top of page

Suramin Trial: What You Need to Know

Results from an N of One: Autism Research Foundation sponsored clinical trial out of University of California San Diego show that a single dose of suramin given to boys with autism resulted in statistically significant, though temporary improvements in the core symptoms of autism.


The importance of the findings go beyond the potential for a new treatment.  They also lend substantial support to the theories put forth by Dr. Robert Naviaux who years ago suggested that underneath the outward symptoms of autism lies a biochemical process that he calls the Cell Danger Response that is amenable to treatment.  An encouraging view, but one that is at odds with the conventional view of autism - as a fixed, genetically-driven condition - that has dominated autism science for decades.

N of One has advocated for years that more funding should be dedicated to exploring Dr. Robert Naviaux's promising research at UCSD as well as other research that lies outside of the conventional view of autism.

N of One: Autism Research Foundation was the first autism non-profit to support the suramin trial at UCSD.  N of One has organized grass-roots fundraisers to support Dr. Naviaux's research and even a letter-writing campaign by parents to the IACC (Interagency Autism Coordinating Committee) encouraging emergency funding for Dr. Naviaux's research.

The trial was successful in: 

  1. Continuing to support Dr. Naviaux's theories that a treatable metabolic disorder may be at the heart of autism (i.e., a common pathway or biology).

  2. Supporting the idea that suramin or other similar compounds might become the first treatments that address the core features of autism.


More work and additional trials need to be done to validate these findings and demonstrate the proven safety and efficacy of suramin.

Understandably, many families that have been touched by autism will have questions about what all of this means.  Below we provide information to address those questions.

Please subscribe so that we can keep you informed about this trial and other promising research.

Here is what you need to know about the suramin trial:



What is suramin and how does it work?


Suramin is a 100 year old drug, originally developed by Bayer. It has been explored for many medical conditions  however today it is used to treat African sleeping sickness, a parasitic condition.  It was chosen by Dr. Naviaux  for this study due to its anti-purinergic properties. Dr. Naviaux theorizes that in autism, a biochemical process he calls the Cell Danger Response (CDR)  persists or gets "stuck" due to purinergic signaling within the body' cells.


He hypothesized that if he could somehow interfere with purinergic signaling with a type of drug called an antipurinergic like suramin, he could turn off the persistent cell danger response and consequently the symptoms of the whole body disorder we know as autism. 


For a more scientifically detailed explanation please refer to our summary of Dr. Naviaux's previous work published in 2015 or our recent conversation with Dr. Naviaux here.


What was the suramin trial?


The suramin trial was the first human testing of research that Dr. Naviaux has been conducting for years.  The trial was primarily about establishing the safety of suramin in children with autism.   The trial was a double-blind, placebo controlled, clinical trial of a single dose of suramin given to 10 boys ages 5-14.  Here is the link to the actual publication. 

Despite it exciting potential for a treatment for autism it was really an effort at continuing to validate Dr. Naviaux's theories about what is the underlying biology of autism.

After decades of genetics focused research providing very few answers for affected individuals and families, the suramin trial was a fundamental shift in the underlying philosophy that autism is a fixed condition and targeted effort to get to the heart of autism in order to advance meaningful treatments. 

In what areas did researchers see the biggest improvements?


Improvements were seen in all three of the core symptom domains of autism spectrum disorders:  communication and language, social interaction, and repetitive behaviors.  Overall Autism Diagnostic Observation Schedule or ADOS scores, which are the gold standard for autism diagnosis improved by 1.6 points which was statistically significant.  In addition to formal documented clinical improvements.  Parents of children in the treatment group reported significant positive improvements in a variety of behavioral areas.  Improvements were not observed in the control group.


Why are the findings so promising?


The findings of this trial are promising for a few reasons:


1.  The research may have identified the core biology of autism, sometimes called the "mechanism."  History has shown us in other conditions that understanding the mechanism of a disease can result in rapid advancements toward effective treatments.

2.  The research not only suggest a mechanism, but is testing a novel treatment.  Typically this leap from mechanism to experimental treatment can take a decade or more.  While suramin will still have to undergo extensive testing to determine if it is safe and efficacious, there are many other antipurinergics that also may be good candidates.

3.  For years N of One: Autism Research Foundation and other researchers have been saying that the conventional view of autism as fixed brain condition primarily caused by genetics does not fit the emerging data.  We have calling for a significant broadening of research funding beyond genetics into other promising areas that hold great promise including cell metabolism, immune function and the microbiome.  This study may finally mark an inflection point where the major autism funders begin to significantly diversify their research funding beyond genetics.

4.  It offers hope to a community of individuals and families affected by autism who have been given few answers and optimism from medicine.

Could this be a fluke?

As with all research, this study will need to be replicated, expanded to test for safety and effectiveness in larger trials. There are no guarantees and there are many examples in science of results not being replicable but consider that Dr. Naviaux spent years developing his theory, then based on his theories, predicted that a certain type of compound would work (anti-purinergics).  He then successfully demonstrated that suramin worked multiple times in multiple mouse models all before this most recent test in humans, which was also successful.  Anything could happen, but the implications of this trial simply cannot be understated for the autism world.


To read more about Dr. Naviaux's previous mouse research, his theories and what he found click here


Can my child take suramin?

No. Suramin is currently not available in the United States and still needs to be further tested for safety and proper use in autism. 


Further the study authors note and N of One: Autism Research Foundation emphatically agrees that:


"Like many intravenous drugs, when administered improperly by untrained personnel, at the wrong dose and schedule, without careful measurement of drug levels and monitoring for toxicity, suramin can cause harm. Careful clinical trials will be needed over several years at several sites to learn how to use low-dose suramin safely in autism, and to identify drug–drug interactions and rare side effects that cannot currently be predicted. We strongly caution against the unauthorized use of suramin."


We understand that many parents and families are desperate but we emphatically beg do not experiment with suramin. Your child's health could be irreparably harmed.


How far off are we from suramin being available to those affected?


The rate at which future trials proceed will directly correlate with the amount of funding that is received.  The next trial will need to be larger and more expensive.  At the moment, funding for this next trial has not been secured. That is why it is imperative to support and advocate for this research in order to advance treatments for those affected.   How can you support that?  In the coming weeks, N of One will be suggesting means by which affected families can advocate for accelerated funding for suramin but in the mean time please consider supporting research like this by making an annual donation to N of One.  Until this this kind of research is well funded by the government, it's up small non-profits, affected families and caring individuals.  You can learn how to help here

Is it likely to be equally effective in older children, teenagers and adults with autism?


Only time and further research will answer this.  However, in one of Dr. Naviaux's mouse studies he was able to demonstrate that suramin also produced improvements in adult mice.  The mice in that particular experiment were 6 months old which according to Dr. Naviaux in the paper would be the biological equivalent to 30 years old in humans.


What is the most important "take away" for affected families?


In our view the most important "take away" for affected families is that this research and others like it provide a promising avenue for meaningful answers and treatments for autism in our lifetime.  However we, parents, families, and the public must be vocal and advocate for more research like this. 


What can I do as a person who is affected by autism to help advance and advocate for this important research?


Funding and awareness are the two most important things in our quest for research and treatments. Please check out our web page and sign up for our mailing list to stay updated on ways you can help by raising funds or helping spread awareness to others who can. We need families to step up and do fundraisers in order for this type of work to continue.  Please follow us on social media N of One: Autism Research Foundation and Rally for Research - an N of One: Autism Research Foundation Initiative) and help spread the word to your friends and family. We need parents to organize fundraisers - walks, bake sales, any type of event to help raise funding for promising research like this because historically this type of research, including Dr. Naviaux's has not been supported by the  handful of large organization that control virtually all the funding.  Together we can make a huge difference in the lives of all who are affected.

bottom of page