Approval for Stem Cells to Begin Clinical Trails for Batten Disease

Batten disease is a rare but fatal inherited nervous system disorder. It strikes in childhood, with symptoms beginning between ages five and ten. Patients suffer from multiple, increasingly debilitating symptoms and usually die in their late teens or early twenties. It is the most common form of neuronal ceroid lipofuscinosis (NCL) disorders, occurring in two to four live births out of 100,000 in the United States. The Institutional Review Board of the Oregon Health & Science University has just given approval to StemCells Inc. to start a phase I clinical trial of its human neural stem cell product (HuCNS-SC) to treat two forms of Batten disease, infantile and late-infantile NCL. Patient enrollment can now start. US Food and Drug Administration cleared the trial in October 2005.

The HuCNS-SC cell therapy product consists of neural stem cells isolated from the human fetal brain. The cells are purified and expanded and stored as frozen cells until they are transplanted. In preclinical experiments in a mouse model that mimics the human form of infantile NCL spread throughout the brain and produced the lysomal enzyme missing in people with the disease. Other lab studies showed that HuCNS-SC also produces the lysomal enzyme missing in late-infantile NCL, the other subtype being studied in the clinical trial.

The phase I trial is designed to evaluate safety and preliminary data on efficacy of the candidate for the two types of the diseases. It will not treat patients with juvenile NCL, a third form of the disease. In addition to safety, the trial may provide initial data on how the agent affects the progression of the disease. Standardised measures of development, cognition, behavior and language will be used to evaluate the children for one year after transplantation. StemCells is committed to a four-year follow up study after that.

The National Institutes of Health has been supporting research at StemCells Inc. and the Reeve Irvine Research Center at the University of California-Irvine (Irvine, CA) to treat spinal cord injury with HuCNS-SC. The candidate also has potential for treating a number of other nervous system disorders, including spinal injury, myelination problems (such as multiple sclerosis), stroke, Alzheimer's disease and Parkinson's disease.


Martin McGlynn, CEO, StemCells Inc.

3155 Porter Dr., Palo Alto, CA 94304-1213

Phone: 650-475-3100

Fax: 650-475-3101


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