StemCells & PMD, Enters Pelizaeus-Merzbacher Disease Study (STEM)
StemCells, Inc. (NASDAQ: STEM) is trading higher this morning on a new study initiation. The stem cell player has announced that it will soon begin a study with the University of California, San Francisco Children’s Hospital. This will be in a Phase I clinical trial, and the goal will be to evaluate the therapeutic potential of StemCells’ proprietary HuCNS-SC® product candidate of purified human neural stem cells as a possible treatment for Pelizaeus-Merzbacher Disease (PMD).
PMD is a myelination disorder that primarily affects infants and young children. Myelin is the substance which surrounds and insulates nerve fibers and enables nerve cells to communicate with one another. Patients suffering from PMD have insufficient myelination of nerve cells in the brain, which leads to loss of neurological function and eventually death in the most severe forms of the disease.
In this trial, patients with a fatal form of PMD will be transplanted with the StemCells’ HuCNS-SC cells. Then the study will evaluate safety and will explore the ability of the cells to myelinate the patients’ nerve axons.
This trial is said to be “the first ever clinical trial of neural stem cells in a myelination disorder,” but it will actually be StemCells’ second clinical trial of its HuCNS-SC cells.
The Company’s first clinical trial was a Phase I trial in neuronal ceroid lipofuscinosis (Batten disease) was completed in January 2009. The company said that this study demonstrated a favorable safety profile along with evidence of engraftment and long-term survival of the HuCNS-SC cells.
What makes this new study unique is that Severe PMD is a very devastating disease and effectively there is no treatment existing today. The good news, if there is any good news, is that it is rare and can often be detected in genetic screenings to avoid passing it on.
Ac cording to data at the The PMD Foundation, there is currently no cure for Pelizaeus-Merzbacher disease, nor is there a standard course of treatment. It does note that outside of the most severe patients, some individuals can survive into their sixties.
So far, we have StemCells trading up almost 3% at $1.75 on the news.
JON C. OGG
SEPTEMBER 10, 2009
