Professor Sir Dr. Mark Pepys explains links between Amyloidosis, Alzheimer's disease, inflammation and C-reactive Protein. Exclusive interview. See more at Online Expert Centers at
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The basis of all our treatment of systemic amyloidosis is to try and get rid of the protein that is forming the amyloid fibrils. If you can get rid of the abnormal protein, or sometimes it's a normal protein but you've got too much of it, whatever it is. If you can get rid of the precursor protein that makes the amyloid deposits, that is the way to stop the disease progressing and to make people better. And so all current treatment consists of keeping the patient alive long enough to get rid of this precursor protein. So we go to extreme lengths to do that, so kidney dialysis, kidney transplantation, liver transplantation, heart transplantation - every possible measure to keep the patients alive long enough to get rid of the protein that's causing the damage. Unfortunately, you can't always do that. The patients, as we said before, have a delayed diagnosis, they are so far advanced you can't do these heroic measures and so you can't save them. Also another problem is, you can't always get rid of the abnormal protein. So we have very good treatment now for monoclonal gammopathy, which is the main cause of the commonest type of systemic amyloidosis, and sometimes those new treatments work, sometimes they do not work, and then of course we have a group of conditions, which are hereditary, where there's a gene mutation that encodes an abnormal protein, and that makes amyloid, and in such cases we don't have any intervention, which can get rid of that. And so these are very challenging cases to manage. We hope that the new treatment which would get rid of existing amyloid deposits will have a big impact and enable people to stay alive long enough to benefit from interventions, which could work and the people with genetic disease, they would need to have obviously repeat dosing of our treatment to keep getting rid of the amyloid, which will otherwise recur. Our proposed treatment is not a miraculous cure in its own right, it requires the other interventions, you always have to be doing the other things. So it's important to understand that. The other thing that's very important to understand is the very very tenuous nature of drug development. Most drug development fails, very very few drugs which start off with an invention in the laboratory and screening of compounds, and the animal pharmacology and toxicology, eventually get into patients - most of them don't go all the way. They fail for a wide variety of reasons, not the subject of this interview, but this is a well-recognized phenomenon. So until the drug is actually licensed by the regulatory authorities we can't really say "it's a drug" or "a medicine" - it is not. - It's a "candidate"... It's something that we're trying to develop, and it's exactly the same with this treatment. So whilst we are optimistic about it and hopeful and the results so far are unprecedentedly encouraging. We've actually shown for the first time there's an intervention that can make amyloid go away in a short space of time, safely, and furthermore, it shows that it benefits the patients. People are still arguing how does exactly amyloid cause disease, and my belief has always been, and I think the evidence is in favor of it, that the major problem caused by amyloid deposits is the disruption of the structure and function of the tissues. Well, we've got a treatment that makes amyloid disappear. What happens? Organ function gets better in the liver, in which it is easy to measure organ function - that's what we've done so far. If we can do the same thing in the heart, it will be wonderful. We haven't got there yet, but at the moment this is a candidate treatment, it's not a medicine yet, we need to be straight about that.
Dr. Anton Titov MD
How might your drug work for the Alzheimer's disease treatment?
Get precise diagnosis and best treatment plan from world's top medical specialists selected to fit your medical problem precisely and perfectly.
The basis of all our treatment of systemic amyloidosis is to try and get rid of the protein that is forming the amyloid fibrils. If you can get rid of the abnormal protein, or sometimes it's a normal protein but you've got too much of it, whatever it is. If you can get rid of the precursor protein that makes the amyloid deposits, that is the way to stop the disease progressing and to make people better. And so all current treatment consists of keeping the patient alive long enough to get rid of this precursor protein. So we go to extreme lengths to do that, so kidney dialysis, kidney transplantation, liver transplantation, heart transplantation - every possible measure to keep the patients alive long enough to get rid of the protein that's causing the damage. Unfortunately, you can't always do that. The patients, as we said before, have a delayed diagnosis, they are so far advanced you can't do these heroic measures and so you can't save them. Also another problem is, you can't always get rid of the abnormal protein. So we have very good treatment now for monoclonal gammopathy, which is the main cause of the commonest type of systemic amyloidosis, and sometimes those new treatments work, sometimes they do not work, and then of course we have a group of conditions, which are hereditary, where there's a gene mutation that encodes an abnormal protein, and that makes amyloid, and in such cases we don't have any intervention, which can get rid of that. And so these are very challenging cases to manage. We hope that the new treatment which would get rid of existing amyloid deposits will have a big impact and enable people to stay alive long enough to benefit from interventions, which could work and the people with genetic disease, they would need to have obviously repeat dosing of our treatment to keep getting rid of the amyloid, which will otherwise recur. Our proposed treatment is not a miraculous cure in its own right, it requires the other interventions, you always have to be doing the other things. So it's important to understand that. The other thing that's very important to understand is the very very tenuous nature of drug development. Most drug development fails, very very few drugs which start off with an invention in the laboratory and screening of compounds, and the animal pharmacology and toxicology, eventually get into patients - most of them don't go all the way. They fail for a wide variety of reasons, not the subject of this interview, but this is a well-recognized phenomenon. So until the drug is actually licensed by the regulatory authorities we can't really say "it's a drug" or "a medicine" - it is not. - It's a "candidate"... It's something that we're trying to develop, and it's exactly the same with this treatment. So whilst we are optimistic about it and hopeful and the results so far are unprecedentedly encouraging. We've actually shown for the first time there's an intervention that can make amyloid go away in a short space of time, safely, and furthermore, it shows that it benefits the patients. People are still arguing how does exactly amyloid cause disease, and my belief has always been, and I think the evidence is in favor of it, that the major problem caused by amyloid deposits is the disruption of the structure and function of the tissues. Well, we've got a treatment that makes amyloid disappear. What happens? Organ function gets better in the liver, in which it is easy to measure organ function - that's what we've done so far. If we can do the same thing in the heart, it will be wonderful. We haven't got there yet, but at the moment this is a candidate treatment, it's not a medicine yet, we need to be straight about that.
Dr. Anton Titov MD
How might your drug work for the Alzheimer's disease treatment?
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