I really hope this is not another one of those false cures. It's time we made some real big-news progress against this horrible collection of ailments we call cancer.
For those who don't want to read through the lengthy human interest story wrapping the news here, from a quick read I gathered they're basically modifying T-Cells:
> In their natural state, T cells usually aren’t able to kill tumor cells, partly because they can’t latch on strongly enough. But June was fascinated by scientific papers showing it was possible to change this. A few researchers—first an Israeli named Zelig Eshhar in the ’80s, then other investigators around the world—had discovered that you could force a T cell to stick to a tumor cell and kill it. To pull this off, you built an “engineered T cell”—a T cell never before seen in nature. You altered the T cell’s genetic blueprint by injecting a new gene into the cell. The new gene would tell it to build a new molecular limb. The limb, called a “chimeric antigen receptor,” would sit partly inside the cell and partly outside, and it could send signals either in or out. One signal it could send was: kill. Another was: replicate.
> June loved this approach. So elegant. Put the immune system on steroids. What if you could train the body to fight cancer on its own? What if, instead of replacing a patient’s immune system (as in a bone-marrow transplant) or pumping him full of poison (chemo), you could just borrow some cells, tweak them, and infuse them back into the patient? In theory, the engineered cells would stay alive in the blood, replenishing themselves, killing any tumors that recurred. It occurred to June that one infusion could last a lifetime.
It seems this research has been going on for 10+ years. How many people have died because of the glacial regulatory pace of cancer research?
There's a book I can't recommend highly enough called "the emperor of maladies" and it's a history of cancer up to ~2008. It's pretty depressing in a lot of ways (both the disease itself and the history of the fight against it), but it's one of the most fascinating books I've read in years.
The kind of trade offs re: safety vs "getting things done" comes up quite a bit. The key optimism I pulled out of it was that cancer research is progressing exponentially, but this is harder to see because the general public only sees through the lens of "cured/not cured". But I think most cancer researchers would say "progress is being made, and faster than ever before with increasing velocity". Then again, the cure was around the corner in the 70s.
But yeah, cancer is bad news and a disease many of us will be forced to face in our lifetimes...
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.
You presume the new treatment will save more lives than the current one. Without adequate research and testing, this is an unsupported presumption. It's as likely to do more harm than good if the preliminary research isn't done.
We have the regulations we do because we caused harm in the past. They didn't spring fully formed from the minds of regulators, they happened because researchers killed and maimed people by not doing the necessary verification before human trials began.
any way you slice it people are going to die and make grave errors. but open transactions between researchers and free human beings would have drugs evolving much faster than unaccountable and corruptible overlords. reputation systems could replace much of their most important functionality.
> reputation systems could replace much of their most important functionality.
I'll start believing this once you show that a "reputation system" (don't these already exist?) actually does anything to stop quacks from peddling diluted water as a cure-all.
If you relied on reputation alone to regulate the medical market, feel-good snake oil treatments would prosper at the expense of legitimate medical advancement.
Do you honestly believe that people with a fatal illness, dealing with people offering a "cure" for that fatal illness, are free to make a rational choice?
You have cancer. You are going to die. Having a terminal illness changes the risk/reward beyond the bounds that most people can imagine. Even so, it seems that most people can make a reasonable decision, as long as the worst of the snake-oil peddlers are kept out. Here are your choices.
Do nothing. Make your time. You are going to die pretty soon. Try to make yourself and your family comfortable.
Take what treatment your insurer allows. You die more slowly, with a small chance of remission, but also with a small chance that the treatment will kill you sooner. Maybe your won't consume your family's assets.
Take what treatment you can afford. Die even more slowly (probably), with a small (but probably better) chance of remission, but also with a small chance that the treatment will kill you sooner. You may consume all your assets, and or incur huge debt/bankrupt your family.
Get yourself into an experimental trial. There is some chance that you will be in the control group (if one is used) in which case, you will die. There is some chance to suffer some adverse reaction in which case, you may be removed from the trial, back to step 1. There is some chance to suffer some catastrophic adverse reaction, in which case you will die, and if you are unlucky the death will be worse than death by cancer/chemo. There is some remote chance the treatment will work as good or marginally better than existing treatments, you win, congratulations. There is some very extremely remote chance the treatment will work amazingly (as in fairy-tale amazing) well, epic win. It may cost little or nothing, but who cares what it cost, you're alive and you just helped save the lives of many others.
Thanks, but I'd rather have medical research systems designed by scientists than libertarians. Free markets are not a panacea, and it's dangerous to think of them as such. In fact, the medical community used to be such a place, and it was dominated by quacks and snake oil salesmen.
you also have to consider that rushing through the trial stages can cause mistakes which leads to the treatment looking like it's ineffective. When this happens, it loses funding any may not then be developed further.
There are cases where early results from a trial were so promising that both sides (of a double-blind trial) were put on the treatment.
eg:
A new prostate cancer drug trial has been so successful doctors have decided to stop it early.
Medics at London's Royal Marsden Hospital were testing a powerful alpha radiation drug on 461 people while another group of the same number were being treated with a dummy drug.
Patients taking the new drug experienced less pain, side effects and lived longer.
Researchers were so astounded with the results they decided to stop the trial and started treating all 922 patients taking part in the study because they said it would be unethical not to.
Yes, but this has to be built into the experiment, or at least considered in the statistical analysis. You cannot just stop something when it's statistically significant.
You have to be extremely careful about this, otherwise you can end up with much worse significance than simple analysis would suggest (and then that kills people).
Yeah, "Stop when the results are positive" is one of the classic ways to fudge a study. Twelve patients (the number mentioned in the article) is way, way premature to end this early.
In this case, it's not even the regulatory environment. As the article mentions, the treatment is as fantastically expensive as it is fantastic. A lot of the skepticism isn't that it can't work, but that it might be too expensive to be viable.
Why has it taken 10 years? Because this stuff is really hard. Is there red tape in science? Sure. But even if all the red tape were removed, science would still move incrementally forward at a slow and steady pace.
Most areas of research are simply not mature enough to move forward at the exponential pace that computer technology leaped forward into our society. What is required? More hard work!
For those who don't want to read through the lengthy human interest story wrapping the news here, from a quick read I gathered they're basically modifying T-Cells:
> In their natural state, T cells usually aren’t able to kill tumor cells, partly because they can’t latch on strongly enough. But June was fascinated by scientific papers showing it was possible to change this. A few researchers—first an Israeli named Zelig Eshhar in the ’80s, then other investigators around the world—had discovered that you could force a T cell to stick to a tumor cell and kill it. To pull this off, you built an “engineered T cell”—a T cell never before seen in nature. You altered the T cell’s genetic blueprint by injecting a new gene into the cell. The new gene would tell it to build a new molecular limb. The limb, called a “chimeric antigen receptor,” would sit partly inside the cell and partly outside, and it could send signals either in or out. One signal it could send was: kill. Another was: replicate.
> June loved this approach. So elegant. Put the immune system on steroids. What if you could train the body to fight cancer on its own? What if, instead of replacing a patient’s immune system (as in a bone-marrow transplant) or pumping him full of poison (chemo), you could just borrow some cells, tweak them, and infuse them back into the patient? In theory, the engineered cells would stay alive in the blood, replenishing themselves, killing any tumors that recurred. It occurred to June that one infusion could last a lifetime.
It seems this research has been going on for 10+ years. How many people have died because of the glacial regulatory pace of cancer research?