Clinical Trial Launched to Assess Safety and Efficacy of Autism Drug Treatment

UC San Diego researchers open first study of suramin in children with autism —

Researchers at the University of California, San Diego School of Medicine have launched a clinical trial to investigate the safety and efficacy of an unprecedented drug therapy for autism.

The phase 1 clinical trial, which is recruiting 20 qualifying participants, will evaluate suramin – a century-old drug still used for African sleeping sickness – as a novel treatment for children with a diagnosis of Autism Spectrum Disorder (ASD). Previous published research by Robert K. Naviaux, MD, PhD, professor of medicine, pediatrics and pathology at UC San Diego School of Medicine, and colleagues reported that a single injection of suramin reversed symptoms of ASD in mouse models. …Read the full story from the UC San Diego Newsroom


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

He co-directs the Mitochondrial and Metabolic Disease Center at UC San Diego.

More about research in the Naviaux laboratory

Clinical Trial Launched to Assess Safety and Efficacy of Autism Drug Treatment

UC San Diego researchers open first study of suramin in children with autism —

Researchers at the University of California, San Diego School of Medicine have launched a clinical trial to investigate the safety and efficacy of an unprecedented drug therapy for autism.

The phase 1 clinical trial, which is recruiting 20 qualifying participants, will evaluate suramin – a century-old drug still used for African sleeping sickness – as a novel treatment for children with a diagnosis of Autism Spectrum Disorder (ASD). Previous published research by Robert K. Naviaux, MD, PhD, professor of medicine, pediatrics and pathology at UC San Diego School of Medicine, and colleagues reported that a single injection of suramin reversed symptoms of ASD in mouse models. …Read the full story from the UC San Diego Newsroom


Senior author Robert K. Naviaux, MD, PhD, is professor of medicine in the Division of Medical Genetics. He co-directs the Mitochondrial and Metabolic Disease Center at UC San Diego.

Visit the Naviaux Laboratory website

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

Drug Treatment Corrects Autism Symptoms in Mouse Model

An old drug gives hope for new treatment in autism

Autism results from abnormal cell communication. Testing a new theory, researchers at the University of California, San Diego School of Medicine have used a newly discovered function of an old drug to restore cell communications in a mouse model of autism, reversing symptoms of the devastating disorder.

The findings are published in the March 13, 2013 issue of the journal PLOS ONE. … Read the full story from the UCSD Newsroom


First author of the study report is Robert K. Naviaux, MD, PhD, professor of medicine in the Division of Medical Genetics and professor in the departments of pediatrics and pathology.

Naviaux leads a medical genetics research laboratory whose two major areas of study are the mitochondrial mechanisms of disease and development and evolutionary systems biology and marine metagenomics. He is founder and co-director of the UC San Diego Mitochondrial and Metabolic Disease Center.

Naviaux earned his MD and PhD degrees at the Indiana University School of Medicine and received his advanced training in virology and molecular biology at the Salk Institute for Biological Studies. There, in the laboratory of Dr. Inder Verma, he conducted investigations in gene therapy and retrovirus biology.

Other Department of Medicine authors of the report are Laura L. Dugan, MD, who is chief of the Division of Geriatrics and Larry L. Hillblom Chair in Geriatric Medicine; research associates Lin Wang and Qingbo Tang; and Mihael Rogac.

Citation for the report:  Naviaux RK, Zolkipli Z, Wang L, Nakayama T, Naviaux JC, et al. (2013) Antipurinergic Therapy Corrects the Autism-Like Features in the Poly(IC) Mouse Model. PLoS ONE 8(3): e57380. doi:10.1371/journal.pone.0057380  |  Read the report