Brain Organoids Uncover Autism Biology, Possible Treatments

Researchers at the Scripps Research Institute have uncovered new information about autism spectrum disorder (ASD). ASD is a developmental and neurological disorder that affects how an individual interacts with others, behaves, learns and communicates. The disorder’s causes are only partly known, with scientists linking a number of genetic variants to it.

For their study, the researchers used stem cells generated from patients with a severe and rare form of the developmental disorder. They focused on MEF2C haploinsufficiency syndrome (MHS), which develops following a genetic variation in the MEF2 gene.

They grew the stem cells into mini-brain organoids, which allowed the researchers to observe how the different brain cell types interacted with each other and learn more about how one mutation in the genes causes autism to develop.

They found that in brains with autism, there was an imbalance in excitatory neurons which promote electrical signaling and inhibitory neurons that blocked this signaling. This is in comparison to healthy brains, which contain a balance of inhibitory and excitatory neurons.

In particular, the researchers observed that the MHS organoids had less inhibitory neurons than usual, causing excessive electrical signaling in the organoids. When they looked into how mutations could cause this imbalance, they found almost 200 genes that were controlled directly by the MEF2 gene.

In particular, they found that 3 of them encoded microRNA molecule genes. This, in turn, prevented the developing brain from making proper connection between nerve cells.

In addition to this, the researchers tested a drug patented under EM-036 (NitroSynapsin) for its ability to restore connections in the brain. Their objective was to see if it could help treat the MHS autism form. They had tested the drug prior to this on its ability to restore brain connections in models of Alzheimer’s disease.

In their report, they explained that their experimental drug could help correct the imbalance and help restore balance to excitatory and inhibitory neurons while also protecting connection between nerve cells.

They noted that this meant that the drug could help reverse some of the brain dysfunction linked to autism in the models.

Dr. Stuart A. Lipton, the senior author of the study and clinical neurologist, revealed that their work demonstrated how this genetic mutation disrupted brain cell balance during development. He added that they also found that there could be ways to address the imbalance.

In their conclusion, the researchers noted that more studies were needed to demonstrate whether the drug could improve MHS symptoms. The study’s findings were reported online in Molecular Psychiatry.

Several drug makers, such as PaxMedica Inc. (OTC: PXMD), are currently focused on developing next-gen treatments against ASD. The coming years could therefore bring breakthroughs that change the paradigm of autism treatment and management.

NOTE TO INVESTORS: The latest news and updates relating to PaxMedica Inc. (OTC: PXMD) are available in the company’s newsroom at https://ibn.fm/PXMD

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