September 23, 2020

The Niche

Knoepfler lab stem cell blog

Duke Launches Large, Debated Stem Cells for Autism Trial

Is there real promise of stem cells for autism?

At this point the jury remains out on that question, but a growing number of kids are nonetheless getting such “treatments” at for-profit dubious clinics. Academic clinical researchers are interested in this area as well.

Dawson Kurtzberg launching stem cells for autism trial
Dawson Kurtzberg are launching a new stem cells for autism trial.

Still, the potential use of stem cells to treat autism is a highly controversial area even in a clinical trial setting.

Today it was announced in a piece on the website of the Simons Foundation Autism Research Initiative that a dramatically larger study involving hundreds of autistic children and adults is now being run by a team at Duke led by Drs. Joanne Kurtzberg, M.D. and Geraldine Dawson, Ph.D. (pictured above in image from Duke PR).

2020 Update: Duke’s Phase 2 trial found no benefit of stem cells for autism, but the group plans to continue anyway.

A very surprising and intriguing aspect of the article on the Kurtzberg trial from the Simons Foundation is that it alternately quoted Kurtzberg and Arnold Kriegstein, Director of the Broad Center of Regenerative Medicine and Stem Cell Research in what feels like a debate format. Kriegstein is highly skeptical of the Duke trial, which he is quoted as calling “premature” and “more like a ‘Hail Mary pass’ than a rational therapy.”

The article quotes him as to why he is skeptical:

“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.

It is unusual and valuable to quote researchers in this way with different opinions. I find it surprising how blunt Kriegstein is in his concerns about this trial. The trial was also announced on the Duke Medicine website here. According to the Duke PR, Dawson was “who was the founding director of the University of Washington Autism Center and then chief science officer at Autism Speaks before joining the Duke faculty in August 2013.” Dawson and Kurtzberg are two top-notch biomedical scientists.

The $40 million stem cell autism trial is funded in part by a $15 million gift from the Marcus Foundation in Atlanta. The official name of the trial is “Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD)”.

The primary outcome measure is safety, while secondary measures are potential efficacy as measured by cognitive and behavioral tests.

The trial will also use umbilical cord stem cells to treat other conditions such as stroke, but the team aims to treat 390 autistic people in that arm.

In fact, the trial apparently already started enrolling patients last month including 20 children ages 2-5 years old treated in an autologous fashion.

For more background on the use of umbilical cord stem cells for autism and other disorders see my two-part interview with Kurtzberg here and here.

Two of the difficult aspects of the idea of using stem cells to treat autism are (1) that autism spectrum disorder is a diverse umbrella group of disorders and (2) that it remains unknown what the various causes might be. Another challenge is that stem cells infused IV rarely if ever make it into the brain. At least in part for these reasons, I’m skeptical that stem cells used in the manners proposed can help autism.

autism stem cells

In 2012 a small trial right here in Sacramento by Sutter was launched using umbilical cord blood stem cells to treat autism. You can see the SacBee headline announcing it that I read at the breakfast table.

The Duke trial official clinicaltrials.gov listing can be read here for more details.

For example, the dose used with be 10-50 million nucleated cells/Kg of subject weight.

I wish we knew more about the pathogenesis of autism, which is a fancy word for saying what causes it, before we proceeded with these kinds of trials. On the other hand, I can see where such a trial could greatly advance knowledge and there is an urgent and seemingly growing need. Overall, I have very mixed feelings.

What are your thoughts on this trial? On the idea of using stem cells to treat autism? Perhaps we can have pro and con readers have a fruitful debate right here in the comments as well.

%d bloggers like this: