AML Treatments and Clinical Trials

When it comes to treatment, AML patients and their care partners have much to consider. There are often many options available, each with advantages and disadvantages. Some people may seek clinical trials, others may have few feasible options. Understanding treatment options, goals, and what to expect are vital to achieving the best possible outcome for you.

More resources for AML Treatments and Clinical Trials from Patient Empowerment Network.

Understanding and Managing AML Treatment Side Effects

Understanding and Managing AML Treatment Side Effects from Patient Empowerment Network on Vimeo.

AML expert, Dr. Jessica Altman, discusses how AML affects the body, and the common side effects patients may experience during varying AML treatment phases.  

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

See More From The Fact or Fiction? AML Series


Related Resources

AML Genetic Testing Explained

What is Targeted AML Therapy?

AML Treatment and Side Effects Program Resource Guide


Transcript:

Patricia:            

Dr. Altman, let’s talk about some common AML treatment side effects. What are some of the things that patients can expect when they begin treatment?

Dr. Altman: 

So, the side effects depend in part on the actual treatment strategy that’s utilized. It’s also important to note that AML itself has symptoms, and so sometimes it’s hard to separate out the symptoms of the Acute Myeloid Leukemia and the symptoms from the treatment. Acute Myeloid Leukemia is a disease where the bone marrow is not functioning normally. The bone marrow is responsible for making healthy red blood cells, healthy white blood cells, healthy platelets, and also is very intimately involved with the immune system. 

And so, patients with Acute Myeloid Leukemia by itself without treatment are at risk for fatigue if the hemoglobin is low, bleeding and bruising when the platelet count is low, and at risk for infections. 

Also, shortness of breath and other side effects from having abnormal blood counts. In addition, the treatment frequently lowers the blood counts further, and the treatment itself increases those risks associated with low blood counts. Patients can be supported with blood transfusions. Patients are also supported with antimicrobial therapy to prevent infections, and if fever or infections occur despite that, patients receive additional antimicrobial therapy based on what the perceived organism is. 

Patients with Acute Myeloid Leukemia, when they receive chemotherapy, are also sometimes at risk for something called tumor lysis syndrome. 

That’s when we kill the leukemia cells, when the leukemia cells are killed quickly, sometimes the contents of the leukemia cells can inflame the kidneys and lead to alterations in the electrolytes and the acids and salts in the body, and that’s something that needs to be monitored for and prevented. 

Patients with Acute Myeloid Leukemia who receive chemotherapy are also at risk for organ inflammation, and that is something that is monitored with the blood counts.

Patricia:     

What can patients or their caregivers suggest to help manage some of these side effects?

Dr. Altman:    

So, I think the biggest side effect that might be the hardest for us to manage and for patients to manage is fatigue. And I’m a believer that energy begets energy, and so trying to be as active as one can throughout all phases of their treatment I think helps the most. And also, the hopeful recognition that the fatigue should be self-limited, and that with time away from treatment, the energy should improve.

I think that’s one of the biggest things I hear from my patients.

AML Treatment Side Effects: What’s Fact and What’s Fiction?

AML Treatment Side Effects: What’s Fact and What’s Fiction? from Patient Empowerment Network on Vimeo.

AML expert, Dr. Jessica Altman, addresses AML treatment side effects, such as nausea and changes in taste, in addition to discussing best practices for researching AML online.  

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

See More From The Fact or Fiction? AML Series


Related Resources

   

Can AML Be Cured?

   

Managing AML Symptoms

   

AML Treatment and Side Effects Program Resource Guide


Transcript:

Patricia:          

All right, a little more fact and fiction now. Here’s what we hear from AML patients about treatment side effects. Tell me if this is true or not. “Treatment side effects are unavoidable.”

Dr. Altman:          

I think it’s probably true, but I don’t think it’s completely true. So, I think they’re a long ways away from being in that Hollywood picture of someone with cancer vomiting over the toilet. We have very good anti-nausea therapy that we give as preventative treatment, and we give the anti-nausea therapy different antiemetics based on the emetogenicity, or the risk of nausea related to chemotherapy.

And we know that. We know how risky an individual and a specific chemotherapy regimen is. In addition, there are additional anti-nausea medications available for all of our patients should they have nausea above and beyond what the preventative medications can handle. So, that’s one that I think, that nausea doesn’t have to occur and we can treat nausea. Many patients with Acute Myeloid Leukemia, with treatment, will experience fever that is related to the low blood counts and related to the chemotherapy itself. That being said, we give preventative antimicrobial therapy to prevent infection as one of the potential causes of fever.

Patricia:          

Is there an increased risk of sunburn and skin cancer with AML?

Dr. Altman:         

So, some chemotherapies increase the risk of sun exposure and damage and sunburns. IN addition, some of the preventative antimicrobial medication that we use also can cause some skin sensitivity. There is a risk, whenever we give chemotherapy, of an increased chance in the future of secondary cancers. The risk of that is very low, but that is a risk that I talk about with all of my patients. Skin cancer is one of the cancers. There also is potential increased risk of thyroid cancer, increased risk of other bone marrow damage. And so, that is part of the conversation that I have with my patients.

Patricia: 

The internet is a wonderful place, Dr. Altman, but for AML patients or anyone looking up medical information it can be overwhelming and infinite.

And confusing. What are some of the things that AML patients should think about when they’re researching their cancer on the internet?

Dr. Altman:          

So, I think the most important thing is to have a conversation with their healthcare practitioners and ask their healthcare practitioners what resources they recommend. And I think being upfront and telling your doctors that you’re utilizing the internet is always welcome by the healthcare provider. So, I think that utilization of the internet is fine, but just making sure that you ask your healthcare provider what resources he or she recommends.

Patricia:          

Right, right. We have a question from Mari. She says, “I had busulfan treatment for my AML with great success. Experienced a side effect of noticeably patchy and thinning hair.”

“Is there hope for finding a cure for this chemo-induced alopecia? Life and self esteem is a huge role in survivorship. It can’t simply be fixed or covered with a wig.”

Dr. Altman:

Thank you, Mari. I appreciate that question. We at Northwestern have a Dermato-Oncology program that we work with. So, we have dermatologists who are very interested in the immediate and long-term side effects of chemotherapy and the skin manifestations of cancer, including blood cancers. So, my recommendation would be to try to seek out a dermatologist in conjunction with your oncologist to help see if there are other options that exist.

Patricia:          

We also had a question from John. He wants to know if there’s a way to combat serious changes in taste and appetite from chemo.

Dr. Altman:       

So, I smirk a little bit because I keep waiting for the food scientist or food engineer to approach me about this. 

The biggest day-to-day complaint that we get from our patients is that the food tastes bad. And we know that while the hospital food might not be the greatest, it’s not just the hospital food. It’s the effect of the chemotherapy on taste buds. I don’t yet have an answer for this, but I’m very interested in finding a food scientist who can develop food that tastes normally for patients who are undergoing chemotherapy. 

What I suggest to my patients during the time period that they’re having chemotherapy is to try foods that maybe they don’t normally eat so that they don’t recognize how different it tastes from what they’re used to. And things that are a bit more bland for patients taste a little bit better, and colder foods don’t induce as much nausea for most of our patients. But another great question that I don’t have the answer to yet.

Patricia:          

I know we talked a little bit about how overwhelming the internet can be, and how confusing a lot of the information is. How can patients identify misinformation and unreliable sources if they don’t have a conversation with their doctor in the wing?

Dr. Altman:

So, I think that as you mentioned, anything on the internet is not a substitute for medical advice. I think the same pearls that I would give to anyone who’s searching anything on the internet – anything that says ‘always’ or ‘never’ is probably not to be trusted, and anything that sounds too good to be true may well be too good to be true. I would start with reputable sources. The partners that you mentioned – the Leukemia and Lymphoma Society and the Aplastic Anemia and MDS Foundation have really good websites with patient information.

And the emerging growth of this organization as well, we anticipate growth of information available to our patients. 

What is Targeted AML Therapy?

What is Targeted AML Therapy? from Patient Empowerment Network on Vimeo.

 AML expert, Dr. Jessica Altman, defines targeted AML therapy and outlines available treatment options. Want to learn more? Download the Program Resource Guide here.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

See More From The Fact or Fiction? AML Series


Related Resources

 

Your Pro-Active AML Patient Toolkit

 

AML Genetic Testing Explained

 

What’s Next in AML Research?


Transcript:

Patricia:

Can you talk a little bit about targeted therapy?

Dr. Altman:

Absolutely. So, targeted therapy – while meant to be specific, because a target is meant to be specific – targeted therapy has become a relatively broad characterization of additional treatments. We think about targeted therapy as the addition of agents that specifically inhibit or target an abnormality associated with the leukemia. The most prominent targeted therapies right now involve specific mutations seen in Acute Myeloid Leukemia. 

For instance, about 30% of adults who have newly diagnosed AML will have a mutation in something called FLT3, or F-L-T-3. There is now an approved drug that is combined with standard intensive induction chemotherapy that improves the
response rate and overall survival in adults with AML with a FLT3 mutation. In addition, there is now an approved agent for relapsed and refractory FLT3 mutating leukemia. 

Patricia:

What about molecular testing? What can you say about that?

Dr. Altman:

Molecular testing is part of the workup for an adult or a child when they’re newly diagnosed Acute Myeloid Leukemia. And molecular abnormalities look for specific known mutations that occur in Acute Myeloid Leukemia cells. 

For instance, that FLT3 that I mentioned. In addition, the IDH mutation. Looking for those mutations has always been important in understanding the prognosis, but it’s now especially important because some specific mutations, we have additional therapies that we can give as part of initial treatment or for relapsed disease that target those mutations. So, not only do they have a prognostic role, but they have a treatment impact as well.

Medical Update on Acute Myelogenous Leukemia (AML)

This podcast was originally published on cancercare.org by Mary-Elizabeth Percival, Eytan M. Stein, Carolyn Messner on June 14, 2019, you can find it here.

 

Topics Covered

  • Overview of Acute Myelogenous Leukemia (AML)
  • Current Treatment Approaches
  • Transplantation as a Treatment Option for AML
  • New Therapies
  • The Role of Clinical Trials: How They Increase Your Treatment Options
  • Clinical Trial Updates
  • Symptom, Side Effect & Pain Management Tips
  • Key Questions to Ask Your Health Care Team
  • Quality-of-Life Concerns
  • Questions for Our Panel of Experts

Panel of Experts

Mary-Elizabeth Percival, MD, MS

Assistant Professor of Medicine (Hematology), University of Washington, Assistant Member (Clinical Research Division), Fred Hutchinson Cancer Research Center, Attending Physician, Seattle Cancer Care Alliance

Eytan M. Stein, MD

Hematologic Oncologist, Clinical Trialist, Acute Myeloid Leukemia, Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center

Carolyn Messner, DSW, OSW-C, FAPOS, FAOSW

Director of Education and Training, CancerCare

Treating Acute Myeloid Leukemia (AML)

This podcast was originally published on The Bloodline With LLS on May 21, 2019, here.

 

There have been few advances in treatment for AML in 40 years. Why is acute myeloid leukemia (AML) so difficult to treat? What is the current treatment for AML? How is The Leukemia & Lymphoma Society (LLS) striving to change that? How are targeted therapies being used for patients? Is immediate treatment for patients necessary for all AML patients? How does a patient’s ethnic background play a role in finding a matching bone marrow donor?

Join Alicia and Lizette as they address these questions and more with Dr. Martha Arellano from Winship Cancer Institute of Emory University in Atlanta, Georgia. On this episode, Dr. Arellano addresses current treatment and treatment advances for AML, including stem cell transplantation and cellular therapy. She also explains the goal and impact of the Beat AML Master Trial, a groundbreaking collaborative and targeted clinical trial for patients with AML. Listen in as Dr. Arellano shares her excitement about the future of treatment for AML.

Why You Should Consider a Clinical Trial

This podcast was originally published on The Bloodline With LLS on September 6, 2017, here.

 

Listen in as Alicia and Lizette from The Leukemia & Lymphoma Society (LLS) chat with John F. Gerecitano, MD, PhD, Clinical Director of Lymphoma Outpatient Services at Memorial Sloan Kettering Cancer Center and Margaret (Peg) McCormick, RN, BSN, MA, Consultant, Clinical Trials Support Center. Hear about the role clinical trials play in cancer treatment, who can participate in a clinical trial and how participants are protected, how LLS’s Clinical Trial Support Center assists patients in finding a trial that is right for them, and why it is important to think of clinical trials as a possible treatment option instead of a last resort.

Mentioned in this episode:

Is It Difficult to Participate in a Clinical Trial?

Clinical Trial Mythbusters

Clinical Trial Mythbusters: Is It Difficult to Participate in a Clinical Trial? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

Three experts discuss the clinical trial process and the difficulty in participating in a trial. Our expert panel includes:

  • Ken Getz, MBA – Founder and Board Chair, CISCRP
  • Andy Lee – Senior Vice President and Head, Global Clinical Trial Operations, Merck
  • T.J. Sharpe – Melanoma Survivor and Patient Advocate

Transcript:

Andrew Schorr:

And greetings from Southern California. I’m Andrew Schorr from Patient Power. And welcome to this Patient Empowerment Network program, another in our series of Clinical Trials MythBusters. Our goal, of course, is to help you get the treatment for you or a loved one that you need and deserve. I want to thank the financial supporters for this program to the Patient Empowerment Network; AbbVie, Inc., Celgene Corporation. Daiichi Sankyo and Novartis for their support. They have no editorial control and we’re going to have a very freewheeling discussion today. And really what it’s about is how can a clinical trial be made easier for you to participate? Are there barriers? We’ve talked about it in previous programs. But specifically, what are the companies—the pharmaceutical industry mostly, who sponsor trials all around the world, what are they doing to make trial participation easier? For you to know about trials. For the people at your clinic to know about it and what to say and how to administer it. For you to have documents that are understandable for you and your family to know whether you want to participate. To keep you informed. And also related to the requirements of trials. How can they be relaxed a little so that there may be a trial that would benefit you, that you and your doctor agree on, and the requirements of it allow you to be in the trial. Okay, and the logistics of it are not so tough either. All right, I’ve been in two clinical trials, and I believe I’m alive today because of that. So, I’m very grateful. We have some wonderful panelists with us over the next hour. Now as you have questions, send them to questions@patientpower.info. And some of you have. So, you’ll be able to interact with us as we go along. First, I want to go to Ft. Lauderdale, Florida, and T.J Sharpe. And T.J. has been on programs with me over the years. Stage four melanoma patient having been in trials. And T. J., you would agree, you’re alive today because you were in trials, right?

T.J. Sharpe:

Absolutely, Andrew. I think both of us are very fortunate that we found a trial that was the right treatment for us and gave us the ability to combat our disease in areas may not have been available to us if we just waited for standard of care therapies.

Andrew Schorr:

Right. And here you are—we should say that you were diagnosed a number of years ago with melanoma, went through trials. And now you’ve had two years without treatment, right?

T.J. Sharpe:

Yes. It’s been five years of treatment followed by now two good years of a clean bill of health.

Andrew Schorr:

Well, great. And I should mention for our audience, many people are familiar with T.J. T.J. goes around the country, gives speeches. He’s been at many events, consults with industry that are developing trials to try to bring the patient perspective forward. So, T.J., thank you for all you do. We really appreciate it.

T.J. Sharpe:

You’re welcome. It’s my honor to be able to represent all these patients.

Andrew Schorr:
Well, most every family—certainly most have been touched by cancer. But our other guests are not cancer patients but are in national leadership programs. And so, let’s go up to Medford, Massachusetts at Tufts University outside Boston, Ken Getz. Ken, welcome to the program. Ken, ladies and gentlemen, is a true national leader when it comes to clinical trials and really helping us move forward with better processes, better understanding. Ken, tell us a little bit about your organization there, CISCRP. What does that stand for?

Ken Getz:

Thank you. And I have to say your pronunciation was nearly perfect. It’s hard to pronounce it. It’s an acronym and it stands for The Center for Information and Study on Clinical Research Participation. It’s a non-profit organization. It was founded 18 years ago. And it’s really there to help patients and their families navigate the whole clinical research progress which for many is completely unfamiliar terrain until they’ve been diagnosed with an illness or when they have exhausted all other treatment options. So, CISCRP really helps people become more educated and informed so that they can really think of the clinical research process with more confidence. And they can navigate this unknown terrain.

Andrew Schorr:

All right. I’m going to come back to you in a minute because you have such an overview, and you’re also an Associate Professor at Tufts. And so, you study all this, and you’ve written books. But I want to introduce the third guest. And that is a leader from the pharmaceutical industry and one of our most respected and venerable companies in the field, and this is Merck. So, joining us in a senior vice president of clinical operations there around the world. And that’s Andy Lee. Andy, welcome. Thank you so much for being with us.

Andy Lee:

Andrew, thank you. And pleasure to be with some prestigious panelists, both of whom I know. And I’ve met you over the last two weeks. And thank you to T.J. and yourself who have been trial participants and who are representing that part of the organization.

Andrew Schorr:

Okay, and we should mention that both T.J. and Andy are working on a couple of levels. And Ken sounds off on this too. There is a group called TransCelerate where pharmaceutical industry is working together on some of the issues they face in having the proliferation of trials. More trials sites, more accessibility, procedures for that. And then, of course, Andy has helped lead that effort at Merck related to breakthrough therapies that they have been trying to develop there in supporting patients who might be in Merck trials. So, we are going to come back to that. But I want to go to you for a second, Ken. Ken, how low is the participation among adults in clinical trials, at least in the U.S. Now, I’ve heard really low percentages. Where are we now with that?

Ken Getz:

Right, it’s a great myth for us to start with, this notion that only three to five percent of patients—eligible patients, participate in clinical research. That’s actually a statistic that was published by the National Cancer Institute in the early 1990’s. The latest research really shows that it varies widely. For example, when we look at pediatric cancers, the participation rates are extremely high, 80 to 90 percent in same cases—pediatric leukemia. In part because those communities have very engaged healthcare providers, very engaged families that really share their information. It’s just an enabled community where all of the stakeholders support participation. And then there are other areas of course. Some cancers where we do see relatively low participation rates. But I want to point out that low participation is driven by so many factors, Andy, including the strict eligibility criteria. And the demanding protocol designs which are a real burden for some people, and they choose not to participate. As well as low awareness, very low accessibility to trials among minorities and underserved communities. So, there are many factors that contribute to this variation in the participation rates.

Andrew Schorr:

Yeah, you’ve ticked off some now. T.J., in your own experience, one of the breakthrough trials you were in you had to go from Ft. Lauderdale in South Florida and move your whole family to Tampa in central Florida, right. I mean that was a big deal.

T.J. Sharpe:

Absolutely. When you have a young family and a stage four cancer diagnosis, relocating simply across the state during the holidays especially, is no big deal. We were fortunate because we had the means to be able to move there with work situation, with family. But too many people can barely go across the county, much less the state or the country to find a trial that might be the best match for them.

Andrew Schorr:

Andy, so we’ve ticked off some of the obstacles, and Ken touched on some about even the proliferation of trials. Is that a lot of what you do is how can we have trials be more accessible, be more widely distributed to a clinic near you?

Andy Lee:

Yes, let me just explain. When we look at a new cancer therapy, we look at the various cancers that may be affected. And what we do is we go for high probabilities of success. And the challenge is if you bring a new cancer agent. You normally start off in very advanced disease. So, patients would have failed multiple lines of therapy, and often it is a last gasp. And you have to show some sort of clinical efficacy. And then you move sort of backwards in the disease, and you go from sort of third-plus line, second line and first line.

And then you may work downwards into earlier stages of the disease into an adjuvant setting and maybe a neoadjuvant setting. So, as we sit down and design a trial, what we need to look at is what is the population that is most likely to show any benefit at all. And quite often when you are developing a new therapy, it’s difficult to show benefit because many of the patients are very ill. So, what we have to do is optimize the opportunity for success of a compound by going to the right target patients.

And quite often as we have learned a lot more about cancer, this does not mean we test a product broadly in anyone with cancer. We typically try and find a profile of a patient that is likely to respond. And many patients now will realize their predictor biomarkers or prognostic biomarkers. So, for example, with immunotherapies, those that work through the PD1 mechanism would probably want to have a PD1 ligand receptor positive patient who is likely to bind to the drug.

And that gives a higher probability of success. So, it sounds counterintuitive that while we want to develop therapies for all cancer patients, when we start clinical trial development, we have to show efficacy in a population that will benefit. And that’s normally predefined and makes the inclusion criteria fairly strict. As we show efficacy and as we can move into broader populations, it makes it a lot easier for us to design more liberal clinical trials. And then we can actually spread those in the geographic domains.

I could talk more about geographic allocation, but let’s hold that for the time being, and let’s see if there’s time later on.

Ken Getz:

Can I just add to what Andy said because I think it’s really important for your viewers to understand just how active drug development activity is today. We’re looking at over 4,000 pharmaceutical and biotechnology companies, some of them very, very small. But in total, we’re looking at nearly 6,000 drugs that are in active clinical trials. And to Andy’s point, many are really targeting a patient with a very specific genetic profile or a specific biomarker. But it should give anyone who believes that a clinical trial may be an important care option for them, they should recognize that there may be many, many trials out there.

In total we estimate as many as 80,000 clinical trials, nearly 50 just conducted in the U.S. alone—50,000. So, it’s just important that we keep all this activity in perspective.

Andrew Schorr:
Right. So, T.J., that’s why all of us as patients need to ask about them, right? Go to different resources, whether it’s an advocacy group that you ultimately spoke with other patients, and obviously quizzing the doctors we go to. Is there something that may line up with my situation, right T.J.?

T.J. Sharpe:

Absolutely. There is a both top down and bottom up approach here that patients as they become educated—and every patient should be the owner of their healthcare as they become educated. Hopefully they are coming across advocacy organizations, other informed patients, patient support groups—all of which will help inform them different options for disease treatment, including hopefully as Ken mentioned, clinical research as a care option. At the same time, there is certainly very much an opportunity from the top down from the sponsors who develop the trials and from the sites that execute them to educate patients as they come in.

Not just at their own site, but at any site, at any medical facility. That if you have a diagnosis and you are looking into your care options, that you should be asking the question. And we should be giving you more information on the possibility of clinical trials and where you may find clinical trials that are appropriate for you.

Andrew Schorr:

Right, the whole enchilada, if you will, of all your options. Andy, so you mentioned about trial requirements. So, first of all, what efforts either at Merck or are you aware in the industry are being made to really talk to patients early on as you are designing trials? Whether it’s the requirements—how many CT scans you’re going to have. How often you are going to have to go to the main trial site. All the different things that sometimes get in the way.

Andy Lee:

Well, firstly we start with design. And we believe in exquisite trial design, quality by design as well. So, what we want is to run the experiment once and not have a sloppy trial design. We want to make it really robust in terms of scientific integrity and operational execution. So, we have a lot of internal design committees and what we do is we co-op with many groups external to our company. So, we speak to people who run clinical trials at cancer institutes.

We speak to the doctors who manage this. We speak to the trial coordinators. We speak to people involved with the transporting and shipping of medicine how they would do that. And then we of course speak to people in the ecosystem. We quite often speak to investigational review boards before we start trials. We talk to them about our design and what would be best to protect the rights and well-being of patients. And then, of course, the patient-centric approach says that we need patient insights.

And I’ve chosen my words very carefully because the insights are really important. Not all patients—and I’m very respectful that some patients are very intelligent and actually may be involved in this. Some patients can contribute to design, not all can. And so, what we do is we take the insights and we impute those. We often have focus groups. We talk about this disease. We talk about the burden of the disease. And then we talk about how that disease is managed in an ecosystem. And quite often in different countries it’s managed differently.

And so, we have to appreciate the global clinical trials have to navigate a path that may not be a linear path as we’d see it at an exquisite elite cancer center in the United States. It’s community-based, it’s all the rest. So, we take that input, and what we try to do is unburden the trial for the patient. We say, “How can we design a trial that requires the least visits to the clinic—the hospital, the least burden for them. And how can we take some of that burden from the clinic and actually transfer that into an easier environment.

So, document reading and review. Perhaps filling in questionnaires about quality of life. These are things that don’t have to be done in the clinic itself. And then often when we work with clinics, we work with them to help them understand how we as sponsors can make their life easier. And some of those things might be simplifying the informed consent. But I want to stress just one point here is that we can do whatever we like in the design at a company.

One of the things is, the patients are not sponsor patients. Okay, we sponsor clinical trials. The patients are managed by a doctor and a professional. And underneath that principal investigator is a whole oncology team. And it involves radiology. It involves pharmacists. It revolves around a 360 multidisciplinary team. They’re exquisite. They help manage the patient, not the sponsor. We provide the enabling functions for them. And then also that the oversight of the patient’s right, safety and wellbeing is the responsibility of an institutional review board.

And while we may provide templates and simplify templates in text and language, we rely heavily on the institutional review boards to help us with things that may make things easier, such as reimbursement for parking, transport, all of these things. And by and large, the institutional review boards are very supportive of these things. But they are very difficult to quantify in exact terms because of different geographic regions and different norms in different places. So, we rely heavily on exquisitely well-trained 360 team who manages oncology patients with a great PI. They manage patients.

And we work collaboratively with the sites who work with patients on our behalf. So, I just wanted to say the myth is that sponsors interactive with patients. That’s a myth. And the truth is that we engage with clinical sites, and we try and make our design and all the elements—the enabling elements, simpler for the trial sites in order to manage the patients in a simpler way.

Andrew Schorr:

Okay. Thank you for that. So, Ken, I want your comment on that. Because okay, we are downstream patients. We have a doctor, healthcare team. And we know somewhere in the background there’s a sponsor that tried to enable good things to happen to get reliable data and hopefully a cure for us. So, how do we—what’s happening? Are we improving things there in that interaction between clinic and patient?

Ken Getz:

Yes, we absolutely are. And I’ll start by just echoing and acknowledging that Andy has really laid out just an incredible amount of input that goes into the design of a protocol. And that’s really for a really large company. We see many, many examples now of patient advocacy groups or smaller companies turning to a variety of approaches to solicit input from patients and healthcare providers. Some virtual approaches through a social media or digital community. So, there’s lots of ways that feedback is being channeled.

And that’s really important. The flip side, to really answer your question, is that our protocol designs are becoming more and more complex, more and more demanding. A much larger proportion of drugs are now targeting rare diseases that have been stricter inclusion and exclusion criteria. And the designs of the studies—the number of procedures and the number of visits. The number of investigators that are involved, all of that has also continued to grow. And as a result, we do see that our trials are taking longer.

We have yet to see a year when we actually witnessed a reduction in the cycle time to conduct a clinical trial. And we just have to figure out new ways of making the participation process less burdensome and more efficient.

Andrew Schorr:

Oh, my. So, T.J., you had been living with stage four melanoma, a life-threatening condition. We have people even on our team who are living with stage four disease. So, when Ken talks about things slowing, that’s not what we want to hear. We want to hear two things. One is, we can accelerate a development of new medicine. And ideally—because this is an issue certainly in the U.S., but I think worldwide, that by speeding the process, cutting through red tape, improving procedures and us participating, the cost can be less as well.

And when we talk about cancer, the costs are going through the roof as you know for people living with chronic cancer. And you know so well, Andy, people who are on some of the medicines that you’ve come out with at Merck. Where people used to die unfortunately in short order, are living a much longer life thanks to new medicines. We want it to happen faster and be financially achievable. Andy, any comment about the pace of science?

Andy Lee:

Yeah, I would like to make a couple of comments about that. We often hear the sort of story that 80 percent of clinical trials don’t recruit on time, et cetera. We do immense feasibility. Once we have designed a protocol, we send it out to all of the countries that could potentially work with us. We have staff in 47 countries. And they look at two areas of interest. One is the medical durability, is the comparator the one we use in our country. Is the protocol designed the way we practice clinical medicine, not clinical research medicine?

And will that enable us to recruit the patients? That’s the first level. The second level we look at is to ask the question, is this operationally feasible? Can we source the comparator? Do the clinical sites have the equipment? How would we have to ship the biological samples around the world? And based on medical durability and the operational durability, we do a site selection. And we run the indicators through a Monte Carlo simulation. And we simulate this trial. What if we took three countries out? What if we added this more sites? What if we changed this inclusion?

And we come up with a model of what the recruitment would look like. And recruit about 80 percent of our trials according to our model. So, about 80 percent of our trials recruit on our model time. And then if we look at the typical time for drug development, it has been from eight to 10 years for many years in the industry. And when we look at some of the development timelines now—the cycle times. Pembrolizumab (Keytruda), for example, from first study until first approval, was 60 percent reduction in time.

We were looking in the four-year time period. And we are looking at five or six years for many indications. And so, we’ve halved that cycle time for some of the newer oncology products. And there are a number of reasons we’ve done that. One is we have found operational efficiencies. Two is the trial design has enabled us to interim analysis with independent data monitoring committees to assist with that. I’d also like to put in a positive plug for the regulators.

I do believe—and I’ll talk specifically about the FDA, because they are the agency for the United States. They have revolutionized the way they approach the designs and the way they review the data. And they have breakthrough designation status they’ll give to compounds that are really looking like they have strong efficacy. And so, the approval process through the agency has improved remarkably. And they’re open to adaptive designs. And they are open to interim analysis. And they are open to all sorts of things.

So, I really wanted to give credit to our agency who has said, “Where there’s a need for breakthrough medications, we’ll try to find the path.” And so, I do believe there’s a real positive side to this. The challenge is the market is saturated. We have now more than 25 PD1s in development. And to put the 25th one in there, they are so far behind in development. I wonder what that does. It clogs up the system. So, when you look at how can we influence sites, at the top sites we only get one or two patients.

And we compete with 50, 60, 70, 80 other sponsors. And so, it becomes so saturated that, that site has to learn to do systems and process with 70 companies. And what they are doing is almost hedging. They are not focusing on certain things. So, in those cancer centers, they offer treatment for all lines of therapy and all types of cancer, the specialized and nonspecialized. And we are moving out of that sort of geography and moving it community-based oncology practices where it’s less saturated, and we can actually have more traction there and be able to engage more with the clinical trial enterprise for the good of the patients.

Andrew Schorr:

Ken, you write books about all kinds of issues around this. So, if we are getting—particularly in oncology to have trials offered at the community practice where those doctors work night and day—the nurses. They are really stretched. More and more cancers, genomic subtypes, most sophisticated testing. How—what would you say the patient can do. T.J. talked about it a little bit. What would you recommend to patients so that at that community oncology practice the patient and the family can kind of discover what may be available for them as Merck and other companies try to get these trials distributed?

Ken Getz:
Right, well you—talk about the whole enchilada, Andrew. You’re really touching on it. It’s also very exciting times for patients, not just cancer patients, but patients that are dealing with any chronic and severe illness today. And it’s really all about more of a partnership with the clinical care environment and clinical research. And of course, at the heart of it is the patients and their family being as informed as possible, sharing their electronic health and medical information so that they can be connected to trials that might be appropriate for them.

But it’s moving—as Andy said, away from the classic places where trials used to be conducted. And in many cases, they were at these dedicated centers that only conducted clinical trials. It’s a very competitive environment now for patients. So, many sponsor companies like Merck and others are looking at clinical care settings and moving into communities or, in some cases, large health systems where you can have clinical research professionals who will supplement and provide support to the healthcare providers, so they’re not stretched too thin.

But so that they have the clinical research capability onsite at the point of care. For patients it’s a great opportunity because now they have the opportunity to get their own healthcare or treating physician and treating nurse involved in a clinical trial as part of their overall care. And we expect to see more of that over time. We expect to see other virtual trials or opportunities for patients to participate in the comfort of their own home tied in with their clinical care setting.

And all of this is relatively new to the whole world of clinical trials and the investigation of experimental medications.

Andrew Schorr:

You touched on something I just want to follow up on. I’ve heard of this term site-less trials where you said you participate in your home. So, T.J. had to go from Ft. Lauderdale to Tampa. I had to go from Seattle to Houston. There are not—this is a big deal, especially if you have little kids as I did, he has. So—and away from work and whatever your situation is. So, is technology going to come in play so Andy can get the data he needs for the FDA, but that we can have technology help accrue that data in a more efficient way.

Ken Getz:

And I’ll say absolutely. And my colleagues here today I’m sure can comment on this as well. But absolutely. We are seeing wearable technologies and mobile applications that now have the ability measure vital signs and other important baseline information in a validated manner. There are ways that you can access a specific facility for a highly specialized test, specialized imaging for example where the technician can evaluate it remotely. Blood can be drawn at remote locations as well.

So, there are lots of places where we have sort of this more flexible environment that can cater more to the patients and less about a specific physical facility where you have to go to participate in a trial.

Andrew Schorr:
T.J., I want to talk to you about diversity. So, you and I are kind of middle-class white guys. But we want to know how new medicines work for a variety of populations, ethnically, economic groups, et cetera. And Andy needs that data. And he goes to the FDA, and the FDA says, “Well, do you have Hispanic people? Do you have Asian people? Do you have African American people?” or whatever the country is because he works globally. And they say, “We want to understand are there differences?”

How are we doing with that. How can we make a difference there so that we really know what medicines make a difference for broader and also distinct populations?

T.J. Sharpe:
I’m sure Ken can back up some of these things with more hard data than I can. I know that different populations have different levels of trust with the medical system. One thing that you and I both experienced was a lack of options—a lack of good options. And when you get into dire straits, you tend to be a little more trustful of anything that comes along. But we have serious or chronic conditions that have proven treatments that might not be the most effective for certain populations.

And we’re not able to broad the scope to these minority populations or populations that don’t have access to NCI designated cancer centers or top-notch medical facilities. They are not able to get either in a trial that is looking for a drug that would help them or even get access to medicines that have been recently approved simply because their healthcare situation doesn’t allow it. Whether that’s a lack of insurance, a lack of healthcare literacy or simply a mistrust of—there’s a lot of generational mistrust I think in some communities of the clinical trial system.

So, as an advocate, I certainly push caretakers especially—and children caregivers for older populations who are maybe first or second-generation Americans to help facilitate a conversation between the medical professional who’s trusted and a patient that might not be able to get or rely on the information they’re given. Because it really will speak to populations that don’t get the opportunities that you and I have gotten simply because they are either not aware, or there is a barrier there to get to that medical professional.

Ken Getz:

I appreciate, T.J., you mentioned CISCRP. That’s one of the things that we’ve focused on for 18 years is bringing clinical research education into major metropolitan areas around the U.S. and parts of northern and western Europe where we plan for several months, and then we put on what we call an Aware for All events. And we really work very hard to encourage participation by—or from patients based within minority or underserved communities.

And I’m happy to say that we’ve had a lot of success with that. These are really difficult communities to reach through a lot of the traditional approaches. We have to rely on community centers and clergy and other approaches to really help these communities, for a lot of the reasons T.J. mentioned, trust the educational information, and come out to learn more. And I’m happy to say we’re seeing more and more people of diverse backgrounds that are curious and interested in learning more about clinical research, especially knowing that representative populations provide more information that can inform treatment for different types of patient sub-populations.

Andrew Schorr:
I want to go to Andy in a second. Andy, just one second. I wanted to mention and call out—and Andy’s company has been a leader in this. He was talking about PD1 and all of that. But drugs that have been breakthrough in immunotherapy for people like T.J. where—and it’s being explored in broader cancers where otherwise life was going to be short. And how to activate the immune system and really fight the cancer in people living long term. So, the people in those trials—and certainly there were people in the melanoma trials like yourself T.J.

Lung cancer trials and increasingly now others who did get tomorrow’s medicine today. Andy talked about accelerated approval which is great. So, that’s the impetus for the patient and the family. Is there the chance to get tomorrow’s medicine today? Now the obstacles may be distrust. You talked about that, Ken. And also, is maybe accessibility. Is it as a clinic near you? And Andy you talked about pushing that out. And then sometimes it’s related to cost.

Now is there anything that sponsors can do, Andy, related to the costs that people may have in being in certain trials? Where do we stand with that?

Andy Lee:

Yeah, so I’ll just touch on the distribution first and then get into the costs because they are linked. When we prosecute global trials—we’ve had a very U.S.-centric discussion so far. But cancers present differently in different geographic regions of the world. And so, when we want speed out of our trials. You want me to shorten that timeline and get drugs to market quickly. I do it internationally and in some cancers like esophageal cancer or some of the gastrointestinal cancers, Asia has a much higher prevalence of these cancers.

And we do a greater proportion of work there. We always include multi-country studies. And U.S. may have a greater proportion in other areas. So, we balance that out to optimize speed. Of course, with clinical trials the cost structure around the globe is very different. But let’s talk about U.S. We have spoken about a saturated core of clinical trial sites that we all go to. And I speak generally now for all sponsors. And we are all looking to optimize and get great efficiency.

At the same time, we realize we have many underrepresented geographies and ethnic groups—and not just ethnic groups, but under resourced populations. And so, what we’ve been thinking about is how can we support people, and support people at all levels. And so, we start off with thinking about the cost structure, and we obviously pay clinical sites for what they do. But we will support all sorts of things. We’ve been negotiating with Uber and Lyft, so we can build that into automated transport for patients.

Again, the IRB has to approve that. We are looking at ways to augment that they are not out-of-pocket for things. And we’ve been talking a lot with a group called Lazarex Foundation who has really expanded into under resourced communities and found ways to ensure that they have daycare and different access for those patients. We have worked extensively now to look at outreach programs into communities that typically wouldn’t be in trials. We are focusing in two areas right now as we speak.

One is next generation of HIV medicines, and the other one is in prostate cancer. And we’ve got a large program rolling out in prostate cancer. So, what we are doing is going into sites and we have put together training videos and training materials. And we are looking at cultural competency. So, it starts at the site. Are they culturally competent to engage a different community? And we’ve spoken about working with the community churches, community education systems.

And so that starts with cultural competency. I have a woman, Madelyn Goday, who works on this day and night in my organization. And she’s very strong at this. It’s early days, but if we can show that it works in one or two therapeutic areas and cancer types, we’d expand it further and further. But we can’t just have a shotgun approach and just go and do 100 sites and hope it works. Hope isn’t a good strategy. We are working systematically to engage different people. And as appropriate and approved by ethics committees, we will support all of these communities and help build infrastructure and capacity.

Those are important things for us. But as I said, where appropriate and where it’s sustainable. We can’t just throw money at something in the hopes something sticks. We have to have something sustainable and it goes to what Ken says, and that’s education and providing resources and materials. And we’ve used quite a lot of Ken’s materials in multiple clinical trials. Thank you for that, Ken. It’s been really helpful for us.

Andrew Schorr:

Great. I wanted to note for your audience. If you have a question, send it to questions@patientpower.info. We have expert panelists here. And this is really—we are all in this together. I think you hear the dedication from Andy at Merck and T.J. as a patient advocate and Ken as a professor and founder of organizations devoted to this. We want obviously accelerate medicines, but have the accurate data of how it affects different people, who is it right for so that the regulators—and thank you for what you said about the FDA here in the U.S., has the information to make a decision on should this medicine be available for people with that diagnosis.

Okay, so what about staying in the trial. So, T.J., how long—let’s take with the Keytruda trial or one of them. How long were you in to for?

T.J. Sharpe:

Nearly four years. Three-and-a-half years.

Andrew Schorr:

Were there ever times when you said, “I’m done. I want to bail out.” You know.

T.J. Sharpe:

I’ll be very careful how I answer this question for Andy’s sake.

Andy Lee:

It’s okay, T.J., we’re friends.

T.J. Sharpe:

No, probably the biggest crossroads I ever came to was when one of my tumors started growing about a year into it. And we weren’t sure if the medicine stopped working or not. We didn’t know what to do. And as it turned out, it was still working. And I think was just one spot that wasn’t responding. But everything else had responded great. However, at the point, as a patient, you’re thinking about yourself first and your family first and the trial second. It’s easy to stay compliant on a trial when things are going well.

But when you’re ahead of the medicine in some ways, and I think patients with chronic illnesses or in some cases rare diseases, are almost more knowledgeable than some of their doctors or the trial protocols about when they’re stopping. They don’t have the luxury of finishing out a protocol and seeing where their disease journey takes them. And the best example I can give of this is a very passionate advocate by the name of Jack Wheelen who we unfortunately lost a couple of years ago, but whose influence has kind of dominated the patient advocacy world for the last decade or so.

And Jack was able to monitor his health almost better than a doctor. And he knew when his trials weren’t working. When we get to that point in a clinical trial setting where we know the medicine is not being effective or where a patient would be better served to move on to another treatment. That’s when we are going to take the next step in clinical research, because now we’re aligning the trial design and the trial goals with a patient and a patient’s family’s treatment goals. And as those two points merge, that’s where clinical research becomes that much more effective as a care option.

Andrew Schorr:

That was well said. And I think with all those trials, you’re right, the team—that care team, what’s right for you at that time. Obviously to get the data, but also not at all costs. In other words, if the data is showing something is no longer effective for you, is there another treatment or a trial? I’ll just share my story for a second. So, I was in a phase two trial of combination therapies—which are increasingly common certainly in oncology. And after three months—halfway in the trial, my blood was kind of cleaned up.

And I had nausea and some other side effects. And I said to the trial coordinator, “You know, I think I’d like to stop.” And she said, “You know, our belief is that you still have microscopic illness in your bone marrow—in this case with the blood cancer, and the additional three months in this protocol will make a long-term difference for you. That’s what we believe.” They didn’t have the answer, but that’s what they believed. You know what? I stuck it out. She was right. I had 17-year remission.

If I’d stopped after three months, would I have? So, it’s a dialogue with the care team Andy, right? It’s this ongoing discussion not just entering the trial, but remaining in the trial, correct?

Andy Lee:

Yes. Absolutely. And I just wanted to impress a really important thing. People talk about people dropping out of trials. In cancer trials we see extremely low drop out. I mean these are potentially lifesaving medicines for all of the companies. But what we do want to make sure about is that when there is progression of disease, and it’s shown that the drug—whichever it is, the control arm or the active arm or the new agent, where there is progression of disease that they get the best available therapy.

And so that often contaminates trials because we have the crossover effect that now they are getting maybe the experimental agent in the standard of care type of thing. But most important thing for us is to track the survival of the patient, regardless of whether they go on another therapy. And we have put a tremendous amount of effort into looking at the informed consent and making sure we work with IRB to track patients long term survival.

Because as you’ve said, you may have a short-term issue that shows that the drug may not be working short term, but long term it may have prolonged and profound effects. Positive or negative, we don’t know that. And so, what we like to do is get long term survival. And we ask patients to consider when they sign the consent for whatever trial and whichever sponsor is sponsoring this, is to consider that knowing their status throughout their treatment—whether it’s on a sponsor’s drug or another sponsor’s drug or x therapy. It is really important — and I ask people to think about that.

Because that really helps us get as much data out of the individual treatment as possible. And that may prevent nonrequired trials in the future or it may say, “Wow, that really informed.” And we’d like to inform all cancer patients. If data we generate can inform other therapies, we certainly want to do that. We do not want to do wasteful clinical trials. So, tracking patients long term or patients—the message to patients is being cognizant of letting the sponsor—and the sponsor could be an institution. Letting them know your status is really important. All they want to know is are you dead or alive.

Andrew Schorr:

In the end, just one thing is, are we partners. In the end, our viewers here, are we your partner? And can we feel that not just for their doctor but you guys behind the scenes with the labs and everything, that in the end we are partners. And unless we see it that way, we won’t get anywhere.

Andy Lee:

Absolutely. I’m glad you used the term partners. Because when we’ve done a prep for this people have said, “Are they investors in the thing?” So, yes, patients invest their time and everything, but they are partners in research. They are contributing so much. They are contributing—they are going into the absolute unknown. And there is an immense trust level that is there. And we owe that back as research professionals is to treat people with respect, dignity and as partners, to make information available, to publish our data to get it out there as quickly as possible. And to make sure we get that back into the participant’s sort of hands.

Andrew Schorr:

So, Ken, how are we doing on that because you go back over the years and people say, “I don’t want to be in a trial because I’ll be a guinea pig,” and respect was not seen as part of it.

Ken Getz:
Well, that’s also a bit of a myth, right? You had a few that claimed that they felt the process made them feel like a guinea pig. The vast majority of people, over 90% of people who participate in a trial, would do it again. So, once they get past that unfamiliar area where they’ve perhaps only heard a few case examples or a few very vocal people who had bad experiences. Once they’ve done it themselves or they’ve been able to work with a group of advocates that really help them think about this process, and they become more educated, generally they’re very impressed with the level of professionalism, the compassion that exists at all levels.

I work with so many professionals—science professionals and pharmaceutical companies and at the research centers, and they all share that kind of commitment that Andy just mentioned. There’s a real desire to partner with the patient to really inform them. I would say one place where we need to see much, much more however is in the return of clinical trial results in a plain language to people who’ve been in trials. That’s a place where as an enterprise—government, research sponsors as well as industry have not really made this a standard practice at this point. And that’s one thing that we’re really working on actively.

Andrew Schorr:

Right. Great. So, T.J., you and I are investors—and Ken used that term and Andy used it, and I’ve always believed it. We are investors of our tissue, our body, our future to help other people and hopefully help ourselves. And certainly, for profit companies that may greatly benefit if they have a blockbuster therapy. But we need to be kept informed in the long term, right T.J.? We want to know what a difference our participation made.

T.J. Sharpe:

Certainly. And I think to echo what both Andy and Ken said is that patients do become partners. Patients who are involved in clinical research, a significant chunk become altruistically invested. I’ve heard more than once, “Even if this doesn’t help me, I’m glad I participated because it might help somebody else.” I know I’ve felt like that, and I’d venture that you’ve had some of that too, Andrew on your journey. So, it’s only—it’s at the very minimal fair, and it’s certainly very justified to expect as a co-participant in this.

And as kind of a co-creator of science with sites and sponsors that we understand what has come of our sacrifice and our time dedication to helping science out. We shouldn’t have to find it out through press releases from ASCO or hope that we hear about it on the nightly news. We deserve to hear what has happened. Not just because it can affect us as people and as patients, but that we put a lot into this too. And then we did our part to further medical research and we want to be part of the—whatever the end of the trial ends up being. We want to be aware of that. Not just for personal knowledge, but to know that it’s going to help this many other people.

Andrew Schorr:

Right, to be honored. So, Andy, at Merck you’ve established some internet platforms in particular related to keeping people informed, right?

Andy Lee:

Well, we’ve got an internet platform that people can log onto. I’m happy to share that with you; in which they can get access to a list of our trials. So, I didn’t prepare this but especially, but I did make a handmade note. And if anyone wants, it’s a very simple log on. Andrew Schorr:
You’re a great artist.

Andy Lee:

And it’s a simple one. What that will get you access to is two main important things. One is it gives access to information about clinical trials. We have a tab on there that tells everyone about the phases of clinical trials and what to expect in a trial. So, it’s an educational part. Then we have a lot of information about the Keytruda clinical trials were, are running, and they’re called keynote trials. And there you can look at the different indications. And you can look up and it has a telephone number you can call.

Now I must stress is that we run over 1,000 clinical trials in oncology. But many of them are not sponsored by us, they are investigator sponsored trials. So, you can go to clinics, and they run their own clinical trials that are not sponsor-related. And the NCI runs their clinical trials. So, there are a lot of different sources. And many companies will have clinical trials. We also have the website clinicaltrials.gov. I’ve had to use that in the last two days for a colleague.

And you can navigate that and look for different types of trials. And you can look at different products and everything. It’s not perfect. But at least it’s a place to go to. And I don’t want to sound as if I’m one sponsor centric. Many other companies have access to websites, and they really want to try and enhance and direct people to the clinical trials sites at which they are working.

Andrew Schorr:

Right, absolutely. And then you were working at the industry level with a group called TransCelerate, and I know T.J. is involved too, to try and establish common procedures as you establish trial sites, as you have communication, as you have training, right? So, that hopefully all boats will rise, right?

Andy Lee:

That’s correct. TransCelerate is a group that formed about eight or nine years ago. There were 10 initial member companies. I was a founder member of that. And we got together to say, “We have to improve operational efficiency.” So, we do not collaborate on molecular structures and those types—that’s competitive. We collaborate on what we call precompetitive, procompetitive aspects which says, “If we all work together to improve something, we’ll all get the benefit of this.” And we share it publicly.

There’s a website, you can look at it. But we’ve looked at standardizing protocols. We have a common protocol template. We’ve adopted that at our companies, so have other sponsors. The protocol can be developed in a standardized way. We’ve looked at standardizing ways where we can improve monitoring. We’re looking now at ways that we can work with investigative sites through i-platforms, shared investigative platforms. So, a clinical trial site has to provide the information for us as a sponsor and use the exact same standardized questionnaire and information for any other sponsor through a standard portal.

So, we are trying to reduce the burden on clinical trial sites. And we’ve plugged away for many years, and we are seeing greater traction there. We are seeing more efficiency, more standardization. We are seeing greater quality, less rework. And so, while it’s hard to quantify this, what we believe is that the sites are freed up of some of the more burdensome things, and they can direct their attention towards patients, patient safety, and access to clinical trials. So, the work may not be directly related to access for a cancer patient into a cancer trial, but there’s a lot of tangential spin-off of making a site more efficient so they can put their resources and energy in the right place.

Andrew Schorr:

Well, thank you for that effort and your leadership. So, Ken, you’ve been around this a long time. And you’ve deal with all the companies and the government and the various agencies. And as you know, in some quarters there’s a distrust for pharma. We mentioned cancer that you get the price tag of a drug, and it’s very expensive. And some people are struggling to pay for it. And there’s just frustration about it. And often in the news media they are the bad guys who are called out for unethical procedure or something that went awry.

So, how are we doing there in overcoming that because we talk to Andy, he seems very ethical, dedicated guy representing a company that’s been around I think well over 100 years. So, how are we doing to move clinical trials on in this area when people aren’t sure what to make of pharma.

Ken Getz:

Yeah. It’s a huge issue, Andrew. And I think part of the challenge is that all it takes is one questionable behavior, and it makes it difficult for the reputation of the entire industry. Right now, we are dealing with major pharma companies that are actually being fined for having contributed—a judgement, having contributed to the opioid crisis. And when you start looking at some companies aggressive marketing tactics, right? It really sort of sheds a darker light on a lot of the great work that companies do.

What we look at, at the Tufts Center for the Study of Drug Development at the School of Medicine. We look at the overall output, the level of innovation that’s coming from the industry today. And we look at the number of complaints that have been filed with the FDA and other regulatory agencies around the world. And what we see is tremendous growth in the innovation and the quality of the innovation—drugs like Keytruda and other cancer immunotherapies. What an exciting area.

We see that the vast majority of companies really support and live by highly ethical, highly professional, highly compassionate approaches because they all know that it takes just one questionable issue that can really tarnish the reputation of every company operating in the industry. So—again, Andy also mentioned just how regulated we are as an industry, the fact that we have ethical review committees and data safety monitoring boards and so many other external agencies that help to oversee the work that’s done here.

So, I would say for patients who are thinking about clinical trials, it’s good to know the history. It’s good to know what you need to do to protect yourself. But the vast majority find that the people they deal with are ethical, they are professional, they are compassionate. And, as I mentioned, over 90% of people who get involved in trials say that they would do it again.

Andrew Schorr:

Thank you. That was a wonderful response. Andy, you mentioned earlier about starting research with the sickest people basically, where there are no options. But one of the questions that came in is, “Are trials only for the sickest people or are there of all those trials you talked about opportunities for people who maybe are newly diagnosed or could be their fairly initial therapy?

Andy Lee:

Yeah, great question. And thank you to the person who asked that. And the answer is that we start in people—because we don’t know if our experimental agent will work. And everyone assumes that new medicines are all going to succeed. And we work in research and researcher because of that many things fail very early on. They fail in phase one before anyone hears of it. It’s normally a code number at that point. And we may just not make the drug soluble enough, or it may not be distributed enough.

So, we may have a thing that works in a test tube or a petri dish. But to get that into humans and make sure that it’s safe at the dosage we use often fails. We just don’t progress far enough. So, what we want to make sure of is that firstly the drugs are safe. And there’s a trade-off between safety and efficacy. We’re constantly trading off. And so, what we do is we look at that and say when someone has no option and we want to get an option going, that’s where we start.

We’ve actually moved down the disease scale, and we’ve come into adjuvant treatment or secondary prevention. And we’ve gone into newer adjuvant is when you have a small tumor is we pre-treat to manage that tumor before surgery is done. And post-surgery we hope that there’s limited treatment or no treatment. And we actually have removed the cancer, and there would be no evidence of disease. But. of course, using the word cured is something we try not to do, because we prefer to use no evidence of disease.

But absolutely. And the next strategy is prevention of cancer. Our company does a lot of vaccinations in women’s health. We have a product that protects against human papilloma virus which is a precursor for cervical cancer. So, people who are vaccinated with this particular product—and I’m deliberately not using brand names for obvious reasons. But when you vaccinate for HPV, you essentially are preventing the likelihood of a cervical cancer. And there are now prospects in many disease areas where either vaccination or early treatment gives you a tremendous positive prognosis of not getting the disease later on in life.

The answer to your question is yes, we are absolutely looking at ways to prevent getting to a very advanced stage which is very costly to manage and very emotional and stressful and difficult.

Andrew Schorr:

I want to thank you. I just want to get a final comment on what you would say to patients or family member. And I want to start with you, Ken. What do you want patients right now to know so that—what tips would you give them so that they’d consider being part of clinical research or stay in clinical research and the benefit it could be for them.

Ken Getz:

I will say really two things. The first is there’s just a tremendous amount of information out there, and we recommend education before participation. So, do your homework and engage family and friends and people you meet and trust to help you make the decision. And the second point comes off of that. And that is this is not a decision you make alone. Really bring in your treating physician, your nurse. Bring in your support network. And chances are you will learn a lot, and you might even find a trial that is right for you.

Andrew Schorr:

Right. And Andy, what about you? A final point—what would you say to a friend or family member or colleague related to considering trials today.

Andy Lee:

We get this question every single day. And we get it from patients in need. And my answer is we are all patients. We are all going to face this as professionals in our job or professionals outside. And so, I say community of practice. And disease hits all of levels of society in all education professions, et cetera. And so, my thing is to encourage people to do what Ken has said. Work as a team. Get multiple inputs.

And I am sponsor agnostic. Get the best therapy that is available. And that may be the best care option—as I said, the ecosystem in which you get the care is really important as well as the medicines that you get. So, have the discussion. Trust the medical professionals, they are very skilled out there. They are extremely well educated. And I just urge people, “Don’t think on two clicks on Google you are going to solve what your treatment option is.” Really discuss it with people because not all the options are public, and there is not enough information available about how to manage the whole disease through the entire enterprise. Trust the professionals.

Andrew Schorr:

Well said. And T.J., you and I are alive today because of trials. What do you want—what’s the thing you want to leave our viewers with?

T.J. Sharpe:

That they don’t have to be involved in clinical research. I think that’s an important distinction to make. And it’s going to pull together what Andy and Ken said that clinical research should not be considered a hail mary or last gasp option. If you are a patient—and we are all going to be patients as Andy mentioned. You want the best care for you. You want to be able to weigh all of your options. And if you are not considering clinical research, if you don’t know about it or aren’t able to get the information you need about it, then you are not going to be able to make the best healthcare decision long term for your health.

So, take that information that you can get. Find the trusted sources. Be able to reach out to advocates or colleagues or someone that you know that would have the disease or can connect you with good information. And be your own advocate—a little cliché, but really own that healthcare information. And once you are able to collect all of the different treatment options, then you consult with your professional medical team as to what the plan forward—the best plan forward for your individual situation would be.

Andrew Schorr:

Right. T.J., my friend, thank you. It’s a delight to see you again. Andy, with Merck, thank you so much for being with us and bringing your years of expertise. And, Ken, being at an independent non-profit center and also at Tufts University there, thank you for all the work you do. I want to thank the Patient Empowerment Network for pulling this all together. And the sponsors who supported us in this effort, AbbVie Inc., Celgene Corporation. Daiichi Sankyo and Novartis.

All these companies and I’m sure many more, working so that research can move forward. We can be true partners in it. And hopefully get tomorrow’s medicine today to make a difference for the community and live a long life, and hopefully a cure, right? I’m Andrew Schorr in California. Remember, knowledge can be the best medicine of all.


Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

AML Treatment Options: What’s Available?

AML Treatment Options: What’s Available? from Patient Empowerment Network on Vimeo.

Dr. Jessica Altman reviews currently available treatments for acute myeloid leukemia (AML), including chemotherapy, stem cell transplant, and clinical trials.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

See More From The Fact or Fiction? AML Series


Related Resources

 

Fact or Fiction? AML Treatment and Side Effects Resource Guide

 

What Causes a Gene Mutation?

 

Can AML Be Cured?


Transcript:

Patricia:                         

Dr. Altman, let’s talk a little bit right now about treatments that are currently available for AML. What kinds of things might patients want to familiarize themselves with?

Dr. Jessica Altman:    

So, we are at a point in AML therapy where there’s not just one choice of treatment.

There are a number of choices that depend on patient characteristics, disease characteristics, and patient goals. So, there’s a lot that the physician with their patient and family members take into account and consider when they’re coming up with a therapeutic strategy.

Patricia:          

So, give us a couple of examples. Chemotherapy is one way to treat AML, correct?

Dr. Jessica Altman:    

Correct. So, the treatments all stem from a chemotherapy backbone. And there are more intensive chemotherapy regimens that usually involve a long, in-patient hospitalization and less intensive chemotherapy regimens. Those chemotherapy regimens can sometimes be combined with targeted therapy based on the genomic structure or the mutations present in leukemia cells. 

Patricia:          

Stem cell transplant is also an option as well?

Dr. Jessica Altman:                

Stem cell transplant is an option that is utilized ideally after the leukemia is in remission as a way of maintaining disease control.

And for some patients, that is the best approach for a curative option, and some patients’ leukemia does not require a stem cell transplant.

Patricia:          

Clinical trials available as well for AML, doctor?

Dr. Jessica Altman:    

So, we feel very strongly that the best treatment strategy for most patients is a well-designed, appropriate clinical trial for all phases of AML therapy. It’s because of research and clinical trials over the last number of years that we have had advances and more approvals for the treatments of Acute Myeloid Leukemia.

How is an AML Treatment Approach Determined?

How is an AML Treatment Approach Determined? from Patient Empowerment Network on Vimeo.

 AML expert, Dr. Jessica Altman, discusses the factors she considers when making treatment decisions for patients. Want to learn more? Download the Program Resource Guide here.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

See More From The Fact or Fiction? AML Series


Related Resources

 Why Should Patients Be Hopeful About AML Treatment Options?  Office Visit Planner  Can AML Be Cured?

Transcript:

Patricia:     

So, when you’re talking with your patients, what kind of things are you considering when determining how to best treat AML?

Dr. Jessica Altman:    

So, that’s a great question. This is something that is the basis for the entire conversation that I have with my patients and their family members. 

I consider patient goals and patient fitness, other medical conditions, and a lot about the biology of the leukemia. If someone has an acute leukemia that is expected to be highly sensitive to intensive chemotherapy, then that is something that we want to think about. Versus if the patient has a disease that is not expected to be as sensitive to intensive chemotherapy, we frequently like to consider other alternatives in that space.

Patricia:     

So, in terms of options, as a patient what kind of things should I be thinking about when I’m working with you as my doctor about what the best treatment for me might be going forward?

Dr. Jessica Altman:    

So, I think the goal of the initial meetings and the initial consultation between a patient and their healthcare provider is to explore those things. We take a detailed history, understanding patients’ other medical issues. In addition to that, the social history and patients’ goals are very important, as things are not always a yes or no.  

They’re not dichotomous choices. And to be able to understand a patient’s goals, and for the healthcare provider to be able to explain what the intent of treatment is helps both parties come to the right decision for that individual patient.

Clinical Trial Toolkit

Clinical Trial Mythbusters: How Does Medicare or Medicaid Impact My Ability to Participate in a Clinical Trial?

 

Clinical Trial Mythbusters: How Does Medicare or Medicaid Impact My Ability to Participate in a Clinical Trial? from Patient Empowerment Network on Vimeo.

Downloadable Guide

Cancer patients are living longer as a result of clinical trials that test new treatments, therapies, procedures, or new ways of using known treatments.

Watch along as a panel of experts from the Diverse Cancer Communities Working Group (CWG) Sustainable Healthy Communities, LLC, Baptist Memorial Hospital–Memphis, and the American Cancer Society Cancer Action Network (ACS CAN) explore the questions:

  • What do patients and their support networks need to know about clinical trials as an option for cancer treatment if they are insured through Medicare or Medicaid?
  • What requirements differ from region to region and what is covered or not covered?

Transcript:

Laura Levaas:             

Hello, and welcome to this Patient Empowerment Network Clinical Trial MythBusters program on a very, very important topic, what impact does Medicaid or Medicare have on a patient’s ability to participate in a clinical trial. My name is Laura Levaas, and I’m the lung cancer community manager for Patient Power. I’m also a Stage 4 lung cancer survivor. I’m two years out from diagnosis, and I’m also on Medicaid. So, this is a topic that’s really important to me on a personal level.

This program is produced by Patient Power.  We want to thank the following companies who provided financial support to make this possible. While they don’t have editorial control, we appreciate the support of AbbVie Inc., Celgene Corporation, Daiichi Sankyo, and Novartis for their support.

Today we are joined by some really amazing guests, the first being Mark Fleury from the American Cancer Society Cancer Action Network out of Washington DC, followed by Jeanne Regnante, also out of Washington DC, and Jeanne is with the Diverse Cancer Communities Working Group, Sustainable Healthy Communities, and last but not least, nurse navigator Laura McHugh from the Baptist Cancer Center in Memphis, Tennessee. Welcome to all of our guests today. Thank you for joining us.

So, Mark Fleury, Mark is interesting because he has an understanding, a very deep understanding, about this issue from a regulatory and research perspective. He’s going to share with us what he’s learned about barriers in clinical trial participation and solutions to overcome some of those options.

Jeanne is going to share her viewpoint as part of the Diverse Cancer Communities Working Group. She helps share information about access to care treatment and inclusion in clinical trials for underserved populations.

And Laura McHugh who is joining us by phone (she is a friend of a friend of mine, and she’s really amazing) is a nurse navigator who has worked in the cancer space for 24 years. And she helps guide people in underserved communities every day as part of her working life. She works with Medicare and Medicaid patients on the daily. So, we’re looking forward to hearing from her.

So, back to our program, patients are living longer as a result of clinical trials that test new treatments, therapies, and procedures, or new ways of using known treatments for new ways. The myth here behind Clinical Trial MythBusters today is that being in a clinical trial isn’t covered by medical insurance particularly for Medicaid or Medicare patients. I know for me personally I’m interested in being in a clinical trial and I’m on Medicaid but I don’t even know what that means. So, I definitely need some guidance.

So, as we’re talking about this today, if you have any questions about if you’re a patient yourself or you’re a support person for a patient that has cancer or any kind of disease wanting to know about clinical trials on Medicare or Medicaid, we’re here to help you. Send your questions to questions@patientpower.info. So, viewers who are joining us today thank you again. If you’re on Medicare or Medicaid, what do you even do if you’re presented with the option to participate in a clinical trial to treat your condition? Let’s talk with Mark Fleury. Hi Mark.

Mark Fleury:              

Hello Laura. Thanks for having me on.

Laura Levaas:             

Yeah. We’re so, so grateful to have you on our program today because you have such a deep knowledge in this industry and on this topic. Can you tell us real briefly what exactly you do for the Cancer Action Network? And then I’d like to talk to you about barriers around Medicare and Medicaid.

Mark Fleury:              

Sure. So, I work for the American Cancer Society Cancer Action Network. We’re the policy and advocacy arm of the American Cancer Society, and we focus on public policy, so that’s regulation, laws that impact cancer patients. And specifically, my work deals with policies around research and drug and device development, so how can we get those findings that happen in the laboratory into the clinic. And specifically, that goes through clinical trials. So, I’ve spent the last couple of years with a large partnership of other stakeholders taking a really deep dive into looking at clinical trials and all of the challenges patients have in getting themselves enrolled as a part of those trials.

Laura Levaas:             

Good. We look forward to hearing more. Can you tell us a little bit about the current state of clinical trial participation in the US right now?

Mark Fleury:              

Sure. So, there’s not real solid numbers, but we believe somewhere between 6 to 7 percent of US cancer patients participate in a clinical trial right now. So, that’s a fairly low lumber overall, and it’s also a fairly low proportion of the patients who would be interested. Research has found that between 50 and 70 percent of patients would say yes to participating in a clinical trial if they were asked. But unfortunately, many are not asked. And some of those who are asked are unable to enroll for a variety of external reasons. One of the things that we do know is that the people who do enroll in clinical trials tend to be less diverse and better off financially than the overall population with cancer.

Laura Levaas:             

Okay. What are some of the barriers around Medicare and Medicaid patients who want to get involved in a clinical trial?

Mark Fleury:              

Sure. So, obviously, first of all, there has to be a clinical trial for the patient based on your clinical characteristics. But assuming that that is the case, for a patient to enroll in a clinical trial, it’s critical that their insurance cover the routine care costs of that clinical trial. In other words, there are costs in a clinical trial that a patient would see regardless if they were in a clinical trial not. Say, for example, the first step of any treatment is a surgery and then the second step in normal care would be one drug but in a clinical trial it’s a different drug.

Well, regardless, you’re always gonna get the surgery. It’s important that insurance cover that routine part of the clinical trial. And unfortunately, historically, that’s not always been the case. Fortunately, in Medicare, they have covered that since 2000. That is not the case universally for Medicaid. And we can talk a little bit more about that later if you’d like.

Laura Levaas:             

Okay. Perfect. I would definitely like to follow up on that topic seeing as I’m a Medicaid person myself. Can you touch briefly on what actually is different between the two programs in terms of clinical trial, the actual coverage? You mentioned routine care; is that for both programs?

Mark Fleury:              

Well, so what’s important to note is that Medicare is a federally administered program. And so, there is one universal federal policy, and if you’re in Medicare, it doesn’t matter if you’re in Florida or if you’re in Idaho, the policies are identical. Medicaid is an insurance program that while partially funded by federal dollars, it’s administered by each state. And as such, each state has quite different policies. So, if you’ve see one Medicare policy, it’s uniform. If you’ve seen one Medicaid policy, it’s only relevant in the state in which you happen to be. So, it could vary significantly from state to state.

Laura Levaas:             

Right. And so, depending on your state, you would need to follow up with your local maybe human services office to get specific questions answered.

Mark Fleury:              

That’s correct. Yeah. There are some resources (and I think we can provide those at the end of the webinar) where generally speaking some states have passed laws or signed agreements in which their Medicaid programs have to cover those routine care costs in Medicaid. And we can certainly make available those states. But even within those states, it’s important to look closely at the policies. For example, in Medicare, Medicare also covers any adverse events. So say, for example, while you’re being treated, you had to be admitted to an ICU for heaven forbid a heart attack or something like that. Medicare pays for all of those unexpected expenses. And that coverage may vary state by state in Medicaid.

Laura Levaas:

Okay. Thank you, Mark. We’re looking forward to those resources. And for those of you watching, we will definitely be providing a downloadable guide with all sorts of resources to help you. Thanks Mark.

Mark Fleury:              

You’re welcome.

Laura Levaas:             

Hi Jeanne.

Jeanne Regnante:        

Hey Laura.

Laura Levaas:             

Okay. I can’t wait to talk to you about this. I have so many questions. I feel like we could talk for an hour. So, aside from the myth, I came into this thinking, “I’m on Medicaid; I probably can’t get into a clinical trial when and if I get to that point.” And then also, “If I am, it’s probably cost prohibitive because I’m on a fixed income.” So, is participating in a clinical trial expensive or cost prohibitive if you’re on Medicare or Medicaid like I thought? I mean, I know Mark touched on some of the issues, but what would you say? How would you answer that?

Jeanne Regnante:        

For low-income patients, the cost of routine care and logistic support needed during a clinical trial is certainly a barrier to participation. And Mark pointed out some of these costs. But specifically in patients in rural communities, remote communities, aging population, children, patients with cognitive disabilities or physical disabilities. These are the same patients who have low access to care in general.

And covering the cost for routine care in a clinical trial and also the logistic support is a clear barrier to participation. So, there are clear barriers there, travel, housing, parking, paying for food, on having access to clinical trials not only for routine care costs like Mark alluded to but also logistical support being included in the clinical trials. So, all of those things are barriers.

Laura Levaas:             

And would you say that seniors are also part of this underserved population?

Jeanne Regnante:        

Absolutely, especially seniors that live alone, that are in remote rural areas in the United States. And remember, that’s 20 percent of the population, aging population, in those areas. So, clearly, we need to do better to engage those patients in care and also clinical trials.

Laura Levaas:             

So, is it possible for us to draw any conclusions about how many people are on Medicare or Medicaid right now in the US? I did a little bit of internet sleuthing mainly through the Centers for Medicare and Medicaid, and it seems like there – the numbers that I came up with were pretty high, and it’s almost like 40 percent of the population is on Medicare or Medicaid. And so, has it –

Jeanne Regnante:        

That’s absolutely true. Look at by the numbers, there is 329 million people living in the United States, and that’s according to the last census, which is a hot topic these days. There is 60 million people on Medicare, beneficiaries, and about 66 million people Medicaid. So, together, that represents about 40 percent of the population. And we have to remember kids. So, there are 7 million patients on CHIP, which is part of the Medicaid program. So, if you include percentage of people on Medicaid plus kids on CHIP, that’s 22 percent of the population.

Laura Levaas:             

So, then circling it back around to clinical trial participation, how can we connect the dots here?

Jeanne Regnante:        

So, I think one of the main issues is clinical trial sponsors and the clinical trial operations folks in the sites working together to do a better job of reaching out to patients, ensuring that everybody is asked to participate, and not just selecting the ones who people think can participate but asking everybody to participate and understanding the eligibility of all patients and working together to help to cover their costs to keep them in chart.

Laura Levaas:             

Got it. Mark, I’m gonna pull you back into the conversation here for a minute. Can you touch briefly on what’s happening in the news right now around Medicare and Medicaid that could potentially impact clinical trials? Or maybe, Jeanne, you can speak better to that.

Jeanne Regnante:        

I’ll let Mark take that one.

Mark Fleury:

Certainly, so, Medicaid traditionally has been a program that has served primarily children in many states, children and pregnant women. Starting close to 10 years ago with the passing of the Affordable Care Act, states had the ability to expand Medicaid eligibility beyond those kids and pregnant women. And now we see many states who have expanded the roles of Medicaid recipients to healthy adults who just happen to be lower income.

And so, what that really has changed is the number of people obtaining their insurance through Medicaid. Obviously, there has been a lot of – it’s a state-by-state decision whether or not Medicaid is expanded. The Affordable Care Act as a whole is hanging in the balance in a court case, and there’s obviously been a lot of discussion about whether it should continue or not. So, certainly, the number of people who are supported through Medicaid is a dynamic number, and that certainly is subject to changing policies that are still under active discussion.

I will say that Medicare, again, the coverage for routine care costs in clinical trials for Medicare, long-standing policy since 2000 that has been relatively stable. And I would expect that to continue unchanged.

Laura Levaas:             

Thank you, Mark. And Jeanne, I’m gonna come back to you in a minute. For viewers that are watching, thank you for hanging in there with us. If you have any questions that you would like us to address in the program, we’ll get to that at the very end after we’ve talked with all of our esteemed panelists. Send your questions to questions@patientpower.info. So, now I would like to talk with Laura McHugh. Are you with us, Laura?

Laura McHugh:          

I am. Thank you so much for having me.

Laura Levaas:             

Hi. I am so excited to have you. I met Laura McHugh because she is a nurse navigator for a friend of mine who is ALK positive, which is the type of lung cancer that I have. And she works very closely with my friend and speaks so highly of Laura. So, I’m excited to have her on the program today. I wonder, Laura, if you could tell us why you think that clinical trials are important.

I wanted to share why they’re important to me personally. The medication that I’m on right now of course went through a clinical trial process, and it wasn’t even around before the year 2011. I was Stage 4 when I got discovered, which happens often with non-small cell lung cancer because many folks are asymptomatic. So, for me, what that means is if I didn’t have people going through the clinical trial process ahead of me, I probably wouldn’t be here today. So, on that level, is there anything that you can say why you think that clinical trials are important especially for people on Medicare or Medicaid?

Laura McHugh:          

Absolutely. I believe that the clinical trials pave the way. All of the genetic testing that’s done now, all of the testing that’s been done all the way down to a molecular level. So, with these clinical trials and all of the things that have been tested, it’s opened up doors beyond what we ever thought we would have for lung cancer. There are so many opportunities and lines of therapy that you never had before.

And across the board, I think clinical trials and participation in clinical trials, all of the people that have done that, just opened the doors for all of the people in the future. We had a lady who was in her 90s, and she met all of the requirements, participated in a clinical trial. And all the way through, she said, “I want to stay on this. I want to do this. It may not help me, but it will help everybody after me.” And that’s just profound.

Laura Levaas:             

Right. And so, Laura, tell the audience who you work with. I know that you specialize in thoracic cancers, and I know that clinical trials don’t always just focus on cancer. They deal with multitudes of diseases and conditions. But can you let us know who you work for because he’s famous in a way, right?

Laura McHugh:          

Absolutely. I’m actually the physician nurse for Dr. Raymond Osarogiagbon. He is well known in the field of lung cancer. That’s our specialty. We have a multidisciplinary meeting every week and a conference. He sits on the board for NCCN and multiple, multiple other things as far as paving the way for lung cancer. I’ve been actually privileged to be his nurse since he came in 2005. We’ve built our practice together, and, oh, the changes are just – the changes that I’ve seen in the years that we’ve done this are amazing. And he is brilliant; he is. He’s known all over the world. And our focus is lung cancer.

Laura Levaas:             

That’s great. Can you shed some light on the role of the patient navigator or the nurse navigator in what you do on a daily basis with your patients especially around clinical trials and folks who are on those government-supported insurances like me?

Laura McHugh:          

Sure. So, we base all of our care – we – or I’m blessed to have a research department and two really dedicated research coordinators that I work with very closely. They’re not nurses like myself, but they do all of the coordinating for the care on the studies and all of the above from patients that are uninsured that are on Medicaid, Medicare, even private insurance. And what we do, we see primarily all of our new patients insurance allowing through our thoracic program.

So, I actually have a coordinator with me when I’m in clinic. And so, if we even think a patient is potentially eligible – not even just for a drug study. There are smoking cessation studies that we have, different protocols for that. So, it really starts at the beginning. There’s the surgical studies, different things like that. And every Wednesday is that clinic. And even during the week, if there’s anything going on, they come to our regular clinics as well and do follow up with the patient.

Laura Levaas:             

So, I hear chatters here and there – when I bring up the subject of clinical trials, I hear things like, “Oh, trials are only for young people,” or, “Trials are only for old people,” or, “Trials are only for this type of person.” Can you speak to that a little bit?

Laura McHugh:          

Wow. Yeah. Well, part of it is if you look at where we sit, there’s always – until now, in recent years, you heard about research but you didn’t really hear about research. So your older population, they were skeptical. It’s a different generation of, “Are you experimenting on me?” And part of your underserved communities, a lot of people didn’t know anything about it. They’re limited on getting to a physician in general much less being able to participate or being in a center that even focuses on clinical trials.

So, I think all of that in the past was very, very real. I believe now people are coming around and seeing, “Wow, anybody can do this.” I think people are still limited. Some people don’t have computer access. It’s hard in a day of electronics, we sit down and we can pull up all of this information, but not everyone can do that.

Laura Levaas:             

Right. We do make a lot of assumptions when it comes to those type of factors. So, being that you’re a nurse navigator, I imagine that when you’re seeing a patient, you’re thinking, “Okay, is there a trial that this person might be good for?” I don’t want to say convince, but how do you help people learn about clinical trials and the importance of it because when you and I spoke yesterday, you said you want to make it clear to patients it’s always voluntary, “We’re not dragging anybody into a study. We want to make sure that you want to be there”?

Laura McHugh:          

Absolutely. So, again, all of our patients are approved during a thoracic conference, and then all of the ones that we can bring to our clinic within our healthcare system we bring through that clinic, and if not, we bring them to our general oncology clinic. The physician will sit down with the patient. Of course, we’ve met with the coordinators, they’ve looked at everything. And they’ll come to us and say, yeah, they like this or this. The physician sits down and talks with them, and then I go in the room and talk with them as well. We tell them, “This is totally voluntary, something that’s open to you if you’re interested,” talk about it.

The coordinators go in and speak with them as well. We tell them to go home, “If you have any questions or concerns, call back.” And a lot of times they will. You have to be able to digest something. It’s a very overwhelming visit to walk in an oncologist office and be told all of this information and try to sort it all out on the spot. So, a lot of times they’ll go home, they’ll think about it, they’ll call back. Basically, communication, I just feel that’s the most important – it’s communication.

Laura Levaas:             

Absolutely. So, to circle back a little bit, do you feel like it’s realistic for patients that are on Medicare and Medicaid to be in a clinical trial?

Laura McHugh:          

Absolutely. I think it’s clinically appropriate for anyone that fits. If everything lines up the way it should and they’re able to participate, I think it would be wonderful if everyone would.

Laura Levaas:             

This may seem like a silly question, but do folks on those programs get the same care as somebody that has a private insurance?

Laura McHugh:

Absolutely, absolutely from our standpoint. Of course, I’m answering from my institution and what I know that we do. And they do, absolutely. And sometimes there are challenges. I mean, we’ve had patients that were uninsured, underinsured. Again, Medicaid, you have to make sure – Medicare’s a little bit different again because all of the guidelines were set state to state. Medicaid’s different because each state has its own – and if you see someone in Mississippi, sometimes they can’t come across to Tennessee to go to the hospital or to do this. So, it’s a patient-by-patient basis, but overall, I think our patients are being treated, being offered clinical trials, and should participate if at all possible.

Laura Levaas:             

Wonderful. And again, just to underline that clear and open communication is important.

Laura McHugh:          

I think communication is No. 1 for everything. People are scared. They have questions. They don’t even know what to ask immediately. So, I think all of the support you can give – everybody has a knowledge base and everybody is empowered with that knowledge. Sometimes it’s all about just listening, communicating, and then answering any question they have no matter how simple it may be to us. To a patient, it’s a very profound thing. And it could be as simple as, “How am I going to get back and forth? Do you have a way to help me?”

Laura Levaas:             

Thank you, Laura.

Laura Levaas:             

Okay. I’m gonna circle back to the group and just ask some questions. I wanted to rewind with Mark and talk about Medicare Advantage. I am on Medicaid for about another year and I’m going to be rolled into Medicare, which under typical – I mean, I’m 44 years old, and so Medicare is typically for people that are 65 and older. And so, for me, it feels a little bit strange, and I’m like, “I just want to know how are they different.” And so, I have called my local CMS office, my local Social Security disability office. And I feel like I get different information. So, it’s sifting through everything. I just wanted to call out Medicare Advantage because you mentioned that. Can you expand on that and how it ties in with clinical trials?

Mark Fleury:              

Sure, sure, happy to. So, traditional Medicare has multiple parts. You have Medicare Part A, which is the hospitalization, and Medicare Part B, which is the physician portion, and then a Medicare Part D, which is the drug portion. A few years back (understand the complexities of all the pieces and parts of Medicare) there was a decision to allow private insurance companies to administer all the programs together on an optional basis.

So, if you qualify for Medicare, you can use the traditional what’s called fee-for-service Medicare or you can go through a private insurance company. So, this might be an Anthem or a Blue Cross or another private insurance company like that who has been authorized to bundle all of your Medicare benefits together under one program. Now because it is a privately run version of Medicare, they’re required to offer the minimum benefits, but they do have some flexibilities in how they administer that.

So, a traditional fee-for-service Medicare, as long as a physician advertises that they accept Medicare patients, you can go anywhere you want to. If you live in Florida and you go on vacation into Los Angeles and become ill and you want to go visit a physician there, as long as they accept Medicare patients, that’s fine. Medicare Advantage on the other hand looks a lot more like private insurance in that they sometimes build closed networks, so, you can only go to certain systems or only go to certain doctors. So, that’s an important difference between the two.

And in terms of with clinical trials, how that’s affected, if you want to enroll in a clinical trial and you’re Medicaid Advantage, right now the current policy is for the portion of your care that is related to the clinical trial, you would revert back Medicare fee-for-service, traditional Medicare. That doesn’t mean that you are kicked off of Medicare Advantage, but anything related to that clinical trial would be handled from a payment and a billing standpoint through traditional Medicare.

So, if you’re on a cancer clinical trial, all those cancer clinical trial bills would go through traditional Medicare. But say, for example, you needed to get your flu shot or had a cold or something like that, that would still be handled under your traditional – or under your Medicare Advantage. You wouldn’t be kicked off of it. It’s just the treatment part of your clinical trial would go through traditional Medicare. So, a little confusing, but that’s where we are from a policy standpoint today.

Laura Levaas:             

Okay. Jeanne, I wanted to ask you – and again, if you want to defer this to one of our other panelists, that’s A-okay. I’m thinking of folks who have some barriers around those additional costs in a clinical trial. Is it typical or acceptable for the, for example, pharmaceutical company or the sponsor of the clinical trial to pick up some of the costs that may not be covered under Medicare or Medicaid?

Jeanne Regnante:        

The answer to that question is yes, it is appropriate for them to do so. And actually, there is an FDA guidance document (it’s Guidance for Industry) that actually reinforces their ability to do so because there has been some concern that covering costs like logistical costs or hotels or travel or giving people a gas card would create undue influence. So, I think the FDA put out a draft guidance that’s clearing that up and basically reinforcing the fact that pharmaceutical companies are able to do that.

I can tell you from our working group, we have 10 active major pharmaceutical company members in the Diverse Cancer Communities Working Group. And I asked them what they usually do in this space, and during the planning phase of the clinical trial, they go out to their sites to ask for a budget and ask them what they need in terms of routine care costs and also logistical costs. And the site sends that information in. And generally, the pharmaceutical companies cover those costs.

What I’ve found to be the case, which is interesting to me, is that the clinical trial operations team in the sites have a lot to do, they have a lot of work to do. And this was brought up to me by a couple of leaders in pharmaceutical companies, that what they’ve learned is that they also need to ask what capabilities do you need, do you need people support or FTE support to be able to adjudicate and track those costs at a site level and validate them and close them out and pay them. And a lot of times, the answer is yes and pharmaceutical companies are paying for those FTEs at the site. So, those costs are being covered when the site asks for support.

Laura Levaas:             

Got it. So, since we’re talking about this topic anyway, that draft to FDA guidance publication, I’m gonna say it. It’s a really long title. It’s a mouthful. But I’m hoping you can break down a little bit of that. So, it’s called Enhancing the Diversity of Clinical Trial Populations, Eligibility Criteria, Enrollment Practices, and Trial Designs Guidance for Industry. What is the meaning –?

Jeanne Regnante:        

So, I do want to paraphrase what the FDA says, but I’m gonna read the portions that I think are appropriate for this discussion. So, there’s a section in this guidance that was put out in June, and it’s a draft guidance, so, it’s open for public comment. And it focuses on study design and conduct considerations for improving enrollment in the industry. There’s a big section. I really would urge everybody on the call to read this section because I think it’s really great and progressive and quite empathetic of a major governmental agency to put out this guidance to industry.

It gives examples. It notes the burden for trial participants in remote and rural locations, for example, and also acknowledges the trial burden on the elderly, children, disabled, and cognitively impaired individuals who require caregiver assistance. So, what the FDA does in this guidance is they encourage industry to reduce No. 1 the number of study visits where possible and use electronic communications or mobile technology to monitor the patient for safety and efficacy because of the challenges of a number of folks in this patient population.

They also encourage industry to make sure that patients are aware of financial reimbursements, and that’s what Laura does. She manages their expectations in the recruiting stage and reinforces the fact – and the guidance also reinforces the fact that the FDA does not consider reimbursement of travel, lodging, parking, time, and other considerations to raise issues concerning undue influence. And they also reinforce that the amount of dollars that might be reimbursed should always be addressed with the local IRB. So, I think this is a very progressive guidance to give the industry so there are no questions on what they can and cannot do.

Laura Levaas:             

Okay. Thank you very much. Laura McHugh, quick question, and Mark touched on this earlier in the program, what if something goes wrong in a clinical trial and a patient has to be hospitalized or treated for an unexpected reason? That’s covered, right?

Laura McHugh:          

It has been for our patients. If it’s Medicare, what you always look at is standard of care. And the Medicaid patients that we’ve had, when they’ve been hospitalized, to my recollection, we’ve not had anyone that we’ve had difficulty substantiating why it should be covered. I mean, sometimes you have to go the extra step and go back and forth with the insurance companies or Medicaid. But we so far have been able to get it covered.

Laura Levaas:             

I have a couple of questions that have come in from the audience, and feel free, Mark, Jeanne, or Laura. I’m assuming that a nurse navigator or a doctor is going to have the best information on where to find out about a clinical trial. But where are the best resources for someone to go? And again, I’m cancer focused because I have lung cancer and I work for Patient Power. And we support all types of folks with cancer. But there are folks that are in clinical trials that are not cancer related. Mark, what would be a source where somebody can find a clinical trial?

Mark Fleury:              

Sure. So, in looking at the current cancer clinical trial landscape, we know that the overwhelming majority, probably 75 to 80 percent of patients, who end up on a clinical trial found that clinical trial because someone on their care team recommended it or someone from the clinical trial team approached them. So, it’s most common that someone from the medical system invites that patient. But we also know that a lot of patients get their cancer care at very small practices (they might be single-doc practices or things like that) where clinical research is not a normal part of what they do. And in that case, you would not necessarily hear about clinical trials from your nurse or from your physician.

In those cases, it’s up to an empowered patient to find the clinical trial on their own. And that’s obviously a little bit harder but certainly not impossible. And there are public-facing websites. Some of them are sponsored by the government, things like ClinicalTrials.gov where all clinical trials whether cancer or not are listed in the United States. And NCI has one, trials.cancer.gov, which is just NCI sponsored, which is the National Cancer Institute. So, it’s federally funded clinical trials.

But additionally, many patient organizations both have general educational materials about clinical trials – so, for example, the American Cancer Society at the website cancer.org has information about clinical trials. At the moment, we don’t have a matching window, if you will, but many patient-advocacy organizations also actively help patients one on one with matching. So, many of these are disease specific. So, there are lung cancer groups who you can call at the hotline, colorectal cancer, etc. Many patient-advocacy organizations will do the direct handholding and navigation if your own provider does not do that for you.

Jeanne Regnante:        

I just want to add to that great list that Mark gave in terms of finding clinical trial sites. So, just a shout out to Stand Up To Cancer, they have a clinical trial matching site for any type of cancer. You can contact them, and they will actually match you to a clinical trial site in your area so you can give that information to your provider so they can call them to see if you qualify. Sometimes it’s difficult for anybody, myself included, to understand what clinical trial I might be eligible for just by looking at a site. So, it’s nice to have somebody do that for you.

Also, all the major pharmaceutical companies have if you happen to know about a given therapy or that you might be looking to be on because you heard about it it’s good to ask for help from somebody to find out what company makes it go to their website. And they all have clinical trial information on their sites as well.

Laura Levaas:

Thank you. And I’d like to share a little bit about my personal experience. When I was diagnosed, I was told about a Facebook group for my specific type of lung cancer mutation. And I learned about clinical trials from that group. And if I had never, like you said, Mark, been an empowered patient and been very curious in wanting the best care for myself, I probably would not have found out about those trials because some of them are just fly under the radar; they’re doing their work.

I think these are some great resources, and thank you for sharing those. One more question that I would like to ask the group before we – we have a couple of questions that came in from the audience, which is awesome. What is one solution (Mark, we’ll start with you) that you would like to put forth to address the issue of better clinical trial participation for Medicare and Medicaid patients which really, I mean, goes out to the larger group, I mean, really for anyone?

Mark Fleury:              

Yeah. Well, I think specifically within the population of Medicare and Medicaid, as I mentioned at the outset, Medicare has a uniform national policy. So, someone like Laura, if she became a clinical trial professional in a different state, the Medicare policy would be the same it doesn’t matter what state you’re in. Whereas Medicaid, it varies so much, and that can be quite a bit of hurdle.

As I mentioned, I work in the policy and advocacy portion of ACS, and so, we focus on legislation. And so, one of the public policies that we have been advocating for (and there’s actually a piece of legislation before Congress right now), it would harmonize all 50 states plus DC Medicaid policies such that standard of routine care costs in cancer clinical trials would be covered in all 50 states in the same way and there wouldn’t be this ambiguity or uncertainty from state to state in terms of how it’s covered. So, that would be my one wish within this question if I could wave my magic wand.

Laura Levaas:             

Yeah. That would very much clarify everything. Ms. McHugh, do you have a solution? What would you like to see happen to get more folks participating in clinical trials specifically those on the Medicares and Medicaids?

Laura McHugh:          

Again, from my nursing background, a lot of it’s communication. And I think it’s sitting down with patients and explaining what some of the benefits are, what the risks are but what the benefits are because truly the benefits outweigh the risks. People worry about money and they worry about all of these things. Well, if it’s Medicare, it’s standard of care. Anything above and beyond, if there’s a problem, then you appeal back to the drug company, the provider.

Opening doors, communicating with patients, telling them, “You have a more active role in your own healthcare when you’re on a clinical trial. You’re empowered. You’re educated. You’re the first to benefit from this drug. You have your health professionals close. You’ve got a research coordinator, your nurse, your doctor, access to new drugs that may not be available.” I just feel like communication and – we’re totally sitting down with someone and explaining and taking some of the fear away from what people think about being on a clinical trial.

Laura Levaas:             

I have a friend in the lung cancer community that was in a clinical trial. I don’t remember the specific drug, but she is still on it after it came out of trials. And she’s been on it for years, which is amazingly successful. And if not for that trial, she wouldn’t be where she is. And so, that’s just amazing. Okay. And then, Jeanne?

Jeanne Regnante:        

You know what, first of all, I agree with what Mark said and what Laura said. First of all, it needs to be legislated. And No. 2, there needs to be better communication amongst trusted providers, trusted community leaders, primary care physicians to talk to patients to have them understand that a lot of these trials now include placebo versus standard of care and also help them to manage their expectations in terms of what will be covered in terms of their cost. And the folks that need to do that are the closest to the healthcare systems and patient navigators and care coordinators who can talk to an individual specific situation.

I think in addition to all those things, I think that generally industry needs to do a better job of placing trials where the patients are. Although that seems quite trite, patients that are in underserved communities or in rural communities, they don’t often have access to these cancer centers which are big academic centers that do a lot of these trials with big innovations.

And I think that we need to get much more creative to make sure that either the reach out from those academic centers go out to community centers or we do a better job placing clinical trials in community research centers to ensure better accessibility because really, logistical support, even if you cover it, even if the industry covers it or cancer care covers it or the American Cancer Society cover it or a laser X organization covers it, it’s still a challenge and a barrier.

So, I think we need to do a better job overall. The infrastructure needs to place trails where the patients are because cancers are not homogeneous across the United States. They appear in different places with higher risk and higher prevalence. And we need to use that data to place trials where the patients are.

Laura Levaas:             

I agree. I’m actually located in Denver, Colorado, and I was doing some research for a blog post recently. And I went to American Cancer Society, Mark, just to look for what are the most recent statistics by state in terms of cancer. And obviously, it’s not lung cancer specific. But I was shocked to find out that Colorado has one of the highest percentages in the country of cancer occurrence. And I was surprised. So, Laura, would it be appropriate – this article that you sent me this morning from ASCO, would this be appropriate to include in our downloadable guide for our guests after the program? This was about the Affordable Care Act because we were talking about how people can get involved if they’re interested. What do you think, should we include this, Jeanne?

Jeanne Regnante:        

Oh, I heard you say Laura.

Laura Levaas:             

Yeah. Sorry.

Jeanne Regnante:        

I think it’s a really well thought out piece to help folks understand how they can get involved with their legislators and understand that this act and this piece of legislation to advocate [inaudible] [00:50:28] specifically for patients that are on Medicaid in the United States so they can get the same benefit of routine care that Medicare patients get.

Laura Levaas:             

I do have a question from Steve, one of our audience members, and he says, “Can Medigap Plan F help with paying for clinical trials? If the clinical trial accepts Medicare, would my out-of-pocket expenses be covered? I’m worried that any extra testing would be my responsibility.”

Mark Fleury:              

Yeah. I’m happy to jump in with a quick answer on that.

Laura Levaas:             

Okay. Thanks Mark.

Mark Fleury:              

So, I mentioned a little bit before about what’s required to be covered. When you think about costs involved in a clinical trial, I’ll put them in three buckets. There is the normal routine medical care that you would get. So, for example, if you would normally get surgery and then followed up by some sort of chemotherapy, everybody’s gonna get the surgery regardless. And then say, for example, ordinarily routine care would be you would get a scan every six months after surgery, but the clinical trial because they want to collect more data wants to have a scan every three months instead of every six months. And the clinical trial is testing a new drug after surgery.

So, Medicare would pay for the routine costs, which would be the surgery and then a scan every six months. The clinical trial sponsor would pay for the drug, which is what you’re testing in the clinical trial. So, the patient doesn’t have any responsibilities for that. And since there’s basically twice the frequency of scans, the sponsor would pay for every other scan.

Now what’s important is that while Medicare covers the routine care costs, it covers them the same way it would cover any other cost. So, if you have a co-pay for a doctor’s visit that is routine, just because you’re on a clinical trial, that co-pay doesn’t disappear. So, if you have a Medigap plan that covers those co-pays, it should cover them the same way as if you were not on a clinical trial because the only responsibility for the patient is the co-pays of the routine care costs, and Medicare will pick those up.

So, anything that’s not normal from a medical standpoint will be paid for by the sponsor. Now as Jeanne aptly pointed out, if you’re coming in twice as often for tests, even if the test itself is paid for, you might be paying for the parking garage twice as often or gas to travel twice as often. And those are nonmedical costs that can add up, but they’re not really involved with insurance, but you can sometimes get money from the sponsor or other third-party support organizations like ACS.

Laura Levaas:             

We have one more. Annie B, “I’m on Medicare. Where do I find a clinical trial in my town?”

Mark Fleury:

Typically, most of the ways that you find clinical trials, again, you can work directly with where you’re seeking care. So, if you have an oncologist, you can ask them about clinical trials. And if they conduct them, they will screen you for the trials that they have open at their site. If they don’t conduct clinical research, then you would either go to one of these public websites like a ClinicalTrials.gov, you could call an advocacy organization. There are several in the lung cancer space, and we can provide a number of different links to different matching engines or third-party organizations that could help match you. But clinical trials typically are not restricted based on insurance types. So, you would use the same search engines as anyone else would.

Laura Levaas:             

Okay. All right. Well, I want to say thank you so much to our esteemed guests for joining us today. We learned so much today about clinical trials, Medicare and Medicaid, the different options. So many takeaways here. We will have a downloadable guide available as well as a replay of the program in case you’d like to dig in a little bit deeper.

Really, I think my takeaway from the whole program is that there are options out there. Clinical trials can be a great solution for your medical care of your disease. I personally am all for it. I know it’s a very personal decision, whether you want to participate or not. But I decided early on that I would definitely enter a clinical trial because I’m willing to sacrifice myself for future generations because there are people that came before me that did the same and I would not be here today if not for that. So, thank you again for joining us Mark, Jeanne, Laura. We very much appreciate you.


We thank AbbVie, Celgene Corporation, Daiichi Sankyo, and Novartis for their support. 

Why Should Patients Be Hopeful About AML Treatment Options?

Why Should Patients Be Hopeful About AML Treatment Options? from Patient Empowerment Network on Vimeo.

Registered nurse Mayra Lee explains why AML patients should be optimistic, emphasizing the positive impact of recent treatment approvals and personalized medicine. Download the Find Your Voice Resource Guide here

Mayra Lee, RN, is an outpatient clinic nurse at Moffitt Cancer Center. More about the expert here.

See More From the The Pro-Active AML Patient Toolkit

Related Resources

 

AML Genetic Testing Explained

 

What Is Personalized Medicine?

 

Finding Resources & Support in AML: A Patient Perspective on Giving Back

 


Transcript:

Mayra Lee:

Other advice that I have for AML patients or reasons to be optimistic is a lot has happened in the last three years in AML that hasn’t happened in 30 years. There has been a lot of drugs that just recently got approved. There has been a lot of research. Cancer itself is moving in leaps and bounds and it’s incredible what’s happening actually in our centers and the academic centers with research and clinical research and personalized medicine based on the cytogenetics of the diseases.

There’s so much of that going on and there is so much focus on that that there is a lot of hope. Recently, in the last two years or so, there have been at least five or six medications approved for AML that didn’t exist before in the market. So, it gives hope to other patients that didn’t have hope before. And clinical trials give hope to those patients as well. We can’t promise you any results on a clinical trial but there’s certainly a medication or a treatment that wasn’t available to you beforehand or that wasn’t available if it’s not outside of the setting of a clinical trial. So, keep optimistic. Keep thinking that you’re going to beat this because if you come into this arena of AML feeling defeated that is going to reflect, actually, on everything.

Patients that have a great attitude, have a positive attitude, think that I’m here, we’re going to do this together do the best.

A Conversation With Becky Pleat

Specialty Pharmacy and the Patient Journey with Specialty Medication

In this segment of A Conversation With, Becky Pleat the Associate Director of Medical Managed Care Oncology Specialist at Sanofi discusses specialty pharmacy and the patient journey. Becky answers the following questions:

  1. What is a specialty drug?
  2. What is a specialty pharmacy?
  3. Where can patients find a specialty pharmacy?
  4. How do patients receive a specialty medication?
  5. Will a specialty medication be covered by a patient’s health plan?
  6. What kinds of services and/or resources are offered at specialty pharmacies?

What Are the Current Treatment Options for AML?

An expert panel help viewers understand more about the evolving field of AML treatment. This includes identifying prognostic factors and determining patient subtypes to setting treatment goals and selecting a suitable course of treatment. The panel was made up Dr. Uma Borate of Oregon Health & Science University, Amanda Fowler of The Leukemia & Lymphoma Society, and patient advocate, Don Armstrong

Downloadable Program Guide


Transcript:

 

Andrew Schorr:

And greetings from Los Angeles.  I’m Andrew Schorr.  Welcome to this Patient Empowerment Network program produced in association with the Leukemia & Lymphoma Society and with support from the following companies:  Celgene, Daiichi Sankyo, Jazz Pharmaceuticals and Novartis.  We thank them for their support, but be advised no outside party has any editorial control. 

In this program we’re going to discuss the latest the treatments for acute myeloid leukemia.  We have a wonderful panel, and over the next hour we will be discussing the options, but of course always discuss this with a healthcare team that you trust so you get the care that’s right for you or a loved one.  Also, if you have a question send it in to aml@patientpower.info, aml@patientpower.info, and we’ll get to questions as we can. 

Today with us with have with us a pretty long‑term survivor of AML joining us, and we also have an information specialist from the Leukemia & Lymphoma Society and a noted physician researcher from Oregon Health & Science University.  So let’s go around the country and meet them.  I’m in Los Angeles. 

Let’s go to Fort Worth, Texas, and joining us now is Don Armstrong, who was treated for AML.  Gee, Don, in 2005 your whole world turned upside down, right? 

 

Don Armstrong:

It absolutely did.  September of 2005 I had no idea there was anything wrong with me until I had a couple ladies tell me on a Friday afternoon I looked horrible.  That ultimately led me to see my general practitioner, and a couple of days later I was diagnosed with acute myeloid leukemia, and from there right into the hospital, right into treatment. 

 

Andrew Schorr:

Right.  And you were in the hospital for a long time. 

 

Don Armstrong:

I was.  I was in the hospital the first time for 33 days. 

 

Andrew Schorr:

Wow.  And ultimately you had a transplant, which is one of the approaches still for AML, and you survived. 

 

Don Armstrong:

I did.  I did.  Yeah, it was interesting.  That first couple of days in the hospital there was so much activity going on around we and I couldn’t understand why I was getting so much attention until one of the nurses kind of pulled me aside and said, you understand you’ve got a 25 to 30 percent chance of surviving this type of leukemia, don’t you?  And I said, now I do, yes.  So it was quite a shock to the system, and it was something that‑‑you just have to kind of hang on and just go with the program as much as you can. 

 

Andrew Schorr:

And a shock to the family.  I mean, it’s a family affair. 

 

Don Armstrong:

No question about it.  Whenever you’re diagnosed with cancer, no matter what the cancer is, it’s just not you.  The entire family is involved.  And I had a great support system.  My dad and my brother and my sister came from different parts of the country just to be with me and support me.  I had an unbelievable group of family and friends that were there with me every single day. 

 

Andrew Schorr:

And you had a transplant.  Where did the donor cells comes from that sort of rebooted your immune system? 

 

Don Armstrong:

That’s a great question.  I’m actually currently still looking for my donor.  My donor, he was happy.  I know he’s a male.  He was excited that he was able to help me, but I have never able to connect with him.  I’m trying again.  I just‑‑so through the Be the Match I got my stem cells, and fortunately there was someone there that was willing to give my life a second chance. 

 

Andrew Schorr:

Now, Don is very active with the Leukemia & Lymphoma Society, which is a partner in this program.  Don, you’ve spent many years now, your career has been in golf and you were a golf superintendent, and yes, you sprayed pesticides on the golf course.  You’re devoted to giving back.  Talk the a little bit about that and what you try to tell patients and families so they can get through this hopefully successfully, as you have. 

 

Don Armstrong:

You know, like we had talked about, I had no idea there was anything wrong with me when I found out I had leukemia, and I was in for a pretty big fight of my life.  After five rounds of chemo and a stem cell transplant, making it through that, I felt like I had been given a second chance, so I wanted to find a way to give back.  And I found the Leukemia & Lymphoma Society.  I found one of their campaigns, Team in Training.  Started doing the endurance events and raising money for that organization.  Just did my 2015 retraining event, but through it all I really wasn’t doing it just to run a marathon.  I was doing it to raise money for research so that somebody else hopefully didn’t have to go through what I went through.  So for me that was a big shift in my mindset.  That eventually led me into being a part of the board of trustees, and I continued along that line.  And I try to stay up on as much about AML as I can, and I talk to as many patients around the country as I can whenever I’m given the opportunity. 

 

Andrew Schorr:

Well, you’re going to hear a lot today, and research has paid off.  Let’s go up to Colorado outside Denver where Amanda Fowler is an information specialist with the Leukemia & Lymphoma Society.  And that means if you call, and I urge you to, whether a patient or a family member, they can help because based on research the world of AML has changed significantly and gives people a great deal more help and is changing those statistics that Don talked about.  Amanda, welcome to the program. 

 

Amanda Fowler:

Thank you it so much for having me. 

 

Andrew Schorr:

So I’m right.  Research has been paying off just in the last year or two and even with more research coming our way, right? 

 

Amanda Fowler:

It’s incredible.  There have been drug approvals in the last two years.  There are numerous clinical trials.  We are really seeing AML treatment change for the better at a pretty rapid pace right now. 

 

Andrew Schorr:

Okay.  So just to be clear, if someone who is watching now calls the Leukemia Society like the national number, and maybe you can tell us again, how do they get to you so you can help them sort this out? 

 

Amanda Fowler:

Absolutely.  It’s really easy to reach an information specialist.  We are open 9 a.m. to 9 p.m. eastern time and our number, which we can repeat at any time, is 1‑800‑955‑4572, and that will take you straight to an information specialist.  And once you reach us we’re really there to talk and figure out exactly what you need. 

So if you’re not sure if you need help or not, give us a call anyway, and we can go over a lot of the resources and services from psychosocial support to financial resources, disease education.  We have a clinical trial support center, so a whole variety of resources, and we encourage people when in doubt to reach out to us. 

 

Andrew Schorr:

Okay.  All right.  Let’s hear about the research.  So joining us from Portland, Oregon, at Oregon Health & Science University is Dr. Uma Borate, who is a hematologist‑oncologist.  Dr. Borate, thanks for being with us. 

 

Dr. Borate:

Absolutely.  Thank you for having me. 

 

Andrew Schorr:

Okay.  So we’ve alluded to changes in AML, payoffs in research.  You’re still in the lab moving research forward with your peers around the world.  You all are making progress, am I right?  The world and the options, the combinations, things you’re researching, that’s all changing incredibly fast. 

 

Dr. Borate:

It absolutely is.  And I just wanted to first just appreciate Don and his journey because we see patients like him every day, and we deliver these very shocking and stressful diagnoses to patients and kind of see the journey of absorbing what this means for them.  The family rallying around them.  And then the treatment and the eventual, you know, the role that takes them hopefully to a cure.  And I just‑‑every day when I talk to my patients I just applaud their courage.  So, Don, I just wanted to put that out there.  You guys are awesome. 

With that said, I think in the last I would say four to five years, and Amanda can attest to this, we’ve had over nine FDA‑approved therapies for AML after about four decades of no progress.  And a lot of this has come with discoveries in the lab where we have identified specific genes that have had genetic changes, what we call genetic mutations, that lead to a patient developing AML. 

And now we have what we call targeted therapies where we can target that specific genetic mutation and therefor destroy the AML cells in a way that doesn’t expose the patient to a lot of additional toxicities.  However, for a certain subset of the AML patients, like Don, we know that these targeted therapies can work for a while, but if you are going for what we call a curative active therapy, for a lot of patients transplant is still right now one of the most, I think, advocated and proven curative therapies out there. 

 

Andrew Schorr:

Okay.  So let’s talk about testing.  So somebody is diagnosed with AML.  What should happen now so that you as a specialist, the doctor, the team that they see, they know what version of AML you have and whether it matches up with either one of these approved therapies or maybe something you at an academic medical center are researching that could be the drug of tomorrow? 

 

Dr. Borate:

So I think that’s a great question, and for patients or family members or anybody else listening out there I think what you a alluded to as the most crucial step in the diagnosis of AML and the subtype of AML is the testing.  So as soon as we identify a patient, like Don described, typically it’s, you know, you don’t feel well.  You go to somebody, they say, oh, my god, your blood work doesn’t look right.  You go to another doctor. 

The first thing that we ask that happens is when a bone marrow biopsy, which is the diagnostic procedure that gives you the diagnosis of AML is done, that it be subjected to adequate genetic testing.  And by this I mean there are many, many laboratories out there that do what we call expanded genetic mutational panels, and they test for all the different genes that could have a mutation that potentially could be targeted or make the patient a candidate for a clinical trial in the future. 

Leukemia & Lymphoma Society is sponsoring an extremely revolutionary trial called BDML, which we are a part of, which does this testing and returns the results back to the clinical provider or the physician in seven days, which is really unheard of in terms of a timeline.  This used to take about two weeks on average. 

And so in seven days I know all the genetic changes in my patient’s leukemia and I can determine, hey, is this the right therapy for them?  Should they go on a clinical trial that we have?  And BDML offers several of what we call (?) Inaudible that are used to match them to the appropriate clinical trial or the appropriate drug.

 

Andrew Schorr:

Okay.  So, Amanda, let’s talk about this for a second.  So, oh, my god, a patient is diagnosed with this acute condition.  Don found himself in the hospital right away, and I understand there can be different versions of AML.  Some like do not pass go, boom, you’re going to the emergency room at the hospital right away, or some there’s a little more time.  But a call comes to you, and people want to feel confident that where they are or where‑‑do they go to this hospital or that hospital, this clinic or that one.  They get someone who is knowledgeable when the whole world of AML has been changing. 

So how do you counsel people so they get the right testing and just all the range of options are considered for them? 

 

Amanda Fowler:

Sure.  Absolutely.  So I should say a lot of the calls, particularly for the acute leukemias, actually come not from the patient but from the caregiver.  The patient is often sick and overwhelmed, and it’s a family member who is making the calls.  Of course we are having to talk to both the patient or the caregiver, but it is really important to be at a center of excellence for this diagnosis. 

Not all hospitals are equipped to handle AML.  If you’re lucky, a local hospital will see that and send you on to the bigger center, but sometimes that work does fall on the family member.  We recommend that people go to university‑type settings or National Cancer Institute comprehensive cancer centers.  These are going to be the larger hospitals that understand what tests are involved.  They will have clinical trials as options, and they will be knowledgeable on the latest treatment options. 

 

Andrew Schorr:

Right.  So, Don, just to you.  You talk to patients, and you speak around the country.  It takes a lot of courage for a patient or family member to say, thank you, Doctor, but I think we’re going to go, take mom or dad, we’re going to go over there because, you know, I mean, hospitals are competitive, right? 

 

Don Armstrong:

Absolutely. 

 

Andrew Schorr:

But you and the family want to make sure that your loved one gets state‑of‑the‑art care.  What would you say to them to be a good consumer? 

 

Don Armstrong:

Well, I agree with what Amanda said.  I think it’s important that you’re in the right hospital, the right setting because not all the hospitals can actually handle the diagnosis of AML.  So I think it’s really important that you’re in a center that can treat that, that’s got experience treating it.  And so I always tell patients call the Leukemia & Lymphoma Society’s IRC and find out where is the best place to go. 

 

Andrew Schorr:

And here’s Amanda.  And, Amanda, you have a medical advisory board.  You keep lists of AML specialists, right?  So you can actually say where in where you live, in Kansas, in California, in Texas, etc., here are centers of excellence, right? 

 

Amanda Fowler:

Yes, absolutely.  We will talk to patients about where to go, wherever it is that they live.  And then occasionally if it’s later on in the treatment and they’re considering transplant they may even look to travel, and we’ll help discuss any option that they want to get them to the center of excellence. 

 

Andrew Schorr:

Okay.  All right.  Dr. Borate, let’s go back to the basics just for a second because we have people who are trying to understand what went wrong.  I’m a leukemia patients too, but with chronic lymphocytic leukemia, but I know that our bone marrow often in our hips and bones is the blood factory.  What went wrong in that blood factory, and how does it show up in AML? 

 

Dr. Borate:

So, thank you, Andrew.  I think that’s the so‑called million dollar questions is we know that there’s a combination of factors that can cause what we call these genetic mutations that then go on to lead to the actual disease. 

So whether it be CLL, which you alluded to, or AML, age is a big factor.  So as all of us grow older the unfortunate reality is as our cells divide they accumulate genetic changes that (?) Inaudible repair, so that’s one thing that happens to all of us.  Environmental and genetic factors play a big role, and I think the new emerging field in this is what patients would ask us, why did I get this?  We would say, well, you were unlucky.  You had this mutation.  Something happened. 

But now we know about 10 to 15 percent of leukemias actually have a genetic or what we call an inherited component.  So if you talk to patients they would have‑‑some patients have a very strong family history, not just of leukemias or lymphomas, which are blood cancers, but other cancers.  And I think it’s really important to nail that down and explore the inherited aspects because for patients like you or Don, if you have kids and grandchildren, you know, those have far‑reaching implications down the road. 

However, 85 percent of these leukemias are what we call sporadic, meaning they just came about because of environmental and genetic factors that sort of played a role in one or two cells developing the mutation and then there is a competitive advantage for these cells.  They start growing, you know, without any checks and balances, and once that happens they start crowding out the healthy cells in your marrow and they sort of replace, as you said, the nice, healthy cells in your bone marrow that should be making your red blood cells, white blood cells and platelets. 

And sometimes the analogy that’s given is you see these weeks on a lawn and once the weeds start growing they kind of take over all the healthy grass because they compete for nutrients and water, and then all you get is a lawn full of weeds. 

 

Andrew Schorr:

Okay.  So somebody comes to the emergency room, like Don looked sick.  Is it fatigue?  Is it bleeding?  Is it just what‑‑how do people present, as you doctors say? 

 

Dr. Borate:

So typically patients present with this feeling that they’re not‑‑they just don’t feel well.  Typically it’s fatigue.  Sometimes they’ll notice bruising or spots all over their bodies.  They’ll notice that their gums are bleeding easily when they brush their teeth or they have nosebleeds when they’ve never had them before. 

A fair number of patients actually present with an infection, so a sew throat that doesn’t seem to go away.  They get swabbed for mono, and the practitioner sees these weird cells in their blood, and they think, well, maybe this is mono because they’ve had fatigue, sore throat and some lymph nodes, and so that’s the way people present. 

Sometimes people present really sick, with a pneumonia or another infection, and then that’s when you go to the ER and you come into the hospital and it’s like, oh, wow something else is going on. 

 

Andrew Schorr:

Okay.  So they get to you, let’s say, at Oregon Health and Sciences in Portland, a major university center, and you run this genetic panel.  Now, it’s seven days.  So, first, what’s going to happen while you’re trying to figure out what version of AML they have and whether you have a therapy. 

 

Dr. Borate:

Yes. 

 

Andrew Schorr:

So let’s just talk about that.  What happens first, and then based on the information what happens then? 

 

Dr. Borate:

So it really depends on what you said before is how sick you are and what type of AML that you have.  So I would say even now about 30 percent of our patients don’t come through the ER and are not that sick.  They come through an outpatient clinic appointment where they’ve had low blood counts, they’ve been tested, there’s some testing that is done which indicates they may have leukemia, and they actually come to my clinic. 

And if they come to my clinic and they’re relatively what we call stable we will perform all this workup, as we call it, as an outpatient.  So we’ll to the bone marrow biopsy as an outpatient.  We will let them be at home for those seven days so they can sort of start preparing for, hey, maybe there’s something going on and this will need some length of treatment in the hospital or multiple visits to the physician, in which case if you’re working you need to start thinking about time off and preparing, and if you have kids how to they get to their activities.  So all the different things that people are struggling with when they get this diagnosis.  So that is about 30 percent of patients.

And in those seven days while they’re waiting, sometimes it’s longer, we keep a really close eye on their blood work.  So if they live close to a hospital or a clinic we make sure they go to the clinic at least two or three times a week to see what their white blood cells, platelets and red blood cells are doing, and then we get those results.  So we monitor them before they come back to get the final diagnosis and what their position is. 

If it’s somebody like Don who ended up in the hospital really sick, then they stay in the hospital while we’re doing this testing.  Typically they will get blood.  They will get platelets.  They would get what we call a workup, meaning we will check their heart, their kidneys, their liver.  We would put what we call a central line, meaning a line or an IV that can stay in their bodies for a longer length of time that can allow them to get treatment and allow them to get blood work and transfusions.  So all this is happening in the background while we are figuring out the subtype of AML. 

The other thing that we also do at that time is we collect what we call HLA typing, and this is to figure out what the tissue type of the patient is.  So like Don, when he went on to the transplant it’s really important for us to know this beforehand.  So while the patients are getting treatment in the hospital we can see if they have matches.  So does your brother or sister, can they be a match for you to donate bone marrow, or does it have to be somebody through Be the Match, as Don said, would that‑‑would it be what we call an unrelated but matched donor that would then be an option for you in the future. 

 

Andrew Schorr:

Okay.  So now the testing comes back.  This next generation sequencing, which is so cool now to see your cancer genes, what cancer genes are active, what could be driving your acute myeloid leukemia, and it says this gene.  And I know they have a lot of different letters, IDH, FLT3.  You could probably name a whole bunch others that different drugs have been developed for.  So it comes back with this letters and you say, ah‑ha, if we have a drug that targets that we can tamp this down.  Right? 

 

Dr. Borate:

Yep. 

 

Andrew Schorr:

Are these drugs infused?  Are they pills?  My understanding is now you have some pills that people can take as well. 

 

Dr. Borate:

So I think that’s one of the really cool things moving forward is most of these new targeted agents are actually oral, so medications, so pills that patients can even take at home to treat their leukemia. 

So just to back up a little bit, once we get back this genetic testing and we know their mutations and like you said IDH1, IDH2, FLT3, these are all mutations that can be targeted, we also determine a little bit‑‑and this can be somewhat arbitrary but is determined more by the patient, their age, their ability to, you know, how able are they to do their day‑to day activities?  Are they somebody who really is not even able to go to the grocery store without being really tired?  We call it, for lack of a better word, performance status.  How do they do in their everyday life? 

So we take all these factors to consider two broad categories:  Is the patient what we call fit versus, and I know this is not the kindest word, we call it unfit.  And I think those broad categories then lead us to what type of therapy should the patient get.  Should they get what we call intensive induction, meaning we still give them very broad chemotherapy to kill all the leukemia, but now we’re adding targeted therapy to the chemotherapy so that you give this double‑whammy?  You knock them with chemo, and you knock it also with the targeted therapy. 

However, if you happen to be 85 and you’re a very functional 85, maybe, but you’re not somebody whose organs can tolerate this heavy intensive chemotherapy or a transplant in the future, then we go with what we call more therapy that’s what we call less intense even though it might be IV, but then we add these targeted agents which they can take at home as a pill and then they’re not in the hospital as much.  They get this therapy as an outpatient while they’re getting treatment for their AML.  So it’s very different based on our goals of care, the patient in front of us and what mutations they have. 

 

Andrew Schorr:

Okay.  So increasingly now you may‑‑maybe somebody would get some chemo, working on approaches where you don’t lose your hair. 

 

Dr. Borate:

Yes. 

 

Andrew Schorr:

You don’t develop mouth sores. 

 

Dr. Borate:

Yes. 

 

Andrew Schorr:

Or ad nauseam.  You’re working a lot on lowering the toxicity. 

 

Dr. Borate:

Correct. 

 

Andrew Schorr:

And, Don, I know you went through that you’re being prepared for a transplant, heavy‑duty.  But that’s been ameliorated to a greet degree for many people. 

 

Dr. Borate:

Yes. 

 

Andrew Schorr:

And then somebody may be on a pill.  Now, do you have a growing range of treatments so if you try one and it doesn’t work or it doesn’t last you have something else?  It’s kind of like an antibiotic.  The doctor says we’re going to try this antibiotic.  No.  Your swollen glands and lymph nodes, it’s not going away.  Let’s go to this one. 

 

Dr. Borate:

We absolutely do.  And I think that’s sort of the next frontier is what we call sequencing.  How do you sequence treatments so that you can continue to get a good response even when the patient fails a treatment and the leukemia decides or figures out how to outsmart that treatment?  And unfortunately that still happens even with targeted therapies that over time the leukemia figures out a way to survive despite a very targeted approach.  And so how do you come back in with a different drug that can still work?  And how do you sequence those drugs to give them maximum effect but the least toxicity as you said, to the patient?  And those are sort of our next frontiers of clinical trials and therapy. 

 

Andrew Schorr:

So at this interim stage, Dr. Borate, one key question:  Don talked about the statistics, which were not good when he was diagnosed. 

 

Dr. Borate:

Yeah. 

 

Andrew Schorr:

Are you seeing a change in quality of life, and you believe in survival based on everything you’re talking about? 

 

Dr. Borate:

Yep.  I’ve seen a huge change in quality of life.  And I will say interestingly in AML more than any other disease we have really pushed this aspect in our older patients.  Because we have heard loud and clear from patients who are 70, 75, 80, that they want to live.  They want to live as long as possible, but they also don’t want to spend all that time in a doctor’s waiting room in or the hospital.  They are very, very determined to have a good quality of life and enjoy whatever it is that they want to do. 

And I think we have really worked hard to deliver that with our targeted therapies, and I want to say the results are astounding.  I have an 89‑year‑old right now who celebrated his birthday and has been outpatient for the last year since his diagnosis.  An 85‑‑oh, an 86‑year‑old, and we celebrated her birthday on (?) Inaudible where she took a pill for 13 months and is in remission and is talking about taking a trip to Bolivia. 

So to me these are huge success stories for my patients because I have a soft corner for my older patients.  They have struggled, they have sort of supported their kids their whole life, and it’s their time now.  And I think that’s so important to deliver that to them. 

 

Andrew Schorr:

Well, Dr. Borate, first of all, thank you for your devotion to patients.  We’re going to talk more about treatments.  We’re going to be taking your questions from our audience along the way, aml@patientpower.info. 

Don, you have been in Team in Training with (?) Inaudible, run I don’t know how many marathons, and it’s for research.  This must make you feel good that research is paying off. 

 

Don Armstrong:

On this side of the screen I am smiling so, so huge, and I’m actually very emotional about it.  It’s great to hear what Dr. Borate is saying because it gives other people a lot of hope and encouragement.  It’s just great to hear because it was not an experience that I would want anyone to go through, and so these are big moments.  So thank you, Dr. Borate, for what you’re doing. 

 

Andrew Schorr:

And if mom and dad want to go Bolivia or Thailand or Europe or wherever it is their time, as you say, Dr. Borate. 

 

Dr. Borate:

It is their time. 

 

Andrew Schorr:

So, Amanda, let me go to you.  So the Leukemia & Lymphoma Society has lots of services.  Some of it‑‑and your cat’s going to help us too, there‑‑is first of all getting information.  Where can I or mom or dad or grandma or grandpa, Uncle Charlie get the right care, okay, state‑of‑the‑art care?  But also then as we get to some of these treatments then they say, oh, my god.  There’s all this medical care and medicines coming in, and there’s expense.  So you all help people with that, too, right? 

 

Amanda Fowler:

Yes, absolutely.  We hear all the time how expensive treatment can be, and these new drugs are amazing but they do come at a cost.  So we have various different options to help patients through a program like copayment assistance that’s available most of the time but is subject to availability.  So we encourage people to call us.  And if there’s something that we don’t have we will work with the patient to find other options. 

It is a big burden to people, and it’s certainly one of the things that people worry about the most.  And this does often come from the caregiver because, like we said, the patient may be feeling pretty ill or be in the hospital, so we just encourage you to call and see what’s available. 

 

Andrew Schorr:

Okay.  Yes, I know.  I take an oral therapy for another leukemia, chronic leukemia, and I’m on Medicare, and I have Medicare Part D and I have a substantial co‑pay.  Now there are foundations, the Leukemia Society, that depending upon your need can help.  And if you’re on commercial insurance and you’re younger and not on Medicare there are other programs that come into play, right, Amanda? 

 

Amanda Fowler:

Yeah.  We will talk to patients, whether it’s through a nonprofit like us or through the pharmaceutical company directly.  And I certainly encourage people when they’re making that transition to Medicare, which often they know about in advance, to call us and we can help talk to you about ways to make that transition a little bit easier because you do find that the cost out of pocket to the patient can sometimes go up when they make that transition. 

 

Andrew Schorr:

Okay.  Dr. Borate, I have questions for you.  Maybe you is tilt your screen down a little.  We’re just losing you.  There we go.  There, that’s good. 

Dr. Borate, so you mentioned clinical trials.  We talked about research.  So you’re doing clinical trials, and many of your peers at other academic medical centers are doing clinical trials, and that’s what led to the approval of these drugs by the FDA based on data that you as researchers and drug companies and National Cancer Institute were able to present.  So talk to us a little bit about what’s in the lab, if you will, that you may be offering patients in clinical trials.  And then we’ll also understand, Amanda, when somebody is in a clinical trial what costs may be covered, too, okay?

So, Dr. Borate, first, what’s going on in research? 

 

Dr. Borate:

So I think, as you said, clinical trials really pave the way to new therapies.  And again I want to have a special acknowledgment and shout‑out to all the patients and their families who participate in these studies because it is a little bit of a leap of faith.  You know, sometimes we’re not always sure these treatments will work and they have side effects, so for patients to put their trust in us and sign off for these studies is I think a big deal.  So thank you. 

When we start somebody on a clinical trial we always collect what we call a pretreatment sample.  So we’ll get a sample of their disease before they’ve had any treatment, and then along the way as this treatment progresses we get multiple what we call post‑treatment treatment samples, one to look at the status of their disease, and secondly to send the sample then back to the lab to understand how these new treatments are affecting the disease.  You know, what pathways in these leukemia cells are being inhibited so that the cells are dying?  What pathways are deactivated, which also helps the cell to die? 

And then thirdly, what pathways are being sort of turned on to help the cell resist these treatments.  We call them mechanism of resistance and it’s similar to the antibiotic analogy you said where you take an antibiotic for a while and it seems to be working initially but then your body develops resistance to it and so the provider or the doctor has to change therapy because now this drug no longer works for your infection. 

And so the same thing happens with leukemia or any cancer, and I think it’s very important for us to observe the samples as the patient progresses through therapy so we can figure out, first of all, why it worked, but also why did it stop working or why did it not work.  And I think that’s where participation in clinical trials is so critical because without this valuable information we really can’t move the field forward. 

 

Andrew Schorr:

Okay.  Amanda, just about costs.  So if somebody signs up for a clinical trial might some of the costs be covered? 

 

Amanda Fowler:

Yeah.  It’s actually a bit of a complicated question, but generally speaking the cost of the actual trial drug is covered through the trial itself.  But it is important to talk to your insurance provider because hospital stays and some other supportive care may still go through your insurance. 

 

Andrew Schorr:

Okay.  So it’s complicated, but it’s something to discuss.  Dr. Borate, one other thing about testing.  So we mentioned somebody gets to the hospital, and ideally there’s this panel, this next‑generation sequencing done, fast track trying to get the results back to the specialist to decide do you have a targeted therapy or what’s going on, what’s your version of AML. 

But cancer is wily, and the cells can change, and the cancer gene that was driving your AML on day one could be different on day 50 or 100. 

 

Dr. Borate:

Absolutely. 

 

Andrew Schorr:

You would know the (?) numerical better than I.  So is retesting sometimes needed if something changes? 

 

Dr. Borate:

Yep.  So I think this is a great question, Andrew, and I think it ties into what Amanda said about centers of excellence because this is something we routinely do in our AML patients every time.  We sort of look back and see what their disease is doing, and we call it re-staging.  And sometimes we see certain genetic changes or mutations come in even when the patient is in remission.  When we can’t see the AML we can see some of the background cells acquire different mutations so we can keep a closer eye on the patient as they continue down the road.  So I think retesting is one of those things that is not done enough if you don’t have experience with the disease. 

Because you’re absolutely right.  The disease is wily.  It does change.  The mutation that was driving your disease to begin with may not be the one driving it when it relapses, and I think that’s really where we need more information, and we need to have this testing done in order to treat the patient appropriately. 

 

Andrew Schorr:

Okay.  So let’s go back to what leads to AML for a minute.  So Don, you worked on golf courses your whole life and eventually became the superintendent of one of the more famous PGA golf courses.  Colonial, is that right? 

 

Don Armstrong:

Correct, yes. 

 

Andrew Schorr:

In Fort Worth.  But over your years devoted to golf you sprayed a lot of pesticide, right? 

 

Don Armstrong:

I did.  I did.  From the time I was 15 years old I get my first job on a golf course and the superintendent knew I wanted to be in the industry, and so he let me do a lot of things that probably somebody else wouldn’t.  And one of them was spray the pesticides, pray the fungicides, herbicides.  You name it, I got to spray everything.  And back in that time frame the pesticides were a lot more potent.  We had a lot of mercury‑based, lead‑based products in those time frames that the EPA had not stopped in terms of use. 

And so I think back about whether that may have had an impact on where I ended up with the leukemia.  It’s hard to say.  I’m sure Dr. Borate would probably agree with that, but it seems to me there could be some correlation, yes. 

 

Andrew Schorr:

Probably so.  So environmental factors.  Dr. Borate mentioned hereditary to some degree.  And I want to ask about another thing.  I am not just living with chronic lymphocytic leukemia but I have another blood condition called myelofibrosis.  And some people with myelofibrosis progress to AML.  My understanding was this secondary version of AML was often harder to treat. 

 

Dr. Borate:

Yes. 

 

Andrew Schorr:

And I also understand, Dr. Borate, you’re researching it. 

 

Dr. Borate:

Yes. 

 

Andrew Schorr:

So are the options, is the chance for hope for people who developed this secondary AML? 

 

Dr. Borate:

So thank you for bringing that up.  I think secondary AML has always been this sort of thing that people don’t want to touch and especially when you’re looking at clinical trials investigating your new agent because we knew that, hey, if you were treating patients with secondary AML with a newer therapy maybe the results wouldn’t be as good and then your clinical trial results overall don’t look at good. 

And to me that is a huge disservice to our AML patients because, as you describe, either because you had a prior cancer, let’s say you had breast cancer and you had chemotherapy for that breast cancer or lung cancer and you survived it, and so you’re a survivor.  And 10 years later you develop AML as a result of exposure to prior chemotherapy, which in many ways does things to your stem cells like a pesticide that Don might have been exposed to.  (?) Sorry, I apologize.

And so I think the thing that secondary AML has taught us is when a patient develops secondary AML there is a large number of genetic mutations already existing in the patient from their prior chemo, from their prior cancer, from their prior exposures.  And so because the treatment is so hard it’s something that we’re looking about very carefully in the lab to understand it much better. 

And especially about secondary AML from myelofibrosis or these diseases we call MPNs, meaning myeloproliferative neoplasms maybe essential thrombocytosis, polycythemia vera, all these big names.  We have a study here at Oregon Health and Sciences University which combines our targeted therapy.  You might have heard about it.  It’s called Jakafi is the commercial name or ruxolitinib is the pharmaceutical name, and it targets a mutation called JAK or JAK2.  And then we combine it with a chemotherapy we call, just called Vyxeos, which has also been recently approved specifically for secondary AML. 

And so we’re taking that approach I describe where we’re combining the therapy that’s already on the market for secondary AML and has shown benefit, but then we’re adding this targeted agent which is also on the market for myelofibrosis but they haven’t ever been combined together.  But we’re doing it in a way that’s slow and careful and cautious because, you know, we have to talk about safety, and we want our patients to get the benefit but not the toxicity.  And we want to see what this does.  The response rates for secondary AML from myelofibrosis are anywhere from zero to 15 percent, which is terrible.  And so we really want to improve on that. 

 

Andrew Schorr:

Okay.  Let’s talk about transplant again.  So, Don, you went through a transplant, and I interviewed a transplant survivor years ago who did well afterwards, but he said, Andrew, it is not a walk in the park.  Now I know there have been a lot of refinements and you had it a number of years ago now, but what was the transplant experience like? 

 

Don Armstrong:

It wasn’t a walk in the park.  As a patient I think we hear what we want to hear, and I didn’t really hear about a lot of the side effects that might occur from having a transplant because I was so focused on just surviving.  I thought I had actually skated through the GvHD, the host‑versus‑graft disease resistance, until probably day 30 or so, and I started having presentation on my skin, and I started having issues with my throat, my eyes. 

So I had to go through all those additional struggles on top of fighting the initial treatment from the leukemia.  So it was tough, and it was something that, it was‑‑it’s difficult to be prepared for that because you’ve already been through all the chemo for the leukemia.  I was grateful that the stem cells did what they were supposed to do, which was graft in my body, but the side effects were difficult.  They made it quite a challenge. 

 

Andrew Schorr:

And you continue to take some medicine related to anti‑rejection? 

 

Don Armstrong:

No, sir.  I haven’t taken anything now probably for, for probably six or seven years. 

 

Andrew Schorr:

Good.  Good.  Dr. Borate, just so we understand with a transplant.  So you’re getting somebody else’s cells to‑‑you get the chemo to knock back the disease in your body, and then you’re having somebody’s healthy cells be infused to try to take offer the immune system.  My understanding is even your blood type can change.  I’m B‑positive.  I get somebody who is O or something that becomes my blood type, right? 

 

Dr. Borate:

Yes. 

 

Andrew Schorr:

So it’s a whole rebooting and a changeover, right? 

 

Dr. Borate:

Right.  I think the one thing I do want to clarify is you mentioned you get a lot of chemo to take care of the disease in your body, but most successful transplants are actually done when you’re in remission, so when the patient is in remission.  Because the chemo that’s given is really given to destroy your own existing bone marrow and immune system and to make a home and to make place for these new donor cells that will then come in and survive.  If you did not do that then your immune system and your bone marrow would immediately reject any of those stem cells that were being given to the patient. 

If you still have disease in your body and a lot of disease, and then you get all this chemo to prepare you for a transplant, the risk of rejection or the disease coming back is actually quite high because when you‑‑when you destroy your own bone marrow including bone marrow that has disease in it and then you infuse somebody else’s stem cells, you have a period of about two to three weeks when you have no immunity.  And the stem cells are trying to grow, but if the patient still has leukemia in their body or had disease when you started this process, those disease cells just grow out of control, and they kill all the donor stem cells, and then it’s just not a good outcome. 

So we really want the patient to have as less disease, preferably no disease in their body when they get a stem cell transplant.  And this is not something that is intuitive to a lot of patients because it’s seen as a treatment for AML, which it is, but you really want to give it when the disease is really under really good control. 

 

Andrew Schorr:

Amanda, could you talk a little bit about the support programs that the LLS, the Leukemia & Lymphoma Society, has, and Don I’ll have talk about it as well, so that somebody going through this, first of all they and their family have never heard of this before.  They don’t know anybody with it.  The treatments can be significant.  Hospitalization could be long or not.  How do you know you’re not alone?  Not just calling you on the phone but there are other resources you have too. 

 

Amanda Fowler:

Sure, absolutely.  I think, like you said, just knowing you’re not alone it’s really important for people both caregivers and patients to connect with other people who have been through this.  It’s very likely until diagnosis a person may have never heard of AML.  They just have a vague idea of what leukemia is.  So we have ways to connect patients and caregivers with other people. 

We have a wonderful program called the Patti Robinson Kaufmann First Connection, and that is a telephone peer‑to‑peer connection.  So we will match you with someone of a similar age and gender who has the same diagnosis and has already been through treatment.  And they are trained volunteers.  We also have in‑person family support groups.  We have online support groups, including ones specifically for leukemias, one specifically for caregivers as well as young adults. 

We also have an online community, so if you’re not available for the live chats you can post there and connect with people all over the world who have been through a similar experience.  While every patient’s journey is really unique there’s going to be some similarities where you can know that you’re not alone.  And I think all of these technologies are great because you can even connect while you’re still in the hospital. 

 

Andrew Schorr:

And, Don, you are a First Connection volunteer.  I’ve done it too, with people with my leukemia.  So you talk to people one‑on‑one. 

 

Don Armstrong:

That’s correct.  By the way, I do want to add that my blood type did change because of my stem cell transplant. 

 

Dr. Borate:

Of course. 

 

Don Armstrong:

You’re absolutely right, yeah. 

The First Connection program is probably one of the most rewarding things that I’ve done since my treatment and making it through that.  To be able to talk to another person that’s going through something that you went through and really just answer any questions they may have is really very rewarding, and I know it’s great for the patient as well. 

When I was going through treatment it was wonderful to hear the doctor say that you’re doing well, it was wonderful to have the support from the nurses, but I really wanted to talk to somebody that had gone through is so I could say, okay, tell me about it.  What was it like?  Did you have this problem?  Did you have that problem?  So it’s just a great one‑on‑one, as Amanda said, opportunity to help answer questions and hopefully give some additional encouragement to patients. 

 

Andrew Schorr:

I want to add some additional resources.  So, first of all, the originator of this program is a group called the Patient Empowerment Network.  Their website is powerfulpatients.org in partnership with the Leukemia & Lymphoma Society.  And we’ve worked with them many times at Patient Power. 

Most recently, we did also a program at the big American Society of Hematology meeting, and that’s where all the hematologists, like Dr. Borate, from around the world come and where research is presented and they talk about it.  This year there was a lot of AML, and so we sat down and had a discussion with peers of Dr. Borate’s, and that is Dr. Kadia from MD Anderson and Drs. Lee and Dr. Ritchie from Weill Cornell in New York, other NCI cancer centers like where Dr. Borate is. 

And so I urge you to take a look at some of these programs.  If you go to patientpower.info and just go to the leukemia and then the AML area you can see the replays of these.  So that’s another resource for you. 

And then I will tell you that some people are connecting on a platform like Facebook, and there are some AML groups there.  A couple of caveats we’ll give you about the internet.  I’m sure Dr. Borate warns people about Dr. Google.  First of all, some of the information sometimes when you just search is not current.  And you’ve heard for the first part of this program things are changing, right?  And it’s very nuanced.  And Dr. Borate may have a meeting with her colleagues tomorrow, and they’ll say, oh, well, now we know this.  So there’s no way some of these services can keep up, particularly just general web searches.  So be careful about that.  That’s why you need to call Amanda because she’s staying on top of that. 

The other thing I’d say is in Facebook you may go to an AML group, which is wonderful, people talking to one another, but we’ve talked about all these different situations in AML so one person’s situation may be different from another.  So there may be general support, but remember, we talked about it at the very beginning of this program, you need to talk to your healthcare team to get a clear picture of your or your loved one’s situation and have a plan that’s, in this age of personalized medicine, is right for you. 

And I’m sure, Dr. Borate, you have people come in sometimes with no information, but often you have people who come in who somehow have wrong information, and maybe you could talk about that, about how people‑‑how can they ask the right questions to get to the right answers. 

 

Dr. Borate:

I think you’ve touched on such an important point because, as you said, patients are scared, their caregivers are scared.  They’re looking for information.  There’s information that they getting from the providers or the physicians, but obviously there is this need to help their loved one and so there is a lot of Google searching, joining groups. 

Some of the big things that we see that are difficult is there’s a lot of information about supplements and alternative therapies, which while I think absolutely can help with many, many things including fatigue, nausea, feeling of well‑being, I think you have to be careful about what that resource is and what studies have been done on it.  So I think those are questions that we get a lot and we try our best to have evidence‑based data on these different‑‑they’re like medications because they are something you’re putting into your body and while some are more natural than others, for example, turmeric is one that is used extensively by people.  They do interact with some of the other treatments you might be getting for your leukemia. 

So just understanding how your supplements are interacting with the treatments that you’re being prescribed I think is important, and there’s some resources that can actually do this in a very evidence‑based manner. 

I think going to societies like the Leukemia & Lymphoma Society, we have the ASH, as you said, American Society of Hematology, ASCO, which is the American Society for cancer, these websites‑‑I think the NCI website, the national center for, you know, all cancers, they’re just really great resources that tell you what you’re up against in a very sort of patient‑friendly way that explains the treatment. 

And I think something that you guys discussed before, the First Connection resource, I think that is something that‑‑I mean, it’s a resource that is so underutilized because even as a provider who treats AML for many years I don’t have the experience that Don had.  I didn’t go through a transplant.  I didn’t go through GvHD.  So while I can discuss side effects I can’t really present a patient’s perspective, and that’s what they are looking for a lot of times.  Just looking at a role model or reassurance that this is what a patient or somebody who looks like me went through and came out on the other side.  And, yes, it wasn’t a walk in the park, and, yes, it was awful at times, but he came out on the other side and this was what he needed to do that.  I think that is so valuable. 

 

Andrew Schorr:

Right.  Just one aside.  GvHD.  You hear all these acronyms.  Graft‑verse‑host disease, where those new cells from somebody else are fighting with your immune system.  They’re going to win because they did for Don, but it’s a fight, and you have side effects with it that could continue for a long time. 

Amanda, what questions‑‑I know it varies by where somebody is in their journey with AML, but what questions do you suggest to people that they ask their doctor or a new center that they go to so that they or mom or dad get the right treatment? 

 

Amanda Fowler:

Sure.  I think that’s actually a big part of what we do in the Information Resource Center is talk to patients about what to ask the doctor.  We can’t always answer their questions, particularly specific medical ones, but we help them get (?) Inaudible to rate that list out to what’s most important to them. 

It really does depend on the (?) fees of where they are on their treatment, but an important question, something that Dr. Borate alluded to earlier, is what the goal of treatment is.  Is my goal curative, or is my goal to extend my life for as long as I might be able to?  Because people are on that borderline sometimes of fit and unfit, and they need to understand which type of drugs the doctors are prescribing for them and why. 

And then of course the question of am I a transplant candidate, why is why not?  I encourage people to ask why because they may think I’m a great candidate and the doctor says well, actually you know, your cardiac function is very poor and you won’t be able to survive a transplant.  I think it’s good for peace of mind to understand the reasons doctors are making these decisions for them. 

 

Andrew Schorr:

Amanda, what about second opinions?  So even if you’re in a major city there could be one big hospital over here and one‑‑I think like New York.  There are more than one NCI cancer center.  So what about that?  What do you tell people about that? 

 

Amanda Fowler:

Absolutely.  So AML can be unique in that sometimes there isn’t a chance for a second opinion upon diagnosis, right?  Sometimes they go in and they need to start chemo right away.  But generally there’s next phase.  There’s a maintenance phase or there’s a transplant phase.  Great time for a second opinion.  I’m a huge believer in second opinions.

With the way treatment is evolving there’s options now.  Really there used to be, like we said, for 40 years there was one option.  Now there’s many, and so you want to be sure that you’re comfortable with your choice, comfortable with your physician because this is going to be a long journey.  No matter whether you have transplant or not this is something‑‑it’s a long relationship, and so we encourage you to call us and we can help guide you to those centers of excellence.  Even if you’re at one you may want to talk to a second one as well. 

 

Andrew Schorr:

Okay.  Dr. Borate, you are a specialist so you’re at a center of excellence.  But how do you feel if somebody or a parent or family member says, you know, we’re going to go over here and see what they say.  Are you okay with that? 

 

Dr. Borate:

So I think any physician or provider who takes care of patients should really be okay with that because we’re all in this for the same reason and that’s to make our patients better and hopefully cure them.  And I think the way we get there shouldn’t be something that you worry about what one person says versus the other.  So absolutely. 

I do agree with Amanda that sometimes for AML, and fact a majority of times for AML when the diagnosis is made time is of the essence, so unfortunately sometimes our patients don’t have the luxury of being able to go for a second opinion or get a second opinion simply because they’re so sick.  They’re in the hospital.  Their disease needs to be treated right away. 

But I completely agree, once that first step is done I think taking a pause, talking about different options with your current physician and then saying, hey, do you mind if I go to the next center?  So for example we’re close to Seattle or California.  Just taking a flight and having a conversation and usually the second opinions really reassure the patients and the caregivers that they’re on the right track, and so they can come back to their original center and continue their treatment. 

The one caveat I have to a second opinion before starting therapy‑‑or after starting therapy, rather, is once you’ve already received a therapy for AML you may not be eligible for a clinical trial for newly diagnosed AML patients.  So we do have patients that come to us after having several cycles of therapy and then want to participate, and unfortunately the way trials are designed you want to get all the information right from the beginning, and so that’s when you enroll on a study when you’re starting your treatment. 

With that said, I mean, it is what it is sometimes just because of the rapidity of the disease and the symptoms, but that is if you’re considering a clinical trial very strongly you may want to go to a center of excellence sooner rather than later.  

 

Andrew Schorr:

One other point I want to make about clinical trials, because I’ve been in two, and about second opinions actually, is don’t drive yourself crazy.  Dr. Borate, and maybe you go to another center, you go to Seattle up the road or California down the road.  And then you say oh, no, now I’m going to the Mayo Clinic and then I’m going to Northwestern and then I’m going to New York, you will go crazy, and you will probably start hearing the same thing, right?  As well as you went through a lot.  So pick a team. 

Amanda, any comment you want to make about that?  Because I’m sure you have people, they’re very stressed out. 

 

Amanda Fowler:

I agree.  There is a number of second opinions that’s too many.  But I also wanted to say on the clinical trial piece we at LLS have a wonderful service called the Clinical Trial Support Center that can help you find those trials.  It’s a team of nurses who do individualized trial searches for people. 

Sometimes that can help inform where you go.  If you’re debating between California and Seattle and our nurses narrow it down and say, you know, there’s a trial in Seattle you could be interested in, it might help inform where you actually go for your second opinion.  And you would reach them the same way you would reach me, through the Information Resource Center. 

 

Andrew Schorr:

I want to recap just a few key points for our audience, and then get some closing comments from everyone.  So, first of all, remember what you heard at the beginning.  You want to, with the help of the Leukemia & Lymphoma Society, you want to get to where they’re really knowledgeable in this changing landscape of AML.  You want to be tested or your loved one so that you know what version of AML you have.  And if things are changing now that treatment has gone on for a while, retesting to say what’s going on now, and consider all your options. 

And Amanda said it just a few minutes ago, so important, discuss your goals for treatment.  What kind of life do you want to have?  Do you want to just knock it back, take pills at home?  Do you want to go to a transplant if you’re qualified for that?  You know, these are all the things to discuss with a knowledgeable team. 

And consider a clinical trial.  And connect with Don or his peers around the country as First Connection people.  So just a few key points.  So first of all, Amanda, what do you want to leave people with?  Probably call the LLS, right?  Call. 

 

Amanda Fowler:

Absolutely.  You know, if you have any questions give us a call.  We’re happy to help.  We’re there to talk to you and help find those important resources that you need.  And also just what a hopeful time it is for AML.  When I started at LLS there had been nothing, and now, as Dr. Borate has said, there’s nine new drugs and (?) evolving quicker than we can almost keep track.  So it’s a really hopeful time right now. 

 

Andrew Schorr:

Okay.  Dr. Borate, first of all, we all want to thank you to you and your peers around the world who are doing research, but a final comment you want to say to this audience and family members and patients who are so worried about this diagnosis. 

 

Dr. Borate:

I do want to echo what other folks have said.  This is a specialized disease, so reaching out to a center that has experience is I think critical.  Even though we might not be able to physically travel at least getting advice from that center, whether it be through your physician or in‑person. 

Secondly, I do want to say participating in clinical trials is a wonderful way not just to get treatment but also to get access to new drugs that could benefit you.  And the other part of a clinical trial that’s never discussed is just by being in a clinical trial you are monitored way more closely and observed way more closely than if you were not on one just because that’s what a clinical trial mandates us to do.  And I think the experience of our patients who are on it is always satisfying, whether the trial is helpful or not.  Just the close connection and the follow‑up that they get is just‑‑it’s a great way to be supported through your therapy. 

And the last point I want to make is something that Don said.  You need a support system.  You need your family members.  Don’t be shy about calling your sister in Florida or your father or mother that live half‑way across the country from you.  This is what family is for.  Surround yourself with your family and friends.  You need it.  You don’t want to do this alone.

 

Andrew Schorr:

Right.  And also remember for you as a family member, there is support for you too.  Leukemia & Lymphoma Society can help.  There are other groups.  The cancer support community on Patient Power.  There’s a care partner section and even with First Connection volunteers there may be some who can help the family as well.

Amanda, thank you so much for the work you do.  Don, final comment from you because here you were treated in 2005 and then took medicines for a while, and you get to speak, but you learned lot today.  Hearing all this what do you want to say to our audience? 

 

Don Armstrong:

I just want to say just be encouraged by all the great new technology that’s come down in the last several years.  Be encouraged by that.  I also want to add to ask a lot of questions.  Make sure that the time you spend with your doctor is quality time.  Don’t let the doctor leave without a question being answered that’s on your mind.  And third, use the resources that are out there that can really benefit you like the Leukemia Lymphoma Society IRC. 

 

Andrew Schorr:

All great advice.  I want to thank everybody for sticking with us.  Remember, there will be a replay of this program.  There will be all sorts of video clips.  There will be a transcript.  All that coming your way.  So look for that. 

Also look for these earlier programs that we’ve produced with support from the Patient Empowerment Network and with the Leukemia Society that really will even broaden your knowledge. 

But we had some great information today.  I want to thank our guests.  I want to thank you for being with us.  I want to thank the companies that have been devoted to research with the physicians like Dr. Borate who have been supporters of this program, Celgene, Daiichi Sankyo, Jazz Pharmaceuticals and Novartis for their support and devotion to the leukemia community. 

And remember, please consider clinical trials in this fast‑changing field as they’re learning to combine medicines.  Does that help you or mom or dad, grandma or grandpa, so they can live a longer, better life?  In Los Angeles with our friends who have been in Colorado and in Texas and in Portland, Oregon, and wherever you are, I’m Andrew Schorr. 

Remember, knowledge can be the best medicine of all. 


Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.