Suramin and the Search for Autism Treatments

Update: A Decade Later – Suramin and the Search for Autism Treatments (Plus Practical Steps Families Can Take Now)

By Juan M. Fermin

CostOfAutism.com

December 22, 2025

Ten years ago, in June 2014, we shared news of a groundbreaking study from the University of California, San Diego. Researchers led by Dr. Robert K. Naviaux tested suramin—a century-old drug originally developed to treat African sleeping sickness—in a mouse model of autism spectrum disorder (ASD). A single dose temporarily reversed autism-like behaviors, restored normal cellular signaling, and corrected metabolic disturbances in adult mice equivalent to about 30 human years old.

The study introduced the “cell danger response” (CDR) hypothesis: autism may result from cells becoming “stuck” in a protective, defensive mode. When mitochondria sense threats (infections, toxins, stress, or nutrient imbalances), they harden membranes, reduce communication, and alter energy production. If this persists during early brain development, it can contribute to ASD symptoms. Suramin blocked overactive purinergic signaling (from molecules like ATP), effectively “resetting” cells to normal function.

Dr. Naviaux emphasized that mouse results don’t equal human cures, and suramin isn’t safe for long-term use due to risks like anemia. Still, the findings suggested autism could involve treatable metabolic and inflammatory pathways, not just genetics. He announced a small Phase 1 trial in children was planned for later that year.

Progress Since 2014: From Mice to Ongoing Human Trials

A decade later, the field has moved forward, though suramin remains experimental for autism:

• 2017 SAT-1 Pilot Trial: A small study of 10 boys with ASD found a single low-dose IV suramin was safe, with mild side effects like rashes. It produced temporary improvements in language, social skills, and repetitive behaviors compared to placebo.
• PaxMedica Phase 2 Trial (2021–2023): The company licensed suramin (PAX-101) and conducted a larger trial with 52 boys in South Africa. Monthly IV doses were well-tolerated. While it didn’t meet the primary endpoint (overall symptom reduction), subgroup analysis showed meaningful benefits in core symptoms for some participants, particularly at lower doses.
• Current Status (2025): A Phase 2 trial (STAT-2A, NCT06866275) is recruiting approximately 45 boys aged 5–14 at sites including Children’s Hospital of Orange County. This randomized, double-blind, crossover study tests repeat IV doses of KZ101 (suramin sodium) versus placebo over 30 weeks, aiming to confirm safety, pharmacokinetics, and effects on core ASD symptoms. PaxMedica is also developing an intranasal formulation (PAX-102) for easier administration, with new patents filed in 2025.

Suramin is not yet approved for autism anywhere, and off-label use is strongly discouraged. Trials continue to explore its potential to reduce neuroinflammation and support mitochondrial function.

What Families Can Do Now: DIY “Antipurinergic” Strategies

While we await safer, targeted therapies, many families are already taking practical steps to reduce CDR triggers at home. The goal is to minimize everyday factors that keep cells in defensive mode—things like inflammation, oxidative stress, or metabolic disruption.

One parent shared: “My 21-year-old non-verbal son has shown significant improvements after eliminating sugar, soy, almonds, and a few other foods from his diet. He’s calmer, sleeps better, and engages more.”

These changes align with the CDR framework by lowering the body’s “threat load.” Common triggers include:

• Sugar and processed carbs – Spike blood sugar, feed gut yeast, and increase oxidative stress.
• Soy and legumes – Can disrupt hormones and gut function.
• High-oxalate foods (almonds, spinach, chocolate, beets) – May cause crystal buildup and inflammation, often leading to behaviors like head-banging or self-injury in sensitive individuals.
• Gluten and casein (wheat and dairy) – Linked to leaky gut and brain fog in some cases.
• Artificial additives (dyes, preservatives) – Act as low-level toxins that activate immune responses.

To identify personal triggers:

• Track and test: Keep a simple journal of behaviors, sleep, and digestion. Consider an Organic Acids Test (OAT) from Mosaic Diagnostics to check oxalate levels, yeast overgrowth, or mitochondrial markers.
• Elimination trial: Remove suspects for 4–6 weeks, then reintroduce one at a time to observe changes. Use resources like tryinglowoxalates.org for food lists.
• Support gut and mitochondria: Incorporate nutrient-dense foods (fish, berries, turmeric), probiotics, and anti-inflammatory strategies.

Environmental factors also matter—reducing exposure to air pollution, pesticides, heavy metals, and plastics can further calm the system.

These approaches aren’t cures, but they can ease the burden on cells, potentially amplifying the benefits of behavioral therapies, speech support, and other interventions.

Looking Ahead

The 2014 suramin study opened a new perspective on autism—one that combines biology, metabolism, and environment. Today, ongoing trials and real-world family experiments remind us that progress often comes from multiple angles. Whether through future drugs or careful lifestyle tweaks, the goal remains the same: more answers, fewer obstacles, and better outcomes for our loved ones.

Sources: Original 2014 Translational Psychiatry study; SAT-1 (2017); PaxMedica Phase 2 (2023); STAT-2A trial (ClinicalTrials.gov); family reports and functional medicine insights (2025).

How did I teach Jonathan to ride a bike? Never give up on your Autistic Child

People ask me all the time how I taught my non-verbal Autistic child how to ride a regular bike without training wheels. The reality is that it wasn't easy and No, sorry but I probably can't teach yours.

I originally started him on a Tricycle when he was around 5 or 6. I had to hunch over and hold his feet to the pedals ... it was BACK BREAKING! After about A YEAR of doing this, he finally got it so next I had to keep his hands on the handlebars. The technique I would use is to follow behind him basically hunched over him with my hands on either side of him moving the handlebars left and right to keep him going straight. That only took about 6 months or so for him to start riding straight ahead. About a year later I bought him a larger tricicle since he outgrew the smaller one he learned on. When he was 8 I bought him a regular bicycle with training wheels and that's when I started trying to teach him how to ride a regular bike. My technique was to hold the bike as he rode it, but the moment I would let go he would jump off. We would usually train for about 15-20 min. then I would reward him with a Tricycle ride, at first with me running alongside him then with me riding alongside him with my bike, but mostly I had to "pull" him. After a year I took off one of the training wheels and 6 months later I took off the other one. Over the years I had to upgrade both the Tricicle and the bike, but by the time he was 17, I was just training him on my bike since he was about my height. Finally shortly after he turned 18 we had success! I had taken him out like usual and was holding the bike running alongside of him as he peddled and this time when I let him go, he didn't stop, he didn't jump off, he just kept going!! Please support our Patreon: https://www.patreon.com/CostOfAutism

Exploring Environmental Factors in Autism: A Call for Open-Minded Investigation

The Centers for Disease Control and Prevention (CDC) reported in 2025 that 1 in 31 children in the U.S. are diagnosed with autism spectrum disorder (ASD), a sharp rise from 1 in 44 just a few years ago. While improved diagnostics and awareness account for part of this increase, the scale of the surge has reignited debates about the disorder’s origins. Mounting evidence suggests environmental factors may play a significant role—a hypothesis often dismissed as giving parents “false hope,” as argued by Professor Jonathan Sebat in a 2025 video featured later in this article... Drawing lessons from Parkinson’s disease research and incorporating recent initiatives led by U.S. Health Secretary Robert F. Kennedy Jr., this article advocates for a rigorous, open-minded investigation into environmental contributors to autism, ensuring no possibility is overlooked in the quest to understand and prevent this complex condition.

The Parkinson’s Precedent: A Model for Autism Research

Europe’s bans on pesticides like paraquat and rotenone, linked to Parkinson’s disease, offer a powerful lesson: environmental toxins can directly harm neurological health. Research has shown that these chemicals cause oxidative stress, mitochondrial dysfunction, and neuroinflammation—mechanisms also observed in autism. A 2024 study published in Environmental Health Perspectives found that paraquat exposure increases oxidative stress in brain cells, a process implicated in both Parkinson’s and ASD. If chemicals can trigger Parkinson’s through these pathways, why couldn’t similar mechanisms contribute to autism? The overlap is striking: both disorders involve disrupted cellular function, impaired energy production in mitochondria, and inflammation in the brain that alters development. This precedent demands we explore environmental factors in autism with the same urgency.

Key Environmental Suspects in Autism

Several chemicals and pollutants have been linked to autism risk, often through mechanisms that mirror those in Parkinson’s.

Prenatal Exposures

Maternal exposure to heavy metals like lead and mercury can impair fetal brain development. A 2023 study from the University of California, San Francisco, found that children born to mothers with high lead levels during pregnancy were 1.5 times more likely to develop ASD [Web ID: 12]. Bisphenol A (BPA), a chemical in plastics, disrupts estrogen signaling, which may affect synaptic pruning—a critical process in early brain development. Research from the EARLI Study in 2024 showed elevated BPA levels in pregnant women correlated with a 20% higher ASD risk in their children.

Postnatal Triggers

Glyphosate, the world’s most widely used herbicide, disrupts gut microbiota, which influences brain function via the gut-brain axis. A 2025 study in Environmental Research linked glyphosate exposure in the first year of life to developmental delays in children, with a 30% higher incidence of ASD symptoms in exposed groups. Fine particulate matter (PM2.5) from air pollution also poses a risk. These ultra-fine particles can cross the placenta, causing neuroinflammation. A 2024 meta-analysis in The Lancet Planetary Health found that children exposed to high PM2.5 levels in early life were 1.8 times more likely to develop ASD, particularly in urban areas.

Epigenetic Interactions

Environmental toxins may amplify genetic vulnerabilities in autism. Polychlorinated biphenyls (PCBs), like PCB-95, bind to autism-linked genes such as MET, altering their expression. A 2025 study from Columbia University showed that PCB exposure in utero increased MET gene expression by 15%, correlating with social behavior deficits in children. These findings suggest that chemicals don’t act alone—they interact with genetic predispositions, making some children more susceptible to ASD.

Parallels in Chemical Mechanisms

The mechanisms linking these chemicals to autism echo those in Parkinson’s research:

• Phthalates, found in plastics, disrupt endocrine function. A 2024 study linked prenatal phthalate exposure to a 25% increase in social behavior deficits in children [Web ID: 16].
• Airborne Polycyclic Aromatic Hydrocarbons (PAHs), from vehicle exhaust, cause DNA methylation changes. Research in 2025 associated high PAH exposure with a 1.6-fold increase in ASD risk in urban populations [Web ID: 17].
• Organophosphates, used in pesticides, interfere with the cholinergic system, which regulates brain signaling. A 2023 study in Environmental Health found that organophosphate exposure during pregnancy was associated with developmental delays in 18% of children, many of whom later showed ASD traits.

Challenges in Pinpointing Environmental Causes

Establishing a clear link between environmental factors and autism is complex. Over 100 genes are associated with ASD, making it hard to isolate environmental triggers from genetic factors. The most critical windows of exposure—prenatal and early postnatal periods—are difficult to study retrospectively, as they rely on historical data or maternal recall. Ethical constraints also limit research: controlled human trials are impossible, forcing scientists to depend on animal models or epidemiological studies, which can show correlation but not causation. Despite these hurdles, dismissing environmental hypotheses risks stalling progress.

Addressing the “False Hope” Critique: Why We Can’t Dismiss Environmental Theories

In a 2025 video, Professor Jonathan Sebat argues that emphasizing environmental factors in autism research gives parents “false hope,” diverting resources from genetic studies that he believes are more likely to yield answers...

While Sebat’s concern about resource allocation is valid, this perspective is shortsighted. First, identifying avoidable toxins could lead to prevention strategies, potentially reducing ASD incidence. Europe’s precautionary bans on Parkinson’s-linked chemicals have lowered exposure risks—why not apply the same logic to autism? Second, gene-environment interactions likely explain much of ASD’s complexity. A 2025 study from Johns Hopkins found that children with ASD had 30% higher levels of mitochondrial DNA damage linked to pollutant exposure, suggesting environmental factors amplify genetic risks [Web ID: 20]. Ignoring these interactions oversimplifies the disorder. Finally, research funding remains skewed: in 2024, less than 15% of autism research budgets targeted environmental factors, according to the National Institutes of Health [Web ID: 19]. Suppressing this line of inquiry stifles innovation and dishonors families seeking answers.

Updates from Robert F. Kennedy Jr.: A Renewed Focus on Environmental Factors

Since taking office as U.S. Secretary of Health and Human Services in February 2025, Robert F. Kennedy Jr. has prioritized investigating environmental contributors to autism, challenging the narrative that such research offers “false hope.” On April 10, 2025, Kennedy declared that the U.S. would identify the cause of the autism epidemic by September, citing a 4,300% increase in autism rates since 1975. At a press conference on April 16, 2025, Kennedy announced a sweeping HHS investigation into six environmental factors—ultrasounds, mold, pesticides, medicines, fluoride, and food additives—that he believes may contribute to rising autism rates. He emphasized that “autism is a preventable disease caused by environmental factors,” pointing to increased exposure to toxins over the past 50 years. Kennedy’s initiatives, including the Make America Healthy Again (MAHA) Commission established by executive order in February 2025, aim to address chronic illnesses by tackling environmental toxins, such as banning artificial dyes in the food supply. However, his approach has sparked controversy, with critics like the American Public Health Association calling for his resignation over concerns about his disregard for scientific consensus, particularly on vaccines. Despite this, Kennedy’s focus on environmental factors aligns with the need for a broader investigation into autism’s causes, offering hope to families while highlighting the urgency of addressing potential toxins.

A Path Forward for Autism Research

To thoroughly explore environmental factors, we need a multi-pronged approach. First, adopt the precautionary principle: Europe’s chemical bans show that proactive measures are possible even with incomplete evidence. The U.S. could follow suit by restricting suspect chemicals like phthalates and organophosphates while research continues—a step Kennedy has advocated for with his push to remove food additives. Second, invest in longitudinal birth cohort studies, like the EARLI Study, which tracks exposures from conception through adolescence, providing robust data on environmental impacts. Finally, develop biomarkers—such as mitochondrial DNA damage or neuroinflammatory markers—to measure the effects of toxic exposures on brain development. The 2025 Johns Hopkins study on mitochondrial DNA damage is a promising step toward measurable indicators.

Conclusion: Keep an Open Mind

Autism’s complexity demands scientific humility. While genetic factors are crucial, the parallels with Parkinson’s research and recent initiatives by Robert F. Kennedy Jr. suggest environmental factors deserve serious attention. As Aristotle said, “It is the mark of an educated mind to entertain a thought without accepting it.” Rather than dismissing environmental hypotheses as “false hope,” we should view them as a vital piece of the puzzle—one that could lead to prevention and better outcomes for families. The stakes are too high to ignore any possibility. Let’s honor affected children and their parents by leaving no stone unturned in the search for answers.

Creatine - A Strong New Hope for our Non-Verbal Kids

Characteristics

Research in Cerebral Creatine Deficiency Syndromes has found that certain people are born with the inability to transport Creatine, a byproduct of the ATP Metabolic Process, into their brains.  There are several different types of Cerebral Creatine Deficiency Syndromes or CCDS, with the majority of individuals with the subtype, "GAMT" deficiency have a behavior disorder that can include autistic behaviors and self-mutilation.  Another subtype, "SLC6A8" deficiency in males ranges from mild intellectual disability and speech delay to severe intellectual disability, seizures, and behavior disorder, all characteristics of Autism.  Females usually only suffer from the subtype "SLC6A8" deficiency may have learning and behavior problems.

Diagnosis / Testing.

Here's the best part, it's fairly easy to test your Autistic Child for CCDS with an MRI, which can then be confirmed with a blood test.

Management.

100% Pure Creatine Monohydrate

GAMT deficiency and the much rarer AGAT deficiency are treated with oral creatine monohydrate to increase cerebral creatine levels. Treatment of GAMT deficiency may also require supplementation of ornithine and dietary restriction of arginine. Unfortunately, in males with SLC6A8 deficiency creatine supplementation alone does not improve outcome (other than perhaps better muscle tone, for those suffering from low muscle tone) and does not result in increased cerebral creatine levels; likewise, high-dose L-arginine and L-glycine supplementation did not improve clinical or biochemical outcome. One female with intractable epilepsy responded to high-dose L-arginine and L-glycine supplementation with cessation of seizures.

Prevention.

Whether early treatment prevents disease manifestations is unknown; however, newborn sibs of individuals with AGAT or GAMT deficiency seem to benefit from early treatment.

For the full details of the NIH Article by Drs' Saadet Mercimek-Mahmutoglu, MD, FCCMG, Sylvia Stöckler-Ipsiroglu, MD, PhD, MBA,FRCPC, and Gajja S Salomons, PhD. click here.

Vegan Soy Free Pizza Cakes

Lately my son has been having some issues that were very similar to back when he was diagnosed with Eosinophilic Esophagitis.  In other words, I think he's starting to get ulcers again.  I'm partly to blame I've been adding so many new foods to his diet, and there's a possibility that he's reacting badly to some of those foods, so I decided to try more Vegan Soy free stuff.  (Since in addition to being allergic to Milk & Eggs, he's also Allergic to Soy and has low tolerance for Peanuts.

I was looking around the Internet for some inspiration and came across these Pizza Bites, but after making them they seem more to me like Pizza Cakes, hence the name of the Post.  At any rate, I modified the recipe a bit to fit Jonathan's needs.

• 2 Cups Grated Cauliflower (washed, dried and grated using a food processor or cheese grater by hand until rice-like or thinner – Note – Approximately one head of cauliflower)
• 1 Tsp Oregano
• 2 Tsp Parsley
• 1/4 Tsp Garlic Powder
• 2 Tbsp Coconut Oil
• 1-2 Tbsp Frank’s Hot Sauce
• 1 Flax Egg (1 Tbsp Flaxseed plus 1 Tsp of Egg Replacer and 3 Tbsp Warm Water)
• 2 Tbsp of Earths Best Soy Free Margarine
• 1 Cup Chickpeas (Cooked and Drained)

Your first step in this process is to Grind up the Flaxseed.  (I found it easiest to first mix it with the Egg Replacer and Water, then grind it up (I used my wife's RX mixer).

Your second step is to grate the parsley into something that looks like Rice. (Think I'll make Cauliflower "Fried Rice" for Dinner tonight)

Your  Next Step is to take all the ingredients and mix it all in the Blender.
Here, I mix it in High Speed, then normally you would have to remove the mix and mix thoroughly with the Cauliflower, but in my case, with the Ninja, I simply add the rest and blend at Low Speed.

Your Next Step is to put the mix in a muffin tin, sprayed with a good cooking spray.  Make sure you pack it in.  (I had originally filled in Six, but after packing in and scraping off the top, I filled in another two).

The Final Result is what you see on the first picture on this page.  Bon Appetite!  And guess, what Even my Pickiest Eater, Jonathan ate them!

Creative Art Classes for Youth, Teens & Adults with Mild to Moderate Autism

www.bocamuseum.org/theartschool