However All May Not Be Lost for the Once Promising Treatment
An original article published in the New England Journal of Medicine has delivered disappointing news for parents and researchers counting on positive results in an oxytocin clinical trial for children with autism. Previous research has suggested that treatment with intranasal oxytocin may reduce social impairment for individuals on the spectrum. This speculation led to high hopes that the hormone could become a possible new ASD treatment. This current placebo-controlled phase 2 trial was conducted over 24 weeks and involved children and adolescents aged 3 to 17 years with autism spectrum disorder. A total of 146 participants were assigned to the intranasal oxytocin group and were administered the hormone up to twice daily. There were 144 participants assigned to the placebo group. Sadly, at the end of the trial period the results showed no significant change in social and cognitive functioning between the exposed group and the placebo group. The study’s disappointing conclusion may seem like an end to a promising autism treatment. However, an accompanying editorial from Daniel H. Geschwind, MD, PhD argued that the study’s findings were premature given the trial’s limitations. Geschwind found many faults with the study’s design. He suggested that the dosing may have been inadequate, the participants’ age range too broad, and the outcome measures too insensitive “to capture social motivation.” Geschwind also pointed out that since autism is “genetically heterogeneous,” oxytocin may provide benefits to some genotypes more than others. In the future, he hopes that precision medicine evolves where treatments will be based on genetically informed disease mechanisms that would leverage genetic and molecular biomarkers in trial designs. In other words, Geschwind still has hopes for intranasal oxytocin as an autism treatment.
