Question: how would you spot a sing that your cure is working?

Blanka Sengerova answered on 4 Jul 2012:

Good question.

I work on a tiny part of a cellular mechanism that *might* be involved in cancer formation. So if I have found that the protein I work on is involved in DNA repair and then stopping it working might damage cancer cells, I can then test it.

First I can find compounds that might stop it from working called inhibitors and test its activity in presence of these compounds in a test tube (where it is on its own). If that works, I can then use human cells grown in culture (in a plate in an incubator) to see if the compound can go into a human cell and do its inhibitory activity in a real-life environment. If that works, the drug can be tested in animal models and later in human patients.

I only work at the first level (testing things in isolation in a tube) so realistically, I won’t know if the compound has become useful in cancer therapy for another few years yet. Does this make sense?