I am not sure why this couldn't go to phase 1 trials right now? I know they don't know why it works but we don't know why a lot of drugs work.
One of the corollaries to not knowing why something works is also not knowing how it can go wrong. Until they know that it's probably not a good idea to risk human trials.
True, but the animal studies should have shed some light into that. They used two different animal models and one cell line? They might not have done any extensive dosing tests, or looked for adverse effects in the animal models. I would assume that they would have done a least some of that since the student would be following the animals status. It depends on how long they think it will take to develop a model. I would expect this would be 3-6 years research in my experience to build a decent model of what is going on. That also assumes that they maintain a sufficient funding level which is never a known factor.
So they have a promising start which invites further research. They'll need investment money, but until they finish their due diligence human trials are contra-indicated.
I guess what I was trying to state, was fully knowing the mechanism isn't required for starting human trials. Knowing potential side effects, dosage, and treatment timelines are required. We often come out with drugs that we don't exactly know why/how they work, but we know they do and have a decent understanding of the risks. I watched many possible treatments die at this stage of research. Funding gets pulled, the researcher simply doesn't want to take it to the next stage, or many other reasons that is never gets moved towards phase 1 trials. Universities and the NIH have set up methods to try and combat this and help things move forward, but I doubt that the particular research on focusing in on the mechanism for treatment will really advance any of the key questions for moving to phase 1 (dosage, safety, and treatment plan). In my experience the basic biology research is critical for understanding what is going on but is not targeted to really move treatments forward.
Ah, then I agree with your point. Science doesn't work in terms of absolutes when it comes to knowledge, it only offers degrees of certainty. Until we know with a reasonable degree of certainty that the proposed treatment won't cause even worse side effects it's best to hold off on human trials.
One of the corollaries to not knowing why something works is also not knowing how it can go wrong. Until they know that it's probably not a good idea to risk human trials.
True, but the animal studies should have shed some light into that. They used two different animal models and one cell line? They might not have done any extensive dosing tests, or looked for adverse effects in the animal models. I would assume that they would have done a least some of that since the student would be following the animals status. It depends on how long they think it will take to develop a model. I would expect this would be 3-6 years research in my experience to build a decent model of what is going on. That also assumes that they maintain a sufficient funding level which is never a known factor.
So they have a promising start which invites further research. They'll need investment money, but until they finish their due diligence human trials are contra-indicated.
I guess what I was trying to state, was fully knowing the mechanism isn't required for starting human trials. Knowing potential side effects, dosage, and treatment timelines are required. We often come out with drugs that we don't exactly know why/how they work, but we know they do and have a decent understanding of the risks. I watched many possible treatments die at this stage of research. Funding gets pulled, the researcher simply doesn't want to take it to the next stage, or many other reasons that is never gets moved towards phase 1 trials. Universities and the NIH have set up methods to try and combat this and help things move forward, but I doubt that the particular research on focusing in on the mechanism for treatment will really advance any of the key questions for moving to phase 1 (dosage, safety, and treatment plan). In my experience the basic biology research is critical for understanding what is going on but is not targeted to really move treatments forward.
Ah, then I agree with your point. Science doesn't work in terms of absolutes when it comes to knowledge, it only offers degrees of certainty. Until we know with a reasonable degree of certainty that the proposed treatment won't cause even worse side effects it's best to hold off on human trials.