Most of the common neuropsychiatric disorders probably represent a collection of less common – even rare -- diseases. We need to begin to think in terms of “lithium-responsive mood disorder” or “clozapine-responsive psychotic disorder.” Such treatment-responsive subgroups may share specific genes or other characteristics. Each of the current diagnostic categories may actually encompass several subgroups for which a new treatment needs to be designed, as underscored by the example of ivacaftor in CFTR therapy summarized above. Autism, which is likely a polygenic disorder, may serve as a good model in developing treatment strategies in the broader realm of neuropsychiatry. Recent work has identified several genomic anomalies associated with autism [reviewed in Malhotra and Sebat, 2012]. Each genetic alteration may therefore implicate a distinct molecular etiology, and hence a different potentially ‘druggable’ molecular target. Depending on the underlying molecular or neural substrates, which may differ even within the same diagnostic classification, effective treatment may require cognitive or behavioral treatments rather than medications. Most may require both.
