Tag Archive for: clinical trial

What Do Genetic Tests Reveal About My CLL Treatment Options?

What Do Genetic Tests Reveal About My CLL Treatment Options? from Patient Empowerment Network on Vimeo.

Genetic testing results can influence a chronic lymphocytic leukemia (CLL) patient’s treatment options and provide a more in-depth understanding into their disease. Dr. Phillip Thompson, a CLL specialist, reviews three important testing results that can impact treatment timing and approaches.
Dr. Phillip Thompson is an Assistant Professor in Medicine in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about this expert here.

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Dr. Philip Thompson:

So, there are three main things we look at before initiating treatment in a patient.

One is what we call the IGHV mutational status of the patient. And this basically splits people into types of CLL. So-called mutated or unmutated. And this is a relatively complex concept. Basically, what happens in normal B-lymphocyte development, so B-lymphocytes are part of your immune system. Their job is they have a probe on the surface of the cell that looks for invading microorganisms. And when they find an invader, this probe binds to the organism. And then the cell actually undergoes, as part of its normal development, a process of mutation so that it makes the best possible antibody to fight that infection. So that’s a normal process that the B-lymphocyte undergoes when fighting infections.

So, CLL can arise from what we call a mature antigen-experienced mutated B-cell, or it can arise from a naive B-cell that has never gone through that process, in which case, it will have an unmutated IGHV. Now, it’s kind of counterintuitive, but the patients with a mutated IGHV generally have better outcomes. That type of CLL is less proliferative, it doesn’t grow as fast, and it also tends to respond better to certain types of treatment. Particularly, it responds better to chemotherapy than patients with unmutated IGHV.

However, the difference between those two is less important if you’re getting some of the newer therapies. Particularly, it seems like if you receive BTK inhibitors, it doesn’t really matter if you have mutated or unmutated IGHV, patients are responding very well. But I like to know whether they have a mutated or unmutated IGHV because it’s helpful for giving the patient an expectation of how their disease is likely to behave biologically.

But also, if they have a mutated they may be a candidate for chemotherapy-based treatment. Whereas if they have unmutated IGHV, I don’t use chemotherapy for those patients.


The second thing is a test called FISH. And FISH looks for chromosome abnormalities. So, we have 46 chromosomes, 23 from our mother and 23 from our father. They contain all of our genetic information. And in malignant diseases, you can have major abnormalities in the chromosomes of the cancer cells. Not in the rest of your body, just in the cancer cells. And they happen because of errors that are made when the cells are replicating their chromosomes.                                                                 

So, in CLL, there are four common abnormalities that we look for in a test called FISH, and they tell us a lot about the patient’s prognosis. And there’s one in particular that we look at that has a major impact on our decision making, and that’s a deletion on Chromosome 17.

So, a missing piece of Chromosome 17. And the reason that that’s important is it tends to be an aggressive form of CLL. It also does not respond to chemotherapy, or if it does, the responses are very, very short-lived. So basically, that’s a contrary indication to receiving chemotherapy for your CLL when you should receive another form of therapy if you have a 17-P deletion.

And then, finally, we look at a type of – we look for individual gene mutations in the cells. And that’s different from IGHV mutational status, although the names are kind of similar.

So, in CLL, there are numerous genes that can be affected by mutations that alter the function of the gene. In some cases, it makes the gene non-functional; in some cases, it changes the function in some way that perturbs the normal functioning of the cell and contributes to the malignant transformation of that cell.

So, the most important one, again, relates to a gene called TP-53. So that’s the gene that is deleted if you lose a piece of Chromosome 17. It’s located on the P arm of chromosome 17. If you mutate that gene, it has the same consequences essentially for the cell as if you delete it by deleting a piece of the chromosome. And the two often go together, so you’ll have a 17-P deletion and a mutation of the TP-53 gene on your other Chromosome 17. Because remember, you have two chromosome 17s. So, if you lose both, it may be even worse than only having one. However, it does seem that if you only have a mutation on the TP-53 gene, but you don’t have a deletion on Chromosome 17, that the responses of those patients to chemoimmunotherapy are still really poor.

So, it’s very important to find out, do you have a TP-53 mutation as well as do you have a deletion on Chromosome 17 before you embark on treatment, particularly if that treatment is going to be chemotherapy. So, those are the three things that we look for before    we start any patient on therapy.

Fact or Fiction? Myeloma Treatment & Side Effects

Fact or Fiction? Myeloma Treatment & Side Effects from Patient Empowerment Network on Vimeo.

When it comes to online myeloma information, how do you separate fact from fiction? Dr. Irene Ghobrial shares facts about current myeloma treatments, common side effects and emerging research. Download the Program Resource Guide, here

Dr. Irene Ghobrial is Director of the Clinical Investigator Research Program at Dana-Farber Cancer Institute and Professor of Medicine at Harvard Medical School. Dr. Ghobrial specializes in multiple myeloma (MM) and Waldenström macroglobulinemia (WM), focusing on the precursor conditions of monoclonal gammopathy of undetermined significance (MGUS) and smoldering myeloma. More about this expert here.

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Welcome to Fact or Fiction: Multiple Myeloma Treatment and Side Effects. Today, we’ll review common misconceptions about myeloma. I’m Patricia Murphy, your host for today’s program. Joining me is Dr. Irene Ghobrial. Dr. Ghobrial, why don’t you introduce yourself?

Dr. Ghobrial:

My name is Irene Ghobrial. I’m a professor of medicine at Dana-Farber Cancer Institute, Harvard Medical School.


Great, thanks so much. Before we get started, just a reminder: This program is not a substitute for medical advice, so please consult your care team before making any treatment decisions. Okay, Dr. Ghobrial, let’s get started.

Let’s talk about some of the things, first, that we hear from patients. You tell me whether or not this is fact or fiction. Here’s one: “There are a number of treatment options for myeloma.”

Dr. Ghobrial:

Fact. It’s amazing because I trained in the old days – and, this shows you how old I am – when we only had bad chemotherapy: Vincristine, Adriamycin, and dex. None of you would even know about it.

Then, we had had high-dose dexamethasone, and that was it, and then we had stem cell transplant, and that’s all we had until suddenly, we had thalidomide, lenalidomide, bortezomib, carfilzomib, ixazomib, and you think about it, we are now in an era where we have 15-20 new drugs, we have another 15-20 coming up, we have an amazing time to completely cure myeloma in the future, and that’s just an exciting time to see that happening in the last 15 years of our lifetime, when patients were living three years, when we had – I remember five percent complete remission rate.

Now, we expect that all of our patients should get into a deep remission into potentially MRD-negative disease, and that’s just the beauty of how myeloma has changed completely.


Well, you’ve already busted our second myth, I guess, that there is no cure for myeloma.

Dr. Ghobrial:

That’s correct. There is no cure for myeloma, but there is a long remission, and the question is if someone lives for 20, 25, 30 years without evidence of myeloma and they die from something else, it’s a step forward. I would love to see us say to a patient, “You are cured,” but until then, we’re getting longer and longer remissions.


How about this one? “Only blood relatives can be donors for bone marrow or stem cell transplant.”

Dr. Ghobrial:

That’s not correct at all. If we think about it, what is stem cell transplant? There are two types. There’s something called autologous stem cell transplant, meaning it’s from myself, so that means that I’m taking my own stem cells, and the whole idea of that autologous transplant is basically high-dose chemotherapy.

So let’s take your own cells before we give you that high-dose melphalan, give the chemo, and then give them back to you, so that you’re not with low blood counts for two weeks, four weeks, you’re only with low blood counts for a couple of weeks. So, that’s autologous transplant; that means I’m giving my own stem cells to myself.

Allogeneic stem cell transplant, which we rarely do now in myeloma, is from another person, and that could be from a relative, but also can be from unrelated donors if they are matching us, but that’s very few cases.


Let’s get an overview of available myeloma treatments.

Dr. Ghobrial:

Oh, boy. Okay, how long do we have here? It depends. The moment I see a patient – and again, maybe we can start with smoldering myeloma because that’s an area I’m really excited about.

If you have asymptomatic disease, it does not mean you have to watch and wait until you fall apart, until you have bone lesions, until you have anemia. We want to see those patients early because we have a lot of clinical trials, and potentially, the cure may actually be in an earlier precursor session when we treat you earlier before you have the disease.

But, the standard of care is when you have symptoms – anemia, hypercalcemia, lytic lesions, and renal failure, or other things like 60% plasma cells – we say you have active multiple myeloma, and in that case, we start saying, “Well, are you a transplant candidate or not?” In the old days, it used to be by age, but now, we say age is just a number, so it really depends on if you have good organ function, are you in an active good state, do you have good lungs, good heart, are you willing to take the transplant, because now, there’s a big discussion whether we should transplant patients or not.

And then, at the end of the day, we’re starting to actually blur that, saying that most of our treatments are almost identical, whether you are old or young, whether you’re a transplant candidate or not. It depends on frailty. Can you tolerate this treatment or not? Maybe a few years ago, we used to say a three-drug regimen is the best way to go.

Now, most of us are starting to say four-drug regimen up front is the way to go, which is an antibody – currently, it’s daratumumab – a proteasome inhibitor – it could be bortezomib or carfilzomib – an immunomodulator – likely, this is lenalidomide – and then, dexamethasone. That’s sort of the option that we have right now, at least in the U.S.

If you go to Europe, you’ll find us using different drugs, like thalidomide or other things, but most of us are thinking of a four-drug regimen to think of our up-front myeloma treatment to get you the best remission, eventually MRD-negative disease, and then we talk about transplant or no transplant, and then, of course, we talk about maintenance.

We want to keep everyone on maintenance therapy; the question is how long, which maintenance, do we use one drug or not? So, there is a lot to be discussed in treatment of myeloma, and that’s the beauty of it. It’s truly an art and science together. It’s not just “Here’s a combination because you have this treatment.” We really personalize therapy for you.

We look at your cytogenetics, your FISH. We say you have high-risk cytogenetics or not, you’re young or not, you have good organ function or not.

There are so many things that we put in consideration when we come up with a treatment plan for a patient.


We’ve been talking a little bit about what patients believe when they come in, some of the things they’re thinking about. What else do you hear from patients that you either have to correct or affirm when they come into your office?

Dr. Ghobrial:

A lot of things. I think the first thing is, of course, they say myeloma is fatal, and they’re so scared, and absolutely, I understand that, but the median survival has become so much better, so much longer. There is a lot of hope, enthusiasm, and excitement right now with the treatments we have. The second thing is most of our treatments are not your typical chemotherapy, so unlike breast cancer or other cancers where you lose your hair, you’re throwing up, you cannot work, you have to take time off, most of our drugs now, people are working full-time, they’re active, you don’t lose your hair, so probably, no one has to know unless you tell them.

And, I think that’s something important for a patient to think about. It’s their own personal life, and not having to interrupt that. I think that’s very unique. So, these are a couple things that, as they come in, that anxiety of “Oh my God, I have cancer,” and then, taking a deep breath and saying, “Now, how do I handle this situation?”


Sure. What about clinical trials? What common misconceptions do you hear from patients enrolling in trials?

Dr. Ghobrial:

There’s a lot of misconceptions, and it’s unfortunate. I would say I would absolutely go on a trial if I can. I’m a believer in clinical trials because they’re the way forward to bring in new therapies and new options. I think a lot of people think that we’re experimenting on them when we’re doing clinical trials, meaning that it’s first in human, meaning it’s the first time we try this drug, and I would say that most of our clinical trials are not first in human.

They’re not the very first time we’ve tried them. Likely, those are drugs we’ve tried, we know the side effects, we know the toxicity, but it’s the first time we’ve put it in a different combination or it’s the first time we’ve put it in a specific subset of patients to look at response or at overall survival.

Most of the trials – so, before you decide “Oh, it’s a trial,” just think – is this a phase 1, a phase 2, or a phase 3? Phase 1 are usually that first time that we try in a population. Phase 2 are usually we know already what happens, we know the toxicity, we’re bringing it to look at the response rate in general or the survival, and then, phase 3s are the bigger studies, going to the FDA for approval.

The second thing is you want to think about is there a placebo arm in it. Most of my patients really worry about “Oh my God, you’re gonna give me the placebo,” and I’m like, “No, we don’t have a placebo arm in this trial. You’re taking the drug that we tell you about.” So again, depending on the trial – read it carefully – there may be a placebo arm, but in most of them, it’s not a placebo arm.

So, I would personally go ask the doctor every time, “So, you’re talking about standard of care. What else do you have? Do you have clinical trial options or not? What’s new?” Almost every single new drug that we’re gonna get approved in the next 5-10 years from now is what we have today in clinical trials. It would be cool to try and get access to those earlier.


So, there’s a significant amount of vetting that goes on before clinical trials are actually in process on humans.

Dr. Ghobrial:              

Oh, absolutely.


What are the common myeloma misconceptions about treatment side effects?

Dr. Ghobrial:              

I think the biggest thing is the loss of hair, the nausea, and fatigue, and to the point that I cannot travel, I cannot see my family, I’m gonna be so immunosuppressed. And again, that’s a huge misconception. Yes, there is toxicity for every drug. Even if you take aspirin, you have toxicity from it.

But, every drug has risks and benefits, and currently, the combinations we have are just impressive that they are well tolerated in general. I’m not saying there is no side effect – there is, for every different class of agents, there are, and you will go through those side effects with your doctor in detail – but in general, yes, you’re slightly immunosuppressed, you have to take care of it, and I said it yesterday to one of my patients – if someone is looking very sick in front of you, don’t go and hug them.

Christmas is around the corner, and we want to make sure people celebrate and enjoy life and enjoy the holidays with their family members.


Dr. Ghobrial, let’s talk about some of the things that patients are concerned about when they come in about treatment side effects, and maybe some of those things aren’t true. You tell me. Treatment side effects are unavoidable – we already talked a little bit about that. How about this one? “Myeloma patients should visit the dentist more frequently.”

Dr. Ghobrial:              

So, there is something about the bisphosphonates that we give patients, and they can cause – in a very rare number of patients – something called osteonecrosis of the jaw.

In the old days, when we didn’t know about that side effect, people would go get a root canal, come back, and have a big problem of osteonecrosis of the jaw with severe pain, and it doesn’t recover.

So, we’ve learned our lesson. We know very well that we hold Zometa or zoledronic acid if they’re getting any procedures. We make sure they don’t get surgical procedures – it doesn’t mean don’t get dental cleaning, please do the usual things for dental health, but don’t go into surgical procedures when you’re getting zoledronic acid – and we’re very careful with that.

We talk to our patients. Most dentists know about it, so I think this is something that in the old days, it was a problem. Now, we know how to medicate that.


Sure. How about this one? “Treatment causes increased risk for blood clots.”

Dr. Ghobrial:              

So, a couple of the drugs that we have – especially immunomodulators – can increase your risk for DVTs, blood clots, or pulmonary embolism, PE. So, the first thing we say is, “Let’s assess your baseline risk.

Are you someone who is at risk of clotting anyways?” Remember, myeloma also increases your risk of clotting, so you’re double. So, if you are at a high risk of clotting, then we would give the full anticoagulation. If you are not, then we would say aspirin is good enough to control that inflammation and endothelial damage that happens early on with therapy, and that can take care of it.


How about this one? “Side effects can be managed by diet and lifestyle.”

Dr. Ghobrial:              

So, I am a big believer that exercise and good, healthy living helps you in general. It makes your mood better, it makes you feel stronger, it gives you that energy because of the fatigue from the side effects, it helps with the dexamethasone because dex is a steroid, so you’re gonna be hungry, you’re gonna be eating more, and the on-and-off makes you fatigued and tired.

So, absolutely, diet and good healthy living – I’m not saying you have to go into extreme starvation and things like that. We say in general, be good, healthy living; exercise if you can.


What do you hear from your patients about side effects and treatments that they may think is true?

Dr. Ghobrial:              

I think neuropathy is very important, and we underestimate the neuropathy, so if you have numbness or tingling, tell your doctor.

That comes from Velcade; it comes from thalidomide when we used to use thalidomide, but it can happen in many patients who have an underlying amyloidosis and we did not diagnose it yet, or it can just happen as you go on from myeloma, rarely. So, tell your doctor about this.

I think the fatigue is very important to know about it because people suddenly change their life, and they want to know about that. I think the rashes that can happen with many of the drugs are very important to know about so that you’re not surprised when you get the rash. We know, for example, Revlimid can cause itching of the scalp, and that’s something that if we don’t tell the patients and they start going like this, then there is a problem.

So, it’s small things, but we want to let them know. We usually tell the patients everything, to a point of just going through all the side effects. It’s better to be aware of it, and then, if you get or not, at least you were aware.


Sure. How does one distinguish treatment side effects from comorbidities like fatigue?

Dr. Ghobrial:              

I think that’s important, and again, talking to your doctor is very important. Keeping a diary on the side is very important because you may have had some of those problems, and that could be from myeloma before you even started the drugs, and making sure that we know what’s from myeloma, what’s from your thyroid issue, what’s from your lung problems if you have asthma or COPD, what’s your diabetes if you have that or your other medications, from what are you doing with those medications.

I think that’s why when you start therapy, we tell you, “Try not to take too many other medications that we don’t know about, herbal medicines and other things, because then we don’t know what are the side effects and what’s causing what.”


Sure. You mentioned neuropathy. Let’s talk a little bit about what that is.

Dr. Ghobrial:              

So, neuropathy can come in different ways, but the most common one is numbness and tingling that you have in your tips of toes and tips of your fingers, and that can happen from medications, as we said, or from the underlying myeloma or amyloidosis. It can be painful, and we’re careful that if you have this, tell your doctor because if it get worse and worse, it’s very hard for us to reverse neuropathy, so just always tell us because we can stop the drug, we can decrease the dose rather than having you go through it.



What about this one? “An MGUS diagnosis will lead to myeloma.”

Dr. Ghobrial:     

Great question. So, let’s talk about MGUS in general. In the general population, once you’re over the age of 50, there’s a three percent change of having MGUS incidentally found, and that’s known from the big studies from Dr. Robert Kyle. Any of us walking around probably may have MGUS, and we don’t know.

We started recently a big study called the PROMISE study where we actually screen for the first time to look for myeloma – or, for MGUS – and the reason for that is we said, “You go screening for mammography with breast cancer, you go screening with a colonoscopy for colon cancer; we don’t screen for myeloma, which is an easy blood cancer with a blood test. Let’s screen for it.” So, that’s available online – promisestudy.org.

The other thing that we said is if you have MGUS, your chance of progression is only one percent per year. That’s very important to know. So, that means that in 10 years, you have a 10% chance of progression to myeloma. In 20 years, you have a 20% chance. So, if you’re 70 or 80, you may have something else that happens before you even develop myeloma or before you are at risk of myeloma.

However, that doesn’t mean that you don’t have the chance. You have a very small chance; it’s a precursor to myeloma, but it’s one of the biggest precursors to myeloma, so we always tell you, “Please go see your doctor, please do follow up with us because the one thing that’s important is we catch it early before it happens.” So, it does not always go to myeloma, but if we live for another 100 years, it may actually progress to myeloma because of the 1% chance per year.


How about this one? “MGUS and smoldering myeloma are the same.”

Dr. Ghobrial:              

That’s not true. That’s a very important question. So, in general, MGUS is diagnosed as having less than 10% plasma cells and a small monoclonal protein, less than 3 grams, and you don’t have any organ damage.

Smoldering myeloma – and, the name says it; it’s almost myeloma, it has a higher chance of progressing to myeloma – in general, it’s about 10% per year, and usually, the bone marrow has more than 10% plasma cells. Now, you start telling me as a patient, “Well, if my bone marrow is nine percent, I’m MGUS, and if it’s 11%, I’m smoldering myeloma, that doesn’t make sense.” So, it’s correct. In general, those demarcations or numbers are more for us as physicians to talk to each other about what we’re calling rather than the patient themselves. The patient is a continuum.

So, you may move from MGUS to smoldering at a certain point, and it’s not really that extra percentage of bone marrow that moves you into the 10% risk. In general, again, smoldering myeloma, you have a higher chance of going to myeloma. So, I saw a patient recently who’s 30 who has smoldering myeloma. The chances of progressing to myeloma is 10% per year. In five years, you have a 50% chance.

You want to make sure that patient is followed up carefully, and you want to offer, potentially, clinical trials because we want to prevent progression. The hope in the future is you don’t want until you have lytic lesions, fractures in your bones, kidney failure, and then we treat. The hope is we treat you earlier and we can make a huge difference in that early intersection for myeloma.


It sounds like staying engaged with your care team is critical.

Dr. Ghobrial:              

Absolutely, and I would say myeloma is a specialty field. Come and see a myeloma expert, wherever it is, even for a one-time consult, because it’s really complicated and it’s not a common disease, so it’s not something easy for everyone to know what to do with MGUS, what to do with smoldering, what to do with overt myeloma. I relax for the first time. All of these things are important, and just like you go and see the best specialist in anything, I would say care about your myeloma in a very specific way, ask your doctor questions, go online and look it up, and always ask an expert if you want to have a second opinion.


Sure. How about this one? “Myeloma is hereditary.”

Dr. Ghobrial:              

It’s a very good question. So, it’s not hereditary specifically. However, there is a 2x increased risk in family members, and that goes back to that PROMISE study.

We are screening people who have first-degree relatives with myeloma. So, what does it mean? Why do I have a higher risk if I have a family member with myeloma? I recently saw a patient who – the patient had myeloma, the mother had myeloma, and the grandmother had myeloma, and you’re thinking, “Okay, there is something we’re inheriting.”

So, we don’t know. There are some susceptibility genes that we could potentially be inheriting, germ line, and we’ve done something called “germ line,” which means you have it from Mom and Dad, that can increase your risk. It could be other factors come in and we’re still trying to understand all of these factors. What are the genes that can increase your risk? Is there an immune factor that can increase your risk, and can we identify those early in the family members?


What about preventing progression from smoldering? Is there anything patients can do?

Dr. Ghobrial:              

I would say enroll on the PCROWD. Study PCROWD is empowering patients themselves to go online. You can look it up – PCrowd with Dana-Farber – so, precursor crowdsourcing.

This is a study where anyone who has MGUS or smoldering myeloma can tell us about their data – so, their clinical information – tell us about their samples – so, give us their samples whenever they’re going to get their peripheral blood or their bone marrow – and by doing that, we can look at 1,000-3,000 people, put it all together, and hopefully give you very soon the answer of what causes progression, what are the specific markers genomically and immune that can predict progression, and can we target them?

Can we develop therapy for you specifically as a smoldering patient and not use the same drugs as myeloma, but target it for one specific patient for one specific operation?


When patients come into your office, they’re learning a lot of new things. Are there terms that are confusing to patients that you need to define for them?

Dr. Ghobrial:              

Absolutely. I think a lot of those terms are very hard. The words “complete remission” – was that a cure or not? It’s not.

We decrease all of your M spike, we decrease your plasma cells to zero, but it doesn’t mean that we’ve cured you. I think progression is very important. We use certain numbers. A 25% increase in your M spike or a 0.5-gram increase – even monoclonal protein is important to understand, that that’s the antibody that your plasma cells are secreting.

So, absolutely, there are so many words that could be very daunting for any patient to go through all of this. I think having an advocate with you – don’t go on your own because there’s so much information you’re getting that first time. I personally think if patients are recording us or taking notes, that’s perfectly fine because you go back and think about it, and you want to make sure that the information is clear.

So, it’s a lot of information to take in, especially if you’re not in the medical field, and I would encourage patients to ask questions, take notes, think about it a lot.


Tell me what an M spike is.

Dr. Ghobrial:    

So, an M spike – a monoclonal spike – is the protein – the antibodies. So, plasma cells are actually antibody-secreting cells, so they secrete the antibody, it goes in the blood, and when you have a lot of it from the same type of cell, they’re monoclonal, so they’re all the same IgG kappa – IgG kappa because they came all from that same kind of plasma cells.

And, when we run a specific gel, called serum protein electrophoresis, all of those antibodies will run in one area, and they will do a spike instead of going into a bigger area, where we call it polyclonal. So, that tiny little spike, which is a very high level of all of them coming together, we can measure it, and we can say, “Your monoclonal spike is 3 grams per deciliter.” If you don’t have all of them the same type of protein, they will just go around in one big area – big lump, basically, on that electrophoresis, and they will not come out as a spike. So, that’s monoclonal spike. 40:44


And, what are some reliable source of information for myeloma? The world wide web is vast.

Dr. Ghobrial:              

Yeah, and it’s unfortunate. So, there is so much information, and you can get lost, and you can also get misinformation. I think some of the big foundations are very important So, I would say the Multiple Myeloma Research Foundation, the International Myeloma Foundation, the Leukemia and Lymphoma Society, and of course, if you go to clinicaltrials.gov, you will find that information, and you’ll find a lot of the clinical trials. But again, ask your doctor. Ask the experts.


There are a lot of online forums – again, we talked about how vast the internet is. How can a patient identify misinformation online? What are some clues?

Dr. Ghobrial:              

That’s a hard one. I would say again, print it and take it to your doctor. Tell him, “Does that make sense? I’ve read this.” This is where you really need to do your research and go to the sites that you have confidence in so that you’re not lost in the middle of so much misinformation.


Do you have patients come in and say things to you that you just have to say, “Whoa, that’s just not accurate”?

Dr. Ghobrial:              

Yeah, but again, this is part of the discussion. I personally think every question is a good question. Even if it sounds completely ridiculous, ask it. That’s why we’re here. We’re here to tell you, “This is right, this is wrong, this one I don’t know, I’m not so sure,” and that’s okay. It’s part of the discussion.


Before we finish up, let’s get your take on the future of myeloma. What are you seeing on the horizon?

Dr. Ghobrial:              

Oh, a lot, and I hope I live long enough to see all of the amazing things. I truly think that we will cure myeloma. I think we should treat patients early. That’s an absolute change.

I think immunotherapy is coming in, CAR-T, bispecific antibodies. We will harness our immune system to kill myeloma, and I think there’s so much to be done there. I think precision medicine is very important. The first study is from MMRF [Multiple Myeloma Research Foundation] coming out now, genotyping, asking the questions “Which mutations do you have?”, and then putting them into different buckets so you can understand which disease should be treated with which drug.

We always say we know there is different subtypes of myeloma, then we treat you the same way, so let’s stop doing that, let’s do precision medicine, let’s individualize treatment specifically for you. So, I think that’s another big thing. So, in the future, there will be so many options. The hope is truly we’ll cure myeloma, we diagnose it early, we screen for it, we diagnose it early, and we prevent it from even causing one lytic lesion for a patient. 41:52


Dr. Ghobrial, let’s end by talking about why you’re so hopeful about the future of myeloma.

Dr. Ghobrial:              

Well, again, I trained – and, I said that 15 years ago – at Mayo Clinic, where we only had few drugs, when the survival of myeloma was three to five years, when we saw patients having severe fractures and severe pain, and now, we look at it, and it’s only 15 years in our lifetime, and we look at it that myeloma is a completely different disease.

We can diagnose it early – in fact, we’re thinking of screening them early – we can make a huge difference in all of the comorbidities, but the most important thing is we have so many amazing drugs that we’re using together to get an amazing, complete remission, MRD-negative disease, and then, in the next 5-10 years, I think we will change, again, immunotherapy with CAR-T. We will have precision medicine and immunotherapy to completely change how we treat myeloma. So, I am extremely hopeful and extremely excited for our patients.


So, how do you talk to your patients about this hope? I would imagine that when they come in, they’re pretty terrified about what’s going on.

Dr. Ghobrial:              

Absolutely. Again, the first thing is you want to say, “Yes, you have a cancer,” and that shocks you. That is a big thing. It makes a big difference in a patient. “I have cancer now” is an important part that you have to acknowledge.

And then, you go to the next step, and now, let’s talk about treatment. Let’s talk about survival. Let’s not say, “I will not see my kids grow up.” These are not things – again, we cannot predict. We’re not gonna play God, and we can never predict if someone will respond or not, but we know from the data that we have so far that we have amazing remissions and long-term survivors. I have many of my patients that I transplanted 15 years ago still alive, doing well. Again, I cannot say that myeloma is cured, but we have a good remission rate currently.


Dr. Ghobrial, thank you so much for taking the time today.

Dr. Ghobrial:              

Absolutely. Thank you.


And, thanks to our partners. To learn more about myeloma and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Patricia Murphy.

Could an MPN Clinical Trial Be Right for You?

Could an MPN Clinical Trial Be Right for You? from Patient Empowerment Network on Vimeo.

Is a clinical trial your best MPN treatment option? Dr. Ruben Mesa explains the clinical trial process and how patients may benefit from participating.

Dr. Ruben Mesa is an international expert in the research and care of patients with myeloproliferative neoplasms (MPNs). He serves as director of UT Health San Antonio MD Anderson Cancer Center in San Antonio, Texas. More about this expert here.

See More From the The Path to MPN Empowerment

Related Programs:

Diagnosed with an MPN? Why You Should Consider a Second Opinion

Could an MPN Clinical Trial Be Your Best Treatment Option?

Improving Life with MPNs: The Latest Research and How to Get Involved


Dr. Ruben Mesa:

There is much exciting research in myeloproliferative neoplasms. First, research trying to understand, why do people develop MPNs, and why do they progress. This is crucial research, and that this basic research to better understand the diseases will help us asses whether our treatments are having an impact slowing down the progression of the disease, and help us better design therapies that, hopefully, can cure these diseases.

Be reassured  that our goal as a scientific community is to cure the MPNs. Now, until we’re able to do that, we want to be able to best control them as best we can. So, the next level of research is really in new therapies; primarily drug-based therapies, but future therapies using the immune system; potentially using vaccine therapy to try to better control the disease to make the disease as neutral in your life as possible.

Our goal, short of curing the disease is to make the disease as invisible in your life as possible. Hopefully, minimal side effects, minimal symptoms, protected against risk of blood clots or bleeding, ideally, decreasing the risk of progression, and hopefully without any significant side effects from the medication your receiving.

So, that really is our goal.

 Clinical trials are a crucial way for us to improve the treatments that we have for any diseases. And in particular, in areas like myeloproliferative neoplasms where we have therapies, but we don’t have cures, clinical trials are crucial. Clinical trials are a structured way for you to be able to receive a new treatment. That treatment is closely monitored, and starts with a strong belief that that treatment is going to be beneficial for you.

Being on a clinical trial has many steps, but you are in the driver seat in each of them. So, you’re able to enroll in a study, and you’re able to decide at any point whether or not you’d like to continue on in that study. You are made clearly aware of what you’re receiving; what dose; what to expect at each and every step of that therapy.

It’s a treatment just like any other, but we use them because we are hoping that it will be better than the treatments that we have, and we do it on a clinical trial so that we can learn from that experience. If that drug is better, then we should probably expand its use and give it to other people, and have it be approved and used around the world. Or for whatever reason that therapy is not as helpful as we would like, then we learn from that, as well.

Why was it not helpful? Was it the wrong therapy? Was it targeting the wrong aspect of the disease? Were there side effects that made the therapy not beneficial? So, we learn a lot about it in either direction. Hopefully, individuals who participate in clinical trials will have a direct benefit themselves by being able to experience a new therapy that is, hopefully, better. But also, they do have the ability to help other patients now and in the future that will be facing the same disease they have.

MPN Treatment Decisions: Which Path is Best for You?

MPN Treatment Decisions: Which Path is Best for You? from Patient Empowerment Network on Vimeo.

Dr. Ruben Mesa provides an overview of available treatments and reviews important factors to consider when choosing a therapy for essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF).

Dr. Ruben Mesa is an international expert in the research and care of patients with myeloproliferative neoplasms (MPNs). He serves as director of UT Health San Antonio MD Anderson Cancer Center in San Antonio, Texas. More about this expert here.

See More From the The Path to MPN Empowerment

Related Programs:

Essential Lab Tests for MPNs

Ready to Start an MPN Treatment? What You Need to Consider

An Expert Summary of Current MPN Treatment Options


Dr. Ruben Mesa:

The treatment landscape for myeloproliferative neoplasms is changing very rapidly. And in a good way, it’s increasingly having many more options for patients with Myeloproliferative Neoplasms. But I would separate it, really, into two groups. First, there are those individuals with essential thrombocythemia and polycythemia vera.

These individuals, we have newer therapies, such as interferons, we have, potentially, use of JAK inhibitors, we have some experimental therapies, as well as prior therapies we’ve used and become accustomed to, including hydroxyurea, phlebotomy, and aspirin.

But we’re learning much more about how to use these therapies; how to combine them; what constitutes success with these therapies; what should constitute a change in terms of therapy.

And there are new therapies being developed in the future that will impact this group of individuals with earlier MPNs: ET and PV.

For patients with myelofibrosis, the treatment is evolving. Patients with Myelofibrosis are affected in different ways. It is, in some ways, a more problematic disease.

There is evolution of our most impactful therapy, of stem cell transplantation. We have a better sense of in which patients we should consider that treatment, and how that can be applied in the safest way. We also have more medical treatments. We just saw in 2019 the approval of Fedratinib as the second specific JAK inhibitor approved for patients with myelofibrosis.

We, additionally, now have, truly dozens of clinical trials of new therapies in development that are in clinical trials right now that might be helpful for patients with myelofibrosis who have either had Ruxolitinib, or have a suboptimal response to Ruxolitinib, or sometimes even newly diagnosed patients. But I would say the future is very bright.

So, it is key with a treatment to first understand what is the treatment, what is the dose, and what is the goal? Each of the treatments have different goals. Some of the goals are to decrease the likelihood of blood clots or bleeding.

And frequently, we assess whether we’re protecting against the blood clots or bleeding by bringing down elevated counts. Is the plate account high, and we’re trying to bring it into the normal range? Is the hematocrit high, and we’re trying to bring that to under 45%? Is the white blood cell count high? Have we lowered each of those? First, it’s around controlling blood counts if that is the goal, as well as trying to decrease at risk of blood clots or bleeding.

 Second, if patients have symptoms associated with their MPN, sometimes itching, sometimes symptoms associated with high courts, sometimes enlargement of the spleen, or symptoms associated with the spleen, have we reduced or nullified those symptoms? Have we shrunk the spleen if the spleen was enlarged?

And then, finally, we assess our goal by trying to be sure that patients are not progressing or getting worse on the disease. So, depending upon the treatment, we first asses what is our goal? Is it to improve counts? Is it to improve symptoms? Is it to shrink the spleen? And have we accomplished one, two, or all three of those goals? Or was only one those our goals to begin with? 

Could an MPN Clinical Trial Be Your Best Treatment Option?

Could an MPN Clinical Trial Be Your Best Treatment Option? from Patient Empowerment Network on Vimeo.

Lindsey Lyle discusses the role of clinical trials as an MPN treatment option and how research is advancing the field.

Lindsey Lyle is a physician assistant at the University of Colorado Cancer Center, specializing in hematological malignancies with a subspecialty in myeloproliferative neoplasms (MPNs). More about this expert here.

See More From the The Path to MPN Empowerment

Related Programs:

Diagnosed With an MPN? Why You Should Consider a Second Opinion.

Improving Life with MPNs: The Latest Research and How to Get Involved

An Expert Summary of Current MPN Treatment Options



When considering treatment, remembering that clinical trials are an option – and often, a very good choice – is something that I really try to communicate to my patients. Generally, there’s a stigma around clinical trials as patients feeling like a lab rat or some sort of a study subject, and there is a perception that they’re not receiving as good of care as they would if they were not on a clinical trial. However, in my patients, I really try to dismiss this thinking because at this point in time, we do have really fairly good options for treatments with MPNs.

However, we do not have a cure for MPNs outside of a transplant, and our treatments are not perfect, and so, enrolling in a clinical trial really should be considered by patients and their providers as a very viable option.

It’s generally introducing perhaps a new way of approaching the disease treatment. Oftentimes, clinical trials are using a combination of agents, which is not necessarily available outside of the clinical trial.

And so, with clinical trials, we’re always trying to make something better. We’re trying to learn something, we’re trying to, No. 1, help the patient – so, my No. 1 goal in enrolling one of my patients in a clinical trial is to, first of all, help them, help control their disease, help them to feel better, and to live a long and good life. No. 2, we learn as we go along. Clinical trials are critical for drug development and for the future of treatment in patients with MPNs.

So, not only are our patients helping themselves, but hopefully, they are helping the future MPN patients who may come along and need a treatment. So, I always like to keep this really in the conversation when discussing treatments, and it may be up front, and it also may be after a treatment has stopped working that we consider a clinical trial.

So, there are also a lot of things that kind of go into clinical trial management and different requirements, so if a patient lives very far away, it may be challenging for them to come back to the academic center on a regular basis for routine clinical trial monitoring that’s required by the study, but if they live close by, I generally do recommend this. They are also associated with clinical research coordinators or clinical trial nurses.

And, these patients are monitored really very closely, and it’s kind of nice to have that extra person in it with you in the clinical trial, just another point person to discuss, perhaps, how you’re feeling or different questions or concerns as the clinical trial proceeds. So, when talking about treatments, in my opinion, especially in MPNs, clinical trials really should be one of the options that is first discussed when thinking about starting treatment, and especially if a treatment has stopped working.

So, there are very many exciting possibilities in MPN research right now. We have a lot of combination therapies, which I think I am most excited about, because we have a decent backbone of therapy at this point, but building on that and trying to maybe enhance the way that the backbone therapy works, and also to perhaps change the microenvironment of the bone marrow – basically, trying to reverse fibrosis.

So, there is currently a drug in clinical trial that is looking at this, and we are proceeding with this trial, and really hoping for the best, but I think that to combination therapies where we can put two things together that we think work really well together to help produce good outcomes – I think I’m most excited about that at this point.

Ready to Start an MPN Treatment? What You Need to Consider.

Ready to Start an MPN Treatment? What You Need to Consider. from Patient Empowerment Network on Vimeo.

Lindsey Lyle discusses the factors that should be considered when choosing a therapy.

Lindsey Lyle is a physician assistant at the University of Colorado Cancer Center, specializing in hematological malignancies with a subspecialty in myeloproliferative neoplasms (MPNs). More about this expert here.

See More From the The Path to MPN Empowerment

Related Programs:

Diagnosed With an MPN? Why You Should Consider a Second Opinion.

Essential Lab Tests for Myeloproliferative Neoplasm (MPN) Patients

An Expert Summary of Current MPN Treatment Options



When deciding about a treatment, it’s really important for the healthcare professional and the patient to discuss the patient’s goals.

The patient really is the key player here, and we as medical professionals are here to support the patient’s goals. So, what might work for one patient is not going to be necessarily the same treatment I would choose for a different patient. So, right off the bat, identifying the patient’s goals – and really, what are we trying to fix in one specific patient is going to look different from the next patient I see in that day.

For example, there are certain clinical manifestations of MPNs that need specific treatment approaches and maybe honing in on trying to help one clinical issue.

So, first of all, identifying the disease process – that’s No. 1. What is the diagnosis? No. 2: Coming up with a goals of care plan with the patient. What is causing them the most difficulty in their everyday life, and how are we going to fix that? That’s generally where I start.

Then, I discuss with the patients the different options for treatment, which either include therapies that are FDA-approved or enrolling in a clinical trial. And then, we really talk about pluses and minuses for each of these therapeutic decisions.

Patients may have different comorbidities, so they may suffer from different chronic diseases that may impact the treatment that is chosen with the patient and their provider, as well as discussing stem cell transplant, which we haven’t talked much about, but stem cell transplant is an option, and at this point, the only curative therapy for patients with myelofibrosis. And so, determining whether or not transplant is in the patient’s best interest is also a topic of discussion when deciding on therapy approach.

Clinical Trial Toolkit

Reinventing the Clinical Trial: Start at Ground Level

If each of us humans is a snowflake, unique in our genomic makeup, where’s my snowflake medicine? I asked that question from the platform at the ePharma Summit in New York in 2013, and have yet to get an answer. The challenge for the bioscience industry is, I believe, the classic randomized clinical trial. That design goes through four phases:

  • Phase 1: a small group of people are given the drug under study evaluate its safety, determine a safe dosage range, and identify side effects
  • Phase 2: a larger group is given the drug to evaluate its efficacy and safety in a larger population
  • Phase 3: large groups – plural – of people are given the drug to confirm its effectiveness, monitor side effects, compare it to other commonly-used treatments, and collect information that will allow the drug /treatment to be used safely
  • Phase 4: the drug is marketed while study continues to assess long-term effects and efficacy

Of course, before they even get to Phase 1, there have to be both the idea for the new treatment, and animal studies to determine what the substance or compound under study might do to a mouse or a monkey.

Science isn’t easy. The phrase “trial and error” came out of science labs, with many trials running up against the error wall by Phase 2. Since bioscience companies can sink about $1 billion-with-a-B into getting just one drug to market, it seems that the traditional clinical trial has turned into a pathway to NOT making scientific discoveries that can benefit humankind.

Then there’s the whole “who’s in charge here?” question. Clinical trials are now a global effort, with US and European pharma companies testing new treatments in Latin America, Russia, and China to gain traction in those emerging markets while simultaneously developing me-too drugs for their domestic markets. So, who’s in charge, the US Food and Drug Administration (FDA)? The European Medicines Agency (EMEA)? A player to be named later? The answer to the question seems to be “all of the above,” which adds to the complexity of the clinical trial process.

As digital technology has made data easier to collect and share, it would seem that clinical trials would be a great place to start intersecting with the quantified-self movement. The shift to electronic health records, the widening adoption of all sorts of health tracking devices, and the rise of (relatively) cheap genomic sequencing should signal an ability to identify conditions, and populations, eager to participate in clinical investigations. But so far, it hasn’t.

What might challenge that stasis? In November 2013, three major pharma companies – Novartis, Pfizer, and Eli Lilly – announced via the White House’s website that they had joined together in a clinical open innovation effort. That page on the White House’s site is gone now – changes in Presidential administrations will do that – but here’s a direct quote from that announcement:

“In order to connect patients and researchers, Novartis, Pfizer and Eli Lilly and Company, are partnering in the U.S. to provide a new platform to improve access to information about clinical trials. The platform will enhance clinicaltrials.gov and will provide more detailed and patient-friendly information about the trials, including a machine readable ‘target health profile’ to improve the ability of healthcare software to match individual health profiles to applicable clinical trials. As part of the project, patients can search for trials using their own Blue Button data.”

Five years later, and we’re still stuck on the slow train when it comes to really reinventing the clinical trial.

I’m one of a growing group of people who think that the entire life-sciences process chain needs to be re-tooled for the 21st century. In my view, the best place to start that re-tool is at ground level, with the patients and clinicians who deal with challenging medical conditions daily. If a doctor has a number of patients who might benefit from some clinical study, why isn’t there an easy way to find a researcher looking into that condition? If a patient has an idea for a clinical investigation into his or her illness or condition, why can’t they find a researcher who’s interested in the same condition to team up and start a science project?

I can only hope that the regulatory agencies involved in life science oversight (hello, FDA!) can move beyond the aftermath of Thalidomide – for which epic disaster we’re still paying a price when it comes to the timeline for drug approval in the US – and toward a process of “all deliberate speed” that doesn’t forsake speed for deliberation. Both are necessary, neither should be more heavily weighted than the other.

We all can, and should, take part in scientific exploration into human life, and human health. Got an idea for a clinical trial? Share that idea in the patient communities you hang out in, and ask your tribe to help you bring that trial to life. To quote Arthur Ashe, “Start where you are. Use what you have. Do what you can.”

We’ve got to start somewhere, right?

Reaping the Benefits of Patient-Centered Clinical Research

This blog post was originally published on Trial Mix, Science 37’s blog, and is reposted here with permission. Learn more about Science 37’s approach to clinical research or view the original post here.

Patient-centered research studies — which reduce the burden of participation — allow patients to reap the benefits by working smarter not harder.

As a scientist, I see the value in clinical research, but my time spent as a cancer patient showed me that priorities realign in times of health crisis. I cared a little less about the scientific value of what I was doing, and more about taking care of myself and spending time with my family. I know that clinical research studies do require extra effort from the participants, but patient-centered studies — those that are designed with the patient in mind — can let the patient work smarter, not harder, while still reaping the benefits.

Clinical Research Requires Some Extra Effort

I have lots of wonderful friends, many of whom have participated in clinical research studies, so I asked them to help me understand some of the hardest parts of their experiences. In addition to hearing about the experiences my friends had as adults, I also got some insight from a friend who’d been in a clinical trial as a child, and another whose son is in a food allergy research study now, giving me little peek at how clinical research impacts kids. Aside from the annoying paperwork like consent forms and surveys, here are the top three hurdles they mentioned:

  • Time: It’s precious and finite, so all those minutes and hours spent in transit, in waiting rooms, and in exam rooms prevent clinical research participants from going about their normal routine.
  • Money: While there should be no additional out-of-pocket cost for medical care to participate in clinical research, there is still a potential financial cost. Several friends talked about the need to pay for parking, miss work, or find childcare for each additional trip.
  • Extra procedures: Shots, additional medications, or daily trips to the nurse for kids who take study drugs during the school day complicate a patient’s routine, and extra scans and invasive examinations really take a toll on a person. Beyond that, of course, no one wants to go through the pain of extra biopsies or the anxiety that comes with additional scans.

Patient-Centered Study Design Can Ease the Burdens of Participation

A more patient-centered study design could remove or reduce some of these hurdles. For example, providing extra assistance like patient navigation to connect patients with resources to provide transportation, childcare, or emotional support could reduce the burden. But since closer monitoring helps the investigators learn more from the research, many of these hurdles can’t be completely eliminated.

More Than Altruism: Clinical Research Benefits Participants

While some people look at all those barriers and decide (totally justifiably) that in their circumstances, the burdens of participation are too much for their family, many of the people I spoke with who had taken part in clinical research told me the extra work was so very worth it.

One friend wrote: “Facing that needle every week and working up the nerve to jab it deep into my thigh was the hard part for me…I still wouldn’t trade the experience for anything. That study gave me my life back.”

From another: “(There were) ups and downs, but I’d do them again. I’ve had substantial shrinkage of my tumor.”

A third shared: “The extra biopsies were painful and scary. But I felt like the doctors were watching me extra so it was worth it.”

My friends could see that not only did they contribute to some particularly awesome science, but each of them were able to reap benefits in their own lifetime — either because a pill could replace the need for a weekly self-injection, the drugs actually slowed their disease, or they knew they were being monitored so much more closely than a patient under standard care.

Working Smarter, Not Harder Improves the Patient Experience

While I’m quick to tell you that the increased monitoring a patient receives from all those doctor visits, blood draws, and surveys is vital to the clinical research process, it would be disingenuous to imply that the system can’t be improved.

Investigators need to design each study to obtain the most valuable data and draw valid conclusions. By involving patients in the design process to make the studies truly patient-centric, the research protocols can be written to ensure each study respects the patient experience and reduces the negative impact on a patient’s life. The Science 37 team partners closely with patients and investigators to ensure that trials are designed thoughtfully to minimize the impact on patients and their families, making it easier for them to benefit from awesome science.

We know it’s worth it to participate in clinical research, but we still want to make it less work for the patients involved.

About the Author

Jamie Holloway is a both a scientist and a survivor, earning her PhD in tumor biology from Georgetown University — where she spent long hours researching breast cancer — a few years before her own breast cancer diagnosis. Now living with no evidence of disease after treatment for early stage triple negative breast cancer, she bridges the gap between scientists and researchers as a Clinical Research Advocate for Science 37, and as the Patient Advocate for the Metastatic Breast Cancer Project at the Broad Institute. She works with researchers as part of the Georgetown Breast Cancer Advocates and writes about her personal experience with cancer on her blog Run Lipstick Chemo, and as a contributor to the Cure Magazine community. A wife, mother, runner, and lipstick addict, Jamie shares her story from the perspective of both a patient and a scientist.

Patient Profile: Elizabeth Carswell

She was 25 weeks pregnant and felt like she had the flu. It was early July 2015 and she made her way to her obstetrician’s office because the antibiotics she’d taken over the Fourth of July holiday hadn’t made her feel better. A couple hours later Elizabeth Carswell was in the hospital being diagnosed with Acute Myeloid Leukemia. She was 33 years old.

“Obviously, the scariest part was the first two months when I was still pregnant,” says Carswell, known as Liz to her friends. She was immediately transferred to Jackson Memorial Hospital in Miami and began chemotherapy. “It was a very tough decision to be treated, but I wanted to meet my baby.”

The first two rounds of chemo were unsuccessful and her doctors wanted her to undergo a more aggressive treatment. That meant it was time to deliver the baby. Liz was 32 weeks along and the surgery was extremely risky. Her blood counts and platelets were considered too low for surgery, but despite it all, baby Hudson was born weighing in at 4 pounds. Liz continued her treatment in the hospital while Hudson was admitted to the NICU for five weeks.

When Hudson was released from the hospital, Liz was not. Her boyfriend, Hudson’s father, Frankie Lightfoot had to return home to work, so Liz’s father and her sister moved to Miami, rented an apartment near the hospital and cared for the baby. Liz was never alone. Her father and sister alternated caring for the baby and spending the night in the hospital with her. Her friends showered her with gifts for the baby and travelled to her bedside to decorate her hospital room. Meanwhile, her doctors were trying to ready Liz for a bone marrow transplant, but could never get her levels where they needed to be and eventually her Jackson Memorial team told her it was time to go home and spend whatever time she had left with her baby.

That’s when a friend connected Liz with the Leukemia and Lymphoma Society which led her to a clinical trial at the City of Hope Cancer Research Hospital in Duarte, California under the care of Stephen J. Forman, M.D., F.A.C.P. She went the next day. The trial involved very aggressive full-body radiation and a stem cell transplant. Her brother, a perfect 10 match, was her donor.

The transplant put her into remission. It was November 2015. Then, in August of 2016, she found some lumps in her breast. The leukemia had returned in mass form. More radiation and seven more rounds of chemo later she is technically in remission. But, due to a gene mutation that makes her highly likely to relapse, Liz continues with regular rounds of chemo for maintenance.

“My doctor expects recurrence,” she says. That’s why she will undergo another stem cell transplant within the next couple months. This time, from an unrelated donor so that a new immune system can be introduced. She will spend another six weeks in the hospital, but she believes this is what she has to do. “I know it’s needed,” she said. “I trust it’s needed. I trust my doctor. He’s pretty amazing.”

Liz is undaunted by what lies ahead. “There are lots of ups and downs, but a positive attitude is a must if you’re going to survive,” she says. Though she credits Hudson, now two, as her main motivation. Early on she felt it was Hudson growing inside her who was keeping her alive and now being his mother keeps her going. “I’m not scared to go through it again,” she says of her upcoming transplant. “I’m excited to do it again. I’ll do anything that will give me a chance to watch Hudson grow up.”

Liz has kept a record of her journey on her Facebook page, Prayers for Elizabeth. She continues to keep her page updated and in addition to helping her, she hopes that sharing her story can help others. You can keep up with Liz here.

Comparing Cancer Treatment to Bug Control

When farmers try to manage insects in their crops, they figure out what dose of pesticide yields the best results. They do this based on understanding which bugs are the most harmful to their crops and what level of poison best combats them. This analogy may send shivers up the spines of organic eaters out there, but it does have relevance to the way chemotherapy is used in treating cancer.Amy Gray

It turns out that not all cancer cells (i.e. insects in the crops) are equally harmful within a tumor.   Some are much more aggressive. When a broad-based chemo is used to kill all the cancer cells, it often leaves behind the most resistant ones, allowing them an opportunity to take over the tumor.

A quick look at evolution explains this phenomenon. Dr. Robert Gatenby, chair of radiology and co-director of the cancer biology and evolution program at Moffitt Cancer Center and Research Institute, says “We tend to think of cancers as a competition between the tumor and the host, but at the level of the cancer cell, cancer cells are mostly competing with each other,” he says. Competition within a tumor reflects a basic evolutionary principle, one which cancer cells utilize as well as normal cells.

Given this theory, Gatenby and his team wondered about the perfect level of chemotherapy that would repress the more aggressive cells, rather than encourage them to overtake the less aggressive cells. In the experiment, they took cells from two different breast cancers and grew them in mice. The mice were then treated to two separate chemo protocols. Group One mice were given lower-dose chemo and then skipped sessions if their tumors shrunk. Group Two mice were given continuous but gradually lower doses of chemo.

The researchers were surprised that 80% of the Group Two mice showed better response in reducing cancer growth. Some of the tumors disappeared completely.

This research is in its early stages and requires quite a bit of personalized chemotherapy analysis. One fly in the ointment – patients have to overcome the mindset that cancer treatment kills all cancer cells. They have to be content with the knowledge that not all cancer cells need to leave the body in order for a treatment to be successful.

In other words, leave the ladybugs and kill the locusts.





Barriers to Clinical Trial Accrual

Interview With Dr. Michael Thompson (@MTMDPhD), Medical Director, Early Phase Cancer Research Program, University of Wisconsin

9 1/2-year CLL patient Carol Preston interviews Dr. Michael Thompson about some of the barriers to clinical trial accrual and how healthcare professionals might overcome them. Today, there is only about 3-5% of adult patients enrolled in clinical trials versus the 60% in pediatrics. Why is that? Watch the video below to find out.

Barriers to Clinical Trial Accrual from Patient Empowerment Network on Vimeo.

The Best of 2015

best of 2015


As 2015 comes to an end, we would like to take a moment to highlight a few of our most popular posts and to thank the people who contributed to the popularity of these posts. We cannot thank the authors enough that have contributed, such as Marie Ennis-O’Connor, Tori Tomalia, Cindy Chmielewski, Dr. Michael Thompson, Edward Leigh, and Deana Hendrickson. Your efforts to Patient Empowerment Network are greatly appreciated.

best of people

From right to left: Edward Leigh, Tori Tomalia, Cindy Chmielewski, Marie Ennis-O’Connor, and Dr. Michael Thompson

PEN would also like to thank our sponsor AstraZeneca and our partner LUNGevity for their support in creating these posts.logos


Most Popular Posts of 2015

1.Virtual Patient Communities

Virtual patient communities offer support and information to cancer patients.

2. The Stigma of Lung Cancer

Tori Tomalia, a lung cancer patient explains the stigma of lung cancer, and that it doesn’t happen to only those who smoke.

3. Can Digital Wearables Help in Clinical Trials?

When applied to clinical trials, wearable technology is a potentially powerful research tool to gather clinical data in real-time and provides remote patient monitoring.

4. Clinical Trials 2.0: Reinventing Research For the Social Age

Clinical research is changing. No longer the sole preserve of clinicians and researchers, the Internet and new digital technologies are reinventing the way in which patients take part in the clinical trials process.

5. Cindy Chmielewski Talks About Clinical Trials

Carol Preston talks to myeloma patient Cindy Chmielewski about her role in advocating for clinical trials. Cindy explains how her cancer journey took her to self-advocacy and advocating for others in her community through social media.

6. Why So Few Adults in Clinical Trials

Dr. Michael Thompson discusses with Carol Preston the reasons behind some patients apprehensiveness towards clinical trials.

7. Spotlight on: LUNGevity – The Meaning of Lung Cancer Awareness Month

What can you do in support of Lung Cancer Awareness Month

8. Activating a Community of People Living With Lung Cancer

LVNG With is a community for people living with a lung cancer diagnosis. These people share their story and help inspire others like them.

9. How to Develop a Personal Medical Resume

Edward Leigh, Founder and Director of The Center for Healthcare Communication, shares his tips for creating a “medical résumé” to ensure great healthcare experiences.

10. Connecting to Cancer Patients Online

In the Empowering Lung Cancer Patients Town Meeting, Deana Hendrickson talks about how important it is to connect with other patients and advocates online.


Also, be sure to check out

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The Clinical Trial Toolkit is a resource to help you find clinical trials and financial assistance. You can view our Patients Helping Patients Blog for various aspects and perspectives of clinical trials. Also, don’t miss our Videos of patients, caregivers, and healthcare professionals!


Improving Clinical Trial Design

Interview With Dr. Michael Thompson (@MTMDPhD), Medical Director, Early Phase Cancer Research Program, University of Wisconsin

Carol Preston’s interview with Dr. Michael Thompson discusses issues related to clinical trial design. The obstacles to good patient-centric clinical trial design are numerous. The informed consent is a text-only legal-type document containing difficult to understand verbiage. Instead, it should be more patient-friendly, with images, graphics and easy-to-understand instructions.

Patients are becoming more empowered and more aware of the issues and are speaking out and willing to participate to make clinical trial design more patient-friendly and effective. More patients are aware of trials and want to educate and inform others. Clinical trial design and informed consent need to be updated and made more efficient so that more patients will be able to easily consider clinical trial enrollment.

Check out the full conversation and the rest of Dr. Thompson’s ideas in the video below:

Improving Clinical Trial Design from Patient Empowerment Network on Vimeo.

How Do I Enroll in a Clinical Trial?

Oftentimes when we hear the word “cancer,” we hear nothing else. Our brains stop processing information. We think we’re going to die, that there is little or no time to weigh options and/or get our affairs in order.

Fortunately, that last part the vast majority of the time isn’t true. We DON’T have to rush into anything. We DO have time to weigh our treatment options. And for many patients, those options can include enrolling in a clinical trial.

What is a clinical trial?

According to PAF, the Patient Advocacy Foundation, clinical trials are research studies in which we patients can help doctors find ways to improve cancer care.

There are several types of trials

  • Treatment: to test a new drug or approach to surgery
  • Prevention: to test new approaches with medicine, vitamins, minerals to help lower the risk of developing certain cancers
  • Screening: Best ways to detect cancer early
  • Quality of Life: Explore ways to comfort, quality of life for patients

Why participate in a clinical trial?

Through clinical trials, doctors and researchers find better ways to prevent, diagnose and treat cancer. Patients can benefit by receiving cutting-edge care or emerging medications. Rarely are placebos, or fake medications, used in clinical trials. Patients for the most part are being given the current standard of care or the new treatment, to determine the following:

  • Phase   I: Is the new treatment safe?
  • Phase II: Does the new treatment work?
  • Phase III: Does the new treatment work better than the current one?

Is a clinical trial right for me?

There are risks and benefits. A good place to start making an assessment is with your physician. Often, we’ll hear about a clinical trial from our doctor. However, you don’t have to have your doc’s OK to enroll in a trial, and it’s key to determine which trial is right for you.

While there is no one source to learn about all cancer clinical trials, you can break it down into clinical trial lists and matching services.


The National Cancer Institute (NCI): 1-800-4-CANCER (422-6237 NCI sponsors most government-funded trials. You can search by the type and stage of cancer, by the type of study (treatment or prevention) or by zip code.

The National Institutes of Health (NIH):   NIH database is larger than NCI but not all trials are cancer studies

CenterWatchCenterWatch provides a list both of industry-sponsored and government-funded clinical trials for cancer and other diseases.

Private companies: Pharma or biotech companies may list studies they are sponsoring, either on their websites or through a toll-free number. You can search a company with the words ‘clinical trials’ in the search. If that company is conducting trials, it will appear in your search. For example:




You get the idea.

As for those clinical trial matching services, each one works differently. Some may charge a fee to the trial sponsor. It could impact the way studies are ranked or presented to you. We suggest you start with the free sites. Sources include:

The American Cancer Society Clinical Trials Matching Service:    1-800-303-5691  ASC works with a company called Eviti  to connect patients with trials. It’s free. It’s confidential. It’s reliable. The lists are updated daily, and it allows patients to contact health care providers running the studies. The website also explains how to determine if you are eligible for a trial, if a trial is right for you.

EmergingMed:  1-877—601-8601  Also free, confidential and reliable for cancer patients seeking a trial.

Various organizations have partnered with EmergingMed and offer widgets that link to the EmergingMed trial finder. For instance, both the sites below link the viewer to the EmergingMed clinical trial finder:



There is a wealth of information here. But you don’t have to become a Medical Doctor to digest and evaluate it. Start with your physician. If he or she doesn’t have enough information, choose a website or two on this list to navigate for information about trials. That’s what I’ll be doing should the day come that I need additional treatment. We are our best advocates about what’s right for us and when.

(We want to help you if you are considering a clinical trial. Please look at our FAQ and Clinical Trial Toolkit pages and browse our Patients Helping Patients blog for articles on clinical trials)

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