StemCells Inc. Receives FDA Approval to Initiate Clinical Trial of HuCNS-SC® Cells in a Myelin Disease
News Dec 19, 2008
StemCells, Inc. has announced that it has received approval from the U.S. Food and Drug Administration (FDA) to initiate a clinical trial of the Company’s proprietary HuCNS-SC product candidate (purified human neural stem cells) to treat Pelizaeus-Merzbacher Disease (PMD), a fatal brain disorder that mainly affects young children.
This Phase I trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for PMD. Currently, there are no approved treatments for this disease.
This is the Company’s second FDA-approved clinical trial to evaluate HuCNS-SC cells as a potential treatment for neurodegenerative diseases. The first such study was the Company’s Phase I clinical trial of HuCNS-SC cells to treat neuronal ceroid lipofuscinoses (NCL), or Batten disease. The Phase I NCL trial will be completed in January 2009.
Patients with PMD are born with a gene mutation that results in insufficient myelination of nerve fibers in the brain, neurological impairment and eventually death. Myelin, which is produced by special cells called oligodendrocytes, insulates nerve fibers to allow electrical signals to be conducted normally. Other, more common, myelination diseases include cerebral palsy, transverse myelitis and multiple sclerosis.
Preclinical studies performed by the Company and its collaborators provide a rationale for potential therapeutic use of HuCNS-SC cells in myelination diseases. The Company has demonstrated that, when transplanted into an animal model of hypomyelination (shiverer mouse), HuCNS-SC cells engraft and differentiate into mature oligodendrocytes and form myelin sheaths around host nerve fibers.
The initial myelination data in the shiverer mouse was published in the Proceedings of the National Academy of Science and the results of additional myelination studies were presented by Nobuko Uchida, Ph.D., the Company’s Vice President of Stem Cell Biology, at the International Society of Stem Cell Research annual meeting in Philadelphia earlier this year.
The Company has begun the process of seeking approval by the Investigational Review Board (IRB) of potential clinical trial sites in order to begin enrolling patients.