Multiple Myeloma Q&A: Why Diagnosis Can Take 12+ Months, MGUS, Costs & FDA News 

Dr. Joseph Mikhael: 

"Why did it take over 12 months to diagnose my multiple myeloma? How could I develop myeloma with perfect checkups and regular visits to my primary care doctor?" Hi, everybody, Dr. Joseph Mikhael here, chief medical officer of the International Myeloma Foundation. I have the privilege of hosting a live Q&A on facebook.com/myeloma, where I try to answer in real time questions from our patients and from their care partners. Of course, I can never get to all of them, so here's a chance for me to try and catch up and answer some of these questions. 

Furthermore, people can submit questions through all of our social media channels using the #AsktheIMF. Whether it's X, or Facebook, or Instagram, or LinkedIn, we want to be here to answer your questions. Our next Facebook Live will be on Saturday, July the 19th. This will be a very special one because I'll be joined by Thomas Goode, a patient with multiple myeloma, and my good friend, Brian McMahon from SparkCures, will be addressing the issue of clinical trials, and you'll have the opportunity to submit questions in the comments, and we'll answer as many of them as we can. Make sure to follow the International Myeloma Foundation on Facebook so you don't miss our next live Q&A. 

All right, let's dive in to these great questions that come to us. The first one is from Elizabeth. "Why didn't my doctor catch my myeloma symptoms sooner? It took a year to get diagnosed." Well, first of all, Elizabeth, I'm sorry to hear that it took so long. 

The sad reality is on average, every myeloma patient sees their primary care doctor three times with signs and symptoms consistent with myeloma before the diagnosis is made. That's even longer in African American and Latino American patients, and a lot of it has to do that the signs and symptoms of myeloma are usually pretty general, things like fatigue, things like pain, things like maybe a low hemoglobin if it was tested for. So many different diagnoses can cause those symptoms, that it's often hard to make the immediate connection to multiple myeloma. That's one of the reasons the IMF is so committed to this, and through our M-Power program, we're working hard to educate primary care providers so that they can recognize those signs and symptoms early on and catch the disease earlier. Let's move to another question here from Denise. 

"Could a simple blood test save lives for myeloma? If myeloma always has early warning signs, why aren't blood tests included in annual checkups?" This is a great question, Denise, and something that we're working very hard to try and figure out in multiple myeloma. First of all, the usual blood tests that people get, which typically includes their CBC or their complete blood count, maybe their cholesterol levels, some of their electrolytes, like their kidney function, those kinds of things are routine. The more specialized testing for myeloma is not a routine test because it's not been proven as a screening tool in big populations to actually make a difference. 

But of course, we've just completed and continued to work on a huge study in Iceland, the iStopMM study, where we've screened over 85,000 people in Iceland to determine, "Should those kinds of tests be in our annual or every two-year checkups to determine if that is actually going to make a difference?" So we're working on it, Denise, and I hope we have an answer for you in the near future. Here's a great question from Tomaso, "If you knew cancer was coming, why would you wait? Why do doctors take a watch-and-wait approach with MGUS, or Monoclonal Gammopathy of Undetermined Significance? Why not treat it before it becomes multiple myeloma?" 

Well, Tomaso, this is actually a fantastic question. We are learning, of course, in cancer in general, that the earlier you catch it, the better that the treatment can be because we can reduce the burden of the cancer when it's smaller. That being said, MGUS is a unique situation, where 5% of everybody over the age of 40 likely has MGUS when we look for it, and more importantly, the vast majority of those people will not develop myeloma. They'll just have this little bit of protein that's there that will never hurt them. So our effort and work has been trying to identify who has the form that is going to grow into multiple myeloma, which is why we follow patients with MGUS, and then they go through a period of what's called smoldering myeloma if they're going to become active myeloma. 

And that's the area we're working on very intently to determine, "When should we intervene, and how should we intervene in smoldering myeloma so that it does not become active myeloma?" But it wouldn't be easy to treat all sorts of people. In fact, we would be over-treating a large number of patients if we treated everybody with MGUS, but as our tests become more sophisticated, as our tools become more predictive of who is going to develop myeloma, we can intervene earlier. Let's go to another question here that says, "Struggling with treatment costs. Is there any financial assistance or foundation support for multiple myeloma patients who can't afford treatment?" 

Well, first of all, I'm terribly sorry to hear this. We know that the treatments in myeloma can be inordinately expensive and a tremendous burden on patients and their families. I would encourage you to reach out to the IMF, call the Info line. We are very much connected to so many different resources around the country based on where you're living, what your experience has been, where you have worked before. There are lots of different ways and options for patients to try and reduce that financial burden, and we want to do everything possible to help through this because it's enough to have multiple myeloma, and all the physical challenges and the psychological challenges that come with it, let alone the financial challenges, and we want to help you through this, and we'll do our very best to support you through this journey. 

Here's another question from Avani, who says, "I had no symptoms, no red flags, then came the diagnosis. Is it possible to miss early signs of myeloma? I had yearly checkups and still wasn't diagnosed until it was advanced." Well, again, Avani, I'm so sorry that this happened. The challenge of multiple myeloma is often, it happens, if you will, under the surface, where people don't feel that that protein is growing in their blood. 

They don't feel that it's starting to thin their bone, and that could even still look normal on our usual follow-ups, and our checkups, and our physical exams, and even most of bloodwork can be continually normal until all of a sudden, it reaches a point that it breaks through the surface and becomes severe. This is another reason why it's so important for us to raise awareness of multiple myeloma, so that even the earliest signs and symptoms can trigger people to test more carefully for myeloma to be able to have the conversations with their healthcare team so that we can catch it earlier. But sadly, there are situations like this where people will not have any clear warning signs, and myeloma still appears. Here's a great comment and question that says, "Smoldering doesn't mean harmless. What does early intervention really mean for smoldering myeloma?" 

"Are we talking about treating it before symptoms appear?" Well, Heidi, great question, and the answer is partially yes. So there's this spectrum, as we've described, of MGUS, or Monoclonal Gammopathy of Undetermined Significance, which really just means someone has this little bit of abnormal protein in the blood, but it's at such a low level we wouldn't want to intervene. And then, there's active myeloma, where we really want to treat, and then in between is smoldering multiple myeloma, where patients, by definition, aren't having symptoms yet. Their organs are not damaged yet by the myeloma, but we know that that can happen. 

Even within smoldering myeloma, we typically divide it into so-called low-risk or standard risk smoldering myeloma, and high-risk smoldering myeloma, where in the high-risk group, these are patients that on average, at least half of them within two years will develop active multiple myeloma. And that's the group we're looking at trying to understand, "Should we partially treat them? Should we fully treat them, and will that make a difference in the long run in either delaying myeloma, or possibly being one of the ways that we can develop a cure?" And that's why so much is going on in clinical trials right now for that high-risk smoldering multiple myeloma. So here's a great question from Tim, "Why does myeloma return, and can we stop it?" 

"What triggers a myeloma relapse? What research is being done to stop it from coming back?" Well, great questions, Tim. Absolutely. In general, myeloma comes back, because even though we treat it well, and even though by the tools we have, it may look like it is gone, there is still some residual myeloma hiding somewhere, and that myeloma slowly grows, and grows and grows, and eventually causes a relapse. 

That's what we think happens in the vast majority of patients that have a relapse, and yes, there is a lot of research going on to try to prevent that relapse primarily by getting rid of it the first time, that we now have such sophisticated testing in a bone marrow, for example, I can look to see if I find one cancer cell in 10 million other cells. That's how sophisticated we become and precise we become. And we believe that if we can get that down to literally no measurable myeloma, we can, if not fully prevent it, at least delay it for a long period of time, and that's the kind of work that we're doing in our research initiatives of the IMF and what the myeloma community is focusing in on, and indeed, we want to prevent it. We don't want just to have it to be an inevitability that the disease will relapse. Here's a great question from Rachel. 

"Has there been any progress in preventing multiple myeloma this year?" Well, Rachel, you speak right to the mission of the IMF, where we want to improve the quality of life of patients as we seek prevention and a cure, because it would be great to actually prevent it from happening, and there are some studies going on, looking in different ways. I wouldn't say that we found a solution yet, or there's been a major breakthrough, but there's been fascinating work in different areas. One work in particular has been from our colleagues in New York City at Memorial Sloan Kettering Cancer Center, where they're studying if nutritional interventions can reduce the movement from MGUS or smoldering myeloma to active myeloma. We know that our guts and all of the flora, as we call it, or the bugs that live within us, have a lot to do with our general health, and maybe something with a dietary change or an intervention may have an influence on that, because we know that perhaps the only truly preventable feature in multiple myeloma or a risk factor is obesity, that we know that there's some connection, and as we study it, we want to understand if nutritional interventions can make a difference. 

But believe me, we are working very hard to try and see if we can prevent multiple myeloma as we try to discover what really causes it. In the vast majority of patients, we really don't know the cause of multiple myeloma. We know that certain exposures like Agent Orange, like firefighters who have been in many fires, and some other chemical or toxic exposures can be associated with myeloma, but that still accounts for a very small number of our patients with the disease. Here's another great question. "What does the FDA ODAC vote on the AQUILA trial mean for patients with high-risk smoldering myeloma? What is the tangible effect of this?" 

Well, Stuart, you're very much in the know here, and just quickly, to get everybody up to speed, the AQUILA trial was just presented and published, where patients with high-risk smoldering multiple myeloma, so these are patients that have features, not only have smoldering myeloma, but features that seem to indicate that the diseases could grow more quickly, were randomized to two treatment choices, either given single-agent Darzalex or Daratumumab for three years, or just close observation, which is typically what we do now, and the study was quite positive. It showed that those who had the Darzalex had a longer time until they developed myeloma, and even indicated the potential for an improved overall survival. Now, before the FDA would approve such a drug, they call what's called an ODAC, or Oncologic Drug Advisory Committee, that look at this a bit more carefully to say, "Is the risk versus the benefit worth it here? Is there a benefit in doing this?" And then there is a discussion, and as you noted, there was a vote that was positive that supported the use of Darzalex in high-risk smoldering multiple myeloma. So now, it is in the FDA's hands. It's not been formally approved yet, but that ODAC was a critical step in moving this to an option that would be available to patients.

Well, that's all I have time for today, but thank you for these amazing questions, and please don't hesitate to reach out to us. Follow us on Facebook, when I do our Facebook Lives, where we answer questions through any of our social media channels using the #AsktheIMF, or just call us on the info line, or if you want in the middle of the night to have an answer to your question, try Milo, our AI chatbot that's available to you at myeloma.org.