Social difficulties are seen to be the most devastating of all the challenges that occur with an autism diagnosis. However, it should be noted that autism condition is incurable. A recent study, involving researchers from the University of Buffalo, reveal it might be possible to use single compounds in an attempt to alleviate behavioral symptoms by meticulously targeting genes that may be involved in the autism condition.
The team demonstrated brief treatment with a very low dose of romidepsin, an FDA approved anti-cancer drug which successfully restored social deficits in autistic animal models in a sustained fashion. The treatment, administered over a time-span of three days, was seen to successfully reverse social deficits in clinical mice that were engineered to have a gene called Shank3, a defect that has a high-risk factor for developing autism condition.
The effect of the treatment lasted for three weeks, a time critical development stage for communication and social skills alike. Researchers claim these results could be equivalent to several years in humans whilst suggesting effects involving similar treatments could be more long-lasting than expected.
Prolonged, Profound effect
Zhen Yan, a Professor based out of Jacobs School of Medicine and Biomedical Sciences explains, “During the course of the study our team has successfully discovered a small molecular compound that shows prolonged and profound effects on social deficit conditions that hold similar ground to autism-like conditions.”
Prof. Yan further comments, “It should be noted that these compounds are already in use to treat a variety of psychiatric diseases, however, have failed to exhibit the right ingredients of therapeutic efficacies for the core symptom involving autism condition.”
The work involved uncovering how loss of the shank 3 gene disrupts neuronal communication ability of an individual by affecting the functions of NMDA receptor, an important player in regulating emotions and cognitive abilities while leading to impaired deficits in social preferences that are common in conditions like autism.
The researchers found they could possibly reverse these social deficits with very low doses of romidepsin, which assists one in restoring genetic expressions and functions using epigenetic mechanisms in place. Prof. Yan further noted that genetics involving humans who have epigenetic abnormalities play a far greater role in autism conditions.
Epigenetics in Autism
Countless mutational factors in autism condition results from chromatin remodeling factors which play a dynamic role in changing the underlying structure of chromatin.
Chromatin is a complex of genetic materials in the nuclei of the cell that condenses into chromosomes. Prof. Yan comments, “These extensive overlaps risk conditions such as cancer and autism.”
Prof. Yan explains, “Autism condition involves loss of many genes. To have the social deficits of an individual rescued, a compound has to affect a large number of genes that are involved in neuronal communication.”
The team concludes, “The underlying advantage of being able to have a set of genes that have autism risk identified explains strong and long-lasting efficacies of therapeutic agents that are meant to cure autism.”
Nevertheless, the team led by Prof. Yan continue their focus on developing and discovering better therapeutic agents for autism condition.