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The Jawaharlal Nehru Centre for Advanced Scientific Research (JNCASR) has found a potential therapy for patients suffering from autism/intellectual disability (ID) that will enable the patient to lead a life less dependent on others.
In India, the estimated prevalence of autism spectrum disorder (ASD) is around 1 in 68 children, with boys being more commonly affected than girls. Now a team of JNCASR researchers has developed a compound named 6BIO, showing promise as a therapeutic agent for ASD and ID. In pre-clinical studies using the Syngap1^+/- mouse model, administration of 6BIO resulted in restored neuronal function, improved learning and memory, enhanced sociability, and a reduction in epileptic seizures.
According to the Department of Science and Technology, current therapeutics prescribed to treat autism spectrum disorder (ASD)/ID are mostly related to alleviating the symptoms rather than correcting the phenotypes observed in neurodevelopmental disorders, especially after brain development.
A team, led by Tapas K. Kundu and James Clement, found that in mice with mutated syngap gene (Syngap1+/- mice) which resembles humans with mutated syngap gene (present in autistic patients) the acetylation of DNA-associated proteins, histones or proteins that provide structural support for chromosomes is repressed in the brain.
The epigenetic enzyme behind this acetylation seems to be KAT3B or p300. Kundu`s group had previously discovered an activator of this enzyme, TTK21. Upon conjugating this activator with glucose-derived nanosphere (CSP-TTK21) and feeding to the Syngap1 autistic mice, the researchers could induce acetylation in the brain,” according to DST.
The team has shown in research published recently in the journal Aging Cell that the CSP-TTK21 restores neuronal function, learning, and memory, and induces neuronal rearrangements in Syngap1+/- mice, mainly when administered after the brain is considered to be developed adolescents in human beings.
“This report not only directly connects histone acetylation with autism, for the first time, but also opens a very optimistic door for ASD therapy. The study provides a new potential therapeutic option by targeting epigenetic modifications in Syngap1-related ID/ASD that can restore the deficits to an extent that will enable the patient to lead a life less dependent on others,” said the department.
Notably, the efficacy of 6BIO was observed not only when administered during developmental stages is equivalent to infancy and childhood but also after mid-childhood in ages 7-11, suggesting its potential for therapeutic intervention at various developmental stages.
These findings offer hope for more effective treatments for ASD and ID, addressing core challenges beyond symptom management.
According to the Paediatrics department in Vani Vilas Hospital part of the Bangalore Medical College, research indicates that genetics play a significant role in ASD. In fact, studies show that if one identical twin has autism, the other twin has a higher chance of being affected compared to fraternal twins or siblings. Some cases are linked to specific gene mutations or chromosomal abnormalities.
Another is pre-natal exposure to environmental toxins like pesticides, air pollution, and certain drugs during pregnancy. These include anti-epileptics and antidepressants which are increasing associated with a high. In addition, certain maternal infections during pregnancy and alcohol consumption in pregnancy may be other reasons why we have greater incidences of babies born with autism, they added.
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