NEWS & COMMENTS

PROHOST    FILTER

We have news that brings hope to children suffering from

a neurodegenerative disease - infantile and late infantile neuronal ceroid lipofuscinosis (NCL),

which is often referred to as:

Batten Disease.

      Neuronal ceroid lipofuscinosis is a rare and 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.

      In infantile and late infantile NCL, the disorder 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 substrates accumulate to the point of interference with normal cellular and tissue 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.

The News

STEM CELLS INC. (STEM)

      The news is about stem cell treatment. It is emanating from the biotech firm StemCells, Inc  (STEM), which has already conducted the first transplantation of its proprietary human neural stem cell product--HuCNS-SC(TM)— to reverse the symptoms and complications of this disease at the Oregon Health & Science University's (OHSU) Doernbecher Children's Hospital. This transplant is the first of six that are planned as part of the firm’s  Phase I clinical trial designed to evaluate the safety and preliminary efficacy of HuCNS-SC as a treatment for Batten Disease.

     Martin McGlynn, President and CEO of StemCells, Inc. said: “Dosing of the first Batten's patient is a major milestone for StemCells, Inc., and for the entire stem cell field. It is important to remember, that this is just the first step in a long and challenging clinical development process. Our focus at this time is on the health and well-being of the patient. We are grateful for the participation of this family in this landmark trial. Children afflicted by Batten disease are gravely ill, and this family's courage advances the hope that this trial will lead to a treatment for this devastating disease."

      The Phase I clinical trial is being led by Robert D. Steiner, M.D., F.A.A.P., F.A.C.M.G., vice chairman of pediatric research and head of the Division of Metabolism at Doernbecher Children's Hospital, and professor of Pediatrics and Molecular & Medical Genetics at OHSU School of Medicine; Nathan Selden, M.D., Ph.D., F.A.C.S., F.A.A.P., Campagna Associate Professor of Pediatric Neurological Surgery and head of the Division of Pediatric Neurological Surgery, Doernbecher and OHSU School of Medicine; and 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 and OHSU School of Medicine.

      HuCNS-SC™ are a cell-based therapeutic prepared under controlled conditions. When HuCNS-SC are transplanted into the brain of a mouse model developed to mimic the human form of infantile NCL, the cells spread throughout the brain and produce the missing lysosomal enzyme. The enzyme level increases and continues to do so over time after the transplant. Thus, placement of HuCNS-SC in appropriate places in the brain provides the prospect of long-term delivery of the missing lysosomal enzyme. In laboratory studies, HuCNS-SC also produces the lysosomal enzyme missing in late infantile NCL, the other subtype being studied in the clinical trial. The production of both enzymes by HuCNS-SC provides a scientific rationale for enzyme replacement and cellular rescue in these two subtypes of NCL.

      The Phase I trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC as a treatment of infantile and late infantile NCL. The trial is open label with two dose cohorts, and is expected to enroll six patients during the next 12 months. Potential patients will be tested for eligibility and then evaluated for baseline disease status prior to transplantation of HuCNS-SC. In addition to measuring the safety of HuCNS-SC, patients enrolled in the study will be evaluated with standardized measures of development, cognition, behavior and language for one year following transplantation.

      Hope, yes, is what this news brings to hopeless children and hopeless families. We cross our fingers.

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