Researchers at MIT and the University of California have discovered a possible treatment for autism spectrum disorder (ASD) in the drug R-Baclofen, known under the brand name Lioresal. Studies with mice show an improvement in ADS-related symptoms.
According to Mark Bear of MIT, who co-authored the study with Jacqueline Crawley of the University of California at Davis,
“Our collaborative teams found that treatment with the drug R-baclofen improved scores on several learning and memory tasks, and on a standard assay of social behaviour, in 16p11.2 mutant mice. This unique corroboration of findings by two independent labs, using two distinct lines of mice with the same mutation, increases confidence that R-baclofen may be an effective pharmacological treatment for some of the symptoms of human 16p11.2 deletion syndrome, including intellectual impairment and autism.”
The condition of human 16p11.2 deletion syndrome is the result of roughly 27 genes missing from chromosome 16.
This deletion is characterized by intellectual disability; impaired language, communication, and socialization skills; and autism spectrum disorder or ASD.
This study, which makes use of the growing understanding of ASD-related gene mutations, shows that the idea of pharmacological treatments to manage or reverse ASD symptoms holds merit.
“These findings are particularly exciting on two fronts. First, the results show that diverse genetic causes of intellectual disability and autism may converge on a limited number of pathophysiological processes that can be ameliorated pharmacologically. Thus, a treatment for one genetically defined disorder may be beneficial for another with phenotypic overlap. Second, R-Baclofen has a well-understood safety profile and is well-tolerated in children and adults, making clinical studies feasible in the near future.”