Sunday, October 23, 2005

Stem-Cell Research: Batten Disease

Trials for stem-cell treatment of brain disease
Clinical testing set to begin on kids with fatal affliction
Cornelia Stolze, Carl T. Hall, Chronicle Staff Writers
San Francisco Chronicle
Friday, October 21, 2005

Researchers in California are about to start the first FDA-sanctioned clinical trial of a stem cell treatment for a brain disease.

The trial will offer regulators one of their first opportunities to reveal what standards should apply for novel "regenerative medicine" experiments in human subjects. Special safety concerns include the long-term cancer-causing potential of stem cells and the risk that they may wire into the brain's circuitry improperly.

"A great deal of attention will be paid to the outcome of this trial," said Dr. Arnold Kriegstein, a neurologist and head of a stem cell program at UCSF, who is not involved in the new study. "It really represents the first trial of its kind. Regardless of the outcome, all of us interested in cell-based therapies will be watching what happens."

The study is sponsored by a small Palo Alto biotech company, Stem Cells Inc., to test the safety of injecting fetal-derived neural stem cells into the brains of children suffering from a rare and always fatal disorder known as Batten disease.

The condition afflicts about 2 to 4 of every 100,000 children born in the United States. Babies may appear normal at first, only to begin losing coordination and suffering seizures several months after birth. Eventually, children become blind, bedridden and unable to communicate before they die at an early age. No therapy is available to stop or even slow down the symptoms.

The problems are caused by a gene mutation, which leads to a lack of an enzyme needed for proper recycling of certain substances that otherwise can build up in the brain. The stem cells are intended to serve as delivery vehicles to get the missing enzyme where it's needed.

Two Stanford physicians -- Dr. Gregory Enns, an assistant professor of pediatrics and director of the biochemical genetics program at Stanford, and Dr. Stephen Huhn, chief of pediatric neurosurgery at Packard Children's Hospital -- helped design the trial protocol. They intend to begin the first procedures sometime next year, if an internal review board at Stanford gives its consent.

Enns said families with Batten disease have little choice but to try a novel approach.

"This is an unremitting disease that is particularly tragic," he said. "Right now there is nothing we can do to stop the progression of this terrible disorder."
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Anthony H. Risser | |

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