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Autism Treatment Research

A. Bailey
Published 2014 · Medicine, Psychology

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Since the launch of Autism Research, there have been exciting advances in the genetics and neuroscience of autism spectrum disorder (ASD). Concomitantly, there has been a significant increase in the number of welldesigned and controlled treatment studies, reflecting both the development of new, more effective developmental and behavioural interventions, as well as pressure for research that addresses the immediate needs of affected individuals. Although the complex neurobiology of ASD is an obstacle to the rapid development of novel biological treatments, the genetic findings implicating synaptic processes have finally stimulated the interest of the pharmaceutical industry in ASD. Indeed, the development of mouse models of Fragile X, tuberous sclerosis, and Rett Syndrome has facilitated drug trials for these conditions, whose findings may be more broadly relevant. In parallel with these advances, the increased attention paid to ASD over the last 5–10 years has highlighted the burden of comorbid psychiatric disorders and the need for effective treatments to improve quality of life. Because of these developments, Autism Research is expanding its scope to include adequately powered treatment studies, with a particular emphasis on research that reveals underlying mechanisms, either by testing causal hypotheses or by revealing variations in responses, which prompts refinement of explanatory models. What are some of the overarching challenges that treatment research will face over the coming decades? From a scientific perspective, variations in severity and persistence into adulthood indicate the need for a range of treatment approaches. Almost since autism was first described, clinicians and researchers have appreciated that there is no one intervention that is effective for all individuals. Additionally, almost regardless of the approach taken, behavioural and educational interventions seem to be most beneficial for individuals with greater intellectual abilities. The early intervention studies raise the questions of the extent to which treatment gains persist into adolescence or early adult life and whether individuals who show the best response to treatment would also have a better long-term outcome without intervention. Of course, long-term outcome is not the only measure against which interventions should be judged, as improved quality of life during childhood for the individual and his or her family is a worthwhile gain in its own right. But undertaking long-term follow-up studies and understanding why less able individuals often show reduced treatment response are key objectives. Possibly, the most handicapped individuals may benefit from novel pharmacological interventions, as they are more likely to be affected by chromosomal abnormalities and single-gene causes of ASD. Indeed, the recent trials of drug treatments for single-gene aetiologies of ASD illustrate the potential of this approach, but neurophysiological and neuroimaging studies to understand mode of therapeutic action are particularly difficult in these populations. The vast majority of treatment research for ASD focuses on childhood. Much of this emphasis is justified by the severe behaviours shown by some young children with autism and also the pressure upon parents and siblings. Nevertheless, as new environmental challenges arise in adolescence and adult life, skills learnt much earlier in development may not suffice and different interventions may be required. The education literature on typically developing adults is replete with examples of the differences between child and adult learners, and the need for modified educational approaches in adult life: there is no reason to suppose that ASD will be the exception to these general observations. The focus on male participants in research is almost as striking as the current emphasis on childhood. The exclusion of female research participants is often because of concerns about statistical power or a wish to reduce heterogeneity, but an unintended consequence of this focus is limited research into whether the brain basis of ASD is identical in both sexes as well as whether interventions should differ in emphasis according to sex. These are important omissions, as we have become increasingly aware that the behavioural and cognitive phenotypes in the most able females can differ from that in males, which may partly explain the apparent underdiagnoses of affected women. Some of these women will go on to raise families and almost nothing is known about how to help these mothers succeed in that role. Additionally, we know much less about the skills of able affected women, whereas the identified mathematical, computing, or physical science strengths of some affected men have been instrumental in developing specific vocational opportunities.

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