Answering Your Myeloma Clinical Trial Questions

Speaker 1:
Hello everyone and welcome to our Facebook Live. This is Dr. Joe here, the Chief Medical Officer of the International Myeloma Foundation, and I am joined here by two colleagues and very dear friends. To my right is Thomas Goode, who is good. He is a patient and a marvelous advocate for the myeloma community and has really just become a tremendous force in the world of multiple myeloma. To my left is my dear friend Brian McMahon, who is the founder of SparkCures. If you don't know what SparkCures is, that's okay. You will know by the end of this Facebook Live and just in case you might think that Brian is just doom scrolling here while I'm doing our Facebook Live. He actually is the one in charge of bringing your questions to us so he can legitimately be playing with his phone throughout the course of this time.

Please realize that this Facebook Live is not really designed for us to just talk to you. This is a chance to have you ask questions. For those of you who are familiar with this, we have a program really, if you will, at the IMF called Ask the IMF or ask Dr. Joe anything. And essentially quarterly we do a Facebook Live where we have the opportunity to have you ask questions and we try to answer them in real time. Sometimes we have a focus to our Facebook Live. Today's focus is clinical trials. We want to help have you understand it, demystify it, promote the concept of research and how critical clinical trials are in multiple myeloma. Furthermore, we happen to be hosting this Facebook Live in absolutely lovely and delicious Minnesota. I have to say that because I married a Minnesotan, I married up and we are at our annual support group leader summit.

The IMF has over 150 support groups across the United States and over 81 of those support groups are represented here by incredible support group leaders and often their partners. And we are here this week to focus in on what we can do to further serve the myeloma community. Long enough intro from Dr. Joe, let's start to talk about clinical trials. So many things we could say about trials and we want you to ask the questions that you have. Please enter in your questions. While you're doing so, tell us who you are, tell us where you're from.

Typically, when we do a Facebook Live like this, we have people from all over the planet, not just here in the United States. Please let us know who you are, let us know your questions about clinical trials and the three of us will try to answer your questions. We'll start answering questions in about five to seven minutes from now. But before we dive right into the questions, first of all, I want to introduce Thomas. As I've mentioned, he is a patient. He is a tour de force in myeloma, welcome Thomas. Always good to see you bro.

Speaker 2:
Thank you Dr. Joe, and thanks for having me here on this Facebook Live. As Dr. Joe said, my name is Thomas Goode, and I've been diagnosed with multiple myeloma this year going on 20 years. Within those 20 years, I have undergone two allergenic stem cell transplants for both clinical trials. Also, I did an autologous prior to the allergenics, but today we're focusing on the allergenics and the clinical trials and please ask any questions that you may have.

Speaker 1:
Sweet. The fact that Thomas is here with us, 20 years of the diagnosis always inspires us, always encourages us, and we know that Thomas is here because of the wonderful care that he's been given from his own resilience and indeed clinical trials. To my left is one of my other homies, Brian from SparkCures. Brian, I'll give you a chance to introduce yourself.

Speaker 3:
Hey everybody, good to see you here. As Dr. Joe said and Thomas. Brian from SparkCures and I have two very special people if I can. Everly and Alice, my two girls are watching this, so they're too young to know a whole lot about clinical trials, but they are learning a lot. Hey girls, love you guys. 10 years ago we started clinical trial matching for myeloma patients and there's a lot of people that know my story and we don't really have time to get into the whole thing, but my mother had myeloma and a clinical trial gave us two years with her, which was really amazing. And this is back in 2005, a very different timeframe, we know what that looked like. No CAR-Ts, no DERA, no Carfilzomib, very different landscape. But we started our partnership about a year ago, actually we kicked it off at the support group leader summit last year and it's been wonderful.

We're helping a lot of people and connecting with a lot of support group leaders and a lot of people that go to these support groups and that are connected with the IMF. Really glad to be here. Thank you.

Speaker 1:
We're so glad to have you Brian. And to fully expound on what Brian just shared with us and what Thomas is experiencing, let me tell you quickly about two critical initiatives related to clinical trials that we have at the IMF. The first, as Brian mentioned, is this beautiful partnership that we have with this wonderful company he started called SparkCures, which really helps patients find clinical trials that are relevant to them. If you go to clinicaltrials.gov that some of you may and you type in multiple myeloma, you're going to get over four or 5,000 hits and it's going to be very challenging to navigate that mass of information. But SparkCures has created this marvelous search engine that can be very specific to you. You can do it anonymously or you can put information about yourself, everything from what line of therapy you have, what treatments you've had before in multiple myeloma, and you can do all this easily at our website, myeloma.org, And you'll see a link there to be able to take you to that SparkCures search engine so that you can search.

And Brian was just sharing with me this week, and I won't put words in his mouth, I'll let him tell us more about it later in the show, but we have had thousands and thousands of patients benefit from this system and be able to search and look as to what are available to them. And many of our patients indeed have gone on to be on these clinical trials. I've been involved in myeloma research for 25 years. I have never seen a time like now in the sheer number of clinical trials and the promising treatments we have for our patients. When I started myeloma 25 years ago, most of our patients sadly lived one to two years max. Now we're seeing the average survival well beyond 12 if not 15 years. There's still a terrible disease. It is still a terrible disease. There's still sadly patients that die rather quickly with this disease.
But clinical trials drive the engine of our research so that we can discover new options for patients. So often people think, they hear clinical trial and they think, "Am I going to be a Guinea pig and am I donating my body to science? Am I doing this for someone else?" They think perhaps about the terrible history that we have of unfortunately in particular populations not providing clinical trials in the correct manner, whether it be Tuskegee or others that have really an unfortunate mark in our history, but yet we have learned over time and now have protections and careful approaches so that all can benefit from these clinical trials. Which brings me to Thomas and brings me to our second major initiative at the IMF. The first was our partnership with SparkCures to facilitate patients finding clinical trials, but we also wanted to create a program to help people learn about clinical trials with all due respect to me, not from Dr. Joe.

If Dr. Joe tells you about trials, that's one thing, but if someone who has been on a trial can share with you their experience, we think it is considerably more powerful. And we took five patients and one care partner who themselves are from communities that are historically underrepresented in clinical trials, and we brought them together for what we call the Diversity in Clinical Trials Academy. And there they shared with each other their experiences and then we were able to capture them on screen to record short and long snippets of them talking about what it was like to be on a clinical trial so that if you hear it from someone who has been through it, you hear it from a peer and not just from a doctor or a nurse or a researcher, you have the opportunity to understand a little bit more.

Thomas, let me just ask you real quick before we start getting to the questions that I'm sure Brian is piling up for us here. And please let me remind everybody, send in your questions, let us know who you are and where you're from. Thomas, let me ask you, how was that experience for you doing that academy that we did and recording your story? How important do you think it is that patients share their story so that others can learn about clinical trials?

Speaker 2:
It was very important to me because first of all, I got to learn from everybody else what they went through within their cancer journey and what they experienced and how they experienced getting on a clinical trial. With me, sharing my story with them, knowing that helping others that look like me is a positive way of getting people of color, getting people in underrepresented areas to be on trials because of some of the things that you said, the history behind clinical trials and why people don't want to be on them. But in a sense, when they see someone that looks like them that has been on a trial, they will be more susceptible about being on a trial themselves.

Speaker 1:
That's beautifully put. And the goal here is not to coerce or to push people into trials, but is to have people realize that trials are part of the spectrum of treatments that we have. When I mentioned I started myeloma 25 years ago, we really had one or two drugs. Now we have over 20 and it's wonderful. We've even just recently had another new approval of a new agent for myeloma. We expect many more in the near future. Having choice, having options that match your interests in life and your lifestyle to the myeloma that our patients have is so fundamental. Part of the goal today is to have us understand that part of that choice should include a clinical trial. Now, there are times when a trial may not be available or may not be suitable or you may not be eligible for one, but we want it to be a consideration because we have learned over years that so many of our patients are literally here today because of clinical trials.

That's enough of hearing from Dr. Joe in the intro. Thanks Brian, thanks Thomas for helping us kick this off. Now, let's get into the good stuff. Let's get us into the questions that you have. The last caveat before we dive into the questions is to remind people we always want questions at the IMF. Please reach out to us, whether it's at Myeloma.org, we have an info line that you can call. We also actually have an AI chatbot affectionately known as Myelo, who if you wake up at two o'clock in the morning because you're on Dexamethasone and you want to ask a question, feel free to ask questions of Myelo.

But we do want to use every realm of our social media, be it on X, be it on Instagram, be it on LinkedIn, be it on Facebook, like here today, use the hashtag, AskTheIMF, when you ask a question and we'll do our very best to answer it whether we answer it during a Facebook Live in one of my videos directly to you, we want to answer your questions. There are always great questions in myeloma and we're here to provide a credible source for you so that you can answer those questions. Dr. McMahon, let's begin with the first question. I'll hand you this mic, and let's dive right into your questions.

Speaker 3:
The first one comes from Tom G. from Wisconsin. Hi Tom, thanks for this. And I start off this one very specifically says, "I have smoldering myeloma. Would a clinical trial benefit me? I have been told it's aggressive, diagnosed in November of 2024 with no treatment. And I think the interesting thing is the spectrum of disease, the spectrum of myeloma, and when to consider a clinical trial."

Speaker 1:
Absolutely, great, great question. Really two questions in one. To start with Brian's editorial question to that which I think is important is that myeloma is a spectrum. We have patients who have pre-myeloma conditions, as it were, something called MGUS or Monoclonal Gammopathy of Undetermined Significance, smoldering multiple myeloma, and then active myeloma. And whether someone has pre-myeloma or myeloma, whether they're a newly diagnosed myeloma or they've had myeloma with multiple lines of treatment before, we try to work and design clinical trials across the whole spectrum. Not only so clinical trials aren't just explicit treatment for myeloma. Sometimes we do clinical trials that involve quality of life, that involve supportive care, the kinds of things that we can do to support patients through their journey. But to specifically answer our friend from Wisconsin who, if I yell loud enough, might be able to hear us beyond the microphone, being here in Minnesota today, that's a great question. I'm sorry to hear of course that you've been diagnosed with smoldering myeloma, but I'm glad it was caught before it was active myeloma.

Technically right now, we do have some options that are available to treat smoldering myeloma, but we all recognize it's a space that we haven't fully understood. And there are outstanding clinical trials right now trying to answer questions like, can we give a patient with smoldering myeloma, basically myeloma-like treatment and maybe nix it early enough that they never developed myeloma and possibly cure them? Or on the other hand, maybe we can give them gentler therapy, a single agent. We've recently had a big trial called the AQUILA trial giving single agent Darzalex or Daratumumab that maybe gives the opportunity to kick the can down the road as it were, or delay someone having multiple myeloma. When we still don't know all the answers, that's when we have to do research.
And I would encourage you to go to the myeloma.org website, use the SparkCure search engine and look for clinical trials if you're interested, because sometimes you may decide on a trial, you may decide on treatment, or you may decide on saying, "Let's continue to wait and watch closely with smoldering myeloma because of course not everyone with smoldering myeloma will develop active myeloma." Next question.

Speaker 3:
Well said. I'm going to combine two. We got Lori B. and Donna C. and they're asking the same thing. "Are clinical trials pointing MRD-negative patients into maintenance versus ACT, stem cell transplant?" And the other part of this is, "If you're in remission or if you're doing well on maintenance, what does that look like?" Or complete response, however we want to use that phrasing today, that's come up multiple times. But are we using MRD to guide those decisions and are there trials looking at that?

Speaker 1:
This is so great. Now we're merging together what is, if you will, an exciting new realm of multiple myeloma along with clinical trials. What is this exciting new realm that Dr. Joe talking about? This concept of MRD or minimal residual disease. Basically, it just means that when someone does a bone marrow test to see what myeloma is left in the body. When we know from the blood tests or the urine test that we can't detect myeloma, we go to the headquarters of myeloma in the bone marrow and can we find any myeloma? And we now have these really specialized tests that can look for tiny amounts of myeloma in the bone marrow. I'm talking about one in a million or one in 10 million cells to see if there's that tiny fraction of myeloma left. Because we believe that if we're going to cure myeloma, we got to get rid of it and get rid of all of it down to that last little schmutz.

Sometimes I say that last soldier to be killed is Rambo. We don't want Rambo to grow and multiply. We got to get rid of that last soldier. And to answer these two individuals asking an amazing question, yes, there are a lot of trials that are looking at this concept of saying, "If I give someone treatment and I get them to MRD negativity..." Sounds pretty attractive, doesn't it, Thomas?

Speaker 2:
Very much.

Speaker 1:
"And I can get them to where really there's no measurable disease." And typically it means that we sustain that for a while because we've also learned that maybe if it seems to disappear, we got to make sure it stays away for a while. There are bits that we may not be able to see. Can it then influence what we do thereafter? Things like maybe it means someone doesn't need a stem cell transplant.

We just saw a great clinical trial from our French colleagues called the MIDAS trial where they took patients who got into MRD negativity after their first six cycles of treatment. And it's a little too early to make a final conclusion, but it looks like there may be a group of patients who may not need a stem cell transplant if they've already achieved that. We may be able to give less therapy.

Similarly, in patients on maintenance, as the question asked is typically we use the word maintenance for someone who's had a stem cell transplant and they're maintaining their response by staying on longer-term treatment. We're now learning that MRD may be able to guide us to either shorten that period of time or reduce the intensity of the maintenance therapy so that patients can of course get off therapy. As I was joking with the support group leaders, my favorite treatment is [foreign language 00:17:41], nothing, zero. I love giving patients nothing, and MRD can help us bring us to that point, then it's very important. Yes, those trials are being done. Yes, we believe we'll be able to alter our strategy for maintenance therapy based on MRD.

Speaker 3:
I think great response. And the one thing to add on there is part of the clinical trial matching tool, you can go on and ask for all the maintenance trials. For anybody that's on first line, going through stem cell transplant, there are these maintenance trials we make it really easy to find. That's something to consider there as well.

Speaker 1:
Well said. Absolutely.

Speaker 3:
Jen from British Columbia. This is a good one. I'm not going to read the whole question, but essentially Jen has been provided a clinical trial that has randomized arms. One is where two of them are going to be the experimental treatment and one is the standard of care, and she's essentially saying, "If I'm in the standard of care arm, what's the disadvantage?"

Speaker 1:
Great question from my homeland of Canada.

Speaker 3:
That's why I threw it out there for you.

Speaker 1:
It's always good to get the Canadians in there. Quickly, to summarize, when we do clinical trials, there are different types of clinical trials. We often talk about different phases, a phase one trial, phase two, phase three trial. Some trials, it's just giving a particular drug or treatment strategy. Some trials you're comparing different strategies. Sometimes when you're comparing them, you may use this thing called randomization, which is really a powerful tool to help us really assess how effective a treatment is. For example, in the trial that was described here, there are three different arms as it were to the trial. A patient doesn't say, "I'm going to pick number one, or I'm going to pick number two, or I'm going to pick number three." Not even the doctor can influence it. It's done in a randomized way like flipping a coin as it were.

And that way there's no bias in the selection, and sometimes we may not even know what arm someone is in, and that way we can ultimately determine later on into the trial that those three arms of patients, the patients that have gone into those three arms have similarities. We haven't favored one arm over the other. When that's happening, a patient needs to be informed of this at the start, that you may well receive the intervention arm, as in this case you got a two-thirds chance of it happening. But let me also emphasize that third arm, we would not ethically allow a third arm in a trial unless that third arm was at least the standard of care. I don't want to offer my patients inferior treatments. When we design and implement clinical trials, that arm, whether it's a third arm or just the second arm, sometimes a lot of trials only have two arms in them, you have to be reassured that we are providing that standard of care, that your prior treatment doesn't prevent you from getting that particular treatment.

And this is such an important question because it often breaks a myth of randomization where people think that, "If I don't get the intervention arm or the new drug, then I'm going to get something very inferior." I'm going to get Thomas to comment on this for a moment too, because Thomas, you as a patient, when that discussion is had, how important is it to you that you know that when you go into a trial, whichever arm you're randomized to, you're still getting standard of care treatment?

Speaker 2:
It's very important because I'm still not lacking the treatment that's needed to treat my myeloma. Granted, a patient wants to get the experimental drug, however, if we don't receive it, then we are still getting the appropriate care to keep our myeloma stable or at rest or to treat it as needed because of the treatment that's being offered.

Speaker 1:
I think that concept is so important because sometimes people hear clinical trials, I'm sure Brian, you hear this all the time in the work that you do in clinical trials, and people think, "I guess I'm taking one for the team, I'm doing it for the myeloma community." Listen, I don't want to be too crass about this, but you want to be selfish. You want to be about you. This trial, this is your myeloma. We want to care for you. And it's important to realize that people go onto clinical trials, not just to educate the scientific community, although that can happen, not just to help other patients, although that can happen to help themselves.

Speaker 2:
To better themselves.

Speaker 1:
Absolutely.

Speaker 2:
Indeed.

Speaker 1:
Brian, let's take the next question, brother.

Speaker 3:
Next one here is interesting. This is Katie who's basically talking about her father who was offered two clinical trials. I think it's important because there's not just... We always say there's no right or wrong. The question is, "When you have these options, how do you decide and how do you go through the determining factors? What does that look like?"

Speaker 1:
Of course by then they've already gone to the SparkCures website and have decided which ones are eligible for. But joking aside, deciding about a clinical trial obviously is a very important thing. A couple of factors that I would throw in here. Number one, it's like making any other decision in myeloma. It has to be what we call shared decision-making. I don't as a doctor look at a patient say, "This is the treatment, you'll take it and you'll like it." That is never going to be the right approach. We want to match the treatment to the patient's preferences, the patient's desires, the nature of the patient's myeloma. That requires a relationship and a conversation with the healthcare team. And so often when we're doing clinical trials, there's more than just the single doctor involved. There's often a research nurse and various other people involved.

You have the opportunity to have that discussion. You have the power as a patient to ensure that the people you want to have there should be there. I think this is particularly important that you remember whether it's a family member, whether it's a friend, whether it's a religious leader. I work in Arizona. I once had a patient who was hesitant to go on a trial until his tribal leader was willing to give the okay. And we engage the tribal leader. This kind of discussion is really important.
The other point I'd make about it is it's wonderful that someone can have more than one clinical trial available to them, whether it's two or more, or even just one trial. Make sure you ask critical questions like, "Is this a randomized trial? What is it being compared to? What are the expected side effects of this? How many times am I going to be coming in? What other tests are going to be a part of the trial?" Because sometimes with clinical trials, you may have a few extra blood tests or even bone marrow tests or x-rays. Those are the kinds of things that we want to look into because we want to make sure we're watching our patients really, really carefully. Great question. Let's take another one. We have about six minutes left, we'll try and go into the speed round now as we answer questions.

Speaker 3:
I have one for you. This is a question that I have. We know that there are reasons that people can join a clinical trial, the fancy term is inclusion-exclusion criteria. When do you think that MRD is going to be used for measurable disease requirement instead of an M spike or free light chains? When and if do you see that occurring from a trial standpoint?

Speaker 1:
Wow, that's an advanced question. Getting to Brian's central point, right now, most clinical trials and understandably so, you have to have a measurable amount of disease. We typically say in myeloma, we generally measure people by either an M spike or a light chain. You have to have enough of something to show a difference. If someone's got just a tiny, tiny amount of M spike, how do I know if the treatments really worked, if I can't measure it? But now that we're good at measuring tiny amounts of disease in MRD, I think it is going to herald today that we might be able to do more of that. I do actually think that we're going to be engaging MRD to more inclusion criteria, depending, of course, on the scenario, much more so.

Speaker 3:
Which I think is important too, because there are people that can't get in because their M spike doesn't get to the one or 0.5 or the free light chains aren't at 10 or a hundred depending on what units they use to make this even more confusing.

Speaker 1:
To add to that, sometimes people have myeloma that we can't measure in the blood or the urine, and we can measure it on an x-ray, what we call extra medullary disease. In fact, I think it's been part of a greater concerted effort to be as inclusive as possible in clinical trials. We can still keep the integrity of showing a change in a trial, but we also can definitely include more patients in the trial.

Speaker 3:
Well said.

Speaker 1:
Now, I know that there was a really great question and I'm going to read this question actually. It came from one of our wonderful advocates. Dana writes and she says, "I have a question for Brian. Brian, what makes the SparkCures clinical trial platform unique? Each of the myeloma organizations now seem to have their own trial search platform. How does SparkCures stand out from the rest?" And by the way, she puts a little PS, "Personally, I believe SparkCures is an exceptional platform among these, and I routinely share it with my Facebook groups." It's lovely. Thank you for that comment. But Brian, what makes SparkCures different?

Speaker 3:
Thanks for that. And thanks Dana for sending that over. And I think that the biggest challenge when we think about this, if we pull back to the 30,000-foot level, the biggest challenge we face is awareness. Letting people know that clinical trials exist. Part of this question is that there are a lot of advocacy groups out there, and everybody's trying to raise awareness. That is a win. I always say to people, you had mentioned, I don't typically say the website, but the government website, which is clinicaltrials.gov. If you can go there and understand it and find a trial, more power to you, we would never take that away. I think just like there's myeloma specialists everywhere, try each one, see what it looks like, see who fits into your care team the way you want to be cared for and the way you want to have these conversations.

But maybe to answer very specifically too, though, I think we've been doing this now for 10 years, and we come from a caregiver and a care partner background. I know what it's like to look for these trials, and you need really, really good data. Number one, we start with really good data. We have to start going outside of clinicaltrials.gov, going to the sponsors. We need to really understand what these trials are looking for, and we also need to understand what's going on with the patients, what treatments did you have? What are your labs? And that's how we're able to go through and navigate this. We're really conservative. We do matching every single day, every time something changes.

There's a lot of quality to this, but we're also very conservative. We are not the clinicians. We're not the Dr. Joe's and the amazing docs out there. Our goal is to have a better conversation or help you to have a better conversation with your care team. And we can do that on one-on-one trial navigation. There's a lot of things that we do. I think wherever you're getting that information, go do it, but I'm of course biased towards what we do in our work here. But thank you, Dana, for the question.

Speaker 1:
Bias aside, I think you provide a comprehensive, yet individualized approach, and that's what I tell my patients, that it really can be helpful. But before we wrap up, I want to ask Thomas or give Thomas the mic one last time and say, Thomas, if you have the chance to talk to the masses out there, because I'm sure there are tens of thousands of people on this Facebook Live, no pressure. But what would you want people to know about clinical trials before we close off?

Speaker 2:
The most important thing that I want everyone to know, that clinical trials is not a death sentence. The fact that you have been asked to be on a trial doesn't mean that you are at the end of your treatment journey. That's the most important thing that I want everyone to know, because that is not true. Clinical trials can help where you're at right now versus the thought of you're on your last leg.

Speaker 1:
I love hearing that because as we commented earlier, we have trials right across the spectrum, and trials have had such a huge impact. I'm sorry we haven't been able to get to all of the questions. We tried to cover as many as we could, but please keep sending them in. We keep track of all of them. We hold them, as I mentioned through our Ask the IMF program so that we can answer your questions in the future.

Want to take a quick moment and thank the sponsors that we've had for today to support this work in our Facebook Lives, AbbVie, Johnson & Johnson, Pfizer, and GSK. We appreciate your support in this. And most importantly, I want to thank all of you for attending today. The fact that you would attend, whether you're watching it right now live today, or the recordings, we know thousands of people watch the recordings, you are already taking a step in the direction of understanding and participating in clinical trials, which we trust is going to be helpful in your myeloma journey or for those that you're caring for. Let me quickly thank Thomas. Let me quickly thank Brian for being my friends, for being fantastic participants today. Thank you again for joining us. Dr. Joe signing off on Facebook Live from the International Myeloma Foundation.