Man survives CJD

[jambo372]

Here is a report on a man who was cured using the controversial drug Pentosan polysulphate.

http://www.rte.ie/news/2004/1214/cjd.html

[apoger]

And you point is?

[jambo372]

It's thought to be invariably fatal, killing absolutely everyone infected. Recently this and a few other isolated cases have slowly recovered following treatment with experimental drugs. This may be a breakthrough.

[Rolfe]

Originally posted by jambo372
Here is a report on a man who was cured using the controversial drug Pentosan polysulphate.

http://www.rte.ie/news/2004/1214/cjd.html A 20-year-old west Belfast man who has variant CJD is no longer regarded as being terminally ill.While this is a most interesting and potentially hugely important case, I think it is premature to use the word "cured". The article says nothing at all about the man's current physical condition or quality of life.

Rolfe.

[richardm]

Originally posted by Rolfe
While this is a most interesting and potentially hugely important case, I think it is premature to use the word "cured". The article says nothing at all about the man's current physical condition or quality of life.

Rolfe.

Indeed. I think it's safe to say that the poor lad's in a pretty poor state (he was in a hospice until the hospice withdrew because he was no longer terminally ill).

Unfortunately:


"No treatment like this is going to reverse damage," Prof Collinge said. "The most we can expect of these first-generation drugs is they slow down or at best stop the damage to the brain."


- so it looks like he's now in something of a limbo. Still, it's a start.

(Quote from here) (http://www.guardian.co.uk/uk_news/story/0,3604,1373806,00.html)

[Rolfe]

"No treatment like this is going to reverse damage," Prof Collinge said. "The most we can expect of these first-generation drugs is they slow down or at best stop the damage to the brain."If I recall correctly, the patient's father had a very protracted legal battle to get permission to use the treatment. With hindsight, it seems a pity the treatment wasn't begun earlier when his quality of life was rather better.

I wonder what would happen if they used the same protocol on someone in the early stages? I wonder if they could, given that I think it involves injections directly into the brain.

Rolfe.

[jambo372]

It might be far more effective if given very early on in the disease.

[Rolfe]

Originally posted by jambo372
It might be far more effective if given very early on in the disease. It might, indeed. But consider the difficulty of being sure of the diagnosis at that stage, and the very real reluctance to consider injecting a drug directly into the brain of someone who might not have that condition at all.

Nevertheless, I think it's likely that someone is going to manage to get permission to try that in the relatively near future.

Rolfe.

[jambo372]

But if it was available without the legal problems, it could be given earlier on in confirmed cases without wasting time in court.

[Rolfe]

Well, if it shows a good result in one or two people, no doubt that will be what happens.

Rolfe.

[MRC_Hans]

Originally posted by jambo372
But if it was available without the legal problems, it could be given earlier on in confirmed cases without wasting time in court. Yes, of course. And that would be good IF it works, but what if it doesn't? What if it causes serious damage to the patient?

I can never come to grips with you alternative people: If a drug, after the best tests that money could buy, still turns out to have a serious adverse effect in the field, you raise hell.

Yet, if a new experimental drug seems to have worked once or twice, you are hollering for it to be released unconditionally.

Do make a serious attempt to get the whole picture, eeehh?

Hans

[Matabiri]

Originally posted by Rolfe
It might, indeed. But consider the difficulty of being sure of the diagnosis at that stage, and the very real reluctance to consider injecting a drug directly into the brain of someone who might not have that condition at all.

Anyone know how diagnosis is confirmed? I've got it in my head that it couldn't be absolutely confirmed without an autopsy, although I may be conflating CJD up with BSE here.

[Rolfe]

Originally posted by Matabiri
Anyone know how diagnosis is confirmed? I've got it in my head that it couldn't be absolutely confirmed without an autopsy, although I may be conflating CJD up with BSE here. To the best of my knowledge, absolute definite confirmation of the diagnosis is by histopathology of the brain, so failing a biopsy (which I suspect surgeons might be a bit reluctant to do) this would imply a post mortem diagnosis. However, I think a fairly confident presumptive diagnosis can be made in the live patient by a certain stage of the disease.

Rolfe.

[Deetee]

Certainly for sporadic CJD, - the isolated, usually middle-old age form, there may be certain EEG characteristics, and a lumbar puncture is a good way of firming up the diagnosis.
A neuronal protein, 14-3-3, is typically found (send samples in UK to the CJD surveillance unit in Edinburgh).

For variant CJD I am not sure if the same criteria apply, but I don't see why not. The days of waiting til the patient dies before getting brain tissue are behind us, and early diagnosis is possible, therefore early treatment should be likewise.

[Matabiri]

Originally posted by Deetee
For variant CJD I am not sure if the same criteria apply, but I don't see why not.

Excellent; thanks.

[jambo372]

MRC Hans
What if it causes serious damage to the patient ?
So. If it doesn't the disease will. CJD causes an inevitable slow painful death. The patient has nothing at all to lose, without the drug they'll die anyway. It's worth the risk.

I don't recall complaining about side-effects either.

I am only advocating the use of experimental treatment in some cases. I woudln't advise it for a mild condition or for a condition with a known effective alternative.

But this is a deadly disease with no known cure - anythings worth a try. If the drug doesn't kill the patient the illness will. In this case it's worth the risk.

[Rolfe]

Originally posted by jambo372
So. If it doesn't the disease will. CJD causes an inevitable slow painful death. The patient has nothing at all to lose, without the drug they'll die anyway. It's worth the risk. First, that's assuming the diagnosis is certain. Second, maybe the patient and his relatives won't see it that way. For example, the relatives of the patient in question seem to prefer his living as a vegetable to his being dead. And human nature being what it is, if the wonder-drug turned out to shorten life or reduce the quality, what's the betting someone would then sue?

These things have to be taken a step at a time.

Rolfe.

[cesium]

If the drug is given early I would think that it would have a greater chance of being effective, but the damage CJD does is completely irreversable (the cells expand and pop, leaving holes in the brain tissue).

It would also be extremely important to figure out how this drug works on the disease, and using this information to assess the possible side effects. I can immagine that large doses of blood-thinner cannot be good for the heart, kidneys, liver, brain, lungs and just about every other organ in the body.

I wonder about the possibilities of euthanasia in cases like these.

[MRC_Hans]

Originally posted by jambo372
MRC Hans
What if it causes serious damage to the patient ?
So. If it doesn't the disease will. CJD causes an inevitable slow painful death. The patient has nothing at all to lose, without the drug they'll die anyway. It's worth the risk.

I don't recall complaining about side-effects either.

I am only advocating the use of experimental treatment in some cases. I woudln't advise it for a mild condition or for a condition with a known effective alternative.

But this is a deadly disease with no known cure - anythings worth a try. If the drug doesn't kill the patient the illness will. In this case it's worth the risk. And they did try it, right? So, what's your problem? That it requres a bit of red tape to be allowed to try it?? Do you feel that terminally ill patients should be free for all guinea pigs???

Hans