What are the ethics of trying this in terminal patients? Surely I’d give it punt and hope for the best should I ever have brain cancer and if not we’d all learn something right? Why is it that we don’t treat terminally ill patients the same way we treat DOA car accident victims and aim to give them the opportunity to contribute?
Even before right to try laws, terminally ill patients were the preferred clinical trial subject. It's much easier to justify putting a risky chemical into a person who's about to die. Unfortunately, this biases the statistics you get, making it less clear whether the treatment would be helpful in less terminal popuations.
Because cancer patients, at the difference of car accident victims, are legally fully capable of consent. In addition, many people will try anything to live longer.
Some patients are genuinely willing to contribute as research subjects in the hope of helping others, and this is a good reason to participate. What many people embarking on studies for new treatments do not realize, is that studies are not designed to benefit them personally (random assignation to placebo group, etc.). It is a common perception that people embarking on a clinical trial hoping for a miraculous cure have the wrong attitude, as there is little chance that you will be cured, and it will be a collateral effect even if it does happen.
PS: the above is a widely accepted opinion in current medical ethics.
But why does it have to be research only ? Why not just make the treatment available, even if it's not proven yet ? If someone is terminal anyway, then what do they have to lose ?
In the past week, I have taken care of 2 patients with multimetastatic cancer. Their history is that basically, they suffered pretty much all complications you could think of from their treatment (surgery and chemo). The US healthcare system went to extreme lengths to ensure that those people are "not let down". Their situation now is that they are dependent on family, breathe through a tracheostomy, are blind, unable to move normally due to shortness of breath and neuropathy, and generally have a dismal quality of life. They still come to the clinic once in a while to have the most recent body part affected by metastasis removed. Yet, those people are not on clinical trials because it is felt (not by me mind, but not my call...) that there are still things to be done with presently known treatment methods.
All that to say, try to walk in a terminal patient's shoes for a bit, and perhaps you'll discover that the cost of increasing lifetime can be higher than you are willing to pay. Plus, we don't have the manufacturing and logistic capabilities to provide every possible experimental treatment to everyone.
Finally, you should realize that a lot of people have financial, academic, or even political interests to incentivize participation in clinical trials. It is precisely to avoid that, that patients must be informed that trials are not designed for one's personal benefit.
As has been pointed out, this does happen. But consider the ethical minefield. It would be very easy for an unethical or poorly governed organisation to fleece these patients. Willingness to try anything no matter the expense, plus slim chance of success, is a terrible equation.
Even with stringent controls in place, there are plenty of examples where med/research companies skirt extremely close to the limits of ethical or exploitative behaviour.
"Might as well try it though"? Well, how about horrible side effects, a life that's longer but more painful and increasingly less dignified? Your last few months in a hospital far from family, vs your last weeks at home?
These are not straightforward issues.
Finally, consider that some terminally ill people simply don't want any more treatment. They're done with it all, and that's fine. This is a limiting factor in recruitment for trials for such situations.
I'm not denying your suggestion -- although these are conscious people with volition, which perhaps doesn't come across in your question. I'm just pointing out that ethics are very, very hard.
>Why is it that we don’t treat terminally ill patients the same way we treat DOA car accident victims and aim to give them the opportunity to contribute?
Cancer patients aren't unconscious and braindead. I agree with what you're saying but this is the argument used by medical boards.
And sometimes you get extremely lucky and a Phase 1 trial of a modified poliovirus injected into your brain tumor---a trial only designed to determine safety and toxicity---kicks ass and saves your life.
1) The control arm of the trial is seldom a placebo, it's more likely to be the current standard of care (i.e. best possible already FDA approved treatment)
2) Not all clinical trials have a control arm. It depends on the phase of the trial among other things.
What about errors or changes in your diagnosis? John Bain (game review youtuber) was diagnosed with Cancer and in 2015 his cancer was determined to be uncurable. But, his diagnosis changed and now he is in remission again. See
https://en.wikipedia.org/wiki/TotalBiscuit
I would think that part of an ethical protocol for testing would include a frank discussion with each patient about the probabilities of various outcomes and trading off actions in that landscape.
This isn't entirely true (not is Wikipedia accurate in its story of his illness).
TB is participating on clinical trials for effectiveness of Panitumumab in stage 4 colon cancer patients, its this drug that produced remission in his illness.
Thanks for the source. I am always happy to see breakthroughs on bio medical researches. I think the world has been over focused on web development and neglecting the researches that could make real impacts.
I've got a friend with stage four glioblastoma. I'd really love to be able to sign him up for one of these trials. Unfortunately, the article says human trials are 18 months away, and I doubt he will live that long.
If your friend is in the United States it's worth reaching out to the following places to see what options might be available for clinical trials or treatment options. If these places are out of reach, at the very least, it's worth looking for a nationally designated comprehensive cancer center with a specialized brain tumor program where they'll have access to the newest treatment options.
I'd love for somebody to explain how you get a group of brain tumor-bearing mice. Can one somehow prompt glioblastoma to occur in some meaningful percentage of mice? I scanned the paper but found nothing.
Want skin cancer? Rub a known causing carcinogen on a patch of skin daily, want brain cancer? Probably injections or controlled exposure to radiation.
There is also a process of transplanting cancer cells (including from humans) into hosts to grow tumors.
Lastly these days there is also mutagenesis (usually gene knockout) in embryos and selective breeding and engineering for predisposition to cancer both general and of specific types.
Overall it's likely a mixture of several methods depending on the exact requirements; breeding mice which are predisposed to cancer by knocking out genes that are known to suppress cancer or help the immune system effectively battle it and then cellular transplant or carcinogenic exposure to cause tumors in specific locations or specific types of cancer.
Yes, mice are bread and engineered with suppressed immune systems and other traits.
There are specific breeds of mice for specific experiments while they aren't technically clones their genetic profile is very specific and their genetic diversity is very reduced.
When you buy mice for research you get a very specific genetic profile many labs today will customize it also for your needs lab mice and rats are not pet store animals.
This goes beyond simply knocking out genes for example transgenic mice and other experimental animals are engineered with human genes that can for example add cellular recptors that do not occur in those animals naturally to test anti virals, vaccines and other drugs.
Not a stupid question. Nude mice are one way of doing this, though without an immune system to interact with a tumor, results need confirmation in other model types.
Yep, we have all sorts of crazy, messed up mice that are used for biomedical research. Humanized mice[1] typically have genes knocked out that code for some essential aspect of the immune system. With important genes being made non-functional, the mice end up being immunodeficient.
