This is the first evidence that shows that it is possible to use a single compound to alleviate these symptoms.
The inability to relate to others is one of the most dramatic consequences of patients with autism spectrum disorder. And, currently, there is no treatment for this symptom. Fortunately, new research conducted by scientists at the University of Buffalo (USA) reveals the first evidence that it is possible to use a single drug to alleviate behavioral symptoms, as it targets sets of genes involved in the illness.
The research, published in the journal Nature Neuroscience, showed that short treatment with a very low dose of romidepsin, an anti-cancer drug approved by the Food and Drug Administration (FDA), permanently restored social deficits in mice with autism.
The three-day treatment reversed problems in social skills in a gene called Shank 3, a major risk factor for autistic spectrum disorder. This effect lasted three weeks, ranging from the juvenile period to the end of adolescence of rodents, a critical stage of development for social skills and communication, the equivalent to several years in humans, suggesting that the effects of A similar treatment could be long-lasting, the researchers point out.
"We have discovered a compound that has a profound and prolonged effect on social deficits similar to autism with no apparent side effects," says Zhen Yan, leader of the work.
The effect of the drug against cancer in mice with autism was profound and prolonged
The study is based on a previous investigation of 2015 that revealed how the loss of Shank 3 interrupts neuronal communications by affecting the function of the NMDA receptor (of N-methyl-D-aspartate), a critical player in the regulation of cognition and the emotion.
To continue these promising findings, Yan founded an emerging company called ASDDR, which received a grant from the National Institutes of Health for more than $ 770,000.
In the new research, the scientists found that they could reverse those social deficits with a very low dose of romidepsin, by restoring gene expression and function using an epigenetic mechanism, where genetic changes are caused by influences other than DNA sequences.
"Autism implies the loss of many genes," explained Yan. "To rescue social deficits, a compound has to affect a series of genes that are involved in neuronal communication."
To do so, the team turned to a type of chromatin remodeler, the histone-modifying enzymes that help organize the genetic material in the nucleus (so that gene expression can be regulated). Since many genes are altered in autism, the scientists were convinced that a histone modifier could be effective.
The rescue effect on gene expression was generalized. When Yan and his team conducted a genome scan they discovered that romidepsin restored most of the more than 200 genes that were deleted in the model of autistic animals that they used in the experiment.
"The advantage of being able to adjust a set of genes identified as key risk factors for autism could explain the strong and long-lasting efficacy of this therapeutic agent for autism," concludes Yan.