3 Reasons Genetically Engineered Mice Are Vital to Drug Development

April 16, 2015 Updated: April 23, 2016

The mouse is one of the most commonly used animals for drug development research, partly because mice and humans share similarities in anatomy, physiology and disease. According to a recent article by the Stanford School of Medicine, mice and humans share amazing genetic similarity in the way their genes are expressed, validating the importance of lab mice in studying and treating human disease.

But the mice used in drug research are typically not the garden-variety type. These lab animal models are often genetically engineered to improve their effectiveness as research tools.

Gavin Cooper, Director of Global Marketing at Taconic Biosciences, a provider of genetically engineered models (GEMs) for research, explains, “Nearly nine out of ten new drugs that reach the clinical testing phase with humans never make it to market. That failure rate has spurred great demand for research animal models that better predict how drug therapies will work in humans. For researchers in commercial and academic organizations, the solution is often GEM mice.”

GEMs are vital to drug development for three key reasons.    

  • GEM mice can be engineered to replicate a particular human disease, like diabetes or Alzheimer’s, making them ideal for studying the disease. Thanks to sophisticated technology, mice can be designed to develop symptoms indicative of specific human diseases. They also can be humanized – made more similar in makeup to man – by adding human genes, cells or tissues, such as tissue from a cancerous It’s even possible to replicate portions of the human immune system in a mouse to study how our immunity impacts disease and drug treatments. Researchers can use these advanced models to gain more confidence that the outcome of a pre-clinical (animal) study will be similar to the outcome of a clinical (human) study.
  • GEM mice can be used to test new drugs or drug treatments for safety or efficacy. It’s not only essential to test if a drug will treat a disease effectively; it’s also important to understand how the body will absorb, distribute, metabolize and excrete the drug, and whether it will pose any safety issues. GEM mice that are engineered for this purpose are invaluable tools for testing drug efficacy, safety and toxicity.
  • GEM mice can serve as avatars to develop personalized treatments for cancer patients. Researchers now know that patients with the same form of cancer may respond very differently to the identical treatment because their genetics and their tumors differ. That finding has fueled demand for oncology treatments tailored to the individual, a trend known as personalized medicine. A GEM mouse can be engrafted with a specific patient’s tumor tissue or cells to test a cancer treatment that’s personalized to the individual, before moving forward.

As researchers continue to find ways to improve human health, GEM mice will increasingly serve as essential tools in helping them better understand diseases and the drug therapies that can treat them.