Tag Archive for: Dana-Farber Cancer Institute

Self-Advocacy in Myeloma Care | Advice From an Expert

Self-Advocacy in Myeloma Care | Advice From an Expert from Patient Empowerment Network on Vimeo.

What questions should myeloma patients ask their doctor about a treatment plan? Myeloma expert Dr. Omar Nadeem shares key advice and emphasizes the importance of patients engaging with their healthcare team in treatment decisions.

Dr. Omar Nadeem is the Clinical Director of the Myeloma Immune Effector Cell Therapy Program and Associate Director of the Multiple Myeloma Clinical Research Program at the Dana-Farber Cancer Institute. Learn more about Dr. Nadeem.

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See More from Evolve Myeloma

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Clinical Trials for Myeloma Treatment | Essential Information for Patients

Clinical Trials for Myeloma Treatment | Essential Information for Patients

What Should Myeloma Patients Ask About Developing Research

What Should Myeloma Patients Ask About Developing Research?

Personalized Medicine for Myeloma Treatment | What Patients Should Know

Personalized Medicine for Myeloma Treatment | What Patients Should Know

Transcript:

Katherine:

What questions should patients be asking their provider about a proposed treatment plan?  

Dr. Nadeem:

Yeah. I think because myeloma therapy’s so nuanced and much of this is still in clinical trials or under investigation about what to do with some of these results, I would say, as a whole, it’s important to know which tests the physician looks at to determine how you’re doing, and kind of what their assessment of that result is. So, for example, if somebody’s had a 50 percent reduction in the amount of abnormal protein in the blood, is that sufficient, or should we be aiming for a number that’s much higher than that? 

Some of that depends on kind of where they are in their treatment course, but that’s a very sort of reasonable question to ask your physician is that where do you see my response now, let’s say six months into therapy, and is this adequate, and what is now, after we have all this information, what is my roadmap going forward to try to keep this disease in check? 

Katherine:

Yeah. Well, that’s great advice, Dr. Nadeem. Thank you. I’d like to turn to self-advocacy, Dr. Nadeem. Why is it so important that patients engage in their care treatment decisions?  

Dr. Nadeem:

Yeah. As I mentioned, myeloma therapy is so individualized now and we can sit here, look at the trial data, get very into the weeds and technical about this therapy with this approach as X or Y higher response rate.  

Or MRD-negative rate, but in reality, we’re dealing with people and we’re dealing with people that have lives.  

They have all their priorities, and until you share that with us, it’s very difficult for us to know exactly what’s important to you. So, what I may consider to be kind of the “best therapy” for you may not make sense for you because of all the priorities that you may have, and I think it’s so important to advocate for yourself and not be afraid to bring that up to your physician because I think many patients kind of hold that stuff in for a long time because they don’t want it to impact their care. But I would argue the other way around.  

Tell us. Tell us exactly what you prioritize. Tell us if you can’t be out of commission for work for X amount of time because of a stem cell transplant. We now have options. We now have options for patients because of all these amazing new therapies for myeloma and we can come up with a very individualized treatment plan for you based on your priorities.  

Katherine:

If a patient is feeling like they’re not getting the best care or they’re uncomfortable with the care they’re receiving, what steps should they take to change that?  

Dr. Nadeem:

Yeah, I think that’s very difficult because this is a complex system. Medical systems are getting even more and more complex. They’re busy. Everybody’s busy: busy offices, labs, radiology. We’re all feeling that. It doesn’t matter where you are. So, I think it’s important to raise those concerns, number one, to your practice that you’re being seen at because they would like to see that feedback, right? So, kind of see what is something that they can perhaps improve upon. I think it’s always important, like we just said, to advocate for yourself and raise some of these issues and not be afraid of that.  

We’re all in this together, right, so I think ultimately, we’re all trying to take the best care of you and we would need to know which part of that may or may not be working so well.   

Personalized Medicine for Myeloma Treatment | What Patients Should Know

Personalized Medicine for Myeloma Treatment | What Patients Should Know from Patient Empowerment Network on Vimeo.

What is personalized medicine, and how can myeloma patients access this type of care? Myeloma expert Dr. Omar Nadeem defines personalized medicine and shares how test results can impact myeloma care and treatment options.

Dr. Omar Nadeem is the Clinical Director of the Myeloma Immune Effector Cell Therapy Program and Associate Director of the Multiple Myeloma Clinical Research Program at the Dana-Farber Cancer Institute. Learn more about Dr. Nadeem.

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See More from Evolve Myeloma

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Self-Advocacy in Myeloma Care | Advice From an Expert

Self-Advocacy in Myeloma Care | Advice From an Expert

Myeloma Research Highlights From ASH 2023

Myeloma Research Highlights From ASH 2023 

What Should Myeloma Patients Ask About Developing Research

What Should Myeloma Patients Ask About Developing Research?

Transcript:

Katherine:

Well, Dr. Nadeem, we’ve been hearing the term personalized medicine more frequently in recent years. How would you define personalized medicine for myeloma, and how can patients access this type of care?  

Dr. Nadeem:

Yeah, personalized medicine or precision medicine is a term that we’ve really sort of used for many oncologic conditions over the last decade or so. I would say, for multiple myeloma, in terms of identifying a target within the myeloma cell that’s unique to the patient. 

And then deploying a certain therapy to that patient because of that target is still lacking. We do have one example where patients have, for example, an 11;14 translocation, which we see in about 15 percent of myeloma patients.  

There’s an agent called venetoclax (Venclexta) that is very active against that particular cohort of patients, although that is still not approved to be used, but that’s one example where that agent specifically benefits that type of myeloma. Other than that, most of the therapies that we have benefit essentially everybody with myeloma, which is great, but it’s not so personalized.  

Where I would say there’s the most personalization happening now, at least in my practice, is looking at which types of therapies an individual patient may receive. What I mean by that is if somebody’s in an excellent response, with quadruplet-based induction therapy, I have a very real discussion with them about the pros and cons of stem cell transplant.  

We make those decisions in real time depending on how the patient doing, depending on how their response is.  

And then kind of deciding a whole kind of what are the kind of risks and benefits and what makes sense for that individual patient. Similarly, when you go on to maintenance therapy, maintenance therapy means that after you’ve gone through the initial phase of your myeloma therapy and the disease is under control, what type of therapy can we keep you on to keep it under control for as long as possible? Historically, that has been lenalidomide or Revlimid. Now we’re adding drugs such as daratumamab (Darzalex) and other agents to Revlimid to see if that can further prolong the response to that initial therapy.  

So, all those decisions are so individualized that you have to discuss with your provider what makes sense for you and what are the pros and cons of doing one approach versus the other.   

Katherine:

Well, if we’re talking about in-depth testing, how do the results of that testing affect treatment options? 

Dr. Nadeem:

So, right now we use conventional blood tests to get a sense of response in the vast majority of patients. That includes the serum protein electrophoresis and the serum free light chain assay.  

Most patients have detectable levels of these proteins, abnormal proteins in the blood at diagnosis and then you can follow them using a blood test. There’s a subset of patients that have disease only that shows up on scans. So, we then kind of incorporate some of those scans and then, also, utilize the bone marrow results both in the beginning and in subsequent analyses to kind of give a big-picture composite response assessment for that particular patient. Nowadays, there are also other tools that we’re using, such as MRD, or minimal residual disease.  

That is a test that is done on a bone marrow biopsy to determine, if you don’t have detectable protein in the blood, do you have myeloma cells present at the deepest level possible? And if you do versus if you don’t, trials have shown that there is a difference in terms of prognosis. Now, while that hasn’t fully been utilized yet to make treatment decisions in patients that are not on clinical trials, we do get prognostic information out of it, and nowadays, more and more of those trials are using these MRD tests to determine what to do with treatment.  

And I think that’s how it’s going to be in the future. So, having those extra tests available but, again, important to discuss with your provider what is the utility of this test. How are we going to use this information for your individual case to make some decisions? 

Next Generation Myeloma Treatment Options

Next Generation Myeloma Treatment Options from Patient Empowerment Network on Vimeo.

What new myeloma therapies are showing promise? Myeloma expert Dr. Omar Nadeem shares an update on emerging myeloma treatment research, including CELMoDs, and shares advice for a having a treatment plan roadmap. 

Dr. Omar Nadeem is the Clinical Director of the Myeloma Immune Effector Cell Therapy Program and Associate Director of the Multiple Myeloma Clinical Research Program at the Dana-Farber Cancer Institute. Learn more about Dr. Nadeem.

Download Resource Guide

See More from Evolve Myeloma

Related Resources:

Personalized Medicine for Myeloma Treatment | What Patients Should Know

Personalized Medicine for Myeloma Treatment | What Patients Should Know

Myeloma Research Highlights From ASH 2023

Myeloma Research Highlights From ASH 2023 

What Should Myeloma Patients Ask About Developing Research

What Should Myeloma Patients Ask About Developing Research?

Transcript:

Katherine:

Are there other emerging myeloma therapies that are showing promise? 

Dr. Nadeem:

Yes. So, I think over the last few years, most of the buzz has been with these immunotherapies. And, again, more work to be done there to see whether combinations, different schedules, different targets, different types, will show more and more benefit in each of these myeloma disease settings.  

But we also have simultaneous development of other agents that are not in this sort of immunotherapy T-cell redirecting therapy realm. We have newer versions of our classic immunomodulatory drugs, such as lenalidomide (Revlimid) or pomalidomide (Pomalyst).  

We now have their next generation agents, called CELMoD drugs and there’s two of them in development. One of them is called iberdomide; one is called mezigdomide.  

These are, again, kind of building up on the success of some of these previous therapies that are kind of cornerstone therapies for myeloma patients and because these are essentially better agents, they’re more targeted, and they also have greater response rates as single agents and as combinations.  

We’re hoping that these would be approved in the not-so-distant future and then perhaps will replace some of these immunomodulatory drugs that we have currently utilized in newly diagnosed and relapsed myeloma. Essentially what this means is things are just getting better and better and better as we get newer versions of some of these therapies. So, those are, I would say, kind of next in line in terms of hopeful approvals.  

And then we’ll add to some of the options that we have for myeloma patients.  

Katherine:

How can patients and care partners stay informed about the latest myeloma research? 

Dr. Nadeem:

Yeah, it’s a lot of moving parts all the time. From one six-month interval to the next, you tend to have nowadays perhaps some drug approvals, which is amazing, but if not updates of all these sort of combination trials, etcetera, of where these things are going. I think kind of talking to your physician, obviously, about some of these updates is really critical. As I mentioned before, having a roadmap in your mind about what the myeloma therapy for you might look like going forward, wherever you are in your disease state, is always important because it gives you time to sort of think about it, learn about it, prepare for it.  

Some of these therapies really require an effort from the patient and their caregivers because, for example, for CAR-T cells. If you’re not near a center, you may have to relocate for a month.  

And it’s very difficult, and we fully understand that and try to help as much as we can, but that’s the kind of commitment that it takes. So, talking to your physician, obviously content like this, reviewing this as much as you can. Online patient support groups are great because you learn from the other patients’ experiences. So, the good news now is we have so many channels of communication, but you have to in a way, in the end, discuss with your physician and verify things you may find on your own.  

Katherine:

Exactly, yeah. You want to make sure you’re getting facts rather than fiction.  

Dr. Nadeem:

Yeah. That’s right. 

Evolving Myeloma Treatment Options | Bispecific Antibody Therapy

Evolving Myeloma Treatment Options | Bispecific Antibody Therapy from Patient Empowerment Network on Vimeo.

What are bispecific antibodies, and how are they advancing myeloma care? Dr. Omar Nadeem of Dana-Farber Cancer Institute discusses the role of this new therapy in myeloma care, shares an update on ongoing bispecific antibody research, and compares this treatment to CAR T-cell therapy.

Dr. Omar Nadeem is the Clinical Director of the Myeloma Immune Effector Cell Therapy Program and Associate Director of the Multiple Myeloma Clinical Research Program at the Dana-Farber Cancer Institute. Learn more about Dr. Nadeem.

Download Resource Guide

See More from Evolve Myeloma

Related Resources:

What Is the Role of Bispecific Antibody Therapy in the Future of Myeloma Care?

What Is the Role of Bispecific Antibody Therapy in the Future of Myeloma Care?

Myeloma Research Highlights From ASH 2023

Myeloma Research Highlights From ASH 2023 

What Should Myeloma Patients Ask About Developing Research

What Should Myeloma Patients Ask About Developing Research?

Transcript:

Katherine:

Well, another therapy that has emerged in myeloma is bispecific antibodies. What patient type is this therapy right for? 

Dr. Nadeem:

So, bispecific antibodies are great because they’re off the shelf. What that means is that CAR-T cells, we first have to collect the T cells and we then have to send them off to be manufactured, and that manufacturing process can take up to a month, sometimes even longer, for some of the current available CAR-T products.   

And then, after the cells are returned to the facility, we then give usually three days of chemotherapy to try to suppress some of the immune systems of the patients. So, that way, when the cells are administered, they can expand robustly and do essentially what they need to do. 

So, that whole logistical process can take a couple of months by the time you identify somebody for CAR-T cells and then, from that moment until they can actually be treated. With bispecific antibodies, if we think somebody’s ready to go, you can basically get it as soon as we can have somebody ready to go either in our clinic or on the in-patient facility.

So, they’re much easier. They also utilize T cells to attack myeloma cells. We now have three approved bispecific antibodies. Two of them are targeting BCMA, the same exact target that we have in CAR-T cells, and one of them is now targeting a new target called GPRC5D, which is also highly expressed on myeloma cells.  

So, having all these bispecific antibodies available is excellent because patients can have access to them a lot faster and now we’re trying to answer the question of sequencing. Can you give bispecific antibodies after CAR-T cells for example? Can you give one bispecific antibody after another, especially if there’s a different target that we now have available?  

As a whole, though, bispecific antibodies tend to have lower response rates than CAR-T cells, particularly cilta-cel (Carvykti), which is cilta-cel that has a very high response rate of close to 100 percent.  

Most bispecific antibodies have response rates somewhere around 70 or so percent, so about two-thirds of patients respond to these therapies, again, in that fifth line or four or more lines of therapy. So, in that space, that’s the response rate. And across the board, generally speaking, patients benefit from these bispecific antibodies approximately a year on average. Some of the studies have shown longer benefit, and it also depends somewhat on response to therapy.  

Patients that have a really deep response can go even way longer than that. So, it is quite mixed in terms of how somebody may do on these bispecific antibodies, but those are the numbers.  

Katherine:

Well, it sounds like bispecific antibodies have really transformed myeloma treatment options.  

Dr. Nadeem:

Absolutely, and what goes hand in hand in this.  

I mentioned the logistics of CAR T, but then there’s also the supply and availability of CAR-T cells. Since the approval, the demand for CAR-T cells has been very high because of all these excellent results, but the supply really hasn’t been there. So, even at a center as busy as ours, we can only treat a handful of patients with CAR T-cell therapies compared to bispecific antibodies, where that is essentially an injection similar to many other approved myeloma agents that you can just readily treat patients with. So, CAR-T cells, while I think, again, have higher efficacy, with that comes slightly higher toxicity as well. It’s a very different kind of treatment program.  

And then, patients get a treatment-free interval, which you don’t see yet with bispecific antibodies. On the other hand, bispecific antibodies are readily available, slightly lower response rates, slightly lower toxicity when it comes to at least the traditional T-cell directing toxicities. And then you have, again, the readily available nature of it, which I think is hugely beneficial for patients.  

Katherine:

You talked about some specifics regarding bispecific antibodies, but are there updates in bispecific antibody research that you’d like to share? 

Dr. Nadeem:

Yeah, so, again, kind of following the theme of what we just said about CAR-T cells, can you bring these antibody therapies earlier? And there’s ongoing trials now looking at it in newly diagnosed multiple myeloma and early relapses, and then we presented our data at ASH this previous year looking at it in high-risk smoldering myeloma. We treated patients with teclistimab (Tecvayli), which is a BCMA bispecific antibody that is approved for relapse refractory patients. And what we demonstrated in that study is that people that got teclistimab had a 100 percent response rate with an MRD-negative rate. So, kind of as deep of a response as we can measure, also at 100 percent.  

So, this is something that we had not seen before. When their immune systems are a lot healthier, they may benefit more. So, hopefully we’ll see confirmation of these results in other trials.  

Particularly in the newly diagnosed space because we do think that these antibody therapies have such huge potential to treat patients, and then hopefully we’ll have durable responses. So, I do think that some of this paradigm may shift over the next few years, and then there’s also combinations that are currently being studied: combinations with traditional myeloma therapies, such as monoclonal antibodies, other immunomodulatory agents, or proteasome inhibitors. All these combination trials are now ongoing to see can you improve upon some of those numbers that I highlighted before with single-agent bispecific antibody therapy. 

Katherine:

Can you share the pros and cons of bispecifics and how it compares to CAR T?” 

Dr. Nadeem:

Yeah. I think we mentioned earlier that as a whole, they’re very similar. They’re both T-cell re-directing therapies, in many circumstances, with the same exact target of the myeloma cell, but because this isn’t a cell infusion – this is a cell injection – that you receive that redirects your T cells to the myeloma cells, you tend to see a little bit of a lower toxicity signal when it comes to the cytokine release syndrome incidents and severity. You see lower neurological toxicity, usually, than you do with CAR  T-cell products as a whole.  

With that comes slightly lower efficacy than you see with at least some of our CAR-T products, but if you respond to therapy, then the durability of response can be as good as you can achieve with CAR-T cells. One thing to note about the bispecifics, though, is that it is continuous therapy, so you are getting it on some regular schedule. Right now the approval is for it to be given weekly and then go to every two weeks after six months of therapy if you’re basically in a good response.   

A lot of that is to try to mitigate the risk of infection. So, that is one of the biggest things that we have seen with bispecifics more so than CAR-T cells. Because it is continuous administration of these therapies, that really suppresses your immune system significantly, and infection rates are quite high. So, we typically give other ways to try to mitigate that using immunoglobulin infusions to try to boost up your immune system. Typically, we do that once a month for patients, making sure you’re on the right prophylactic medications and then really adjusting the therapy and the schedule to you depending on your tolerability.  

So, as we said before, it’s an excellent option. I think bispecific antibodies are going to be the mainstay of myeloma therapy going forward because CAR-T cells, again, we can’t really treat everybody with CAR-T cells just simply because of the dynamics of how the process is. So, having the bispecific antibodies available for patients is excellent.   

Evolving Myeloma Treatment Options | CAR T-Cell Therapy

Evolving Myeloma Treatment Options | CAR T-Cell Therapy from Patient Empowerment Network on Vimeo.

What is CAR T-cell therapy, and who is it right for? Dr. Omar Nadeem of Dana-Farber Cancer Institute discusses the role of this therapy in myeloma care and shares an update in ongoing CAR T-cell therapy clinical trial research.

Dr. Omar Nadeem is the Clinical Director of the Myeloma Immune Effector Cell Therapy Program and Associate Director of the Multiple Myeloma Clinical Research Program at the Dana-Farber Cancer Institute. Learn more about Dr. Nadeem.

Download Resource Guide

See More from Evolve Myeloma

Related Resources:

Clinical Trials for Myeloma Treatment | Essential Information for Patients

Clinical Trials for Myeloma Treatment | Essential Information for Patients

Personalized Medicine for Myeloma Treatment | What Patients Should Know

Personalized Medicine for Myeloma Treatment | What Patients Should Know

What Should Myeloma Patients Ask About Developing Research

What Should Myeloma Patients Ask About Developing Research?

Transcript:

Katherine:

Well, I’d like to talk about some new and emerging therapies in myeloma, starting with CAR T-cell therapy. Can you talk about who this treatment option might be appropriate for?  

Dr. Nadeem:

So, yeah, just to kind of give folks background, CAR T-cell therapy is a form of immunotherapy, where we take out an individual’s T-cells and then re-program them, essentially, to recognize myeloma cells. Right now there’s two approved CAR-T products for multiple myeloma, both in the relapse refractory setting. It’s really for patients that have had four or more lines of therapy.  

So, that’s a lot of different combinations that we currently have available. Those therapies stop working before patients are actually eligible for CAR-T cells at the moment. Both of these CAR T-cell products have been gamechangers in terms of improving prognosis for patients.  

The good thing about CAR-T cells is that it is a one-and-done treatment. So, patients, when they go through that initial phase of therapy, they are then off therapy, although we are now starting to study certain therapies that we may administer after CAR-T cells to get them to last even longer than they currently do, but that’s still in, for example, that’s one of the clinical trials or many of the clinical trials that are currently ongoing now, to try to answer that question.  

So, a lot of patients can be eligible for CAR-T cells. They have to have the prerequisite amount of therapies. Again, there are some sort of baseline fitness characteristics that we look at for patient’s ability to tolerate it. But as a whole, I consider CAR T-cell therapy more broadly applicable to myeloma patients than compared to, let’s say, a stem cell transplant.  

Katherine:

How has this therapy revolutionized myeloma care? 

Dr. Nadeem:

Yeah, before the first approval, now a few years ago, in this space we didn’t really have anything like this to offer patients. So, many of the combinations and other compounds that were in clinical trials would have a response rate somewhere around, let’s say, 30 percent. So, 30 percent of patients may respond to that therapy in that space, and that may only last a few months, and that was considered successful not that long ago. Now, with CAR T-cell therapy and bispecific antibodies, these therapies are highly efficacious.  

You see response rates of 70 to 100 percent in some of these immunotherapies, and what that’s translating into is patient’s disease staying away for a year or two years, even three years in some of these clinical trials. And again, this is completely unprecedented compared to what we had before.  

Katherine:

I understand that there are a number of clinical trials for different types of CAR T, or even using it earlier in the disease. Can you share updates in CAR T-cell therapy research? 

Dr. Nadeem:

Yeah, so, exactly as you pointed out, there have been trials already, actually, that have been completed, Phase III studies looking at CAR T-cell therapies in earlier relapses.  So, patients that have had either one of two lines of therapy. 

Both our CAR-T therapies have been compared to standard of care in that space and have shown superiority, and this is something that we all have been kind of waiting for to see if you deploy it earlier, perhaps you’re going to see even greater benefit, and that seems to be the case in some of these trials, and now we’re awaiting, hopefully, approval of some of these CAR T-cell therapies to be administered earlier because in fifth line, it’s very different than treating patients in second or third line, which I think will really vastly improve our ability to deliver this therapy to many patients, as it can be quite challenging for patients that are in fifth line, to allow them to go through the process of CAR-T cells and then having them be administered.  

I was looking at it head-to-head with stem cell transplant, as I mentioned before, and this is in the context of quadruplet and induction therapy followed by either CAR-T cells or stem cell transplant, and then followed by maintenance therapy. So, really trying to see if I can overcome what we typically have achieved with stem cell transplantation.  

We also are doing some studies even before that. So, patients, again, in high-risk smoldering myeloma, which we know have an increased risk of developing newly diagnosed disease in the next few years, perhaps that could be the time where we can give some of these immunotherapies, and that’s some work that we have going on at our center. 

Clinical Trials for Myeloma Treatment | Essential Information for Patients

Clinical Trials for Myeloma Treatment | Essential Information for Patients from Patient Empowerment Network on Vimeo.

How do clinical trials advance treatment options for myeloma? Dr. Omar Nadeem discusses the important role of clinical trials in improving patient care, key questions to ask your care team about trial participation, and the benefits of seeing a myeloma specialist. 

Dr. Omar Nadeem is the Clinical Director of the Myeloma Immune Effector Cell Therapy Program and Associate Director of the Multiple Myeloma Clinical Research Program at the Dana-Farber Cancer Institute. Learn more about Dr. Nadeem.

Download Resource Guide

See More from Evolve Myeloma

Related Resources:

Next Generation Myeloma Treatment Options

Next Generation Myeloma Treatment Options

Personalized Medicine for Myeloma Treatment | What Patients Should Know

Personalized Medicine for Myeloma Treatment | What Patients Should Know

Myeloma Research Highlights From ASH 2023

Myeloma Research Highlights From ASH 2023 

Transcript:

Katherine:

I’d like to start with the importance of a patient’s healthcare team. What are the benefits to seeking care with a myeloma specialist, even if it’s just for a second opinion or a consult? 

Dr. Nadeem:

Yeah, so, myeloma is a little less than 2 percent of all cancers, and it’s the second most common blood cancer, so certainly not rare. With that being said, if you go to a general community practice, they don’t typically see too, too many patients with this disease. So, alongside that, we have so many different treatment options and combinations and these, as I mentioned, immune therapies.  

And other therapies that are only actually carried out at academic centers for now, such as stem cell transplants, and CAR T-cell therapy.  

I think it’s important to kind of meet with an academic provider just to get a sense of what the patient may be facing, both in that immediate time, but also in the future, because a lot of myeloma therapy is lifelong. And in that case, you do have to come up with a plan for your whole treatment in a way early. So, it’s important to kind of one: hear it from another person, and then two: really sort of figure out what the outlook would look like for the individual patient.  

With that being said, many of our myeloma regimens that are approved can very easily be given at the local provider, and that’s usually our preference, for patients to be treated closer to home. So, ultimately, this is another way for patients to get input about their treatment program, but also talk about the future.  

Katherine:

That makes sense. Specialists at academic medical centers are typically more involved in research and clinical trials. 

And patient participation is essential to advancing medicine. So, how do clinical trials impact myeloma care? 

Dr. Nadeem:

Well, everything that we have available today for myeloma therapy was once in a clinical trial. So, all these promising therapies usually start in early phase studies and move on to Phase II and Phase III studies, and then those are the ones that the FDA uses to approve a particular combination.  

So, it all depends on kind of where someone is in their disease course. It also kind of depends on what their preferences may be in terms of taking on something that is beyond standard of care. So, as part of any clinical trial in whatever phase it may be, whether its newly diagnosed multiple myeloma, even smoldering myeloma, which is one step before that, relapsed/refractory myeloma…  

At each step of the way, there are clinical trials that are there trying to improve upon what’s already out here, right? So, we are, despite all these amazing advances, unfortunately, the disease is still not curable for a vast majority of patients.  

In that case, how do we move to that cure, or how do we kind of advance the disease even beyond this? And a clinical trial is a way to do that.  

Katherine:

What type of patient is most appropriate for a clinical trial? 

Dr. Nadeem:

So, there are criteria that each clinical trial uses in terms of eligibility. Some of that has to do with the disease characteristic itself, kind of where somebody is in their disease course, but many times it’s also patients’ fitness, organ status in terms of kidney function, their blood count to some extent, heart function, etcetera. There are some sort of minimal prerequisite guidelines that we have to enroll patients in trials. So, it really, again, depends on where somebody is in their disease course and what they may be willing to take on beyond what may be offered to them as part of standard of care.  

Katherine:

What questions should patients be asking if they’re entrusted in participating in a clinical trial? 

Dr. Nadeem:

I think the important thing is to sort of first recognize what’s available to them as part of standard of care and then what the clinical trial is trying to answer.  

So, for example, if it’s newly diagnosed multiple myeloma, we now have quadruplet regimens that we give to patients at the time of their diagnosis, and then the next natural question for eligible patients that now comes up is whether they should do a stem cell transplant or not.  

And alongside that goes with all these advances in immune therapies, such as CAR T-cell therapies and bispecific antibodies. And there are now trials looking at those therapies and comparing them, for example, to stem cell transplant to try to answer the question “Can we get even beyond something like a stem cell transplant?”  

So, that’s one example of a trial where a patient may be interested in saying “Okay, well, a transplant may be my standard path, but what if I try to enroll in this study and get randomized, for example, to the CAR-T arm? Then, perhaps, I’m getting access to some of these therapies early and maybe that’s going to improve my outcomes.” 

Evolving Myeloma Treatment Options: How You Can Access Cutting-Edge Care

Evolving Myeloma Treatment Options: How You Can Access Cutting-Edge Care from Patient Empowerment Network on Vimeo.

With the quickly evolving landscape of myeloma treatment and care, it’s important to work with your healthcare team to determine a care plan. In this program, Dr. Omar Nadeem discusses the latest updates in research and clinical trials, the role of new and emerging therapies– including bispecific antibodies and CAR T-cell therapy–and shares advice for accessing quality myeloma care.

Dr. Omar Nadeem is the Clinical Director of the Myeloma Immune Effector Cell Therapy Program and Associate Director of the Multiple Myeloma Clinical Research Program at the Dana-Farber Cancer Institute. Learn more about Dr. Nadeem.

Download Resource Guide

See More from Evolve Myeloma

Related Resources:

What Is the Role of Bispecific Antibody Therapy in the Future of Myeloma Care?

What Is the Role of Bispecific Antibody Therapy in the Future of Myeloma Care?

Questions to Ask Your Doctor About CAR T-Cell Therapy

Questions to Ask Your Doctor About CAR T-Cell Therapy

Considering CAR T-Cell Therapy for Myeloma_ Key Questions to Ask Your Doctor

Considering CAR T-Cell Therapy for Myeloma? Key Questions to Ask Your Doctor 

Transcript:

Katherine:

Hello, and welcome. I’m your host, Katherine Banwell. As patients collaborate on treatment decisions with their healthcare team, it’s important that they understand all of their options and how these options may be impacted by research developments. That’s why the Patient Empowerment Network created the Evolve series, to arm you with the latest information and help you feel empowered and confident during conversations about your myeloma care.  

In today’s program, we’re going to hear from an expert in the field about the evolving treatment landscape and discuss how you can play an active role in your care. 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 may be best for you. Well, let’s meet our guest today. Joining us is Dr. Omar Nadeem. Dr. Nadeem, welcome. Would you please introduce yourself.  

Dr. Nadeem:

Thank you. Hi, everyone. My name is Omar Nadeem from the Dana-Farber Cancer Institute. It’s my pleasure to be here.  

Katherine:

Thank you so much for joining us today. Before we get into our discussion, would you share with the audience how the field of myeloma care has changed over the course of your career?   

Dr. Nadeem:

Yeah, and things are changing so rapidly. My career started after my training in 2015 and at that time, daratumumab (Darzalex) just had its approval in relapsed/refractory multiple myeloma. That, along with several other monoclonal antibodies a few immunomodulatory drugs and proteasome inhibitors.  

At that time, it felt like myeloma was at the forefront of significant advances and change in practice, which it was. Little did we know that we were right around the corner with the next renaissance of myeloma therapy, which is these immunotherapies that have been approved over the last three to four years now. So, safe to say things are changing so, so fast and it’s leading to excellent outcomes for patients.  

Katherine:

Yeah, it’s great news. So positive. I’d like to start with the importance of a patient’s healthcare team. What are the benefits to seeking care with a myeloma specialist, even if it’s just for a second opinion or a consult? 

Dr. Nadeem:

Yeah, so, myeloma is a little less than 2 percent of all cancers, and it’s the second most common blood cancer, so certainly not rare. With that being said, if you go to a general community practice, they don’t typically see too, too many patients with this disease. So, alongside that, we have so many different treatment options and combinations and these, as I mentioned, immune therapies.  

And other therapies that are only actually carried out at academic centers for now, such as stem cell transplants, and CAR T-cell therapy. I think it’s important to kind of meet with an academic provider just to get a sense of what the patient may be facing, both in that immediate time, but also in the future, because a lot of myeloma therapy is lifelong. And in that case, you do have to come up with a plan for your whole treatment in a way early. So, it’s important to kind of one: hear it from another person, and then two: really sort of figure out what the outlook would look like for the individual patient.  

With that being said, many of our myeloma regimens that are approved can very easily be given at the local provider, and that’s usually our preference, for patients to be treated closer to home. So, ultimately, this is another way for patients to get input about their treatment program, but also talk about the future.   

Katherine:

That makes sense. Specialists at academic medical centers are typically more involved in research and clinical trials. 

And patient participation is essential to advancing medicine. So, how do clinical trials impact myeloma care? 

Dr. Nadeem:

Well, everything that we have available today for myeloma therapy was once in a clinical trial. So, all these promising therapies usually start in early phase studies and move on to Phase II and Phase III studies, and then those are the ones that the FDA uses to approve a particular combination.  

So, it all depends on kind of where someone is in their disease course. It also kind of depends on what their preferences may be in terms of taking on something that is beyond standard of care. So, as part of any clinical trial in whatever phase it may be, whether its newly diagnosed multiple myeloma, even smoldering myeloma, which is one step before that, relapsed/refractory myeloma.

At each step of the way, there are clinical trials that are there trying to improve upon what’s already out here, right? So, we are, despite all these amazing advances, unfortunately, the disease is still not curable for a vast majority of patients.  

In that case, how do we move to that cure, or how do we kind of advance the disease even beyond this? And a clinical trial is a way to do that.  

Katherine:

What type of patient is most appropriate for a clinical trial? 

Dr. Nadeem:

So, there are criteria that each clinical trial uses in terms of eligibility. Some of that has to do with the disease characteristic itself, kind of where somebody is in their disease course, but many times it’s also patients’ fitness, organ status in terms of kidney function, their blood count to some extent, heart function, etcetera. There are some sort of minimal prerequisite guidelines that we have to enroll patients in trials. So, it really, again, depends on where somebody is in their disease course and what they may be willing to take on beyond what may be offered to them as part of standard of care.  

Katherine:

What questions should patients be asking if they’re entrusted in participating in a clinical trial? 

Dr. Nadeem:

I think the important thing is to sort of first recognize what’s available to them as part of standard of care and then what the clinical trial is trying to answer.  

So, for example, if it’s newly diagnosed multiple myeloma, we now have quadruplet regimens that we give to patients at the time of their diagnosis, and then the next natural question for eligible patients that now comes up is whether they should do a stem cell transplant or not.  

And alongside that goes with all these advances in immune therapies, such as CAR T-cell therapies and bispecific antibodies. And there are now trials looking at those therapies and comparing them, for example, to stem cell transplant to try to answer the question “Can we get even beyond something like a stem cell transplant?” 

So, that’s one example of a trial where a patient may be interested in saying “Okay, well, a transplant may be my standard path, but what if I try to enroll in this study and get randomized, for example, to the CAR-T arm? Then, perhaps, I’m getting access to some of these therapies early and maybe that’s going to improve my outcomes.” 

Katherine:

Well, I’d like to talk about some new and emerging therapies in myeloma, starting with CAR T-cell therapy. Can you talk about who this treatment option might be appropriate for? 

Dr. Nadeem:

So, yeah, just to kind of give folks background, CAR T-cell therapy is a form of immunotherapy, where we take out an individual’s T-cells and then re-program them, essentially, to recognize myeloma cells. Right now there’s two approved CAR-T products for multiple myeloma, both in the relapse refractory setting. It’s really for patients that have had four or more lines of therapy.  

So, that’s a lot of different combinations that we currently have available. Those therapies stop working before patients are actually eligible for CAR-T cells at the moment. Both of these CAR T-cell products have been gamechangers in terms of improving prognosis for patients.  

The good thing about CAR-T cells is that it is a one-and-done treatment. So, patients, when they go through that initial phase of therapy, they are then off therapy, although we are now starting to study certain therapies that we may administer after CAR-T cells to get them to last even longer than they currently do, but that’s still in, for example, that’s one of the clinical trials or many of the clinical trials that are currently ongoing now, to try to answer that question.   

So, a lot of patients can be eligible for CAR-T cells. They have to have the prerequisite amount of therapies. Again, there are some sort of baseline fitness characteristics that we look at for patient’s ability to tolerate it. But as a whole, I consider CAR T-cell therapy more broadly applicable to myeloma patients than compared to, let’s say, a stem cell transplant.   

Katherine:

How has this therapy revolutionized myeloma care? 

Dr. Nadeem:

Yeah, before the first approval, now a few years ago, in this space we didn’t really have anything like this to offer patients. So, many of the combinations and other compounds that were in clinical trials would have a response rate somewhere around, let’s say, 30 percent. So, 30 percent of patients may respond to that therapy in that space, and that may only last a few months, and that was considered successful not that long ago. Now, with CAR T-cell therapy and bispecific antibodies, these therapies are highly efficacious.  

You see response rates of 70 to 100 percent in some of these immunotherapies, and what that’s translating into is patient’s disease staying away for a year or two years, even three years in some of these clinical trials. And again, this is completely unprecedented compared to what we had before.   

Katherine:

I understand that there are a number of clinical trials for different types of CAR T, or even using it earlier in the disease. Can you share updates in CAR T-cell therapy research? 

Dr. Nadeem:

Yeah, so, exactly as you pointed out, there have been trials already, actually, that have been completed, Phase III studies looking at CAR T-cell therapies in earlier relapses.  So, patients that have had either one of two lines of therapy. 

Both our CAR-T therapies have been compared to standard of care in that space and have shown superiority, and this is something that we all have been kind of waiting for to see if you deploy it earlier, perhaps you’re going to see even greater benefit, and that seems to be the case in some of these trials, and now we’re awaiting, hopefully, approval of some of these CAR T-cell therapies to be administered earlier because in fifth line, it’s very different than treating patients in second or third line, which I think will really vastly improve our ability to deliver this therapy to many patients, as it can be quite challenging for patients that are in fifth line, to allow them to go through the process of CAR-T cells and then having them be administered.  

I was looking at it head-to-head with stem cell transplant, as I mentioned before, and this is in the context of quadruplet and induction therapy followed by either CAR-T cells or stem cell transplant, and then followed by maintenance therapy. So, really trying to see if I can overcome what we typically have achieved with stem cell transplantation. 

We also are doing some studies even before that. So, patients, again, in high-risk smoldering myeloma, which we know have an increased risk of developing newly diagnosed disease in the next few years, perhaps that could be the time where we can give some of these immunotherapies, and that’s some work that we have going on at our center. 

Katherine:

Well, another therapy that has emerged in myeloma is bispecific antibodies. What patient type is this therapy right for? 

Dr. Nadeem:

So, bispecific antibodies are great because they’re off the shelf. What that means is that CAR-T cells, we first have to collect the T cells and we then have to send them off to be manufactured, and that manufacturing process can take up to a month, sometimes even longer, for some of the current available CAR-T products. And then, after the cells are returned to the facility, we then give usually three days of chemotherapy to try to suppress some of the immune systems of the patients. So, that way, when the cells are administered, they can expand robustly and do essentially what they need to do. 

So, that whole logistical process can take a couple of months by the time you identify somebody for CAR-T cells and then, from that moment until they can actually be treated. With bispecific antibodies, if we think somebody’s ready to go, you can basically get it as soon as we can have somebody ready to go either in our clinic or on the in-patient facility. So, they’re much easier. They also utilize T cells to attack myeloma cells. We now have three approved bispecific antibodies. Two of them are targeting BCMA, the same exact target that we have in CAR-T cells, and one of them is now targeting a new target called GPRC5D, which is also highly expressed on myeloma cells.  

So, having all these bispecific antibodies available is excellent because patients can have access to them a lot faster and now we’re trying to answer the question of sequencing. Can you give bispecific antibodies after CAR-T cells for example? Can you give one bispecific antibody after another, especially if there’s a different target that we now have available? 

As a whole, though, bispecific antibodies tend to have lower response rates than CAR-T cells, particularly Cilta-cel (Carvykti), which is cilta-cel that has a very high response rate of close to 100 percent.  

Most bispecific antibodies have response rates somewhere around 70 or so percent, so about two-thirds of patients respond to these therapies, again, in that fifth line or four or more lines of therapy. So, in that space, that’s the response rate. And across the board, generally speaking, patients benefit from these bispecific antibodies approximately a year on average. Some of the studies have shown longer benefit, and it also depends somewhat on response to therapy.  

Patients that have a really deep response can go even way longer than that. So, it is quite mixed in terms of how somebody may do on these bispecific antibodies, but those are the numbers.  

Katherine:

Well, it sounds like bispecific antibodies have really transformed myeloma treatment options.  

Dr. Nadeem:

Absolutely, and what goes hand in hand in this.  

I mentioned the logistics of CAR T, but then there’s also the supply and availability of CAR-T cells. Since the approval, the demand for CAR-T cells has been very high because of all these excellent results, but the supply really hasn’t been there. So, even at a center as busy as ours, we can only treat a handful of patients with CAR T-cell therapies compared to bispecific antibodies, where that is essentially an injection similar to many other approved myeloma agents that you can just readily treat patients with. So, CAR-T cells, while I think, again, have higher efficacy, with that comes slightly higher toxicity as well. It’s a very different kind of treatment program.  

And then, patients get a treatment-free interval, which you don’t see yet with bispecific antibodies. On the other hand, bispecific antibodies are readily available, slightly lower response rates, slightly lower toxicity when it comes to at least the traditional T-cell directing toxicities. And then you have, again, the readily available nature of it, which I think is hugely beneficial for patients.  

Katherine:

You talked about some specifics regarding bispecific antibodies, but are there updates in bispecific antibody research that you’d like to share? 

Dr. Nadeem:

Yeah, so, again, kind of following the theme of what we just said about CAR-T cells, can you bring these antibody therapies earlier? And there’s ongoing trials now looking at it in newly diagnosed multiple myeloma and early relapses, and then we presented our data at ASH this previous year looking at it in high-risk smoldering myeloma. We treated patients with teclistimab (Tecvayli), which is a BCMA bispecific antibody that is approved for relapse refractory patients. And what we demonstrated in that study is that people that got Teclistimab had a 100 percent response rate with an MRD-negative rate. So, kind of as deep of a response as we can measure, also at 100 percent.  

So, this is something that we had not seen before. When their immune systems are a lot healthier, they may benefit more. So, hopefully we’ll see confirmation of these results in other trials.  

Particularly in the newly diagnosed space because we do think that these antibody therapies have such huge potential to treat patients, and then hopefully we’ll have durable responses. So, I do think that some of this paradigm may shift over the next few years, and then there’s also combinations that are currently being studied: combinations with traditional myeloma therapies, such as monoclonal antibodies, other immunomodulatory agents, or proteasome inhibitors. All these combination trials are now ongoing to see can you improve upon some of those numbers that I highlighted before with single-agent bispecific antibody therapy. 

Katherine:

Oh, I was just going to ask you the next question, which is are there other emerging myeloma therapies that are showing promise? 

Dr. Nadeem:

Yes. So, I think over the last few years, most of the buzz has been with these immunotherapies. And, again, more work to be done there to see whether combinations, different schedules, different targets, different types, will show more and more benefit in each of these myeloma disease settings.  

But we also have simultaneous development of other agents that are not in this sort of immunotherapy T-cell redirecting therapy realm. We have newer versions of our classic immunomodulatory drugs, such as lenalidomide (Revlimid) or pomalidomide (Pomalyst).  

We now have their next generation agents, called CELMoD drugs and there’s two of them in development. One of them is called iberdomide; one is called mezigdomide.  

These are, again, kind of building up on the success of some of these previous therapies that are kind of cornerstone therapies for myeloma patients and because these are essentially better agents, they’re more targeted, and they also have greater response rates as single agents and as combinations.  

We’re hoping that these would be approved in the not-so-distant future and then perhaps will replace some of these immunomodulatory drugs that we have currently utilized in newly diagnosed and relapsed myeloma. Essentially what this means is things are just getting better and better and better as we get newer versions of some of these therapies. So, those are, I would say, kind of next in line in terms of hopeful approvals.  

And then we’ll add to some of the options that we have for myeloma patients.  

Katherine:

How can patients and care partners stay informed about the latest myeloma research? 

Dr. Nadeem:

Yeah, it’s a lot of moving parts all the time. From one six-month interval to the next, you tend to have nowadays perhaps some drug approvals, which is amazing, but if not updates of all these sort of combination trials, etcetera, of where these things are going. I think kind of talking to your physician, obviously, about some of these updates is really critical. As I mentioned before, having a roadmap in your mind about what the myeloma therapy for you might look like going forward, wherever you are in your disease state, is always important because it gives you time to sort of think about it, learn about it, prepare for it.  

Some of these therapies really require an effort from the patient and their caregivers because, for example, for CAR-T cells. If you’re not near a center, you may have to relocate for a month.  

And it’s very difficult, and we fully understand that and try to help as much as we can, but that’s the kind of commitment that it takes. So, talking to your physician, obviously content like this, reviewing this as much as you can. Online patient support groups are great because you learn from the other patients’ experiences. So, the good news now is we have so many channels of communication, but you have to in a way, in the end, discuss with your physician and verify things you may find on your own.   

Katherine:

Exactly, yeah. You want to make sure you’re getting facts rather than fiction.  

Dr. Nadeem:

Yeah. That’s right.  

Katherine:

Well, Dr. Nadeem, we’ve been hearing the term personalized medicine more frequently in recent years. How would you define personalized medicine for myeloma, and how can patients access this type of care? 

Dr. Nadeem:

Yeah, personalized medicine or precision medicine is a term that we’ve really sort of used for many oncologic conditions over the last decade or so. I would say, for multiple myeloma, in terms of identifying a target within the myeloma cell that’s unique to the patient.  

And then deploying a certain therapy to that patient because of that target is still lacking. We do have one example where patients have, for example, an 11;14 translocation, which we see in about 15 percent of myeloma patients.  

There’s an agent called venetoclax (Venclexta) that is very active against that particular cohort of patients, although that is still not approved to be used, but that’s one example where that agent specifically benefits that type of myeloma. Other than that, most of the therapies that we have benefit essentially everybody with myeloma, which is great, but it’s not so personalized.  

Where I would say there’s the most personalization happening now, at least in my practice, is looking at which types of therapies an individual patient may receive. What I mean by that is if somebody’s in an excellent response, with quadruplet-based induction therapy, I have a very real discussion with them about the pros and cons of stem cell transplant. We make those decisions in real time depending on how the patient doing, depending on how their response is.  

And then kind of deciding a whole kind of what are the kind of risks and benefits and what makes sense for that individual patient. Similarly, when you go on to maintenance therapy, maintenance therapy means that after you’ve gone through the initial phase of your myeloma therapy and the disease is under control, what type of therapy can we keep you on to keep it under control for as long as possible? Historically, that has been lenalidomide or Revlimid. Now we’re adding drugs such as daratumamab and other agents to Revlimid to see if that can further prolong the response to that initial therapy.  

So, all those decisions are so individualized that you have to discuss with your provider what makes sense for you and what are the pros and cons of doing one approach versus the other.  

Katherine:

Well, if we’re talking about in-depth testing, how do the results of that testing affect treatment options? 

Dr. Nadeem:

So, right now we use conventional blood tests to get a sense of response in the vast majority of patients. That includes the serum protein electrophoresis and the serum free light chain assay.  

Most patients have detectable levels of these proteins, abnormal proteins in the blood at diagnosis and then you can follow them using a blood test. There’s a subset of patients that have disease only that shows up on scans. So, we then kind of incorporate some of those scans and then, also, utilize the bone marrow results both in the beginning and in subsequent analyses to kind of give a big-picture composite response assessment for that particular patient. Nowadays, there are also other tools that we’re using, such as MRD, or minimal residual disease.  

That is a test that is done on a bone marrow biopsy to determine, if you don’t have detectable protein in the blood, do you have myeloma cells present at the deepest level possible? And if you do versus if you don’t, trials have shown that there is a difference in terms of prognosis. Now, while that hasn’t fully been utilized yet to make treatment decisions in patients that are not on clinical trials, we do get prognostic information out of it, and nowadays, more and more of those trials are using these MRD tests to determine what to do with treatment.   

And I think that’s how it’s going to be in the future. So, having those extra tests available but, again, important to discuss with your provider what is the utility of this test. How are we going to use this information for your individual case to make some decisions? 

Katherine:

What questions should patients be asking their provider about a proposed treatment plan?  

Dr. Nadeem:

Yeah. I think because myeloma therapy’s so nuanced and much of this is still in clinical trials or under investigation about what to do with some of these results, I would say, as a whole, it’s important to know which tests the physician looks at to determine how you’re doing, and kind of what their assessment of that result is. So, for example, if somebody’s had a 50 percent reduction in the amount of abnormal protein in the blood, is that sufficient, or should we be aiming for a number that’s much higher than that? 

Some of that depends on kind of where they are in their treatment course, but that’s a very sort of reasonable question to ask your physician is that where do you see my response now, let’s say six months into therapy, and is this adequate, and what is now, after we have all this information, what is my roadmap going forward to try to keep this disease in check? 

Katherine:

Yeah. Well, that’s great advice, Dr. Nadeem. Thank you. PEN has also created a downloadable office visit planner to help you organize your thoughts and communicate effectively with your healthcare team. You can find these at Powerfulpatients.org/myeloma.  

I’d like to turn to self-advocacy, Dr. Nadeem. Why is it so important that patients engage in their care treatment decisions? 

Dr. Nadeem:

Yeah. As I mentioned, myeloma therapy is so individualized now and we can sit here, look at the trial data, get very into the weeds and technical about this therapy with this approach as X or Y higher response rate.   

Or MRD-negative rate, but in reality, we’re dealing with people and we’re dealing with people that have lives. They have all their priorities, and until you share that with us, it’s very difficult for us to know exactly what’s important to you. So, what I may consider to be kind of the “best therapy” for you may not make sense for you because of all the priorities that you may have, and I think it’s so important to advocate for yourself and not be afraid to bring that up to your physician because I think many patients kind of hold that stuff in for a long time because they don’t want it to impact their care. But I would argue the other way around.  

Tell us. Tell us exactly what you prioritize. Tell us if you can’t be out of commission for work for X amount of time because of a stem cell transplant. We now have options. We now have options for patients because of all these amazing new therapies for myeloma and we can come up with a very individualized treatment plan for you based on your priorities.  

Katherine:

If a patient is feeling like they’re not getting the best care or they’re uncomfortable with the care they’re receiving, what steps should they take to change that?  

Dr. Nadeem:

Yeah, I think that’s very difficult because this is a complex system. Medical systems are getting even more and more complex. They’re busy. Everybody’s busy: busy offices, labs, radiology. We’re all feeling that. It doesn’t matter where you are. So, I think it’s important to raise those concerns, number one, to your practice that you’re being seen at because they would like to see that feedback, right? So, kind of see what is something that they can perhaps improve upon. I think it’s always important, like we just said, to advocate for yourself and raise some of these issues and not be afraid of that.  

We’re all in this together, right, so I think ultimately, we’re all trying to take the best care of you and we would need to know which part of that may or may not be working so well.  

Katherine:

Let’s get to a few audience questions that we received prior to the program. This one is from Rita. “Is there an age limit on CAR T-cell therapy?” 

Dr. Nadeem:

So, no, there isn’t. A lot of age-related cutoffs that we’ve historically used for transplants or even the CAR T originally don’t really apply because we all know there’s patients that are in their late 70s that may be more fit and robust than somebody in their 50s. We see this all the time. So, frailty is something that we assess quite a bit in patients in determining whether they can handle some of the toxicities that may come from these therapies. So, there’s no age cutoff.  

Again, we look at certain other medical problems you may have, how fit you are, your organ function and things like that, but ultimately the goal is can you tolerate the chemotherapy you get before CAR-T cells and then can you tolerate some of the acute toxicities of CAR-T cells, such as the cytokine release syndrome, some risk of neurological toxicity, things like that. All of those are usually short-term, and if you feel confident that we can get you through that, then you’re eligible.  

Katherine:

Yeah. Laura sent in this question: “I’m considering bispecific antibody therapy. I know some of the side effects are similar to CAR T-cell therapy. Can you share the pros and cons of bispecifics and how it compares to CAR T?” 

Dr. Nadeem:

Yeah. I think we mentioned earlier that as a whole, they’re very similar. They’re both T-cell re-directing therapies, in many circumstances, with the same exact target of the myeloma cell, but because this isn’t a cell infusion – this is a cell injection – that you receive that redirects your T cells to the myeloma cells, you tend to see a little bit of a lower toxicity signal when it comes to the cytokine release syndrome incidents and severity. You see lower neurological toxicity, usually, than you do with CAR  T-cell products as a whole.  

With that comes slightly lower efficacy than you see with at least some of our CAR-T products, but if you respond to therapy, then the durability of response can be as good as you can achieve with CAR-T cells. One thing to note about the bispecifics, though, is that it is continuous therapy, so you are getting it on some regular schedule. Right now the approval is for it to be given weekly and then go to every two weeks after six months of therapy if you’re basically in a good response.  

A lot of that is to try to mitigate the risk of infection. So, that is one of the biggest things that we have seen with bispecifics more so than CAR-T cells. Because it is continuous administration of these therapies, that really suppresses your immune system significantly, and infection rates are quite high. So, we typically give other ways to try to mitigate that using immunoglobulin infusions to try to boost up your immune system. Typically, we do that once a month for patients, making sure you’re on the right prophylactic medications and then really adjusting the therapy and the schedule to you depending on your tolerability.  

So, as we said before, it’s an excellent option. I think bispecific antibodies are going to be the mainstay of myeloma therapy going forward because CAR-T cells, again, we can’t really treat everybody with CAR-T cells just simply because of the dynamics of how the process is. So, having the bispecific antibodies available for patients is excellent.  

Katherine:

Thank you for this information, Dr. Nadeem. And please continue to send in your questions to questions@powerfulpatients.org and we’ll work to get them answered on future programs.  

We’ve definitely learned today that the field of myeloma care is advancing quickly. As we close out the program, what would you like to leave the audience with? Why are you hopeful? 

Dr. Nadeem:

Yeah. I think you all can see the tremendous progress that’s been made and, again, I still think it’s sort of the tip of the iceberg. These immunotherapies that are really showing this kind of activity, we’re just learning about them, and we’re going to improve them, not just the way we administer them. We’re going to make them even better and better and better and our hope is that a cure is not so far in the future. And perhaps even now we can cure a subset of patients if we deploy some of these therapies in the right person at the right time. So, I think that is really what I am hopeful for, that we have all these options available.  

Now it’s up to us to figure out which one fits in where and then, as we do that, hopefully we’ll see even better and better outcomes. And my hope is, over time, that this is a disease that we can cure at least in a subset of patients, which means that they get fixed duration therapy with whatever that we have.  

And then they’re done, and then hopefully never have to have therapy for this disease because it’ll be gone, and then, in patients that develop a disease relapse, we then treat them with some of these other agents. So, this is starting to hopefully mirror what we see in other blood cancers, such as lymphoma, for example, where you give the initial therapy and cure a subset of patients. Hopefully we can get there with myeloma in the not-so-distant future.  

Katherine:

It’s a very promising outlook to leave our audience with. Dr. Nadeem, thank you so much for joining us today. 

Dr. Nadeem:

Thank you so much for having me.   

Katherine:

And thank you to all of our collaborators. 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 today.  

Combination AML Therapy for Newly Diagnosed Patients | What Are the Long-Term Effects?

Combination AML Therapy for Newly Diagnosed Patients | What Are the Long-Term Effects? from Patient Empowerment Network on Vimeo.

A Patient Empowerment Network community member wants to know the length of time that patients can stay on the combined treatment of azacitidine (Vidaza) and venetoclax (Venclexta). AML specialist Dr. Jacqueline Garcia responds, sharing an update on the long-term follow-up data for this combination treatment.

Dr. Jacqueline Garcia is an oncologist and AML researcher at the Dana-Farber Cancer Institute. Learn more about Dr. Garcia.

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Transcript:

Katherine Banwell:

Jerry had this question. “How long can patients stay on azacitidine (Vidaza) and venetoclax (Venclexta) before relapse or toxicities force them to abandon treatment?”  

Dr. Jacqueline Garcia:

So, this is a good question. I would say azacitidine and venetoclax just got FDA-approved just shy of five years now, and it’s totally changed our treatment paradigm in many great ways. It was initially approved for patients that could not get intensive chemotherapies or were above 75. We call these our older patients, our more vulnerable.   

And we demonstrated and compared to azacitidine alone. It was given with placebo. We saw that the combination of azacitidine and venetoclax not only was safe, well-tolerated, it led to two-and-a-half times higher complete remission rates and impressively longer survival. That’s all we care about, patients are living longer. So, one of the things that we are appreciating in 2023 now, now that we have more patients on azacitidine and venetoclax, is that we have many patients that are long-term responders.  

So, in the original clinical trial we’ve been reported – and we just submitted the update for the long-term follow-up that we presented at the American Society of Hematology meeting in 2022, in December.   

We presented the long-term follow-up data that shows that responses can be durable and even as long as two years or three years in some patients. The average amount of time the patients are on therapy is somewhere between one-and-a-half to two years. But not every patient performs like an average patient.  

We have some that respond for less time. We have some that respond for a longer time. So, I definitely have a few patients that have been on combination therapy, and we’ve gone to year three, then four, and two that got to year five. And that was using the original indication of older the 75, no intensive chemotherapy. Most of those patients in the original trial and led to the approval were not transplant candidates. But once those drugs got approved, more patients that were older started getting this therapy.  

And so, the durability of this treatment might be longer for people that don’t have competing health problems and for specific mutation subtypes. There are a couple of mutation subtypes that include IDH2 and NPM1, where we’ve seen some extreme long-term responders.  

And then, there are others that are much shorter. So, I would say it’s very individual. In terms of toxicities in general, the regimens very well-tolerated. And if it’s not, often it’s because there should be supportive care, prophylaxis, and adjustments to the dosing strategy, which has been well-published. Sometimes, if you have a treating oncologist that is less familiar, they won’t know the nuances of how to adjust the doses, so I would ask your local oncologist to reach out to anybody that was part of the original trials. Often, a lot of us are very responsive to helping out our colleagues to help patients to stay on treatment.   

But at the end of the day, if a patient loses response or has a bad toxicity that makes it very difficult, we have to move on to another therapy.   

AML Specialists and Second Opinions | Expert Advice to Patients

AML Specialists and Second Opinions | Expert Advice to Patients from Patient Empowerment Network on Vimeo.

If you seek a second opinion, will you hurt your current doctor’s feelings? Dr. Jacqueline Garcia shares advice for seeking a consultation from an AML specialist, emphasizing timing and clear communication.

Dr. Jacqueline Garcia is an oncologist and AML researcher at the Dana-Farber Cancer Institute. Learn more about Dr. Garcia.

See More from Thrive AML

Related Resources:

Acute Myeloid Leukemia Care | Who Are the Essential Team Members

Expert Perspective | Key Advice for AML Patients

How Can You Thrive With AML Advice for Navigating Care.


Transcript:

Katherine Banwell:

If a patient is feeling uncomfortable with the direction of their treatment plan or their care, should they consider a second opinion or even consulting a specialist?  

Dr. Jacqueline Garcia:

Oh, 100 percent. I would say – I think that I’m spoiled. I’m a leukemia specialist, so they’re already seeing a specialist when a patient sees me. I don’t take care of any other cancers. But, I would say, for anyone seeing any oncologist in general, I would – number one, it doesn’t do the medical team any favors if you withhold any feelings of how the treatment’s going. Meaning, if you feel uncomfortable or that you’re having symptoms or people are taking too long to get back to you based on your experience.  

I would just make sure you do your best to at least let them know so that they have the ability to adjust or accommodate whatever need you might have that might be different than what they’re used to, because every patient’s different. Some people have a really great support system. Or they have a little bit of experience of being a patient. Different coping mechanisms. Everyone’s different. There’s no right or wrong. But I would just make sure that it’s clear with your existing team, because they’re actively seeing you. Give them a chance to make the experience better.  

I would for sure seek a second opinion. Don’t delay – I will just put this disclaimer. I would not delay treatment for an AML if your current doctor is giving you a good plan, and you feel confident that they have looked into whether or not you need to go to a bigger leukemia center and all that other stuff. But if you feel like they are giving you a good plan, don’t delay your therapy in the beginning, because you might get sick.  

If, however, there is demonstration of safety and time to see someone within a short timeframe for a second opinion at the time of diagnosis before treatment started, then that’s okay. But wouldn’t wait a few months to go looking around, because that could put your health at risk. Once you’re on treatment, seeking a second opinion, if you’re dissatisfied with your ongoing team, it’s fine. I always want patients to feel comfortable with their treatment plan.  

But I would recognize that you want to make it clear to your current team that they’re still helping you and responsible for your treatment. Because if you, for instance, started seeing multiple doctors and they won’t know who should be helping to follow up on certain things, who’s going to be scheduling the next round of therapy. And that ends up putting more ownership unnecessarily onto the patient where they might not have needed to have all that extra responsibility. So, I would just say just make sure that’s clear.  

AML Treatment Decisions | Understanding Factors That Impact Your Options

AML Treatment Decisions | Understanding Factors That Impact Your Options from Patient Empowerment Network on Vimeo.

An acute myeloid leukemia (AML) diagnosis can be different for each individual patient, so how is a treatment approach determined? AML specialist Dr. Jacqueline Garcia provides an overview of factors taken into consideration when choosing therapy, including age, overall health, and the patient’s preference. 

Dr. Jacqueline Garcia is an oncologist and AML researcher at the Dana-Farber Cancer Institute. Learn more about Dr. Garcia.

See More from Thrive AML

Related Resources:

Acute Myeloid Leukemia Care | Who Are the Essential Team Members

Understanding AML Treatment Categories


Transcript:

Katherine Banwell:

With all the treatment options available, how do you decide who gets what? Tell us what is considered when choosing treatment for a patient. 

Dr. Jacqueline Garcia:

When I – this is a complicated question, because it’s not like you follow any particular algorithm. But when I meet a patient, I make a decision on what’s important to the patient and what’s  their goal. If I know – I need to understand their overall health to get a sense of are there ongoing competing risk factors that are active and more likely to impede with response, ability to deliver chemo, ability to get to transplant, something that tells me that’s not a possibility, or is their age too advanced – meaning greater than 75 – where we know that some of the treatments are not safe to deliver in that setting?   

So, I take a look at a patient’s overall health and age to make a decision. I take a look at bone marrow biopsy and lab findings to understand the flavor of their leukemia, from chromosomes to mutations. And because I am familiar with the data to give me a sense of what’s safe, what’s tolerable, and importantly what types of diseases, or subtypes of AML, would respond to one therapy over another, that’s how I formulate a recommendation.  

And based on all of that, all together, I’ll talk to them about treating the AML in steps. The first step is getting them into a remission, which can be done regardless of therapy type. That means to get their bone marrow under control, blood counts to recover. The second step, which is a more involved conversation that I often give a little bit of a hint of, but I go into greater detail over time, because we will see each other quite a lot, whether in the hospital or in clinic, is how to keep them in remission.  

And that’s where details about things like transplant come into play. I do my best to not overwhelm them, because when a patient hears the word transplant – and that’s often what they hear from family and friends because that’s what you can Google – they don’t know that there are many things, or many weeks of therapy, that have to happen in advance of transplant even being considered or happening. And transplant can’t even happen until someone’s in remission.  

But that is always on the forefront of a leukemia doctor’s mind, “Can I bring this patient to a transplantation? How successful will I be and what else do I need to give them to get them there sooner, safer, with a deeper response?” So, that way transplant could be successful. Transplant, by the way, is when we give a patient someone else’s stem cells that match their HLA typing, or their white blood cell signature.  

And it helps us to use someone else’s immune system to completely irradicate any microscopic leftover leukemia in a patient. But that is only successful when patients have good disease control or remissions. And that is only also successful if we have a donor for the patient, both of which  require at least several weeks to a couple of months of therapy. But that process is always initiated and ongoing in the background. And so, we often do this in piecemeal, because getting a diagnosis is already overwhelming. Learning about treatment is overwhelming.  

Learning about the frequency of labs, transfusions, being hospitalized, and then details about what a transplant would entail can be also overwhelming. But a lot of family and friends like to ask, because they feel like that is one way they might be able to help a patient. So, I know that they often eagerly ask the patient, “Well, what about this? How can I help?”  

Katherine Banwell:

Right. I can imagine that patient preference is also considered. But what kind of questions should patients ask about their treatment regimen?  

Dr. Jacqueline Garcia:

I always tell patients that I care very much about things like travel, hotels, all that jazz. But I always tell them let’s first talk about their health, what treatment I would recommend based on the available options and what their disease would mostly respond to, because I want it to be successful. And I always tell them let’s reserve questions on how it’s going to be done for last. I call that the logistics. I will never bring up or recommend something that could never be possible. But that being said, I try not to let the commute determine the decision.  

Whether or not there needs to be a hospitalization versus a hotel stay. I always consider then the background, but that financial decision should not drive the best treatment choice for a patient. Very fortunately, we’re in a country where patients have the ability – often, not always – to seek second opinions or to travel to academic centers.  

And because AML is an emergent or life-threatening disease, many insurance providers allow patients to come up to a big center to be treated, which I think is more than appropriate. So, we get into details of logistics last, because that’s the one thing that we can often overcome by providing additional resources and support. In terms of patient preference, if that’s what you mean with that, I would say I leave logistics to last, but we always consider and we do our best to accommodate.  

And that might be where we inform them we will look into getting a local partner to help us with additional therapies after the first month or upon discharge. So, it totally depends on the scenario for a patient, whether or not they have a local provider and a local hospital that could accommodate acute leukemia. I always tell patients ideally you don’t want to go to a place that only sees this once per year. You want to go to a place where everyone has seen it multiple times, including the nurses on the floors.  

So, that way, when there’s a complication, everyone knows what to do. We don’t want any “surprises” when it’s really just run-of-the-mill standard stuff for us every day. In terms of what patients desire, we always keep that in the conversation of their level of support. Can they swallow pills? Are they able to cope with being in and out of the hospital? All that stuff gets considered, but I think if they hear about the plan, about what’s required, when my expectation would be for a response, when the frequency of trips to a big city would decrease, how I could get a local partner to help with some of the lab or transfusion burden.  

Many of those preferences that they thought they had diminished, because they recognize that we found a way to make it work.  

Katherine Banwell:

Dr. Garcia, you mentioned earlier the fact that some therapies can cause a lot of side effects, like nausea. And certainly, speaking up and telling your healthcare team how you’re feeling and what some of the symptoms and side effects are, that’s really essential. What is the impetus for someone to consider changing treatment if something is just absolutely not agreeing with them?  

Dr. Jacqueline Garcia:

So, there are many reasons to change a treatment. One is a patient doesn’t tolerate it. It depends on what the issue is. Is it something that’s serious, like a liver or enzyme abnormality that is very abnormal, or a new cardiac problem where it would warrant a change or a dose reduction? That makes sense. There is definitely – often, there’s a lot of guidance in the package inserts or within a clinical trial and how to manage that. But if patient has some intolerabilities that could be overcome with standard supportive care methods, I would make sure we’ve done that.   

So, I would make sure you give you medical team the chance to fix any nausea. We have so many great antinausea drugs. I would want to make sure – or if constipation or diarrhea. It’s often a GI issue that patients get really bothered by.  

I would try to delineate whether or not the side effect was really from the chemo or is from the leukemia that is not yet under control. Or is it another medical condition or a drug-drug interaction that was missed. So, I would do my best to make sure there wasn’t something that was fixable or something else that should be addressed. We otherwise would recommend changing therapy for an extreme intolerability if there was another equivalent better option. And if someone’s disease does not respond to treatment, then we would consider another therapy, too.  

New and Emerging AML Therapies Being Studied in Clinical Trials

Are there newer AML treatments that patients should know about? AML researcher Dr. Jacqueline Garcia discusses therapies being studied and how recent clinical trials have advanced care for patients.

Dr. Jacqueline Garcia is an oncologist and AML researcher at the Dana-Farber Cancer Institute. Learn more about Dr. Garcia.

See More from Thrive AML

Related Resources:

Understanding AML Treatment Categories

AML Treatment Decisions | Understanding Factors That Impact Your Options

Combination AML Therapy for Newly Diagnosed Patients | What Are the Long-Term Effects


Transcript:

Katherine Banwell:

As a researcher, Dr. Garcia, you’re on the frontlines of AML treatment. Are there new and emerging therapies that patients should be aware of?  

Dr. Jacqueline Garcia:

Yeah. I think we’re at this really exciting point now where we had for a long time just been giving people standard two agent intensive chemotherapy. We have been studying in Phase II and Phase III settings, and even in Phase I – which means testing safety out for the first time. We’ve been moving a lot of treatments to more mature settings where we’re testing the addition of a third drug. So, for people that are getting intensive chemo, we’re looking at, “Can we add a pill to augment responses deep in them to reduce risk of disease returning?”  

For less intensive chemotherapies, one of the most common regimens we now use is something called azacitidine (Vidaza), which is a hypomethylating agent that is given by IV or subcutaneous administration. Plus, a pill called venetoclax (Venclexta).  

We helped to get that FDA-approved a couple of years ago. That combination of therapy, we call that a doublet, meaning it’s two drugs – because it’s so well-tolerated and active, we’re now asking the greedy question of, “Well, can we make it more active for patients since we’re seeing how well-tolerated it is?”  

So, there have been a lot of therapies that are currently under investigation that are adding a third drug to these less-intensive doublets. So, there’s a lot of therapies under investigation to test, “Can we add an immunotherapy target? Is there another pill that we can add? Is there another targeting mutation to add to the doublet?” So, we’re looking at AML therapies from different angles. We’re looking at adding something to the existing new standard of care – those are these new, so-called, triplets.  

We’re looking at still the role of cellular therapy or CAR Ts targeting leukemia cells from an immunotherapy standpoint.  

That remains underdeveloped overall, and we have not succeeded as well, like our lymphoid colleagues in the lymphoma and acute lymphoblastic leukemia realm where there are drugs that are active and FDA-approved.  

So, we’re still trying to identify the right target. But those are some of the areas that are currently under study. 

Understanding AML Treatment Categories

 

Understanding AML Treatment Categories from Patient Empowerment Network on Vimeo.

What are the available classes of therapy for acute myeloid leukemia (AML)? Dr. Jacqueline Garcia reviews AML treatment options, ranging from chemotherapy and stem cell transplant to supportive care. 

Dr. Jacqueline Garcia is an oncologist and AML researcher at the Dana-Farber Cancer Institute. Learn more about Dr. Garcia.

See More from Thrive AML

Related Resources:

New and Emerging AML Therapies Being Studied in Clinical Trials

New and Emerging AML Therapies Being Studied in Clinical Trials


Transcript:

Katherine Banwell:

In your experience, what does it mean to thrive with AML?   

Dr. Jacqueline Garcia:

I think that’s a really great question, and I’m glad you’re asking me now as opposed to a decade ago. In the last several years, we’ve had a tremendous number of drugs that got FDA-approved and a lot of exciting clinical trials that have not only shown efficacy and safety but really some long-term responses. So, we can now focus on not just finding what drug can work, which used to be our problem 10 years ago, since we had very limited therapeutic tools, meaning treatments. We now have several treatments available.  

So, when I think of what it means to thrive, it’s identifying the right treatment for each individual patient with acute myeloid leukemia, because what might be recommended for one patient may not be the right for another. And there are many different patient- and disease-related factors that go into that decision-making.  

Katherine Banwell:

Can you walk us through the classes of treatment that are considered when choosing an AML treatment approach?  

Dr. Jacqueline Garcia:

Yeah. In terms of the different classes of treatments, I would say we think of probably three broad categories. One would be – sorry, four broad categories. One would be intensive chemotherapy. And that involves generally hospitalization. Another would be less intensive therapy. That could involve a mixture of inpatient or outpatient therapy. That could also include targeted therapy. The third would be clinical trials, which can include any of the former options I recommended, but they would be in an experimental study. And the fourth would be focusing solely on supportive care or hospice for patients that are too sick to receive therapy.  

Other aspects that are specific, such as pills, versus IV, versus role of transplant, I don’t see it as being separate. You don’t go right to transplant when you have a diagnosis of AML. You have to be in remission. So, transplant, for instance, would come after an intensive therapy or after the less intensive chemotherapy. So, I see that as being the second step once I choose the right treatment option for the patient.  

Katherine Banwell:

And when you’re talking about transplant, you’re talking about stem cell transplant, right?  

Dr. Jacqueline Garcia:

Yes. Stem cell transplant, bone marrow transplant – they mean the same thing. We recruit stem cells from donors that are related or unrelated, and we mobilize them from bone marrow to blood. And so, we can collect stem cells either from blood or bone marrow at this point. So, that’s exactly right.  

Katherine Banwell:

And what about targeted therapy?  

Dr. Jacqueline Garcia:

We have targeted therapy available that’s IV or pill form. And so, any one of these options can be considered. But everything is very patient-specific, and I am very happy to tell you some of the categories and nuances of things that I look at, because I don’t usually just offer patients a menu.  

I tell them what’s appropriate based on their patient characteristics, meaning what their liver function is, their heart function, their history, medical history, what their labs show. And then, I look at their disease history. We are now in an era where we have options. So, I look to see are there mutations that are targetable. Are there not? Are there markers on the surface of their leukemia cells that suggest that there’s a target for an immunotherapy?  

So, we don’t offer classes per se without it being specific. So, I always look to see what are the patient disease-specific characteristics, and then I start the conversation about what the potential options could be and then what I think the best option would be for that particular case.  

Acute Myeloid Leukemia Care | Who Are the Essential Team Members?

Acute Myeloid Leukemia Care | Who Are the Essential Team Members? from Patient Empowerment Network on Vimeo.

Acute myeloid leukemia (AML) care is not just monitored by an oncologist or hematologist – there’s an entire medical team. Dr. Jacqueline Garcia, an oncologist and AML researcher, shares an overview of the various members of the healthcare team and the role they play in overall care.

Dr. Jacqueline Garcia is an oncologist and AML researcher at the Dana-Farber Cancer Institute. Learn more about Dr. Garcia.

See More from Thrive AML

Related Resources:

Understanding AML Treatment Categories

AML Treatment Decisions | Understanding Factors That Impact Your Options

How Can You Thrive With AML Advice for Navigating Care.


Transcript:

Katherine Banwell:

Typically, there are a number of team members to care for a patient. Who is part of an AML healthcare team?  

Dr. Jacqueline Garcia:

Absolutely. We definitely cannot work on our own. Our team is very large, and it’s because these patients require a lot of support. At a bare minimum, a healthcare team will include at least one physician or an oncologist. The AML healthcare team might also include a second oncologist – that could be a bone marrow transplant doctor.  

Other members that are very critical include having a mid-leveler available that’s a physician assistant or a nurse practitioner. Often, an oncologist who runs a busy practice, who takes care of patients that could be very sick, like AML, they work in partnership with often very talented physician assistants and nurse practitioners. I know I do.  

In addition to that, I’m at an academic center so I’m super fortunate. I have really amazing and very smart hematology oncology fellows and residents that also follow to learn how to take care of patients. But we also, in the background, that patients don’t see – we have a pharmacist that helps us with making sure that drugs are prescribed correctly. They often call the patients with oral therapies to follow up. We have financial resource teams to help patients, to link them to LLS for support for bills that might come up, or transportation, or linking them up to other services that could help to defray or reduce costs.  

So, the healthcare team is quite extensive. But in terms of those that are patient-facing, it’s primarily the MDM that are mid-leveler. Some teams operate also with a nurse or a nurse care coordinator. That’s pretty common, too. And that person helps to not only schedule but also to answer pages or phone calls from patients if the medical team is not doing that.  

Katherine Banwell:

What about a social worker or psychologist? 

Dr. Jacqueline Garcia:

Oh. Yes. Yes. So, absolutely. So, every patient can be offered, if needed, access to an inpatient or outpatient social worker. Often, if my patients are admitted we have them see a social worker because that’s fairly seamless. Otherwise, for outpatient, if we identify any particular needs or there’s an interest, we’ll link them up with a social worker. This is the same that goes for physical therapy, or nutritionists, or those other ancillary services that can be really critical when patients are getting started.  

PODCAST: Thriving With AML | Tips and Support for Navigating Treatment

 

How can you navigate care and thrive with acute myeloid leukemia (AML)? In this webinar, Dr. Jacqueline Garcia, an AML specialist and researcher, discusses the treatment and management of AML. Dr. Garcia will review factors that impact therapy choices and shares advice and resources for people living with AML.
 
Dr. Jacqueline Garcia is an oncologist and AML researcher at the Dana-Farber Cancer Institute. Learn more about Dr. Garcia.

Download Resource Guide

See More from Thrive AML

Transcript:

Katherine Banwell:

Hello, and welcome. I’m Katherine Banwell, your host for this webinar. Today’s program is about how to live and thrive with AML. We’re going to discuss how to live well with AML and why you should play an active role in your care. 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.  

Well, let’s meet our guest today. Joining me is Dr. Jacqueline Garcia. Dr. Garcia, welcome. Would you please introduce yourself?  

Dr. Jacqueline Garcia:

Yes. Hi. My name is Jacqueline Garcia. I’m an oncologist at the Dana-Farber Cancer Institute. I’m a clinical translational investigator. And what this means is I take care of patients with acute and chronic leukemias. I focus mainly on patients with acute myeloid leukemia. The investigator part means, in addition to seeing patients, I spend a lot of time writing, developing, and executing clinical trials in the AML space. We know that there have been so many wonderful therapies that we helped to move froward and bring to the field and so there is more work to be done. So, having active investigations is a key part of this role.  

Katherine Banwell:

Excellent. Well, thank you for taking the time out of your schedule to join us today. We really appreciate it. We start all of our webinars in our Thrive Series with the same question. In your experience, what does it mean to thrive with AML?  

Dr. Jacqueline Garcia:

I think that’s a really great question and I’m glad you’re asking me now as opposed to a decade ago. In the last several years, we’ve had a tremendous number of drugs that got FDA-approved and a lot of exciting clinical trials that have not only shown efficacy and safety but really some long-term responses. So, we can now focus on not just finding what drug can work, which used to be our problem 10 years ago, since we had very limited therapeutic tools, meaning treatments. We now have several treatments available.  

So, when I think of what it means to thrive, it’s identifying the right treatment for each individual patient with acute myeloid leukemia, because what might be recommended for one patient may not be the right for another. And there are many different patient- and disease-related factors that go into that decision-making.  

Katherine Banwell:

Thank you for that, Dr. Garcia. It helps guide us as we move into our conversation. Typically, there are a number of team members to care for a patient. Who is part of an AML healthcare team?  

Dr. Jacqueline Garcia:

Absolutely. We definitely cannot work on our own. Our team is very large, and it’s because these patients require a lot of support. At a bare minimum, a healthcare team will include at least one physician or an oncologist. The AML healthcare team might also include a second oncologist – that could be a bone marrow transplant doctor.  

Other members that are very critical include having a mid-leveler available that’s a physician assistant or a nurse practitioner. Often, an oncologist who runs a busy practice, who takes care of patients that could be very sick, like AML, they work in partnership with often very talented physician assistants and nurse practitioners. I know I do.  

In addition to that, I’m at an academic center so I’m super fortunate. I have really amazing and very smart hematology oncology fellows and residents that also follow to learn how to take care of patients. But we also, in the background, that patients don’t see – we have a pharmacist that helps us with making sure that drugs are prescribed correctly. They often call the patients with oral therapies to follow up. We have financial resource teams to help patients, to link them to LLS for support for bills that might come up, or transportation, or linking them up to other services that could help to defray or reduce costs.  

So, the healthcare team is quite extensive. But in terms of those that are patient-facing, it’s primarily the MDM that are mid-leveler. Some teams operate also with a nurse or a nurse care coordinator. That’s pretty common, too. And that person helps to not only schedule but also to answer pages or phone calls from patients if the medical team is not doing that.  

Katherine Banwell: What about a social worker or psychologist?  

Dr. Jacqueline Garcia:

Oh. Yes. Yes. So, absolutely. So, every patient can be offered, if needed, access to an inpatient or outpatient social worker. Often, if my patients are admitted we have them see a social worker because that’s fairly seamless. Otherwise, for outpatient, if we identify any particular needs or there’s an interest, we’ll link them up with a social worker. This is the same that goes for physical therapy, or nutritionists, or those other ancillary services that can be really critical when patients are getting started.  

Katherine Banwell:

Yeah. Of course, getting appropriate care and treatment is essential to thriving. Can you walk us through the classes of treatment that are considered when choosing an AML treatment approach?  

Dr. Jacqueline Garcia:

Yeah. In terms of the different classes of treatments, I would say we think of probably three broad categories. One would be – sorry, four broad categories. One would be intensive chemotherapy. And that involves generally hospitalization. Another would be less intensive therapy. That could involve a mixture of inpatient or outpatient therapy. That could also include targeted therapy. The third would be clinical trials, which can include any of the former options I recommended, but they would be in an experimental study. And the fourth would be focusing solely on supportive care or hospice for patients that are too sick to receive therapy.  

Other aspects that are specific, such as pills, versus IV, versus role of transplant, I don’t see it as being separate. You don’t go right to transplant when you have a diagnosis of AML. You have to be in remission. So, transplant, for instance, would come after an intensive therapy or after the less intensive chemotherapy. So, I see that as being the second step once I choose the right treatment option for the patient.  

Katherine Banwell:

And when you’re talking about transplant, you’re talking about stem cell transplant, right?  

Dr. Jacqueline Garcia:

Yes. Stem cell transplant, bone marrow transplant – they mean the same thing. We recruit stem cells from donors that are related or unrelated, and we mobilize them from bone marrow to blood. And so, we can collect stem cells either from blood or bone marrow at this point. So, that’s exactly right.  

Katherine Banwell:

And what about targeted therapy?  

Dr. Jacqueline Garcia:

We have targeted therapy available that’s IV or pill form. And so, any one of these options can be considered. But everything is very patient-specific, and I am very happy to tell you some of the categories and nuances of things that I look at, because I don’t usually just offer patients a menu.  

I tell them what’s appropriate based on their patient characteristics, meaning what their liver function is, their heart function, their history, medical history, what their labs show. And then, I look at their disease history. We are now in an era where we have options. So, I look to see are there mutations that are targetable. Are there not? Are there markers on the surface of their leukemia cells that suggest that there’s a target for an immunotherapy?  

So, we don’t offer classes per se without it being specific. So, I always look to see what are the patient disease-specific characteristics, and then I start the conversation about what the potential options could be and then what I think the best option would be for that particular case.  

Katherine Banwell:

As a researcher, Dr. Garcia, you’re on the frontlines of AML treatment. Are there new and emerging therapies that patients should be aware of?  

Dr. Jacqueline Garcia:

Yeah. I think we’re at this really exciting point now where we had for a long time just been giving people standard two agent intensive chemotherapy. We have been studying in Phase II and Phase III settings, and even in Phase I – which means testing safety out for the first time. We’ve been moving a lot of treatments to more mature settings where we’re testing the addition of a third drug. So, for people that are getting intensive chemo, we’re looking at, “Can we add a pill to augment responses deep in them to reduce risk of disease returning?”  

For less intensive chemotherapies, one of the most common regimens we now use is something called azacitidine (Vidaza), which is a hypomethylating agent that is given by IV or subcutaneous administration. Plus, a pill called venetoclax (Venclexta).   

We helped to get that FDA-approved a couple of years ago. That combination of therapy, we call that a doublet, meaning it’s two drugs – because it’s so well-tolerated and active, we’re now asking the greedy question of, “Well, can we make it more active for patients since we’re seeing how well-tolerated it is?”  

So, there have been a lot of therapies that are currently under investigation that are adding a third drug to these less-intensive doublets. So, there’s a lot of therapies under investigation to test, “Can we add an immunotherapy target? Is there another pill that we can add? Is there another targeting mutation to add to the doublet?” So, we’re looking at AML therapies from different angles. We’re looking at adding something to the existing new standard of care – those are these new, so-called, triplets.  

We’re looking at still the role of cellular therapy or CAR Ts targeting leukemia cells from an immunotherapy standpoint.  

That remains underdeveloped overall, and we have not succeeded as well, like our lymphoid colleagues in the lymphoma and acute lymphoblastic leukemia realm where there are drugs that are active and FDA-approved.  

So, we’re still trying to identify the right target. But those are some of the areas that are currently under study.  

Katherine Banwell:

You touched on this earlier, Dr. Garcia, but I’d like to get into a bit more detail. With all the treatment options available, how do you decide who gets what? Tell us what is considered when choosing treatment for a patient.   

Dr. Jacqueline Garcia:

When I – this is a complicated question, because it’s not like you follow any particular algorithm. But when I meet a patient, I make a decision on what’s important to the patient and what’s  their goal. If I know – I need to understand their overall health to get a sense of are there ongoing competing risk factors that are active and more likely to impede with response, ability to deliver chemo, ability to get to transplant, something that tells me that’s not a possibility, or is their age too advanced – meaning greater than 75 – where we know that some of the treatments are not safe to deliver in that setting?  

So, I take a look at a patient’s overall health and age to make a decision. I take a look at bone marrow biopsy and lab findings to understand the flavor of their leukemia, from chromosomes to mutations. And because I am familiar with the data to give me a sense of what’s safe, what’s tolerable, and importantly what types of diseases, or subtypes of AML, would respond to one therapy over another, that’s how I formulate a recommendation.   

And based on all of that, all together, I’ll talk to them about treating the AML in steps. The first step is getting them into a remission, which can be done regardless of therapy type. That means to get their bone marrow under control, blood counts to recover. The second step, which is a more involved conversation that I often give a little bit of a hint of, but I go into greater detail over time, because we will see each other quite a lot, whether in the hospital or in clinic, is how to keep them in remission.   

And that’s where details about things like transplant come into play. I do my best to not overwhelm them, because when a patient hears the word transplant – and that’s often what they hear from family and friends because that’s what you can Google – they don’t know that there are many things, or many weeks of therapy, that have to happen in advance of transplant even being considered or happening. And transplant can’t even happen until someone’s in remission.  

But that is always on the forefront of a leukemia doctor’s mind, “Can I bring this patient to a transplantation? How successful will I be and what else do I need to give them to get them there sooner, safer, with a deeper response?” So, that way transplant could be successful. Transplant, by the way, is when we give a patient someone else’s stem cells that match their HLA typing, or their white blood cell signature.  

And it helps us to use someone else’s immune system to completely irradicate any microscopic leftover leukemia in a patient. But that is only successful when patients have good disease control or remissions. And that is only also successful if we have a donor for the patient, both of which  require at least several weeks to a couple of months of therapy. But that process is always initiated and ongoing in the background. And so, we often do this in piecemeal, because getting a diagnosis is already overwhelming. Learning about treatment is overwhelming.  

Learning about the frequency of labs, transfusions, being hospitalized, and then details about what a transplant would entail can be also overwhelming. But a lot of family and friends like to ask, because they feel like that is one way they might be able to help a patient. So, I know that they often eagerly ask the patient, “Well, what about this? How can I help?”  

Katherine Banwell:

Right. I can imagine that patient preference is also considered. But what kind of questions should patients ask about their treatment regimen?  

Dr. Jacqueline Garcia:

I always tell patients that I care very much about things like travel, hotels, all that jazz. But I always tell them let’s first talk about their health, what treatment I would recommend based on the available options and what their disease would mostly respond to, because I want it to be successful. And I always tell them let’s reserve questions on how it’s going to be done for last. I call that the logistics. I will never bring up or recommend something that could never be possible. But that being said, I try not to let the commute determine the decision.  

Whether or not there needs to be a hospitalization versus a hotel stay. I always consider then the background, but that financial decision should not drive the best treatment choice for a patient. Very fortunately, we’re in a country where patients have the ability – often, not always – to seek second opinions or to travel to academic centers.  

And because AML is an emergent or life-threatening disease, many insurance providers allow patients to come up to a big center to be treated, which I think is more than appropriate. So, we get into details of logistics last, because that’s the one thing that we can often overcome by providing additional resources and support. In terms of patient preference, if that’s what you mean with that, I would say I leave logistics to last, but we always consider and we do our best to accommodate.  

And that might be where we inform them we will look into getting a local partner to help us with additional therapies after the first month or upon discharge. So, it totally depends on the scenario for a patient, whether or not they have a local provider and a local hospital that could accommodate acute leukemia. I always tell patients ideally you don’t want to go to a place that only sees this once per year. You want to go to a place where everyone has seen it multiple times, including the nurses on the floors.  

So, that way, when there’s a complication, everyone knows what to do. We don’t want any “surprises” when it’s really just run-of-the-mill standard stuff for us every day. In terms of what patients desire, we always keep that in the conversation of their level of support. Can they swallow pills? Are they able to cope with being in and out of the hospital? All that stuff gets considered, but I think if they hear about the plan, about what’s required, when my expectation would be for a response, when the frequency of trips to a big city would decrease, how I could get a local partner to help with some of the lab or transfusion burden.  

Many of those preferences that they thought they had diminished, because they recognize that we found a way to make it work.  

Katherine Banwell:

Okay. Well, that’s really good to know. You touched on oral therapies a bit ago, and I know that they’re available for certain patients. Do you have any advice for patients who are in charge now of administering their own therapy?  

Dr. Jacqueline Garcia:

Yeah, I think that taking pills in general is hard for anybody, whether they’re naïve to pills. I definitely have patients that have never been on anything, and suddenly they’re on many medicines, to other people that are managing multiple medical conditions and this is yet another burden to add. I would say having an oral regimen is wonderful. It offers a lot of convenience. But we are all very thoughtful, and we all need to be proactive about looking for drug-drug interactions, because often there could be increases in the chemotherapy presence when another drug is on board.  

Sometimes, antibiotics are added on but they don’t realize it can add to side effects to chemotherapy. So, I would say number one is always make sure your oncology team is aware of the medications you are on or get recommended to add on in the midst of therapy, so we can make sure there are appropriate dosage estimates or if a particular drug should be avoided, then we can do that.  

I would say, too, having oral therapies is great, but there’s also financial toxicity that comes with it. Sometimes copays can get hefty. So, just because it’s oral, it’s not always convenient financially. Also, when things are oral it can add to more GI or mal gut toxicity. So, we’re always keeping in mind how many oral therapies, what drugs they are, so we don’t increase nausea and diarrhea, which can happen frequently when you’re requiring the GI tract to absorb the therapies that are necessary to eliminate the disease.  

So, all these things are under consideration. But to help people that are on oral therapies, it’s helpful to let your providers know if you’re noticing a pattern of nausea, so we can premedicate, have you take a nausea medicine before you take the chemo. You could also put a timer on your phone if you’re not used to taking medicines to serve as a reminder. You could create little calendars or check off on a paper calendar when you’ve taken a drug if you need help with reminding.  

So, there are little tricks like that. I always consider using a pillbox if you don’t have other pills to mix in and if you’re the only one touching it. I don’t want anybody to be exposed to therapies that they shouldn’t be otherwise.  

Katherine Banwell:

That’s good advice. Thank you. If a patient is feeling uncomfortable with the direction of their treatment plan or their care, should they consider a second opinion or even consulting a specialist?  

Dr. Jacqueline Garcia:

Oh, 100 percent. I would say – I think that I’m spoiled. I’m a leukemia specialist, so they’re already seeing a specialist when a patient sees me. I don’t take care of any other cancers. But, I would say, for anyone seeing any oncologist in general, I would – number one, it doesn’t do the medical team any favors if you withhold any feelings of how the treatment’s going. Meaning, if you feel uncomfortable or that you’re having symptoms or people are taking too long to get back to you based on your experience.   

I would just make sure you do your best to at least let them know so that they have the ability to adjust or accommodate whatever need you might have that might be different than what they’re used to, because every patient’s different. Some people have a really great support system. Or they have a little bit of experience of being a patient. Different coping mechanisms. Everyone’s different. There’s no right or wrong. But I would just make sure that it’s clear with your existing team because they’re actively seeing you. Give them a chance to make the experience better.  

I would for sure seek a second opinion. Don’t delay – I will just put this disclaimer. I would not delay treatment for an AML if your current doctor is giving you a good plan and you feel confident that they have looked into whether or not you need to go to a bigger leukemia center and all that other stuff. But if you feel like they are giving you a good plan, don’t delay your therapy in the beginning, because you might get sick.  

If, however, there is demonstration of safety and time to see someone within a short timeframe for a second opinion at the time of diagnosis before treatment started, then that’s okay. But wouldn’t wait a few months to go looking around, because that could put your health at risk. Once you’re on treatment, seeking a second opinion, if you’re dissatisfied with your ongoing team, it’s fine. I always want patients to feel comfortable with their treatment plan.  

But I would recognize that you want to make it clear to your current team that they’re still helping you and responsible for your treatment. Because if you, for instance, started seeing multiple doctors and they won’t know who should be helping to follow up on certain things, who’s going to be scheduling the next round of therapy. And that ends up putting more ownership unnecessarily onto the patient where they might not have needed to have all that extra responsibility. So, I would just say just make sure that’s clear. Yeah.  

Katherine Banwell:

Dr. Garcia, you mentioned earlier the fact that some therapies can cause a lot of side effects, like nausea. And certainly, speaking up and telling your healthcare team how you’re feeling and what some of the symptoms and side effects are, that’s really essential. What is the impetus for someone to consider changing treatment if something is just absolutely not agreeing with them?  

Dr. Jacqueline Garcia:

So, there are many reasons to change a treatment. One is a patient doesn’t tolerate it. It depends on what the issue is. Is it something that’s serious, like a liver or enzyme abnormality that is very abnormal, or a new cardiac problem where it would warrant a change or a dose reduction? That makes sense. There is definitely – often, there’s a lot of guidance in the package inserts or within a clinical trial and how to manage that. But if patient has some intolerabilities that could be overcome with standard supportive care methods, I would make sure we’ve done that.  

So, I would make sure you give you medical team the chance to fix any nausea. We have so many great antinausea drugs. I would want to make sure – or if constipation or diarrhea. It’s often a GI issue that patients get really bothered by.  

I would try to delineate whether or not the side effect was really from the chemo or is from the leukemia that is not yet under control. Or is it another medical condition or a drug-drug interaction that was missed. So, I would do my best to make sure there wasn’t something that was fixable or something else that should be addressed. We otherwise would recommend changing therapy for an extreme intolerability if there was another equivalent better option. And if someone’s disease does not respond to treatment, then we would consider another therapy, too.  

Katherine Banwell:

Dr. Garcia, I want to make sure that we get to some of the audience questions that were sent to us prior to this program. Let’s start with this one.  

 Jerry had this question. “How long can patients stay on azacitidine and venetoclax before relapse or toxicities force them to abandon treatment?”  

Dr. Jacqueline Garcia:

So, this is a good question. I would say azacitidine and venetoclax just got FDA-approved just shy of five years now, and it’s totally changed our treatment paradigm in many great ways. It was initially approved for patients that could not get intensive chemotherapies or were above 75. We call these our older patients, our more vulnerable.  

And we demonstrated and compared to azacitidine alone. It was given with placebo. We saw that the combination of azacitidine and venetoclax not only was safe, well-tolerated, it led to two-and-a-half times higher complete remission rates and impressively longer survival. That’s all we care about, patients are living longer. So, one of the things that we are appreciating in 2023 now, now that we have more patients on azacitidine and venetoclax, is that we have many patients that are long-term responders.  

So, in the original clinical trial we’ve been reported – and we just submitted the update for the long-term follow-up that we presented at the American Society of Hematology meeting in 2022, in December.  

We presented the long-term follow-up data that shows that responses can be durable and even as long as two years or three years in some patients. The average amount of time the patients are on therapy is somewhere between one-and-a-half to two years. But not every patient performs like an average patient.  

We have some that respond for less time. We have some that respond for a longer time. So, I definitely have a few patients that have been on combination therapy, and we’ve gone to year three, then four, and two that got to year five. And that was using the original indication of older the 75, no intensive chemotherapy. Most of those patients in the original trial and led to the approval were not transplant candidates. But once those drugs got approved, more patients that were older started getting this therapy.  

And so, the durability of this treatment might be longer for people that don’t have competing health problems and for specific mutation subtypes. There are a couple of mutation subtypes that include IDH2 and NPM1, where we’ve seen some extreme long-term responders.   

And then, there are others that are much shorter. So, I would say it’s very individual. In terms of toxicities in general, the regimens very well-tolerated. And if it’s not, often it’s because there should be supportive care, prophylaxis, and adjustments to the dosing strategy, which has been well-published. Sometimes, if you have a treating oncologist that is less familiar, they won’t know the nuances of how to adjust the doses, so I would ask your local oncologist to reach out to anybody that was part of the original trials. Often, a lot of us are very responsive to helping out our colleagues to help patients to stay on treatment.  

But at the end of the day, if a patient loses response or has a bad toxicity that makes it very difficult, we have to move on to another therapy.  

Katherine Banwell:

Of course. Carrie sent in this question. “What percent of patients relapse and what percent of patients relapse more than once?”  

Dr. Jacqueline Garcia:

Okay. So, this is a question that I can certainly answer, but I would say it depends on the context. So, if I was taking – any time a patient asks me that, I always ask them what they want to know and what they don’t want to hear, because sometimes hearing numbers can be really daunting to patients overall.  

So, a very large number of patients with leukemia can go on to relapse, which is why, if you’re on a treatment like azacitidine and venetoclax, we continue it every month as long as we can with dose reductions to help with tolerability.  

And that’s why, if you got that regimen or intensive chemo or another clinical trial and you get into remission, we ask the question of can we transplant this patient to do our best to cure them long-term to avoid and reduce the chance of a relapse. So, even with transplant, which remains our gold standard for long-term curability – it’s the only treatment we have that has a guaranteed track record of cure – not every patient that goes to transplant will remain in remission.  

If I were to be asked, “Well, how many relapse,” I would say it depends. I would say if I took the average patient, maybe 40 to 50 percent will relapse. But if you ask me for certain mutation types it could be 90 percent are cured or only 20 percent are cured. So, it’s very individual. It depends on age. It depends on mutations. It depends on the level of response they had before they go to transplant.  

So, I would say even though the word relapse is very scary or disease coming back is definitely a scary thing, there are a lot of people, including me, that are working on ways to reduce risk of relapse, improve how we transplant, improve the treatments around and after transplant, and improving frontline and relapse therapies.  

I think you had a second question of what happens if you relapse once and then what about if relapse happens again? I would say that getting into remission the first time is always the easiest. The way I always think about it is, you kill off all the bad cells that are the easiest to die the first time around with chemotherapy. Anything that’s left behind are often the resistant types. And so, getting into a second remission or responding the second time around with treatment is doable, but it’s much harder.  

So, I would say the majority of patients that relapse the first time will relapse the second time, unless we can successfully bridge them to a transplantation.  

Katherine Banwell:

Yeah. Dr. Garcia, as we close out this conversation, I wanted to get your thoughts on where we stand with progress in helping people live longer and thrive with AML. What would you like to leave the audience with?  

Dr. Jacqueline Garcia:

I think that this is – I feel very lucky with when I entered the field, that in this last decade, as I’ve developed – my time at Dana-Farber, for instance – I’ve seen that there have been so many drugs that we helped to get approved that are now in the hands of local oncologists and other academic oncologists, suggesting that the clinical trials are a gateway to improving treatments and offering new options.  

 We’ve gotten better at understanding what mutations and chromosomes means and personalizing medicines, and that has allowed us to develop smarter and better clinical trials, which we hope we will get to keep approving and making more available to patients. So, I think that this is a really good time for AML, meaning we have more than one option, that is for sure. We can now think about what the patient wants, what the patient, and what their patient disease has in order to make a decision. We weren’t able to do that before.  

So, we can really involve patients so they understand why we would recommend one option versus another. And we are still not done with investigation, even though many drugs got approved in the last five years. There’s a lot more progress to be made, especially in areas that we touched upon, from approving getting patients to transplant, reducing relapse risk, keeping people in remission. Those are all things that I’m personally working on in the clinical trial space and things a lot of my colleagues in the world are working on, too.  

It’s very important to all of us. So, I would say be hopeful that we are not done. There’s a lot of great options out there. We really can personalize. There are a lot of options out there, but everyone will get offered their best therapy and the first-line therapy is the most important. And I am very hopeful that we will keep getting better at prolonging remissions and durability of those responses.   

Katherine Banwell:

Dr. Garcia, thank you so much for taking the time to join us today. It’s been a pleasure.  

Dr. Jacqueline Garcia:

Thank you.  

Katherine Banwell:

And thank you to all of our collaborators. To learn more about AML and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for being with us.   

Thriving With AML | Tips and Support for Navigating Treatment

How can you navigate care and thrive with acute myeloid leukemia (AML)? In this webinar, Dr. Jacqueline Garcia, an AML specialist and researcher, discusses the treatment and management of AML. Dr. Garcia will review factors that impact therapy choices and shares advice and resources for people living with AML.
 
Dr. Jacqueline Garcia is an oncologist and AML researcher at the Dana-Farber Cancer Institute. Learn more about Dr. Garcia.

Download Resource Guide

See More from Thrive AML

Related Resources:

How Can You Thrive With AML Advice for Navigating Care.

How Can You Thrive with AML? Advice for Navigating Care

The Benefits of Being Pro-Active in Your AML Care

What Are the Phases of AML Therapy


Transcript:

Katherine Banwell:

Hello, and welcome. I’m Katherine Banwell, your host for this webinar. Today’s program is about how to live and thrive with AML. We’re going to discuss how to live well with AML and why you should play an active role in your care. 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.  

Well, let’s meet our guest today. Joining me is Dr. Jacqueline Garcia. Dr. Garcia, welcome. Would you please introduce yourself?  

Dr. Jacqueline Garcia:

Yes. Hi. My name is Jacqueline Garcia. I’m an oncologist at the Dana-Farber Cancer Institute. I’m a clinical translational investigator. And what this means is I take care of patients with acute and chronic leukemias. I focus mainly on patients with acute myeloid leukemia. The investigator part means, in addition to seeing patients, I spend a lot of time writing, developing, and executing clinical trials in the AML space. We know that there have been so many wonderful therapies that we helped to move froward and bring to the field and so there is more work to be done. So, having active investigations is a key part of this role.  

Katherine Banwell:

Excellent. Well, thank you for taking the time out of your schedule to join us today. We really appreciate it. We start all of our webinars in our Thrive Series with the same question. In your experience, what does it mean to thrive with AML?  

Dr. Jacqueline Garcia:

I think that’s a really great question and I’m glad you’re asking me now as opposed to a decade ago. In the last several years, we’ve had a tremendous number of drugs that got FDA-approved and a lot of exciting clinical trials that have not only shown efficacy and safety but really some long-term responses. So, we can now focus on not just finding what drug can work, which used to be our problem 10 years ago, since we had very limited therapeutic tools, meaning treatments. We now have several treatments available.  

So, when I think of what it means to thrive, it’s identifying the right treatment for each individual patient with acute myeloid leukemia, because what might be recommended for one patient may not be the right for another. And there are many different patient- and disease-related factors that go into that decision-making.  

Katherine Banwell:

Thank you for that, Dr. Garcia. It helps guide us as we move into our conversation. Typically, there are a number of team members to care for a patient. Who is part of an AML healthcare team?  

Dr. Jacqueline Garcia:

Absolutely. We definitely cannot work on our own. Our team is very large, and it’s because these patients require a lot of support. At a bare minimum, a healthcare team will include at least one physician or an oncologist. The AML healthcare team might also include a second oncologist – that could be a bone marrow transplant doctor.  

Other members that are very critical include having a mid-leveler available that’s a physician assistant or a nurse practitioner. Often, an oncologist who runs a busy practice, who takes care of patients that could be very sick, like AML, they work in partnership with often very talented physician assistants and nurse practitioners. I know I do.  

In addition to that, I’m at an academic center so I’m super fortunate. I have really amazing and very smart hematology oncology fellows and residents that also follow to learn how to take care of patients. But we also, in the background, that patients don’t see – we have a pharmacist that helps us with making sure that drugs are prescribed correctly. They often call the patients with oral therapies to follow up. We have financial resource teams to help patients, to link them to LLS for support for bills that might come up, or transportation, or linking them up to other services that could help to defray or reduce costs.  

So, the healthcare team is quite extensive. But in terms of those that are patient-facing, it’s primarily the MDM that are mid-leveler. Some teams operate also with a nurse or a nurse care coordinator. That’s pretty common, too. And that person helps to not only schedule but also to answer pages or phone calls from patients if the medical team is not doing that.  

Katherine Banwell: What about a social worker or psychologist?  

Dr. Jacqueline Garcia:

Oh. Yes. Yes. So, absolutely. So, every patient can be offered, if needed, access to an inpatient or outpatient social worker. Often, if my patients are admitted we have them see a social worker because that’s fairly seamless. Otherwise, for outpatient, if we identify any particular needs or there’s an interest, we’ll link them up with a social worker. This is the same that goes for physical therapy, or nutritionists, or those other ancillary services that can be really critical when patients are getting started.  

Katherine Banwell:

Yeah. Of course, getting appropriate care and treatment is essential to thriving. Can you walk us through the classes of treatment that are considered when choosing an AML treatment approach?  

Dr. Jacqueline Garcia:

Yeah. In terms of the different classes of treatments, I would say we think of probably three broad categories. One would be – sorry, four broad categories. One would be intensive chemotherapy. And that involves generally hospitalization. Another would be less intensive therapy. That could involve a mixture of inpatient or outpatient therapy. That could also include targeted therapy. The third would be clinical trials, which can include any of the former options I recommended, but they would be in an experimental study. And the fourth would be focusing solely on supportive care or hospice for patients that are too sick to receive therapy.  

Other aspects that are specific, such as pills, versus IV, versus role of transplant, I don’t see it as being separate. You don’t go right to transplant when you have a diagnosis of AML. You have to be in remission. So, transplant, for instance, would come after an intensive therapy or after the less intensive chemotherapy. So, I see that as being the second step once I choose the right treatment option for the patient.  

Katherine Banwell:

And when you’re talking about transplant, you’re talking about stem cell transplant, right?  

Dr. Jacqueline Garcia:

Yes. Stem cell transplant, bone marrow transplant – they mean the same thing. We recruit stem cells from donors that are related or unrelated, and we mobilize them from bone marrow to blood. And so, we can collect stem cells either from blood or bone marrow at this point. So, that’s exactly right.  

Katherine Banwell:

And what about targeted therapy?  

Dr. Jacqueline Garcia:

We have targeted therapy available that’s IV or pill form. And so, any one of these options can be considered. But everything is very patient-specific, and I am very happy to tell you some of the categories and nuances of things that I look at, because I don’t usually just offer patients a menu.  

I tell them what’s appropriate based on their patient characteristics, meaning what their liver function is, their heart function, their history, medical history, what their labs show. And then, I look at their disease history. We are now in an era where we have options. So, I look to see are there mutations that are targetable. Are there not? Are there markers on the surface of their leukemia cells that suggest that there’s a target for an immunotherapy?  

So, we don’t offer classes per se without it being specific. So, I always look to see what are the patient disease-specific characteristics, and then I start the conversation about what the potential options could be and then what I think the best option would be for that particular case.  

Katherine Banwell:

As a researcher, Dr. Garcia, you’re on the frontlines of AML treatment. Are there new and emerging therapies that patients should be aware of?  

Dr. Jacqueline Garcia:

Yeah. I think we’re at this really exciting point now where we had for a long time just been giving people standard two agent intensive chemotherapy. We have been studying in Phase II and Phase III settings, and even in Phase I – which means testing safety out for the first time. We’ve been moving a lot of treatments to more mature settings where we’re testing the addition of a third drug. So, for people that are getting intensive chemo, we’re looking at, “Can we add a pill to augment responses deep in them to reduce risk of disease returning?”  

For less intensive chemotherapies, one of the most common regimens we now use is something called azacitidine (Vidaza), which is a hypomethylating agent that is given by IV or subcutaneous administration. Plus, a pill called venetoclax (Venclexta).   

We helped to get that FDA-approved a couple of years ago. That combination of therapy, we call that a doublet, meaning it’s two drugs – because it’s so well-tolerated and active, we’re now asking the greedy question of, “Well, can we make it more active for patients since we’re seeing how well-tolerated it is?”  

So, there have been a lot of therapies that are currently under investigation that are adding a third drug to these less-intensive doublets. So, there’s a lot of therapies under investigation to test, “Can we add an immunotherapy target? Is there another pill that we can add? Is there another targeting mutation to add to the doublet?” So, we’re looking at AML therapies from different angles. We’re looking at adding something to the existing new standard of care – those are these new, so-called, triplets.  

We’re looking at still the role of cellular therapy or CAR Ts targeting leukemia cells from an immunotherapy standpoint.  

That remains underdeveloped overall, and we have not succeeded as well, like our lymphoid colleagues in the lymphoma and acute lymphoblastic leukemia realm where there are drugs that are active and FDA-approved.  

So, we’re still trying to identify the right target. But those are some of the areas that are currently under study.  

Katherine Banwell:

You touched on this earlier, Dr. Garcia, but I’d like to get into a bit more detail. With all the treatment options available, how do you decide who gets what? Tell us what is considered when choosing treatment for a patient.   

Dr. Jacqueline Garcia:

When I – this is a complicated question, because it’s not like you follow any particular algorithm. But when I meet a patient, I make a decision on what’s important to the patient and what’s  their goal. If I know – I need to understand their overall health to get a sense of are there ongoing competing risk factors that are active and more likely to impede with response, ability to deliver chemo, ability to get to transplant, something that tells me that’s not a possibility, or is their age too advanced – meaning greater than 75 – where we know that some of the treatments are not safe to deliver in that setting?  

So, I take a look at a patient’s overall health and age to make a decision. I take a look at bone marrow biopsy and lab findings to understand the flavor of their leukemia, from chromosomes to mutations. And because I am familiar with the data to give me a sense of what’s safe, what’s tolerable, and importantly what types of diseases, or subtypes of AML, would respond to one therapy over another, that’s how I formulate a recommendation.   

And based on all of that, all together, I’ll talk to them about treating the AML in steps. The first step is getting them into a remission, which can be done regardless of therapy type. That means to get their bone marrow under control, blood counts to recover. The second step, which is a more involved conversation that I often give a little bit of a hint of, but I go into greater detail over time, because we will see each other quite a lot, whether in the hospital or in clinic, is how to keep them in remission.   

And that’s where details about things like transplant come into play. I do my best to not overwhelm them, because when a patient hears the word transplant – and that’s often what they hear from family and friends because that’s what you can Google – they don’t know that there are many things, or many weeks of therapy, that have to happen in advance of transplant even being considered or happening. And transplant can’t even happen until someone’s in remission.  

But that is always on the forefront of a leukemia doctor’s mind, “Can I bring this patient to a transplantation? How successful will I be and what else do I need to give them to get them there sooner, safer, with a deeper response?” So, that way transplant could be successful. Transplant, by the way, is when we give a patient someone else’s stem cells that match their HLA typing, or their white blood cell signature.  

And it helps us to use someone else’s immune system to completely irradicate any microscopic leftover leukemia in a patient. But that is only successful when patients have good disease control or remissions. And that is only also successful if we have a donor for the patient, both of which  require at least several weeks to a couple of months of therapy. But that process is always initiated and ongoing in the background. And so, we often do this in piecemeal, because getting a diagnosis is already overwhelming. Learning about treatment is overwhelming.  

Learning about the frequency of labs, transfusions, being hospitalized, and then details about what a transplant would entail can be also overwhelming. But a lot of family and friends like to ask, because they feel like that is one way they might be able to help a patient. So, I know that they often eagerly ask the patient, “Well, what about this? How can I help?”  

Katherine Banwell:

Right. I can imagine that patient preference is also considered. But what kind of questions should patients ask about their treatment regimen?  

Dr. Jacqueline Garcia:

I always tell patients that I care very much about things like travel, hotels, all that jazz. But I always tell them let’s first talk about their health, what treatment I would recommend based on the available options and what their disease would mostly respond to, because I want it to be successful. And I always tell them let’s reserve questions on how it’s going to be done for last. I call that the logistics. I will never bring up or recommend something that could never be possible. But that being said, I try not to let the commute determine the decision.  

Whether or not there needs to be a hospitalization versus a hotel stay. I always consider then the background, but that financial decision should not drive the best treatment choice for a patient. Very fortunately, we’re in a country where patients have the ability – often, not always – to seek second opinions or to travel to academic centers.  

And because AML is an emergent or life-threatening disease, many insurance providers allow patients to come up to a big center to be treated, which I think is more than appropriate. So, we get into details of logistics last, because that’s the one thing that we can often overcome by providing additional resources and support. In terms of patient preference, if that’s what you mean with that, I would say I leave logistics to last, but we always consider and we do our best to accommodate.  

And that might be where we inform them we will look into getting a local partner to help us with additional therapies after the first month or upon discharge. So, it totally depends on the scenario for a patient, whether or not they have a local provider and a local hospital that could accommodate acute leukemia. I always tell patients ideally you don’t want to go to a place that only sees this once per year. You want to go to a place where everyone has seen it multiple times, including the nurses on the floors.  

So, that way, when there’s a complication, everyone knows what to do. We don’t want any “surprises” when it’s really just run-of-the-mill standard stuff for us every day. In terms of what patients desire, we always keep that in the conversation of their level of support. Can they swallow pills? Are they able to cope with being in and out of the hospital? All that stuff gets considered, but I think if they hear about the plan, about what’s required, when my expectation would be for a response, when the frequency of trips to a big city would decrease, how I could get a local partner to help with some of the lab or transfusion burden.  

Many of those preferences that they thought they had diminished, because they recognize that we found a way to make it work.  

Katherine Banwell:

Okay. Well, that’s really good to know. You touched on oral therapies a bit ago, and I know that they’re available for certain patients. Do you have any advice for patients who are in charge now of administering their own therapy?  

Dr. Jacqueline Garcia:

Yeah, I think that taking pills in general is hard for anybody, whether they’re naïve to pills. I definitely have patients that have never been on anything, and suddenly they’re on many medicines, to other people that are managing multiple medical conditions and this is yet another burden to add. I would say having an oral regimen is wonderful. It offers a lot of convenience. But we are all very thoughtful, and we all need to be proactive about looking for drug-drug interactions, because often there could be increases in the chemotherapy presence when another drug is on board.  

Sometimes, antibiotics are added on but they don’t realize it can add to side effects to chemotherapy. So, I would say number one is always make sure your oncology team is aware of the medications you are on or get recommended to add on in the midst of therapy, so we can make sure there are appropriate dosage estimates or if a particular drug should be avoided, then we can do that.  

I would say, too, having oral therapies is great, but there’s also financial toxicity that comes with it. Sometimes copays can get hefty. So, just because it’s oral, it’s not always convenient financially. Also, when things are oral it can add to more GI or mal gut toxicity. So, we’re always keeping in mind how many oral therapies, what drugs they are, so we don’t increase nausea and diarrhea, which can happen frequently when you’re requiring the GI tract to absorb the therapies that are necessary to eliminate the disease.  

So, all these things are under consideration. But to help people that are on oral therapies, it’s helpful to let your providers know if you’re noticing a pattern of nausea, so we can premedicate, have you take a nausea medicine before you take the chemo. You could also put a timer on your phone if you’re not used to taking medicines to serve as a reminder. You could create little calendars or check off on a paper calendar when you’ve taken a drug if you need help with reminding.  

So, there are little tricks like that. I always consider using a pillbox if you don’t have other pills to mix in and if you’re the only one touching it. I don’t want anybody to be exposed to therapies that they shouldn’t be otherwise.  

Katherine Banwell:

That’s good advice. Thank you. If a patient is feeling uncomfortable with the direction of their treatment plan or their care, should they consider a second opinion or even consulting a specialist?  

Dr. Jacqueline Garcia:

Oh, 100 percent. I would say – I think that I’m spoiled. I’m a leukemia specialist, so they’re already seeing a specialist when a patient sees me. I don’t take care of any other cancers. But, I would say, for anyone seeing any oncologist in general, I would – number one, it doesn’t do the medical team any favors if you withhold any feelings of how the treatment’s going. Meaning, if you feel uncomfortable or that you’re having symptoms or people are taking too long to get back to you based on your experience.   

I would just make sure you do your best to at least let them know so that they have the ability to adjust or accommodate whatever need you might have that might be different than what they’re used to, because every patient’s different. Some people have a really great support system. Or they have a little bit of experience of being a patient. Different coping mechanisms. Everyone’s different. There’s no right or wrong. But I would just make sure that it’s clear with your existing team because they’re actively seeing you. Give them a chance to make the experience better.  

I would for sure seek a second opinion. Don’t delay – I will just put this disclaimer. I would not delay treatment for an AML if your current doctor is giving you a good plan and you feel confident that they have looked into whether or not you need to go to a bigger leukemia center and all that other stuff. But if you feel like they are giving you a good plan, don’t delay your therapy in the beginning, because you might get sick.  

If, however, there is demonstration of safety and time to see someone within a short timeframe for a second opinion at the time of diagnosis before treatment started, then that’s okay. But wouldn’t wait a few months to go looking around, because that could put your health at risk. Once you’re on treatment, seeking a second opinion, if you’re dissatisfied with your ongoing team, it’s fine. I always want patients to feel comfortable with their treatment plan.  

But I would recognize that you want to make it clear to your current team that they’re still helping you and responsible for your treatment. Because if you, for instance, started seeing multiple doctors and they won’t know who should be helping to follow up on certain things, who’s going to be scheduling the next round of therapy. And that ends up putting more ownership unnecessarily onto the patient where they might not have needed to have all that extra responsibility. So, I would just say just make sure that’s clear. Yeah.  

Katherine Banwell:

Dr. Garcia, you mentioned earlier the fact that some therapies can cause a lot of side effects, like nausea. And certainly, speaking up and telling your healthcare team how you’re feeling and what some of the symptoms and side effects are, that’s really essential. What is the impetus for someone to consider changing treatment if something is just absolutely not agreeing with them?  

Dr. Jacqueline Garcia:

So, there are many reasons to change a treatment. One is a patient doesn’t tolerate it. It depends on what the issue is. Is it something that’s serious, like a liver or enzyme abnormality that is very abnormal, or a new cardiac problem where it would warrant a change or a dose reduction? That makes sense. There is definitely – often, there’s a lot of guidance in the package inserts or within a clinical trial and how to manage that. But if patient has some intolerabilities that could be overcome with standard supportive care methods, I would make sure we’ve done that.  

So, I would make sure you give you medical team the chance to fix any nausea. We have so many great antinausea drugs. I would want to make sure – or if constipation or diarrhea. It’s often a GI issue that patients get really bothered by.  

I would try to delineate whether or not the side effect was really from the chemo or is from the leukemia that is not yet under control. Or is it another medical condition or a drug-drug interaction that was missed. So, I would do my best to make sure there wasn’t something that was fixable or something else that should be addressed. We otherwise would recommend changing therapy for an extreme intolerability if there was another equivalent better option. And if someone’s disease does not respond to treatment, then we would consider another therapy, too.  

Katherine Banwell:

Dr. Garcia, I want to make sure that we get to some of the audience questions that were sent to us prior to this program. Let’s start with this one.  

 Jerry had this question. “How long can patients stay on azacitidine and venetoclax before relapse or toxicities force them to abandon treatment?”  

Dr. Jacqueline Garcia:

So, this is a good question. I would say azacitidine and venetoclax just got FDA-approved just shy of five years now, and it’s totally changed our treatment paradigm in many great ways. It was initially approved for patients that could not get intensive chemotherapies or were above 75. We call these our older patients, our more vulnerable.  

And we demonstrated and compared to azacitidine alone. It was given with placebo. We saw that the combination of azacitidine and venetoclax not only was safe, well-tolerated, it led to two-and-a-half times higher complete remission rates and impressively longer survival. That’s all we care about, patients are living longer. So, one of the things that we are appreciating in 2023 now, now that we have more patients on azacitidine and venetoclax, is that we have many patients that are long-term responders.  

So, in the original clinical trial we’ve been reported – and we just submitted the update for the long-term follow-up that we presented at the American Society of Hematology meeting in 2022, in December.  

We presented the long-term follow-up data that shows that responses can be durable and even as long as two years or three years in some patients. The average amount of time the patients are on therapy is somewhere between one-and-a-half to two years. But not every patient performs like an average patient.  

We have some that respond for less time. We have some that respond for a longer time. So, I definitely have a few patients that have been on combination therapy, and we’ve gone to year three, then four, and two that got to year five. And that was using the original indication of older the 75, no intensive chemotherapy. Most of those patients in the original trial and led to the approval were not transplant candidates. But once those drugs got approved, more patients that were older started getting this therapy.  

And so, the durability of this treatment might be longer for people that don’t have competing health problems and for specific mutation subtypes. There are a couple of mutation subtypes that include IDH2 and NPM1, where we’ve seen some extreme long-term responders.   

And then, there are others that are much shorter. So, I would say it’s very individual. In terms of toxicities in general, the regimens very well-tolerated. And if it’s not, often it’s because there should be supportive care, prophylaxis, and adjustments to the dosing strategy, which has been well-published. Sometimes, if you have a treating oncologist that is less familiar, they won’t know the nuances of how to adjust the doses, so I would ask your local oncologist to reach out to anybody that was part of the original trials. Often, a lot of us are very responsive to helping out our colleagues to help patients to stay on treatment.  

But at the end of the day, if a patient loses response or has a bad toxicity that makes it very difficult, we have to move on to another therapy.  

Katherine Banwell:

Of course. Carrie sent in this question. “What percent of patients relapse and what percent of patients relapse more than once?”  

Dr. Jacqueline Garcia:

Okay. So, this is a question that I can certainly answer, but I would say it depends on the context. So, if I was taking – any time a patient asks me that, I always ask them what they want to know and what they don’t want to hear, because sometimes hearing numbers can be really daunting to patients overall.  

So, a very large number of patients with leukemia can go on to relapse, which is why, if you’re on a treatment like azacitidine and venetoclax, we continue it every month as long as we can with dose reductions to help with tolerability.  

And that’s why, if you got that regimen or intensive chemo or another clinical trial and you get into remission, we ask the question of can we transplant this patient to do our best to cure them long-term to avoid and reduce the chance of a relapse. So, even with transplant, which remains our gold standard for long-term curability – it’s the only treatment we have that has a guaranteed track record of cure – not every patient that goes to transplant will remain in remission.  

If I were to be asked, “Well, how many relapse,” I would say it depends. I would say if I took the average patient, maybe 40 to 50 percent will relapse. But if you ask me for certain mutation types it could be 90 percent are cured or only 20 percent are cured. So, it’s very individual. It depends on age. It depends on mutations. It depends on the level of response they had before they go to transplant.  

So, I would say even though the word relapse is very scary or disease coming back is definitely a scary thing, there are a lot of people, including me, that are working on ways to reduce risk of relapse, improve how we transplant, improve the treatments around and after transplant, and improving frontline and relapse therapies.  

I think you had a second question of what happens if you relapse once and then what about if relapse happens again? I would say that getting into remission the first time is always the easiest. The way I always think about it is, you kill off all the bad cells that are the easiest to die the first time around with chemotherapy. Anything that’s left behind are often the resistant types. And so, getting into a second remission or responding the second time around with treatment is doable, but it’s much harder.  

So, I would say the majority of patients that relapse the first time will relapse the second time, unless we can successfully bridge them to a transplantation.  

Katherine Banwell:

Yeah. Dr. Garcia, as we close out this conversation, I wanted to get your thoughts on where we stand with progress in helping people live longer and thrive with AML. What would you like to leave the audience with?  

Dr. Jacqueline Garcia:

I think that this is – I feel very lucky with when I entered the field, that in this last decade, as I’ve developed – my time at Dana-Farber, for instance – I’ve seen that there have been so many drugs that we helped to get approved that are now in the hands of local oncologists and other academic oncologists, suggesting that the clinical trials are a gateway to improving treatments and offering new options.  

 We’ve gotten better at understanding what mutations and chromosomes means and personalizing medicines, and that has allowed us to develop smarter and better clinical trials, which we hope we will get to keep approving and making more available to patients. So, I think that this is a really good time for AML, meaning we have more than one option, that is for sure. We can now think about what the patient wants, what the patient, and what their patient disease has in order to make a decision. We weren’t able to do that before.  

So, we can really involve patients so they understand why we would recommend one option versus another. And we are still not done with investigation, even though many drugs got approved in the last five years. There’s a lot more progress to be made, especially in areas that we touched upon, from approving getting patients to transplant, reducing relapse risk, keeping people in remission. Those are all things that I’m personally working on in the clinical trial space and things a lot of my colleagues in the world are working on, too.  

It’s very important to all of us. So, I would say be hopeful that we are not done. There’s a lot of great options out there. We really can personalize. There are a lot of options out there, but everyone will get offered their best therapy and the first-line therapy is the most important. And I am very hopeful that we will keep getting better at prolonging remissions and durability of those responses.   

Katherine Banwell:

Dr. Garcia, thank you so much for taking the time to join us today. It’s been a pleasure.  

Dr. Jacqueline Garcia:

Thank you.  

Katherine Banwell:

And thank you to all of our collaborators. To learn more about AML and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for being with us.