6 ms·

No. Pharmaceutical research does not and cannot work this way. Over the last few years, there has been much reasearch on molecular interactions. The current con

by patall 11y ago

No. Pharmaceutical research does not and cannot work this way. Over the last few years, there has been much reasearch on molecular interactions. The current consensus is that everthing interacts with pretty much everthing at some point, it is just the degree of these interaction that varies. Molecular pathways become more and more vague (which does not mean that they disappear, its just very complex). For example, we do not even know how many existing drugs interact with each other, because who would fund that? Some reasearch hospital now do it but it is tedious when you have hundreds of potential interacting partners. For clinical trials, this leads to very strict guidelines. One of my university professors once told us about a drug they had to test in dogs for some side effects. Nothing happend to the dogs but afterwards they would have been eutanized because it was regulated that these exact dogs cannot enter another study. They finally adopted them, but still. Similar, people entering clinical trials are usually not allowed to take certain drugs, may need to abstinent from alcohol etc. Keep in mind. This is not an experiment, this is a clinical trial in a human.

Dylan16807 11y ago
Giving a drug to one person to see what happens is definitely an experiment.
gumby 11y ago
> Keep in mind. This is not an experiment, this is a clinical trial in a human. (Note: I have one drug in clinical trials, for which I designed the phase I and phase II protocols, and have appeared before the FDA in support of clinical trials of new medications.) Actually this is not a trial, it is considered an experiment. Trials are controlled (not always blinded or tasted against placebo, as many people think). Strictly speaking you cannot give an unlicensed medication without getting in trouble -- in fact permission to do a trial is actually agreement by the FDA that they won't prosecute you for breaking this law, basically because you followed one of the procedures spelled out in the law and the FDA agreed with your proposal. You are completely right on interactions. And yes, the animals are pretty much always sac'ed at the end of the experiment, even those in placebo arms :-( There are a few quasi-informal exceptions to the no unapproved administration. One is if you develop the drug yourself -- hard to prosecute. In a modern corporation however you can't really do that -- if you're a research scientist developing a drug, lawyers could claim that you were forced into it by your boss (another example is that the FDA requires that QC be an independent function). So in practice the only person who can try such a drug is the CEO. In my case I was both the CEO as well as one of the developers so was confident that the in vivo data we had showed that I would be fine. As a result I was a pincushion (these were true experiments in PK, diffusion etc -- I didn't have the indication we were treating for). The drug hasn't yet gone for approval, but if it does, somewhere in the approval application there will be a description under "previous human experience". You can do other experiments and trials someplace not subject to the FDA (i.e. other countries). Unless they are also formally part of your FDA process you can't use them in support of approval -- but if you paid for them you have to include them as previous human experience as well. And there are also various "compassionate use" cases though they are also somewhat-formalized now because some people try to stretch them into trial territory. By definition they are not adequately powered. There are amusing examples of previous human experience. My favorite is someone who shall go nameless who argued in support of an erectile disfunction treatment in front of his board by dropping his trousers and injecting his penis in front of them. I have only heard of this second hand but I bet there was quite a reaction! NB for anyone else in pharma: yes I glossed a few terms like calling the NDA an "approval application" in the interest of clarity for those not in the industry.
- aab0 11y ago
  > My favorite is someone who shall go nameless who argued in support of an erectile disfunction treatment in front of his board by dropping his trousers and injecting his penis in front of them. I have only heard of this second hand but I bet there was quite a reaction! That was Brindley's (in)famous lecture on papaverine: "How (not) to communicate new scientific information: a memoir of the famous Brindley lecture" http://onlinelibrary.wiley.com/doi/10.1111/j.1464-410X.2005.05797.x/full http://onlinelibrary.wiley.com/doi/10.1111/j.1464-410X.2005.... The reaction was just as you suppose and everyone here might enjoy reading Klotz's recollection of witnessing it.
  - YeGoblynQueenne 11y ago
    Please submit that, it's delightful :)
    - aab0 11y ago
      It's been submitted before: https://news.ycombinator.com/item?id=3478971 https://news.ycombinator.com/item?id=3478971
darawk 11y ago
You're completely missing the point. Those two things are not comparable. Yes, it's not a controlled study. But it still has enormous value. If I tell you I invented a cure for cancer, you should be highly skeptical of me. But if I tell you I have a cure for cancer, prove to you that I have cancer, then cure my own cancer - you shouldn't give that treatment FDA approval, but you should sure as hell start the clinical trials process. Whereas if I do not treat myself or anyone else, you should just write me off as a crank. That is the difference between the world before and after this self-experiment. It upgrades the probability in everyone's mind that this is a real thing and it can really work. It doesn't mean we should all go blindly get the treatment done, but it does mean we should take this research much more seriously. That is its value.
- semi-extrinsic 11y ago
  Except in this case, the proof that you've cured cancer (and that you had cancer in the first place) comes from a non-reputable lab (SpectraCell), doing a telomere length test which has not been documented in the literature. It's even on Quackwatch's list of labs doing "Dubious diagnostics tests".

by patall 11y ago

Dylan16807 11y ago

Giving a drug to one person to see what happens is definitely an experiment.

gumby 11y ago

> Keep in mind. This is not an experiment, this is a clinical trial in a human. (Note: I have one drug in clinical trials, for which I designed the phase I and phase II protocols, and have appeared before the FDA in support of clinical trials of new medications.) Actually this is not a trial, it is considered an experiment. Trials are controlled (not always blinded or tasted against placebo, as many people think). Strictly speaking you cannot give an unlicensed medication without getting in trouble -- in fact permission to do a trial is actually agreement by the FDA that they won't prosecute you for breaking this law, basically because you followed one of the procedures spelled out in the law and the FDA agreed with your proposal. You are completely right on interactions. And yes, the animals are pretty much always sac'ed at the end of the experiment, even those in placebo arms :-( There are a few quasi-informal exceptions to the no unapproved administration. One is if you develop the drug yourself -- hard to prosecute. In a modern corporation however you can't really do that -- if you're a research scientist developing a drug, lawyers could claim that you were forced into it by your boss (another example is that the FDA requires that QC be an independent function). So in practice the only person who can try such a drug is the CEO. In my case I was both the CEO as well as one of the developers so was confident that the in vivo data we had showed that I would be fine. As a result I was a pincushion (these were true experiments in PK, diffusion etc -- I didn't have the indication we were treating for). The drug hasn't yet gone for approval, but if it does, somewhere in the approval application there will be a description under "previous human experience". You can do other experiments and trials someplace not subject to the FDA (i.e. other countries). Unless they are also formally part of your FDA process you can't use them in support of approval -- but if you paid for them you have to include them as previous human experience as well. And there are also various "compassionate use" cases though they are also somewhat-formalized now because some people try to stretch them into trial territory. By definition they are not adequately powered. There are amusing examples of previous human experience. My favorite is someone who shall go nameless who argued in support of an erectile disfunction treatment in front of his board by dropping his trousers and injecting his penis in front of them. I have only heard of this second hand but I bet there was quite a reaction! NB for anyone else in pharma: yes I glossed a few terms like calling the NDA an "approval application" in the interest of clarity for those not in the industry.

aab0 11y ago
> My favorite is someone who shall go nameless who argued in support of an erectile disfunction treatment in front of his board by dropping his trousers and injecting his penis in front of them. I have only heard of this second hand but I bet there was quite a reaction! That was Brindley's (in)famous lecture on papaverine: "How (not) to communicate new scientific information: a memoir of the famous Brindley lecture" http://onlinelibrary.wiley.com/doi/10.1111/j.1464-410X.2005.05797.x/full http://onlinelibrary.wiley.com/doi/10.1111/j.1464-410X.2005.... The reaction was just as you suppose and everyone here might enjoy reading Klotz's recollection of witnessing it.
- YeGoblynQueenne 11y ago
  Please submit that, it's delightful :)
  - aab0 11y ago
    It's been submitted before: https://news.ycombinator.com/item?id=3478971 https://news.ycombinator.com/item?id=3478971

darawk 11y ago

You're completely missing the point. Those two things are not comparable. Yes, it's not a controlled study. But it still has enormous value. If I tell you I invented a cure for cancer, you should be highly skeptical of me. But if I tell you I have a cure for cancer, prove to you that I have cancer, then cure my own cancer - you shouldn't give that treatment FDA approval, but you should sure as hell start the clinical trials process. Whereas if I do not treat myself or anyone else, you should just write me off as a crank. That is the difference between the world before and after this self-experiment. It upgrades the probability in everyone's mind that this is a real thing and it can really work. It doesn't mean we should all go blindly get the treatment done, but it does mean we should take this research much more seriously. That is its value.

semi-extrinsic 11y ago
Except in this case, the proof that you've cured cancer (and that you had cancer in the first place) comes from a non-reputable lab (SpectraCell), doing a telomere length test which has not been documented in the literature. It's even on Quackwatch's list of labs doing "Dubious diagnostics tests".