Admittedly it seems like a bit of a gamble to just start popping drugs whether they've been studied or not, but people spend lots of money on "natural" supplements which are at least usually reasonably safe but are even less likely to work.
There's not much worse than having your mind die while your body is still alive.
Just to pick another unrelated chemical example that is well known, 30ml of methanol will kill you but 10ml will make you permanently blind.
How is this relevant?
People tend to vary wildly in choices and opinions. Who knew!?
But some people think their choice or opinion is somehow the "correct" one and should be imposed on everyone ;-)
I know there's very good reason to be wary of mouse models, but in cases (as this appears to be) where the physiological mechanism of disease is both (a) understood and (b) the same from mouse to human, a little less skepticism may be warranted.
We should be far more concerned about side-effects of a drug intended to treat acne or erectile dysfunction than for drugs targeting life destroyers such as cancer and Alzheimer's.
At some threshold of disease viciousness, society (and the FDA) should relax substantially on the risk-aversion, because the patient is already facing the worst-case scenario.
It's also now possible to do clinically-useful testing of targeted treatments using genetically-edited control tissue cultures against unedited tissue cultures.
The point is that it's better to test something that is as close to the patient as possible, not guess with other species or even other people whom express genes differently.
FYI: making a new mouse models requires lots of chopping off heads of mice whom don't possess the desired gene. While transgenic, highly-edited living models might be nice, there's probably a more humane/simpler/reproducible way to do the exact same thing.
No they aren't. Transgenic mouse models are a $billion industry. We do not understand biological mechanisms nearly enough to not use live animal models. We are not even able to recreate single cell organisms with our level of knowledge and engineering.
>>FYI: making a new mouse models requires lots of chopping off heads of mice whom don't possess the desired gene. While transgenic, highly-edited living models might be nice, there's probably a more humane and simpler way to do the exact same thing.
Ah, the real root of your argument. Do you even know what the word humane means? I work in a labroatory and the animals are treated better than the people. Yes, a lot of mice are killed.. but for a reason. You say there's probably a simpler way to do the exact same thing, but there simply isn't. I wish more people were actually educated in this matter, but emotional responses tend to get more results.
That said, regardless of whether or not they're proven/approved/tested, drugs are never magic bullets. The ones used to treat the most serious chronic diseases always come with serious risks and profound side-effects. I don't expect that to change anytime soon.
In the future, medical clinicians won't have to guess as much because adult stem cells can now be created from red blood cells, allowing personalized medication with a petri model of actual patient tissue, to screen problems like interactions and allergies before administration.
"Statins are beneficial, but some have questioned whether their benefits are due to their ability to lower cholesterol or to their anti-inflammatory effects, or both. There are two competing hypotheses, the LDL hypothesis and the statin hypothesis. A new study in The New England Journal of Medicine sheds some light on that controversy and tips the balance in favor of the LDL hypothesis."
https://www.sciencebasedmedicine.org/update-on-cholesterol-a...
For example, Ben Goldacre. He is cynical about 'Big Pharma', and has written about it in one of his books[0]. At the same time he appears to support efforts to open up private medical research -- he recently cofounded AllTrials[2] along with the BMJ, Cochrane, PLOS, and a few other high-profile reputable sources[3].
[0]: https://en.wikipedia.org/wiki/Bad_Pharma [1]: http://www.cochrane.org/ [2]: http://www.alltrials.net/ [3]: http://www.phc.ox.ac.uk/news/ben-goldacre-joins-oxford-unive...
