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

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

Using microRNA Fit to a T (cell)

Researchers show B cells can deliver potentially therapeutic bits of modified RNA

Researchers at the University of California, San Diego School of Medicine have successfully targeted T lymphocytes – which play a central role in the body’s immune response – with another type of white blood cell engineered to synthesize and deliver bits of non-coding RNA or microRNA (miRNA). … Read the full story from the UC San Diego News Center


Dr. Maurizio ZanettiPrincipal investigator Maurizio Zanetti, MD, is emeritus professor of medicine in the Division of Hematology-Oncology and director of the Laboratory of Immunology at the UCSD Moores Cancer Center.

Dr. Zanetti is the director of tumor immunology for the UCSD Center for Immunology, Infection and Inflammation. He directs the immunology course in the Biomedical Sciences graduate program.

Citation for the study report in Proceedings of the National Academy of Sciences:

Gonzalo Almanza, Veronika Anufreichik, Jeffrey J. Rodvold, Kevin T. Chiu, Alexandra DeLaney, Johnny C. Akers, Clark C. Chen, and Maurizio Zanetti. Synthesis and delivery of short, noncoding RNA by B lymphocytes. PNAS 2013 ; published ahead of print November 25, 2013, doi:10.1073/pnas.1311145110  |  Abstract (Open access)  |  Full text (UCSD only)

Other UCSD news stories about Dr. Zanetti’s work:

Herpes Virus Exploits Immune Response to Bolster Infection

Researchers at the University of California, San Diego School of Medicine and colleagues report that the herpes simplex virus type-1 (HSV-1), which affects an estimated 50 to 80 percent of all American adults, exploits an immune system receptor to boost its infectivity and ability to cause disease. … Read the full story from the UCSD Newsroom


Dr. Richard Gallo

Dr. Richard Gallo

Richard L. Gallo, MD, PhD, left, is principal investigator of the study, which is reported in the online-only journal Nature Communications.

Gallo is professor of medicine and chief of the Division of Dermatology.

Citation for the study report:  Daniel T. MacLeod, Teruaki Nakatsuji, Kenshi Yamasaki, Lester Kobzik & Richard L. Gallo. HSV-1 exploits the innate immune scavenger receptor MARCO to enhance epithelial adsorption and infection. Nature Communications 4, Article number: 1963. doi:10.1038/ncomms2963. Published 06 June 2013.  |  Abstract

More Information:

Cancer Cells Co-opt Immune Response to Escape Destruction

Researchers at the University of California, San Diego School of Medicine report that tumor cells use stress signals to subvert responding immune cells, exploiting them to actually boost conditions beneficial to cancer growth. … Read the full story from the UCSD Newsroom


Dr. Maurizio ZanettiThe report comes from the laboratory of Dr. Maurizio Zanetti (left), with graduate student Navin R. Mahadevan as lead author and Dr. Zanetti as senior author.

Maurizio Zanetti, MD, is emeritus professor of medicine in the Division of Hematology-Oncology and director of the Laboratory of Immunology at the UCSD Moores Cancer Center.

Dr. Zanetti is the director of tumor immunology for the UCSD Center for Immunology, Infection and Inflammation.  He directs the immunology course in the Biomedical Sciences graduate program.

Drs. Zanetti and Mahadevan published a Science Magazine Perspectives article on the connection between immune surveillance and chromosomal chaos in September.

On the PLOS ONE report, coauthors from Dr. Zanetti’s laboratory are undergraduate student Veronika Anufreichik, graduate student Jeffrey J. Rodvold and research associate Kevin T. Chiu.

Read the report in PLOS ONE

Citation for the report:  Mahadevan NR, Anufreichik V, Rodvold JJ, Chiu KT, Sepulveda H, et al. (2012) Cell-Extrinsic Effects of Tumor ER Stress Imprint Myeloid Dendritic Cells and Impair CD8+ T Cell Priming. PLoS ONE 7(12): e51845. doi:10.1371/journal.pone.0051845

More Information:

  • Science Magazine Perspectives article, September 2012:

Zanetti M, Mahadevan NR. Immune Surveillance from Chromosomal Chaos? Science 337 (6102): 1616-1617, 28 September 2012. DOI: 10.1126/science.1228464.

  • Other UCSD news stories about Dr. Zanetti’s work:
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Sex Hormone Precursor Inhibits Brain Inflammation

UCSD scientists say molecule moderates condition linked to neurodegenerative diseases

Researchers at the University of California, San Diego School of Medicine have discovered a steroid hormone that inhibits inflammation in the brain. The findings, to be published in the May 13 issue of the journal Cell, have implications for understanding the exaggerated inflammatory responses that are characteristic features of numerous neurodegenerative diseases. … Read the full story from the UCSD Newsroom

Christopher K. Glass, MD, PhD

Senior author of the study is Christopher K. Glass, MD, PhD (pictured above), Professor of Medicine in the Division of Endocrinology and Metabolism and Professor of Cellular and Molecular Medicine. Kaoru Saijo, MD, PhD, project scientist in the Glass laboratory, is the study’s first author.

Read the published study in Cell (free full text).