Some of the nation’s leading experts in basic and clinical neuroscience gathered at USC’s Davison Conference Center June 11-13 to discuss the overlap between autism and other developmental disorders in hopes of pinpointing their genetic and neurobiological overlap, which ultimately may lead to important drug discoveries and therapeutics.
The three-day conference explored “the new frontier for neuroscience research,” according to organizer Steven Moldin, executive director of USC’s Washington, D.C. Office for Research Advancement.
“Understanding autism and its related disorders really provides a window to understand all of developmental neuroscience,” Moldin said.
With considerable funding being made available for autism research, Moldin said the field is undergoing a true renaissance, and the conference aimed to help the scientific community determine the direction of hundreds of millions of dollars in future research funding for autism and related disorders.
The conference boasted an impressive roster of presenters from across the country, including two of USC’s own researchers, Roberta Biaz Brinton and Michel Baudry.
Brinton, holder of the USC School of Pharmacy’s R. Pete Vanderveen Endowed Chair in Therapeutic Discovery and Development, presented evidence proposing a link to a subpopulation of autism to a deficit in vasopressin, a small protein that promotes social behavior, learning and memory within the brain.
Brinton showed that animals lacking vasopressin exhibit strikingly similar symptoms to a subgroup of autistic children. The symptoms include impaired memory and learning, decreased social recognition and emotional bonding, repetitive behavior and abnormal kidney function.
She suggested that such children might respond to vasopressin therapy. Of the approximately 300,000 autistic patients in the United States, Brinton estimated that 10 to 20 percent might be responsive to vasopressin therapy. A vasopressin nasal spray, already used for other conditions and shown to be safe in children, could be a “reasonable approach,” she said.
If the treatment proved effective in the subpopulation that is vasopressin deficient, then it might be reasonable to extend this therapy more broadly to the autistic population. But, she added, “I think there are multiple ways to develop autism and thus successfully treating the entire autistic population will require multiple treatment strategies.”
Baudry, professor of biological sciences in USC College, discussed the potential of computer simulations for testing new drugs. Baudry, one of USC’s most frequently cited reseachers, leads a group of neuroscientists working with a small start-up company to develop realistic simulations of the human cognitive process.
Using the simulations, researchers could block a specific receptor or pathway and track the consequences for the brain. The method could make it easier to identify the most promising drug targets before proceeding to expensive and time-consuming early-stage trials.
The conference was sponsored by four institutes at the National Institutes of Health, the Office of Rare Diseases in the NIH Office of the Director and six private foundations: Autism Speaks, Fragile X Research Foundation, Rett Syndrome Research Foundation, Southwest Autism Research and Resource Center, Simons Foundation and Tuberous Sclerosis Alliance.