Could This 100-Year-Old Medication Be the Cure for Autism?

Vials containing suramin. NIMR London/Flickr

Suramin is a medicine that was first developed back in 1916, and it has proven so reliable as medication that it's on the World Health Organization's List of Essential Medicines. Today it's primarily used as an anti-parasitic drug for treating African sleeping sickness and river blindness, but now scientists think it might also offer treatment for one of the fastest growing developmental disorders in the United States: autism.

Research led by Dr. Robert Naviaux of the University of California, San Diego School of Medicine has found that when suramin was administered to children showing symptoms of autism, those symptoms were significantly alleviated after just a single dose, reports Seeker.

"After the single dose, it was almost like a roadblock had been released," remarked Naviaux.

One of the parents of a child helped by the medication was elated: "Suramin produced the most dramatic improvement in autism symptoms that we have ever seen with anything we have tried."

Naviaux and colleagues became curious about how suramin might influence autism symptoms after developing an unconventional theory about the cause of the condition, which can be debilitating in some cases. While genetic and environmental factors certainly play a part in the autism equation, Naviaux believes the root cause of the disorder comes down to a metabolic problem, which he has labeled the “cell danger response” (CDR).

Theoretically, CDR happens when a cell responds to external stressors or toxins by hardening its membranes and ceasing interaction with neighbors. Essentially, it's a defense mechanism where a cell withdraws into itself and waits for the perceived danger to pass. This is a healthy cell response most of the time, but occasionally it gets "stuck" in this mode. According to Naviaux, when this process sticks during crucial stages of early child development, autism can result.

The reason Naviaux believed suramin might be able to un-stick cells from unhealthy CDR is because it's also the only drug used in humans that can "inhibit extracellular ATP signaling." That's important because it has the power to normalize the CDR response.

The drug was first tested on mice showing autism-like symptoms to great success back in 2013. This is the first time the drug's effects have been studied on autistic human children, however. While the results are encouraging, there's also plenty of reason for caution. Due to financial restraints, Naviaux's team was only able to perform the study on 10 subjects. Only five of those actually received the suramin so that the rest could be used as a control. So the sample size of the experiment is extremely small.

Furthermore, all of the children who were administered suramin experienced a rash, an alarming side effect. And although the children showed remarkable positive cognitive and emotional improvements, the effects were only temporary. After three weeks, the children all regressed back to the norm. Even so, the study warrants further research.

“If the future studies show that there’s continued health benefits,” said Naviaux, “this could be a game-changer for families with autism.”

We'll just have to optimistically wait and see.

The research was published in the Annals of Clinical and Translational Neurology.