Century-Old Drug Reverses Autism-like Symptoms in Fragile X Mouse Model

Autism spectrum disorders (ASD) affect 1 to 2 percent of children in the United States. Hundreds of genetic and environmental factors have been shown to increase the risk of ASD. Researchers at UC San Diego School of Medicine previously reported that a drug used for almost a century to treat trypanosomiasis, or sleeping sickness, reversed environmental autism-like symptoms in mice.

Now, a new study published in this week’s online issue of Molecular Autism, suggests that a genetic form of autism-like symptoms in mice are also corrected with the drug, even when treatment was started in young adult mice. … Read the full story from the UC San Diego Newsroom


Chief investigator of the study is Robert Naviaux, MD, PhD, professor of medicine in the Division of Medical Genetics and professor in the departments of pediatrics and pathology.

Dr. Naviaux is co-director of the Mitochondrial and Metabolic Disease Center at UC San Diego.

Read the abstract of the article in Molecular Autism

More about research in the Naviaux laboratory

Single Dose Reverses Autism-like Symptoms in Mice

Old drug used for sleeping sickness may point to new treatment in humans

In a further test of a novel theory that suggests autism is the consequence of abnormal cell communication, researchers at the University of California, San Diego School of Medicine report that an almost century-old drug approved for treating sleeping sickness also restores normal cellular signaling in a mouse model of autism, reversing symptoms of the neurological disorder in animals that were the human biological age equivalent of 30 years old. … Read the full story from the UC San Diego Newsroom