Julia Schueler, Head of in vivo Operations, Oncotest, a Charles River company 10.11.16
Preclinical studies in oncology are frequently based on the use of mouse models. On average, the protein-coding regions of mouse and human genomes are 85% identical and the mouse genome can be easily manipulated.
But as the saying goes, close only counts in horseshoes.
The genomic identity between H. sapiens and M. musculus cannot compensate for significant species differences that, unfortunately, influence the outcome of experiments in not-so-beneficial ways. One good example is the failure of 9-AC in Phase II clinical trials despite very impressive preclinical data: the compound failed due to its small therapeutic window in humans as compared to mice.1
To overcome this limitation, multiple efforts have been undertaken to establish and characterize large collections of patient-derived tumor xenograft (PDX) models—mice implanted with cancerous tissue from human patients—for cancer research.
Although this model system dates back to the late 1980s, it recent
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