Proteinopathies are diseases caused by protein misfolding and self-aggregation, which leads to altered neuronal function and neurodegeneration. Examples of proteinopathies include Parkinson’s Disease (PD), Alzheimer’s Disease (AD) and Amyotrophic lateral sclerosis (ALS).
Proteinopathies are complex diseases difficult to model in vitro and in vivo. Many therapies that showed promise in animal studies have failed in human clinical trials, emphasizing a translation gap in the drug discovery process.
Developing human physiologically relevant disease models is of high importance to identify and validate drugs therapeutic potential with higher confidence of clinical success. Human induced pluripotent stem cells (hiPSCs) have the potential to be differentiated into any cell type, retain patient-specific genetic backgrounds, mimic clinically-relevant human pathophysiology and respond appropriately to candidate therapeutics. These characteristics make hiPSCs an excellent tool for drug discovery.
In this study, Ncardia developed three in vitro assays based on hiPSC-derived neurons to model the aggregation of α-synuclein, Tau and TDP-43 – key hallmarks for diseases like Parkinson’s, Alzheimer’s or Amyotrophic Lateral Sclerosis.