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Cancer drug may reverse anti-social symptoms in autism patients 

An anti-cancer drug could help people with autism establish and maintain friendships and relationships, new research claims.

A mice study revealed low doses of romidepsin, an FDA-approved drug used to treat lymphoma, restores the genes associated with social and communication skills.

People on the autism spectrum usually have difficulty establishing and maintaining relationships, however there is currently no treatment for this primary symptoms.

The study, conducted by researchers at the University of Buffalo, suggests people with autism could be prescribed the cancer drug to treat anti-social symptoms.

An anti-cancer drug can reverse the anti-social symptoms associated with autism, according to a new study

‘We have discovered a small molecule compound that shows a profound and prolonged effect on autism-like social deficits without obvious side effects,’ said Dr Zhen Yan, professor in the department of physiology and biophysics at the University of Buffalo.  

For the study, Dr Yan and her colleagues randomly assigned mice to drug and placebo groups. They then conducted experiments on mice’s social preference and  self-grooming behaviors.

They found romidepsin made mice that were deficient in a gene called Shank 3, a hallmark of autism, more sociable. 

Researchers also found that the effect of the three-day treatment lasted for three weeks in mice – from the juvenile to late adolescent period. In humans, this is equivalent to several years, researchers said.

These findings build on Dr Yan’s previous research in 2015 which found Shank 3 disrupts how neurons communicate by reducing the function of the NMDA (n-methyl-D-aspartate) receptor, a protein that regulates cognition and emotion.

This causes people to be anti-social, a symptom that is common in people on the autism spectrum.

The new study shows that romidepsin reverse those social deficits by restoring gene expression and function.

Dr Yan said many of the cell mutations that occur in people with autism are a result of chromatin remodeling factors, which are involved in changing the structure of genetic material.

‘The extensive overlap in risk genes for autism and cancer, many of which are chromatin remodeling factors, supports the idea of repurposing epigenetic drugs used in cancer treatment as targeted treatments for autism,’ said Dr Yan.

She and her colleagues knew that chromatin regulators – which regulate gene expression – were key to treating the social deficits in people with autism.

‘Autism involves the loss of so many genes,’ Dr Yan explained. ‘To rescue the social deficits, a compound has to affect a number of genes that are involved in neuronal communication.’  

To do so, the team needed to target histone modifiers, a type of chromaton remodeler that alters proteins called histones that help regulate gene expression.

The anti-cancer drug romidepsin, inhibits histone modifiers.

‘In the autism model, HDAC2 is abnormally high, which makes the chromatin in the nucleus very tight, preventing genetic material from accessing the transcriptional machinery it needs to be expressed,’ said Dr Yan. ‘Once HDAC2 is upregulated, it diminishes genes that should not be suppressed, and leads to behavioral changes, such as the autism-like social deficits.’

Romidepsin loosened the chromatin in the nucleus, allowing the genetic material to gain access to the transcriptional machinery so they can be expressed.

Dr Yan and her team found that romidepsin restored the majority of the more than 200 genes that were suppressed in the autism animal model they used. 

‘The advantage of being able to adjust a set of genes identified as key autism risk factors may explain the strong and long-lasting efficacy of this therapeutic agent for autism.’ Dr Yan explained. 

About one in 68 children in the US have autism, according to the Centers for Disease Control and Prevention. People with this developmental disability have significant social, communication and behavioral challenges. 


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