Portland - A safety panel reviews the first treatments to battle Batten disease, a deadly destroyer of brains
Tuesday, June 19, 2007
ANDY DWORKIN
Portland doctors are halfway through a dramatic medical experiment that injects stem cells inside the skulls of children dying from the brain-destroying illness Batten disease.
Since November, three children have been treated at Oregon Health & Science University with stem cells purified from fetuses. In animal tests, the cells made the missing enzymes that cause Batten disease. Scientists hope for the same in people.
The trial's sponsor, California-based StemCells Inc., is limiting information on the results so far. But an independent safety committee reviewed the cases and let the trial continue.
"The fact that they've looked at the (data) does not mean that there have not been any serious adverse events," said Rodney Young, chief financial officer of StemCells Inc. Passing the review does mean no problems have been serious enough to stall the trial, which is mainly meant to judge the treatment's safety. Full information on safety and possible hints about the treatment's effectiveness won't be available until after the trial ends in late 2008 or 2009.
Dr. Nathan Selden, the Doernbecher Children's Hospital neurosurgeon who performed the operations, said doctors are thrilled to be "halfway through the trial, and extremely pleased that the monitoring board has validated the ongoing safety of the trial." Selden and Young declined to discuss the three patients treated so far.
The father of 7-year-old Daniel Kerner, the first child treated, said none of the three children has had serious problems related to the treatment.
Daniel "is getting better and stronger," said Marcus Kerner, an assistant U.S. district attorney in Santa Ana, Calif. "We know the other kids. They are the cutest kids you could possibly imagine, and they are doing wonderfully."
"At this point, the way things are going, it looks like all the kids are going to make it," he said.
The stem cell study is the model of high-stakes science. Study subjects are the most vulnerable imaginable: Children with an incurable, deadly disease that leaves them unable to fully understand their treatment. In Batten disease, fatty substances build up in the brain, killing nerve cells and sapping a person's ability to see, speak, think and move. Affected children generally develop seizures and die by their teens.
The treatment is risky: StemCells purifies cells from the brains of aborted fetuses donated for research. The cells are injected through eight holes in the skull in a long surgery, whose risks include bleeding in the brain and infection from drugs used to control the immune system post-transplant.
Experimenters are moving into the trial's second phase, which will treat three different children with a higher dose of stem cells. They declined to say when the next surgery is scheduled but hope to finish all three this year.
Kerner gladly discusses what he calls "a miracle that's continuing to unfold."
Before surgery, Daniel had lost much of his ability to speak and move. Soon after, his parents noticed language improvements. He said "Dad" for the first time in two years. The other night, Kerner said, after watching the movie "Brother Bear," Daniel said, "I'm a bear."
Daniel has gained enough leg strength to stand for more than a minute, using his hands to steady himself. "His legs are so strong that he was able to break the straps off the foot pedals" on his wheelchair, Kerner said.
The changes have not all been dramatically good. "Before the surgery he was seizure-free," Kerner said. "Now, depending on how we adjust his medicines, he has minor issues with seizures. But nothing major." But a weeklong follow-up visit at Doernbecher Children's Hospital last month showed "no problem at all associated with the stem cells," Kerner said.
Between doctor visits, Daniel leads a busy life. For his seventh birthday in January he went biskiing, schussing the slopes with guides in a sort of wheelchair on skis. He finishes second grade with his special education class on Thursday.
"It is like he's growing along with the cells," Kerner said. "We know there's a long road ahead, that you don't wave a magic wand over a killer disease and make it evaporate. What you do is what these kids are doing . . . These kids are fighting as hard as you can possibly believe."
Andy Dworkin: 503-221-8239; andydworkin@news.oregonian.com