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

Enzyme Restores Function with Diabetic Kidney Disease

Mouse findings reverse prevailing theory; point to potential treatment options

Researchers at the University of California, San Diego School of Medicine say that, while a prevailing theory suggests elevated cellular levels of glucose ultimately result in diabetic kidney disease, the truth may, in fact, be quite the opposite. The findings could fundamentally change understanding of how diabetes-related diseases develop – and how they might be better treated. … Read the full story from the UC San Diego News Center


Kumar Sharma, MD, FAHA Kumar Sharma, MD, FAHA, senior author of the study report, is professor of medicine and director of the Center for Renal Translational Medicine in the Division of Nephrology-Hypertension.

Dr. Laura DuganFirst author Laura L. Dugan, MD, at right, is professor of neurosciences and professor and chief of the Division of Geriatrics in the Department of Medicine. She holds the Larry L. Hillblom Chair in Geriatric Medicine.

Second author Young-Hyun You is an associate project scientist in the Division of Nephrology-Hypertension,

Other DOM faculty coauthors are Sameh S. Ali, assistant professor of medicine in the Division of Geriatrics, and Robert K. Naviaux, MD, PhD, professor of medicine in the Division of Medical Genetics.

Citation for the study report:

Laura L. Dugan, Young-Hyun You, Sameh S. Ali, Maggie Diamond-Stanic, Satoshi Miyamoto, Anne-Emilie DeCleves, Aleksander Andreyev, Tammy Quach, San Ly, Grigory Shekhtman, William Nguyen, Andre Chepetan, Thuy P. Le, Lin Wang, Ming Xu, Kacie P. Paik, Agnes Fogo, Benoit Viollet, Anne Murphy, Frank Brosius, Robert K. Naviaux and Kumar Sharma. AMPK dysregulation promotes diabetes-related reduction of superoxide and mitochondrial function. J Clin Invest. 2013;123(11):4888–4899. doi:10.1172/JCI66218.  |  Full text (UCSD only)

Related UC San Diego news stories:

$5.9 Million Grant to UC San Diego for Paradigm-Shifting Diabetes Research

Kumar Sharma, MD, FAHA, professor of medicine at the University of California, San Diego School of Medicine and director of the Center for Renal Translational Medicine, has received a $5.9 million grant from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the National Institutes of Health, to study kidney complications related to type 1 and type 2 diabetes…. Read the full story from the UCSD Newsroom


Kumar Sharma, MD, FAHADr. Sharma’s project, “Novel Paradigms in Diabetic Complications,” is a five-year Type 1 Diabetes Targeted Research Award.

Read the project abstract

Other Department of Medicine investigators in the project: