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I run a foundation; I just realized that tonight.

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Th1onein Donating Member (1000+ posts) Send PM | Profile | Ignore Mon Nov-20-06 11:52 PM
Original message
I run a foundation; I just realized that tonight.
I do it part time, aside from my regular "job."

It's a research foundation. For research into cystic fibrosis. It's small; only about 300 people strong, but lots of those people are very smart, and very motivated. They get done what needs to be done.

I do the biochemistry research. In my spare time. After eleven years, I've come to a "stopping place." Although, of course, you never stop wanting to learn about something that you're interested in. But, now, I've found a compound that might be able to stop the disease. So, now, of course, I have to go through all of the bureacracy that the FDA requires. We can't do it, in our homes, ourselves, so we contracted it out to an independent lab, which required $30,000, which our foundation raised and paid for.

It is not a big trial, because the bigger the trial, the more the money. We can only afford five or six subjects. An extremely small trial by any standards. When you have such a small sample size, your results mean very little. And, if you don't screen your sample size very well, for confounding variables, your results mean nothing. That costs more money.

Now, there are two avenues open to us--

1. We have the patent for the use of this compound in cystic fibrosis. We can tell people how to get the compound, independently, with their doctor's help, and then take statistics from that. This is legal, although the logistics of getting statistics is formidable. But we do have someone who will gather this for us, if necessary.

2. Or we can raise more money, to do the trial, with an independent firm.

The first one gives us freedom, but also much more scrutiny, in terms of credibility for our scientific model. If you don't have those initials behind your name in this world, you are not given much credence. Still, if you make enough of a wave of this kind, people are going to pay attention, credibility or not.

The second gives us freedom, but it will put our trials off, which everyone is waiting for, and of course, it costs more, so we have to raise more money.

Tell me what you think, please?
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Redstone Donating Member (1000+ posts) Send PM | Profile | Ignore Mon Nov-20-06 11:56 PM
Response to Original message
1. Christ, I don't know. My first inclination is to say "do what will get it to the most
people who will benefit from it the soonest," but I don't know the ins and outs of your choices.

I'm sure you'll make a good decision.

Redstone
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NNadir Donating Member (1000+ posts) Send PM | Profile | Ignore Tue Nov-21-06 12:25 AM
Response to Original message
2. I really think you need to do the clinical trials.
Clinical trials exist as they do for a reason.

Lots of people "feel" as if their treatments work, but they cannot be sure that they do unless they are proved. This of course, requires a double bind study. Without such a study, the results are speculative.
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Th1onein Donating Member (1000+ posts) Send PM | Profile | Ignore Tue Nov-21-06 01:52 AM
Response to Reply #2
3. I think you're right.
Most people, when they take a new drug, worry that it will kill them. That it will be toxic. I think that it is important to have this trial, even though the population in it is small, in order to let people know that this drug won't kill them. In reality, it is in such small doses, that it could hardly kill anyone.

It is specific enough to change their basic biochemistry, though. Enough to, hopefully, change the course of their disease.

It will work for some, most probably, and it might work for most, less probably (depending on their genetics for certain other modifying protein and enzymes), but it will work to such a degree and at low enough dosages so that most people are not afraid to try it. And it should work, when it works, quite dramatically.

Thanks for your input.
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