Burying the lede a bit: the drug can’t be used because its safety profile is unacceptable, but the possible mechanism might be targetable by other future drugs. Translation: come back in 2040.
It's "lead" not "lede."
MS treatment a step closer after drug shown to repair nerve coating
51–60 of 141 posts
Re: MS treatment a step closer after drug shown to repair nerve coating
#52While it is sad that this drug cannot continue for MS directly, I love that this is science work at its best. Experimental drug, through the efforts of terminally ill, are able to discover/show/validate quite powerful benefits, but the drug is too dangerous to use itself. So we stop it. It is progress and many times, progress is not easy nor cost free. Thank you to all those who, even in their terminally ill state, h…
One thing I’ve always wondered is: how many drugs have we rejected like this, which were actually bimodal in their safety profile, the way many drugs are bimodal (or polymodal) in their effect profile.
I.e., that there’s some inherent difference between the people the drug harms, and the people it doesn’t; and if you can identify that difference, and test for it, then you can safely give the drug to only the people it doesn’t harm.
The way we currently investigate drug safety in clinical trials doesn’t really allow for this approach. We only seem to do it when a drug is initially declared as safe; introduced into the market; and then we find that it has side-effects that clearly correspond to some subpopulation.
Re: MS treatment a step closer after drug shown to repair nerve coating
#53Earlier quoted context omitted.
There is no research showing that stem cells are effective for these conditions. That's why it's a scam.
I just pointed you to a clinic in the Ukraine who 30 years ago initially did 5 years research before clinically offering fetal stem cell treatments for 25 years - where their research, and I'd recommend you read through their site + you can email to ask them for whatever evidence or 3rd party research by others they could point you to to satisfy you. Edit to add: or even better for actual experiential learning - find…
This https://quackwatch.org/research-projects/stemcell/ isn't encouraging.
The fact that they've been running for 30 years proves nothing. See e.g. https://www.mcgill.ca/oss/article/quackery/hoxsey-hoax for another example.
Anything's possible. But without much better evidence than these guys seem to be offering, I wouldn't touch them with a barge pole.
Your "even better" suggestion seems pretty ridiculous to me, by the way. "Want to know whether this thing that seems like a ghoulish fraud preying on people desperate for a cure is real or not? All you have to do is find someone with the disease they claim to be able to cure, persuade them to go and do this costly, risky, probably fraudulent thing, and see what they say about their quality of life is afterwards!" And even if they do manage to persuade someone to do that -- all they get is an n=1 single sample, which is pretty hopeless evidence for anything, especially for a disease like multiple sclerosis which commonly comes and goes in mysterious ways.
Re: MS treatment a step closer after drug shown to repair nerve coating
#54To me, following modern MS research progress is as exciting of an inward journey as space exploration is an outwards one. In fact to the point where I have considered leaving the tech field within the next 5-10 years to partake in it so it's fun to see this here on HN. Just 20 years ago the first disease modification treatments were introduced. These were recurring injections of interferon-beta (1b/1a) for a 18-38% r…
“Just 20 years ago..." Personally, when people think we are making good progress on something, I’m reminded of these words: “Before this decade is out...” Sure, great and rapid progress might require a couple percent of US GDP, and a million people working on a problem, but advancing medical research a few decades in the next 10 years, would benefit everyone.
The thing about clinical trials, is there is a minimum time it takes to run one. You can up concurrency but it only gets you so far - you really need to know the long term impact before you can iterate much.
Re: MS treatment a step closer after drug shown to repair nerve coating
#55Layman here. I can't find a report about this clinical trial, maybe I didn't try hard.
It is a phase II, in a phase II the goal is not to test the efficacy of the drug, it is to test the presence or absence of side effects.
A phase III role is needed to test if the drug is helpful to patients. It involves several hundred people to avoid statistical errors. Here there will be no phase III trial.
Another thing is that nerves can repair in the peripheral nervous system (PNS), but (until now) not in the central nervous system (CNS). If a drug can help nerves to repair in the CNS, it is certainly something revolutionary.
Re: MS treatment a step closer after drug shown to repair nerve coating
#56While it is sad that this drug cannot continue for MS directly, I love that this is science work at its best. Experimental drug, through the efforts of terminally ill, are able to discover/show/validate quite powerful benefits, but the drug is too dangerous to use itself. So we stop it. It is progress and many times, progress is not easy nor cost free. Thank you to all those who, even in their terminally ill state, h…
The drug used in this study, Bexarotene, is already approved for cancer treatment and available in the United States and Europe.
Using Bexarotene in cancer patients makes sense because the side effects are more tolerable than dying from cancer.
Using Bexarotene in Multiple Sclerosis patients doesn’t make sense if the net effect is a worsened quality of life (significant thyroid disease) and a shortened lifespan due to accelerated heart disease (hyperlipidaemia and hypercholesterolaemia).
Re: MS treatment a step closer after drug shown to repair nerve coating
#57It's interesting that this drug is a Vitamin A derivative.
The structure is very close to another popular drug, Isotretinoin (Accutane), with similar side effects.
I imagine Accutane probably doesn't have this effect or we'd have caught it, but how wild would it have been if acne medication also ended up being the cure to MS.
Re: MS treatment a step closer after drug shown to repair nerve coating
#58Layman here. I can't find a report about this clinical trial, maybe I didn't try hard. It is a phase II, in a phase II the goal is not to test the efficacy of the drug, it is to test the presence or absence of side effects. A phase III role is needed to test if the drug is helpful to patients. It involves several hundred people to avoid statistical errors. Here there will be no phase III trial. Another thing is that…
Actually there is a recent publication which hints that bexarotene reduced the number of pro-inflammatory microglia/macrophages while increased the number of anti-inflammatory microglia/macrophages in mice:
Re: MS treatment a step closer after drug shown to repair nerve coating
#59It’s kind of disappointing that this drug won’t be used as a treatment. I get that this drug has dangerous side effects but life with MS is terrible and the life expectancy is short 5-10 years. I fully believe that for chronic life threatening diseases people should be allowed to try all sorts of treatments that would be deemed unsafe.
The drug is already available on the market as a cancer treatment.
However, don’t underestimate the impact of those side effects. If the drug accelerates heart disease (hypercholesterolaemia and hyperlipidaemia are among the primary side effects) and causes thyroid issues, then it might very well have the effect of worsening quality of life and shortening overall lifespan.
This drug doesn’t appear to treat the root cause of MS, but instead shows some efficacy in reversing one of the downstream manifestations of MS. That implies it would have to be taken indefinitely or at least cycled throughout life.
I know people want to be free to try different medications, but in this case it would be shortsighted to trade one set of serious, life-threatening health complications for another that might actually be worse.
Re: MS treatment a step closer after drug shown to repair nerve coating
#60To me, following modern MS research progress is as exciting of an inward journey as space exploration is an outwards one. In fact to the point where I have considered leaving the tech field within the next 5-10 years to partake in it so it's fun to see this here on HN. Just 20 years ago the first disease modification treatments were introduced. These were recurring injections of interferon-beta (1b/1a) for a 18-38% r…
My partner suffers from relapsing-remitting MS and it's horrible.
Out of nowhere, body and brain half-quit working. Needs wheelchair, constantly confused. And then suddenly one day it stops.
I like to hope we'll figure this out during our lifetime.