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

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:

Urine Biomarkers Reveal Mitochondrial Dysfunction in Diabetic Kidney Disease

Researchers at the University of California, San Diego School of Medicine have identified 13 metabolites – small molecules produced by cellular metabolism – that are significantly different in patients with diabetes and chronic kidney disease compared to healthy controls. … Read the full story from the UC San Diego News Center


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

Citation for the study report:

Metabolomics Reveals Signature of Mitochondrial Dysfunction in Diabetic Kidney Disease. Kumar Sharma, Bethany Karl, Anna V. Mathew, Jon A. Gangoiti, Christina L. Wassel, Rintaro Saito, Minya Pu, Shoba Sharma, Young-Hyun You, Lin Wang, Maggie Diamond-Stanic, Maja T. Lindenmeyer, Carol Forsblom, Wei Wu, Joachim H. Ix, Trey Ideker, Jeffrey B. Kopp, Sanjay K. Nigam, Clemens D. Cohen, Per-Henrik Groop, Bruce A. Barshop, Loki Natarajan, William L. Nyhan, and Robert K. Naviaux JASN ASN.2013020126; published ahead of print October 10, 2013, doi:10.1681/ASN.2013020126  |  Full text (UCSD only)

Related UC San Diego news stories:

Potential Nutritional Therapy for Childhood Neurodegenerative Disease

Researchers at the University of California, San Diego School of Medicine have identified the gene mutation responsible for a particularly severe form of pontocerebellar hypoplasia, a currently incurable neurodegenerative disease affecting children. Based on results in cultured cells, they are hopeful that a nutritional supplement may one day be able to prevent or reverse the condition…. Read the full story from the UCSD Newsroom


Robert K. Naviaux, MD, PhD, is a coauthor of the study. He is 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.

Citation for the report:  Naiara Akizu, Vincent Cantagrel, Jana Schroth, Na Cai, Keith Vaux, Douglas McCloskey, Robert K. Naviaux, Jeremy Van Vleet, Ali G. Fenstermaker, Jennifer L. Silhavy, Judith S. Scheliga, Keiko Toyama, Hiroko Morisaki, Fatma M. Sonmez, Figen Celep, Azza Oraby, Maha S. Zaki, Raidah Al-Baradie, Eissa A. Faqeih, Mohammed A.M. Saleh, Emily Spencer, Rasim Ozgur Rosti, Eric Scott, Elizabeth Nickerson, Stacey Gabriel, Takayuki Morisaki, Edward W. Holmes, Joseph G. Gleeson. AMPD2 Regulates GTP Synthesis and Is Mutated in a Potentially Treatable Neurodegenerative Brainstem Disorder. Cell, Volume 154, Issue 3, 1 August 2013, Pages 505–517 http://dx.doi.org/10.1016/j.cell.2013.07.005  Read the report

Related Stories:

Drug Treatment Corrects Autism Symptoms in Mouse Model – March 14, 2013

$5.9 Million Grant to UC San Diego for Paradigm-Shifting Diabetes Research – October 3, 2011

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

$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: