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Oregon Health & Science University to Try Stem-cell Shots for Child Disease
By Andy Dworkin
Knight Ridder/Tribune Business News
10 March 2006
Mar. 10--Oregon Health & Science University will be the first, and maybe only, hospital to give a risky experimental stem-cell transplant to children dying of a rare nerve-destroying disease.
In the coming months, as many as six children with Batten disease will travel to OHSU for injections of neural stem cells, primitive cells that can form new brain and nerve cells.
The stem cells are taken from human fetuses, with their mothers' agreement, and processed and purified by a California company, StemCells Inc. At OHSU, a surgeon will inject the stem cells into eight areas of each child's brain.
The theory is that the stem cells will take root and grow in the brain, making enzymes missing in children with the two forms of Batten disease being studied, said Dr. Robert Steiner, the principal investigator at OHSU's Doernbecher Children's Hospital.
The roughly one in 100,000 children with these incurable diseases can't clear toxins that gather in their cells. They generally lose their vision, develop seizures and dementia, and die before their teens.
That grim outlook helped persuade OHSU's scientists to join the trial, even though its benefits are unclear. No one has ever put these stem cells in a person, and the procedure may not help anyone. This early-stage human trial aims mostly to see whether the treatment is safe, as opposed to effective.
"These cells have never been transplanted into a human being before, nor anything like them," said Martin McGlynn, StemCells Inc. president and chief executive. "These are uncharted waters."
In tests on mice with a Batten-like disease, the stem cells grew and helped make the missing enzyme, limiting nerve damage and extending the mice's lives, McGlynn said. The chance that people could see similar results weighs against the trial's risks, which include bleeding in the brain and infection.
McGlynn said other health centers are deciding whether to join the trial, but he would not name them. The company withdrew an application from Stanford University after its board asked for more information on possible benefits, which McGlynn said would take too much time to provide.
For parents of children with Batten disease, the start of this trial "is like coming out of the darkness into brilliant sunlight," said Marcus Kerner, an assistant U.S. attorney in Trabuco Canyon, Calif. Kerner's 6-year-old son, Daniel, can't speak or walk, though he can see and think, his father said. The family has applied to join the trial, and Kerner hopes to travel to Oregon for Daniel's treatment.
"Despite this being phase one safety research, we believe this is going to be one of the most profound medical breakthroughs in history, and we believe it's going to save his life," he said.
Doctors stress that there's no way to know whether the stem cells will help until the trial is over. But the hope that the trial may help create a new kind of treatment inspires them to move ahead, said Dr. Nathan Selden, the Campagna Associate Professor of pediatric neurological surgery at OHSU.
"We constantly face diseases for which there is no really good treatment," Selden said. "And we live for the days when we can see that change."
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Copyright (c) 2006, The Oregonian, Portland, Ore.
Distributed by Knight Ridder/Tribune Business News.
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