Understanding cure in myeloma

[music] A few weeks ago, uh the international myoma society did organize a specific meeting uh on uh the uh definition of cure and that's really something totally new in fact for multiple myoma you know in a lot of papers a lot of articles a lot of manuscripts we are always writing myoma is an unccurable disease and that's not true you know so it is time to think about differently about the outcome of patients [snorts] uh with this disease why because we have now a lot of new agents uh new drugs And recently uh the development of new imunotherapies is uh very fast. And when we are combining in fact those new drugs uh together with autoguous stem cell transplantation etc. We have now incredible results in term of response to the disease and uh long-term outcomes are really uh now improving. So can we speak about cure for all patients? Uh is it good to speak about cure in the relapse setting for example? So we looked at a lot of database, a lot of clinical studies and we did analyze uh the results of these studies uh with a very long follow-up and we also have now the tools to look at the depth of response, the quality of response to a specific treatment. And we are calling this minimal residual disease assessment MRD. And we know that there is a very strong correlation between MRD negativity. So we are not able to detect any myoma cells, any tumor cells within the bone marrow and most importantly not only MRD negativity but sustain MRD negativity. If we are doing sequential assessment of the disease within the bone marrow and we are not seeing any abnormal tumor cells during a long period of time, maybe that could be the definition of cure. So we are now proposing a definition of cure. This definition is very simple and that's a consensus uh between all the experts that were attending this meeting. the patient has to be out of treatment. He is not receiving any treatment and he must [music] be MLDD negative during 5 years. Uh so that's a very very uh important point because we need to stop the treatment. Someone that is receiving treatment during a long period of time or until disease progression this is not cure. [snorts] And also we need to have a very long followup uh to be sure that the disease is not coming back in fact after 1 2 to 3 years. So how to achieve cure and I mentioned this previously we need to combine the classes of agents that are [snorts] very active if possible with autogu stem cell transplantation [music] an intensive treatment for younger patients. We are proposing autotolog stem cell transplantation up to the age of 70 years and after highdose therapy we can propose a maintenance with eventually two drugs possible 2 years. So overall we can have a treatment duration of 3 years and then we are stopping the treatment and we are carefully following the patients. So what is the cure rate? In fact, the cure fraction uh this is very very difficult in fact to uh speak about this because this rate is really depending on the drugs that we are using and drug access is really key. You know that um the uh reimbursement of drugs or combinations the access to uh drugs is not unfortunately the same all over the world. you know you have all the drugs available very quickly in the US in Germany uh and in Europe we are fighting you know one country after the other you know for the reimbursement etc and the one drug or one combination can be approved by the European authorities by IMA but that's subsequently a country per country decision for reimbursement and we Sometime the time from the uh approval by IMA and the time to the reimbursement can take very long. It can be 2 years, 3 years, 4 years. So access is really key. But if we are combining now all the agent that we do have, we can expect to cure uh and we are probably already curing uh something like 30% 40% of young patients. We know also that with the same treatment the cure rate will not be identical according to some characteristics of the disease. If you have a disease with let's say standard risk I mean no cytogenetics abnormalities the outcome will be much better. On the opposite if you have poor cytogenetics we know that it will be rather difficult um not to reach a response but to have the cure uh at the end of the day. So this is not that simple and when we are speaking about with patience you know we can give hope definitely the most important word to my opinion is hope because we have de we are developing a lot of new agents and they are becoming more and more effective you know so with this uh clinical developments with this new approvals the outcome is really uh um improving uh very very fast. So in front of one patient we cannot say based on your clinical characteristics based on your biology you will be cured. No [snorts] we can say the probability of response is very high. The depth of response is very important and if we can reach MRD negativity therefore we can expect a very very long overall survival for you you know and um we can also compare the outcome of myoma patients with the outcome of the general population without the disease with the same age. And now we are showing that we are close to reach the same remaining life expectancy. Uh and this is also one way of curing patients. You know your life expectancy will remain the same of the population without multiple myoma. Obviously you have to have uh to receive drugs. You have to unfortunately uh to experience some toxicity, some side effects. But at the end of the day, what we want to propose you is the same life expectancy as the general population without multiple myoma. >> [music]