StemCells, Inc. (NASDAQ: STEM) announced today that it has commenced patient recruitment for a Phase I clinical trial designed to test the safety and preliminary efficacy of its HuCNS-SC® purified human neural stem cells in Pelizaeus-Merzbacher Disease (PMD), a neurological disorder that primarily afflicts children. The study is being conducted at the University of California, San Francisco (UCSF) Children’s Hospital, one of the leading medical centers in the United States for neonatology, pediatric neurology and neurosurgery.
PMD results from a defective gene and is characterized by a lack of myelin, a substance that surrounds and insulates nerve cells’ communications fibers. These fibers function much like electrical wires, and without sufficient myelination are unable to properly transmit nerve impulses, leading to the loss of neurological function and eventually death in the most severe forms of PMD. Currently, there are no effective treatment options for patients with PMD.
“Initiation of this first ever human study of neural stem cells in a myelination disorder is a significant milestone, both for our Company and for the stem cell field,” stated Stephen Huhn MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. “We are investigating a cell therapy approach to assess the ability of HuCNS-SC cells to produce the myelin needed for nerve cells to communicate with one another and maintain healthy functioning of the central nervous system. It is our hope that this approach will one day lead to a viable treatment option for these afflicted children, and possibly also for patients suffering from other myelination disorders, such as multiple sclerosis, transverse myelitis and certain types of cerebral palsy.”
The trial is being directed by a team of prominent academic researchers at UCSF. The principal investigator is David H. Rowitch, M.D., Ph.D., Chief of Neonatology at UCSF Children’s Hospital, Professor of Pediatrics and Neurological Surgery, member of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, and a Howard Hughes Medical Institute investigator. The study co-investigators are Nalin Gupta, M.D., Ph.D., Chief of Pediatric Neurological Surgery, and Jonathan B. Strober, M.D., Director of Clinical Services for Child Neurology and Director of the Muscular Dystrophy Clinic at UCSF Children's Hospital.
“We feel very fortunate to be carrying out this landmark study at a world-class medical center in collaboration with a team of highly regarded specialists, all interested in advancing stem cell therapy for neurological disorders,” added Huhn. “The health and well-being of the children participating in this trial is always the primary focus, so we are pleased that such respected clinicians will be providing their significant expertise to these patients.”