Phase I Clinical Trial therapy of connatal PMD with Hu-CNS-SC® cells. To read the article, click here. Although these results are preliminary and there were no control subjects for comparison, there is a small but positive indication of progressive myelination in all four test patients. There is also a level of confidence in the safety of the stem cell transplant regimen. Evaluation and monitoring will continue for an additional 4 years. Stem Cells Inc believes these initial results provide a compelling rationale to begin planning a controlled Phase II study. They are working in collaboration with UCSF in hopes of publishing a study of their findings.
Additional updates on the status of the studies will be monitored and posted. For additional information, click on any of the following: