 Ik zou willen welkomst de groep, de Amulodos groep van Spanje, die van Spanje is. Welkomst, Italië, oké, welkomst hier. Israel, daar, oké, welkomst, en de groep van de Nederlandse, welkomst. We hebben vier Amulodos groepen hier, en de Amulodos is ook een speciaal topic in onze masterklaas. Natuurlijk praten we over Mioloma, maar Amulodos is absoluut een relatieve ziek, en dat is een van de topics we ontdekken in de MPE, hoe we het kopen en wat de treatmenten zijn, wat de relatie met Mioloma is. Wel, je ziet het als je onze masterklaas volgt. Ook wil ik remarkten dat we drie potentiële nieuwe meisjes hebben, maar ik denk dat ze heel erg interesseerd zijn. Armenië, en ik denk dat ze daarvan zijn. Oké, bedankt voor het komen. En dan hebben we Suiden ook, maar ze hebben het al gezegd dat ze andere plekken hadden, maar ze zijn heel welkom en ook een nieuw groep van Spanje. Ik denk dat ze niet in de moment zijn, misschien de volgende HM. We beginnen vandaag met twee subjecten. We beginnen met de eerste Will Alfonso en Ananda. We zullen jullie door de hele konferentie, de hele masterklaas. En vandaag beginnen we eerst met de MPE dialogues. Dat is meer over Mioloma en hoe we het nu kijken. Hoe we het nu kijken. Het is in de toekomst. En we vragen een paar echt experten om ons te praten over dit en te geven ons een update op dit. En dan hebben we onze Prostitius Atlas project, die we hebben al voor een paar jaar. En we willen je update op dit. Zodat het dag meer een lange programma zal zijn, de hele dag. En dan gaan we meer in depte op een aantal aspecten om de treatmenten, de cures en de care van Mioloma patients. Je kunt het op je programma zien. De staff heeft dit gemaakt, heel praktisch, denk ik. Misschien heb je het gezien. Dat is de hele programma in één kleine slag. Het is ook heel handig als je moet spelen. Je hoeft niet te spelen als we je papers etc. Dus dat zal onze programma zijn op zaterdag. En ik hoop dat je onze masterclass vandaag en tomorom heel veel genoemd hebt. En ik hoop dat je ook op zondagmorgen op de AGM gaat zien. Dank je. Oké, welkom, iedereen. En ik bedoel, tot alle van jullie die me al kennen. En ook meer van die die voor het eerst naar de MPE-community komen. Ik hoop dat je op de huis voelt en dat je een deel van de familie voelt. Mijn naam is Ananda Plate, ik ben CEO van Mioloma patients Europe. En als je vragen of hulp nodig hebt, laat me dat weten. De eerste seizoen zal door de conferentie rijden. En vooral wat het voor het eerst gaat zien. Kijken, problemen die je zal vinden door de conferentie. Alfonso zal in de detail van de scientific expressions, de acronyms en de concepten die je niet te bekend zou zijn. En ik zal je een overview van de organisatie geven. En wat we over de laatste jaar hebben gedaan. De goede van de seizoen is om te weten over de organisatie, of je niet al bent. Verkrijg een update, als je ons weet en hebt met ons gehad voor de vorige jaren. Help je navigeren over het meeten. Ook helpt je de goede die je hebt binnen je organisatie. In termen van leiding. Dus waarom heb je naar de masterclass gehad? Waarom is het voor je nodig? En ook misschien welk van de MiloMind, AL en Melodosis concepten die je hebt. En die moet je over de conferentie. Dus, als summary van wat de NPE is en hoe het al begint. Dus we hebben in 2011 gemerkt van de twee pre-existing organisaties die er toen existed, die was MiloMai Euronet en de Europese MiloMai platform. Ze hebben beide besloten dat het niet voedselig was om dezelfde dingen te doen, te duplikeren, separate. Dus ze hebben besloten om te merken en de NPE familie was gecreëerd. Dus we hebben gegaan sinds 2011. Hier is een van de foto's, de eerste foto's. Ik zie Christa en Mika in de foto's als dit was gecreëerd. We hebben al veel masterclass gehad. De eerste was in 2012 in Edinburgh. Voor jullie die met ons geweest hebben, dingen ontwikkelde. Ik weet niet of de start daarin zit. Hij is niet. Maar daar is hij. De eerste seizoen was echt... Homemade. Laten we het zo doen. En we wilden mensen die het ooit konden kopen, die het ooit konden. Ze zouden in de vond van iedereen staan en praten over het. Het was een mooie event. Een mooie get-together, maar het was niet dezelfde kwaliteit in terms van kennisstandards die we nu hebben ontwikkeld. Dit is meer en meer professioneel over de jaren. Dit is de resultaat van 2017. Hopelijk zal dit nog steeds gaan. We hebben op de moment, ik denk, 41 mensen. 38 volle mensen. En we hebben drie aandrijven. Dit in 20... Aan 20 zikere landen in Europa. En zoals je heen hebt van Hans, we hebben drie nieuwe membrijkeerplikaten. Een van Spanje, een van Zweden, die we heel blij zijn om het te nemen. We hebben nog niet een club in Zweden. En Arminia, van het vaste Europese regione. Dit is de bevrijdingstructuur van de NPE, dus we hebben een board die was electeerd in oktober 2016. We hebben vijf boardmembers aan de moment en voor die van jullie die op de AGM gaan blijven, we hebben nog twee bevrijdingstructuur gekregen, een was Mika en de andere was Maïet, dus we hebben twee bevrijdingstructuur gekregen om de boardpictuur weer te completen. De board werkt heel snel met de executieve bevrijdingstructuur, die de bevrijdingstructuur zet de strategie en de bevrijdingstructuur krijgt de bevrijdingstructuur uit van een executieve punt van view. En die zijn mijn collega's Alfonso, Ana Vallejo, Ana Rovira en mijzelf. En we hebben kate Morgan daar te joinen. Ze bevrijdingstructuur aan de NPE en zal aan de 1e september joinen, waarin we heel blij zijn. Vanaf een Europese perspectief, en dit is iets van onze industriekollega's die we in de morning meten, voelt het een beetje bood om dit weer te horen. Dus er is nogal... Er is geen dubbeel over de fact dat we voor Miloma werken en voor Miloma patiënten. Maar sommige activiteiten die we doen zijn cross-disees. En voor dat is het heel belangrijk om te align met andere associaties, andere staakhaldergroepen en zelfs andere organisaties, cancerorganisaties of zelfs patiëntorganisaties in general, zoals het E.P.F. of Uratis zou zijn, bijvoorbeeld. Ook medicinaalsocijties in order om de widerpictuur te ontvangen. Dus we hebben, over medicinaalsocijties, we zijn in de Esmopac, de ECCO, rare cancers Europe. We're involved in the European Medicines Agency, Patient and Consumer Working Party. We also are part of the new initiative, which is the Europe Bloodnet, which is a part of the European Reference Networks, which we won't cover because of time limitation during this masterclass. But if you have any questions regarding that, please feel free to contact us. En informaal, we can group of cancerpatient organisations, where all the umbrella organisations of Europe are part of. These are our strategic objectives. They haven't changed over time. Basically, the key elements of this is to create a strong organisation, but also to facilitate the creation of sustainable and strong organisations at a national level. What MP is really about is not only advocating at European level for access or for early diagnosis, or whatever priorities we might have at a European level, but to also enable and build capacity and capability at a regional and national level. Our member groups there are able to do the same thing at a national level. To really enforce and encourage them and give them the right instruments so they can move at a national level. And then we have several other strategic objectives, but I would focus on access to treatment, which is an issue in every region of the European Union and wider Europe, and early diagnosis. I think that we can say our matter to all of the ones that are sitting in this room. So that's why I highlight them. We're currently developing the new, well, actually the board is developing the new strategic objectives, and those will be presented to you when they're ready, so you can give input and will be voted at the next AGM. And from this, I think I gave the overview of MPE, and I'll pass on to my colleague Alfonso, who will drive you through the dirty science bit. Thank you, Ananda, for the introduction. And well, as I'm based here in Madrid, I would like to give you a warm welcome to the city that I really love. I hope you, all of us, enjoy these two exercises in the masterclass, and also for those at the National Assembly, which I'm sure we will enjoy in a different way on Sunday. So welcome here. So what I would like to do in the next few minutes is just to give you a brief overview and to drive you through the AGM. For those who have been attending here for many years, you may notice that there have been some slight changes in order to make this more interactive and more useful for all of you attending. And also for those of the newcomers, I hope it will help you to understand better what we are here, what we are going to do. So, first of all, when we are organizing this masterclass, our main object is that you make the most out of the whole meeting. And we are here in order to basically know that the landscape of Maya Loma has changed completely and it's changing at an incredibly high speed. And also we are going last year for those of you attending the annual masterclass. We were talking for the first time about LA Milioidosis. This is something that some of you have asked to talk about. So one of the main objectives is to know about the science, to know about the latest and exciting updates in both fields, as well as in other related diseases. Of course, we have also thought about including some sanctions to cover one of our main goals, to help you building capacity in your organization so you are able to advocate and to support better your patients in a daily basis. And this is something that we had in mind when developing this masterclass. Also, I think this is a great opportunity because we don't usually have the time to meet many times a year. So we have here two, three days to network or set of experiences to talk to each other. We have in the room not only patient groups, but also some of very good speakers, physicians and also from the advocacy community as well as some pharma representatives. So I think it's a great opportunity for you to engage with each other and so on. And of course, one other thing that I think is one of the most important keys of this AGM, which is to report back to your community. Don't say what you're going to hear today for yourself and try to share with the others. So if you have the chance to go through the agenda, this year, listening for your feedback from other years, we have tried to make an agenda which allows you to customize your journey through the conference. We're going to start today. We have five panelists which are going to accompany us in our first multi-stakeholder panel and our first NPE dialogue in which we are going to try to set the basis of what are the current status in myeloma and how does the future look like. And we will have the perceptions and the opinions from different perspectives, not only from the physician side, but also including the patient representative side, the industry, the regulator and the payer, and also to promote the debate and the discussion. And you are very welcome to participate in the discussion. Apart from that, we will have scientific sessions. We have a plenary session by Dr. Sonia Speckman to set up the basis of the current diagnosis and treatment guidelines in myeloma, AL-Amelioidosis and other health-related diseases. And then we will have for the first time four different breakout sessions. So you will be able to choose whether you want to go to one or to the other in order to cover your interest. One of them will be on AL-Amelioidosis. We will have Jean-Paulo Merlini here, who is one of the best genome experts in the world in this field. And the other one by Dr. Gabor Michala will be about the role of stem cell transplant in myeloma. After that, we will have again two breakout sessions, one of clinical trials and drugs under development, currently in myeloma and AL-Amelioidosis. And also a panel, at the same time a panel on how to treat myeloma in the light of access virus. This is something that you have requested as many times. You have told us it is great to know about the new drugs, but I certainly have some problems in my country in terms of access, so I really need to get this information. And also we will have some advocacy sessions. Two of them will be plenary, one today, about the Atlas to give you an update. Also to hear back from you, because we want this AGM to be very interactive. We really want and need to hear back from you. And the other one on a project that we started this year, which is the MPE Advocate Development Program. En my colleagues Anna, they ask you to choose some of the rotating sessions, to mark three rotating sessions for tomorrow, because we will be having small groups working on different topics, from cross-border health care, the drug approval route, engaging with industry and best practices in supporting patients and caregivers. So I really hope that this year we are providing you a quality agenda that really meets your expectations. And right now, even though I'm not a scientist, I don't have a scientific background. Many of you are aware of that, but I would like to... We are going to hear a lot of scientific terms here. We are going to have, great as a speaker, talking about very important and very interesting topics in both myeloma and amyloidosis. And to set the ground, I would just like to give a brief overview about myeloma and amyloidosis. So even though, I guess, of course, you are very familiar with myeloma, I would just like to make a brief overview. So as you know, myeloma is a type of blood cancer which is arising from plasma cells. The plasma cells are responsible for creating antibodies also called immunoglobulins, which are the kind of cells that help us to defend against infections. In the case of myeloma, these plasma cells start to grow in a normal way without any kind of control, releasing only one type of antibody which is known as paraprotein, which has no useful function. And then, if this continues growing, then you have what is called the CRAP criteria. You have heard about this. You are starting to have the common symptoms of myeloma, hyperchalcemia, renal problems, denemia, debon pain, enzovoort. In de laatste few years, we have witnessed something which is incredible. In that sense, myeloma has been a winner. Today we have reached a level of understanding better how myeloma develops, how myeloma works, what are the genetics beyond the disease. Right now, we have been able, and the researchers have been able to develop different drugs with different mechanisms of actions. Even though we are still at a point in which myeloma, we have always said that this is a very complex disease. Even though we are knowing it better, how it works, if you think about some other blood cancers such as chronic myelolukemia, which tends to be more easy to manage, and the pathway is easier to handle, myeloma is still a complex disease. But certainly we are getting to a point in which we are knowing better about that. And also if you think about what has happened, I wouldn't go too far away in the last five years, three years, something like that. First since the appearance of thalidomide, handboxesomid in the early 2000s, and now with the novel agents, we are the armamentarium to fight against myeloma, has grown greatly. Also there is a better understanding of the earlier stages of myeloma, also how the complete responses has changed with minimal residual disease, which I will explain later, and also Sonia, Zbekman, Johan, Gardered, en Gabo, Mikara, we'll explain tomorrow, as always, to really fight the disease. So we had a past scenario with the, you know, most of you have been in touch with myeloma patients, or myeloma patients by yourself, but like a decade ago, not so long, this was a devastating disease with a very acute journey, which really had a tremendous impact on the quality of life in patients, while right now we are achieving higher survival rates, it is the management of side effects and also the symptoms of the disease is better, so we are able now to offer a better quality of life, a better wellbeing to the patients, and also it has been a great effort, and it is thanks to you in the community to empower patients to provide patient education information. So right now, we are winning little by little the battle against myeloma. As I was saying, one of the main achievements during the last few years is that certainly a few years, about a decade ago or so, there were some drugs from the same family, there were no many ways to fight against myeloma. You have all the patients there, you try to achieve a remission, then if it relapsed, you try again, but there were certainly not so many options. If you think about the new mechanisms of actions of drugs who have been approved in the myeloma portfolio, it is incredible because it is giving a lot of weapons and a lot of combinations, and right now the scientists are trying to understand how to treat myeloma in the best way possible, which patient might take more benefit for receiving a certain regime of other. Here you have some examples, both deeply into details, because this is something that will be happening tomorrow in the masterclass, but certainly it's impressive what has happened in the last few years. Another important thing that has happened, and we have the chance to discuss this morning in an internal meeting we held, is the MRD assessment. MRD stands for minimal residual disease en the thing is that in the recently has been discovered using some very precise techniques it has been made possible the fact of analyzing how many abnormal plasma cells are in the bone marrow. So what does this mean? Now we are able to locate a few thousand cells or to measure a few thousand or even a few hundred cells in the bone marrow which is given us the possibility to know if the response that we have achieved with the treatment is good or how good it is. So here this is a graph published in the Blood Journal by Joaquin Martínez and his team in which it shows how important MRD can be. So you can see in the blue line patients which has achieved the traditional complete remission they treat you with a certain set of drugs you response to the treatment so we are not we don't need to treat you anymore until you relapse and that also happens in the red line but as you can see the blue line are patients in which the MRD measurement is negative that means they are not able to locate many plasma cells abnormal plasma cells in the bone marrow and the red line shows the red graph shows patients in which there is still a little disease even the complete response has been achieved and as you can see the time to progression it's incredibly much better in those patients which has achieved the MRD negativity compared to the other ones so this is something that it will be discussed during this master class and I think it has been one of the great achievements in my aloma Here it is shown I didn't quote here I think this slide is from Jesus and Miguel but I love it very much and I think it really explains really well what is the exact meaning of minimar recidual disease as you can see my aloma and the symptoms is just the tip of the iceberg but in our way to the cure we are trying to have better drugs which allow us to have better and better responses and there are no abnormal plasma cells in the bone marrow and even though now this slide for me shows something great for the first time in many since my aloma has been there physicians and clinicians are starting to talk about are we now in the current way to cure my aloma and this is something great for me it will be good to retire before 40 because my aloma is not a problem hopefully who knows if that will happen but I like it very much and also I would like to put this slide we see the names on the list and that's a good new because this is the list of molecules that are currently under development in phase 2 and phase 3 trials in my aloma I mean for those of you who has been there 20 years ago you know what was my aloma there was basically a few chemotherapy drugs nothing much more than that if you think that some of probably not all these drugs will get approved probably not all the drugs will work but this is amazing how this thing has changed we have still a lot of challenges out there but really science is working and we are getting better and better in my aloma so now I will go on to alien amyloidosis which I know this may be a little bit more unknown for some of you and as I said we have started working on alien amyloidosis since last year I mean this has been something from the board and also from the executive office we are very passionate about doing things for alien amyloidosis patients because sadly the things doesn't look as good as in my aloma and we are really trying to do things for patients and try to help the patient community and hopefully work together with scientists, with industry with everybody involved to really be witnessing what has happened with my aloma hopefully will happen with alien amyloidosis but let's start by explaining what is amyloidosis so amyloidosis is not just a single disease it is a group of diseases in which one means for the proteins which are called amyloids deposits and bills in organs causing damage the problem is in certain types of amyloidosis it usually builds up in organs such as the heart liver and causing damage to this organ can be potentially fatal and why we started working with alien amyloidosis basically because as Hans stated before between 10 and 15 percent of my aloma patients will develop alien amyloidosis at a certain point so this is a real problem one of the major issues has to be with diagnosis symptoms in amyloidosis is not a very frequent disease in this rare disease and the symptoms are quite vague and there is not only just one single test to diagnose so the scientific communities and GPs and so on are not very aware of what is amyloidosis and that leads to a certain problem of amyloidosis and they are also in contrast to what happened with my aloma there are currently no approved treatments by the FDA or by the EMA for treating this disease so how frequent it is this disease well there was a set of records done by the Mayo Clinic one of the major senders all over the world in which whether were analysing a set of service of patients that they have been treated since 1962 2013 and as you can see alien amyloidosis is the most common type of amyloidosis there are also some other types but this represents at least in this study around 70% of the cases and what is the link and here you can see if you remember I was talking that my aloma the problem was where plasma cells were starting to be produced in a controlled way started to grow without control and when this plasma cell was produced without control it started to free certain kind of proteins called light chains they can deposit directly on certain organs or they can accumulate in amyloid fibrils which starts to the organ so this is basically the link between my aloma and amyloidosis and how it works in very simple works of course as I said tomorrow we will go deep into this about that and also this graphic shows a little bit the reality that we are facing today this is also a graph from the myoclinic and as you can see in this set of service of patients you have 4 cubes showing the overall survival for patients since they are diagnosed and you can see that even those patients diagnosed in the 70's or 60's I mean there is a better a little better overall survival improving 5-year terms if you notice in the first year the outcomes are very poor and one of the main research has been this infrared diagnosis but there is still around from 40 to 60% of the patients passing away in 6 to 12 months within the diagnosis so we are here to try to do something but there are also good news and last year for the first time many of you might be familiar with the annual with the American Society of the Autology Congress for the first time in its long history they had a session on ALI milioidosis so it started to grow certain interest in the community and there has been some trials en studies conducted with new drugs which might be potentially a proof in the future for the treatment of this disease so how has been treated in very few words how has been treated the milioidosis right now one option was to go through and stem cell trust plan but some patients may not be eligible for this and traditionally has been treated with chemotherapy basically with malphalan but there has been some trial en studies showing up activity of some drugs that you might be familiar with in the field of myeloma like teledomide it was showing some activity with some proteasomy inhibitors there was a study presented about the usage of proteasomy in combination with malphalan plus dexamethasone compared to malphalan and dexamethasone alone there were promising results as well as a study called turmaline AL1 in which they were trying to to show the effect of the use of exasomy in the treatment of alamelioidosis also there has been some trials on Dara tumumab this monoclonal antibody which is also used for the treatment of certain types of certain patients of myeloma and the good news is that the other monoclonal antibody which has been which has some efficacy in reducing the amelioid deposits which is called NIOC01 there has been some promising results in two clinical trials in phase 1 and phase 2 and now they are going with the phase 2 phase 3 trials so hopefully in the upcoming years we will see new drugs to treat these patients en well that was basically everything from my side we still have some time if you have any question regarding the conferencing