Naviaux and his team first tried suramin in mice models with autism-like features in 2013, and found that normal mouse social behaviors were restored. The UC San Diego trial was initially meant to be a phase one trial that only tested the safety of suramin in children, noted John Rodakis, the founder and president of N of One: Autism Research Foundation, which supported the trial.
But, Rodakis told Seeker, the results were “mind-blowing.”
The study had a very small sample size; only ten children (all of whom were boys) were involved in the double-blind, placebo-controlled trial. They were matched together based on factors including severity of symptoms, IQ, and age. Then, one child in each pair was given an intravenous infusion of suramin, for a total of five children that took the medication.
“After the single dose, it was almost like a roadblock had been released” in the treated children, Naviaux remarked. All five of the boys who received the drug began to show improvements in symptoms, including displaying increased communication and social activity.
The parents of these five children were also thrilled, according to a batch of anonymous statements from them that the lab released to accompany a press release.
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“Suramin produced the most dramatic improvement in autism symptoms that we have ever seen with anything we have tried,” one parent wrote.
“Nothing has come close to all the changes in language and social interaction and new interests that we saw after suramin,” said another.
Yet even proponents of the study are quick to note that there are limitations to the trial, with the most obvious being the small sample size of the children studied.
“We would have loved to have done a much larger trial,” said Rodakis. “We just didn’t have the funding for it.”
Both Rodakis and Naviaux noted that they did not receive any federal funding for the trial, and had to rely on donations and grassroots support from autism parents and advocates.
All of the children who received the medicine exhibited a rash, the authors noted, which may have compromised the double-blind nature of the research. They also pointed out that the children who showed benefits from suramin only improved temporarily. Naviaux said that the most improvement was reported three weeks after the infusion — children began to regress to previous levels of functionality after that point.
Finally, while the study designers tried to control the placebo effect as much as possible, autism research has a notoriously difficult time parsing genuine benefits from false positives. It’s not uncommon for small studies to claim that they have identified a potential cure for autism, but then have these hopes dashed after more rigorous study. One such case was the hormone secretin, which in the late 1990s was briefly thought to produce an improvement in ASD symptoms. Upon more testing, the science didn’t hold up.
"It's a really interesting study — very small, but well done," said Dr. Sanford Newmark, a pediatrician who specializes in treating autism. He noted that the trial's findings are "premature," however. Newmark, who is the medical director of the Osher Center for Integrative Medicine at the University of California, expects that his patients are going to start asking about suramin, but he doesn't think that they should experiment with it yet until more is learned about its possible effects.
Regardless of the ultimate validity of these new preliminary results, Naviaux and Rodakis believe that this research has shown enough promise to warrant follow-up trials that will test suramin on more children at various doses.
“If the future studies show that there’s continued health benefits,” Naviaux said, “this could be a game-changer for families with autism.”
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