Century-Old Drug Is Promising for Autism

Barbara Geller, MD

In animals, results with suramin are intriguing, but how will they translate to humans?

In a mouse model of autism, investigators studied symptom improvement with the drug suramin, a century-old drug that is primarily used for parasitic infections and which acts at purinergic receptors.

The mice were offspring of laboratory-mated animals and prenatally received either saline or poly IC, a compound that produces maternal immune activation and autism-like behaviors in offspring. At 6 months, an age equivalent to 30 years in humans, male mice were randomized to suramin or saline.

Poly IC was associated with loss of sociality, a need for sameness, and impaired motor skills. With suramin, the asociality and sameness behaviors ceased in poly IC mice; 5 weeks after suramin was discontinued, these behaviors returned. In a separate postmortem study, suramin was present in brainstem areas that can be reached without crossing the blood-brain barrier. In metabolic analyses, suramin partially normalized the pathways affected by poly IC, including those related to neurodevelopment.

Citation(s):

Naviaux JC et al. Reversal of autism-like behaviors and metabolism in adult mice with single-dose antipurinergic therapy. Transl Psychiatry 2014 Jun 17; 4:e400.