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.
Details
Martin McGlynn, CEO, StemCells
Inc.
3155 Porter Dr., Palo Alto, CA 94304-1213
Phone: 650-475-3100
Fax: 650-475-3101
URL:
www.stemcellsinc.com
To
comment on this article, write to us at
tiresearch@frost.com
To find
out more about Technical Insights and our Alerts, subscriptions and research
services, access
http://ti.frost.com
|