New Autism Therapy Shows Promise in Enhancing Cognitive Abilities: study finds

According to a recent study, cancer drugs could make thinking easier for individuals with Rett syndrome, a rare disorder linked to autism. This discovery could lead to therapies for patients with other neurological conditions.

The findings published in Stem Cell Reports highlighted the role of microglia, a type of white blood cell found in the central nervous system, in the formation of the human brain. While research on these cells has advanced significantly in neurodegenerative disorders such as Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), and multiple sclerosis, their role in early neural development has been less understood due to limited access to foetal tissue.

For the study, researchers used brain organoids — “mini brains,” essentially, that mimic the developing brain of an embryo — grown from skin-derived stem cells of consenting patients. Such organoids were created from individuals with Rett syndrome — a disorder primarily found in females that features loss of speech, purposeful use of hands, mobility and muscle tone, among other symptoms as well as from neurotypical individuals.

Results showed that the synapses of typical neurons experienced impaired functioning when Rett syndrome microglia were introduced, further confirming the role of the immune cell in brain function and development.

Faulty microglia make cognition even harder for Rett syndrome patients, who already contend with fewer and impaired synapses due to a loss of function in the MECP2 gene, implicated in other types of neurodevelopmental conditions as well.Other studies on Rett syndrome have highlighted potential therapeutic targets. But none so far have identified a potential treatment involving human microglial cells.

This study highlighted that a cancer drug may offer cognitive benefits for some individuals with autism. This finding opens up new possibilities for therapeutic interventions, potentially enhancing cognitive function and quality of life for those affected by autism. Preliminary results indicate that this drug could improve cognitive function and overall quality of life for those affected by autism. If confirmed, this approach could offer a new, effective option for addressing cognitive challenges associated with autism, representing a major step forward in therapeutic development.