In Brief
A small clinical trial has demonstrated that 100-year-old drug can relieved symptoms of autism in children. The work has also generated a unifying theory of ASD linking the disorder to altered metabolic processes.

Old Drug, New Tricks

A small clinical trial from the University of California, San Diego, has just yielded some promising results for those living with autism spectrum disorder (ASD). The results indicate that suramin, a 100-year-old drug used to treat African sleeping sickness, can measurably, albeit transiently, improve ASD symptoms in children.

This has led the research team to conclude that ASD in many children may be caused by a treatable metabolic syndrome and that, for some people with ASD, the right treatment can improve symptoms since they are not necessarily permanent.

*4* 100-Year-Old Drug May Be the Key to Treating Autism
Image Credit: Abhijit Bhaduri / Flickr

ASD is just that — a spectrum, and many children fall somewhere on that spectrum. According to the World Health Organization, about one in 160 children worldwide have ASD, although the CDC estimates that number to be one in 68. While it is not entirely clear whether the incidence of ASD is increasing, or detection of ASD is changing, or some other mechanism is at work making the numbers grow, there is no doubt that many people are affected.

The UCSD team is now focusing on metabolism — the shared language of the brain, immune system, and gut which allows the three linked systems to communicate. In people with ASD, each of these systems works differently, and the communication between them is altered.

The researchers chose to test suramin because it inhibits purinergic signaling, a cell communications process that takes place in metabolism. Within seven days, all five of the children treated with suramin showed a steady improvement of symptoms, with no change at all shown in the placebo group.

New Approaches To ASD

These results mark the first time any drug has shown the potential to actually alter symptoms of ASD. Of course this is a small first trail, and the treatment may never be available depending on further research outcomes. Even so, these results are likely to prompt a major shift in the way we think about autism.

If the researchers are right, abnormally persistent cell danger response (CDR) is what’s producing the metabolic syndrome causing ASD. Both environment and genes are factors in the CDR, so it’s possible that genetic causes alone might produce the metabolic syndrome and ASD. However, if a metabolic syndrome is what’s behind ASD symptoms, it can be treated, even though the genes can’t be.

This research also provides the first real unifying theory for the root cause of ASD. The lack of such a theory has been a huge factor in pharmacologic failures in treating aspects ASD. Treatments weren’t targeting the aspect of autism that could lower people’s quality of life and were sometimes worse than symptoms.

However, if this unifying theory is right that CDR and problems in purinergic signaling play an important role in some forms of ASD, then doctors should be able to treat some symptoms of ASD — such as difficulties with verbal communication, fear of changes in routine, and social anxiety — without suppressing the traits that sometimes make people with ASD exceptional.