Ten years ago, if someone had suggested that we organize a translational meeting on autism and related disorders, we would have regarded the idea as too premature. The fact that the Roche–Nature Medicine Translational Neuroscience Symposium on Autism and other developmental brain disorders, held on 17 and 18 April in Buonas, Switzerland, was one of the best meetings we have ever been involved in clearly illustrates how far this discipline has come in a decade.

The goal of the meeting was to explore how other neurodevelopmental diseases, particularly those with a monogenetic basis, could inform autism research. This goal was certainly met, but the meeting also highlighted the spectacular progress that has been made toward understanding and treating forms of mental retardation.

Successful translational research transforms molecular understanding of disease into a new therapy, a process that the accompanying figure (Fig. 1), which several speakers showed during their talks, depicts as a cycle. It was quite exhilarating to see how, for diseases such as neurofibromatosis type I, tuberous sclerosis and fragile X syndrome, researchers are tantalizingly close to completing their first full turn of this circle.

Figure 1
figure 1

Translational research as a cycle that begins and ends with human disease.

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For these diseases, investigators have a good genetic grasp, informative animal models, a working molecular understanding of pathology and 'druggable' targets. Clinically available compounds that can treat aspects of disease pathology in preclinical models are beginning to be tested in people. The challenge for researchers in this field is now twofold: to design rigorous clinical trials to investigate the true therapeutic effect of the existing molecules and to move away from the existing pharmacopoeia, instead developing the new generation of small molecules and other therapies that will make it to the clinic.

For some diseases, such as Rett syndrome, progress has also been solid, but is presently a couple of steps behind in the translational cycle. Although the animal models of Rett syndrome have been useful to the point of showing that aspects of the disease might be reversible, we still lack mechanistic insight and specific molecular targets.

In the case of autism, spectacular progress has been made in understanding its genetic basis. Some of the alterations found in people with the disease, such as mutations in members of the shank and neuroligin protein families, have already been introduced into mice, with very intriguing results. But the fact remains that there is much work to do in creating animal models before investigators can figure out the molecular pathophysiology of autism. However, research into the other neurodevelopmental diseases is leaving a clearly marked trail for the autism field to follow as guidance.

We encourage you to visit the meeting's website (http://www.nature.com/natureconferences/tns2009/), where you can find a write-up of the event and, in due course, information about the next Translational Neuroscience Symposium. We are very proud of our partnership with Roche to create this symposium series, and we look forward to future, equally stimulating, meetings.