Mice don't get Alzheimer's, so why test Alzheimer's drugs on them?

6 min read
Lab-grown human cells offer a revolutionary new model for bio-medical research.
In the course of delivering the 2013 Cartwright Lecture at Columbia University, molecular biology pioneer Sydney Brenner made a startling prediction. The Nobel Prize winner had done foundational work in genetic regulation of development — and as a side project, laid the groundwork for the development of the C. elegans worm as a model organism for genetic research — so most in the audience expected his lecture to cover some aspect of the evolution of simple nervous systems. Instead, he spent the majority of his time arguing that the age of the model organism was coming to an end. In short, thanks to advances in human genomics and tissue cultivation, the future of biomedical research would not require the necessary evil of animal testing, which, on top of ethical concerns, was a poor predictor of a drug's efficacy in human subjects.

This did not go over particularly well with the large group of biologists in the room, most of whom had built their careers conducting research on worms, flies, mice and rats, all standardized organisms designed to provide a controlled model for investigating biological processes. At the time, there was simply no foreseeable alternative to model organisms. Less than a decade later, however, that is starting to change, and Brenner's prediction is starting to be realized. In the near future, we may be able to conduct all biomedical research directly on lab-grown human cells.

Mice lie, Monkeys exaggerate

Animals are not always the ideal stand-in when studying potential treatments for diseases that represent the biggest threats to human health. In some cases, model animals may, in fact, present an impediment to crucial research.

Take the case of Alzheimer's disease (AD). Drugs designed to treat Alzheimer's have been remarkably unsuccessful, with a 99% overall rate of failure in clinical trials. This is a huge problem because the US alone is projected to see 12.7 million cases of Alzheimer's dementia by 2050 if no effective treatment is…
Peter Weinberg
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