Doctor: China's Stem-Cell Therapy for Kids Is Risky Optic nerve hypoplasia is a condition in which nerves in and around the eye fail to develop properly. Some parents of affected children are seeking controversial stem cell treatments in China. Dr. Mark Borchert, head of the Vision Center at Children's Hospital Los Angeles, urges caution.
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Doctor: China's Stem-Cell Therapy for Kids Is Risky

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Doctor: China's Stem-Cell Therapy for Kids Is Risky

Doctor: China's Stem-Cell Therapy for Kids Is Risky

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In this part of the program yesterday we heard about a controversial treatment for a condition that is a leading cause of blindness in children. The medical term is optic nerve hypoplasia, a condition where nerves fail to develop properly.

Yesterday NPR's Louisa Lim followed an American mother in China seeking an untested treatment for her baby using stem cells. Jenna Teague of Georgia believed her daughter had been helped.

Ms. JENNA TEAGUE: When we got over here we've had so far three injections of a stem-cell through IV. And now she sees light out of one eye, and the other one is actually dilating almost to where she can see light.

LIM: So with such a small baby, how can you tell what she's seeing?

Ms. TEAGUE: I can't tell anything. I'm just - that's what the doctors are saying.

MONTAGNE: Jenna Teague tried the treatments against the advice of specialists back home. Dr. Mark Borchert is head of the Vision Center at Children's Hospital here in Los Angeles. When parents ask him about this treatment for their children, this is what he says.

Dr. MARK BORCHERT (Children's Hospital, Los Angeles): I tell them I have a great deal of difficulty with the therapy on two levels. First of all, from a scientific point of view it makes no sense that stem cells harvested from cord blood if they are injected into the spinal cord, which is what they were purportedly doing - and more recently reports are that it's been given by IV injection - it makes no sense that those cells could migrate to the eye, form new neurons within the optic nerve, and also form accurate connections within the brain. And certainly it makes no sense that that's possible within the timeframe that they are describing.

MONTAGNE: Which is weeks...

Dr. BORCHERT: Which is days to weeks. It doesn't even happen that quickly in the developing fetus. And these neurons have to grow much further in fully developed child.

MONTAGNE: That's what you're telling...

Dr. BORCHERT: I'm telling them that's the problem from a scientific point of view. I have much bigger problems with it from an ethical point of view. First of all, you should not subject people, much less innocent children, to therapies that are not yet proven in animals or in the laboratory.

And second of all, such radical therapy really should be done under careful monitoring, with independent observers validating that the techniques that are used are proper and that the outcomes in terms of benefit are clearly documented in an objective fashion. And that is not the case of what's going on in China.

MONTAGNE: Although if you're getting calls from parents who have a child with this condition who are probably looking for any treatment they can get, and since you, a Western medical doctor, are not offering them any treatment, is it hard to make your case?

Dr. BORCHERT: It is hard, because this is a desperate situation for these parents. Unfortunately, desperate situations often lead to vulnerability of these parents. It really makes no sense, for instance, if they were truly doing what...

MONTAGNE: That is the Chinese doctors.

Dr. BORCHERT: If the Chinese doctors are truly doing what they're claiming, which is merely harvesting stem cells from cord blood and injecting it in the vein, it makes no sense that they should charge $20,000 for such a simple treatment.

MONTAGNE: You know, just one last thing. In Louisa Lim's story, there was some talk of some of these young children getting better based on a placebo effect. Is that likely in your experience?

Dr. BORCHERT: The placebo effect is not an effect on the child. The placebo effect would be effect on the parent. If the parent believes that their child has absolutely no vision and no potential for vision, and then they start to notice visual behavior afterwards, they are likely to attribute it to the treatment for which they spent thousands of dollars for. In fact, more than 50 percent of children with optic nerve hypoplasia have spontaneous improvement in their vision without any treatment, and to the level that is being reported in the kids from China and sometimes even to greater levels than what is being reported from the kids in China.

MONTAGNE: Thank you for joining us.

Dr. BORCHERT: Thank you.

MONTAGNE: Dr. Mark Borchert heads the Vision Center at Children's Hospital Los Angeles. For more on optic nerve hypoplasia, options for treatment and to hear our earlier report, go to This is NPR News.

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