During an ongoing search for autism spectrum treatments, recent findings have shown promising results for drug-based therapy. 

To date, there are no known cure or possible treatment for autism spectrum disorder. However, a drug that is currently being tested on deletion mice by a group of researchers showed promising results that could potentially treat the said disorder.

Autism spectrum disorder (ASD) is the name for a group of developmental disorders. According to National Institute of Mental Health, ASD includes a wide range or a ‘spectrum’ of symptoms, skills, and levels of disability. People suffering from ASD often show the following characteristics:

  • Ongoing social problems that include difficulty communicating and interacting with others
  • Repetitive behaviors as well as limited interests or activities
  • Symptoms that typically are recognized in the first two years of life
  • Symptoms that hurt the individual’s ability to function socially, at school or work, or other areas of life

According to a statistics released early this year by Talk About Curing Autism (TACA), an organization dedicated to spreading awareness about autism, 1 out of 42 boys and 1 in 189 girls in the United States are diagnosed with autism.

New research suggests a possible treatment for #autism spectrum disorder #ASDClick To Tweet

Available treatments today only help in alleviating many of the symptoms suffered by people diagnosed with autism. While it’s still early to say, a possible treatment for autism spectrum disorder could give hope to millions of people affected by this condition around the world.

A New Hope: Possible Treatment for Autism Spectrum Disorder

ASD is commonly associated with human chromosome 16p11.2 deletion syndrome, a condition wherein 27 genes on chromosome 16 is absent. This deletion is characterized by different intellectual disability such as impaired language, communication, and socialization skills.

Chromosomal Deletion which the possible treatment for autism spectrum disorder hopes to treat
Genetics Home Reference | ghr.nlm.nih.gov

A very recent study conducted by Mark Bear at Massachusetts Institute of Technology (MIT) and Jacqueline Crawley at the University of California at Davis suggested a potential treatment for ASD. It appears that the drug R-Baclofen was able to reverse the cognitive deficits and improve the social interactions in two lines of 16p11.2 deletion mice. The study read:

“16p11.2 deletion syndrome includes symptoms of intellectual impairment and autism. Cognitive and social deficits were previously detected in three independently generated lines of 16p11.2 deletion mice. We employed two of these lines to test the hypothesis that the GABAB agonist R-baclofen could improve performance in behavioral domains relevant to symptoms of the human 16p11.2 deletion syndrome. Our two separate laboratories used the same R-baclofen dose and treatment time course to test 16p11.2 deletion mice and their wildtype littermates on distinct but complementary behavioral assays.”

Mark Bear
Mark Bear | Picower Institute | picower.mit.edu

Apparently, the findings mean that humans with 16p11.2 deletion syndrome and ASD could potentially be treated.

“Our findings that R-baclofen improved performance on three cognitive tasks and reversed deficits on two measures of male-female reciprocal social interactions suggest the drug may have therapeutic utility in humans with 16p11.2 microdeletion syndrome.

Racemic baclofen has been used clinically for many years for the treatment of spasticity in children and adults with cerebral palsy, and placebo controlled trials in fragile X and autism using R-baclofen have shown the drug is safe and well-tolerated in patients with other developmental brain disorders,” the researchers explained.

The two teams found that R-Baclofen could improve scores on several learning and memory tasks of deletion mice. Crawley said:

“This unique corroboration of findings by two independent labs, using two distinct lines of mice with the same mutation, increases confidence that R-baclofen may be an effective pharmacological treatment for some of the symptoms of human 16p11.2 deletion syndrome, including intellectual impairment and autism.”

Bear, on the other hand, said that the findings are particularly exciting on two fronts.

“First, the results show that diverse genetic causes of intellectual disability and autism may converge on a limited number of pathophysiological processes that can be ameliorated pharmacologically. Thus, a treatment for one genetically defined disorder may be beneficial for another with phenotypic overlap. Second, R-Baclofen has a well-understood safety profile and is well-tolerated in children and adults, making clinical studies feasible in the near future.”

While the test showed positive results, it is still unknown how R-Baclofen alters the biology in 16p11.2 deletion mice and if the findings are also applicable to human physiology. In some clinical trials, R-Baclofen was found to ease social problems, irritability, and other characteristics in some children with autism. However, the drug appears to work in a small group of individuals only, with some findings falling short of statistical significance.

Elizabeth Berry-Kravis, a professor of child neurology at Rush University in Chicago who was not involved in the new study, said:

“We have a lot of positive effects in mouse models, and they’ve been very challenging to translate clinically.”

Right now, only further studies and clinical trials will prove if R-Baclofen could be the possible treatment for autism spectrum disorder.

What do you think about this potential ASD treatment?

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