Researchers at the UC San Diego School of Medicine said Thursday that a century-old drug corrects genetic autism symptoms in laboratory mice, which might suggest a pathway for treatment in humans.
The drug is suramin, which has been used for decades to treat trypanosomiasis, a parasitic disease found in sub-Saharan Africa that’s also known as Sleeping Disease. The illness comes in two stages, the first of which doesn’t show symptons, but the second — which strikes years later — impacts the central nervous system and upsets the sleep cycle.
UCSD scientists previously found that suramin corrected environmentally caused autism symptoms in mice, but this is the first time the same result has been seen in genetic autism.
Autism spectrum disorders affect 1 to 2 percent of children in the United States, and hundreds of genetic and environmental factors have been shown to increase the risk, according to UCSD.
“Correcting abnormalities in a mouse is a long way from a cure in humans, but our study adds momentum to discoveries at the crossroads of genetics, metabolism, innate immunity, and the environment for several childhood chronic disorders,” said Dr. Robert Naviaux, a UCSD professor of medicine. “These crossroads represent new leads in our efforts to understand the origins of autism and to develop treatments for children and adults with ASD.”
Naviaux, also a co-director of the Mitochondrial and Metabolic Disease Center at UCSD, said suramin blocks a defensive response by cells that senses danger from viral or other infections, toxins or genetic mutations.
That response, which causes a reduction in communication between cells, is an underlying mechanism in autism spectrum disorder, he said. A consequence is interference with brain development and function.
Suramin can only be given to patients for a couple of months before levels of the drug become toxic. Naviaux said a new class of medication might be provided intermittently to unblock metabolism, restore more normal neural network function, improve resilience and permit improved development.
The study was supported, in part, by the Jane Botsford Johnson Foundation, the UC San Diego Christini Foundation, the UC San Diego Mitochondrial Research Fund, and the Wright Family Foundation.
— City News Service