"The OHSU Doernbecher research team worked very hard to carry out this highly complex research and is heartened to see that this approach appears to be safe. We are delighted that this first trial of human neural stem cells was successful and offers hope for effective treatment of NCL and other neurodegenerative disorders," said Robert Steiner, M.D., F.A.A.P., F.A.C.M.G., co-principal investigator, professor of pediatrics and molecular and medical genetics, and vice chairman for pediatric research at Oregon Health & Science University Doernbecher Children's Hospital.
The Phase I trial of HuCNS-SC (purified human neural stem cells) conducted at OHSU Doernbecher Children's Hospital included six children with advanced stages of infantile and late-infantile neuronal ceroid lipofuscinosis (NCL), often referred to as Batten disease. The study participants were transplanted with HuCNS-SC cells and followed for 12 months.
Overall, the Phase I data demonstrated that high doses of HuCNS-SC cells transplanted directly into multiple sites within the brain followed by 12 months of immunosuppression were well tolerated by all six patients. The patients' medical, neurological and neuropsychological conditions following transplantation appeared consistent with the normal course of the disease.
"It was a privilege for our team to care for these precious children. We are indebted to our patients and their families for taking us into this new era of therapy for the central nervous system. We hold out great hope in the future for them and for others around world with similar diseases that today have no cure," said Nathan Selden, M.D., Ph.D., F.A.C.S., F.A.A.P., co-principal investigator, Campagna Associate Professor and Head, Division of Pediatric Neurological Surgery, OHSU Doernbecher Children's Hospital.
The OHSU Doernbecher team was the first in the world to implant neural human stem cells directly into the brains of patients with NCL.
"We are grateful to the multi-specialty team of investigators and clinicians at OHSU for their key role in conducting this landmark trial. We appreciate the dedication they demonstrated both to the patients enrolled in the trial and to the importance of this research as we work to find a treatment for this devastating disease," said Stephen Huhn, M.D., F.A.C.S., F.A.A.P., vice president and head of the CNS program at StemCells, Inc.
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 HuCNS-SC® Cells
StemCells' lead product candidate, HuCNS-SC cells, is a purified composition of normal human neural stem cells that are expanded and stored as banks of cells. The Company's preclinical research has shown that HuCNS-SC cells can be directly transplanted; they engraft, migrate, differentiate into neurons and glial cells; and they survive for as long as one year with no sign of tumor formation or adverse effects. These findings show that HuCNS-SC cells, when transplanted, act like normal stem cells, suggesting the possibility of a continual replenishment of normal human neural cells.
About StemCells, Inc.
StemCells, Inc. is a clinical-stage biotechnology company focused on the research, development and commercialization of products derived from stem cell technologies. In its therapeutic product development programs, StemCells is focused on developing cell-based therapeutics to treat diseases of the central nervous system and liver. StemCells has pioneered the discovery and development of HuCNS-SC® cells, its highly purified, expandable population of human neural stem cells. StemCells has completed a six-patient Phase I clinical trial of its proprietary HuCNS-SC product candidate as a treatment for neuronal ceroid lipofuscinosis (NCL), a rare and fatal neurodegenerative disease that affects infants and young children. StemCells has also received approval from the Food and Drug Administration (FDA) to initiate a Phase I clinical trial of the HuCNS-SC cells to treat Pelizaeus-Merzbacher Disease (PMD), a rare and fatal brain disorder that mainly affects young children. StemCells, through its wholly owned subsidiaries Stem Cell Sciences UK Ltd and Stem Cell Sciences Australia Pty, is also pursuing applications of its cell-based technologies to develop research tools, such as cell-based assays, media and reagent tools, which the Company believes represent nearer-term commercial opportunities. StemCells has exclusive rights to approximately 55 issued or allowed U.S. patents and approximately 200 granted or allowed non-U.S. patents. Further information about StemCells is available on its Web site at: www.stemcellsinc.com.
About Oregon Health & Science University Doernbecher Children's Hospital
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 120,000 outpatient visits, discharges, surgeries and pediatric transports annually. In addition, nationally recognized physicians ensure that children receive exceptional care at OHSU Doernbecher, 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 2,800 children at more than 154 outreach clinics in 13 locations.