Zebrafish: A predictive model for assessing drug toxicity and efficacy

TOXI 6

Patricia McGrath, pat@phylonix.com, President & CEO, Phylonix, 100 Inman Street, Cambridge, MA 02139
Zebrafish have several advantages for assessing drug toxicity and efficacy, including transparency, rapid development, easy drug delivery, and use of small quantity of drug per experiment. In addition, a statistically significant number of animals can be assessed in each experiment. Here, we describe in vivo assays for assessing drug induced organ toxicity, including, heart, GI system, kidney, and CNS. After compound treatment, organ specific phenotypes in zebrafish are strikingly similar to defects observed in mammals. We also describe several disease models for assessing drug efficacy using a chemical phenocopy approach. Models for most major diseases, including those involving angiogenesis, oxidation, and neurodegeneration, have been generated in zebrafish. Toxicity vs. efficacy can also be easily assessed in large structurally diverse chemical libraries. Studies in this model organism are designed to complement in vitro and rodent methods.