Stemcells, Inc. Announces First Human Neural Stem Cell Transplant in Myelination Disorder Trial

StemCells, Inc. has announced that its proprietary HuCNS-SC® human neural stem cells have been used to treat the first patient enrolled in its Phase I clinical trial in Pelizaeus-Merzbacher Disease (PMD), a myelination disorder that afflicts male children.

The stem cells were administered yesterday at the University of California, San Francisco (UCSF) Children’s Hospital by direct injection into the brain of a patient with connatal PMD, the most severe form of the disease. This marks the first time that neural stem cells have been transplanted as a potential treatment for a myelination disorder, and the second clinical trial involving the use of HuCNS-SC cells in a neurodegenerative disease.

President and CEO Martin McGlynn stated, “We are extremely grateful to the family of this child, and to the clinicians and staff at UCSF, for bringing us one step closer to our goal of realizing a cell-based treatment for devastating myelination disorders that impact the central nervous system.”

The trial is being directed by a team of prominent researchers at UCSF Children’s Hospital, one of the leading medical centers in the United States for neonatology, pediatric neurology and neurosurgery. 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.