The development of drugs for brain disorders is plagued by failures to translate lab-based research into effective therapies in patients. To address this problem, we have established several platforms incorporating the testing of patient-derived human brain tissue and cells into the drug discovery and development pipeline. We are using these platforms with molecular pharmacology, chemical biology, medicinal chemistry, as well as in silico and phenotypic screening assays to uncover novel drug targets and drug candidates for brain disorders. These platforms and examples of their use will be presented in this seminar.