New Milestone In Stem Cells Trial (6/21/2007)

StemCells, Inc. (NASDAQ: STEM) today announced the Phase I clinical trial of its proprietary HuCNS-SC™ product candidate (purified human neural stem cells) has successfully completed enrollment of the low-dose cohort and will proceed to the high-dose cohort.

This trial is designed to evaluate the safety and preliminary efficacy of the HuCNS-SC product candidate as a treatment for infantile and late infantile neuronal ceroid lipofuscinosis (NCL), often referred to as Batten disease. To date, three patients out of a planned total of six have been transplanted with HuCNS-SC cells. A review of the trial data to date, conducted by an independent Data Safety Monitoring Committee comprising experts in pediatric neurosurgery, pediatric neurology, solid organ transplantation and genetics, has identified no safety issues that would preclude advancing the trial to the next dose level.

"This is a significant milestone for the trial and StemCells, Inc. We are halfway through the planned enrollment in the first FDA-approved clinical investigation of purified human neural stem cells. The first patient enrolled in the trial has now reached the halfway point of the study and completed a number of important assessments. To date, all three patients have tolerated the transplantation and have returned home," said Stephen Huhn, M.D., F.A.C.S., F.A.A.P., Vice President and Head of the Neural Program of StemCells, Inc.

"We are encouraged by the progress of the trial, but remain mindful of the difficult challenges involved with the development of novel therapeutics. We are also grateful for the participation of the families in this study and the commitment of the research staff at Doernbecher Children's Hospital at OHSU. Batten disease is a terrible affliction, and this trial is the first step on the path toward a treatment for this devastating disease and potentially other lysosomal storage disorders."

The clinical trial is being led by co-principal investigator Robert Steiner, M.D., Vice Chairman of Pediatric Research at Doernbecher Children's Hospital, and Professor of Pediatrics and Molecular & Medical Genetics in the Oregon Health & Science University (OHSU) School of Medicine; co-principal investigator Nathan Selden, M.D., Ph.D., Campagna Associate Professor of Pediatric Neurological Surgery and Head of the Division of Pediatric Neurological Surgery, Doernbecher, OHSU School of Medicine; co-investigator Thomas K. Koch, M.D., F.A.A.P., F.A.A.N., Director of Pediatric Neurology and Professor of Pediatrics and Neurology at Doernbecher, OHSU School of Medicine; and co-investigator Amira Al-Uzri, M.D., M.C.R., associate professor of pediatrics (pediatric nephrology and hypertension), and director of the pediatric kidney transplant program at Doernbecher, OHSU School of Medicine.

About Neuronal Ceroid Lipofuscinosis (Batten Disease)

Neuronal ceroid lipofuscinosis is a fatal neurodegenerative disorder brought on by inherited genetic mutations. The disorder afflicts infants and young children, and the three most common forms of NCL — infantile, late infantile and juvenile onset — are often referred to as Batten disease. All forms have the same basic cause—lack of a lysosomal enzyme—and have similar progression and outcome. Children with NCL suffer seizures, progressive loss of motor skills, sight and mental capacity, eventually becoming blind, bedridden and unable to communicate.

Infantile or late infantile NCL is brought on by inherited mutations in the CLN1 gene, which codes for palmitoyl-protein thioesterase 1 (PPT1) or in the CLN2 gene, which codes for tripeptidyl peptidase I (TPP-I), respectively. The consequence of these gene mutations is either a defective or missing enzyme that leads to accumulation of lipofuscin-like fluorescent inclusions in various cell types. These non-degraded lysosomal inclusions accumulate to the point of interference with normal cellular function and ultimately lead to the pathological manifestations of the disease. One way to treat the disease is to provide the brain with a replacement source of functional enzyme that can be taken up by the enzyme-deficient cells.

Note: This story has been adapted from a news release issued by Oregon Health & Science University

Post Comments: