Stem cell trial for autism: hope vs. caution

Last week brought a media and social media frenzy over the announcement that a new clinical trial would be testing the effect of injecting cord blood into kids with autism.

The trial—and the conversation surrounding it—illustrates a tension in stem cell science between the hope of the patients and caution on the part of scientists.

What’s important to remember is that this is just a trial. Often, I see new trials heralded as proof that stem cells are working, especially by those who really want to believe in that trial. New trials testing adult versions of stem cells, for example, are heralded as proof of the effectiveness of adult stem cells compared to their embryonic counterparts.

A trial is just that—a trial. More early stage clinical trials fail than succeed. That’s how science moves forward. Right now it’s easier to get a trial started with adult stem cells than with embryonic stem cells, and so there are many more of them. But trials don’t equal success.

Given that this trial may or may not provide any relief for kids with autism or their parents, it’s too soon to be celebrating.

Scientist and science writer Emily Willingham has written about her concerns regarding the science behind this trial. She characterizes the excitement as “stem cells are hot and autism is hot so lets throw some stem cells at autism.” Her blog entry is worth a read. In it, she discusses her concerns about the scientific rational for using cord blood as a therapy for autism.

However, a person commenting on Willingham’s blog makes exactly the point we often struggle with at CIRM. That is, when is it the right time to take risks. The commenter Chris August notes, “As a parent with a child with mild Autism I cheer progress.” He goes on to make the point that even if the stem cells aren’t a magic wand, there’s no harm in testing them. (Willingham herself has a child with autism, which she mentions in her response.)

In a story in Nature’s blog, the lead investigator of the trial Michael Chez gives his own reason for why it’s important to be testing blood cells for autism even if the science behind the trial isn’t strong. Cord blood cells and fat stem cells are being administered overseas as a possible treatment for autism.

“Our research is important because many people are going to foreign countries and spending a lot of money on therapy that may not be valid.”

He’s right. CIRM is one of many organizations and individuals who are worried about the rise of stem cell tourism, in which unregulated clinics overseas promise miracle cures at a steep cost (here’s information on our website about stem cell tourism). Those miracle cures are often based on shaky science, or have no scientific basis at all. They aren’t part of clinical trials and outcomes aren’t recorded. At least in the case of this trial, people who participate will be followed and any benefit or lack thereof will be recorded and could help guide future trials.

On our own governing board this tension between funding the very best science and taking risks to push the science forward for life-threatening diseases comes up routinely. Our patient advocate board members, who make up 10 of the 29 members, sometimes push for funding for science that didn’t get the highest scores from our scientific reviewers. Their point: maybe it’s not a sure thing, but it might help us learn something important.


2 thoughts on “Stem cell trial for autism: hope vs. caution

  1. I think the real controversy came when people were saying that this new study would be a 'cure' for autism. That is a very, very big claim and shouldn't be said when the study hasn't even been conducted yet.

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