Zebrafish as tool for drug discovery

As the only vertebrate model organism amenable to scalable screens, the zebrafish has long been touted as having the potential to deliver new drug candidates. In the past few years, drug candidates discovered in zebrafish have finally begun to reach human trials, suggesting that the potential may be turning to reality.

The webinar lead by Dr. Randy Peterson titled “Drug Discovery: Can Zebrafish Screens Really Lead to New Human Medicine?” reviewed the benefits of Zebrafish in the development of new medicine.

So why should we look at Zebrafish? As vertebrates, they share a high degree of sequence and functional homology with mammals, including humans. Due to the conservation of cell biological and developmental processes across all vertebrates, studies in fish can give great insight into human disease processes. As mentioned, Zebrafish also provide researchers a high degree of scalability and phenotypic richness. The ability to screen the whole organism grant scientists access to a variety of complex phenotypes relative to disease. In a recent review, Swinney and Anthony found that approximately two-thirds of the first-in-class drug developed in the last decade was based on the phenotype-based method instead of the typical target-based approach (Nature Review Drug Discovery, 2011).

According to Dr. Peterson, roughly eight to twelve compounds currently in clinical studies started with a zebrafish screen.

Dr. Peterson anticipates many opportunities for impact from therapeutics for nervous system disorders, methods of assessing and predicting drug exposure, and methods for screening in juveniles and adults.

To answer the question posed at the beginning of the webinar, “Can Zebrafish Screens Really Lead to New Human Medicine?” the answer is perhaps. The number of compounds now in clinical trials that originated with zebrafish screening shows that it’s gaining traction and leads one to believe that in the near future, we’ll be able to point to available drugs that all started with a zebrafish.


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