Efficacy & Toxicology

Researchers must demonstrate not only that a new therapeutic candidate is effective against a target disease (i.e., elicits the desired effect) but also that it is safe at the dose intended for human treatment (i.e., does not cause significant toxic side effects).

To address efficacy testing, NCATS scientists in the Assay Development and Screening Technologies (ADST) program aim to develop disease-specific models, primarily for rare diseases, against which large libraries of compounds can be screened to identify potential therapeutic candidates.

Additionally, through the Tissue Chip for Drug Screening program, the Center supports academic researchers who are developing 3-D human tissue chips that accurately model the structure and function of human organs, such as the lung, liver and heart. Researchers can use these model systems at earlier stages of discovery and development to more efficiently predict whether a candidate agent is safe or toxic in humans.

In another area of toxicity testing, NCATS scientists in the collaborative Toxicology in the 21st Century (Tox21) program conduct assay development and toxicity testing for thousands of environmental chemicals to which humans are routinely exposed.