StemCells gets board OK to start phase 1 clinical trial in Batten disease

By Elaine Rigoli

Tampa, Fla., March 9 - StemCells, Inc. announced Thursday that it has received approval from the Institutional Review Board of the Oregon Health & Science University to start a phase 1 clinical trial of the company's proprietary human neural stem-cell product, HuCNS-SC.

This product is intended as a treatment for infantile and late-infantile neuronal ceroid lipofuscinosis (NCL), two forms of a group of disorders often referred to as Batten disease, according to a company news release.

StemCells announced in October 2005 that it received clearance from the Food and Drug Administration to start a phase 1 trial, and this review board approval means that patient enrollment may now begin, the release said.

StemCells said this will be the first trial using a purified composition of neural stem cells as a potential therapeutic agent in humans.

"[Institutional Review Board] approval of this groundbreaking trial removes the only remaining regulatory requirement between us and the initiation of clinical testing in this very needy patient population. It is the culmination of many years of meticulous basic science followed by diligent preclinical research conducted by our scientists and collaborators around the world," said Martin McGlynn, president and chief executive officer, in a statement.

Batten disease, named after the British pediatrician who first described the juvenile form of NCL in 1903, is a rare and fatal neurodegenerative genetic condition. The name is used to encompass the three most common forms of NCL, infantile, late infantile and juvenile onset. All forms have the same basic cause - a lack of a lysosomal enzyme - and have similar progression and outcome.

StemCells' HuCNS-SC is a cell therapy product prepared under controlled conditions. Neural stem cells are isolated from the human fetal brain, purified, expanded and then stored and frozen in cell banks until they are transplanted as HuCNS-SC doses, the release said.

When HuCNS-SC are transplanted into the brain of a preclinical mouse model developed to mimic the human form of infantile NCL, they 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, the release said.

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 Batten disease.

StemCells, located in Palo Alto, Calif., is a clinical stage biotechnology company focused on the discovery, development and commercialization of stem cell-based therapies to treat diseases of the nervous system, liver and pancreas.

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