StemCells initiates HuCNS-SC second clinical trial for Batten disease in children

Oct 29 2010

StemCells, Inc. (Nasdaq:STEM) announced today that it has initiated a second clinical trial of its HuCNS-SC^® product candidate (purified human neural stem cells) in neuronal ceroid lipofuscinosis (NCL, also often referred to as Batten disease), a fatal neurodegenerative disorder in children. The trial is designed to evaluate the safety and preliminary efficacy of the cells in patients with either infantile or late infantile NCL. The trial will enroll six patients with less advanced stages of the disease than those who participated in the Company's first NCL trial. Like the first NCL trial, this second trial is being conducted at Oregon Health & Science University (OHSU) Doernbecher Children's Hospital, a leading medical center with nationally recognized programs in pediatric neurology and neurosurgery.   

 "The initiation of this trial represents a key advancement in the evaluation of our human neural stem cells as a potential therapeutic product," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "In this second trial, we will continue to assess safety, but will also look at certain measurements to evaluate the impact of these cells on disease progression. The trial is designed to enroll patients with less neuronal degeneration, which we believe will enhance the prospect of detecting clinical benefit. The data gathered from this trial will also help us begin to assess possible clinical endpoints for use in a pivotal clinical trial in NCL, and may provide important information about the potential for the use of these cells in other neurodegenerative disorders."

The trial is being led by Nathan Selden, MD, PhD, FACS, FAAP, Campagna Professor of Pediatric Neurosurgery and head of the Division of Pediatric Neurological Surgery at OHSU Doernbecher Children's Hospital and OHSU School of Medicine. Dr. Selden was co-principal investigator of the Company's first NCL trial.

"We are pleased to work with StemCells in the pursuit of an effective therapy for this devastating disease," stated Dr. Selden. "The first study conducted here at OHSU Doernbecher Children's Hospital revealed a favorable safety profile for human neural stem cell transplantation directly into the brain. This second trial is an important next step, and may allow us to see evidence of clinical efficacy from the transplanted cells. We look forward to further evaluating the potential of these cells to truly help children facing a desperate illness."