Stem Cells, Inc.

Clinical Trials for PMD Patients


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:

Link to UCSF website

PMD clinical trial brochure

Stem Cells Inc. clinical trial sites

Pelizaeus-Merzbacher Disease (PMD) Phase I Results this Weekend (Mar. 2012)

First stem cell clinical trial for treating brain’s “communication highway” to begin (Sep. 2009)

First stem cell clinical trial for Pelizaeus-Merzbacher disease

StemCells provides update on PMD Phase I clinical trial  (Oct. 2010)

Stem cell research heads to the clinic (Oct. 2011)

News at Clinical (Mar. 2011)

StemCells, Inc. Completes Dosing in Second Trial of HuCNS-SC(R) Neural Stem Cells (Feb. 2011)

Neural Stem Cell Trial Gives Hope for Myelin Disorders (Spring 2011)