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

Anti-Leukemia Drug May Also Work Against Ovarian Cancer

An antibody therapy already in clinical trials to treat chronic lymphocytic leukemia (CLL) may also prove effective against ovarian cancer – and likely other cancers as well, report researchers at the University of California, San Diego School of Medicine in a study published in the Nov. 17 online early edition of the Proceedings of the National Academy of Sciences (PNAS).

The findings extend the anti-cancer potential of an experimental monoclonal antibody called cirmtuzumab, developed at UC San Diego Moores Cancer Center by Thomas Kipps, MD, PhD, and colleagues. Cirmtuzumab is currently in a first-in-human phase 1 clinical trial to assess its safety and efficacy in treating CLL. … Read the full story from the UC San Diego Newsroom


Dr. Thomas Kipps

Dr. Thomas Kipps

Thomas Kipps, MD, PhD, Evelyn and Edwin Tasch Chair in Cancer Research, is professor of medicine in the Division of Hematology-Oncology and deputy director of research at the UC San Diego Moores Cancer Center.

Dr. Kipps directs the multi-institutional, National Institutes of Health (NIH)-sponsored CLL Research Consortium (CRC) and UC San Diego Blood Cancer Research Fund.

Clinical Trial To Test Safety of Stem Cell-Derived Therapy for Type 1 Diabetes

UC San Diego is initial site for first-in-human testing of implanted cell therapy

Researchers at the University of California, San Diego School of Medicine, in partnership with ViaCyte, Inc., a San Diego-based biotechnology firm specializing in regenerative medicine, have launched the first-ever human Phase I/II clinical trial of a stem cell-derived therapy for patients with Type 1 diabetes. … Read the full story from the UCSD Newsroom


Principal investigator in the study is Robert R. Henry, MD, professor of medicine in the Division of Endocrinology and Metabolism at UC San Diego and chief of the Section of Endocrinology, Metabolism & Diabetes at the Veterans Affairs San Diego Healthcare System.

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

Blacks Have Less Access to Cancer Specialists, Treatment

UC San Diego Study Suggests Racial Inequality Leads to Higher Mortality

Researchers at the University of California, San Diego School of Medicine say metastatic colorectal cancer patients of African-American descent are less likely to be seen by cancer specialists or receive cancer treatments. This difference in treatment explains a large part of the 15 percent higher mortality experienced by African-American patients than non-Hispanic white patients. … Read the full story from the UC San Diego News Center


Department of Medicine co-investigators on the project are Samir Gupta, MD, MSCS, associate professor of clinical medicine in the Division of Gastroenterology; Gregory Heestand, MD, Health Sciences assistant clinical professor in the Division of Hematology-Oncology and Paul Fanta, MD, MS, Health Sciences associate clinical professor in the Division of Hematology-Oncology.

Samir Gupta, MD, MSCS  Gregory Heestand, MD  Paul Fanta, MD, MS
Above, from left: Drs. Samir Gupta, Gregory Heestand, and Paul Fanta

Citation for the study report in the Journal of the National Cancer Institute:

Daniel R. Simpson, María Elena Martínez, Samir Gupta, Jona Hattangadi-Gluth, Loren K. Mell, Gregory Heestand, Paul Fanta, Sonia Ramamoorthy, Quynh-Thu Le, and James D. Murphy. Racial Disparity in Consultation, Treatment, and the Impact on Survival in Metastatic Colorectal Cancer JNCI J Natl Cancer Inst first published online November 14, 2013 doi:10.1093/jnci/djt318  |  Full text (UCSD only)

Clinical Trial Evaluates Engineered Smallpox Vaccine as Potential Liver Cancer Killer

As part of a multicenter clinical trial, researchers at University of California, San Diego School of Medicine are evaluating Pexa-Vec (JX-594) to slow the progression of hepatocellular carcinoma (HCC) or liver cancer. Pexa-Vec is a genetically engineered virus that is used in the smallpox vaccine.. … Read the full story from the UCSD Newsroom


Dr. Tony ReidPhysician-scientist Tony Reid, MD, PhD, is principal investigator of this clinical trial of Pexa-Vec (JX-594) at UC San Diego, which is one of 42 sites participating in the study.

Reid is professor of medicine and medical oncologist in the Gastrointestinal Cancer Unit of the UCSD Moores Cancer Center. He conducts research in the center’s Solid Tumor Therapeutics Program.

For more information about the Pexa-Vec (known as TRAVERSE) clinical trial, call 858-822-5354 or visit http://traversetrial.com.

Study Identifies Potential New Class of Drug for Treating Ulcerative Colitis

Oral Drug Shows Clinical Response and Remission in Some Patients

An investigational drug currently under FDA review for the treatment of rheumatoid arthritis has now shown positive results in patients with moderate-to-severe ulcerative colitis, according to researchers at the University of California San Diego, School of Medicine. The study will appear in the August 16, 2012 issue of the New England Journal of Medicine (NEJM). … Read the full story from the UCSD Newsroom


Dr. William J. SandbornThe principal investigator of the study is Dr. William J. Sandborn, professor of medicine and chief of the Division of Gastroenterology at UCSD.  |  Read his academic profile  |  Read his clinical profile

Read the abstract of the study report in the New England Journal of Medicine

Citation of study report:  Sandborn WJ, Ghosh S, Panes J, Vranic I, Su C, Rousell S, Niezychowski W; Study A3921063 Investigators. Tofacitinib, an oral Janus kinase inhibitor, in active ulcerative colitis. N Engl J Med. 2012 Aug 16;367(7):616-24.

Scott M. Lippman, MD, Named New Director of UC San Diego Moores Cancer Center

Scott M. Lippman, MD, chair of Thoracic/Head and Neck Medical Oncology at The University of Texas (UT) MD Anderson Cancer Center, has accepted the position of director of Moores Cancer Center at the University of California, San Diego, beginning May 1, 2012…. Read the full story from the UCSD Newsroom

Non-Invasive Technology Evaluated to Treat Cardiac Chest Pain

A clinical trial headed by Dr. Anthony DeMaria is the topic of the UCSD Newsroom story, “Non-Invasive Technology Evaluated to Treat Cardiac Chest Pain.”

The story has appeared in PhysOrg and EmaxHealth.

Anthony N. DeMaria, M.D., M.A.C.P., is the Judith and Jack White Professor of Medicine in Cardiology. He recently accepted the position of Associate Dean for Outreach and Development for Health Sciences. He served as founding director of the Sulpizio Family Cardiovascular Center and is on its governing board.