The Phase 1 trial will take place at UCSF Children’s Hospital and will evaluate StemCells Inc.’s HuCNS-SC product candidate (purified human neural stem cells) as a treatment for Pelizaeus-Merzbacher Disease (PMD).
“There is a compelling rationale for this trial, and it is a critical first step towards potentially helping seriously ill children who today have no treatment options, as this trial may provide proof-of-concept regarding the extent of myelination in these patients following transplantation of HuCNS-SC cells,” said Martin McGlynn, President and CEO of StemCells, Inc.
Stephen Huhn MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc., added, “Our clinical development strategy has been to use our HuCNS-SC cells to provide what the patient’s own cells cannot, whether that is a missing enzyme, as in NCL, or myelin, as in PMD. The goal is to have our cells protect the patients’ existing neurons and maintain their function. I believe that if we can show that these cells preserve neurological function in patients with PMD, then it is conceivable that patients suffering from other, more common, myelination disorders, such as multiple sclerosis, transverse myelitis and certain types of cerebral palsy, may also benefit from this approach.”
The trial will be conducted at UCSF Children’s Hospital, which is one of the top centers for pediatric neurology and neurosurgery in the
Other myelination disorders include multiple sclerosis and cerebral palsy. Accordingly, positive results in this study may pave the way for studies of HuCNS-SC in these other areas.