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Zebrafish Discoveries Leading to Clinical Trials

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Numerous drug treatments that have recently entered the clinic or clinical trials have their genesis in zebrafish. Zebrafish are well established for their contribution to developmental biology and have now emerged as a powerful preclinical model for human disease therapeutic development.

Publication: PMC6570580
Title: Visualizing engrafted human cancer and therapy responses in immunodeficient zebrafish.
Indication: Cancer (Ewing’s Sarcoma; OMIM: 612219)
Clinical Trial: NCT01858168
Drug: Olaparib and temozolomide
Reported Observations: Zebrafish robustly engraft a wide array of human cancers at 37°C that grow with similar kinetics and have similar histology as those engrafted into NSG mice.

Publication: PMC5994396
Title: Preclinical Animal Models for Dravet Syndrome: Seizure Phenotypes, Comorbidities and Drug Screening.
Indication: Epilepsy (Dravet syndrome; OMIM: 607208)
Clinical Trial: NCT04462770
Drug: Clemizole
Reported Observations: Given the evidence that the zebrafish scn1labs552 model recapitulates salient genetic, behavioral, electrophysiological phenotypes observed in DS patients, and has been pharmacological validated against known AEDs, this model appears ideal for screening compound libraries for antiepileptic activity.

Publication: PMC5752378
Title: Neuroleptics as therapeutic compounds stabilizing neuromuscular transmission in amyotrophic lateral sclerosis.
Indication: Neurodegen (amyotrophic lateral sclerosis; OMIM: 105400)
Clinical Trial: NCT03272503
Drug: Pimozide
Reported Observations: We identified a class of neuroleptics that restored motility in C. elegans and in zebrafish, and the most potent was pimozide, which blocked T-type Ca2+ channels in these simple models and stabilized neuromuscular transmission in zebrafish and enhanced it in mice.

Publication: PMC2265478
Title: Identification of genetic and chemical modulators of zebrafish mechanosensory hair cell death.
Indication: Deafness (drug-induced hearing loss, OMIM: 580000)
Clinical Trial: NCT05730283
Drug: ORC-13361
Reported Observations: We selected compound 90 as our lead compound for full pre-clinical evaluation based on its superior protective activity in the zebrafish assay.

Publication: PMC6958429
Title: Zebrafish Models of Diamond-Blackfan Anemia: A Tool for Understanding the Disease Pathogenesis and Drug Discovery.
Indication: Blood disease (Diamond Blackfan anemia; OMIM: 105650)
Clinical Trial: NCT01362595, NCT01464164, NCT03966053
Drug: l-leucine, Sotatercept, Trifluoperazine
Reported Observations:Treatment of rps19-knockdown zebrafish with l-leucine improved the anemia and developmental defects associated with DBA.
– We investigated the ability of the murine ortholog of sotatercept (RAP-011) to restore erythroid levels in zebrafish models of DBA.
– A chemical screen using zebrafish rps29-/- embryos identified several calmodulin (CaM) inhibitors that can rescue hemoglobin levels in mutant embryos.

Publication: PMID27213289
Title: NANS-mediated synthesis of sialic acid is required for brain and skeletal development.
Indication: Metabolic (Spondyloepimetaphyseal dysplasia; OMIM #605202)
Clinical Trial: NCT03545568
Drug: Sialic acid
Reported Observations: In zebrafish embryos, exogenously added sialic acid was able to partially rescue the developmental phenotype caused by NANS knockdown.