PORTLAND, Ore. — Oregon Health & Science University Doernbecher Children's Hospital will lead the next phase of a landmark clinical trial to further assess the safety and preliminary effectiveness of purified human neural stem cells (HuCNS-SC®) as a potential treatment for infantile or late-infantile neuronal ceroid lipofuscinosis (NCL), a rare and currently fatal neurodegenerative disorder that affects infants and children.
The Phase Ib trial, sponsored by StemCells, Inc., is designed to further assess the safety and preliminary effectiveness of StemCells' HuCNS-SC as a potential treatment for NCL, also referred to as Batten disease. This second trial is expected to enroll six children whose NCL is less advanced than those enrolled in the initial Phase I trial, also carried out at OHSU Doernbecher, and, in addition to safety, is also designed to evaluate the impact of HuCNS-SC on disease progression.
"The hard work of testing neural stem cells for their safety and effectiveness in clinical use began here at OHSU Doernbecher Children's Hospital. We are very pleased to carry on this important effort with a second major trial. Our great hope is that this work will eventually yield significant benefits for patients who suffer from devastating nervous system diseases," said Nathan Selden, M.D., Ph.D., FACS, FAAP, Campagna Professor of Pediatric Neurosurgery and head of the Division of Pediatric Neurological Surgery at OHSU Doernbecher Children's Hospital, OHSU School of Medicine.
Selden was co-principal investigator on the groundbreaking initial Phase I trial and will lead the Ib study.
The initial trial was the first-ever FDA-authorized clinical trial of purified human neural stem cells as a potential therapeutic agent. Begun in March 2006, its aim was to test the safety and preliminary efficacy of transplanting HuCNS-SC in six children with advanced stages of NCL. Once transplanted, the children were followed for 12 months. Data from the Phase I trial, completed in January 2009, demonstrated that high doses of HuCNS-SC transplanted directly into multiple sites within the brain followed by 12 months of immunosuppression were well tolerated. The data also included evidence of engraftment and long-term survival of the donor cells.
Participants in the new study will be transplanted with HuCNS-SC via a neurosurgical procedure and their immune systems will be suppressed for nine months. Following transplantation, the children will be monitored and regularly evaluated for 12 months to assess the safety and tolerability of the HuCNS-SC, the surgery and the immunosuppression. In addition, participants will be evaluated and assessed at regular intervals using a comprehensive range of clinical and X-ray tests, both before transplantation to establish a baseline, and over the course of 12 months following transplantation.
StemCells, Inc. will initiate a separate four-year observational study at the conclusion of this trial.
For information about enrollment in the new trial at OHSU Doernbecher, call 503-494-4495 or visit www.stemcellsinc.com. Additional information about this clinical trial can also be found at www.clinicaltrials.gov.
About Neuronal Ceroid Lipofuscinosis (Batten disease)
Neuronal ceroid lipofuscinosis (NCL) is a fatal neurodegenerative disorder that afflicts infants and young children. The disorder, often referred to as Batten disease, is caused by genetic mutations, and children who inherit the defective gene are unable to produce enough of an enzyme that processes cellular waste substances that accumulate in a part of cells known as the lysosome. Without the enzyme, the cellular waste builds up, and eventually the cells cannot function and die. Children with NCL appear healthy when born, but as their brain cells die, they begin to suffer seizures and progressively lose motor skills, sight and mental capacity. Eventually, they become blind, bedridden and unable to communicate or function on their own. There currently is no cure for the disease. The infantile and late infantile forms of NCL are caused by different genetic mutations. As the names imply, the two forms begin to afflict patients at different stages of infancy, but both have similar disease progression and outcomes.
About OHSU Doernbecher Children's Hospital (www.ohsudoernbecher.com)
OHSU Doernbecher Children's Hospital is a world-class facility that each year cares for tens of thousands of children from Oregon, southwest Washington and around the nation, including national and international referrals for specialty care. Children have access to a full range of pediatric care, not just treatments for serious illness or injury, resulting in more than 145,000 outpatient visits, discharges, surgeries and pediatric transports annually. Nationally recognized physicians ensure that children receive exceptional care, including outstanding cancer treatment, specialized neurology care and highly sophisticated heart surgery in the most patient- and family-centered environment. Pediatric experts from OHSU Doernbecher travel throughout Oregon and southwest Washington to provide specialty care to some 3,000 children at more than 154 outreach clinics in 13 locations
About StemCells, Inc. (www.stemcellsinc.com)
StemCells, Inc. is engaged in the research, development, and commercialization of stem cell therapeutics and tools for use in stem cell-based research and drug discovery. In its therapeutic product development programs, StemCells is targeting diseases of the central nervous system and liver. StemCells' lead product candidate, HuCNS-SC® cells (purified human neural stem cells), is in clinical development for the treatment of two fatal neurodegenerative disorders that primarily affect young children. StemCells also markets specialty cell culture products under the SC Proven® brand, and is developing stem cell-based assay platforms for use in pharmaceutical research, drug discovery and drug developments.