TABLE 3.
Drugs identified for ALS treatment in zebrafish models.
| Compound | Classification | Model | Effects on phenotype | Behavioral method | Molecular mechanism | References |
|---|---|---|---|---|---|---|
| FPL64176 and Bay K8644 | L-type calcium channel agonists | TARDBP-G348C-Tg | Rescue impaired locomotor function | Touch-evoked escape response, 52–56 hpf | Rescue motoneuron excitability, decrease synaptic fidelity, NMJ abnormal structures | Armstrong and Drapeau, (2013) |
| Pimozide | T-type Ca2+ channels antagonist | TARDBP-G348C-Tg; SOD1-G93A-Tg; FUS-R521H-Tg | Improve motor defects, stabilize neuromuscular transmission | Touch-evoked escape response, 52–56 hpf | Not applicable | Patten et al. (2017) |
| TRVA242 | Pimozide derivatives | TARDBP-G348C-Tg; SOD1- G93A-Tg; C9ORF72 | Restores aberrant spinal motor neuron outgrowth; recovers NMJ structures and synaptic deficits | Locomotion test, 5 dpf | Not applicable | Bose et al. (2019) |
| Riluzole | Sodium current INaP inhibitor | SOD1-G93R-Tg | Reverse abnormal movements | Touch-evoked escape response, 96 hpf; locomotion test, 12 months | Reduce neuronal stress | Benedetti et al. (2016) |
| Methylene blue | Cationic thiazine dye | TARDBP-G348C-Tg and FUS-R521H-Tg | Protect impaired motor phenotypes | Touch-evoked escape response, 48 hpf | Inhibit TDP-43 aggregates, suppress oxidative stress | Vaccaro et al. (2012) |
| 1-Fe | Corrole iron complex | SOD1-G93R-Tg | Improve motor defects | Light-dark test, 6 dpf | Anti-antioxidant | Soll et al. (2021) |
| Ciprofloxacin and celecoxib | Antibiotics and anti-inflammatory | SOD1-G93R-Tg; TARDBP-G348C-Tg | Rescue motor defects and axonopathy, recover NMJ structures and ramified morphology of microglia | Touch-evoked escape response, 54–56 hpf; light-dark test, 6 dpf | Anti-inflammatory | Goldshtein et al. (2020) |
| Terbutaline sulfate | β2-adrenergic receptor agonist | TARDBP-Q331K-Tg | Prevent axonal defects and NMJ degeneration | Not applicable | Activate β2-adrenergic receptors | Paik et al. (2015) |
| PFI-1 | Bromodomain inhibitor | PR20 | Improve embryos viability | Not applicable | Not applicable | Corman et al. (2019) |
| Na-Phen | Histone deacetylase inhibitor | PR20 | Improve embryos viability | Not applicable | Not applicable | Corman et al. (2019) |
| Telbivudine | Uracil-like nucleoside compound | SOD1-W32S-Tg | Rescue axonopathy and motor neuron deficits | Touch-evoked escape response, 48 hpf | Interact with W32 residue | DuVal et al. (2019) |