List of diseases with a known mechanism but no cure/treatment

[poetinsf]

And what about the heterogenous nature of the patients - don’t we need to assess the risks on a case by case basis?

The heterogenous nature is why I suspect some drugs may work for some people even if it doesn't for the majority. Most drugs probably won't do anything for most of the patients. But you are right, the effect could go either way and you wouldn't know which way ahead of time. At this point however, certain safe drugs making ME/CFS permanently worse is as much (case-by-case) anecdotes as making it better. So it should be left as a matter of belief for each patient to evaluate and decide.

There is a certain amount of risk in being overly risk-averse too, at least for the moderate to mild end of the spectrum. If you stick to strictly safe routine, for example, you are not going to discover anything about the personality of your ME/CFS. Some of the discoveries could allow you to better manage your ME/CFS or even improve. What are the chances of that? And What are the chances of making your ME/CFS permanently worse in the process? Each patient will have to decide on their own. I just think it would be nicer to make it easier for those willing to experiment. Who knows, new insights or solution may even emerge through these experiments.

 

[poetinsf]

I don't know how the science comes into it, but for the record I've never thought it's coming to rescue me.

Even if I did, it's a struggle not to take offence that anyone would think for a second I'd be so selfish that it would affect my position on the social and medical ethics of this. I understand no offence is meant, and none's taken of course; I just need to point out how morally compromising that suggestion appears.

I don't quite understand. If you have an intractable disease, you can either wait for the science to find the solution or do something on your own. Why shouldn't your belief effect your choice? It's just a matter of evaluating the odds according to your belief; there is no social/medical ethics there as far as I can see.

 

[Utsikt]

I don't quite understand. If you have an intractable disease, you can either wait for the science to find the solution or do something on your own. Why shouldn't your belief effect your choice? It's just a matter of evaluating the odds according to your belief; there is no social/medical ethics there as far as I can see.

There is if you’re asking someone to help you get the medications.

It’s a bit like the assisted suicide debate in that specific regard.

 

[Trish]

I don't quite understand. If you have an intractable disease, you can either wait for the science to find the solution or do something on your own. Why shouldn't your belief effect your choice? It's just a matter of evaluating the odds according to your belief; there is no social/medical ethics there as far as I can see.

This disregards the prescribing doctor's professional ethics and insurance. A responsible doctor is surely unlikely to risk their career and reputation by prescribing unevidenced drugs for a patient to try without being provided with some evidence to support that experiment.

 

[Kitty]

Why shouldn't your belief effect your choice? It's just a matter of evaluating the odds according to your belief; there is no social/medical ethics there as far as I can see.

Because it's obviously not about me. It's a much bigger ethical issue, and I'm not a special case who can be exempted from it.

As soon as doctors start experimenting on patients—and they would be doing that, prescribing drugs makes them active participants—the whole ethical paradigm begins to break down. Thin end of a wedge and all that.

 

[Chestnut tree]

But what about the ethical issue of abandoning, and refusal of funding biomedical research into the disease of very ill people? Even worse forcing people to undergo treatments that make them worse?

Isn’t very cruel to expect the very ill patients to wait for biomarker research, trials when the funding that is needed has not come in the last 30 years and will not likely come in the next few years?

To expect people not to try anything, is almost just as unethical.

The issue is not as black and white, in regard to medications being unsafe
- if there are medications that are safe for human use
- if these medications are of low toxicity and few adverse effects
- if these medications have been tested as safe for similar complaints

It would not be like shooting in the dark, because testing has been there.

Than these medications should be available for the individual patient who wants to, to carefully try.

 

[Kitty]

But what about the ethical issue of abandoning, and refusal of funding biomedical research into the disease of very ill people? Even worse forcing people to undergo treatments that make them worse?

Yes, I do think it's unethical. But two wrongs don't make a right.

Isn’t very cruel to expect the very ill patients to wait for biomarker research, trials when the funding that is needed has not come in the last 30 years and will not likely come in the next few years?

Of course, but the same applies to many causes of disease and disability. In some cases it probably won't be a wait of a few years to get solutions, it could be a few lifetimes.

I've lived with ME/CFS for 49 years without any help, and a relative lived with it for 73 years. The fact we were unlucky enough live in a time before there was a treatment—just as her brother was unlucky enough to live in a time when there was no treatment for Type 1 diabetes, so he died in pain at the age of four—feels cruel, but it's how it goes.

I struggle to think about it without considering the wider context, and that is that we won't improve science by allowing doctors to experiment on people outside of clinical trials. We'll just erode the ethical framework further by allowing the existing wild west situation to gain momentum, and reward them for ever-diminishing standards. Our children and grandchildren won't thank us for that legacy, and they certainly won't benefit from it.

I understand your perspective and I know how much people need progress to be made on treating ME/CFS, but at the same time I can't un-understand that it's not only about us. I hope that makes some kind of sense, anyway.

 

[poetinsf]

It’s a bit like the assisted suicide debate in that specific regard.

Is the off-level use of safe drugs really comparable to assisted suicide? Or is it just your belief? If it is your belief, is it fair to impose that on others? The ethics here is no different than any other off-level use of safe drugs. The only difference is the possibility of making your ME/CFS permanently worse. That, as I said before, is as much a belief as improving permanently.

 

[Utsikt]

Is the off-level use of safe drugs really comparable to assisted suicide? Or is it just your belief? If it is your belief, is it fair to impose that on others? The ethics here is no different than any other off-level use of safe drugs. The only difference is the possibility of making your ME/CFS permanently worse. That, as I said before, is as much a belief as improving permanently.

I did not say it was comparable to assisted suicide in general.

I said it was comparable to assisted suicide in that regard, in context of the previous sentence that referenced the ethical considerations in play when another person has to act in order for you to be able to test a treatment.

A person with a medical, legal and ethical responsibility to protect your wellbeing has to agree do let you try something that has the potential to cause harm. They will bear the personal responsibility if something goes wrong.

Therefore, the choice to try the restricted treatments can’t be entirely yours. You can’t force them to write a prescription if they don’t want to.

And by extension of that, the choice falls to the society through their governing mechanisms, through which they decide the legal, medical and ethical framework in which the practitioner is allowed to make a judgement about what they will and won’t allow you to try.