Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial.
Level 2 - randomized trial
Individual randomized, placebo-controlled trial.
PubMed 28695149 · doi:10.1002/acn3.424
What was done
Ten male subjects with autism spectrum disorder (aged 5–14 years) were matched by age, IQ, and autism severity into five pairs. They were randomized in a double-blind design to receive a single intravenous infusion of low-dose suramin (20 mg/kg, n = 5) or saline placebo (n = 5). Primary outcomes were the ADOS-2 comparison score and the Expressive One-Word Picture Vocabulary Test (EOWPVT). Secondary outcomes evaluated aberrant behavior, repetitive behaviors, and clinical global impression.
What was found
Blood suramin concentrations were 12 ± 1.5 µmol/L at 2 days and 1.5 ± 0.5 µmol/L after 6 weeks, with a terminal half-life of 14.7 ± 0.7 days. A self-limited, asymptomatic rash occurred, with no serious adverse events. ADOS-2 scores improved by -1.6 ± 0.55 points (95% CI -2.3 to -0.9; Cohen's d = 2.9; P = 0.0028) in the suramin group, whereas the placebo group showed no change. EOWPVT scores did not change. Secondary outcomes showed improvements in language, social interaction, and repetitive behaviors, but exact numerical values were not reported in the abstract.
Why it matters
This study provides initial preliminary human pilot data suggesting that purinergic signaling modulation via low-dose suramin is safe and may improve core ASD symptoms.
Limits
The study is extremely small (n = 10 total; 5 per arm) and included only male pediatric subjects, limiting generalizability and statistical certainty. It evaluated only a single infusion over a short observation period, and longer-term efficacy, dosing regimens, and safety remain unmeasured.
Cited by
- partial Dr. Suzanne Goh's work identified autism spectrum disorder as involving a mitochondrial energy deficit and demonstrated clinical improvements using mitochondrial therapies.