Educated Patient® Multiple Myeloma Summit Looking Ahead Panel: November 13, 2022

This panel was moderated by Kristie L. Kahl, and included Dr. Francesco Maura, from Sylvester Comprehensive Cancer Center, University of Miami Health System, Dr. Marcella Kaddoura, from University of Miami Sylvester Comprehensive Cancer Center, and Mary DeRome, from the Multiple Myeloma Research Foundation.

Kahl: Mary, I want to start with you. We've had a lot of presentations today that have been great. And I think we share a lot of hope in this space. But to help educate our patients further, Mary, can you talk about the resources that the MMRF has available for them?

DeRome: Thank you so much for having me with you today. And I was able to see the last two presentations, which were particularly interesting to me regarding personalized medicine. And we can talk more about that. But as a patient-focused organization, the MMRF has a lot of different resources available for patients.

So certainly from an education perspective, we offer many programs for patients, from patients – summits that are really all-day events that happened in various cities. And patients can either attend in person if they live in that city, or certainly we're doing all live webcasting now, so patients can always join those online. And they talk about all the different facets of myeloma.

We have webinars, usually monthly, talking about different topics in myeloma, such as maintenance therapy, MRD or stem cell transplant. We have Facebook Live sessions that take place after our webinars. And the goal of those is to really answer more questions. And we're able to really get to those after the webinars. And one of the things that I really think is useful with those Facebook Live sessions is that we often have a member of a faculty from an academic medical center, we have one of the advanced practice providers who are working with that doctor in their practice and then one of the patients that works with that advanced practice provider who's either a nurse practitioner or maybe a physician assistant and the physician themselves. And it's great to see these three people as a care team interact with each other and talk about the different phases of the myeloma journey that the patient has been through, and how the patient has played a role in helping with the decisions that are made on, having to do with their health, which is something that Valerie was talking about in the last session, which I think is extremely important for patients to do.

We also have podcasts that patients can listen to. And we do live updates with faculty from all of the major meetings, from ASCO, ASH and also from IMS. So along with those educational resources, we also have our Patient Navigation Center, which is staffed by a number of talented oncology professionals. They can be reached by phone at 884-1663. And their hours are Monday through Friday from 9 in the morning till 7 in the evening, Eastern time. So those folks are available to answer any questions and offer any resources to myeloma patients and can help with clinical trials etc.

We also have a Myeloma Mentors Program where we have myeloma patients and caregivers who have been trained to be mentors to other patients and caregivers. And that's a phone-based program, you can go on our website, look at the different bios of the different mentors that we have in our meta core, and sign up directly online to have a phone-based chat with that mentor and just talk about experiences, which many patients find really, really helpful in their experience.

Kahl: Great, thank you so much. And Dr. Maura, we had someone asked if the IRRM model has been validated?

Maura: So the answer is not yet. We will be done with this week. So we got right now 200 patients from Heidelberg University, enrolling the clinical trial. And those will be the validation of our model. The model was proved cross validation. So it is kind of complicated, but basically you run 30 or more around of your model around 30 more times, getting a piece of the dataset and use that as a validation. So it's kind of like internal validation for designs are more so that's good, it better and external. And that's what we're getting now. At ASH, for people that will attend we will present all sorts of addition. I want to spend one second, just because probably people imagine that knows data. And what we're using almost less than half of almost applications, including the model are actually part of the combat, which was a team, a monumental effort from EMRs, where patients were sequenced, and the data were shared for free for all the users and scientific academic centers.

DeRome: That's what we do. So that's our thing, providing data to researchers like yourself Dr. Maura and also Dr. Kaddoura talked a lot about our My Drug Study as well. So, this is what we like to do is provide the studies and information to researchers to help them improve outcomes for all patients.

Kahl: Thank you. And Dr. Kaddoura, are there ongoing studies looking at quiescent cells so that more patients can go off maintenance if MRD negativity lasts for three consecutive years?

Kaddoura: I'm not aware of that. Yeah, I'm not sure about that.

Kahl: Mary, we had this question come in earlier, from the advocacy side and just in the myeloma space in general, what are the biggest hurdles that we're still having and finding a cure for myeloma?

DeRome: I think that some of the information that has been discussed in this session, particularly around the area of personalized medicine is really the key to be being able to provide the best outcomes and potential cures for patients as well. So what it really rests around is collection of data from as many patients as possible. And that data can include their genomic data, it can include all of their clinical information, as well as what's happening in their immune system. So all of those pieces of the puzzle of a patient coming together, and then being able to be utilized by researchers to be able to as Dr. Maura was talking about a sort of a predictive cure, certain types of patients would have a certain type of outcome. So there's a number of different ways that therapies can be personalized, they can be personalized through risk, as Dr. Maura was talking about, they can be personalized through targeted therapies that would be appropriate for patients who had certain mutations, as Dr. Kaddoura was talking about, they can also be personalized, based on the landscape of the immune system of each patient. This is really a brand new area, which we as the MMRF, and a number of other centers are really looking into right now, which is going to be really very important. And then for the other area of personalization is really in sequencing of therapies. So in multiple myeloma as a community, we really almost have an embarrassment of riches of so many therapies that are available to patients at this time that are approved. And the question then becomes, which is the best therapy for which patient? And which should you get first? And which do you get next, and this becomes increasingly important with these immunotherapies because many of them are targeting the same molecule BCMA. And I think we're still struggling a little bit to find out which therapy that's a BCMA targeting therapy should be used first. And then what can go after that? So this type of gathering of data for researchers to use to answer these questions is super important. And we as an organization, encourage every patient to donate their data whenever they can, and wherever they can. So that so that investigators like Dr. Maura and many other investigators can utilize this data to provide better outcomes for myeloma patients, the more data we have the closer we're going to get to a cure faster. And that's the way these things work.

Kahl: Absolutely. And Dr. Maura and Dr. Kaddoura, do you guys have anything to add to any of those challenges we're seeing?

Maura: I agree with everything Mary said, in particular, about how the research should be performed, like sharing data. A lot of journals now put as a mandatory rule, an obligation to share the data. Some groups don't like to do it. But as far as we're concerned, we are very old, our data available, and we collaborate with many centers and share the data. So that I think is essential. And in terms of curing myeloma, I think some patients are cured. Because when you have 34%, or 10 years of remission, a much higher intensity of your core, I was very curious to do the genetic study, the different cancer, like saying phenotype, but two different cells. So it's what I believe I don't have any data, but that's what you would expect. The question is why we have encoded yet, because in some cancers, if you look at the formula, acute leukemia was the worst leukemia ever in the war, that center in the 70s and 80s. And then they found the molecular mechanism, and then they found the drugs. And basically, most of the patients now are cured completely. So that's not what we're talking about precision medicine for people not in the field that may or may not know these people. It's not like fantasy science movies. These are things that happen already in certain tumors. So we have to do the same. The question is, again, why we haven't done yet, because it's complicated. Myeloma is a very bad disease, it's like probably the metrological cancer, it must be recurrent one more similar to the solid tumors, which again, are very difficult to cure with drugs. And so I think we need to think out of the box, we need to try new avenues, stop doing odd predicament like FISH, like clinical trials based on FISH, clinical trial based on 65 year olds as a criteria, these things are the past. And it didn't work. Actually, it works in improving the outcome significantly with a history of success that's done that. But he didn't cure the disease for most of the patients. So we need to get rid of what helped. But now he's old. And think about new things like this prediction model, like what you mentioned before, mid adaptive strategy, with channel cost all aligned and all the trials that master all these experiences point in a new direction, but somehow this new direction struggles to keep the right speed, because we are keeping all this burden of all the concepts. And so I think the community really needs to try something new, try to risk sometimes need a bit more, and get rid of the old concept and criteria to really get more understanding and improvement on the one that we already achieved.

Kaddoura: Yeah, expanding on one of the things Dr. Maura mentioned, and that myeloma is just a very complicated disease. And while some other diseases in the past were certainly very aggressive, because we didn't know the exact driver. Once we figured that out, those patients actually had very good outcomes, and we were able to start curing patients. The challenge with myeloma is that because it's complex, there can be a lot of things going on at once. And it's just hard to identify what that dominant thing is and what we should be targeting. And so I think that's why we're a little behind in in this disease compared to some others.

Kahl: Dr. Maura, we had a patient ask, is the APOBEC and chromotripsis methods, do they require marrow sample?

Maura: So far? The answer is yes. I think liquid biopsy and the development of liquid biopsies is the future. In myeloma, it's very hard to get what you need for the technology we have. So you get DNA, you get material, but a significant fraction of patients that studies have not shown yet. But we can imagine, show that a lot of patients don't have enough or don't even have. And so right now, we are still working is needed to do all these investigations in the future, we are working on trying to get from peripheral blood, the same information. But right now, bone marrow sorted cell always sorted. That's another important point. And we've arrived to see and that's important, because a lot of data are published yet and still published with very low quality genomic analysis. That's something we, for example, there's a network with Gareth Morgan, The German group and other people with the computational pipeline for whole genome sequencing for free, available for everyone is a Docker so you can stop everywhere in every center to make myeloma studies reproducible, and accessible by the community. So my days are not different, because we are two centers and people wonder who is the best the data generated in the same way. So that's a big effort. Not everybody will jump on this boat. But a lot of people will actually start to use this and to install this tool in their server. So we encourage people to reach us out the tool is free available. And this I think, is one of the things we need, like more consistently more share sharing.

Kahl: We had a question come in, and I know we didn't necessarily touch on this today, but we actually have it asked pretty frequently, so I'm hoping maybe somebody could answer it for me. Is there any study that indicates that multiple myeloma can be transmitted genetically to family members?

Maura: So there are definitely partners as families where myeloma is in reach. Now, there are needs which means patients that are so genetic to pass through man to and father to their progeny, that seems to be associated with a higher risk of multiple myeloma. Some of those are generally higher risk in cancer, there are some things that just increase the risk of cancer in general, there is still a lot of things to do about this, we still need a lot of information. And again, to really understand this, you need like hundreds of thousands of data. And we don't have yet that power. So we're still working on that. But I believe there is definitely a predisposition. And even if there is nobody's position in the risk, the type of the myeloma to develop high risk, low risk APOBEC. Yet, I would like to know, the genetic system, how important and we need to do more. But again, we need more data. And this is not always easy for barrier in shedding people that don't put the data label and so I think that's a very important question regarding the ancestry and the racial disparity, we have run out and NIH Chawan funded with Garrett Morgan, addressing this question called Smart study, and we are working with the Polytechnic 1000 in New York State to look at genomic determinants of passion targeting multiple myeloma. What we have so far is that our hypothesis and data suggests that is not genetic, but mostly is such economic. So we believe the increased risk is due to a more germinal center stimulation that generate more errors in the visa. And that's ingredient higher risk of multiple myeloma that is still in a pot that is weakly supported by the luminary data. But hopefully, we will present some of those data in like more mature data.

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