By Michelle Diament
June 17, 2014
New research suggests that a decades-old drug may be able to reverse symptoms of autism and now the medication is set to be tested in children with the developmental disorder.
The findings come from a study looking at a mouse model of autism published Tuesday in the journal Translational Psychiatry.
Researchers report that the drug called suramin effectively restored typical communication between cells in mice who had a human equivalent age of 30 and led the once-reclusive animals to seek out unfamiliar mice and explore new parts of their surroundings.
The results were temporary — with a single dose of the drug remaining effective in the mice for about five weeks — and researchers said suramin cannot be used long-term since it can lead to serious side effects like anemia and adrenal gland dysfunction.
Nonetheless, those behind the study said the findings are promising enough to warrant a small clinical trial of children with autism starting later this year.
Suramin has been used for nearly a century to treat a parasitic disease called African sleeping sickness. In the autism mouse model, the drug worked by addressing metabolic disturbances common in those on the spectrum that can impact language and social skills.
“Obviously correcting abnormalities in a mouse is a long way from a cure in humans, but we think this approach — antipurinergic therapy — is a new and fresh way to think about and address the challenge of autism,” said Robert Naviaux of the University of California, San Diego, a senior author of the study.
Naviaux indicated that further understanding of suramin’s effects on autism could lead to better treatments that may be used in conjunction with behavioral therapy to improve outcomes for individuals on the spectrum.