Many parents of children with autism have expressed to me their dismay that the anti-vaccine lobby is sucking all the oxygen out of the room for autism awareness. They feel that just being a parent of a child with autism makes others assume that they are anti-vaccine. They also worry that resources and attention are being diverted from promising legitimate research because of all the attention being paid to the failed vaccine hypothesis.

So it is good to occasionally focus on mainstream autism research to show that progress is being made, despite the unfortunate anti-vaccine sideshow.

A recent study published in the latest issue of Pediatrics shows that early intervention in toddlers with autism can have significant benefits. The study is a randomized controlled trial of  the Early Start Denver Model compared to conventional treatment in 18-30 month old children with a diagnosis of autism spectrum disorder (ASD). The study is a reasonable size for this kind of intervention – 48 children were randomized – and this is sufficiently powered to get statistical significance. But it should be noted this is still a smallish study and replication to confirm the results is welcome.

Another potential weakness is that the control group was “referral to community providers for intervention commonly available in the community.” Therefore the control group was not standardized and it’s possible this group was sub-optimally treated. Further, while the groups were randomized they were not blinded.

The results of the study are quite robust:

Compared with children who received community-intervention, children who received ESDM showed significant improvements in IQ, adaptive behavior, and autism diagnosis. Two years after entering intervention, the ESDM group on average improved 17.6 standard score points (1 SD: 15 points) compared with 7.0 points in the comparison group relative to baseline scores. The ESDM group maintained its rate of growth in adaptive behavior compared with a normative sample of typically developing children. In contrast, over the 2-year span, the comparison group showed greater delays in adaptive behavior. Children who received ESDM also were more likely to experience a change in diagnosis from autism to pervasive developmental disorder, not otherwise specified, than the comparison group.

The results of this study are plausible and encouraging, and hopefully will lead to further studies to confirm the results.

The concept behind ESDM is this (from the EarlyDevelopment Studies Lab website):

Autism impedes this process of child learning and adult scaffolding, in several ways.  Children with autism are less attentive to other people and so miss many learning opportunities by watching. Young children with autism have impaired communication skills, so they have difficulty learning from the communications that adults provide. Young children with autism also have difficulty imitating others, and so miss opportunities to learn by copying what others do. Young children with autism may not be quite as flexible in their play skills as others, and may enjoy repeating favorite actions with objects, rather than generating new play ideas and learning through discovery, and this limits their learning. And finally, young children with autism may not find social experiences as inherently rewarding as other children, which can result in reduced time in interactions of all sorts. Since interactions are a primary learning opportunity for toddlers, fewer interactions mean fewer learning opportunities.

The ESDM is designed to make learning for young ASD toddlers more fun, dynamic, and child-directed rather than repetitive. The purpose is to compensate for the specific areas of difficulty of ASD children.

While not a “cure” if these results hold up ESDM can be a very effective early intervention for children with ASD. It highlights the potential benefit of early detection and intervention programs.

It also brings to mind the broader context (which is relevant to many neurological diseases and disorders) that at times focus on finding a cure might overshadow research and even clinical attention paid to interventions that improve quality of life and mitigate the impact of a disorder. Finding a cure is, of course, always the long term goal. But we have to be realistic in recognizing that for many complex disorders it is a very long term goal. Meanwhile there is much that can be done to significantly improve outcome, and these types of interventions should not be neglected simply because they are not a “cure.”

Patients and families need to recognize that often there is much that can be done, and they should not neglect medical attention under the false belief that “nothing can be done” simply because a cure does not exist yet. Sometimes, even clinicians need to be reminded of this fact.

Also, research priorities should reflect a reasonable balance between searching for a cure and improving outcomes in the meantime. Grassroots advocacy groups that raise funds for clinical research, in my opinion, often over-emphasize research that has the potential to find a cure in the short term, and may in fact slow overall research progress by upsetting the balance between various kinds of research (basic, therapeutic, and symptomatic).

So it is good to see a solid symptomatic trial like this one get attention, and to remind people that we can make a significant difference with science-based interventions in the lives of children with ASD, even if we don’t fully understand the disorder or know how to cure it.