The promise of using patient-derived neurons to create disease models for drug screening is starting to bear fruit across a variety of neurological disorders vastly underserved by available therapies. New examples of human inducible pluripotent stem cell (iPSC) derived models of neurological disorders have identified a range of disease-relevant phenotypes by using specifically differentiated neuronal sub types, including Alzheimer’s disease, Amyotrophic lateral sclerosis (ALS), Parkinson’s disease, Rett syndrome, and several copy number variants (CNVs) associated with neuropsychiatric disorders and autism. The quality of patient care and research at BCH provides well-studied psychiatric and neurological patient populations that will facilitate additional development of preclinical disease models using iPSCs to support drug screening.

The recently formed Translational Neuroscience Center (TNC) at BCH created a Human Neuron Differentiation Service (HNDS) to prepare human neurons for researchers at BCH and the surrounding area using iPSC lines created from well-phenotyped and genotyped patients using optimized protocols for specific neuronal cell types. This service has begun addressing a large unmet need in the research community. The existing Assay Development Screening Facility (ADSF) core facility within the FM Kirby Neurobiology Center at BCH also provides complementary resources in the form of a high-throughput screening instrument to investigators for monitoring functional responses of neurons and other cell types. These paired facilities enable faculty to create neurons, develop assays, and run drug screens for new therapeutic approaches to disease.