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Sometime in the next few weeks, a surgeon in Portland, Oregon, will drill a series of small holes into a young child's skull. The child has a rare, fatal brain disease and through those holes, the surgeon will inject neural stem cells.
It's the first time neural stem cells will have been used to treat a human disease, but it's also possible that the treatment itself will kill the child. And that raises a difficult question. When is it appropriate to test unproved treatments on children?
NPR's Joe Palca reports.
JOE PALCA: If you've never heard of Batten Disease, count yourself lucky.
Mr. LANCE JOHNSON (Batten Disease Support and Research Association): I had a daughter that had juvenile Batten Disease that started when she was five.
PALCA: Lance Johnson knows a lot about Batten Disease. It's an inherited disease that affects the nervous system. Patients lack an enzyme for breaking down certain fat and protein compounds. Over time, the compounds build up inside nerve cells. Eventually, the cells stop working.
The first sign of trouble is usually vision problems. That was the case with Lance Johnson's daughter. But that was just the beginning.
Mr. JOHNSON: She had her first seizure at age nine. She was totally blind at age 13. She started having mobility problems at that time. She started having severe dementia. Cognitive functioning problems, behavioral problems at 15. She couldn't walk any longer at 17. She had a feeding tube put in at 19. She was totally bedridden at 20, and she died at 22.
PALCA: That was in 1993.
Today, Johnson is director of the Batten Disease Support and Research Association. He says if his daughter were diagnosed today, the outcome would be the same. There is no cure for Batten Disease. And since the disease is rare, there's not a ton of pressure to find a cure.
But a company called Stem Cells, Inc. thinks it may have something. Studies in mice suggest human neural stem cells produce the missing enzyme. Maybe, just maybe these stem cells might produce enough of the enzyme to stop the damage to the nervous system.
Mr. JOHNSON: Out of the darkness of desperation, there's a glimmer of light. It's something that you can reach out and grasp and hold on to and pray that it will work and save your child's life.
PALCA: But for now, it's just a glimmer. Scientists have no idea how many stem cells will be needed to halt the damage to the brain. They also have no idea how many is too many. Too many could be harmful. So the plan is to start cautiously and establish that the procedure is safe.
Robert Steiner is a pediatrician at Oregon Health and Science University. He's in charge of the first study with human patients. The children in the study have more aggressive forms of Batten Disease. One form kills by age 12, the other by age five. In this study, the children are mostly too young to make an informed decision about whether to participate, so it's up to the parents.
On a speaker phone at a recent teleconference, Steiner said he and his colleagues do everything they can to explain to families that this is a safety study, not a study designed to help their child's illness.
Dr. ROBERT STEINER (Oregon Health and Science University): It's really to see if putting these stem cells into the brains of these children is safe and doesn't cause side effects. We hope that there may be some benefits, but the families have to understand going into this we're not promising by any means any benefit.
PALCA: But you can say that and say that and say that, and a parent can hear it and still not hear it.
Alan Fleischman is a bioethicist at the New York Academy of Medicine.
Dr. ALAN FLEISCHMAN (New York Academy of Medicine): The real challenge for the scientists, is not only to inform the families, but to be sure that the families don't have exaggerated expectations as to what's possible.
PALCA: And because you can't be certain families don't have exaggerated expectations, Fleischman says you have to go beyond simply laying out the facts, especially when you're talking about a potential treatment for a fatal disease, and especially when you're talking about children.
Dr. FLEISCHMAN: Research projects like these require what some of us have called special scrutiny.
PALCA: In other words, taking a second and a third and a fourth look at a study to come up with the best estimate of the likelihood that someday the procedure will keep sick children alive and weigh that potential against the risks - the risk any brain surgery involves and the unknown risk of injecting stem cells into the brain.
Fleischman says it may even be worthwhile to bring in an independent advocate to make sure parents are being realistic about the risk they're exposing their kids to. Lance Johnson says the parents of children with Batten Disease know the score.
Mr. JOHNSON: Parents who are going to have their children treated have done their background work. They've made informed decisions about this. They understand the risks that are being taken. And in spite of the risks that are inherent to any type of invasive procedure like this, they're willing to take these risks in the hope that the procedure, the treatment, will save their child's life.
PALCA: It's not as if everyone will say yes to an experimental therapy. But Johnson says those who do know what they're going. Even if it doesn't help their child, some parents will participate because it might help others in the same boat.
And Eric Kodish says that's a good thing. Kodish is at the Cleveland Clinic Foundation. He's just written a book about children and research. He says if anything, children are being overprotected when it comes to medical research.
Mr. ERIC KODISH (Cleveland Clinic Foundation): That's resulted in lack of information about medicine for kids. Pediatricians in their offices don't know the right dose to use all the time. We don't get new cancer drugs, we don't get new drugs for neurologic diseases, we don't have drug development or device development for that matter for kids the way that we have it for adults. And that's an ethical problem.
PALCA: Kodish says there is one area of medicine where children have participated. The majority of children with leukemia can now be cured.
Mr. KODISH: It's one of the great success stories of modern medicine and it's because of careful clinical research that's been done. And it's a tribute to the children and their families that have been the participants in these studies really.
PALCA: For the parents of children with Batten Disease, the choice is to do nothing or enroll your child in an experimental research study at the very earliest stage. Lance Johnson understands that it can be alarming to be at the frontier of medical science.
Mr. JOHNSON: We don't really know for sure what will happen once the stem cells are injected into the brain. Because once they're injected, you can't take them back. So the children and the families who are going to have this done are in fact pioneers.
PALCA: The scientists carrying out this research are reluctant to talk about it. They won't give interviews and they're encouraging the families who are participating not to talk about it either. They have promised to publish the results of the research, good or bad.
Joe Palca, NPR News.
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