The Difficult Path to the Discovery of Novel Treatments in Psychiatric Disorders

CNS diseases, including psychiatric disorders, represent a significant opportunity for the discovery and development of new drugs and therapeutic treatments with the potential to have a significant impact on human health. CNS diseases, however, present particular challenges to therapeutic discovery efforts, and psychiatric diseases/disorders may be among the most difficult. With specific exceptions such as psychostimulants for ADHD, a large number of psychiatric patients are resistant to existing treatments. In addition, clinicians have no way of knowing which psychiatric patients will respond to which drugs. By definition, psychiatric diagnoses are syndromal in nature; determinations of efficacy are often self-reported, and drug discovery is largely model-based. While such models of psychiatric disease are amenable to screening for new drugs, whether cellular or whole-animal based, they have only modest face validity and, more importantly, predictive validity. Multiple academic, pharmaceutical industry, and government agencies are dedicated to the translation of new findings about the neurobiology of major psychiatric disorders into the discovery and advancement of novel therapies. The collaboration of these agencies provide a pathway for developing new therapeutics. These efforts will be greatly helped by recent advances in understanding the genetic bases of psychiatric disorders, the ongoing search for diagnostic and therapy-responsive biomarkers, and the validation of new animal models.