Myelination Disorders / PMD: Pre‑clinical Studies

Published pre-clinical data demonstrates the therapeutic potential of the StemCells HuCNS-SC® (purified human neural stem cells) platform technology to treat myelination disorders such as Pelizaeus-Merzbacher disease (PMD).

This is the second clinical trial of StemCells HuCNS-SC human neural stem cells. The first study, a Phase I Clinical Trial in NCL (neuronal ceroid lipofuscinosis), often referred to as Batten disease, was completed in January 2009. Data from the NCL trial demonstrated a favorable safety profile, along with evidence of engraftment and long-term survival of the HuCNS-SC cells.

This Phase I trial is designed to assess the safety and preliminary efficacy of the HuCNS-SC product candidate as a potential treatment for PMD, and is expected to enroll four patients with connatal PMD, the most severe form of the disease. While the primary focus in this first trial is safety, StemCells will also be looking for evidence of new myelin formation in the patients' brains following transplantation, as well as any signs of improved neurological function. All patients will be transplanted with HuCNS-SC cells, and will be evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC product candidate and the procedure. In addition, MRI examination of the brain post-transplant may enable the measurement of new myelin formation. StemCells plans to follow the effects of this therapy long-term so, as with its Phase I NCL trial, this trial will also be followed by a separate, four-year observational study.

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Pre-clinical Proof of Concept

When transplanted in an animal model of hypomyelination (shiverer mouse), StemCells human neural stem cells have been shown to restore myelin and form myelin sheaths with tight density lines.

Download PDF of published research; see page 7, Figure 6…

PMD Image

Brain

Axon Cross Section

Axon
(red)
Myelin
(green)
Myelin
(grey ring)

Pre-clinical studies performed by StemCells and its collaborators provide a rationale for potential therapeutic use of the Company’s HuCNS-SC product candidate in myelination disorders. StemCells has demonstrated that, when transplanted into an animal model of hypomyelination (shiverer mouse), its neural stem cells engraft and differentiate into mature oligodendrocytes and form myelin sheaths around host nerve fibers. StemCells is using this same approach to treat spinal cord injury, which is often associated with neuron loss and demyelination. Pre-clinical studies have shown that when transplanted into the spinal cord of injured mice, StemCells neural stem cells form myelin around the damaged nerve axons and restore lost motor function.

Learn about our completed PMD clinical trials…

View our publications on myelination disorders…

Learn more about PMD…

About PMD

Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, results in a progressive loss of neurological function. Today there is no cure or effective treatment.

Learn more about PMD…

Clinical Program

StemCells, Inc. completed a Phase I clinical trial of HuCNS-SC® human neural stem cells in Pelizaeus-Merzbacher disease (PMD) in February 2012.

Learn about our completed PMD clinical trials…

Publications

Findings from studies performed by StemCells, Inc. and its collaborators on the use of human neural stem cells for the treatment of myelination disorders have been published.

View our publications on myelination disorders…