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Comment by tripletao

> This means the drug has gone through the actual drug development lifecycle- Terrence is not talking about this.

I don't understand what you mean by this. Nobody is conducting phase 3 clinical trials of a new drug except as part of that regulatory drug development lifecycle, to gain legal permission to market the drug. The idea that it could be "unclear who conducted" a >$10M trial closely supervised by multiple government agencies does not make sense.

> No pharmaceutical is testing drugs today which have no scientific theory or reasoning or seminal papers behind it.

Even today, new drugs are routinely identified by screening with assays that give no mechanistic insight, as e.g. for ganaplacide. They found that drug by screening directly for whether the compound killed Plasmodium (the genus of parasites that cause malaria), by applying it to infected cultured liver cells[1]. That was probably a lot more expensive than something like a protein binding assay against a prior known target, but probably more fundamentally accurate. That also creates potential to discover an entirely new mechanism of action, without potential for cross-resistance with drugs developed against prior known targets, and that apparently happened here.

The first studies in humans were conducted around 2014[2], at which time I believe no mechanism had yet been proposed. Some progress towards a mechanism was proposed in 2020[3] and perhaps it's correct; but if it's later shown to be incorrect then nobody will care, and that in no way drove the discovery or initial human testing of the compound.

1. https://pmc.ncbi.nlm.nih.gov/articles/PMC3473092/

2. https://pmc.ncbi.nlm.nih.gov/articles/PMC4249437/

3. https://pubmed.ncbi.nlm.nih.gov/32286267/

parent tripletao 16 days ago

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