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Microbiome: From Fringe Idea to a New Frontier for Autism (Finally)

We want to highlight two recent studies and their press coverage that indicate potentially promising developments for autism.

Two Line Summary:

  • Stool (gut bacteria) from children with autism caused mice to develop autism-like symptoms.

  • Stool from healthy donors improved autism symptoms in both mice AND CHILDREN WITH AUTISM.

Got your attention yet? Here is a little more about the important findings.

Study #1: Fecal Microbiota Transfer (FMT) in Children with ASD

The first report is the recent release of the two-year follow-up data from the autism fecal microbiota transplant (FMT) trial that was done at Arizona State University. The original trial, completed in 2017, showed meaningful clinical and behavioral improvements in the children that received an FMT.

What is new is that when they recently followed up with these children two years after their last dose of FMT, the results continued to improve! This opens up the provocative notion that the FMT may have restored a healthy microbiome and led to improvements in core autism symptoms.

Some attention grabbing headlines include:

Autism symptoms reduced nearly 50% 2 years after fecal transplant

At the start of the study, 83% of participants were rated as “severe” autism. At the end of the study, only 17% were “severe,” 39% were “mild/moderate” and 44% no longer met the criteria for autism.

Here is a link if you'd like to read more

It's important to bear in mind that this trial did not have a control group so there is always the potential for a placebo effect. Also, the trial began with a treatment of vancomycin, which as we have covered in this blog and in our Wall St. Journal Op Ed was shown in a small trial in 1999 to result in a dramatic, albeit temporary improvement in autism symptoms. An open question is whether the improvements being seen because the FMT allows for the vancomycin benefits to be more durable?

More trials will need to be done to better understand how safe is FMT, and how well does it work in the general population of autism, but the early results look very encouraging and seem to validate the idea that microbiome matters, at least for some with autism.

Study #2: FMT in Mice

Cal Tech's Sarkis Mazmanian, Ph.D

The second study we highlight was done by a dream team of autism researchers led by N of One supported researcher Sarkis Mazmanian at Cal Tech. In this study Dr. Mazmanian and his team asked the question "what would happen if you transplanted stool from children (with and without autism) into genetically identical mice?"

What happened is pretty remarkable and noteworthy. When the stool from children with autism was transplanted into mice, the offspring of those mice developed autism-like behaviors (such as decreased vocalization or increased repetitive behaviors). Interestingly these behaviors were more pronounced in male offspring than female.

Mice that that received stool from typically-developing children, did not develop autism-like symptoms

What was particularly interesting is that when the mice with the autism-like symptoms received stool from the typically-developing children, there was a marked reduction in autism-like behaviors (kind of like what happened in the human study (#1) above).

The Media Coverage and Why it Matters

Much is being written about these studies and what it means but probably the most important and encouraging comes from the Economist which has long been at the leading edge of autism research.

Here is the opening paragraph from the Economist article just published titled: More Evidence That Autism is Linked to Gut Bacteria

"PARADIGM SHIFT is an overused term. Properly, it refers to a radical change of perspective on a topic, such as the move from the physics of Newton to the physics of Einstein, or the introduction of plate tectonics into geology. Such things are rare. Something which history may come to regard as a true paradigm shift does, however, seem to be going on at the moment in medicine. This is a recognition that the zillions of apparently non-pathogenic bacteria on and in human bodies, hitherto largely ignored, are actually important for people’s health. They may even help to explain the development of some mysterious conditions. One such condition is autism"

If this sounds vaguely familiar to long-time N of One supporters, it's because when we launched N of One in 2014 I wrote the following Huffington Post:

"I believe we are on the verge of a paradigm shift in autism where the new view is that, while some genetic factors may be important, the underlying condition is more of an acquired syndrome that arises from externally-induced changes in metabolism, immune function, and the microbiome."

The encouraging thing about this view is there is reason to believe that many of these children can get better. We need to fund this kind of research. Our children deserve better answers."

So in the past five years we have moved from being "on the verge" to being in the midst of the much needed broadening of our autism research focus, and it's already paying dividends!

Beyond the encouraging news, there's an important, perhaps broader message here:

When I speak with parents, and being a parent myself, I understand that it can be hard to make the connection between supporting research and having something tangible that can help your child. The two can seem too far removed.

But in less than 5 years, we have gone from the microbiome in autism as being a "fringe idea" to having a promising treatment undergoing FDA clinical validation - and we are just at the beginning! There are many more treatments and prevention strategies that will come from research focused on the microbiome.

To me, this is proof that the strategy of funding and advocating for research that goes beyond the traditional views, but is in-line with the emerging data, works and can result in treatments for our children in their lifetime.

We hope you see it similarly and will join us in sharing these encouraging developments and supporting more research like this. If you've been a supporter from the beginning, Thank You! We hope you will continue to stand with us. If you have not supported our efforts before, we would ask that you join us and make your first donation of $10 right now be part of our effort for better answers and treatments for our children.

N of One: Autism Research - We have an innovative approach

to autism research and we are getting results!

Want to learn more about microbiome in autism? Read our blog post Why The Microbiome Matters in Autism

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