Stemcells, Inc. Advances to Second Clinical Trial in Batten Disease
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.”
About the Phase Ib NCL Trial
The Phase Ib trial is designed to further assess the safety and preliminary efficacy of HuCNS-SC cells as a potential treatment for NCL. All patients will be transplanted with HuCNS-SC cells via a neurosurgical procedure, and will be immunosuppressed for nine months. Following transplantation, the patients will be evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC cells, the surgery, and the immunosuppression. In addition, MRI evaluations will focus on potential measures of effect from the donor cells, including assessing and tracking cerebral volume and unique neuronal metabolites, both of which are negatively impacted by disease progression. As the Company intends to follow the effects of this therapy long-term, a separate four-year observational study will be initiated at the conclusion of this trial. For information on patient enrollment, interested parties may call (503) 418-4495. Additional information about this clinical trial can also be found at www.stemcellsinc.com and www.clinicaltrials.gov.
About Neuronal Ceroid Lipofuscinosis (Batten Disease)
Neuronal ceroid lipofuscinosis (NCL) is a fatal lysosomal 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 independently. There currently is no effective treatment 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.
StemCells has shown in preclinical studies that its HuCNS-SC cells produce the enzyme missing in NCL and protect neurological function. The Company’s clinical development strategy in NCL may also provide important information on the applicability of HuCNS-SC cells for other lysosomal storage disorders that affect the central nervous system, as well as for a broad range of other neurological diseases.