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

Next Generation: Thomas Ohno-Machado Lobbies for Federal Research Funding

One in a Series of Occasional Stories About Faculty Members’ Children

Dr. Lucila Ohno-Machado and Thomas Ohno-Machado with Senator Barbara Boxer Lucila Ohno-Machado, MD, PhD; her son Thomas and California Senator Barbara Boxer.

Recently, to his mother’s wonderment, 17-year-old Thomas Ohno-Machado revealed that he is interested and quite active in politics.

It seemed to happen overnight, his mother said, and without any particular encouragement from his parents. “It doesn’t run in the family,” she mused.

Thomas, a senior at Torrey Pines High School, is the oldest of Dr. Lucila Ohno-Machado’s three sons. Lucila Ohno-Machado, MD, PhD, is associate dean for informatics for the UCSD School of Medicine and professor and chief of the Division of Biomedical Informatics in the Department of Medicine.

Thomas is now the founder and president of the Torrey Pines High School Autism Awareness Club and an advocate for children with disabilities. He and his friends have successfully organized a funding drive and raised money to benefit a local school for children with special needs.

One day out of the blue Thomas asked Dr. Ohno-Machado, “Mom, can we go to Washington, DC?”

It turned out he had written to Senator Barbara Boxer’s office and arranged to visit and express his concerns about federal support for biomedical and behavioral research, particularly for the National Institutes of Health, and for the increasing numbers of children who have special needs.

Dr. Ohno-Machado agreed to go. She arranged for him to add a one-day visit to Washington DC to a trip she had already scheduled. He purchased his first suit, from Macy’s, and carried it on the plane so that it would not wrinkle in flight.

She suggested he prepare for his audience with the senator, and he assured her, “Don’t worry.”

They arrived at Senator Boxer’s office and, with the Archbishop of California and a California judge who had come in with them, waited their turn to speak with one of the senator’s staff members.

The time came. Prepared with statistics to back him up, Thomas Ohno-Machado expressed his concern and asked to know what the senator was going to do about federal medical research funding and the rising numbers of special needs children in the United States.

The senator’s staffer responded by showing Thomas documents detailing how Senator Boxer and another senator are addressing these issues.

Said Dr. Ohno-Machado of the meeting, “I didn’t speak a single word.”

She and Thomas had tried but weren’t able to set up a visit to California Senator Diane Feinstein’s office on the same trip. In a separate solo trip, Thomas visited the governor of Massachusetts and a school for special needs children in the Boston area. Recently, he pressed his causes at the San Diego mayor’s office.

He is headed to UC Santa Cruz this fall. He plans on a political career.

Dr. Ohno-Machado is still marveling at how a strong interest can reveal itself so suddenly in one’s offspring.

“He’s very excited about it,” she said. “He has a bright future.”

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