Myelination Disorders / PMD (Pelizaeus-Merzbacher Disease)
StemCells, Inc. is conducting a Phase I clinical trial of HuCNS-SC® human neural stem cells in PMD, a fatal myelination disorder in children. Learn more about our clinical trial in 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.
Preclinical 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.
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Axon Cross Section
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Preclinical 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. Preclinical 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.
