How can you actively participate in your myeloma care and treatment decisions? Engaging with your healthcare team is essential and may lead to better overall outcomes. In this program, Dr. Rafael Fonseca provides tips for how best to advocate for yourself or a loved one, as well as tools for making treatment and care decisions.
Dr. Rafael Fonseca is the interim director of Mayo Clinic Cancer Center and serves as the director for Innovation and Transformational Relationships at Mayo Clinic in Arizona. Learn more about Dr. Fonseca here.
Hello and welcome. I’m Katherine Banwell, your host for today’s program. Today we’re going to explore how to engage with your healthcare team when diagnosed with myeloma, and we’ll discuss the patient’s role in care decisions. Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. Let’s meet our guest today. Joining me is Dr. Rafael Fonseca. Dr. Fonseca, welcome, and would you please introduce yourself?
Dr. Rafael Fonseca:
Yes, of course. Happy to do that. Thank you very much, Katherine.
I am a hematologist/oncologist, but I specialize in the area of multiple myeloma. I work at the Mayo Clinic in Arizona. I currently serve also as interim executive director for the Mayo Clinic Cancer Center that is at large across the Mayo Clinic enterprise. But at heart, I’m a myeloma doctor and I love to take care of myeloma patients. I devote my research and the rest of my academic activities to the field of myeloma.
Excellent. Thank you so much for joining us today. Let’s start with a question that’s on the mind of many of our audience members. We’re hearing that the COVID-19 vaccine is safe, but how effective is it for myeloma patients?
Thank you. I think that’s a fundamental question. It’s hard to know precisely how to gauge effectiveness when it comes to vaccination because historically, we know that is done by measuring antibodies and there’s a number of publications that are addressing this.
The concern has been two-fold. One is that because the disease itself is something that starts from the person’s immune cells become cancerous, that perhaps that would prevent them from having a very good response. Number two, and perhaps more importantly, will the treatments that are used for myeloma, etc. or lymphoma, can they interfere with our ability to mount an effective immune response? I think the response is mixed right now. I think I tell all my patients the upside is much better than the downside. I think we have a good record now of the safety of this product. I encourage everyone to get their vaccination.
I think it’s important to discuss this with your healthcare provider because sometimes people say, “Should I stop a little bit so that I can get a better response?” While it’s theoretically possible, we don’t want people to stop treatment if they don’t have to do that. Just my very last quick comment, the good news is that the community transmission is clearly going down as more and more people have participated in the vaccination.
We have more people who now have participated in this level of immunity that we have in the community. Hopefully, for patients as well as for their families, the risk of contracting this will continue to decrease.
Yeah. We can only hope. Well, let’s learn a little bit more about the disease itself. Dr. Fonseca, to level set with our audience, can you help us understand myeloma?
I’m happy to do so. Multiple myeloma is a cancer form of the bone marrow that arises when the cells that under normal circumstances protect us by the formation of antibodies. These are called the plasma cells. They become malignant. Myeloma is the last stage of a process where a plasma cell can go through a benign tumor or benign phase, if you may, something we call the monoclonal gammopathy, which by the way is quite common. About two percent of people over the age of 50 have this abnormality. Think of it like the colon polyp, a precursor condition.
There’s an intermediate stage that we call smoldering multiple myeloma, which is just more growth, but not quite at the level that it creates problems for the individual.
Then lastly, what we just simply call multiple myeloma, and that is when the growth of those cells becomes of such magnitude that a person starts having problems or starts having symptoms related to that. These cells live predominantly inside the bones in the space we call the bone marrow. They can do a number of things that actually lead to the symptoms and to the clinical presentation. As they grow in the bone marrow, they take some of that real estate.
A person may experience fatigue and that is because they have anemia.
The myeloma cells are also very characteristic because they can erode into the structure of bones, so destruction of bone is another feature that we see in patients with myeloma. That can be either seen on x-rays or sometimes people will present with symptoms related to bone pain or discomfort with movement or weight bearing. Those are signs that we look for.
Lastly, the myeloma cells product proteins and some of the fragments of those proteins can be damaging to the kidneys. Occasionally, people will present with decreased kidney function and sometimes outright failure of the kidneys. Those are the common presentations. It is a disease that mostly affects people in their 70s. It is not something that you can detect through routine testing; it’s just indirectly we start seeing abnormalities and then we do the right testing. If anyone is hearing this, of course, they need to have a detailed discussion with their own provider.
Of course, yeah. When a person is diagnosed with myeloma, they usually have a whole healthcare team. Who is typically on that team?
Absolutely. Let me start by saying the key to the successful management of myeloma is to have a well-organized team. It’s a disease that requires an integrated approach that usually brings around the patient a physician.
As part of my team, we also have advanced practice providers. We work with nurse practitioners that help us do the longitudinal care of patients. We have the nursing team. Every time I meet a new patient, I make it a point to bring my nursing team into the room so they can put a name and a face together, as patients will be interacting, of course, with a nursing team through the portal and the various visits. We have a team that is in charge of the chemotherapy administration. That is usually a separate a nursing team that is in charge of the administration of the medications. But we really don’t stop there.
We have pharmacists who help us review the medications for our patients. Very importantly, we have social workers that help us address psychosocial needs, as well as some of the practicalities that become inevitable when one deals with a serious diagnosis like multiple myeloma.
Yeah. Lately, we’ve been hearing this term, “shared decision making,” which basically means that patients and clinicians collaborate to make healthcare decisions, and it can help patients to take a more active role in their care.
I’d like to get your thoughts, Dr. Fonseca, on how best to make this process work.
We are very fortunate to live in this time of medicine, where ultimately, we recognize that the patient is the person expert. It is the patient decisions that should drive what is to be done in a situation. Whenever I interact with patients, I tell them, “Listen, I’m going to be like your counselor. I will provide you with options of what I think is reasonable. I will go to different degrees of effort in trying to convince you one way or another for a particular intervention. But at the end of the day, I only do a good job if I present you with the options and the pros and cons of those various approaches.”
I weave that into my language on every single conversation we have with patients. I think we’re way past the time where a physician would come and say, “This is what you’re going to do,” or “This is what will happen.” My language always includes, “I would recommend this.”
“I think the next best step for you to consider would be X, Y, or Z.” But ultimately, I look at patients and not infrequently at the person next to them, a family member or a close friend, and I say, “You’re the boss and with the person next to you providing additional support, comment, and guidance, we can together reach the best decision of what should proceed.” I think we’re incredibly fortunate because patients have access to sophisticated information, especially patients that have serious conditions such as would be cancer and, in my case, myeloma.
As an example, when I work with general internal medicine residents that work with me learning about hematology, I sometimes tell them, “You’re gonna walk into a room. Are you gonna be seeing what I say, this is like a tennis match between professionals. Are you gonna see the level of questions that patients are going to be asking me? They’re going to be asking me about the latest study that was presented at this meeting and the P value and this and that.”
“I can guarantee you that you would not have the tools to be able to address all those questions, simply because there’s such an in-depth understanding of the disease.” I realize this is not everyone. I’m giving you an extreme example. There are individuals that need additional support, more resources. But just to interact with someone who has such commitment to understand their disease and to help us by that understanding make the right decision makes my job so much more rewarding.
What do you think is the role of a patient then in their care?
I think it needs to be … I’m describing in some detail and there’s a lot to unpack there. Of course, patients are dealing with a very serious diagnosis. It’s okay to have periods where they are in a pause moment and they’re reflecting of what their facing, and that they can gather information from close family members.
I think we, as providers and the medical team, need to deliver a message that provides clear options for them as far as what the best next phase of their treatment or their management might be, including observations or supportive care. But the patient ultimately is a person who has to make that decision. I frequently get the question, and this is not surprising, and it happens all the time. A patient tells me, “What would you do if this was a family member?” I always tell them, “I always talk to you as if you were my family member, as if you were my brother, my mother, my father.
So, I try to live deeply to that fiduciary responsibility I have to your well-being. I recognize that there are circumstances, and that’s part of the finesse and the art of medicine, that I have to help a little bit more walk you through that step. Sometimes, it’s just human that one may want to say, I just want to disconnect. Maybe I’m not the person that wants to go and read in detail. But perhaps I have my daughter or my son who are helping me and understand better where things are.”
I think one of the key aspects of my role is to make sure that I have a sense that the person has a good understanding to be able to make an informed decision. At the end of it all, if the person decides to proceed in such way that doesn’t necessarily align with what I’m trying to do, I’m deeply respectful of that choice. I will go to extra lengths. So, if someone is foregoing treatment, when I know their treatment has a high likelihood of improving their quality of life, relieve a symptom, or improve survival, I don’t think I would do a good job if I don’t present why that’s so important. But ultimately, it is the patient’s decision.
Related to what you’ve just been speaking about, we have a question from the audience. This one is from Sarah. Her question is, “What advice do you have for caregivers? How can I be supportive during appointments?”
That’s a great question.
I have experienced this both as a physician, as well as a caregiver myself to someone who has had a cancer. I think I’m gonna say that there are several roles that caregivers play. Some of them are obvious and I’m gonna call them practical or perhaps even pedestrian, you know, organizing the activities of every day. That’s important, but a lot of people can do that. The second role is to be in assistance for the knowledge that is needed for some of this decision making. Sometimes patients can be overwhelmed, and we need some support and some vetting and peer process from a trusted and loved person so you can go through that.
That is very helpful, but what is essential, and the number one thing is you are first and foremost the loving family member or friend of that individual who is living through a very profound human experience. I think the first role of a caregiver has to be to express that role.
I, myself, reflect on moments where perhaps in a quick, reactive way I wanted to solve some of the immediate practicalities and what was needed most was a direct support. Even if I face a situation today, if I was, again, a caregiver for someone with a serious diagnosis with cancer, I would start with that priority. Number one, you are the support and the loving person. Number two is I will try to provide information. And number three, hopefully you can help with meals and the driving and what have you. But there’s many more people who can come and help in that regard. Not a lot can do the first part.
Right, absolutely. Yeah, those are excellent points. Let’s talk about treatment goals. What are the goals of myeloma treatment from a clinical perspective?
I’ve been very fortunate, also, to live through this era when we have seen a plethora of studies and new drugs being approved for the treatment of myeloma.
When I first started, I used to say no one wanted to do myeloma because we didn’t have good treatments. People wanted to study leukemia, lymphoma. It just turns out that this is probably one of the most vibrant areas of hematology from a science and from a clinical research perspective, of course. If I see young patients who have multiple myeloma, I have essentially two goals. The first one is to induce the deepest possible response I can do so in a safe manner. I also repeat, “in a safe manner.” But I really have the goal to try to induce the deepest response possible because that has translated and continues to translate, and in many ways proven to be associated with an improvement on their longevity and the time we can control the disease.
And it leads me to second goal, and that is that I firmly believe there is a subset of myeloma patients that are cured from their disease.
Now, this is possible because of the availability of these new treatments. I will only be able to say that in 10 and 15 years from now, when we have monitored patients for a long period of time, and we have been able to see that became true. But by all indicators, we have patients that are living many, many years without the disease coming back. I think that would be important. Now, we have patients that with more advanced age sometimes it’s difficult to propose some of the most intense form of treatments like stem-cell transplants.
We don’t do a lot of that in individuals over the age of 72 just because the toll that it takes on a person is very high, and the risks become higher. But still, in that population, providing the best treatment possible becomes a goal because I think more and more, we’re seeing patients in that age category that can start to get close to what normal life expectancy would be. It’s not there. It’s not perfect, but you start to get close. Lastly, if someone asked me, I have that balance between quantity and quality, the good news in myeloma, if you do it right, quantity and quality go hand in hand.
So, effective treatment provides symptom relief and provides durability of responses.
That’s excellent. What other factors do you consider when determining a treatment approach?
The human experience that comes to the bedside as we consider treatments is so multi-factorial and multi-complex that all that needs to be brought into consideration. Whenever I walk into the room, I tell residents usually the medical part can be resolved pretty quick, but we’re reading how much we can communicate? What’s the level of understanding? What do I understand about the support system for this person? Is there someone who can drive to the treatment center? Is there someone perhaps whose other medical conditions would create certain challenges in how they’re gonna be treated?
This person is telling me they do daily hikes for four miles. Well, that’s different from someone who I see comes into the clinic and has to use a cane. We try to integrate all of that information to make the right decisions. I’ve made a lot of my career in the early years working and showing how, for instance, genetic factors are important. I’ve come to realize later in my career and through some of the very elegant work that other colleagues have done, that these factors are just as important in determining the ultimate outcome of patients. Whenever I talk about that clinical experience, there’s two things I always tell the residents.
I use the residents a lot because I think it’s a good example of how we aspire to interact with patients. Number one is every single encounter is a final exam. You have to put your best foot forward. Every single encounter should be considered a final exam. Number two is when I walk into that room, there are three things I do, particularly the first time I meet a person.
Number one is connect, right? We cannot have a conversation and I’m not gonna be able to move forward unless we have a human connection and I have gained the trust of the patient and the family members that are there. That’s number one. The second point is decide. That is usually okay, we’re gonna do this treatment or that. That is a small part. Most of the time for me, that’s a very small fraction of the time and of the mental energy that I consume. There’s cases that are more complicated, but most of the time it’s pretty straightforward. So, it’s connect, decide do very small, and then on the other end is explain.
So, that’s how I can connect. I propose we do this, and then why we are gonna do it and what can you expect. If you can do those three things, I think that goes a long way in establishing a fruitful and a productive relationship with a patient and their families.
I would suspect that you also take into consideration the patient’s health, their age, maybe test results, side effects, things like that?
Of course. So, we look at the medical record and with the advent, of course, of the electronic record and all the tests that we do, our consideration is quite complex. We have to look at all those factors, and the age, and comorbidities. It’s rare that we would take one factor alone that would trump everything else. We usually have to integrate the information. The same is true when we manage myeloma patients and we’re monitoring their protein levels and their response to treatment. I tell patients, they ask me, “What would you do? What’s the magic number for this or that?”
I say, “It’s a little bit like you’re flying a Cessna plane and you have all these dials in your dashboard, and that’s how we manage the situation is the integration of all of that information.”
Right. Can you help us understand, Dr. Fonseca, how test results may affect treatment options?
Sure. Happy to do that. In myeloma, we are very fortunate in that we have, and it’s not the topic for today, but we have the best biomarker that exists for any cancer. That is that we can measure the proteins that are associated with the growth of the cells. We have multiple tests that we can do. We do them in the blood and we do them in the urine. They’re simple tests that have been done for decades now that allow us to monitor how a person is doing with regards to their disease. I use the following analogy. Myeloma cells live inside the bones, as I mentioned, in the bone marrow.
They don’t come out into the blood. So, we cannot measure them. Indirectly, we can measure how many they are and how they are behaving by measuring this protein. I use an analogy of imagine you’re walking in a street, and you see smoke coming out of a building. There are two things you can do. First is you diagnose that there is a fire inside the building, right? We see that with myeloma by measuring these abnormal proteins.
Then as a firefighting team comes on, you can gauge whether they’re making progress or not by the amount of smoke that comes out. That’s exactly what we do when we monitor myeloma. We monitor the M-Spike, the serum free light chain, the urinary proteins. That’s how we make those determinations.
At the same time, we do that, we have to look indirectly at the rest of the body. We have to look at the kidney function. We have to look at the blood counts. We have to look at the hemoglobin and the red cell count because that can (A) start on the wrong foot because of the myeloma itself, but (B) can also suffer as a consequence of our treatment.
It is, again, that idea of having the multiple dials in the dashboard that allow us to reach our practice. We have to be adjusting. So, if we measure the proteins and we’re doing great, but then at the same time we see we’re suffering in blood counts, and we may need to adjust those as we provide supportive treatment. If we don’t see the proteins go down, then that may mean we need to change to a different form of treatment or that the person is unfortunately a refractory or relapsing to something.
So, that’s how we integrate the test results into our management.
What sort of questions should patients consider asking about their treatment plan?
I think it’s important that patients understand a few things. They can be described in multiple ways. Number one is, of course, what? What is it that is being used? I think that includes a description of what to expect, the practicalities, the names of the medications, their side effect profile, and what to report when you use those medicines. I think that’s very important because if you’re empowered with that information, you’re gonna be better off as you react for symptoms that may come along. I always tell patients when you have a cancer diagnosis, your self-awareness goes through the roof because we’re gonna be paying attention to everything, every skin change, every pain we have.
So, I think having a bit of that proactive discussion becomes important as they think about the treatments that they want. I think the how-to on the practicalities are very important. The best where the nursing team and the pharmacists help us a lot too. Do you take the medicines at night? Do you take them with meals? Is there something that you shouldn’t be mixing? How much time would it take for me to get a refill? It’s different to get a medication from a specialty pharmacy versus your down-the-street Walgreens. So, all of those things are important that patients, again, participate in the understanding.
If not them, at least the caregivers that are a part of this team. I think it’s important that patients ask also some brief descriptions of (A) the biology of the disease. If I have myeloma, what type of myeloma do I have? Does that matter as far as what treatments I’m going to be using? What treatment options may be available to me because of my specific subtype? We have subsets of myeloma that have options that are not available to others.
Also, I think it’s important that patients also ask a sense from the physicians as to where they are. I’d like to describe this a little bit more. Sometimes, patients ask us specific questions about, am I in a complete response? Am I in a very good partial response? What is a PFS? Those terms work very well when we talk about clinical trials, but they don’t necessarily describe in a great way the situation for an individual patient. I’d use a lot more objectives than I’d use technical terms when I describe where patients are. I say, “You have an excellent response. You have a very deep response.”
Then I’d provide more details if they want. “Yes, you’re MRD-negative at 10 to the -6.” But sometimes I find that it’s harder for patients to understand where they are if they completely focus on the staging system or the response criteria, etc. Because maybe a VGPR, a very good partial response, doesn’t sound very good.
But then you can be in a very good partial response for 15 years and it doesn’t matter. You my want to be in an MRD-negative status, but you still have a good outcome. That’s why the general description of the status by a physician becomes important.
Do you think patients should get a second opinion consult with a specialist?
In general, my answer is going to be yes. This is not self-serving. I think myeloma has become so complex that trying to integrate at least once, or if not, in some infrequent basis, an opinion of a myeloma specialist becomes important. This is no one’s fault. If you’re a community oncologist somewhere where myeloma represents only a small fraction of your practice, I can guarantee you, you cannot stay on top of the literature. I cannot stay up with everything that goes on with myeloma, even though that’s what I do 100% of the time.
I get an email every week with all the articles, all the publications, and I have to integrate that. I have to think, okay, does this matter or not? I go to the professional meetings. I see all the abstracts and I still feel like I’m missing out. How could you do that if that is only a small fraction of your practice? I’m sure that the same applies for other cancers, breast and colon. You can’t move. You cannot uproot yourself and leave your community and your family, but I think there should be ways by which patients at least have an opinion from someone who has more expertise. Fortunately, there are many centers across the nation now that have that expertise for the management of myeloma.
Dr. Fonseca, we have a question from a newly diagnosed myeloma patient. Barbara says, “I am just about to begin my first myeloma treatment. What can I expect?”
Thank you, Barbara, for the question. I think if you start on treatment, first of all I hope they already went through a good description of what the treatments are, the frequency by which you’re gonna have to go to the center, and also what are the toxicities to look out for.
One of the most common toxicities that we face and one of the most challenging parts of initial treatment is the use of steroids. So, we use dexamethasone as part of every single regimen we use for myeloma. I tell patients, “Dexamethasone is a simple drug at first glance, but it’s oftentimes the most complicated part of treatment.”
The human brain works at triple speed when you’re on dexamethasone. So, it’s hard to sometimes be able to sleep properly. People can become anxious and even the sweetest person in the world can become a little bit edgy on dexamethasone.
I always say Mother Teresa on dexamethasone would be an edgy person. Just be patient. Work with the team. Just know that on the other side of treatment there is a return to normal life.
Our goal as we embark on treatments and, for instance, is I see patients that are going to go through transplant, I tell them, “Our goal is you finish, you recover, and you go back to your life. You back to work. You go back to your family, your kids, your sports.” That’s really what we strive for when we treat patients with myeloma.
Yeah. Once on therapy, how is the disease monitored and how do you know if the treatment is working?
Well, fortunately, we use the same markers. Once a person is in therapy, we will be monitoring. We monitor at least on a monthly basis of those myeloma protein markers. Once a person reaches a great level of response, sometimes we complement that with an analysis of the bone marrow. Of course, it’s more invasive, so we don’t like to do a lot of them, but we do them as needed. As we go forward and monitor patients, we will be looking for signs that those proteins remain in a low level as stable as an indicator that the disease is under control.
Now, if I saw someone and then I start seeing that there’s an increased concentration of those proteins or we see something else clinical, we might need to do a little bit of a regrouping and test again in great detail to determine if the person is experiencing regrowth and the disease is so-called relapsed.
Why is it so important for patients to speak up when it comes to symptoms or treatment side effects?
Well, that’s a great question. If you don’t speak about them, we don’t know about them. It seems very obvious, but then we cannot make the proper adjustments. I’ll give you a couple of examples. I already talked about dexamethasone, but a common drug we use is something called bortezomib. Bortezomib is a proteasome inhibitor.
That’s a mouthful, but it’s one of the key type of drugs we use. It’s given as an injection under the skin. Not to be confused, by the way, with daratumumab. Faspro is the name of that medication, so not to be confused with that is bortezomib, which we have been using for many years.
Bortezomib has a potential toxicity that is called peripheral neuropathy. If patients have peripheral neuropathy, that can go from very mild where you have some numbness and tingling, to the more extreme cases that it’s associated with pain, discomfort, even weakness and disability.
Well, if we don’t know that’s happening, then we can’t react to it and we can’t adjust doses or switch to something different altogether. You can imagine now we have more options, but in the old days, I always tell patients, “You might be tempted not to say anything about this because you might be thinking, boy, this is working. I don’t want to interfere with my treatment. I can live with the peripheral neuropathy.” But if it gets worse, despite the fact that the treatment is working, the person might have a very significant impingement on their quality of life.
More so now that we have so many alternatives, it’s important not to get us into a path that we might reach a point of an irreversible chronic complication from treatment.
No, and that would be awful.
Before we end the program, Dr. Fonseca, have there been any recent developments in myeloma treatment in research that make you hopeful?
Absolutely. I would say that the one area of work that makes me most hopeful is what we’re seeing with immunotherapy. We have seen that both as the ASH meeting, as well as the ASCO meeting in this year, where people are presenting updates with the various clinical trials with either bi-specific antibodies or CAR T cell therapy as a new avenue for the treatment of myeloma.
In fact, at the last ASH meeting, we had 14 presentations of different compounds or different constructs that are active. I think the future is bright in that regard. We’re seeing their application right now. A lot of these updates have also been made as ASCO.
We’re seeing the update of the treatment of treatments with fairly advanced and aggressive disease where we can still show very significant responses. I participate in some of these trials. I can tell you in my institution, using some of the bi-specifics, I see patients who have previously exhausted all of their options and now are MRD negative at 10 to the -6.
If we’re seeing that in the very advanced disease, I cannot wait to see what happens when we start using these treatments in either early relapse and why not in the near future as frontline part of our therapy? I think to me, that whole field of T-cell engagers, where there’s bi-specifics or the CAR T cells remains one of the most exciting areas for future research.
How can patients stay up to date on information like this?
I think what we alluded to before is very important to work with groups like yours and other patient support organizations that can keep them up to date. I think they’re doing a very good job at also providing updates post some of the large meetings. I know there’s a lot of patients out there that are very sophisticated that will even join the medical meetings. That happens with some frequency; that they want to learn, and patients that go and ask me details about the statistics of the trial. That’s a whole spectrum, right?
But at the minimum, I would say a strong connection with a support group, or a patient support organization becomes an imperative as you deal with this. Also, that would help you because with this whole concept of the information not always being complete and truthful, that can be scary as well, too.
If someone goes and just looks for, I would say even some of the resources that are out there in a textbook today, just keep in mind that textbook was probably written five years ago, and it represents the studies of about 10 or 15 years ago. How that relates to you, it’s very distant. So, it is because of this continuous process of research that we know better what’s going on at the present time.
Dr. Fonseca, thank you so much for taking the time to join us today.
Oh, it’s my pleasure. Thank you for the opportunity.
And thank you to all of our partners. To learn more about myeloma, and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for joining us.
For those cancer patients who have COVID-19 vaccine hesitancy, what information is there about vaccine development? Expert Dr. Shaji Kumar shares details about development and clinical trials of the COVID-19 vaccines – and the reasons why testing could be carried out at a rapid rate compared to other vaccines.
Can you speak to those who might be hesitant about the speed of vaccine development around COVID. I’ve heard this often from other people saying, “Well, they develop this so quickly, how can we trust it?”
Dr. Shaji Kumar:
I think those concerns are quite valid, I think vaccines have always been a very controversial topic and not just COVID vaccination but even for childhood vaccinations. There have been long-standing concerns that some of those vaccinations may be responsible for some of the issues that we see in the children and even in the late adulthood. I think what we really want to get across is, again, taking that question apart, and there are multiple different aspects to it, one is the whole concept of how we created the vaccine so quickly, we kept telling everyone from the time that it started that it takes five to 10 years to develop a good vaccine, and now we have something in a year, so obviously that raises concerns amongst people. I think it’s just a testament to how far technology has come. In the past, we had to isolate the protein and use that protein to develop the immune response, and what has been really unique about the COVID situation has been the Pfizer vaccine and the Moderna vaccine, both of which uses a new technology called the mRNA-based technology. And this is something that has been developed over the past decade to decade-and-a-half, and I would say this is a platform that was perfect, just waiting for the right opportunity to come along.
And the COVID situation really presented that. And even though it was the speed with which this was developed, is just because the technology has come along so much and we can actually do that, and the second is how fast the clinical trials have been done, and I think that speaks to, again, the infrastructure that they have been developed over the years to rapidly develop and implement a clinical trial. So the clinical trials, both Pfizer and Moderna trials had 40 to 50,000 people enrolled in a quick phase and the community transmission that was happening at a very high rate. We could get these trials done in a very rapid manner, so the patients or the people who enrolled in this clinical trial the fact that they were not getting infected could be determined in a much, much faster fashion than what you would have done in the past with any of the other vaccines. So I think the technology is robust. The [COVID] trials are very well-conducted and the end point in terms of efficacy has been very well-determined or very accurately determined. And given the size of these trials and the number of people who have been a goal, I think we can feel fairly confident that the risk associated with this vaccine is pretty low, so you can argue that one of the risk of a particular side effect is only 1 in 80,000. So maybe to the 40,000 people enroll in the trial, they may not have adequate numbers of that and that was certainly a concern when they started vaccinating. And we just know a couple of days ago, there was a publication that looked at almost like 63 million vaccine doses that have been given, and overall the risk of vaccine related side effects have been very, very minimal.
But on the other hand, we all heard about what would happen with some of those vaccines and the blood clots, and I think that even though…yes, it is, as it is a risk. It is a very, very small risk. And the fact that you were able to identify them right away again, I think tells us that should there be rare side effects, you’re going to find it, and we are going to figure out the mechanics of why those side effects happen. And we’re going to figure out how to avoid those things.
So, I think the information flow is so fast and all the data related to vaccines and the side effects are being captured in a real-time fashion that you’d immediately be aware of side effects should that happen.
Check out Part I of Sujata’s story: Normalizing the Word Cancer
Empowered multiple myeloma patient, Sujata Dutta, shares an overview of her treatment from a stem-cell transplant to a clinical trial, and how she chooses to see the positives in her journey.
So once I was diagnosed with multiple myeloma and I was actually informed about the standard of care. So standard of care with multiple myeloma today is typically a couple of cycles of chemo. So I had about five or six cycles of chemo to bring the M-spike to as low as you can, and then that’s followed with like a stem-cell transplant (an SCT) or bone marrow transplant – both are the same. In my case, it was an autologous stem-cell transplant which meant that I use my own stem cells which were extracted and stored and then given back to me.
So then post-transplant, if the counts look good then you go into a maintenance routine. So I didn’t have succession of chemo before the stem-cell transplant. I had my stem-cell transplant at Mayo in Rochester, Minnesota and unfortunately, in my case, we did not achieve the results that we were expecting so my disease actually did actually not come down as much as we would have hoped.
So, I had to go back on a chemo routine and I’m on that one right now. However, I actually am part of a clinical trial. I signed up to be part of a clinical trial that’s looking for newer ways of treatment which are shortening the time of treatment and also with the goal of improving the standard of you know care or like better lifestyle for the patients and like obviously longer life.
So, I’m part of a clinical trial that’s combining Revlimid and Daratumumab, which is like usually you would have an 8-hour hospital visit for the chemo, but in this I am just getting a subcutaneous injection in my belly. It’s a 5-minute injection so that’s not pleasant, but 8 hours compared to 5 minutes, it’s great.
So yes, I am back on chemo just so that we can bring the disease under control. But typically with standard of care with multiple myeloma is like couple cycles of chemo followed by a transplant. If you are eligible for one, and if you are ready for one, and then followed by maintenance. So that’s typically what happens with multiple myeloma.
But there are loads of other treatments that are coming up and researches that are happening, clinical trials that are happening, I would highly encourage it if you come across a clinical trial that interests you, speak to your doctors and see what they say. And if you’re eligible, it would be a great thing to do. I personally wanted to get involved in some kind of volunteering activity. I know that folks before me have done so much and I’m benefiting from that, I wanted to give back as well so I actually signed up for the trial. But other than that, that’s pretty much what the standard of care is today for multiple myeloma or what I know of.
I think one of the biggest takeaways from my cancer journey, I would say is learning to be appreciative of what I have. Learning the value of what I have, not that I did not know that, but I think this life changing kind of event that has happened has taught me even more of the value. For myself, what’s my worth? What’s the worth of somebody else in my life? What’s the worth of things around me in my life? And it has, so my journey has actually helped me understand these things and be appreciative of what I have.
My husband he’s been my primary caregiver throughout this journey and we have actually like been on the journey together, so it has been an amazing journey I would say.
We have discovered like a new relationship between us, like going for chemo, going to Mayo for 6 weeks, and we stay together and you know how much I appreciate what he has had to go through because of me. Like looking at me not being able to walk or not even being able to talk or even drink water because of the amounts of … that I had and supporting me through all of that. I really appreciated it. I appreciated my boys, like I have a 7th and a 6th grader, and for them to understand what I was going through and for them to be able to accept in the form that I was, has been great.
I have friends, I have family who have supported me throughout this so I really appreciate them being with me, being around me, supporting me, rooting for me, praying. There’s one thing that I tell everybody like you know there have been so many people known and unknown that have like you know helped me or prayed for me or rooted for me that I have no choice but to get better.
So you know I really appreciate what I have and I think I also appreciate the value of what I have, and like not think about what I don’t have. I am a believer that divine intervention happens, you don’t know why but everything has a reason and I think whatever happens, happens for the best. For even cancer, I think happens for the best.
For me to understand like what all I had and like how grateful I was for everything that I had. For me to go back to a hobby that I had almost forgotten. I paint, I used to paint and I’d almost given up on that through my journey. I was like I need to go back and do something else and I went back to painting. So like so many good things have come out of this, so you know I’m really grateful for whatever has happened and I’m quite positive for the future so I am looking forward to what’s in store for the future and I’m going to be positive keeping my fingers crossed. That’s my story for you.
On April 27, 2020, I received an email plea for help from Debra after she had read my book. Deb’s husband, Jeff, was struggling with a very malignant form of parotid cancer called Acinic Cell Carcinoma that, despite surgery and radiation, had spread to his chest and spine. Worse yet, there were no clear treatment choices available. Over the next 11 months, Deb & I have maintained an almost constant contact via emails and telephone chats. It has been my honor & privilege to get to know Deb. I am most impressed by her innate intelligence, rock solid determination and steadfast perseverance. Jeff is alive today primarily due to Debra’s tireless efforts to find a solution.
On my request, Deb has penned this story of Jeff’s illness. I sincerely hope that it will inspire other patients and caregivers to become more empowered. Remember, Knowledge is Your Superpower. Sajjad Iqbal, M.D.
My husband, Jeff, was diagnosed with high-grade acinic cell cancer of the parotid gland in February of 2018 at the age of 65. He was a very young, healthy 65, who rarely saw a doctor and needed no regular medications. For 37 years he was a teacher and coach at a small school in Iowa. We have now been married for 47 years, have three children and three grandchildren. Jeff retired early from teaching when he was 61, but continued coaching for several more years. He also did small construction jobs with our son. We spent a lot of time traveling by car throughout the United States. It was a shock to both of us to hear that Jeff had this disease since he seemed to be so healthy.
Several years before Jeff was diagnosed, he mentioned a small lump behind his ear. During a brief physical he had, he asked his doctor about it and was told to keep an eye on it and, if it got bigger, to see a doctor. In January of 2018, he noticed it was getting bigger so he saw the doctor. He was told he needed to get a biopsy but it was probably just a blocked salivary gland. As soon as I heard that, I figured it was cancer as Jeff’s mother had been diagnosed with salivary gland cancer many years before. Hers was a slow growing adenoid cystic cancer that was treated with surgery only. He had his biopsy done at a local hospital and when they said it was cancer, we had them make him an appointment at Mayo Clinic in Rochester, Minnesota which is only a couple of hours from our home.
He had further testing done at Mayo which also showed a lesion at the top of his spine. In March of 2018, he had two separate surgeries to remove the tumors. Cancer was also found in 9 of 21 lymph nodes. He came through the surgeries with no problems. Soon after, he received six weeks of radiation on both of those spots. This was much tougher on him than the surgeries. His neck was badly burned, nausea, no appetite, etc. He made it through and slowly got back to feeling normal. At that time, we were told that chemo wouldn’t help him so he never received any. Three months later, a scan showed a nodule on his chest wall. They did a biopsy and found it to be the same type of cancer. He had a cyroablation on that spot.
Two months later, we found out that the cyroablation had not worked, the spot was bigger and there were several spots on bone. He had Foundation One testing done on his tumor and it showed very few mutations. There was only one mutation, RET, that had a possible treatment at that time. There was a clinical trial at Mayo for a targeted drug for that mutation and they were able to get him in. He started on that in February of 2019. He experienced no side effects and the chest wall tumor stayed about the same the entire time he was on the trial. Unfortunately, though, it was not stopping the bone mets. He had radiation three days in a row on a couple of them when they started causing him pain. Because it was not stopping the bone mets, he discontinued the trial. His oncologist told us that he didn’t know of any clinical trials at that time that would help him. The only thing he had to offer was chemo and possibly Keytruda but he was doubtful they would help very much. Needless to say, this left us feeling lost as to what to do next.
The Mayo oncologist had told us that, in his opinion, clinical trials were the best way to go as you could get the newest treatments and you would be closely monitored. That is what I decided to look for first. Luckily, since Jeff was first diagnosed, I had been doing research on his cancer and possible treatments. There wasn’t a lot as it is a rare cancer. I have no medical background but was determined to figure things out as much as I could and find something that might be able to help. I found three clinical trials that I thought might work for Jeff. These trials did not exist when Jeff was first diagnosed. I sent them to his Mayo oncologist who had told me that he would be willing to look over a clinical trial if I found one. He agreed that the one I was most interested in looked like a good possibility and one of the trial locations was Iowa City which is about 3 hours from us. This is a trial that focuses on the genetic makeup of the cancer instead of the type of cancer. One of the mutations that Jeff has is FANCA and this trial was the first one I found where FANCA was one of the mutations they were looking for. Also, Jeff’s mother, who also had salivary gland cancer, is a carrier of the FANCA gene. There is no known relationship between the FANCA gene and salivary gland cancer but I feel there must be a connection. It is a rare cancer and to have a mother and son have it must be extremely rare. Our children have been tested for this gene and we discovered that our son is also a carrier.
It was in February of 2020 when we went to Iowa City to try to get Jeff into the trial. We found out that they had changed the requirements for the trial and now you had to have had chemo in order to be accepted. The doctor started Jeff on the oral chemo drug, Xeloda, and told us that if anything grew, he would stop the chemo and try to get him in the trial. Jeff was also having some rib and back pain and that was treated with five days of radiation therapy. Following those treatments, he had some heartburn issues for a couple of weeks after which it slowly resolved.
At first, the chemo wasn’t too bad. Soon though, there were many nasty side effects; peeling palms and bottoms of feet, nausea, no appetite, etc. He did not feel up to doing much and spent a lot of time sitting or lying down. He was on this about five months and decided to stop due to the side effects. He was having some back pain during his chemo and was prescribed a narcotic pain reliever. It helped the pain some, but caused constipation, so he had to take more medication for that. He told the doctors he did not like taking the narcotic drug and wanted to find another alternative. They tried one drug and the first night he took it he ended up fainting and having make a trip to the hospital. Needless to say, we stopped that drug right away! They said he was having nerve pain from his spine but were not able to find the exact source. He ended up having a vertebroplasty on his spine as they thought it might help his pain.
Unfortunately, it didn’t help the pain and he also started having a weird feeling of a tight band around his abdomen. We made a trip back to the Mayo Clinic to see a pain specialist there. He thought Jeff might be helped with a nerve block on either side of his spine. He had this done and, not only did it not help, it made the band feeling we were trying to get rid of feel even tighter! This was very disheartening as we really thought it would help. Iowa City had started him on Gabapentin for his nerve pain and had been slowly increasing the dosage. He was also started on a low dose of Lexapro and, between those two drugs, he started to feel less pain in his back. The “band” feeling is still there, but not as bad as it once was. He was finally able to get into the clinical trial in August of 2020. The drug he is on now is a parp inhibitor that targets the FANCA pathway. He has been on this drug for about seven months now with almost no side effects. The targeted tumor has shrunk quite a bit and the bone mets have stayed the same. Unfortunately, on his last scans, there was a new spot on his liver. He was allowed to stay in the trial as it is working on his targeted tumor and he is scheduled soon for microwave ablation on his liver.
When one treatment stops working, I always look for a new clinical trial first.
It is hard, however, as so many of the trials are for certain types of cancer. Even though you discover (from the mutations) that a certain drug may help your cancer, you can only be in that trial if you have a certain type of cancer. I hope in the future there are many more trials based on the genetic makeup of the cancer rather than the type of cancer. The other problem is that the majority of trials are held at larger hospitals that are just too far away to go back and forth as often as needed. It would be great if there were a way to have some of the treatments done at a larger hospital in your own state. Also, if you have a rare cancer, it is much harder to find clinical trials.
I have a library background and have always relied on books and articles to find information about various topics. Now that the internet is available that has been my most important tool at this time. Also, websites like PEN, providing patient’s stories, healthy recipes and classes are very helpful. These types of sites have really helped me feel not so alone and have given me much more hope than I have ever received from any oncologist. It is also over the internet that I connected with Dr. Sajjad Iqbal after reading his book “Swimming Upstream.” He has been very generous with his time and willing to give suggestions and advice as he has a cancer similar to Jeff’s. It has been a great comfort to me to be able to e-mail him to get his opinion on something or ask a question. He has also helped me feel more hopeful than anyone else I have talked to – not only by his words but by his courageous example.
When Jeff was first diagnosed, he was still coaching track. The entire track team wanted to have a benefit for him and sold t-shirts and wristbands, and had a meal and dodge ball tournament to raise money for him. Jeff is a very popular guy in this rural school district and I know it meant a lot that his team did this for him. We have support from our family and friends and feel that we have people we can call if we need something. The pandemic has kept us from getting together with people as often as we would like but we are looking forward to that in the future.
We know that there is a good chance that Jeff’s cancer may never be cured. If that is true, I would like the next best thing – for him to live as long as possible, as well as possible with the cancer. We have had three very good years living with it and working around his medical appointments. I will do everything I can to help him have more of those years.
Jeff has handled this whole situation very well from the beginning. He is a pretty laid-back person who takes things as they come and isn’t much of a worrier. He has kind of set an example for me just by taking things as they come. I feel his job is to fight the cancer and my job is to help him fight the cancer. Our lives are pretty much the same as they were before he was diagnosed – only with a lot more doctor appointments!
Sajjad Iqbal is a physician and cancer survivor. Almost 17 years ago, Sajjad learned that he had an extremely rare and most malignant type of parotid cancer called Salivary Duct Carcinoma. He was given a 30% chance of surviving for 2 years. Because of Sajjad’s medical training and his unwavering sense of hope & determination, he was no ordinary patient. Sajjad researched his condition and proposed a treatment method for himself that stopped the progression of cancer cells. Although Sajjad is still living with, and still fighting, the cancer, he continues to defy the odds with no end in sight. To inspire other cancer patients, Sajjad Iqbal has chronicled his amazing cancer journey in the form of a book titled, “Swimming Upstream, My Struggle and Triumph over Cancer and Medical Establishment.”
Myeloma research is advancing quickly. Dr. Joshua Richter, a myeloma expert, shares his excitement about emerging treatments in development.
Dr. Joshua Richter is director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai. He also serves as Assistant Professor of Medicine in The Tisch Cancer Institute, Division of Hematology and Medical Oncology. Learn more about Dr. Richter, here.
When it comes to myeloma research and emerging treatment options, what are you most excited about, specifically?
So, I think the big thing that I’m excited about from myeloma that we’re on the cusp of is T-cell engagers and T-cell based therapies. And, essentially, we all have T cells in our body, and T cells are a part of our immune system. They attack bacteria, viruses, and cancer.
And one of the best cancer fighters that exists is our own immune system. And the old way of treating cancer and blood cancers like myeloma was just to give medicines that suppressed all of the immune system, the good and the bad. Now, we’re trying to be more precise, and there’s certain parts of the immune system that we don’t want down, we want up. So, they help attack the cancer.
And the two biggest technologies are something called CAR T and something called bispecific antibodies. CAR T stands for chimeric antigen receptor T cells.
And, basically, what that is is we collect your T cells, we engineer them in the lab to rev them up and target the cancer. And we can put them back into you and they attack the cancer, very exciting. And then we have something called a bispecific antibody that has two arms. And as we infuse this medicine into you, one arm grabs onto the cancer cell, the other arm grabs onto your T cell and makes that T cell activate and attack the cancer cell.
And a lot of these drugs are in clinical trials as well. So, we’re very excited about moving from, you know, just lowering everything, the good and the bad, to being more precise and saying, no, no, no. There are some cells that we want way, way up.
Right. Right. So, you’re – you’re being much more specific now.
When deciding on a myeloma treatment, what factors affect your choice? Dr. Joshua Richter shares key considerations, the patient role in making decisions, as well as key questions to ask about treatment
Dr. Joshua Richter is director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai. He also serves as Assistant Professor of Medicine in The Tisch Cancer Institute, Division of Hematology and Medical Oncology. Learn more about Dr. Richter, here.
Dr. Richter, would you please start by introducing yourself?
Sure, my name is Dr. Joshua Richter. I’m an Assistant Professor of Medicine at the Tisch Cancer Institute Icon School of Medicine at Mount Sinai and the Director of Myeloma at The Blavatnik Family Medical Center at Chelsea at Mount Sinai.
Great. Thank you. When making a treatment choice, what are three key considerations for myeloma patients?
Absolutely. So, whenever we decide on treatment options, we consider three main topics: patient-related factors, disease-related factors, and treatment-related factors. So, patient-related factors are easy. How old or young are you? How fit or frail? Do you have any comorbidities, meaning other medical problems like heart disease or diabetes?
Disease-related factors are another important one. How aggressive is your disease? Is it rising up very quickly? Is it very slowly? Do you have something that we call extramedullary disease which means myeloma outside the bone marrow in the mass that we call a plasmacytoma? And that influences how we treat things.
And the last is treatment-related factors. What treatments have you, previously, had, how did you respond to them, and what side effects did you have?
If you developed a lot of neuropathy with one drug, we may not want to choose a drug that continues to have that type of side effect profile.
What’s the role as a patient in making treatment decisions?
The role, from my standpoint of the patient, is honesty. You don’t get extra points for being in pain. I want to hear from you. I want you to tell me what your concerns are, short-term, long-term. I want you to tell me about little problems that you don’t – it’s not that you don’t want to bother your care team, we want to know.
Because something little may mean something big to us. So, all we want is for your well-being. And the better we keep those lines of communication open, the better.
Are there questions that patients should consider asking about their treatment plans?
Absolutely. I think in a day and age where there’s so many different options, I think it’s always important to ask the care provider, what are the alternatives to this? Or why did you select this treatment for me? Because many times, there are alternative answers. So, in myeloma, there are a lot of options that may be good for someone. And the physician team may say we recommend this drug, and the patient may have trouble getting back and forth to clinic for logistical reasons. And there may be an all-oral alternative that if you don’t ask, we may not know that that’s going to be your preference. So, really that dialog is crucial.
What tests will you have following a myeloma diagnosis? Are there additional tests you should request? Dr. Joshua Richter provides an overview of key testing for myeloma and why each test is necessary.
Dr. Joshua Richter is director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai. He also serves as Assistant Professor of Medicine in The Tisch Cancer Institute, Division of Hematology and Medical Oncology. Learn more about Dr. Richter, here.
What standard testing follows a myeloma diagnosis?
So, the standard testing that follows a myeloma diagnosis is multifaceted. So, the first one is blood work. And we draw a lot of blood tests to look at the bad protein that the cancer cells make. So, we send tests like a protein electrophoresis which tells us how high that bad protein is. We send immunofixation. That test tells us what type of bad protein it is. You’ll hear names like IgG kappa and IgA lambda.
These are the different types of bad proteins made by myeloma cells. Oftentimes, we’ll send urine tests to find out how much of that bad protein that was in the blood is coming out in the urine. We will, typically, do a bone marrow biopsy. It’s a test where we put a needle into the back of the hip bone to look at the marrow itself. And we’ll use that marrow to figure out how much myeloma there is, any other characteristics like the genetic changes in those cells.
The other big thing is imaging. So, the classic imaging that we do with myeloma is something called a skeletal survey. It’s, basically, a listing of X-rays from head to toe. But nowadays, we have newer techniques, things like whole body low-dose CAT scans, something called a PET-CT scan, and MRI scans. And your care team may have to figure out which one is right for you at what given time.
Mm-hmm. Are there additional tests that patients should ask for?
Absolutely. One of the most important things from myeloma has to do with the genetic risk stratification.
So, for almost all cancers, the staging has a very big impact. And people will often think of cancer in stages I, II, III, and IV, and they’re managed very differently depending upon what stage it is. Myeloma has three stages, stage I, II, and III. But the most important thing is, actually, beyond the staging is what’s called the cytogenetics risk stratification. So, it’s really important when the bone marrow is sent to be sure that it is sent for, kind of, advanced techniques. Because you really want that snapshot of exactly what the genetic profile is, because that gives us information of A) how to treat, and B) prognostic, you know, who will tend to do better or worse based on this information. And even though that may not tell us which drugs to use, specifically, it may say, should we do something like a transplant or not? Should we consider a clinical trial early or not?
I see. How do test results affect treatment choices?
So, test results can affect treatment choices in a number of ways. Probably, the most common one is thinking about the routine blood tests like your CBC or complete blood count and your chemistry, which looks at things like your kidney function. Some drugs tend to have more toxicity to the blood counts. So, if your blood counts are very low, we may choose drugs that don’t lower the blood counts very much.
Kidney function which we, usually, measure by something called the creatinine. Creatinine is made by the muscles and cleared out by the kidneys. So, if your kidneys aren’t working very well, you don’t pee out creatinine, and that creatinine level will rise in the blood. If your creatinine level is high, we may choose certain drugs that don’t affect the kidneys or not metabolized or broken down by the kidneys.
The genetic studies that we use – we’re not quite at this base yet where we can say, if you have this genetic abnormality in your myeloma, we should use this drug except there’s some really great data on the cutting edge about a drug called venetoclax.
Venetoclax is a pill that’s used to treat other diseases like lymphoma and leukemia. And it turns out that people who have what’s called a translocation (11:14) which means part of the 11th chromosome and part of the 14th chromosome in the cancer cells swap material.
Those people respond amazingly well to venetoclax. So, we’re starting to have what we would call precision medicine where we find your genetic abnormalities, not that you got from your parents or passed to your kids, but the genetics inside the tumor cells to tell us which treatments will work best for you.
What are the essential tests that should follow a myelofibrosis diagnosis? Dr. Joseph Scandura reviews the necessary laboratory testing, along with a discussion of next generation sequencing, and explains how often bone marrow biopsies should take place.
Dr. Joseph Scandura is Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura, here.
What testing should take place following a myelofibrosis diagnosis?
So, a diagnosis of myelofibrosis always comes after a bone marrow exam and a physical examination. Often, patients have an enlarged spleen and blood count testing and a variety of other laboratory tests. So, after that and a diagnosis is made of myelofibrosis and, sort of, coincident with the diagnosis, we often look for molecular markers of myelofibrosis. So, these are malignancies of the bone marrow, cancers, if you will, on the bone marrow, although the term is scarier than or is different than what we think of for many malignancies in how it acts. But the myelofibrosis, this is a disease that’s characterized by, really, mutations in the malignant cells, the abnormal cells.
They’re really just one of three genes. And so, JAK2 being one of the genes, calreticulin or CALR being another one, and MPL one.
And more than 90 percent are people having mutation in just one of those three genes. And so, often at the time of diagnosis, tests for those mutations are done, and they help eliminate the possibilities of other causes of myelofibrosis – infections, rheumatological diseases. Sometimes, you can have marrow fibrosis but they don’t go along with mutations and the same clinical situation. And so, at the time of diagnosis, we usually know something about a mutation in JAK2, CALR, or MPL.
More commonly now, and it’s increasingly common over the past 10 years in, I would say, in New York City and many places across the country, we also look more broadly for other common mutations in the MPN cells. And these are what we refer in the batch as next generation sequencing or NGS panels, and we use the term panels because we’re looking at from a few tens to even 100 or a couple hundred genes for mutations that occur far less frequently than in JAK2 or MPL or CALR.
But they occur often enough that some of them we use to help guide treatment decision-making or approach to therapy. The reality of it is that that the technology to sequence and identify mutations has really outstripped our knowledge of what to do with all of that information.
And, for the vast majority of people, it comes down to do you have a marker, a genetic marker that tends to go along with higher risk, meaning a higher likelihood of something that we don’t want to have happen. And in that instance, although it may be looking at a hundred or so genes, it comes down to a binary thing – either you have or you don’t have.
Is there any other testing that you usually want to do?
Laboratory testing, for sure and, as I mentioned before, a bone marrow exam. But physical examination, some people might do imaging of the spleen size. Honestly, I don’t routinely do that outside of the setting of the clinical trial. I don’t really think it dictates therapy very often.
And if the spleen is so small that you can’t feel it on physical exam, it probably isn’t clinically meaningful anyway in terms of something to treat. It might be there, but it doesn’t really change things too much.
How often should patients have a bone marrow biopsy?
So, I’ll answer there is no standard in terms of monitoring for myelofibrosis with the marrow or otherwise. My personal approach is I do a marrow when I think it’s going to help medical decision-making. And so, for a patient who’s got early myelofibrosis, who’s been very stable, responding well to therapy, that could be three, five years between marrow exams.
For somebody who’s being considered for a clinical trial, oftentimes, a marrow exam is required before they start on the clinical trial and at various intervals afterwards. If there’s somebody who had been stable and something is changing, like the blood counts are changing or his symptoms are changing, or any of a number of clinical features, then I might look in the marrow to see what’s happening there, to see if explains and can help guide a treatment approach to help people feel better. So, there is no single standard, but my personal approach is to do a bone marrow exam when I think it’s going to help make a decision.
With a lack of staging in prostate cancer, which patients can benefit the most from telemedicine visits? Dr. Leanne Burnham maps out factors that may make some patients lower risk and situations that may warrant other patients to be seen in person to receive optimal prostate cancer care.
Dr. Leanne Burnham
So, prostate cancer is a very diverse disease. It presents itself differently in the clinic in each individual patient, so who is considered low risk, who is considered high risk is really a personal conversation that you have with your physician one-on-one, and it’s based on a lot of different factors. It’s not as cut and dry as some other cancers where you may break the disease down by just stage, simply stage I, stage II, stage III, stage IV. There’s a lot that goes into determining how aggressive someone’s prostate cancer tumors are. That being said, if you are considered to be low risk, you may be undergoing active surveillance by your physician or watchful waiting and in that situation, telemedicine would probably be a perfect approach where you get your labs done every few months or whatever your physician decides. And they can track your PSA velocity or doubling time and seeing if your PSA is growing, by growing I mean increasing in circulation or if it’s not which would be ideal. If you are more high risk, then you may need to increase your telemedicine visits. And, of course, if you are taking therapies that cannot be done from home, then you would need to go to a clinical setting, so that would include radiation of course, and chemotherapy, immunotherapy, perhaps. If you’re enrolled in a clinical trial where you need to go on-site to receive the medication, then that’s something that cannot just be done by telemedicine, you would have to go in-person.
“I had a clinical trial in my hip pocket already because I had taken the extra steps. I think it saved my life and got me in the right place,” says empowered patient Bob Lawson who is currently participating in a clinical trial for his non-small cell lung cancer, which recurred a little over three years after his initial cancer diagnosis and treatment. While Bob is in the right place now, his cancer journey has not been an easy one, and he has had to be actively involved in his treatment to ensure the best possible outcome. “It’s so difficult to know what to do, it’s overwhelming. You have to do research on your own,” says Bob, who recommends the Patient Empowerment Network (PEN) website for patients looking for a place to start. “For patients seeking out advocacy, PEN has something that’s very important,” he says referring to the free, online resources provided by PEN. Bob especially likes the PEN-Powered Activity Guides, found here, and says they are powerful tools for patients to find hope and ways to benefit from the new technology and treatments available. His best advice, though, is to do what he’s done all along. “I would say ask questions. A lot of them. You want to ask the right questions and the right questions change,” he says. “If you don’t ask the questions, you’ll never know.”
When he was first diagnosed with lung cancer, Bob says he had no idea what to expect. “At that time, I didn’t know anything about anything,” he says, but he adds that he did know enough to ask questions, and by asking questions, he improved his treatment outcomes. He was diagnosed as the result of some testing that was done for a medical episode that ultimately turned out to be a transient ischemic attack (TIA), which is often referred to as a mini stroke. During the testing, an x-ray revealed a shadow on his left lung, and a fine needle biopsy confirmed that it was cancer.
Bob says when he talked to the surgeon at the hospital, he wasn’t comfortable with the treatment plan, which included a surgery where his ribs would be cracked. He asked the doctor if the invasive surgery was really the only option, and that question got him referred for a second opinion. “Most doctors encourage a second opinion,” he says. “You have to become educated very quickly about what you’re dealing with.”
Bob says the second doctor won his confidence almost immediately. He recommended a minimally invasive lobectomy of the upper left lung and, although his cancer was stage 1B and did not require chemotherapy, the doctor strongly recommended it. The surgery was successful as was his round of chemotherapy. “I did the entire regimen, rang the bell on my way out the door, and they said I was cancer free,” says Bob. That was seven years ago.
A few years after being declared cancer free, Bob decided it was time to address the TIA he’d experienced. The cause of the TIA had never been discovered so Bob had a scan of his carotid artery. His artery was clear, but the technologist noticed something on his thyroid. That something turned out to be cancer. It was a nodule classified as malignant neoplasm, and it was unrelated to his lung cancer. Fortunately, the nodule was completely encapsulated in the tissue and was removed. However, the treatment protocol at the time was to completely remove the thyroid. “I didn’t like the sound of that,” says Bob who once again asked a question. “What would you do,” he asked his doctor. Later that night, Bob says his doctor called him. Because of Bob’s question the surgeon dug a little deeper and spoke with the pathologist who said he wouldn’t remove the thyroid. Bob kept his thyroid and, with the assistance of minimal medication, he’s had no problems with his thyroid since. He says five months after he turned down surgery to remove his thyroid, the nodule he had was declassified as cancer.
However, just ten months after the issue with his thyroid, Bob lost his voice and noticed he was coughing a lot. At the same time, he was experiencing significant pain in his right hip. He attributed the hip pain to not getting any younger and the natural consequences from old injuries, but it bothered him enough that he went in for some tests. The tests revealed a little bit of discoloration on the bone that the doctor said he normally wouldn’t worry about, but since Bob had a history of cancer, he wanted to do a scan of the area. “The scan lit up like a Christmas tree,” says Bob. His lung cancer was back, and it had spread to his right femur and hip. Bob had successful radiation treatment on his hip, but he couldn’t have radiation on the tumor in his lung because of its location near the vagus nerve and vital organs. The tumor location also meant surgery was not an option, so his doctor recommended a targeted monoclonal antibody immunotherapy.
Once again, Bob sought a second opinion. “What I had done when they discovered the hip tumor was called the insurance company and got permission to get a second opinion,” he explains. He actually got the opinion of two other oncologists who both agreed with his doctor’s recommended treatment course, but the second doctor, with Johns Hopkins Medicine, went a step further and said, if the treatment stopped working, he had a clinical trial that Bob might want to consider. So, Bob took home the paperwork for the trial and began immunotherapy treatment with his doctor.
It was a relatively new therapy at the time, and, for a while, it worked to reduce the size of the tumor in his lung. Then, he had two months in a row where his scans showed disease progression, and he was taken off the therapy and given a prognosis of 10 to 18 months to live. He asked his doctor what they were going to do, and his doctor said he could do chemotherapy again. “That was the last thing on earth I wanted to do,” says Bob, who asked his doctor about a clinical trial. The doctor agreed that a trial was probably the best course of action for Bob, but he didn’t have any to recommend.
That’s when Bob pulled the Johns Hopkins clinical trial out of his hip pocket. He’s been having infusions every month for the past 17 months, and he’s really happy with the results. “The tumor is steady or reducing all the time, which is great, obviously,” he says.
Bob remains vigilant about his healthcare and continues to pay attention to what other treatments might be available to him should this trial stop working. He continues to research other trials, sometimes reviewing as many as 20 in a week. “Most people just trust the doctor, and that’s the wrong approach,” he says. “Get a second, third, and fourth opinion, and have something ready to go in case what you’re doing doesn’t work.” Bob has learned that approach through experience, but he hopes his story will help make it easier for others. “How can I best support someone who’s like me,” he often asks himself. “I think that’s something I’d like to do,” he says.
Patients who want to ask questions like Bob did, but aren’t sure what the right questions are, can start here with the PEN downloadable office visit planners available for free at powerfulpatients.org.
Dr. Peter Forsberg discusses how clinical trials help improve care for myeloma patients and shares advice to patients who are fearful about joining a trial.
Dr. Peter Forsberg is assistant professor of medicine at the University of Colorado School of Medicine and is a specialist in multiple myeloma. More about Dr. Forsberg here.
How Can Myeloma Patients Advocate for the Best Care?
Where do clinical trials fit in as a treatment choice?
So, I do clinical trials in myeloma, I am certainly an advocate for the important role of clinical trials in myeloma. It is how we learn more about how best to treat patients. So, clinical trials are the foundation on which our decision-making has been built and continues to be refined. We are at a place where clinical trials don’t mean one thing. There are different types of clinical trials. Different stages of trials. Some that may be what we call, early phase that’re looking at brand new medicines or medicines in entirely different ways.
And ones that are late phase, where they may be comparing a well validated standard of care, versus a new approach. So, understanding what the potential clinical trial is and what that entails and what its goals are, are an important factor for patients as they consider participating. But beyond that, trials are a really critical area for us to evaluate new therapies and to get better at using the medicines we have in novel or improved ways.
So, they can be a really useful piece for not only the myeloma community, but for patients as they navigate through. So, I haven’t had many patients who I take care of who participated in clinical trials and been disappointed that they did so. Usually, it’s a positive experience.
Even if it is one where you want to understand what you may be embarking upon as you begin the process.
Some patients can be fearful when it comes to clinical trials. What would you say to someone who might be hesitant to consider participating in one?
Well, like I said, I would say that one of the most important things is making sure you understand what the goal of the trial is. What it entails. Clinical trials may have one name, but they’re very different things. And the right type of trial may be very different in different clinical circumstances. So, feeling comfortable with what it is. Making sure you feel comfortable asking your provider what the rationale for the trial is.
But also, as I mentioned, trials are a unique process and one that can often be very fulfilling for patients. Understanding that not only may you be trying a new treatment approach, but that you’re hoping to contribute to our improvement for how we manage multiple myeloma. It’s an altruistic goal. But it can be one that can be pretty meaningful for patients if they’re comfortable moving in that direction.
Dr. Peter Forsberg outlines options in the myeloma treatment toolkit, including targeted therapies, chemotherapy, immunotherapy, and combination approaches —and explains how the recovery process from stem cell transplant has improved.
Dr. Peter Forsberg is assistant professor of medicine at the University of Colorado School of Medicine and is a specialist in multiple myeloma. More about Dr. Forsberg here.
Would you walk us through the currently available myeloma treatment approaches and who they might be right for?
At this point, we’re lucky that we have a much broader toolkit to treat myeloma than we have had in the past. Myeloma is one of the successes in modern oncology in that way. At this point, we have a number of targeted therapies. Some of those are pill-based options, some are injections or infusional medicines. We have some immunotherapies, which are things like monoclonal antibodies, which help to work.
We use some conventional or older fashioned chemotherapy, often lower doses and as part of combinations. And steroids. Steroids are always the medicine that is one of the backbones of our combinations. In myeloma, we do often use combinations. So, it’s usually a mixture of targeted therapies. Sometimes immunotherapies or chemotherapies.
As well as steroids to try to treat the myeloma. And some of the considerations are, which combination makes the most sense. Are there other medical problems or disease related factors like disease aggressiveness that may influence which ones we wanna choose or how many. Also, is a three-drug combination the right fit or is a four or a two drug the right. And it does continue to evolve.
Our options and our ability to use multi-agent regimens has continued to improve as we’ve gotten better and better therapies that’re well tolerated and that allow us to use really active combinations, even in patients who may have substantial other medical problems. So, I think it’s been something that continues to evolve over time and will continue to evolve. But the good news is that it’s been an issue of just how to incorporate more and better options.
How do we bring these good new tools into the mix as early as is appropriate? To control the myeloma in really substantial ways. And again, as I mentioned, the question of the role of stem cell transplant continues to be an important one. That is a way for us to still use older fashioned chemotherapy at a high dose to help to achieve a more durable remission. But usually, the way that we parse through these targeted immunotherapies and chemotherapies, is something that may be individual.
Although, we have some broad principals that help guide us for how we manage patients across different types.
How do you decide who stem cell transplant might be right for?
The good news in the United States is that we’re able to be fairly broad in terms of our consideration of stem cell transplant. There is no age restriction above which it’s not. We’ve gotten better and better at supporting patients through stem cell transplant. We have better medicines to deal with potential toxicities. And so, patients do better and better in going through transplant. But it is still an intensive treatment modality. So, in considering it, it is an option for a large portion of myeloma patients at diagnosis. After we get the myeloma under control. But the decision remains an individual one. Some patients may prefer to defer stem cell transplant until a second line therapy or later.
Whereas others feel very comfortable moving forward with it in the first-line setting. I would say that it is certainly something that we try to demystify for patients. It can sound a little bit intimidating, certainly because it is a little more intense and requires more support. But it is something that we have gotten quite good at navigating patient and supporting them through.
What about maintenance therapy, how does that fit in?
Following initial treatments to get the myeloma under control, whether that includes stem cell transplant or not. Usually we transition into a maintenance therapy. Maintenance therapy is a way for us to sustain control or remission of the myeloma. And make that longer lived. So, what we use for maintenance may be different patient to patient. But it is a important part of our treatment approach for many patients.
Are some therapies less intense than others, and what are some possible side effects of those?
So, certainly there are treatments with varying degrees of intensity or potential toxicities. The good news is that as we’ve gained more and more treatment options, we’ve also gotten better at using the ones we have had for a while now to minimize some of their toxicities. So, by adjusting dosing schedule and routes of administration, we’ve gotten better at fine tuning the tools we have toward minimizing those toxicities.
So truthfully, many myeloma patients after you start treatment, actually feel better than before they started chemotherapy because the myeloma itself is a destructive process and the treatments are quite often well tolerated. That being said, certainly over time, treatment related side effects often emerge. Some of the treatment toxicities may cause some challenges in terms of managing patients through their myeloma process. But usually, those can be overcome. Even if that means needing to adjust the treatment protocol.
Adjust doses, change medicines. And so, while there are varying degrees of intensity, we’re usually able to find the right balance for any given patient to still have a very active anti-myeloma regimen while trying to be very cognizant of potential treatment toxicities and taking steps to mitigate that.