Biological Mechanisms in the Treatment of Neuropsychiatric Diseases

Dear Colleagues,

Diseases affecting the nervous system are the leading cause of overall disease burden in the world, affecting 3.4 billion persons. Although research in basic neuroscience has thrived for several decades, interest in applied neurosciences—developing new therapies—has lagged behind until the recent few years, when we have seen a resurgence of commitment to therapeutics development. The urgent need for progress in the understanding of molecular, circuit-related, and genetic causal factors for serious neurological and psychiatric conditions is again attracting the considerable governmental and private investments needed to progress an empirically founded idea into a successful therapeutic.

This Special Issue of Cells focuses on novel methodologies, promising cellular and molecular mechanisms, and treatments that offer hope for patients with a range of disorders and diseases for which there is a considerable need for safer, better tolerated, more effective medical treatments. Successful clinical trials require valid, quantitative outcome measures that are sensitive to the physiologic and biological features of the disease under study. However, limitations in outcome assessment methodology are believed to have hampered the discovery of effective treatments. Advances in the understanding of the molecular mechanisms of genetic diseases have ushered in a renaissance of interest in rare neurological conditions for which, unlike most neuropsychiatric conditions, the pathology is well-characterized and can thus inform rational therapeutic development. The application of precision medicine approaches within highly heterogeneous neuropsychiatric conditions is being pursued as a potentially more successful strategy where the “one size fits all” approach has clearly failed. Moreover, novel methods for preclinical drug screening, which are more relevant to human biology, have come to the fore as potentially more predictive of human safety outcomes.

With this selection of ongoing work in the field, we hope to encourage an optimistic mindset toward the possibility that the following decade will see the creation of many new and better therapies to improve the lives of millions of people with these conditions.

Dr. Randall D Marshall
Dr. Frank S. Menniti
Guest Editors

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• neurodevelopmental conditions
• rare neurological disease
• molecular mechanisms of novel CNS treatments
• precision medicine in neuropsychiatry
• organoids
• molecular mechanisms of effective treatments