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Stem cells to treat autism: premature trial or worth the attempt?

Researchers from Duke led by Joanne Kurtzberg are testing to see whether treating children recently diagnosed with autism will benefit from an infusion of stem cells. For early trials, these stem cells will have come from the cord blood of the specific child, which was harvested and stored at birth. Later on, this can be attempted with stem cells from a separate donor, which have more clinical use for those who didn't save their cord blood.

This approach isn't without its criticism, as reported today by SFARI.org. Arnold Kriegstein of UCSF is very vocal as to why it's a potentially bad idea to set up such a trial at this time:

"These are not cells that can treat a laundry list of diseases," he says. Because the stem cells are similar to those that normally give rise to blood cells, he says, it is unlikely that they can repair or replace neurons in the brain. Also, because autism results from errors during development, it is unlikely that the stem cells can reverse those effects.

"I think it would be marvelous if this trial worked, but it really seems more like a 'Hail Mary pass' than a rational therapy," he says.


Current research suggests that autism is a complex state with many potential factors affecting its development. Plus so little is known as to how effective stem cell treatments will be, and what effects these cells will have in the body.

At least preliminary results seem to show few ill-effects in those who received an infusion of stem cells. If parents are well aware as to how unlikely this is to work (a huge if!), is it OK that this trial is being attempted on the off chance it does work? Or is this not worth it until we know a little more about the biology of autism?


My gut leans towards this is premature and not worth the cost, the raising of hopes, and the potential long-term risks to the kids. What do you guys think? Any compelling arguments for it?

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