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How might your drug work for the Alzheimer's disease treatment?
- Alzheimer's disease is a very very different disease from systemic amyloidosis, which I have been talking about up till now. So in Alzheimer's disease there are amyloid deposits in the brain substance, which you never get with systemic amyloidosis. You can have amyloid in systemic amyloidosis in the membranes that surround the brain, and in the blood vessels, which supply the brain, but you don't actually have it within the brain substance. In Alzheimer's disease there are amyloid deposits in the brain substance, but they are microscopically small. The total amount of amyloid in the brain in Alzheimer's disease is in milligrams, a small number of milligrams, maybe a 100 milligrams, something like that. It's absolutely tiny! - In clinically manifesting people with Alzheimer's disease?
- It's a very very small amount of amyloid in Alzheimer's. There are tiny microscopic plaques, they're called little amyloid deposits, which the neuropathologists call plaques, and there are other abnormal protein structures, which are called neurofibrillary tangles, which are not actually bona fide amyloid, but are very similar in terms of the way that proteins are folded, and so on. So these are the neuropathological hallmarks of Alzheimer's, but they are very very small in amount. In the liver of someone with systemic amyloidosis you might have 5 kilograms of amyloid, OK? It's a completely different situation. - 5 kilograms vs. milligrams?
- Yes. So I don't call Alzheimer's disease "amyloidosis". It is very important in medicine - and this is quite a controversial area - because the neuroscientists and a lot of the biochemists and biophysicists who work in this area, who are not clinical, they use words in a very loose way. If you're a clinical doctor looking after patients, what you call things really matters. If you use the word, which is one diagnosis, and has one treatment, and you use that same word for somebody else, who has got a different disease, which requires completely different treatment, it's a disaster! So unfortunately, there is a fashion nowadays, which is probably impossible to overcome, in which this influence from the basic scientists has strayed into the clinical world to some extent, and people call Alzheimer's disease, Parkinson's disease, Huntington's disease, they call those "amyloidosis", because they all share at the molecular level certain similarity, the particular way of abnormal structures of proteins, OK? But they are not amyloidosis. In Huntington's disease the abnormal stuff is in the nucleus of the cell, in Parkinson's it's in the cytoplasm of the cell, and in Alzheimer's disease it's outside the cells, which is correct, most like amyloidosis is elsewhere. But they are completely different, they are more different than trying to do a long jump on Earth, a long jump on the Moon, and a long jump on Mars. Those are different for obvious reasons: no atmosphere, different gravity, OK? But the environment inside the nucleus, in the cytoplasm, and outside the cell is totally different, and it's very important not to conflate these things. Conflation in this way is very dangerous, and even within systemic amyloidosis, if you make a mistake and you call somebody AL amyloidosis, the treatment then is cytotoxic chemotherapy, which can kill the patient easily, and does kill patients, because it's very toxic, and if they've got a gene mutation, and do not require cytotoxic chemotherapy, to make this mistake is a disaster. So as clinicians, we are very sensitive to using words properly.
- Which is very important, and I think this underscores the point of not only having a general diagnosis, "amyloidosis", "cancer of this organ", but a precise and complete diagnosis, at the molecular level. - Absolutely! Dr. Anton Titov MD
best treatment for damaged hair Alzheimer’s Disease - Amyloidosis link: confusion of definitions (5) | |
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