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Autism Speaks & GI Research: Listen to Families, Build on Science

By Autism Speaks Chief Science Officer Rob Ring and Paul Wang, Autism Speaks senior vice president for medical research


This week we’re very excited to announce that Autism Speaks is funding not just one but two proposals for research into autism-related gastrointestinal (GI) issues. (See our related news story here.) This increases the funding of our new GI initiative to $2.3 million, over 3 years.

For many years, our families have been educating the medical community on how very common it is for autism to be accompanied by GI problems. They’ve told us how the pain of these conditions can worsen their children’s autism symptoms.

Research conducted through the Autism Speaks Autism Treatment Network (ATN) has helped to convince the medical community that these family anecdotes are both accurate and important. In the fall of 2012, this progress was reflected in a series of guidelines on the management of autism-related medical conditions – including chronic constipation – published in the respected journal Pediatrics.

However, we saw that publishing these guidelines was not enough to ensure that they were widely followed beyond the doors of our ATN centers. We knew that one of the best ways to increase the use of medical guidelines would be to develop irrefutable evidence of their benefits. In the case of autism and constipation, these benefits include more than relief of the associated pain and discomfort. They also include improvements in behavior and daily function.

The first of our two new GI grants addresses this issue. It’s just the kind of “roll up your sleeves and get the basics done” science that will ensure that more individuals with autism-related GI issues will get the effective treatments they need.

The second of our new GI grants follows up on the exciting findings of an Autism Speaks Trailblazer grant. This investigation showed that, in a mouse model of autism, changes in intestinal bacteria (the “microbiome”) can affect social behavior. On top of that, treating the mice with a probiotic both altered the bacteria living in their gut and improved their social behavior.

To follow-up these breakthroughs, Autism Speaks issued a “Request for Applications” to the research community earlier this year. It came with a commitment to provide at least $1.5 million, over 3 years, to support new investigations aimed at advancing our understanding of autism’s “gut-brain connection” and put these findings to work for our families.

More specifically, we asked for clinical research proposals. Clinical research refers to studies that involve people – in this case, individuals who have autism. We felt this vitally important because we want research that can improve our understanding and treatment of autism-related GI problems in the near future.

The response to our request for research applications was enormous. We received letters of interest from more than sixty top research teams across North America, Europe, Asia and Australia. Many of the GI experts who applied are new to the field of autism research. This is a great achievement for us. We need researchers at the top of other fields to see the unmet needs of the autism community as an opportunity to use their expertise to help our large community.  

The science team evaluated this first round of brief applications, which we call Letters of Interest, or LOIs. This initial triage allowed us to narrow down a group of finalists based on the soundness of their project’s science and potential benefits for our community. We asked the finalists to submit full research proposals. With the help of scientific experts and family advisors, we have been poring over these final applications ever since.

The first research grant will go to research teams headed by James Versalovic, at Baylor Medical College. The second grant goes to teams led by Pat Levitt, at Children’s Hospital of Los Angeles (CHLA) and University of Southern California. We are proud to note that both research teams include scientists and clinicians from three of our ATN sites – CHLA, Nationwide Children’s Hospital (Columbus, Ohio) and one of our newest ATN sites – the University of California, Irvine.

In this blog post, we’d like to give you some background on why we considered their research proposals the most promising of the many quality projects submitted.

Autism and the human microbiome
Dr. Versalovic’s project follows up on the recent mouse microbiome discoveries described above. He and his team will examine the microbiome of 375 children, using stool samples. They will also carefully characterize these children’s GI and behavioral symptoms to look for associations with their intestinal bacteria. Importantly, the participants will include some children with autism and others without. It will include both children with GI symptoms and those free of such problems. In this way, the researchers will be able to determine whether specific types of gut bacteria are tied to GI symptoms or behavioral symptoms or both.

What does this research promise when many families are already giving probiotics to their loved ones with autism? The answer lies in the complexity of the microbiome. Thousands of different types of bacteria live in the human gut. The probiotics currently on the market contain only a few types of bacteria. A small number of these probiotic products contain bacteria that have shown clear health benefits in scientific studies outside the field of autism. However, none of them have been rigorously tested and shown to benefit individuals with autism. We can’t assume that just because something works in one population, it will work in the same way, or with the same dose, or at the same time in a different population. One of the most important questions is which bacteria will be of benefit for specific symptoms. So if a company wanted to create a new probiotic for autism, it would need such answers.

Dr. Versalovic’s research team will determine exactly which bacteria – if any – are abnormally high or low in persons with autism. They will identify the metabolites, or byproducts, that these bacteria produce. This will help us understand possible biological effects.

Importantly, the researchers will look for associations between gut bacteria and their metabolites and the children’s behavioral and GI symptoms. We expect their findings to guide the development and improvement of new evidence-based probiotics tailored for autism – products that have the real potential to improve quality of life.

Delivering relief “here and now”
While Dr. Versalovic’s microbiome research lays the groundwork for future treatments, Dr. Levitt’s research promises to deliver “here and now” benefits for our community. In the words of one of our scientific reviewers, Dr. Levitt’s project has the potential for “quick and meaningful impact on the well-being of many children with autism.” 

Past research by our ATN and others has made clear that constipation is one of autism’s most common medical complications. Our ATN specialists have even pioneered gold-standard guidelines on how best to treat the problem. (See “Helping Pediatricians Improve Treatment of Autism-Related Constipation.”)

Unfortunately, outside of autism clinics such as the ATN, our children’s GI problems go unrecognized far too often. We know that many of our families struggle to have their child’s GI issues properly recognized and adequately treated by their physicians. Too often, we see physicians use psychiatric drugs to treat behaviors that may be associated with GI pain. This needs to change. The best way to effect such a change is to invest in research that is laser focused on generating evidence that GI treatments work.

Then we must make sure this information is disseminated to as many physicians as possible. This would seem obvious, but there really isn’t any documented scientific evidence that GI treatments help with associated autism symptoms.

We expect that Dr. Levitt’s research will clearly demonstrate – for the entire medical community – the wide-ranging benefits of effectively treating constipation in individuals with autism. His team will treat 120 children with autism and constipation, following gold-standard guidelines. They will track these children for an entire year, assessing both GI symptoms and any changes in behavioral symptoms.

We believe that they will find that effectively treating constipation brings significant improvement in behavioral symptoms. If this is, in fact, the case, their report will be invaluable in motivating healthcare providers to invest the time and effort to assess for constipation in individuals with autism and treat it thoroughly.

We need this clear evidence if we’re going to get the medical community to implement effective treatment for everyone with autism – just as we needed clear evidence on the benefits of early intervention to get it implemented across the country.

In addition, Dr. Levitt’s study will have an interesting twist. Many of us have heard about the importance of including anti-oxidants in our diet. According to one theory, autism can be caused or worsened by “oxidative stress” – an imbalance of antioxidants vs. oxidants in the body. Dr. Levitt and his team have published research showing that oxidative stress is elevated in children with autism. They likewise found elevated oxidative stress in children with constipation. They found the highest levels of all in children with both autism and constipation.

In their new, Autism Speaks-funded study, they will be assessing oxidative stress levels before and after successful treatment for constipation. They’ll look for associations between oxidative stress levels and both GI and behavioral symptoms.

The road to new treatments tends to be long and bumpy. But when it comes to treating constipation in children with autism, it’s not a question of how to treat – it’s a matter of getting treatment implemented. We are very hopeful that Dr. Levitt’s project will generate the evidence to make this happen. At the same time, Dr. Versalovic’s project promises to speed the day when we’ll have effective probiotic treatments for autism.

With both of these approaches, we hope to turn autism’s gut-brain connection in our favor – to improve the well-being of individuals with autism.

Unfortunately, we couldn’t fund all of the great project proposals we received during this round of applications. They included research on a variety of dietary interventions and other treatment approaches. We will continue to work with many of these researchers to develop their research ideas. It’s important to emphasize that this research initiative is only beginning. It’s the start of a long-term commitment to grow and mature research into autism and GI health. In the process, we are showing other major research funders the importance of this area of investment. We look forward to their joining us in supporting this important research.

We want to thank the entire Autism Speaks community of families, fundraisers and volunteers for making this research possible. We’d love to hear from you in the comment section below. 

The Autism Speaks blog features opinions from people throughout the autism community. Each blog represents the point of view of the author and does not necessarily reflect Autism Speaks' beliefs or point of view.