Understand Translational Research Tools

Cell and Animal Models

A disease model is an animal or cell which displays all or some of the disease processes that are observed in the actual human. 

  • Studying disease models aids understanding of how the disease develops and testing potential treatment approaches.
  • Preclinical studies in animal or cell models demonstrating the safety and efficacy of a potential treatment must be submitted to the FDA prior to being granted approval for trials in humans.
  • Cell and animal models include:
    • Primary or patient-derived immortalized cell lines.
    • Patient-derived induced pluripotent stem cell (iPSCs).
    • Organoids, which are tiny, self-organized, three-dimensional tissue cultures that are derived from stem cells. Such cultures can be crafted to replicate much of the complexity of an organ, or to express selected aspects of it like producing only certain types of cells.
    • Yeast.
    • Slime mold.
    • Roundworm.
    • Fruit fly.
    • Zebra fish.
    • Frog.
    • Mouse. 
    • Rat.
    • Rabbit.
    • Pig.
    • Other mammals.
  • Animal Model Qualification Program provides information about the steps to have an animal model qualified by the FDA for preclinical studies.

Resources

Bioassays
Assay Guidance Manual National Center for Advancing Translational Sciences (NCATS) (link)
Biomarkers
CDER Biomarker Qualification Program U. S. Food and Drug Administration (FDA) (link)
Cell and Animal Models
Animal Model Qualification Program U. S. Food and Drug Administration (FDA) (link)
Biorepository
NCI Best Practices for Biospecimen Resources National Cancer Institute (NCI) (link)