3 ms·

Truth is that we don't even know what [disease] is. ALS could be several etiologically distinct diseases that manifest similarly. And there are indications of

Truth is that we don't even know what [disease] is. ALS could be several etiologically distinct diseases that manifest similarly. And there are indications of this in the fact that certain drugs -- like the recently approved Tofersen -- have some effect on a subset of ALS cases but have no effect on others. On that note, even Tofersen is of very questionable efficacy: https://www.cnbc.com/2023/03/22/fda-advisors-vote-against-effectiveness-of-biogen-als-drug.html https://www.cnbc.com/2023/03/22/fda-advisors-vote-against-ef... I'm usually hugely sympathetic to "Right to Try" cases. But, in the article, you've got people without the disease in question (whatever it is) seeking the right to try drugs that are of very questionable efficacy to begin with. I say let them have it, for whatever good (or harm) it'll do. People can decide for themselves whether the side effects they might experience are worth the amelioration of fear and glimmer of hope they receive.

klipt 3y ago
> ALS could be several etiologically distinct diseases that manifest similarly. What's the common factor - are they all autoimmune diseases? Would the "inverse vaccine" approach discussed here recently help?
- A_D_E_P_T 3y ago
  It's unclear whether or not ALS (any form) is an autoimmune disease. Autoimmune diseases are typically associated with autoantibody production. These are in evidence in (-- at least some forms of --) ALS, but in an unusual way that makes their role highly uncertain. They are potentially beneficial. See: https://pubmed.ncbi.nlm.nih.gov/25344935/ https://pubmed.ncbi.nlm.nih.gov/25344935/
margalabargala 3y ago
> in the article, you've got people without the disease in question (whatever it is) Honestly my takeaway from the article, and from a lot of the comments here, is that we should be (but aren't) updating our definition of "disease" for some diseases as our understanding grows. If you have partially blocked arteries, you're not actively suffering from heart disease, but it's normal to give that person drugs and advise lifestyle changes as soon as that's detected. You don't wait for them to have a heart attack. Of course, the cause and effect is much better understood with heart attacks and isn't at all clear with ALS. But the fact that ALS doctors were bewildered that someone might want to take prophylactic measures says to me that they have some blinders on. Imagine getting some tests done, learning your coronary arteries are 60% blocked and your LDL levels are at the 98th percentile, and having a cardiologist tell you "Don't think about it for now, come back once your heart stops beating".
- carbocation 3y ago
  I think you are nicely describing the field of preventive medicine. You picked a particularly good example (CAD) because we know that preventive efforts work. Hopefully that will expand to all diseases in time. In the circumstances where we really understand the disease process, is seems much better to prevent symptom onset than to wait to treat after things are so bad that the patient notices them.
  - margalabargala 3y ago
    What's worrying is that based on what the article describes, it sounds like branches of medicine that aren't used to practicing preventative medicine are resistant to beginning to do so.
NoMoreNicksLeft 3y ago
I know very little about ALS, other than I'm aware that it exists and is a nervous system disease. How quick is the onset once someone does develop it? Is it a gradual decline, a rapid decline, or do they just wake up one morning and can't move (falls off a cliff)? "Right to try" might be worthless in the last case. (Unless there's reason to believe that it's reversible with some as-of-yet uninvented treatment.)
- SoftTalker 3y ago
  It varies. Stephen Hawking lived with it for many years. For most people, 2-5 years of steady decline is normal between diagnosis and death. In some people it progresses more rapidly, in others much more slowly. I don't think we really know why.
deleted 3y ago
[deleted]

klipt 3y ago

> ALS could be several etiologically distinct diseases that manifest similarly. What's the common factor - are they all autoimmune diseases? Would the "inverse vaccine" approach discussed here recently help?

A_D_E_P_T 3y ago
It's unclear whether or not ALS (any form) is an autoimmune disease. Autoimmune diseases are typically associated with autoantibody production. These are in evidence in (-- at least some forms of --) ALS, but in an unusual way that makes their role highly uncertain. They are potentially beneficial. See: https://pubmed.ncbi.nlm.nih.gov/25344935/ https://pubmed.ncbi.nlm.nih.gov/25344935/

margalabargala 3y ago

> in the article, you've got people without the disease in question (whatever it is) Honestly my takeaway from the article, and from a lot of the comments here, is that we should be (but aren't) updating our definition of "disease" for some diseases as our understanding grows. If you have partially blocked arteries, you're not actively suffering from heart disease, but it's normal to give that person drugs and advise lifestyle changes as soon as that's detected. You don't wait for them to have a heart attack. Of course, the cause and effect is much better understood with heart attacks and isn't at all clear with ALS. But the fact that ALS doctors were bewildered that someone might want to take prophylactic measures says to me that they have some blinders on. Imagine getting some tests done, learning your coronary arteries are 60% blocked and your LDL levels are at the 98th percentile, and having a cardiologist tell you "Don't think about it for now, come back once your heart stops beating".

carbocation 3y ago
I think you are nicely describing the field of preventive medicine. You picked a particularly good example (CAD) because we know that preventive efforts work. Hopefully that will expand to all diseases in time. In the circumstances where we really understand the disease process, is seems much better to prevent symptom onset than to wait to treat after things are so bad that the patient notices them.
- margalabargala 3y ago
  What's worrying is that based on what the article describes, it sounds like branches of medicine that aren't used to practicing preventative medicine are resistant to beginning to do so.

NoMoreNicksLeft 3y ago

I know very little about ALS, other than I'm aware that it exists and is a nervous system disease. How quick is the onset once someone does develop it? Is it a gradual decline, a rapid decline, or do they just wake up one morning and can't move (falls off a cliff)? "Right to try" might be worthless in the last case. (Unless there's reason to believe that it's reversible with some as-of-yet uninvented treatment.)

SoftTalker 3y ago
It varies. Stephen Hawking lived with it for many years. For most people, 2-5 years of steady decline is normal between diagnosis and death. In some people it progresses more rapidly, in others much more slowly. I don't think we really know why.

deleted 3y ago

[deleted]