High-Content Drug Screening for Rare Diseases
June 2017
in “
Journal of Inherited Metabolic Disease
”
TLDR High-content screening is useful for finding new treatments for rare diseases and has led to FDA-approved drugs.
The document from 2017 reviews the use of high-content screening (HCS) in the development of treatments for rare diseases, highlighting its role in drug repositioning and the potential for personalized medicine. It notes the advancements in HCS technology, which allow for rapid compound screening and multiplex measurements, and the use of computational modeling to predict drug responses. The paper acknowledges the challenges of HCS, such as the need for extensive data storage and analysis, but also points out its successes, including the FDA approval of six drugs identified using HCS in 2016. Quality control strategies and target deconvolution processes are discussed as essential components of HCS. Examples of HCS in rare disease research are given, including the identification of treatments for FOP, Huntington disease, and Von Hippel-Lindau disease. The document also mentions financial support from foundations and the Horizon 2020 program, and confirms the absence of conflicts of interest and adherence to ethical standards in the research presented.
