June 6, 2020

The Niche

Knoepfler lab stem cell blog

Why I do not believe in stem cells for autism today

There is a critical need for treatments for children with autism. 2020 update: see this more recent post on my thinking on stem cells for autism.

autism stem cells trial
Autism stem cells trial made news in the Sac Bee.

About a year ago I wrote about a clinical trial at Sutter Health in Sacramento that was approved by the FDA for stem cells for autism.

At that time I was relatively less concerned about the trial. Over time, as I’ve learned more, I’ve gotten quite worried about it, a concern I discuss in my new book on stem cells.

My concern has been amplified by the sharp increase in the number of parents contacting me with questions as they are considering getting stem cell “treatments” for their autistic children. Some of the clinics offering such treatments are right here in the US, while others are in foreign countries. I believe that hundreds, perhaps even thousands of autistic children are being infused with stem cells around the world each year.

I just don’t see how such an approach would be successful. I’ve become concerned the Sutter trial is being used indirectly (through no fault of those doing the trial itself) by the sketchy for-profit clinics selling snake oil stem cell cures for autism. Some parents have mentioned the Sutter trial as a rationale for getting their kids stem cell transplants from clinics.

For another, extremely helpful view of this trial and great background see this piece by Emily Willingham. This passage from her article especially resonated with me:

“What we have is a few studies suggesting an autism–immunity link, although not all findings support one [paywall], some partial effectiveness of cord blood cell infusion for cerebral palsy, and no data regarding what effect, if any, a cord blood infusion would have in autism. The rationale for the work appears to be the CP trials and Patterson’s work with a mouse model of inflammation.

By Patterson she is referring to Caltech neurobiologist Paul Patterson.

The families with children who have autism that contact me almost weekly now have 2 main questions that they ask me:

“Should we get a stem cell treatment for our son (or daughter)?”

“What specific clinic do you recommend?”

I find myself in the difficult position of telling them gently that I do not believe in treating autism currently with stem cells. I briefly tell them why. I explain that there is hope for the future in this area, but we are many years if not decades away.

The reactions range from appreciation for my honest opinion to anger over my skepticism. I understand.

So why don’t I believe in stem cell therapies for autism today?

Basically, there is no convincing data to support the idea.

Let me preface my deeper explanation by saying again that some day stem cells may help autism, but that day is not today and I strongly doubt it’ll be in less than a decade. Willingham is even more skeptical and in an excellent piece last month listed stem cells as one of the top 5 scariest autism interventions. She says bluntly, “There’s not a strong rationale for using stem cells as therapy for autism…”

On the other hand using stem cells such as induced pluripotent stem cells (iPSCs) to study autism, for example by examining a patient’s neurons made from their own iPSCs), to try to better understand the disease has great potential. Still that is very basic even if clinically relevant science meaning that treatments based on such research would be far down the road. Parents of autistic kids do not feel they have time to wait. I understand that.

The biggest problem stem cell treatment wise for autism is that even autism experts do not know what causes this spectrum of disorders. If you don’t know what causes a disease it is awfully tough to treat it with much hope. Most autism experts believe there are multiple, perhaps even diverse causes to autism spectrum disorders, which complicates treatment even further.

What this all means is that doctors and clinics promoting stem cell-based “treatments” for autism today have very little in the way of evidence and frankly sometimes no evidence at all that the treatments will work.

The main rationale given by proponents of stem cells for autism to support the use of stem cells in treating this disease is based on the unproven idea that autism is an autoimmune disease and that stem cell interventions will calm down the immune system. The most prevalent stem cell intervention for autism is IV injection of stem cells into the blood stream through a vein in the arm.

Absent any real data, this is essentially a long shot, a shot in the dark. And the stem cells given to the children have risks that we do not entirely understand today. Further, those risks may last a lifetime since stem cells, unlike traditional chemical medicines, are alive.

Therefore, in the end, I strongly recommend against getting any stem cell “therapy” for autism from a clinic.

Even clinical trials (see list here) for autism using stem cells will have risks and are likely long shots to work as well. However, at least in that context your child has a relatively greater chance of being treated by trained physicians, followed up on carefully, and not be exploited for profit. In addition, you and your child may contribute to helping future generations of autistic children through the knowledge gained from the trial.

The bottom line is that I urge parents to use great caution when considering stem cell interventions for autism or other childhood neurological disorders such as cerebral palsy.

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