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New Drug Trial May Help Autism Patients

New Drug Trial May Help Autism Patients


Two companies have joined forces to develop disease modifying treatments for autism spectrum disorders and Fragile X syndrome.

National Autism Society LogoSwiss pharmaceutical company Roche and Seaside Therapeutics based in Cambridge, Massachusetts in the US are hoping that the drug once developed will target the molecular basis and in turn the core symptoms of these neurodevelopmental disorders.

The drug is not being touted as a cure for autism as there is no known cure for the disorder

And it will be some years before the drug will be able to help sufferers in the UK where there are more than 500,000 people with autism – 1 in 100 of the population.

Tests will have to take place in the US first before a green light is given for the drug to be used.

The two firms have drugs in late-stage tests targeting Fragile X syndrome, the most common form of inherited intellectual disability and the most common known genetic cause of autism.  And though Fragile X accounts for only a small percentage of autism patients, early studies suggest the drugs may work in other forms of autism, too.

The drug on trial will act by blocking mGluR5, a brain receptor implicated in both fragile X and autism.

The number of studies of the drugs so far remains small, and the trials themselves have been relatively small. Of those that have been completed, the effect has been dramatic in some patients but negligible in others.

Researchers have seen some improvement in the behavior of trial participants – in particular, a decrease in the anxiety that some of these patients have.

Fragile X syndrome, a rare genetic disease with symptoms similar to autism. It is an inherited intellectual disability in boys that is caused by a change in the gene FMR1 on the X chromosome.

A spokesperson for the National Autistic Society said: “Autism is a serious, lifelong and disabling condition for which there is no known cure.

“There are no specific drugs for the treatment of autism.

“In trying to minimise challenging behaviour, the NAS would always advocate providing the right environment and strategies to suit an individual’s needs, with appropriate support from trained staff.  We need to try and understand what a person’s behaviour is communicating, and then where possible help them to learn more appropriate means of communicating this.

“In some instances, drugs can be used to control issues like anxiety levels enabling the person to use the strategies they have been taught. These should be used appropriately and be carefully monitored.”

 



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