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Experts balk at large trial of stem cells for autism

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Nidhi Subbaraman
14 July 2014

Cold comfort: Researchers are trying to find out whether stem cells taken from frozen cord blood can improve autism symptoms.

A team at Duke University in Durham, North Carolina, is set to launch a $40 million clinical trial to explore stem cells from umbilical cord blood as a treatment for autism. But experts caution that the trial is premature.

A $15 million grant from the Marcus Foundation, a philanthropic funding organization based in Atlanta, will bankroll the first two years of the five-year trial, which also plans to test stem cell therapy for stroke and cerebral palsy. The autism arm of the trial aims to enroll 390 children and adults.

Joanne Kurtzberg, the trial’s lead investigator, has extensive experience studying the effectiveness of cord blood transplants for treating various disorders, such as leukemia and sickle cell anemia1. Most recently, she showed that cord blood transplants can improve the odds of survival for babies deprived of oxygen at birth. A randomized trial of the approach for this condition is underway2.

“To really sort out if [stem] cells can treat these children, we need to do randomized, controlled trials that are well designed and well controlled, and that’s what we intend to do,” says Kurtzberg, professor of pediatrics and pathology at Duke. “We firmly believe we should be moving ahead in the clinic.”  

Early animal studies have shown that stem cells isolated from umbilical cord blood can stimulate cells in the spinal cord to regrow their myelin layers, and in doing so help restore connections with surrounding cells3. Autism is thought to result from impaired connectivity in the brain. Because of this, some groups of children with the disorder may benefit from a stem cell transplant, Kurtzberg says.

But others are skeptical of the approach. Autism is a complex disorder with many possible causes. Also, it’s unclear how stem cells derived from cord blood can improve connections in the brain. Given these important caveats, it’s too soon to conduct a test of this scale and investment, some experts say.

“It’s probably premature to run large trials without evidence that they have a therapeutic effect that [we] understand,” cautions Arnold Kriegstein, director of the Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, San Francisco.

Pilot trials:

In June, Kurtzberg launched the first phase of the trial, with 20 children between 2 and 5 years of age. Her team plans to infuse the children with a single dose of their own cord blood cells, banked at birth and preserved by freezing.

For this stage of the trial, the team is recruiting children who have an autism diagnosis but who do not have other genetic disorders, such as the related Rett syndrome.

In later stages, the trial will deliver cord blood cells from donors to children and adults with autism. For individuals with a known genetic link to autism, Kurtzberg expects that donor stem cells — which presumably will not have the mutations that might have caused autism — are more likely to treat the condition than the individuals’ own banked cells. Also, donor cells will allow more people to benefit from this procedure, she says.

In animal models — and in one postmortem brain from a child who had been treated for a different disorder — Kurtzberg’s team has found that donor cord blood can cross the blood-brain barrier that keeps the vast majority of molecules and cells out of the brain. The cells enmesh themselves in brain tissue and develop into specialized cells, such as microglia, Kurtzberg says. The stem cells may also send chemical signals that travel through the blood and stimulate recovery of inflamed or injured sites, or encourage connections to form where there were none, she says. Depending on the need, these signals vary.

Kriegstein is skeptical of these proposed mechanisms, however.

“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.

What is clear so far is that the treatment at least causes no harm to people treated with their own or donor cells for a range of disorders, according to pilot studies by Kurtzberg and others2, 4.

In the meantime, the first stem cell trial for autism in the U.S. is about midway through its course. That trial includes 30 children with autism between 2 and 7 years of age and began in 2012. In the trial, the children, who have a diagnosis of autism but no known genetic risk factors, receive a single infusion of their own banked cord blood.

After six months, the researchers record any changes in behavior and learning abilities, as well as any changes to their autism symptoms, measured by tests such as the Autism Diagnostic Observation Schedule.

“We hope to demonstrate to people [whether] this is worth pursuing or not,” says principal investigator Michael Chez, a pediatric neurologist affiliated with the Sutter Health Institute, a nonprofit health network based in Sacramento. Chez says he anticipates that some subtypes of autism may respond better to this procedure, and part of the goal of his pilot study is to flag those groups.

Kurtzberg’s trial “will have much better statistical information than our smaller study,” Chez says. “I just hope that there’s data there to justify this investment even before pilot studies like ours [have concluded].”

Several clinics outside the U.S. also offer cord blood stem cell treatments for autism. Parents travel to secure the procedure for their children with autism, and some have informally reported signs of improvement.

Kurtzberg sees her trial as an answer to the calls from researchers for a more rigorous assessment of this procedure.

“There’s no question Kurtzberg is the right person to do this,” says Emanuel DiCicco-Bloom, professor of neuroscience, cell biology and pediatrics at the Rutgers Robert Wood Johnson Medical School in New Jersey. Still, he says, “I think it’s early times.”

News and Opinion articles on SFARI.org are editorially independent of the Simons Foundation.


1. Kurtzberg J. et al. Blood 112, 4318-4327 (2008) PubMed

2. Cotten C.M. et al. J. Pediatr. 164, 973-979 (2014) PubMed

3. Dasari V.R. et al. J. Neurotrauma 24, 391-410 (2007) PubMed

4. Sun J. et al. Transfusion 50, 1980-1987 (2010) PubMed


Name: passionlessDrone
14 July 2014 - 1:44PM

I'm not a neuroscientist, but this seems insane.

If I remember correctly, the Chez study was based on the idea that because of altered immune biomarkers in some children with autism and the immunomodulatory/immuno-normalizing behaviors of stem cells, then maybe they could help. Somehow. It would seem that we have *other* agents capable of producing similar immune modulating effects with far well more established safety profiles compared to stem cells. This study doesn't seem to be taking that track, but rather, a notion that somehow the stem cells will just know how to repair 'impaired connectivity'; as if the *problem* with autism is that it's all impaired connectivity all over the brain, in the same way, as opposed to different connectivity in different areas of the brain.

The idea that we know that this is a 'safe' therapy is a total joke; it is at least admitted that the other studies on safety were 'pilot', with around 200 children having been given the cells with subsequent monitoring of a year. The cancer risk in the treatment group? Who knows! At least the money train is rolling.

It would be great if stem cells can cure autism. But this looks like a very expensive stab in the dark.

Name: Paul Knoepfler
14 July 2014 - 11:26PM

This was a thoughtful article that presented both sides to a controversial area. I've been studying stem cells for years. For my take on this study (and this article) see here on my lab's blog: http://www.ipscell.com/2014/07/duke-launches-large-debated-stem-cell-trial-for-autism/


Name: Wanda
15 July 2014 - 2:17AM

gostaria de saber se vai valer para os autistas adultos, como é o caso do meu filho q tem 21 anoos!

Name: Amanda
15 July 2014 - 3:49AM

Dr. Chez has been at the forefront of treatment for ASD children and adults with epilepsy and immune dysfunction for a decade. He has personally helped the ASD children of so many parents I know. While we are so eager to throw millions at Seaside and various blind pharma trials why shouldn't Dr. Chez move forward here? He really understands the immune aspect of autism and treats some of the toughest ASD cases. Chez is a real innovator and ASD research desperately needs more scientist/ clinicians like him.

Name: ApraxiaDad
15 July 2014 - 5:05AM

Absolutely agree with Amanda - frankly this trial is a damn sight more relevant than the truly insane Chelation Therapy. Early times, of course, but it has to start somewhere, some time.

Name: Saadia Akhtar
15 July 2014 - 7:28AM

It is a strange world when researchers work for and get funding to test if chimps can write a Shakespeare (http://news.bbc.co.uk/2/hi/3013959.stm) and yet not a single soul from west is interested in growing claims from Pakistan that farm fresh foods reduce autism and baby cereals, baby milk and vaccines together worsen autistic traits. Surprisingly, they are also not interested in the cases where autistic children are having their hearings restored in Pakistan.
The money spent on chimps would have been enough to confirm these claims originating from Pakistan ( http://www.amazon.com/Reducing-Autism-Poisoning-Impacts-Adeerus-ebook/dp/B00LHIJLAY ). And a fraction of millions of dollars spent above would be enough to test the hypothesis in a controlled environment in any western nation.
But then, this is how the world is. If the solution is easy, straight forward and in front of your eyes, it cannot be. It has to be complicated, difficult and expensive - something with which big pharmas can make money and scientists can get famous.

Name: ASD Dad
15 July 2014 - 11:43AM

Whilst stem cell trials are needed one feels that a more specific focus be bought to two areas that intersect - biomarkers and phenotype before we move ahead on treatment regimes that may target the 'wrong' subset. One hopefully can avoid the mistakes that 'blunt tool' of epidemiology has given us.

Thus whilst clinical trials must go ahead they should gather as much 'ecological' data of each patient particularly data surrounding biological biomarkers such as c-reactive protien and urinary p-cresol come to immediate mind.

There is also some solid evidence that we can change immune function and key autism behaviors by addressing gut/immune issues as per Sarkis Mazmarian
at SFARI webinar with what might be less elaborate and as one commentator potentially dangerous stem cell interventions.


Perhaps a hierarchy of intervention be planned at a higher level with substantial funding and resources provided by government. There is a need to move quickly on a disorder of childhood that is looking to effect at least 2% of the population in the very near future.

As always just some thoughts.

Name: Dadvocate
15 July 2014 - 4:19PM

One correction, Amanda. Chez has been a positive factor in autism/epilepsy circles for a lot more than a decade. However, I don't think he gets the respect or acknowledgment he deserves. His open mind to new ideas (like stem cells) while issuing firm warnings to avoid potentially risky or harmful non-evidenced based therapies, makes him a voice worth listening to, but that approach doesn't endear him to all, especially those who think they have "the answer". As an outgrowth of his large scale clinical practice, Chez has championed (and published for many years)on the critical importance of autism subtypes (which likely require different therapeutic interventions) as well as the general autism/epilepsy connection. His book is well worth reading.

Chez's primary role as a clinician may have biased some academic institutions and grant makers, but I think he's added a lot more to the body of knowledge of ASDs with a lot less funding than many of the "leading names".

Name: Al
4 August 2014 - 6:46AM

Come up with a good idea and you will find 10 people tell you why it won't work.

Name: tmcd
28 October 2014 - 6:23PM

My severely autistic 7 yr old nephew is part of the study at Sutter. After 8 months of placebo and testing he was finally given his own chord blood in May. We have definitely seen large improvement starting around June. We have greater attention and comprehension from him and recently he requested a song be played again. He has also learned to tell you his name when asked. Surprised to read the comment about cancer risk from passionlessdrone. these children are receiving their own chord blood. If they were at risk for cancer, they would be so without having the treatment as well. We are greatly encouraged by the improvement we see. We saw no change the first 8 months not knowing whether or not he got the actual blood. Now it is confirmed that he received it in May. The experts said the cells start to triplicate in approximately 6 weeks and that's when we would see changes if the treatment worked. And that is what happened.

Name: ASD Father
22 July 2015 - 12:31AM

reading your story make me so optimist, and I really wanna try this for my 3 years old son who has severe autism and he's non verbal.can you please share more of your story.
you can also reach me at:

Name: Sarah
8 December 2014 - 4:30PM

Our son has received stem cell therapy to treat his autism and is now recovered. Stem cells do work and they work on much more. You can find us at www.stemcelltherapyforautism.com and on FB. Best wishes, Sarah

Name: Judy
24 January 2015 - 5:23PM

Hi Sarah, would you be willing to share your story with me? I have a 13 year old son with PDD-NOS, and we've tried almost everything, with some improvements along the way, but no recovery. I feel so desperate for more drastic improvement.

Name: amelie
31 January 2015 - 3:28AM

would love any information on how your children imoroved. my son is 10, I am very willing to try and desperate to try any safe treatment option.

Name: ASD Mama
19 June 2015 - 5:55PM

We are in the Duke Trial. Our son has had amazing results. I have spoken with families all over the world. For certain sub groups of autism, this works.

Name: WJB
17 July 2015 - 3:34PM

Hi. We have a 10 year old non-verbal son with autism. He communicates with sign language and has a couple of spoken words but is not conversational. Is there a way we could communicate about your child's experience in the trial? My email is williambrittain@ymail.com and I'm on FB, but I don't know how you'd locate me there, lol.

Name: ASD Dad
22 July 2015 - 12:23AM

reading your story make me so optimiste and I really want to try this for my 3 years old who has autism and he's non verbal, can you please share more of your story, you can also reach me at my email:

Name: DCK
23 July 2015 - 2:01AM

Please provide information on how to participate in the Duke trial.

Name: Greg Boustead
6 August 2015 - 2:36PM


Joanne Kurtzberg, who runs the trial at Duke, gave us this update: "The trial is ongoing but has enrolled all of the patients already. We are in the follow-up phase. A larger trial is planned for 2016."

-Greg Boustead

Name: Anita B
28 August 2015 - 3:16PM

Would you be so kind and provid me with Dr. Joanne Kurtzberg's e-mail address. It's not listed on the hospital's website. Any assistance is greatly appreciated.niconichole@gmail.com

Name: Greg Boustead
1 September 2015 - 9:18PM

Hi Anita,

You can use the following email address: cordbloodtherapyinfo@dm.duke.edu

-Greg Boustead

Name: Michelle
25 August 2015 - 11:50PM

Yes, I am having difficulty of finding contacts at Duke. My son just turned 3 and this is something very new to us. I am open and willing to learn about all avenues that can potentially help. Any guidance or help would be tremendously appreciated. Thank you!

Name: Greg Boustead
25 August 2015 - 11:59PM

Hi Michelle - The Duke study discussed in the article is no longer enrolling participants.

-Greg Boustead

Name: Anita B
28 August 2015 - 2:48PM

Regarding Duke study. The first round is closed, however, recruitment for Phase 2 has not started; confirmed today by someone who works on team.

Name: Greg Boustead
1 September 2015 - 9:17PM

For anyone interested in contacting the research team at Duke about to inquire about enrollment for possible future phases of this or related studies, they provided the following email address: cordbloodtherapyinfo@dm.duke.edu

- Greg Boustead
SFARI.org, community manager

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