The vast majority of new treatments fail in Phase 2 - that is, in showing effectiveness in a human model. A huge chunk of the remainder fail in phase 3.
People whine about how slow the pace of research is, but that's not because clinical trials are so slow - it's because most drugs fail. The vast majority of drugs that work in animal models get bounced in Phase 1 (when they show unacceptable toxicity in humans so great it can't be dosed) or Phase 2 (where it just shows no effectiveness in humans at all - not even some level of effectiveness that fails in cost/benefit analysis). this is the problem with people complaining "But every delay kills people!"
No. You don't know going in whether it's going to work - that's why you're doing a trial. And from a naive position, the probability is pretty clear that it's very unlikely it's going to work. There's absolutely no reason to think any particular drug is worthwhile, and every to worry about its side-effects. With a sample size of 12, this one may yet became a last-ditch drug with toxicities worse than chem/rad. We don't know yet. Using this is an example of over-cautiousness is ridiculous.
Additionally, the slowest part here wasn't the clinical science - it was the basic science. Gene therapy has been a failure for decades, and we've been repeatedly going at it to try and get it to work. It's not as though this is the first clinical trial for a gene therapy: it's just one of the first to be worth anything.
>Gene therapy has been a failure for decades, and we've been repeatedly going at it to try and get it to work. It's not as though this is the first clinical trial for a gene therapy: it's just one of the first to be worth anything.
Glybera is a gene therapy which has been approved for medical use.
Your post is misleading. You're not providing counter-evidence for the 'failure for decades' claim, and are in fact confirming the 'one of the first' part.
Yes, it has been approved, but only very recently, in late 2012. It is also the first such drug that has been approved (and to date, the only one), and this happened under 'exceptional circumstances'. The usual standard of supporting evidence was forgone because the disease it treats is so rare. The company is explicitly required to supply further supporting evidence, and the rules for its use are very strict.[1]
And even the Cancer drugs that "work" and are registered only improve stuff like PFS and OS (overall survival) a couple of months in most cases. Cancer treatments tend to work for some time until the illness finds a new pathway rendering the treatment ineffective. It's frankly pretty depressing to look at the survival curves with and without a new drug. You don't save many people anyway.
You really can't make such statements about "cancer". Stage II lung cancer? You will probably die in a few months, surviving five years isn't very likely. Stage 4b Hodgkin's lymphoma at the age of 18? You have a decent chance of getting to know your grand children.
People whine about how slow the pace of research is, but that's not because clinical trials are so slow - it's because most drugs fail. The vast majority of drugs that work in animal models get bounced in Phase 1 (when they show unacceptable toxicity in humans so great it can't be dosed) or Phase 2 (where it just shows no effectiveness in humans at all - not even some level of effectiveness that fails in cost/benefit analysis). this is the problem with people complaining "But every delay kills people!"
No. You don't know going in whether it's going to work - that's why you're doing a trial. And from a naive position, the probability is pretty clear that it's very unlikely it's going to work. There's absolutely no reason to think any particular drug is worthwhile, and every to worry about its side-effects. With a sample size of 12, this one may yet became a last-ditch drug with toxicities worse than chem/rad. We don't know yet. Using this is an example of over-cautiousness is ridiculous.
Additionally, the slowest part here wasn't the clinical science - it was the basic science. Gene therapy has been a failure for decades, and we've been repeatedly going at it to try and get it to work. It's not as though this is the first clinical trial for a gene therapy: it's just one of the first to be worth anything.