JNCASR Researchers Develop 6BIO for Autism Spectrum Disorder Treatment

A promising therapeutic option has been proposed at the Jawaharlal Nehru Centre for Advanced Scientific Research for treating Autism Spectrum Disorder (ASD) and intellectual disability(ID), thus contributing toward the possibility of independent functioning by the patient.

In India, ASD is estimated to affect 1 out of 68 children, with a higher incidence in boys. Preclinically, the compound 6BIO has shown excellent potential in Syngap1^+/- mice-an animal model representing the genetic mutations found in certain autistic patients. 6BIO treatment has also enhanced neuronal function, learning, memory, and social behavior while decreasing seizure activity.

Presently, therapies associated with ASD/ID are more symptom-oriented and less focused on treating the pathology itself, which essentially starts after brain development. Under the leadership of Tapas K Kundu and James Clement, the research team has elucidated how diminished acetylation of histones in Syngap1^+/- mice has led to impaired brain function under the control of the epigenetic enzyme KAT3B/p300.

The team had previously rediscovered TTK21, which activates KAT3B and had made a new formulation of it (CSP-TTK21) that exploits glucose-derived nanospheres as a suitable delivery vehicle.

The department stated, "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".

In Aging Cell, they showed that CSP-TTK21 was able to restore brain function even when provided post-development, which, in humans, corresponds roughly to the adolescent period.6BIO may, interestingly, not have been limited to treating those with early childhood onset but may be applicable even later in phases (ages 7-11) still inclusive of treatment).

According to professionals at Vani Vilas Hospital, genetic risk factors, prenatal exposure to toxins, infection, and some drugs may contribute to ASD. This research represents a ray of hope for treating the core implications of ASD and ID rather than merely symptomatic treatment.