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Hormonal therapies have been found to help communication skills and social interactions in children and men with autism.

When the US Food and Drug Administration recently asked people with autism and their caregivers what drugs they would find most useful, the top result was overwhelmingly something that would help with communication and socialisation behaviours, says Paulo Fontoura of the pharmaceutical firm Roche.

Some people with autism take ADHD medication for help with attention, or anti-psychotics for help with aggression, but there are no drugs available to help with things like social difficulties.

Now two separate studies have tested approaches that target the body’s system for regulating vasopressin, a hormone known to affect social interactions.

In the first study, 30 autistic children aged 6 to 12 were given a nasal spray to use daily for 4 weeks. Around half were given a placebo spray, while the others got one containing vasopressin.

The children’s social abilities were assessed at the start and end of this period by getting their parents or guardians to answer a standard questionnaire. Doctors also rated the children’s social skills using a standard assessment scale and the children took tests that measured their ability to interpret the emotions of other people based on their facial expressions – a skill often diminished in people with autism.

Emotional recognition

The children who received vasopressin showed a greater improvement in their social abilities after 4 weeks than those who received the placebo, as rated by both doctors and parents. They also improved at recognising the emotional states of faces.

The team found that those who naturally had higher concentrations of vasopressin in their blood before the study showed more of an improvement than the others.

The hormone nasal spray also seemed to reduce symptoms of anxiety, an effect that was magnified in the children with higher pre-study vasopressin levels. For this same group, parents or caretakers also reported diminished repetitive behaviours, another common symptom for people with autism.

It’s not clear yet why this may be, says Karen Parker at Stanford University, who led the study. It could be that people with the lowest natural levels of vasopressin didn’t benefit as much because they would need a higher dose, she says.

“What they’re doing is supplying vasopressin. What we’re doing is blocking vasopressin signalling,” says Fontoura, who worked on the second study.

Fontoura and his colleagues worked with 223 adult men with autism, who took either a placebo or a low, medium or high dose of a drug called balovaptan every day for 12 weeks.

Improved communication

When the participants were rated on the same scale used to assess the children’s social abilities, there was no sign of an improvement in the men’s social skills.

However, when the team used a different test that assesses daily living skills, their analysis suggested that there were some improvements in communication and social abilities that increased in line with the dose of the drug.

These are small, early studies, and we don’t know yet whether targeting vasopressin pathways will have meaningful or lasting effects. One unanswered question is why both boosting and blocking vasopressin signalling appears to have beneficial effects.

“The best way for me to reconcile that might be that there’s an optimal band of vasopressin functioning, and you don’t want to deviate too high or too low from that,” says Parker.

Fontoura stresses that the vasopressin-suppressing drug isn’t intended as a “cure” for autism. “We’re not saying by any means that we’re curing something or treating diseases, we’re helping people with certain aspects of their behaviour that they may find challenging,” he says.

But some think drugs aren’t necessarily the best way to do this. “Before moving to long-term hormone treatments to do something like cause an autistic person to make eye contact, we would encourage researchers and clinicians to think about what an autistic person is actually experiencing and what they need,” says Zoe Gross of the Autistic Self Advocacy Network in Washington DC.

“Teaching an autistic person ways to signal attention without making eye contact, or non-autistic people ways to communicate more directly, may be effective in improving socialisation without resorting to medications with unknown long-term effects, or focusing on ‘normalising’ the autistic person’s behaviour,” says Gross.

Journal reference: Science Translational Medicine,