The latest advances in autism medication

People compare children with autism to stars in the sky, shining brightly in their own world and not caring about how the outside world changes. The cause of autism is still unclear, and no drugs have been developed to treat autism, but studies have shown that autism is hereditary. Many scientists are studying the factors that cause autism and the drugs that can treat it. In 2016, there was a major breakthrough in the latest drug development for autism, with the discovery of drugs that are effective for the restrictive and repetitive behaviors of children with autism.

One in every 160 children in the world suffers from autism. It was once believed that its cause may be related to social and psychological factors, but current research shows that autism is caused by genetic genes, brain disease or trauma and other physiological reasons. In recent years, the medical community has tried to use traditional or new antipsychotic drugs to intervene in autism.

In 2006, the US FDA approved risperidone for the treatment of autistic children aged 5 to 16 years old. In addition, there are also methylphenidate, carbamazepine, etc., but the effectiveness still needs further investigation, especially the side effects are very large.

A 2016 study found that low-dose buspirone was effective for restrictive and repetitive behaviors in children with autism spectrum disorder. But so far, there is no specific drug to treat the core symptoms of autism.

In 2007, scientists found that 83% of children with autism showed temporary improvement when they had a high fever. But the timing of a fever is crucial: A fever in the mother can increase the risk of autism in her unborn baby. As an expert in the metabolic processes that cells use to maintain life, Robert Naviaux of the University of California San Diego School of Medicine was intrigued by the idea that autism symptoms might be caused by metabolic dysfunction, leading to a breakdown in communication between the brain, the gut, and the immune system at the cellular level.

This abnormal cellular danger response can be maintained by purinergic signaling, a metabolic process that requires the use of ATP and adenosine molecules. To test this hypothesis, Naviaux and his team turned to a known "anti-purinergic drug" - suramin, a drug molecule that can suppress purinergic signaling. Suramin is a century-old "old drug" that has been used since 1916 to treat protozoan infectious diseases caused by trypanosome infection - African trypanosomiasis, also known as sleeping disease.

Through preclinical mouse studies, Naviaux et al. found that a single dose of suramin could significantly improve the symptoms of autism-like mice. This study, published in Translational Psychiatry in 2014, was the first to suggest that this was a promising drug for the treatment of autism.

This also paves the way for human trials, and this study is also the first human study of suramin in the treatment of autism. The researchers used a double-blind and placebo-controlled trial to divide 10 boys diagnosed with ASD (5-14 years old) into a suramin group (n=5, 20mg/kg suramin) and a control group (n=5, saline) according to age, IQ and autism severity.

People with autism spectrum disorders also have a core set of characteristics - difficulties with social interaction, communication, narrow interests and stereotyped repetitive behaviors. The researchers used standardized assessment tools to evaluate these core characteristics before, during, and after treatment. In addition, parents of boys with autism (who did not know whether their children were taking suramin or a placebo) reported improvements in their children's behavior, language, developmental goals, and social interactions.

By monitoring the blood drug concentration, it was found that the blood level of suramin was 12±1.5μmol/L on the second day and 1.5±0.5μmol/L after 6 weeks. The half-life is 14.7±0.7 days. A self-limited asymptomatic rash was seen, but no serious adverse events.

The five boys who received suramin showed stable improvements in autism symptoms in just seven days, while the placebo group showed no changes. Two of the boys showed surprising changes, Naviaux said: "After receiving suramin infusion, two children, aged 6 and 14, spoke their first words in their lives. The children who received the placebo showed no changes." But unfortunately, these improvements were only temporary. After six weeks, as the drug was gradually metabolized and excreted in the body, autistic symptoms reappeared.

In addition to the rapid improvement in symptoms, most importantly for the researchers, this positive result further strengthens the hypothesis that metabolic dysfunction is related to autism and that this dysfunction is treatable.

But it’s important to note that the study included very small numbers of people, and suramin is not currently a commercially available drug, and in the United States it’s not even approved for any therapeutic use. Therefore, be sure not to attempt to use it on your own. Like many IV drugs, suramin can cause harm if administered improperly by untrained personnel, if the wrong dose or time of administration is used, and if drug levels and toxicity are not monitored.

It will take several more years of careful clinical trials of suramin to understand how to safely use low doses of suramin in autism and to identify drug interactions and rare side effects that are currently unpredictable. The sample size of this current study was small, and larger studies are needed to confirm these preliminary results and investigate the potential risks and side effects of suramin, as well as whether suramin is suitable for long-term use.

“We plan to do five more studies over the next five years to gather all the data needed for FDA approval of suramin for autism,” Naviaux said. He also noted that even if suramin itself is not the right treatment for autism, these preliminary results may spark interest in developing anti-purinergic drugs.

We also eagerly await results from larger-scale trials and specific drugs for treating autism. Currently, in addition to comprehensive special education training, there are several other possible treatment methods for autism: behavioral correction, drug therapy, sensory integration therapy, auditory integration therapy, and acupuncture therapy.