So if I summarize correctly they re-evaluated already “FDA passed” but abandoned substances for anti-cancer workings using new techniques (CRISPR) and got 50 hits. That’s amazing! Would this work in other fields as well?
That's a good summary but it's missing the context that these kinds of "hits" will most likely fail clinical trials. Many drugs look good at this stage in preclinical development and almost none of them show both safety (ph1 trial) and efficacy (ph3 trial).
There aren't actually that many low hanging therapeutic fruit out there, mostly we have to actually do the work to discover new mechanisms and make new compounds to target them.
The genes essential for cancer cells to grow in vitro could easily be quite different from those required to establish the tumor micro-environment, invade new tissues and fight off the immune system. They'll have many false negatives from the mismatch in context. Simultaneously, they might have false positives from e.g. differences in layout and density of tumor cells (2D/uniform vs. 3D heterogeneous tumor).
And even if they get the right targets, finding drugs with an effective therapeutic index is hard.
This might be a useful screening technique...but it's not the first high throughput screening method. They might have some successes, only trials will tell.
There’s a specific regulatory pathway for this process, section 505(b)(2). It allows you to use the safety data for the approved treatments which can save you a lot of time and money (I have used this route for a drug myself T one of my companies).
You still have to prove that your formulation is safe,of course, and prove efficacy for the situation you intend to market it for.
Typically years. We had a program for a drug that caused bladder cancer in rats. The original mfr had spent a couple of years figuring out that it was specific to rats.
I think the current stats are that the average drug time to approval is 14 years.
