Amyloidosis treatment principles. Leading expert explains. 4

Amyloidosis treatment principles. Leading expert explains. 4

Amyloidosis treatment principles. Leading expert explains. 4

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We have good treatment now for monoclonal gammopathy. It is the main cause of the commonest type of systemic amyloidosis. We hope that the new treatment would get rid of existing amyloid deposits and will have a big impact and enable people to stay alive long enough to benefit from interventions”. Leading amyloidosis treatment expert. The basis of all our treatment of systemic amyloidosis is this. We try and get rid of the protein that is forming the amyloid fibrils. Professor Dr. Mark Pepys, MD. We work on a cure for amyloidosis. Dr. Anton Titov, MD. Sometimes you can get rid of the abnormal protein. Sometimes it is a normal protein but you've got too much of it. Whatever it is. Sometimes you can get rid of the precursor protein that makes the amyloid deposits. That is the method to stop the amyloidosis progressing and to make people feel better. All current treatment consists of keeping the patient alive long enough to get rid of this precursor protein. We go to extreme lengths to treat amyloidosis. Professor Dr. Mark Pepys, MD. Kidney dialysis, kidney transplantation, liver transplantation, heart transplantation. We pursue every possible measure to keep the patients alive long enough to get rid of the amyloid protein. It is causing the damage in amyloidosis. Unfortunately, you can't always do that. The patients with amyloidosis have a delayed diagnosis. Dr. Anton Titov, MD. Amyloidosis is so far advanced that you can't do these heroic measures. So you can't save them. Also another problem is this. You can't always get rid of the abnormal protein. Professor Dr. Mark Pepys, MD. We have very good treatment now for monoclonal gammopathy. This is the main cause of the commonest type of systemic amyloidosis. Sometimes those new amyloidosis treatments work. Sometimes they do not work. Then we have a group of hereditary amyloidosis diseases. There is a gene mutation that encodes an abnormal protein. That makes amyloid. In such cases we don't have any intervention that treats amyloidosis. These are very challenging amyloidosis cases to manage. We hope that the new amyloidosis treatment would get rid of existing amyloid deposits. It will have a big impact and enable people to stay alive long enough to benefit from amyloidosis therapy. This therapy could work for the people with genetic amyloidosis disease. They would need to have obviously repeat dosing of our treatment. Patients can keep getting rid of the amyloid. Amyloidosis will otherwise recur. Our proposed treatment is not a miraculous amyloidosis cure. Professor Dr. Mark Pepys, MD. It requires the other interventions. You always have to be doing the other things to treat amyloidosis. It is important to understand that. The other thing that is very important to understand is this. Dr. Anton Titov, MD. The very tenuous nature of medication development. Most medication development fails. Very very few amyloidosis medications this start off with an invention in the laboratory. There is a screening of amyloidosis compounds. The animal pharmacology and toxicology is done. Eventually amyloidosis treatment candidate gets into patients. Most of them don't benefit from amyloidosis treatment. They fail for a wide variety of reasons. It is not the subject of this interview. But this is a well-recognized phenomenon. Until the medication is actually licensed by the regulatory authorities we can't really say "it is a medication" or "it is a medicine”. It is not. It is a “candidate". It is something that we are trying to develop for amyloidosis. It is exactly the same with this amyloidosis treatment. Professor Dr. Mark Pepys, MD. We are optimistic about it. We are hopeful. The results of amyloidosis therapy so far are unprecedentedly encouraging. We have actually shown for the first time there is an intervention that can make amyloid go away. It removes amyloid in a short space of time. It can remove amyloid safely. Furthermore, it shows that it benefits the patients with amyloidosis. Dr. Anton Titov, MD. People are still arguing how does exactly amyloid cause amyloidosis. My belief has always been that the major problem caused by amyloid deposits is the disruption of the structure and function of the tissues. The evidence is in favor of this amyloidosis theory. Well, we've got a treatment that makes amyloid disappear. Dr. Anton Titov, MD. What happens? Organ function gets better in the liver. It is easy to measure organ function. That is what we have done so far. Sometimes we can do the same thing in the heart. It will be wonderful. Professor Dr. Mark Pepys, MD. We haven't got there yet. But at the moment this is a candidate amyloidosis treatment. It is not a medicine yet. We need to be straight about that. How might your medication work for the Alzheimer's disease treatment?

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