LC Treatments and Clinical Trials Archives

When it comes to treatment, lung cancer 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 Lung Cancer Treatments and Clinical Trials from Patient Empowerment Network.

Talking To Your Family About Clinical Trial Decisions

Hearing your name and the word “cancer” in the same sentence is a world-shaking moment. After getting a cancer diagnosis, telling your family about it is another big step, one that can be fraught with as much emotion as hearing that diagnosis yourself.

Once the emotional dust has settled, talking with your family about treatment options, including clinical trials, can raise the emotional temperature again. If your family is like mine, everyone has an opinion, and is more than ready to share it. Even in families where everyone is calm about big issues like this – I question that those families exist, but I’ve heard they might – talking about clinical trials as a treatment option means being ready to field questions, and guide the conversation.

The American Cancer Society has a great set of resources for people who are assessing whether clinical trials are a good option for their treatment. I’ll use some of those as a framework for a discussion guide you can use to walk your family through your decision to explore clinical trials for your cancer:

  • Why do I want to participate in a clinical trial?
    • Your reasons can be anything from “I want to try cutting edge treatments” to “my cancer is advanced stage, and I want to throw everything but the kitchen sink at it.” The key here is to have an answer ready to this question when you discuss treatment options with your family.What are the risks?
  • What are the risks?
    • Here’s another question you’ll want to gather answers for, for yourself, before opening a conversation with your family about enrolling in a trial. Your oncology team can help you put together a risk profile for trials, and further help you target the right trials via molecular profiling of your cancer.
  • Will my insurance cover the trial?
    • Federal law requires that most insurers cover routine costs of cancer trials. However, like so much about US health insurance, the answer can still be “it depends.” There’s a great tip-sheet on the National Cancer Institute’s site that addresses this topic. You, and your family, and your oncology team, will be working together to make sure your costs are covered, either by your insurer or the trial sponsor.
  • What happens if I’m harmed by the trial – what treatment will I be entitled to?
    • Here’s another “it depends” situation. Addressing harm to trial participants is an ongoing ethics issue in the US. The key here is to review all trial enrollment documentation fully – with help from a medical ethicist or legal eagle who’s not involved with the trial, or your oncology team – and have any potential harm scenario fully spelled out, including who will address the remedy for harm, and how that remedy will be delivered.

Having solid family support is a key factor in managing cancer treatment, and in thriving as a cancer survivor. Getting your family involved in your care by talking through your options and decisions with them will give them a sense of involvement in your care, and its outcome. They can help you through the down days when side effects have you feeling punky, and celebrate the bright days with you when scans show progress against your cancer.

Curing cancer is a team sport. You, your family, and your oncologists are all on that team. Work together toward a win, which often includes unlocking the power of precision medicine via clinical trials – which can become a win for other cancer patients, too.

Clinical Trial Mythbusters: Does the Clinical Trial Process Need an Extreme Makeover?

Does the Clinical Trial Process Need an Extreme Makeover?

Clinical Trial Mythbusters: Does the Clinical Trial Process Need an Extreme Makeover? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

Many cancer patients feel that the clinical trial process is in need of a serious makeover. One of them is Jim Omel. Jim, a retired oncologist living with multiple myeloma, turned patient advocate, makes it his business to understand myeloma from the inside out. He joins this program to share his experience in clinical trials and how he learned about his vulnerabilities as a cancer patient.

Also joining the discussion is, Dr. Michael Thompson, medical director for the Early Phase Cancer Research Program at the Aurora Research Institute and an active clinical researcher developing new treatments, particularly early phase (Phase I and II) molecular biomarker-driven clinical trials.

Join us for a meeting of the minds on debunking myths around clinical trials. How are patients protected within a trial? Will I as a patient be lost in the clinical trial system? Can I select my own arm in a trial? The questions are endless and, left unanswered, contribute to the barriers to trial enrollment.


Transcript:

Andrew Schorr:

Welcome to this Patient Empowerment Network program. I’m Andrew Schorr from Patient Power. I’m joining you from near San Diego, Carlsbad, California, and I’m so excited about this program, Does the Clinical Trial Process Need an Extreme Makeover? Having been in a clinical trial, and I’ll talk about my experience in a little while. I am a big fan, but I know that people have concerns, and I know that the percentage of cancer patients who are in clinical trials among adults is very low. How does that affect drug development and having the chance to get closer to cures for us?

I want to thank the financial sponsors for this program who provided assistance to the Patient Empowerment Network. They are Celgene Corporation, Astellas and Novartis. They have no editorial control, so what happens in the next hour is what we say, the questions you ask, what we hear from our experts who are joining us.

If you have a question, send it in to questions@patientpower.info. Again, if you have a question, send it in to questions@patientpower.info, and our wonderful producer Tamara will take a look at it, forward it to me, and as we can over the next hour we’ll be discussing questions you have already sent in. And we’ll have a very inspiring, I think, and provocative dialogue between our experts.

So let’s meet them. I want to take you to Grand Island, Nebraska, where my dear friend Jim Omel is there. He’s a retired now family practice physician. And, Jim, for years you’ve been a myeloma patient. When were you diagnosed with myeloma, and what’s happened along the way? You’re taking regular treatment now, I think, some treatment for the bone complications. How are you doing, and when were you diagnosed?

Jim Omel:

Andrew, I was diagnosed in 1997. It started off with a plasma cytoma at T10. I broke my back, I underwent a stem cell transplant in 2000 and had six years of remission. It came back in 2006, and I had radiation and lenalidomide (Revlimid), and it went away a while. Came back again in 2010, and I had radiation, bortezomib (Velcade), Revlimid, dex, and it went into remission. And since then, Andrew, I’ve been so fortunate that all I’ve been taking is bone-protective bisphosphonates.

Andrew Schorr:

Oh, good for you. Now, you were in a trial, but you decided not to continue, but yet you’re a believer in trials.

Jim Omel:

Oh, absolutely. Without trials our treatment wouldn’t change. When I had a full evaluation at Arkansas they suggested that I join their trial, and I did, and at the end of that trial was a tandem transplant. And I got to thinking and reading, and I didn’t really want to get that extent of treatment. I had a single transplant, and I dropped out of the trial. And that’s one of the things that I would certainly tell our listeners, that they can stop a trial at any time. They’re not bound to it. Ever since then, Andrew, I’ve had the good fortune of having fairly responsive myeloma, and when I had my treatments they responded to standard therapy. I certainly would have rejoined another trial if necessary, but I was fortunate that it responded the way it did.

Andrew Schorr:

Okay. And before we meet our next guest, I just wanted you to list some of the committees you’re on, because you’re very active locally and nationally on behalf of patients. So what are some of those activities you’re doing?

Jim Omel:

Well, I’ve been doing this since about 2000, so that involves a lot of activity. Peer review with the NCI was one of my main ways to get started.

Andrew Schorr:

National Cancer Institute.

Jim Omel:

Yes, and I progressed on to the Board of Scientific Advisors, which was a really good, important work with the director of the NCI. I’ve been an FDA patient representative for many years and was on the advisory board that brought Kyprolis or carfilzomib to us. I spend a lot of time each month for sure with the Alliance Cooperative Group working with Paul Richardson as we bring you new trials to patients. I’ve been with CINBR, Center for National Bone Marrow Transplant research for several years, several advisory boards. I’m on two pharma accompany advisory boards as they seek patient input.

Andrew Schorr:

Wow. All right. Well, the point of this, what I wanted our viewers to get, is that Jim is—trained as a physician, worked many years as a family physician, became a patient, eventually had to retire. He’s been through a lot of treatment and is very much an advocate for all of us, particularly in this process of trials. So we’re going to talk about the unvarnished truth about trials and see how we can make it better. Okay.

Let’s skip over to Milwaukee, Wisconsin, where we’re joined by Dr. Mike Thomson, who is very involved in research, and Mike has been very involved in all sorts of programs related to education. So, Mike, first of all, welcome to the program, and tell us a little bit about your involvement both locally in research and in education of other physicians nationwide and worldwide.

Dr. Thompson:

Sure. So not as impressive as Jim, but he’s one of my heroes who has really dedicated himself to improving the clinical trials process. I have an MD, PhD. My PhD is in pharmacology, and I was interested in pharmacogenetics and how individuals vary in their response to drugs, especially cancer drugs. I did my fellowship at MD Anderson and worked with a lot of myeloma doctors there and have worked in the community setting seven years in one place and about five years now where I’m located at and Aurora Healthcare in Milwaukee. I have been on the NCI Myeloma Steering Committee. I’m currently on the NCI lymphoma steering committee. I helped organize the ASCO 2016 meeting. I was the Chair of Education. As of June, I’m one of the editors for cancer.net around myeloma, so taking over from Paul Richardson who did that. So I’ll have about three years doing that and probably asking people like Jim for help to provide educational materials for people. And in the world of myeloma, I’ve created the MMSM or Multiple Myeloma Social Media hashtag to have Twitter chats, which I know some people don’t think are the optimal form of communication, but it is a way to get information out from experts and some opportunity for patients to ask questions. So I’ve been highly involved in social media, highly involved in the NCI and NCORP for increasing access to clinical trials in the community. And right now I am in the middle of an NCI designated clinical trial called EAA172 for multiple myeloma, which has gone through ECOG Executive Committee, the NCI Myeloma Steering Committee, and now we’re discussing with the companies and with Ctap how to bring that forward. And I think that’s—one of the things is how much effort it takes to bring some of these trials from concept to activation.

Andrew Schorr:

Okay. Now, we’ve mentioned this more rare cancer, multiple myeloma, not rare if you have it, but Jim has it, Mike specializes in it a lot. But what we’re talking about applies to the clinical trial process about broadly. So we may have people with us living with lung cancer and hoping to live longer and better, prostate cancer, chronic lymphocytic leukemia, like me, are also myelofibrosis. I’m a two-fer, if you will. There may be many different cancers among our audience, and the process applies to all. So we’re going to talk about that. So whatever it is, ask your questions, questions@patientpower.info. I’m just going to share a little personal story for a second, because I’m very passionate about it, and I wanted to mention it. And this is part of our Clinical Trials MythBusters series, and we have previous programs on Patient Power with lung cancer experts, experts in other conditions about the clinical trial process, so look that up on patientpower.info. There will be a replay of today’s program and also a downloadable guide with highlights that you can share, talk about it with your doctor, with other patients, with people you know and for your review. Okay.

So now my own story. I was diagnosed with chronic lymphocytic leukemia, the most common adult leukemia, in 1996—terrified, had no idea what it was. Didn’t know anything about what a trial was, didn’t know what the treatments were. Quite frankly, thought I’d be dead like within a week. I didn’t know. And so you start getting educated, and eventually that led to me connecting with academic medicine specialists and ultimately suggestion at the appropriate time of being in a Phase II clinical trial. I didn’t know what the phases were, we may talk about that along the way, and it was 2,000 miles from my house. So I traveled a number of times to be in that trial, and I had my local oncologist collaborating on that. And the end result was I had a 17-year remission. I had treatment again for chronic lymphocytic leukemia. It wasn’t until last year, 17 years. And I got the combination of medicines 10 years before that combination was approved. So I’m a believer.

The second thing I’d say about trials was I was in a second trial along the way, and I had deep vein thrombosis, blockages in the veins in my legs, for a blood thinner trial. And by being observed in that trial, that led to them discovering a second cancer which was at work related to those clots, myelofibrosis, and I was observed, so I liked the attention. It had nothing to do with what they were testing. It had to do with the observation you get. So, again, I love the attention of being in a trial. It may give you access to tomorrow’s medicine today, but there are things that may be broken. So, Jim, let’s start with that. Jim, what has been some of the frustration points for you the way the process has been today?

Jim Omel:

Well, I think one of the main things, Andrew, is that clinical trials tend to be designed to answer scientific questions. I think what they should do is be patient friendly. I think they should be designed to help patients. If you ask any researcher, what is the purpose of the scientific trial, clinical trial, they will say, to answer a question. If you ask a patient, they’ll think the purpose of the trial is to help patients. The—it may seem like a minor point, but it’s not. Patients need to be the center of them. We need to help patients understand what their contribution is to a trial. For instance, hardly ever does a patient hear how their outcome, what they did during a trial improved the final outcome of a trial. The patient needs to be centered. If we get the trial to a point where some of the questions are pretty obviously answered, rather than continuing to recruit patients just to be statistically valid, I think trials should close sooner. I think they should be more focused on getting patient care without necessarily the scientific question. I’m not a radical. I’m certainly a fan of trials. We wouldn’t be where we’re at without trials, but I think they should just become more patient-centered and patient-friendly.

Andrew Schorr:

Okay. Now, Mike, Dr. Thomson, so we know we can’t have new drugs approved by the FDA unless there are trials, Phase I, Phase II for sure, and often, typically, Phase III and sometimes even monitoring after a drug has been approved. I think you call those Phase IV trials. But from where you sit having been around this a long time what are some of your frustrations? What would you like to see be improved?

Dr. Thompson:

I agree a lot with Jim. I think another word to put on it is pragmatic trials. So I’ve been on a number of advisory committees, NCI investigator-initiated studies and pharma-directed studies. And when you have an advisory group with a bunch of academics they often think about the theories, and they think about what would be interesting to know. And increasingly both the NCI and others are getting not only patients but community physicians who will say I don’t really care about this question here. And we don’t think that it will fly and won’t accrue, and we know a lot of trials don’t complete accrual, so therefore patients are wasted, if you will, because we won’t have the information, we won’t be able to answer questions. So I agree. There are so many things get to involved it’s hard to break them all down, but part of the issue is answering a clinically meaningful question. I think the meaning should be patient-centered. Within those questions you can ask scientific questions that are imbedded in what are sometimes called secondary imports or co-relative studies. But I just last week was talking to some pharmaceutical leaders, and I said, you have to design a trial to answer a question people care about, and that’s patients and physicians. Because sometimes the trials are designed to get FDA approval, and they’re comparator arm if it’s a randomized study, is an arm that we don’t think is the current standard of care, and we have to do them in countries where they don’t have as many therapies and they don’t have as much access, so they’ll get them done. But then when they’re approved in the U.S. we don’t know what to do with the trial, because it’s not a question we’re asking. So that’s important. And I think if more studies are done not to get FDA approval but to go on pathways and to ask, what are the clinical branch points for decision-making, I think that’s when you’ll start getting good trials.

There are a number of other issues around the pragmatics. So there’s this NCI Match study, tons of people screened, very few people on the matched drugs, and they switched over to a strategy more like an ASCO TAPUR, where they waited for people that already had testing and then the people that had already kind of pre-screened couldn’t get evaluated for the study. And many, many more people went on study. The imaging and other things in the middle were not as rigorous as a usual clinical trial. It rolled quickly, and I think the point is you’re looking for big end points. Where you have to sort of go back to the classical, randomized, Phase III large study is when you’re trying to make incremental improvements, so, for instance, breast cancer where the cure rate or progression-free survival rate may be in the 90-something percentile rate, or even CML or other things where we’re doing so well you’d need a lot of patients and probably a standard design. But in many other areas you can do a variety of different techniques—Bayesian analysis, continuous reassessment models.

And one thing Jim mentioned was stopping for futility or if there’s an obvious benefit, and that is done but probably not as often as it should be. And the designs using what are called interim analyses or futility analysis with data safety monitoring boards or DSMBs, probably could be more robust. There could be more of them. I think people are afraid to do them, because they do slow the trial down, they slow accrual, and that has to do with stuff both within the trial as well as extrinsic to it. So there are a number of barriers and issues, but I think Jim’s pinpointed them as well.

Andrew Schorr:

Okay. Well, folks, you can tell that Dr. Thomson is a scientist. We’re going to unpack this and get down to the nitty-gritty. So, okay. So, Jim, so first of all, we mentioned this term “randomization.” So people wonder in cancer am I going to get the good stuff? I know that I’m sick, maybe like in your area, multiple myeloma, there have been lots of new medicines, but in some other areas not, like pancreatic cancer, for example.

So, say, I understand the standard therapy, and you’re testing it maybe against that, but I want to get the good stuff, because I’m really hopeful. I want to be a believer. So could you just describe where we are with randomization, because that’s a concern people have?

Dr. Thompson:

Absolutely, Andrew, and thanks for asking that question. That’s a real red, red hot button item for me. I maintain that if the patient has gone through the effort of studying their cancer, studying the possible treatments, and they’ve learned of a trial that’s opened that they would qualify for, they’re excited, they go talk to the principal investigator, and they say I want to be in this trial. And the PI turns to them and they say, well, we’ll flip a coin. You may get the medicine we’re going to be using, or you may get standard therapy. Just imagine how disappointing that would be. And when it comes to randomization, Andrew, there’s many, there are many trials that absolutely lack equipoise. And I’m afraid that scientists often use equipoise.

Andrew Schorr:

Now, tell us what that means. You’ve got to define that for us.

Jim Omel:

Equipoise basically means equal, equal balance within the arms. In other words, technically, officially the principal investigator doesn’t know which arm is best. And yet look at it from the patient’s standpoint. Let me give you an example. There was a trial in which patients had the choice of three oral drugs in one arm versus a stem cell transplant in another arm. Now, think about that. Think of the insurance ramifications. Think of the fact that it takes almost a year to really totally recover from a stem cell transplant, versus taking three oral drugs. How can anyone say that there’s equipoise in a trial like that? So how can you pattern your life with the flip of the coin or a computer randomizing you into one of those arms?

Andrew Schorr:

Wow. That’s, that’s an important issue. Another one is, Mike, you know, people are—one of the ladies wrote in on Facebook I posted about this program, and she said, well, the trials are not really accessible to me because I live in a rural area, and they’re only in the big cities. You’re in one, Milwaukee. But Jim’s in Grand Island, Nebraska, and some people if you set requirements for the trial, well, you’ve got to come see me, you’ve got to come to the clinic for a variety of tests with some frequency and somebody has to drive four or five hours and take off work and get babysitters and all that, it just makes it impractical. Where are we with more trials being available or having an aspect of it, like testing, closer to home?

Dr. Thompson:

So I work at a community setting. I’m at our kind of flagship hospital but we cover most of the population centers of Wisconsin, so I think we cover about 70 or 80 percent of the population. So that’s a huge issue for our site is that we—when I talk to sponsors including as recently as last week I say if we can’t do it at all our sites I’m not really interested in doing your trial.

There are exceptions of course. We’re doing a surgical trial or a radiation trial that has to be at one site or sometimes a Phase I trial with just a lot of blood monitoring, very intensive, they can only be done at a few sites. But in general I completely agree that we should try to have the drugs available to people in the community they live in, because that’s where their social networks are, right? So that’s where their family is. They can stay at home. They don’t have to just go into a hotel. They don’t have to pay for travel, and I think it’s better for everyone. And for companies, I’ve been trying to tell them that it’s more generalizable to the reality of where cancer patients are. So

85 percent of cancer patients are in the community setting and are treated there, and drugs should be accessible to them there. So, you know, both the using the CCOP mechanism or NCCCP, and now we have the NCI Community and College Research Program or NCORP. The whole idea is to increase that access to community sites. So this has been going on a long time. I think there were budget cuts, and so the U.S. and the way we’ve established our cancer budgets has been to decrease access at least NCI trials and usually need some of those NCI trials to support the research infrastructure to do other studies. So I think part of that, you know, a lot of these things you follow the money. And if there was more money for community research sites, you could hire more research staff to get these things done.

But I think we need to get them done in the community, because we know if you do early phase studies and they look promising in highly selective patients, then when you expand them and put them in the community you go from efficacy to effectiveness, and the effectiveness isn’t there because the patients are different. So there are all these things with real-world data and comparative effectiveness research at ASCO’s cancer link trying to get at some of that not on study to just try to get the data.

But we need to have access to people, and the way to make drugs cheaper, make them develop faster and answer more questions, both scientific and patient-oriented, is to get more people on trial. There’s a big example for immunotherapy drugs where there are so many immunotherapy drugs and trials there are not enough patients to get it done. So we’re going to enrolling in trials which don’t complete, or we’re not going to be able to answer these questions, so it’s going to stall and move it out the process of moving faster. In myeloma, we move very fast, but we need to do this in other areas too.

Andrew Schorr:

Right. So let’s talk about that. So, Jim, you know, the president had a big kick-off, HHS Secretary Azar I think just yesterday as we do this program, was before Congress and part of it was the discussion of can we lower the cost of drugs ultimately? And one aspect of it is can we speed drug development. So instead of all these trials languishing at the cost of millions of dollars, hundreds of millions of dollars, how do we speed it up?

So one is participation, certainly, but can the process be simplified as well, Jim? What work is going on there, so we can try to get these answers and get to the FDA and present the data quicker, and hopefully there’s been lower cost in getting to that point?

Jim Omel:

Well, as we’re learning more and more about each individual patient, personalized medicine and targeted therapy, we certainly should start relying more on biomarkers. Biomarkers can be a way to select patients that would particularly fit a given treatment.

We need to lower costs. We need to make trials slicker and faster. Single-arm trials are those in which a patient just get—all the patients get the therapy. They all get the same treatment. And FDA has actually approved drugs based on single-arm trials, a much faster and efficient way to get an answer.

The problem is that the costs are going to be there. When I think about Mike and all the work that he does in developing his venetoclax (Venclexta) trial that he mentioned, Mike has put in months or years, and it’s all above and beyond his normal time. I mean his day job is to take care of patients, so all of the work that he does to develop a trial is just remarkable in the extra hours it takes and the consistency that Mike gives to doing his work. We need to make the trials more efficient.

We need to use biomarkers. We need to make them shorter. We need biostatisticians to come up with ways to give us an answer without having to approve so many hundreds or thousands of patients to all these potential new treatments.

Andrew Schorr:

So, Mike, let’s talk about that. And, Mike, first of all, I want to thank you for your—well, both of you, but, Mike, certainly in the clinic, thanks for your devotion to this.

But continuing on that, so this was brought up by Jim, biomarkers, and I know in some of the blood cancers now we’re talking about more and more minimal residual disease testing, and we’re doing genomic testing to see what genes have gone awry, what’s our version of lung cancer or a breast cancer or a myelofibrosis or whatever it is.

And then do we qualify for a trial? What’s our specific situation? Do you feel that that sort of precision medicine testing and analysis can help refine this, so we know which trial is right for which person at which time and also some analysis along the way of how is it going?

Dr. Thompson:

Yeah, so at my site I’m the director for precision medicine, and I gave a talk at ASCO on precision medicine and barriers in the community setting, so I’m very passionate about that. And I think that is one of the ways you can try to get things done with smaller numbers of patients and things done faster. And part of this is alignment, right? So there’s different perspectives, a patient perspective, a payer perspective, a pharma sponsor perspective, the physician. There’s all these different perspectives, and I think it’s trying to get them all aligned and trying to get things done faster.

So, you know, there are some areas where we don’t know enough, and we can’t use biomarkers. But there are other areas where we have a biomarker, and there’s feasibility, and we can test that quickly. And if we are looking for a large effect size—here I am in jargon mode—but if you’re looking for a big, big hit, a home run, is to look for an alteration that is very specific and we think is—a drug can target. So-called targeted therapy—it’s a little bit of a misnomer.

So—and lung cancer has been one of the hottest places for this. So there’s ALK inhibitors, ROS1 inhibitors, EGFR inhibitors, and now BRAF inhibitors, HER2 targets. So lung cancer has exploded with precision medicine therapy, and the same with melanoma and BRAF. So, you know, I think even skeptics will say you don’t really need statistics if the prior therapies, nothing worked, and you give something, and 80 percent of people respond.

There are issues with precision medicines, but the main thing is not response rate but durability. And I think that’s going to be the next iteration of the NCI Match study, which is a large precision medicine study, is stop doing just these small groups of people who are showing activity, but then they relapse quickly. And I think it’s going to look at systems analysis, and how do we overcome resistance.

But one way to get at this and another different take on it is inclusion and exclusion criteria. So this has to do with access and individualizing and being patient-centric. Many of the inclusion and exclusion criteria, when somebody says, oh, I have lung cancer, oh, here’s a lung cancer trial, and they say, oh, you can’t go on the trial. And much of that is because there’s language that’s been cut and pasted from a previous trial which is not really pertinent.

So if the new drug is metabolized by the kidney, you don’t necessarily need to look at the liver studies. And we did a small study or I was aware of a small study done by Kaiser where if we improve the inclusion-exclusion criteria, accrual rate can go up 30 percent—so no cost to that.

Andrew Schorr:

Wow.

Dr. Thompson:

And Ed Kim led a publication about six journal articles in JCO about different aspects of inclusion-exclusion criteria including function, HIV status, age, etc.

Andrew Schorr:

Well, yeah. We had Ed Kim on the program just a week ago, as a matter of fact.

So, Jim, inclusion, exclusion, so first of all, we’re in this age where electronic medical records, it would seem that at your fingertips there could be some analysis of your record and some matching or offering of trials that could come out of an analysis of your results, genomic results. Do you have ALK or ROS or whatever, if it’s lung cancer, whatever it may be maybe JAK2 positive in myelofibrosis, what is various status for us?

And also broader inclusion criteria, and Mike was getting at that, saying some was just—excluding was just cut and pasted. And a lot of us patients would feel, well, that’s just unfair. So what’s your comment on all that, about inclusion and exclusion and analysis so we can be matched with trials more easily, can be offered to us?

Jim Omel:

Inclusion and exclusion criteria are really important parts of trials. They’re what get people into trials, they’re what keep people from being in trials. And, unfortunately, Andrew, many times the criteria are very defined, very narrowed, and drug companies especially want to do it this way to get the best effective appearance of their drug. They want to get approval. And yet in the real world, in fact most times, patients who would not even need inclusion criteria are the very patients that are going to be taking these drugs.

And Mike’s right. There’s too much cut and paste. If a trial takes a thousand patients to write a proposal or protocol, too many times researchers will just take the exclusion criteria that might have been from a previous trial and, like Mike said, cut and paste it when perhaps it’s not even necessary to have creatinine values or kidney values measured so precisely on this particular drug compared to the other one.

So those are the criteria that let people in or keep people out of trials, and they absolutely need to be widened. To make a drug more applicable to the general population we need to reflect the general population more in trials.

Andrew Schorr:

Right. Right. It’s sort of a Catch-22. So if somebody is at a drug company and they’re investing hundreds of millions of dollars maybe to develop a drug, and then that trial is languishing or taking longer to get there, somebody ought to go back and say, well, can I loosen up this criteria, get the big answer and do benefit to patients who may be very willing to be into a trial that doesn’t have all of these requirements that are not really necessary? And we get the answer and get it quicker, and help people along the way. I mean, it’s pretty obvious to me, and I hope they’re watching, folks.

So, Mike, here’s a question for you, though, and you work with people in the community setting. So we have patients who have written in and said, you know what, where I go to the cancer clinic they never mentioned trials to me, and Jim alluded to the extra time it may take for physicians and their teams is when there are trials. You have just treating people with current therapies, and then you’ve got research layered on top of that. It’s very time consuming.

But what about just awareness at the community level? What can we do about that so that wherever I go into a clinic they have a clear picture of what I’m dealing with, and if there is important research going on that relates to me I hear about it? Now, maybe they say, you’ve got to go to a university center, you’ve got to go to Milwaukee, wherever you have to go, but there’s that discussion.

Dr. Thompson:

Yeah, so with all of these, you know this has been analyzed in multiple different papers. We were on one looking at a trial log, trying to look at some of these issues, and what seems to be clear is when people are offered trials they tend to go on them at about the same rate, and that has to do—seems to be somewhat independent of socioeconomic status, race, etc., or geographic area.

So one of my colleagues, Dr. Verani, told us about—about this, about rural settings how do you get people on trial. So there are different barriers. So one is the trial, and like Jim said, if you can only do some therapy that you have to come in quite a bit for that limits the geographic area you can accrue to for most people.

There are site issues where if you don’t have enough research staff to be there enough the doctor doesn’t feel supported to spend time on it. There are physician issues where they may not care about trials, or they have too much people scheduled in clinic, they’re an hour behind, and they can’t stop to spend time on it.

Also in the community setting you may be seeing every type of cancer, and you can’t remember everything, versus at many academic settings you may only see one or a cluster of types of cancer. So if you’re seeing lung cancer all day and you have 10 trials open, you probably know those trials very well for lung cancer, because you don’t care about the CLL or myeloma trials, you only care about lung.

And then there are patient factors. So patients that are in rural Wisconsin may have different characteristics, and the reason they’re in rural areas, you know, the motivations is about, you know, going in for things and stuff like that may be different than people who have the capabilities to fly to Boston or Houston or New York, and they can do that. So all of those areas are important.

Now, one potential way to help mitigate some of those things is we have got a clinical decisions support tool, which is an IT product, which our physicians have to enter in what they’re going to do with the patient. So it could be observe, no treatment, hospice or various therapies. And when they put in the cancer and the stage it pops up with the clinical trials, the first thing that pops up. And so the physician doesn’t have to do the trial, but they have to say why they’re not doing it. And so we can track over time. It doesn’t necessarily help that individual patient, but that doctor has been aware of the trial, and we kind of get an idea of why people are not going on studies, and so that’s one way to do it.

Something we just did the last week is we had a different IT product where the NCI-matched precision study opened up five new arms with different targets for different drugs. So we looked back at the number of patients that had those targets identified within our entire system, and then we screened those to see how many people were still alive, and were their organ functions still good enough to go on these trials because of the inclusion-exclusion criteria, and we found several. So we’re now able to contact the physicians and the research staff to go back for these patients that had screened for molecular testing and now they have new options.

So I think there are IT issues that you can do systemically to try to take some of those barriers away, and then each of those points does have barriers which probably have different solutions and different ways of tackling. But one reason, you know, the accrual rate hasn’t gone up a lot is it’s not easy. It’s a complex problem, so there’s not going to be one single thing you do. There’s going to be many different ways to try to improve things, including patient education.

Andrew Schorr:

Yes, well, okay. To let’s flip that over. Jim, you and I are patients. So what do we want to say, and from your perspective?

So back at the clinic and from group has, so Mike is working on IT to identify trials and have it pop up on the screen for the doctor. Okay. Great. But we’re the ones living with the condition. What can we do so that promising research that we may learn about is available to us? We can see whether it matches up with us. Maybe we have to go down the road. Maybe we have to have a discussion with our doctor to even encourage them to have you us be in a trial. How do we make it happen, okay?

Jim Omel:

Well, of course, we all need to educate ourselves about our cancer. When I was in medicine school I had heard about myeloma, but I certainly wasn’t any expert in it. I had two patients in my practice that had myeloma. I knew sort of how to take care of them. But since I developed my myeloma, I have become my own expert. And as I lead my support group, Andrew, I make them experts. I teach this cancer to them so that they can make educated decisions.

Patients are very likely to go on the Internet, watching Patient Power. In my particular cancer, they’re going to go to the IMF and MMRF to look at myeloma trials and see what’s available. And they will take that information to their doctors, many times making their doctor aware of trials that perhaps they aren’t each advocating or aware of.

So, Mike’s right. There are many factors that keep patients from trials, but one of the things that patients really do themselves is educate themselves and perhaps even to the extent of bringing or educating their doctor about what can be available for their treatment.

Andrew Schorr:

Mike, I want to ask you about cost. So you mentioned different inclusion, exclusion, or what’s your liver function or this or that. So there is a problem where maybe certain drugs or certain aspects of a trial are covered, but then your insurance company, you know, that you have or Medicare or whatever, they say, oh, no, we don’t pay for that, but yet it’s part of the trial or it goes along.

So people have a concern about cost. I want to ask you about two aspects of cost related to testing sometimes. And then also are there programs that can assist with the logistical costs for patients as well?

Dr. Thompson:

So when I trained at Mayo Clinic and MD Anderson, and when I got—first went into practice I prided myself in not caring about cost. And then I realized you have to think about these things because you can bank—you know, we bankrupt, about 40 percent of people with cancer get bankrupted. So these are huge issues for people who want to keep their houses, that want to hand something down to their kids, and cost is huge, right? So that can either be throughout the whole course of standard treatment, or it can be trying to meet the cost of going places, trying to find clinical trials.

So the Affordable Care Act and various other national and state legislative initiatives have tried to make insurance companies pay for the standard costs in clinical trials. There are some carve-outs for smaller companies and things like that, and so this is, you know, not perfect, but in general insurance companies should pay for the standard cost of clinical trials. They should pay for standard imaging stuff too, and they try to get out of that. So it’s not a perfect world, but that should be covered. And any research-associated costs should be covered by the company. Even in some NCI trials some people disagree with what should be covered and isn’t, and it’s complicated. But in general, a patient, the research cost should be covered.

Now, that does not include travel, lodging and a lot of incidentals. So there are a variety of foundations, that could be The Leukemia & Lymphoma Society, that could be other organizations which could help with that. Individual hospitals or health systems might have ways of approaching that. And sometimes there are things you can do within the various companies. So there’s a new target called Entrek, and the company Loxo, I’ve heard will fly people who wherever there’s a site and pay for them to go on the study, which I think is amazing. That’s not true for every company and every drug being developed. But that’s one way to do it.

One of the issues that comes up with IRBs if you’re giving people money, are you coercing them? And, you know, if you’re just recovering the cost to travel, I don’t think you are, right? But those are one of the things that come up. But certainly there are lots of disparities. And just like in different countries, they don’t have access to the drugs we have as standard drugs here, and not all of these disparities are going to be fixed because we have—outside of cancer we have lots of disparities in the United States, but cost is a big issue.

And then value, which we’ve been increasingly talking about in the oncology community, which is utility over cost. And that’s more for once we’ve done the trial figuring out even if shows like it works, how do we figure out how to use it based on those characteristics?

Andrew Schorr:

Thanks. And also I wanted to mention that Mike Snyder is sending that question, answering why it cost so much. I hope that answers it.

We have—you know, some people wrote in as we were preparing for this program and they were bitter because they thought they had a spouse, let’s say, that had died in a clinical trial. And that relates to a couple of things. One is transparency. Is the data from a trial and any dangers that show up, is that reported and analyzed in public, Jim? And also what are the risks being in a trial, and what is the monitoring to try to have trials be at safe as possible. So, Jim, maybe you could talk about that from a patient perspective.

I want to make sure I know what I’m getting, I know what the risks are, and if any have come up along the way I want it to be reported, and I want to know that there’s a team looking out for me.

Jim Omel:

You have every right to expect that, Andrew. If you’re in a trial you have the right to get that knowledge if there’s new things that come up that we’ve learned about. And part of every trial as it’s being written, there has to be a data safety monitoring board. These are the experts who will do what you’ve asked be done. They will monitor the trial as it goes along. They will look for any safety issues. If there are patients who are developing liver toxicities, they will find this. They will point this out and perhaps see if the trial needs to continue or if something needs to be revised.

The presence of institutional review boards review whether trials should go forward or not. Patients who are in trials actually get very, very good medical care and medical coverage. In fact, I would maintain, Andrew, that they get better care than just standard care. They have experts that are watching them even more carefully than would be in a general routine care setting because they’re looking for these concerns and problems.

The person who mentioned the bad outcome, we can’t ever say that every trial is going to be perfect. There are going to be concerns. That’s why trials are done. But they’re relatively rare, and we do have boards and review organizations during the trial, not afterwards, but during the trial to be looking out for your benefit, Andrew, so that you’re not hurt by the trial.

Andrew Schorr:

All right. But let’s say this—and, Mike, for you. So, first of all, admittedly a lot of these trial start, and people are sick people, and they’re feeling maybe the trial is their last hope. We had a friend, Lisa Minkove, who died in the CAR-T trial for CLL not long ago. She had been very sick with CLL, so we’d hoped that it would work. It didn’t work for her, whether CLL won. And we know other people whereas the learning is going on about often powerful new medicines they didn’t benefit. Or in one case, there was a drug, venetoclax we know about, there were some deaths early on when the drug was far more powerful than was originally understood. So what do we do? I mean that’s the real world I guess of scientific study, but that’s a concern, you know, Mike, of people saying, oh, my God, I’m worried about being a guinea pig the unknowns on the subject of dangers.

Dr. Thompson:

So there are a couple of things. So whenever people say—it doesn’t come up as much recently about being a guinea pig, I say, well, guinea pigs don’t have choices, so. And so like Jim has said you can drop off a trial if you want to drop off it. But—so I think for adverse events and things that can happen, one reason to randomize people is that you do understand then if you treat someone with one thing and then another and the death rate the same in both, the drug is not causing it. That’s just the disease. And a couple years ago, there was a presentation from the group at Dana-Farber on the precision medicine program, and the issue was they were taking so long to get people evaluated that their performance status or how well they felt was good, and by the time they got through the evaluation many of them had died. Because the disease, you know, when you get to fifth, sixth, seventh-line therapy it can often progress very rapidly.

And so I think that’s one of the issues, that people can feel the drug did it, and it’s hard to know. And we get these—doctors get these things called adverse events reporting forms, and we have to try to come up with is this probably related, possibly related, and we also get these forms that say you have a patient on the study. The study is open in three countries, thousands of people on it. One person died of a heart attack, and you have no idea as the physician, well, is that the same rate as—you know they’re 70 years old. Is that the same rate as this other 70-year-old. So you need the enumerator and the denominator, and that’s what the DSMB or the Data Safety Monitoring Board is supposed to do, which is look at the data and say, is this beyond what we would expect? And they can stop the trial. They can do expanded cohorts. They can do things to try and figure that out. Now, we know from like even car companies lying about their exhaust systems that if the Data Safety Monitoring Board gets false data, well, you can’t fix that. But that’s pretty nefarious. Like that I think is not something that’s commonly happening and would be a very serious thing to happen.

Now, one thing for transparency is that almost all studies I’m aware of get registered on clinicaltrials.gov or maybe some other sites but usually that site, and they’re supposed to report out the outcomes. It’s not also a perfect process, but you should be able to see how long the study has been open, are there any complications related to it and those types of things. So this whole process is not perfect, but I would say in general the people at the companies are trying to develop something they think is going to work. They’re trying to do it safely, both to help develop their drug well as well as to avoid a bunch of regulatory issues, and the people on the Data Safety Monitoring Board are trying to do their best to answer these questions. But the smaller the number of patients which increasingly will take the trials we are doing and almost are aiming for, it’s harder to be definitive about when these things happen and what caused it.

Andrew Schorr:

Right. Right. It’s imperfect, as we said. So, Jim, Mike Thompson mentioned earlier, gave lung cancer as an example and, of course, across immunotherapy, there are so many companies endeavoring to move this research along. So let’s say you had lung cancer or one of these others where this is big, although it’s going on in the hematology area too, so a patient says, oh, my god, there are all these trials, and I might qualify for one, two, three, four. How do I prioritize? What do I bet on? And maybe my own doctor is doing more than one. So what do you say to patients if they become receptive to being in a trial and there’s more than one trial that they qualify for?

Jim Omel:

That’s a very good question, and it’s a nice kind of problem to have, to have choices of trials. I think, Andrew, the best answer is the patient needs to look at what they are looking for. Are they looking for longevity? Are they looking for something that’s going to expend their life? Are they looking for a trial that maybe will greatly improve their quality of life? Perhaps they’re looking for a trial that gives them one pill per week versus two injections a week. So there are certainly effectiveness end points. There are different things that patients find of value.

But to answer your question it really comes down to each patient needs to ask themselves, what is it I’m looking for in a trial? Do I want something that makes my burden lighter? Do I want something that’s going to extend my life? How much am I willing it accept as far as potential problems versus the standard of care that I know what the problems exist with if I don’t go on a trial?

Andrew Schorr:

Right. So that’s a question we got in, is they’re trying to assess that. One was about how do I prioritize? The other is, by being in a trial, Mike, is it going to make me sicker? Like, to do I have to go through the valley of the shadow of death to get, hopefully, to a better place, and how do you discuss that with your doctor when not everything is known?

Dr. Thompson:

Yeah, maybe I’ll kind of step back and say for phases of trials, Phase I, the intent—both ASCO and NCI say the intent of a Phase I trial is therapeutic. But the statistical design is to evaluate safety. A Phase II is to look at initial efficacy or how well it works, and Phase III is to compare versus standard of care the efficacy. So there’s other types of designs, phase 0, Phase IV and other things, but it used to be, I think, you know, I—we would say don’t go on a Phase I unless that’s the last option because you’ve already gone through the safety initial efficacy if it’s a Phase III trial. It costs a lot of money to do Phase III trials so fewer are being done now, and we’re kind of finding that in this era of precision medicine people are going on trials, and there’s no one rule, but I look at it as if it’s a study involving a lot of different groups of patients, a lot of—you know, it’s not individualized to you, I don’t know, but I think it will have less of a benefit probably than if it’s something like a study designed for BRAF melanoma back when that was a study and you have BRAF. Well, it’s targeted for you. It doesn’t mean it will work, but even if it’s an early phase, a Phase I or II trial, it’s really aimed at your disease.

And we’re finding this with venetoclax, with T1114, and there’s other markers, FLT3 in AML, all these things, and sometimes we find that the drug doesn’t work like we think it’s going to work. The ALK and ROS story in lung cancer, it may benefit other people that we didn’t recognize before, and that’s part of–we’re trying to find people besides T1114 that respond to venetoclax in myeloma because it looks like some people will. But I think as we’re getting more targeted therapy it doesn’t mean there’s no toxicity, but it at least has the suggestion that we’re targeted more at your specific cancer. And some of these pills can have as much toxicity as IV chemo s, but our aim is to decrease toxicity and increase efficacy. And I think, like Jim said, you’ve got to look at different trials and hopefully with a physician who has time to sit down and run through several scenarios. And some people will take the most aggressive therapy because that’s what they’re after, and some people will try something that’s easier and closer to home. So everyone’s values are a little bit different, and you have to try to individualize as a patients.

Andrew Schorr:

Right.

Dr. Thompson:

One thing about trial matching is besides clinicaltrials.gov, there’s myeloma and other groups that are doing these matching, so you can put in characteristics of your cancer and you can try to filter out and get a closer approximation, including at clinicaltrials.gov you can click on the states in the surrounding area or how many miles you’re willing to travel.

Andrew Schorr:

Right. I would mention, put in a plug for our advocacy group friends, whether it’s Lung Cancer Alliance, Bonnie Addaria Lung Cancer or the International Myeloma Foundation with The Leukemia & Lymphoma Society, you can be in contact with them directly and talk about your situation, and they will often be very aware of trials and how it’s starting to line up with these sub groups, subtypes of illness. Here’s a question we got it in with Jack. I just want to get in a couple more before we have to go. This relates to what you were talking about the National Cancer Institute’s Match trial, as I understand it, Mike. He said, regarding precision medicine I thought I heard that initial results have been disappointing for the NCI trial which treats patients with a specific mutation with a specific drug for that mutation. How does this impact precision medicine? You want to talk on that? Mike?

Dr. Thompson:

Yeah, so the people who are opponents of precision medicine would say that the SHIVA trial in Europe and the NCI Match trials were failures. I think you need to look at it a little more carefully. And if you do a huge screening and you don’t have many drugs you don’t have many matches and not many people are going to benefit. So there are some arms in match that match the accrued the number they wanted, and the drugs didn’t work well. So those were truly we think negative studies. But I think the things about Match are there is a huge interest in the community, and they had thousands or several hundred people screened when they only had a few arms opened, and those people weren’t matches, and it basically overwhelmed the system. And then they had to rejigger it to open up more arms. So I think we could—you know, pick holes in the design of the initial study, but I think it took everyone by surprise how much interest there was in trying to personalize these molecular therapies. And other iterations such as ASCO TAPUR, there’s company versions of it like Novartis Signature, and I think the new design of Match do allow for better match rates, and we’ll see how after they’ve adjusted how well they can hit their targets.

Andrew Schorr:

Okay. So that’s an example, where we’re going through a makeover there. Before we go, Jim, we have people watching from all over the world, and Mike alluded to sometimes trials done in other countries. Certainly they are. So we have somebody from New Zealand, we have people from other countries now. How do I access trials? Does it have to be in my country? Or what would you say to an international audience as far as finding out what’s available to them?

Jim Omel:

That’s a difficult question because every country has their own standards. Each country has their own boards that review. What is allowed in some countries are not even allowed. Observational trials can have more importance in some countries than others. Again, it’s a tough question. I think perhaps the person who asked it really needs to be again their own advocate and go online, go with their physician, go to their local support groups, go to their national groups, because they’re the ones that can give that local person their answer. There’s no one set answer for every country because there are some many variances.

Andrew Schorr:

Right. I do want to tell one of my favorite stories. I had a friend Jan Rin in Dublin, Ireland. She had a tremendous problem with more advanced chronic lymphocytic leukemia, one of the conditions I have, no trial for her there. She heard about Imbruvica being studied in Leeds, England, different health system, national health system. She was in Ireland, didn’t have it. She got permission from the Irish government to go over to Leeds and be in Dr. Hellmann’s trial there, and I think it saved her life. She would tell you that. So she had to be pushy. There were newspaper articles. She had to do lots of things to make it happen. It’s going to be varied by country but it starts with…

Jim Omel:

…drug like the one you mention, and it’s not available in the country, and there’s so much of that in myeloma. We have many, many drugs in the US that they don’t have in other parts of the world, and it would be so sad to be a patient in those countries, know that a treatment like that is available but not have access to it. So we all need to work to get these drugs available to patients wherever they’re at.

Andrew Schorr:

Right. Amen. I want to just get some final comments from you. We may just go a couple minutes over. So, Mike, the process is improving, I hope, you’re working on it. Can we feel confident that these gaps, if you will, improving it for prevision medicine, more awareness among the doctors wherever we may go, financial assistance, working with the insurance companies, are you working on it so that this process, we can have some improvement and hopefully have higher levels of enrollment and can get drugs approved quicker?

Dr. Thompson:

Yeah, I think we’re all very concerned about it. We should all be aligned in having more patients on trial, moving things faster and getting it done more cheaply. And I think we’re making progress. It’s not as fast as any of us want, but we’re all trying to move the ball forward.

Andrew Schorr:

Okay. So, Mike, it comes—excuse me. Jim, it comes down to us then as patients. We have to push, right? We have to see what’s within ourselves, what are we willing to do, understand our clinical situation and what’s going on for our cancer, and we’ve got to push, right?

Jim Omel:

And one of the things we need to push for are more interesting trials. We need to make pharma companies put up their drug against another pharma company’s drug. I think it’s so troubling when they’re afraid to take big steps. They just take little, incremental steps with their trials. If we can put drug A of one company versus drug A of another company—pharma companies are really reluctant to do those kinds went trials, and yet those are the kind that would be exciting to patients. I could give certain names of myeloma drugs, but we won’t get into that. It just needs—we need to get better, more interesting trials, and that will attract patients.

Andrew Schorr:

Okay. So I want to just put in a plug for something. We started something at Patient Power called the Patient Power Ambassador Program, and you can see it listed on our site, where you can share your voice. So we can all work with Jim, work with Dr. Thompson, and we cannot just be getting what’s right for us, but we can push on this process. So please consider doing that. Because I want to thank you, Jim Omel, for not just getting what’s right for you as a myeloma patient, but working on these government panels and with advocacy groups to try to advance it for all of us. Jim Omel, thank you for doing this.

Jim Omel:

Thank you, Andrew. It’s a pleasure to do this, and I’ll keep doing it.

Andrew Schorr:

Yes. And long life, Jim. Thank you.

Jim Omel:

Thank you.

Andrew Schorr:

And, Dr. Mike Thompson, thank you, Mike, for your leadership too and those extra hours put in, not just for programs like this but all the clinical research speaking to industry and the government to try to improve this process. Thanks for being with us, Dr. Mike Thompson.

Dr. Thompson:

Thanks for having me on, and I think this is the some of the most powerful patient educational material that people can get, this type of program.

Andrew Schorr:

All right. Thank you so much. So, folks, we’re all in this together. So you have your own issues about whether you know about trials, whether you want to be in a trial, that’s right for you or a loved one, whether it’s close to home, not close to home, so—but we have these discussions. So please look ongoing at the clinical trials mythbuster series. The let us know how we did today. You can always write to me, andrew@patientpower. Our producer, Tamara, T-A-M-A-R-A, at patientpower.info. And talk to your own doctor and your own healthcare team about clinical trials and where they line up, what are the obstacles, for you participating. And let’s see if we can improve this process and ultimately have more medicine that can lead to a cure for us be available sooner. Thank you for watching. We’ve done our best today, but this is an ongoing discussion. In Carlsbad, California, I’m Andrew Schorr. Jim joined us from Nebraska, Dr. Mike Thompson joined us from Wisconsin. Worldwide, we’re here for you. Remember, knowledge can be the best medicine of all. Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. 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.


Please remember the opinions expressed on Patient Empowerment Network are not necessarily the views of our sponsors, contributors, partners or Patient Power. 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.

Notable News: June 2018

There’s a little something for everyone in the news this month. Immunotherapy looks promising for men; lung cancer does not. More women can forego chemo, and African Americans and Latinos have a new warning sign. Preventable cancers are on the rise, but your amount of alcohol consumption might help you change that. There’s a lot of news this month, and it’s all right here so you can pay attention and stay empowered.

Speaking of paying attention, African Americans and Latinos have a new pancreatic cancer warning sign. Recent findings show late-onset diabetes, after age 50, is an early sign of pancreatic cancer in African Americans and Latinos, according to this report from accessatlanta.com. The link between diabetes and pancreatic cancer is still unclear, but the study showed that African Americans were three times as likely to get pancreatic cancer after developing diabetes, and Latinos were four times as likely. While pancreatic cancer is rare, you should discuss your risk with your doctor should you get a late-onset diabetes diagnosis.

Another new report offers good news for women. New evidence shows that many women with breast cancer can forego chemotherapy as part of their treatment, reports washingtonpost.com. Findings from the federally sponsored, largest ever breast cancer trial indicate that women who have the most common type of early-stage breast cancer, with low and moderate risk of recurrence, don’t require chemo after surgery and won’t be subject to the often harmful side effects. The study previously showed that women with low-risk of recurrence didn’t need chemotherapy, but there was some question about those with moderate risk. After further study of patients with moderate risk, researchers determined that those who did not undergo chemo did as well as those who did. The type of cancer studied is hormone-driven, has not spread to the lymph nodes, and does not contain the HER2 protein. The findings affect more than 85,000 women per year and are expected to change the way early-stage breast cancer is treated. More information can be found here.

There’s also good news for some men. An early stage trial that was presented at the annual meeting of the American Society of Clinical Oncology finds that immunotherapy looks promising as a treatment for some prostate cancer patients, reports bbc.com. Unfortunately, the treatment doesn’t work for the majority of patients, with only 10 to 15 percent of patients having any response to the treatment. Researchers are hoping to determine which patients are most likely to respond. More information can be found here.

However, worldwide the news is not quite as positive. Lifestyle cancers are on the rise and increased prevention is needed, reports sciencedaily.com. Lung, colorectal, and skin cancers have all increased worldwide over the past ten years while other cancers have decreased, according to the Global Burden of Diseases (GBD) study in which researchers analyzed 29 cancers and then reported their findings based on age and sex for 195 countries and territories. Lung and colorectal cancers are the leading causes of cancer deaths worldwide despite the fact that they can be preventable with such things as dietary changes and reduction in tobacco usage. The GBD also found that the United States was the third leading country in new cases of cancer per 100,000 people in 2016. Australia and New Zealand were the first and second respectively. Syria was the lowest in both new cases of cancer and cancer deaths per 100,000 in 2016. The country with the highest rate of cancer deaths per 100,000 in 2016 was Mongolia. Here you can find the full list of cancers analyzed in the GBD and where they are most likely to occur worldwide.

In other lifestyle news, your alcohol intake may be affecting your health. Less alcohol means less cancer or death, reports livescience.com. A new study reveals that light alcohol drinkers (fewer than seven glasses per week) had a lower risk of cancer and death than those who drank more alcohol or no alcohol at all. The study combines the risks of cancer and death from other causes whereas most studies pertaining to cancer risk and alcohol don’t factor in various causes of death. The combination of the two addresses the role of alcohol in overall health. More about whether or not you should put down your wine glass can be found here.

Finally, those cancers pertaining to lifestyle are often likely to come with stigmas attached to them. While most people believe lung cancer is preventable and caused by smoking, forbes.com contributor Bonnie J. Addario offers a different perspective about the stigma of lung cancer and how it has hindered research. Smoking is not the only cause of lung cancer, Addario points out. In fact, she states, 70 percent of lung cancer patients have long-since quit smoking or never smoked at all. Lung cancer, as we learned above, is the leading cause of cancer death worldwide, and Addario notes it is the leading cause of cancer death for both men and women in the United States. Perhaps it’s time we look at lung cancer differently, as Addario advocates here. It’s worth the read.

 

ASCO 2018 Lung Cancer Roundtable

A Lung Cancer Roundtable: Takeaways from ASCO 2018

Lung cancer experts Dr. Jeffrey Crawford from Duke and Dr. Edward Kim from Levine Cancer Institute speak about key take-aways from this year’s ASCO meeting including immunotherapy updates, newly identified genes, the role of liquid biopsies and specific questions patients/care partners should be asking as the lung cancer landscape continues to evolve.


Transcript

Andrew Schorr:

Okay.  Here we go.

Hello and welcome to this Patient Empowerment Network program produced by Patient Power.  I’m Andrew Schorr from Patient Power, and we’re discussing an update from the big American Society of Clinical Oncology meeting, ASCO, and what it means for patients and family members dealing with lung cancer today.  I want to thank our financial supporters for making grants to support this program, Celgene and Pfizer.

So we have two noted experts with us.  We have Dr. Jeffrey Crawford from Duke University and the Duke Cancer Institute in Durham, North Carolina, and Dr. Edward Kim from the Levine Cancer Institute down the road also in North Carolina, in Charlotte, North Carolina.  Dr. Crawford, welcome to Patient Power and the Patient Empowerment Network.

Dr. Crawford:

Andrew, thank you.  I’m glad to be here.

Andrew Schorr:

Dr. Kim, welcome to you.

Dr. Kim:

Pleasure, Andrew.

Andrew Schorr:

Okay.  Gentlemen, let’s start.  So I walked into the ASCO exhibit hall, which is many football fields wide and long, and I was impressed with so many companies devoted to helping doctors and their patients understand the specific biology, molecular composition of the tumor that somebody might have for example with lung cancer.  Dr. Kim, is this where it’s going, is that sort of precision medicine?  And why is it so critical for patients and their doctors?

Dr. Kim:

Yeah, thanks, Andrew.  I think it’s really important to know how the new standards are changing.  We’ve been used to a lot of therapies and how we assess folks for decease such as biopsies and histological diagnoses, and now it’s not just about that.  It’s about trying to figure out what genes exist that are unique to each person’s individual tumor.  And we know that these genes are differently made up in different folks, so just to call somebody who has a non‑small cell lung cancer, and that’s the area that myself and Dr. Crawford cover, is really not the whole picture any more.

We’ve seen this in breast cancer.  We’ve just kind of come to accept it over the last couple decades, that you’re either a hormone receptor‑positive breast cancer patient or your tumor is HER2 positive or not or you’re a triple negative, and that’s means none of those markers are present.

Well, we were never that sophisticated in lung cancer, frankly, to have the equivalent of a triple negative even though we did, and we started is seeing this in the early 2000s, especially as we looked at first the mutations like EGFR and translocations like ALK and ROS1, and now that number is just really exploding as far as the number of markers that a clinician has to check just at baseline to make the proper assessment to treat a patient with non small‑cell lung cancer these days.

And that’s exciting, but it’s also daunting in that the data and the drugs and markers are changing so frequently that it’s hard to keep up, and even as an expert it’s hard.

Andrew Schorr:

Now, Dr. Crawford, you’re in research a lot as well, and so this multiplying of genes, you keep identifying new ones, right, and then it’s a matter of finding out, well, which genes are important at which time for which patient, right?

Dr. Crawford:

Correct.  As Ed was saying, it’s a complicated task, and I think we get now a lot of information.  When we do next‑generation sequencing, we get literally hundreds of genes.  Some of them are actionable, some aren’t, and really understanding which are and which aren’t and now to interpret that is becoming a field of its own.  So molecular tumor boards have started to try to dissect this at the institutional level so people can sit down with pathologists, (?) like the pathologist‑clinicians, try to work through how to move forward on an individual patient basis.

Andrew Schorr:

So, Dr. Kim, we hear about immunoncology, immunotherapy, and drugs that are being tested in many cancers to try to help the immune system be boosted, I guess, to fight the cancer.  Maybe you could explain that because there was news about that at ASCO, wasn’t there, for lung cancer?

Dr. Kim:

Yeah.  And certainly it seems like every major meeting, Andrew, has news about immunotherapy.  And the really nice part about it, speaking very selfishly, is that there has been a lot of news about immunotherapy and lung cancer, and I get to tease my melanoma colleagues, that, yeah, you know, we know it’s been around for greater than five, six years in melanoma, but it required a large scale sort of cancer to take this into the main stream.

And lung cancer is one of the largest.  It affects so many people out there, and to have these trials testing immunotherapies and these FDA indications, has really transformed things.  What we explain to people is that it’s not like the vaccine programs in the past in that the immune system is a very sort of gray area for a lot of folks.  Some people think you can take vitamins and boost your immune system.  Other people think you just have healthy living it will do it, and all those things contribute because your immune system is really like your micro environment throughout your entire body, and a lot of things affect it, and it affects a lot of things.

But what’s really cool about these newer generation drugs that are impacting the cancer process is that cancers have become smart.  They are able to build up defenses to be sort of stealth inside the body, and so even though there were bad things happening to you your body couldn’t tell that they were cancer cells versus normal cells.  And so these new checkpoint inhibitors have focused on trying to break down the stealth or the defenses that these cancer cells have been using to invade the immune system.

And so now you’re really empowering your own body’s immune system to fight the cancer.  And that’s really exciting.  The side effects, there are some but have generally been very well tolerable.  There are always a percentage of patients who can get a hyperactive immune system, and that’s usually what causes a lot of symptoms we see, but all in all‑‑you know, we use Jimmy Carter as a poster child, he’s like 150 years old, and he’s on an immunotherapy being treated for a stage 4 melanoma and doing very well.  So that’s what my patients see out there, that’s why they’re asking about it.  We have to select the right people who is appropriate.

Andrew Schorr:

Well, Dr. Crawford, let’s talk about selection.  So we’ve alluded to testing to understand what’s at work or what sort of immune levels, we hear these terms PD‑1 and PD‑L1, and they’re even mentioned on telephones commercials for lung cancer drugs.  So how do we know whether this changing world of immunotherapy applies to an individual patient?

Dr. Crawford:

Well, that’s a good question.  So I think we’re learning as we go about biomarkers for immunotherapy, but certainly the one that’s out there most notably is PD‑L1, and so that’s a marker of this protein that Dr. Kim was talking about.  It’s an immune checkpoint, so PD‑L1 when it finds the PD‑1 receptor down regulates or lowers the immune system, and that’s a natural, naturally occurring process.  It’s important so our immune system does get overly revved up, but what happens in cancers it often gets overly depressed and suppressed, so we have inhibitors, drugs that work by inhibiting that reaction that allow the immune system to emerge and attack the cancer.

So what’s really cool about this is that the immune system itself is what destroys the cancer when you take these agents.  This is not like chemotherapy or even targeted therapy where there’s a direct cytotoxic effect on the cells.  This is really enabling your immune system to take over and attack the cancer and destroy it.  So it’s remarkable when we see an x‑ray with cancer disappearing based on restoring the immune system.

So PD‑L1 is clearly an important marker because it’s the way these first‑generation immune checkpoint inhibitors work through that process.  So one would assume that the PD‑L1 measurement would be predictive of who is going to benefit and who is not.  And in some sense it is, but it’s not at all like EGFR testing, where we are pretty confident when we have an EGFR mutation we’ll have a very high response rate, while with PD‑L1 even in patients with expression above 50 percent only about half of them get a good response.

And on the other end patients with very low response, very low levels of PD‑L1, they still have a response of 8 or 10 percent.  So it’s not a perfect marker by any means, but it has been helpful in identifying patients likely to benefit.  And what’s come out of ASCO is more and more about how to select patients for immunotherapy or a combination of chemo and immunotherapy or other options.

Andrew Schorr:

Dr. Kim, let’s talk about biopsy for a minute or how you get the information from the patient as to what’s going on and then what to do about it, if you will.  So getting a lung biopsy is not easy, and I know sometimes there’s a problem getting enough tissue to do all the analysis you want, and now we’ve been hearing about more and more companies that are doing liquid biopsy.  Okay.

So here’s Mr. Jones, you want him to have a lung biopsy.  Would there also be a liquid biopsy or‑‑and not just at diagnosis but would you be doing some of this along the way to see if treatment is working?

Dr. Kim:

Yeah, we’ve always been attracted to some of the other cancers that utilize liquid tests, ovarian cancer, CA125, PSA, prostate cancer, although we’re still not really clear on where we’re supposed to be using that to screen patients, but that has given people is principle that they like to follow things.  And that’s why cholesterol, for instance, was such a powerful sort of marker even though the relevance of it has been questioned by cardiologists.  People can see there is an effect.

So, first of all, we have to say that nothing has completely replaced tissue.  That is really the gold standard.  It still is.  I tell our interventionalists, whether it’s a pulmonologist, interventional radiologist or anyone, I don’t want a diagnosis.  I want tissue.  Because they can make a diagnosis by doing some brushings or some cytology, and they can tell me it’s an adenocarcinoma favoring lung.  That is not helpful.  We need to absolutely have data that allows us to send for these molecular tests which includes, as Jeff mentioned, PD‑L1.

We need EGFR mutation, ALK, ROS1, BRAF.  These are all very important markers now that need to be sent.  And in some cases, at some centers they send for the larger panels.  What you get are 3‑ to 500 genes.  I don’t need 3‑ to 500 genes, but there are certainly clinical trials out there that can help match patients into trials based on these genes, so it is some utility.

But the blood‑based markers and the biopsies are improving.  There are definitely very‑‑there are good data that show concordance when they’re positive.  So if you do a blood test and it shows a positive mutation for EGFR, for instance, you can be pretty confident that the tissue has that as well.  The problem is that when you get a negative result.  And the negative result, those percentages aren’t disconcordant because (?) really show the amount of accuracy, and so you can’t take a negative test at face value.  We don’t standardly do liquid biopsies in patients unless the patient really has a contraindication to doing a traditional tissue biopsy.

As far as the surveillance aspect, as you mentioned, we do that on research.  So on our research studies we do follow patients at every cycle with another blood draw, in addition to what they give in labs, so it’s not an extra stick.  It’s just extra biopsy.  And we do try to follow to see if we can see some of these different mutations either go up or down based on how the treatment is working or not working.  And we’re hopeful that this type of research down the road can lead to more predictive assays that are easier to gather so we can either surveil patients to see if they have cancer, if it’s gone away, if it’s come back.

You can imagine somebody who has been treated for cancer, who has no evidence of disease on a CAT scan but maybe with blood surveillance we can get an early sign if something is coming back.  These are all possibilities and are being investigated, but right now it’s really a backup plan if tissue can’t be adequately gathered.

Andrew Schorr:

Dr. Crawford, of course you’re doing research as well.  Do you agree with this, where we are now and where we’re headed?

Dr. Crawford:

Absolutely.  I think what’s happened in lung cancer is because of this need for tumor tissue, as Dr. Kim has pointed out, it’s really transformed all the interventional things we’ve been doing.  We were moving in the 90s to smaller and smaller biopsies, smaller and smaller needle aspirations just to make a diagnosis, but now we’ve gone back the other way where we’re retraining our pulmonologists to get larger cores of tissues.  They’re developing new techniques to get more tissue, endobronchial biopsies.  CT interventional people have been enormously helpful for getting core biopsies so we get adequate tumor tissue to do the molecular tests we’ve been talking about.

So that’s really fundamentally important and important to have at every institution hospital across the country.  It’s one thing for Levine or Duke to be able to do this, but it really needs to be done in smaller community hospitals and done well by interventional people who can get the tissue we need because the samples can always be tested at a central site if the pathology labs can’t do it locally.  We have to be able to get the tumor tissue.

Andrew Schorr:

Let’s pull this together for a little bit.  I want to see if I’ve got this right.  So you’re having a revolution now in more genes being identified and trying to decide what’s actionable, whether you have approved medicines or combinations or drugs in trials, that both of you have alluded to, could for research purposes you identify something and where that could offer hope to a patient where otherwise the existing therapies might not match up.

So what actions should patients and family members be talking about?  And you said, Dr. Crawford, like at the community level or if they have a university hospital as a choice to go.  What should they be doing now because obviously anybody diagnosed with lung cancer or their family member, we want the longest life and the best chance right now, and yet you have an evolving field.  So what would‑‑Dr. Crawford, how would you counsel patients and family members so that with what you have available, either as approved therapies or in trials, could be available to them?

Dr. Crawford:

Well with, first, let me back up a second to say we’ve been talking mainly about advanced lung cancer.

Andrew Schorr:

Right.

Dr. Crawford:

So it’s important that patients get diagnosed early.  It’s important that patients who are eligible for CT screening and to go that so we can detect lung cancer at an earlier stage and hopefully offer them curable surgery, and then for them to get evaluated by a multidisciplinary team if they’re in early stages to see is surgery alone the right thing, surgery and chemotherapy, a combination with radiation, so all those standards are still present in early‑stage disease.

Now, as we may talk about, immunotherapy and targeted therapy may have a role there as well, but I think our curative strategies remain intact there.  So it’s very important to have availability of a multidisciplinary team that can really assess cancer at all stages.

For the advanced cancer patients then, what’s particularly important is for every patient to get molecularly defined tumor testing being done.  So we not only need to know the pathology, as Dr. Kim has said.  We really need to know the molecular phenotype of cancer to really make the best treatment approach for patients with advanced disease.  And in most patients that should happen before they ever talk about chemotherapy.  We need to know are there better approaches for that patient, and we’re not going to know that without these tests being done.

Andrew Schorr:

How about you, Dr. Kim?  I mean, still chemotherapy is still around, still in combination.  People understand there are side effects, not that there are not side effects with the new immunotherapies, but people would like to skip to the most effective treatment first.  So what recommendations would you have for our listeners?

Dr. Kim:

Yeah.  You know, we’re talking strictly about the advanced lung cancer patients.  The new standards in non‑small cell, both nonsquamous and squamous, now contain an immunotherapy combined with chemotherapy in markers that are lower selected or unselected.  I agree with Jeff.  You know, the biggest struggle we always want to tell our patients is be patient.  Do not let the chemotherapy start without having the results of your markers.

And that’s where sort of this new diagnosis of cancer comes in, the fear of it growing while you’re waiting a couple of weeks for the results of these markers, but we have to reassure patients it’s okay because if you just wait the extra one to two weeks.

And I understand it could take longer getting the biopsy to get enough tissue, sending it away, taking three weeks, and then your doctor, who is maybe not as sophisticated at reading these very, very, 18‑page reports, take some time to evaluate it.  It could be five weeks right there very easily, and we don’t like to wait that long.

But if you do have a marker present, and if it is‑‑and now almost 50 percent of the patients with non‑small cell have this, have a marker, maybe we’ll be able to give you something in lieu of chemotherapy that’s not a pill, single‑agent immunotherapy.  And certainly as a default now we’re seeing again new standards of care.  New standards of care are combination therapy, chemotherapy with immunotherapy based on data that’s been presented in the last couple months.

And so as a biomarker person I love seeing marker‑enriched populations receiving less therapy, but as we begin to incorporate these drugs in our standard regimens we’re seeing improvements that are undeniable and are forcing us now to readjust or new standards.

Andrew Schorr:

Dr. Crawford, so I’ve heard along the way, and I know knowledge is expanding, whether or not some of these newer approaches apply to people whether‑‑you know, whether they smoked or not, whether they had a history.  Where are we now with having the widest array of approaches for the widest array of people whether they’re smokers or not?

Oh, we lost your audio.  Go ahead.

Dr. Kim:

Am I back?

Andrew Schorr:

Yeah.

Dr. Kim:

So smoking is clearly an important factor in outcome for patients, and it’s also somewhat predictive of likelihood of different things.  We know smokers have a lower rate of EGFR and ALK translocations, mutations.  We also know that they have a higher rate of PD‑L1 expression and may be more likely to respond to some of these immunotherapies, but those are just generalized statistics.  And we have smokers who have EGFR mutations, and we have never smokers who respond beautifully to immune checkpoint therapy, so the answer is we have to do the molecular testing and sort out who has what.  Smoking may influence that frequency, but on any individual patient basis we have to have the tests to know how to best to treat them.

Andrew Schorr:

That’s good news.  So, Dr. Kim, you had referred earlier about cancer being kind of wily, if you will.  So is it possible that the molecular testing results at time of diagnosis further down the road may be different?  In other words, some other gene is driving the cancer should it come back or it’s still going, and you need a different approach.  In other words you have to change horses, if you will.

Dr. Kim:

Yeah, that’s a great point, Andrew.  You know, back in 10 years ago, almost 11 years ago when we initiated this trial while I was at MD Anderson called BATTLE, the whole principle was to rebiopsy patients once they completed or once the first line of therapy stopped working.  And for that very point you brought up is that these tumors change.  If you use a baseline tissue that’s a very different environment that that tissue was exposed to.  It has not been treated with chemotherapy, it’s not been under different stressors, and nor has it now begun growing after getting chemotherapy.

So a patient, just as you say, who has been treated maybe there was some success but then it‑‑with chemotherapy it’s always a little transient, and then now the tumor is growing despite being treated, that could be a different tumor.  It’s been shown also by the Boston group that you get transformation to small cell, of all things, in about 15 percent of patients.  And so different histologists altogether.  So who knows what will evolve out of the cancer that’s been treated that is now beginning to grow.

And so I think it’s really important to have a repeat biopsy when this occurs to help again drive the appropriate treatment.  And, as we talked about earlier, if it’s difficult sometimes a liquid biopsy can even be done at this setting if it’s difficult or the patient is has a difficult area to get tissue.

Andrew Schorr:

So, Dr. Crawford, you have lung cancer meetings throughout the year, but the ASCO meeting with like 40,000 people across all cancers from around the world, it’s a big meeting.  You’re involved in research and, of course, with existing therapies as well, how positive do you feel about change and even the rate of change to offer hope for people dealing with lung cancer today?

Dr. Crawford:

I’m as excited about lung cancer as I’ve ever been, and I’ve been doing this for quite a while.  The rate of change is, as Ed has pointed out, is dramatic.  The number of new agents that we have seen over the last year, both targeted therapies and immunotherapies, and the rate of change, it’s not just ASCO every year.  AACR, a meeting that’s normally more basic research, had major breakthrough discoveries (?) inaudible, as well I’m sure this year, and Europe will have additional new discoveries as they did last year.

So it’s really changing every few months, our guidelines through NCCN have to be changed almost monthly, and I think that’s a good thing.  It’s telling us that new knowledge is really being moved very quickly into the patient care arena.

Andrew Schorr:

Dr. Kim, so we’ve talked largely about non small‑cell lung cancer, and you’ve rattled off some of the different types.  There’s a percentage of people, smaller percentage, but people with small cell‑lung cancer.  Were there things you were hearing there at ASCO that could offer hope or in research to help this population as well?

Dr. Kim:

And certainly Jeff is the expert here.  He’s had a long career with it.  Small cell has always been that tough cancer where you get teased a little bit.  Again, if you’re fortunate enough to find someone in limited stage you can try to deliver curative intent therapy.  If they happen to be in an extensive stage it really becomes about trying to give chemotherapy that has a high response rate, and so you feel good about that, but then the difficult aspect of it is that in fact it doesn’t last forever.  And so when it does again not respond, it’s not responding, we’ve got to figure out some things.

The immunotherapies have been very widely tested, and so there are some therapies that are coming.  There are some that are approved, nivolumab, ipilimumab have been used.  They’re trying to incorporate in combination with chemotherapy with these immunotherapies.  There are some other drug classes, (? Phonetic) roba‑T and others that are being looked at very closely in small cell.  So I love the fact that there’s spillover in the small cell because it wasn’t really a high area of importance for a lot of development of drugs, which was unfortunate because we still see those patients, but it’s nice to see that there’s a lot of studies been looking at these types of drugs.

Andrew Schorr:

Okay.  Dr. Crawford, any other comment you wanted to make about small cell?

Dr. Crawford:

I would say it’s an area that’s been difficult to see advances.  Small cell presents generally at more advanced stage, so very few patients can have surgery.  Chemo and radiation can still be curative for early‑stage patients with lymph node involvement who don’t have distant disease, but in the advanced stage setting we’ve been using the same chemotherapy for 20 years.  Our supportive care has gotten better, we’ve made some advances, but we’re hoping immune therapy and others will make a difference.

It’s kind of interesting.  Small cell, you would think, since it’s prevalent largely in smokers, people with smoking exposure, could be very‑‑a lot of mutations being present.  We know that total mutation burden is a nice predictor of benefit in non small‑cell lung cancer, so we think that would‑‑might play out here.  There is PD‑L1 expression in small cell but it’s not as intense.  And there is some separation by PD‑L1 score of benefit for immune checkpoint therapy in small cell, but the responses in general are less than they have been in non‑small cell.  So we’re going to need more, more homework to figure this one out, but I think we’re taking some steps in the right direction.

And as Dr. Kim pointed out, roba‑T is a targeted therapy, maybe one of the first targeted therapies we’ve had in small cell that attacks antigen present on a lot of small cell called (?) B L L 3, and there are other therapies being developed against that B L L 3 because we know that’s an important marker.  So I hope we will see agents that are truly targeted therapies in small cell in the next few years.

Andrew Schorr:

Okay.  So I think as we pull this together, and I think you were rattling off some acronyms, and that’s sort of what we’ve been seeing a lot in lung cancer now.  We’ve talked about EGFR and ALK and ROS1, and we talked about also PD‑L1.  So I know for patients it can be confusing, but look back, review this program with Dr. Crawford and Dr. Kim were saying about if you have someone diagnosed with advanced lung cancer to get that molecular test (? Inaudible) and make sure that the experts like this in your major center like this, that they have the information.  And then if you need to (? Inaudible) you may get (? Inaudible).  So (? Inaudible) but there’s help in second opinions from people like this.  Dr. Crawford, did I get it right?

Dr. Crawford:

I think you did.  You’re a good student.

Andrew Schorr:

Okay.  All right.  Well, we have two professors with us, Dr. Edward Kim from the Levine Cancer Institute in Charlotte, North Carolina, my old home down, and Dr. Jeffrey Crawford from Durham and Duke University.  I’ll say that even though I went to the University of North Carolina eight miles down the road.

Dr. Kim:

You had to say that.

Andrew Schorr:

Yeah.  Thank you.  Thank you both for your work in treating patients and in researching, helping give us a window into this ASCO conference, but I get the sense you‑‑you said it, Dr. Crawford‑‑you’re having meetings every couple of months and talking to your peers all the time, and this is a faster changing field.  Thank god, right?  So thank you so much.  Dr. Crawford from Duke, thank you so much for being with us.

Dr. Crawford:

Andrew, thank you so much and thanks to all the patients who are joining in today.  It’s for you we do all that.

Andrew Schorr:

Yeah, thank you.  And Dr. Kim, thanks.  I interviewed you years ago, and you were at MD Anderson.  Now you’re in Charlotte and you have a wonderful program there.  Thank you for being with us.

Dr. Kim:

Thank you, Andrew.  It’s our pleasure, and again, we’re just as excited as the patients because we get to offer them these really cool therapies and research studies.

Andrew Schorr:

Right.  Okay.  All right.  All the best to our patients and family members watching.  For the Patient Empowerment Network, I’m Andrew Schorr from Patient Power.  Remember, knowledge can be the best medicine of all.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. 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.

Talking to Your Oncologist About Clinical Trials

You’ve gotten a cancer diagnosis. You’ve selected an oncologist as your partner, working toward “No Evidence of Disease,” or NED (NED is every cancer patient’s very best friend). Your and your oncologist are working up a treatment plan, and you want to talk about clinical trials as part of that plan. Should you kick off that discussion, or wait until your onc brings it up?

YES, definitely bring up clinical trials yourself, if your oncologist hasn’t started that conversation. If you’re not sure how to kick off that discussion, here are some tips.

  • “Just do it.” Lace up your mental Nikes, and just ask the question. Have some resource links handy at your next oncology team visit, or start the conversation before the visit via your onc’s patient portal. Start with the information on the Patient Empowerment Network’s Health Centers and Programs hub, take a dive into gov, or check out the American Society of Clinical Oncology’s Cancer.Net trials site.
  • There’s an article in the Journal of Oncology Practice, “Identifying and Selecting a Clinical Trial for Your Practice,” that talks clinicians through the process of selecting clinical trials for their oncology practice. Reading through that can help you craft some great questions, and open a productive conversation with your treatment team about clinical trials for your cancer.
  • The National Institutes of Health has a great tip-sheet for oncologists on how to talk to their patients about clinical trials. You can use that to frame the conversation you’d like to have with your own oncologist about your clinical trial options. I’ve often found that reading articles and tip-sheets aimed at the clinical side of the equation have helped me accelerate discussions with my own clinical teams about treatment options, for cancer and for other medical conditions.

When you’re dealing with a cancer diagnosis, you want to have all your options on the table, and make the most informed decisions possible. Opening up a dialogue with your oncology team about clinical trials early in the treatment process will give you the information you need for those “most informed decisions.”

Another reason to open those discussions early is to gauge your oncologist’s response to shared decision making, and participatory medicine. If your oncologist doesn’t welcome self-advocacy on your part, it’s better to know early in the treatment process so you can shift to another, more participatory practice.

You are the focus of your cancer treatment team’s work. Lead the discussions, share your perspective, and participate fully in your treatment planning. Opening the discussing of clinical trials is a great way to get your team on your page about treatment and outcome preferences, and to unlock the power of precision medicine.

Clinical Trial Helps Lung Cancer Patient Live Active Life

Editor’s Note: This blog originally appeared on the American Lung Association’s Blog. You can view it here.


Donna Fernandez’s father died of adenocarcinoma at the age of 49, just six months after he was diagnosed, so when she learned she had the same disease, she knew exactly what it meant.

“They told my husband that I would live for four months,” she recalled.

Adenocarcinoma is a form of non-small cell lung cancer often found in the outer area of the lungs and makes up about 47% of lung cancer cases, and usually grows and spreads to other parts of the body more slowly than small cell lung cancer does. It develops in the cells of the outer layers of lung tissue, which line the cavities and surfaces of the body and form glands.

Donna was put on traditional chemotherapy and the cancer tumors did shrink, but the moment she stopped chemotherapy, they came back. “I was most worried about my dogs,” Donna said. “They can be pretty evil, and no one is going to love these little devils the way that I do.”

Like many patients, Donna, who worked through her treatment, had a tough time with chemotherapy. If she had treatment on Thursday, she would feel fine until the steroids from the treatment wore off by the weekend.

“I’d be so nauseated that I could not bring myself to drink anything,” she remembered, “I wouldn’t be able to walk from the couch to the refrigerator.”

During chemotherapy in 2013, Donna was approached with the opportunity to join a clinical trial, and it changed her life.

“I had no idea that it would help me,” she said. “I thought that I was helping future generations.”

Still an active participant in her clinical trial for treatments, Donna checks in every two weeks for an infusion and extensive blood tests. With the personal care administered from her healthcare team, to the tailored medical regimen, down to the feeling of being among family when she visits the Cancer Center at University of Texas Southwestern, Donna admits that she went from surviving to thriving.

“I can’t say enough about the ramifications [of the clinical trial],” she said. “I’m living proof. I’m not just alive, I’m living.”

Donna and her dogs travel often as they participate in dog agility competitions throughout the country. They went to Tennessee last year, and Donna is now preparing for a trip to Missouri, all thanks to the tremendous treatment received as a result of a clinical trial.

“I’m living my life and that’s so significant,” she said. “Just a few year back, I probably would not have made it.”

Donna’s story is proof that clinical trials are a valid treatment option and should be considered as an option throughout treatment.

A How-To On Reading Scientific Papers

“Be skeptical. But when you get proof, accept proof.” – Michael Specter

That quote is from Denialism: How Irrational Thinking Hinders Scientific Progress, Harms the Planet, and Threatens Our Lives, where New Yorker staff writer Michael Specter examined the distrust of science that’s turned discussion of scientific topics into a potential minefield. Some good examples of that minefield are climate change, and childhood vaccinations.

Anyone interested in scientific progress – full disclosure, I’m in that group – needs to understand the ideas being explored in scientific papers, the dispatches from the front lines of scientific thinking and discovery. To arrive at that understanding, you have to be able to understand what you’re reading, and I’ll be the first to admit that isn’t easy.

Scientific papers are written by scientists, for scientists, and follow a set of rules and formal structures that can feel like they’re designed to prevent any understanding by the average Joe/Jane “just plain human.” In this post, my goal is to help anyone interested in, but not formally trained in, science tackle reading – and understanding! – an article in any scientific journal.

10 steps to scientific (article) understanding

  1. Check the source

    • What journal is publishing the article? Check Beall’s List, and if the journal appears there, you can stop reading – it’s a fake journal.
    • Who is the lead author, and what organization or institution is s/he affiliated with? If it’s an established university or research institute (University of Chicago or Scripps Institute, for example), keep reading.
  2. Read the introduction first, not the abstract

    • The introduction will reveal the Big Question, the one that the research project worked to reveal the answer to. For instance, an article in the Christmas 2017 issue of The BMJ reports on research into the effects of pet ownership on human biomarkers of ageing; the introduction clearly lays out the Big Question as “ we examined the prospective link between pet ownership and a selected range of objective biomarkers of ageing proposed for use in large scale population based studies of older people.”
  3. Write out your own summary of what the research was examining

    • This will give you a grasp of why the researchers wanted to ask the Big Question, and a framework for assessing what their answers to that question are.
  4. Identify the null hypothesis

    • The null hypothesis could really be better termed the “nullifiable” hypothesis, since the purpose of the research project is to nullify the hypothesis that there are no differences in possible answers to the Big Question.
    • An example of a null hypothesis is “the world is flat,” which is what Copernicus worked to scientifically disprove a while back. He was successful, but there are some people who still reject his conclusions. (Warning: opening that link might be hazardous to your sanity.)
  5. Look at the approach, and the methods, used in the research study or experiment(s)

    • What did the researchers do to answer the Big Question? What specific experiments did they run?
    • Sketch out diagrams of each experiment or data crunch.
  6. Read the results section of the article

    • Look at the written results, as well as all charts and figures related to those results.
    • What are the sample sizes? Really small sample sizes are a red flag.
    • What results are listed as “significant,” and what as “non-significant”? If you want to totally geek out on this topic, this post will make your geeky day.
  7. Do the results actually answer the Big Question?

    • Using your own judgment, do you think the study authors have answered the question asked in the introduction?
    • Do this before you read the paper’s conclusion.
  8. Does the conclusion make sense, in light of everything you’ve read and evaluated while going through the paper?

    • Do you agree with the conclusion?
    • Can you identify an alternative explanation for the results in the article?
    • What are the next steps the authors see emerging from their research?
  9. Read the abstract at the beginning of the paper

    • In light of the work you did in Steps 1 through 8, does the abstract line up with what the authors said their research purpose was?
    • Does it fit with your own interpretation of the paper?
  10. What are other scientists saying about the paper?

    • Have other scientists written about this paper?
    • What other research is referenced in the paper?
    • Have the authors of that research weighed in on the paper you’re evaluating?

Reading, and understanding, scientific papers takes practice. It’s also fun, if you’re a science nerd, or just interested in new scientific discoveries. And it’s work worth doing, because the more you know, the more likely it is that you yourself might make a discovery that makes a difference.

How Do I Stay Strong During Lung Cancer Treatment?

How Do I Stay Strong During Lung Cancer Treatment? from Patient Empowerment Network on Vimeo.

How can I stay strong during lung cancer treatment? Can I tackle fatigue, nausea and anxiety through lifestyle changes? Two noted experts, Dr. Christine Lovly, Assistant Professor of Medicine in the Division of Hematology-Oncology at Vanderbilt-Ingram Cancer Center and Dr. Ishwaria M. Subbiah, Assistant Professor of Palliative, Rehabilitation & Integrative Medicine at The University of Texas MD Anderson Cancer Center both discuss how they work with lung cancer patients and their families daily to address effective evidence-based interventions that can be used to help patients stay strong during treatment.

Clinical Trial Mythbusters: Do Patients Have A Voice While Participating in a Clinical Trial?

For survivors Roberta Alberle and T.J. Sharpe and so many others, cancer was an unwelcome intruder that suddenly demanded their attention. Both became proactive and engaged, vocal patients, doing research about their treatment options and gaining access to clinical trials that made a HUGE difference.

Watch to learn:

  •       Do patients still have a voice during the clinical trial process?
  •       What is a clinical trial navigator?
  •       How can patients help their healthcare team be more effective in locating trials for them?
  •       More about clinical trial resources

Clinical Trials Mythbusters: Do Patients Have A Voice While Participating in a Clinical Trial? from Patient Empowerment Network on Vimeo.

 

Clinical Trial Mythbusters: Are Clinical Trials a Gamble for Me or My Loved One?

Is a clinical trial right for me or is it a gamble with my health? How will my loved one be affected? Do the risks outweigh the benefits? Watch as a panel of experts, including an oncologist, trial coordinator, and patient advocate as they debunk some of the myths around clinical trials. Listen to hear the patient voice and perspective for getting the best care-making decisions about clinical trials.

Watch to learn:

  • What are common clinical trial myths?
  • Why should patients participate?
  • How can patients navigate the system?
  • How can I or my care partner work with my medical team?

Clinical Trial Mythbusters: Are Clinical Trials a Gamble for Me or My Loved One? from Patient Empowerment Network on Vimeo.

 

ASCO 2017 Roundtable: Progress in Lung Cancer on Multiple Fronts

At the recent American Society of Clinical Oncology meeting (ASCO), Dr. David Carbone, Director of the James Thoracic Center, James Cancer Hospital and Solove Research Institute at Ohio State University Comprehensive Cancer Center and Dr. George Simon, Professor in the Department of Thoracic/Head and Neck Oncology at The University of Texas MD Anderson Cancer Center, share highlights from the meeting including what they call “progress on multiple fronts in lung cancer.” Dr. Carbone discusses how patients should demand a genetic workup on their tumor and his free genetic testing initiative at Ohio State. Dr. Simon shares how some immunotherapy and chemotherapy combinations are showing promise and how small cell lung cancer patients could potentially also benefit. Watch the video below to hear from two lung cancer experts.

ASCO 2017 Roundtable: Progress in Lung Cancer on Multiple Fronts from Patient Empowerment Network on Vimeo.

Clinical Trial Mythbusters: Are Clinical Trials a Last Resort Treatment Option?

Are clinical trials only for patients who run out of treatment options? Watch as our expert panel answer questions as they debunk common myths around clinical trial participation. Tune in to hear the patient perspective and expert advice for making decisions about clinical trials.

Watch the video and learn:

  • What is a trial and when should I consider one?
  • What are common clinical trial myths?
  • If my cancer center does not offer a trial, what should I do?
  • How can I stay informed?
  • Is there financial assistance to be in a trial?

Clinical Trial MythBusters: Are Clinical Trials a Last Resort Treatment Option? from Patient Empowerment Network on Vimeo.


Transcript:

Andrew Schorr:

Hello and welcome to this Clinical Trials MythBusters program.  I’m Andrew Schorr from Patient Power joining you all the way from Barcelona, Spain.  We’re here for a conference.  You’re about to meet folks from across the U.S. and wherever you’re joining us.  Thank you so much for joining us.

Thanks to our wonderful partner, the Patient Empowerment Network, and also the Coalition for Clinical Trial Awareness and the Alliance for Patient Access.  And thank you to our sponsors, they all start with A, Astellas, Amgen and AbbVie.  They help make this program possible.

We have a lot to talk about in helping debunk the myths about clinical trials and hopefully raising awareness and understanding for you and your family, so you can consider a clinical trial and see whether it’s right for you.  And I can tell you, in so many areas of cancer now there’s exciting research going on. But if you want to get the possibility of tomorrow’s medicine today, and it happened for me with chronic lymphocytic leukemia, being in a Phase II trial way back in 2000, 10 years before the drug combination I received was approved.  I know it was life-saving for me.

And I want you to meet our first guest.  It was life-saving for him, and that is Pat Gavin.  Pat joins all the way from Marne, Michigan, which is outside Grand Rapids.  Pat, thank you so much for joining us on this program.

Pat Gavin:

Thanks for having me, Andrew.  Glad to be here.

Andrew Schorr:

Now, Pat, I want to go over a little background about you.  I believe that you’ve been treated for three cancers, right?  Pharyngeal head and neck cancer, in 2007, right?

Pat Gavin:

Right.

Andrew Schorr:

And also you were treated for melanoma 2008, and then in 2014 prostate cancer.  Now, you were in a Phase II trial for that pharyngeal head and neck cancer.  Do you believe it made a big difference for you?

Pat Gavin:

Well, that trial is absolutely the reason I’m here today.  My oncologist described it as we had the experience of witnessing a miracle.

Andrew Schorr:

Let’s meet one of our medical specialists who is joining us, who has been on our Patient Power programs before and our lung cancer programs, and that’s Dr. Charu Aggarwal.  She joins us from Penn Medicine, the Abramson Cancer Center in Philadelphia.  She’s a lung cancer specialist and also a head and neck cancer specialist, very active in trials.  Dr. Aggarwal, thank you for joining us.

Dr. Aggarwal:

Thank you, Andrew.  Thank you for having me here.  I’m delighted to be part of this program.

Andrew Schorr:

Dr. Aggarwal, you have a lot of research going.  It takes patients wanting to participate for us to ever have approved medicines, right?

Dr. Aggarwal:

Absolutely.  And I think that’s key in clinical trial participation, to get drugs to patients early.

Andrew Schorr:

All right.  And certainly in the area of lung cancer and many other cancers now there’s a lot happening, and smart researchers like Dr. Aggarwal are trying to prove some things that really seem like they would make a lot of sense, but we patients have to participate, be their partner.  I’ve seen that.

Dr. Aggarwal, lung cancer is a good example, but you’re an oncologist, and you see many different areas that are changing fast.  What would you say to patients about the opportunity, like I said, did it give me tomorrow’s medicine today?  Or, Pat, who feels he’s alive because of that.  You must see that a lot.  Doesn’t always work out, but it’s happening more and more, isn’t it?

Dr. Aggarwal:

It is definitely happening more and more.  Clinical trials are really accelerating our ability to get patients, like you said, tomorrow’s medicine today.  In the last five years, we’ve had upwards of eight to 10 drugs approved for lung cancer alone, and it would not have been possible without patients’ participation on clinical trials.

As we understand the biology of diseases better and as more medicines are available to us, the only way to access them and the only way to get FDA approval is through clinical trials.  And we’ve certainly seen that for lung cancer, but we’ve also seen that for head and neck cancer, and immunotherapy is now possible because of clinical trial participation.

Andrew Schorr:

Right.  And I’m living with two cancers, chronic lymphocytic leukemia, where there are many new drugs now, and now we’re looking at trials with combinations of new drugs.  And then I have another condition, scarring in the bone marrow, myelofibrosis, and I was very grateful that a new drug had been approved for that. And I’ve taken that drug now four-and-a-half years, a genetic inhibitor, and I’ve met patient number one, who is in that trial, and I give him a big hug.

Now, Pat, what do you think are some of the myths?  You know, you meet people all the time.  What do you think are some of the things that people just think are true but really aren’t?  Maybe you could tick some off.

Pat Gavin:

Well, one of the big myths out there is that there’s going to be a placebo arm, and there are not placebo arms to treatment trials, unless the standard of care would be a wait and watch, which is relatively rare.  So you’re always going to get either standard of care or a combination that includes standard of care and the test drugs or the—test drugs.  You’re never going to be left out there with just taking a sugar pill.

Andrew Schorr:

Right.  So let me go over that with Dr. Aggarwal.  People I think are—have heard about trials for other illnesses, but we’re talking about cancer now.  Your patients don’t get just a little white pill with nothing in it, right?  They either get quality care, standard of care, or they get something new.  Is that correct?

Dr. Aggarwal:

That’s correct.  So the era of placebo?controlled trials is almost over, and I say almost because in the metastatic setting or in the stage IV setting or incurable setting we almost always never use placebo anymore.  We are either randomizing patients to standard of care or meeting standard therapy, the chemotherapy, be it a pill, be it targeted therapy or immunotherapy, and we compare patients to that approach and introduce the experimental approach on the other hand.

Now, if there are patients that have standard of care as observation, then, of course, that observation arm does become our randomized arm.

Andrew Schorr:

Okay.  And may I ask what Pat was taking, or like I know in leukemia we have people who are in watch and wait.  So we have some people who are in watch and wait, okay.  So I get that.

Pat, what’s another myth, do you think?  So one was where you get a placebo, so we heard no.  So what’s another myth, do you think?

Pat Gavin:

I think there’s always a feeling that I’m going to be just a guinea pig, and that’s the one thing I think I hear most often from people is I don’t want to be a guinea pig.  I want to make sure that I’m getting a treatment and not being exposed to things that are unsafe.  Of course, there’s always a certain amount of risk with any trial that we participate in, but the chances of some of the things happening that you might see on the comedy TV shows just aren’t going to be there.

Andrew Schorr:

Okay.  Dr. Aggarwal, let’s go over that.  So, first of all, you’re at a major university center, University of Pennsylvania and Abramson Cancer Center.  What sort of panels in decision?making of smart minds are there going to whether you’re even going to go ahead with a certain trial?  I think you call it an investigational review board.  Tell us a little about the process of deciding whether you’re going to have a trial at your institution at all.

Dr. Aggarwal:

Yes.  So there’s a very thorough review of clinical trials, and these are vetted through several committees both in terms of ethical review as well as scientific review.  And, you know, when my patients say to me I don’t want to be a guinea pig, I really try and figure out what is it about the trial that they don’t want to do?  Is it the fact that they don’t want to get the investigational drug, or is it the number of tests that are involved in association with receiving that drug?

And I think, you know, most of the time, 80 to 90 percent of the time, I’m able to answer patients’ questions and concerns regarding their guinea pig concern, and most of the times actually it’s related to the fact that they don’t want to undergo extra tests or procedures that they wouldn’t have otherwise.

As soon as they hear that this is actually a drug where it may benefit them and they’re not just going to get a sugar pill, most patients are actually interested in clinical trial participation, because they’re here to really help themselves and to get something that can help their cancer.

Andrew Schorr:

So, Pat, another concern—well, I guess one limitation of people being in trials is people don’t even know about them, you know, not only don’t understand what a trial is but have not even been told that it’s an option, and that’s a problem in the U.S. today, isn’t it?

Pat Gavin:

Absolutely.  I even think it was a problem for me.  I didn’t know that a trial was going to be available in my home town.  If it wouldn’t have been for my oncologist recommending it to me, I probably wouldn’t have joined.  Fortunately, today I think patients are getting more knowledgeable about trials that are out there, and they’re hearing more and have the interest in joining a trial, and they’ll recommend it to their oncologists and tell them that they are interested.  But not knowing about them is a big problem.

Andrew Schorr:

Okay, Pat.  So for our viewers today, what question or questions would you urge cancer patients or family members to ask today so that they have the awareness of trials that might be right for them?

Pat Gavin:

Well, the first thing I would do is I would offer to my oncologist that I’m interested in being in a trial.  And I would ask what type of trials are available for people with my cancer, and what would you recommend as far as the trials that you see out there that you think is right for what I’m facing today?

Andrew Schorr:

Okay.  All right.  Well, now joining us I think is Mary Ellen Hand, who has been at the Rush University Medical Center in Chicago for many years and also works with lung cancer patients but has been in oncology for many years.  Mary Ellen, first of all, thank you so much for being with us.

Mary Ellen Hand:

You’re welcome.  Sorry for the technical difficulty.

Andrew Schorr:

It’s okay.  Thank you for being with us, Mary Ellen.  So from your point of view, what’s a myth that comes up a lot for people?  We’ve been talking a little bit with our other guests about whether with you get a placebo, no, whether you’re a guinea pig, no.  Are there other myths that you can think of that you really want to talk about now?

Mary Ellen Hand:

I think that people sometimes come to this thinking I don’t want to do something because I don’t—as you’re saying, a guinea pig or be in uncharted territory as opposed to having an opportunity to have a therapy that may be more impactful in their disease and help control their cancer better.

And, secondly, I will have people who have an out?of?network insurance or something that doesn’t allow them the flexibility to maybe even come to our institution or somewhere else for their therapy, and they think cancer trials are free care, anything you get if you’re on a trial is free.  And what is true is that ordinary customary charges for things like blood tests and scans and doctor appointments and the medicines that are approved are billed to your insurance, and what the company might provide that’s being tested would be the thing that’s provided free to you.  And so I think that that’s a misconception that many people have.

Andrew Schorr:

Okay.  Now, can a major medical center like yours help a patient discover the financial issues related to them, maybe even work with their insurance company to see are there options for them related to being in a trial?

Mary Ellen Hand:

I think over the last couple years in particular things have become much more complicated for people.  You know, some people sign up for Medicaid or Medicare replacement policies in the different states.  There’s Medicaid with places—Medicaid policies that don’t allow people flexibility.  But certainly that’s our job is to help people find out where they could go and if they’re eligible for a trial to help them get to that trial, and some of that is people who have—fit a particular niche.

And some people need to be well enough to travel, you know, if they need to—if the trial is out of our ZIP code.  Here in Chicago we’re very collegial in head and neck cancer and lung cancer and, you know, multiple other cancers.  You know, if the trial exists five miles from here, we’ll facilitate the patients getting on that trial there.

I think that medical records, one of the many—one of the most common medical records systems is available at many institutions across the country, so people can have access to the reports for another hospital.  Otherwise, there are coordinators and people who can make sure that all of that gets to the research nurse and gets in the hands of the person who is going to take care of that.

And then at our hospital, and I’m sure across the country, we make sure that they get the imaging so that they have something to compare it to, and then that’s uploaded into your chart, you know, at the other facility so that everybody has the right information to take care of the patients.

 

Andrew Schorr:

Okay.  Dr. Aggarwal…

Dr. Aggarwal:

I would just add…

Andrew Schorr:

Go ahead, please.

Dr. Aggarwal:

I would just add that this is a very common concern about the financial responsibility for clinical trials.  And here at Penn we are actually trying to make this process very, very transparent so that when I discuss a clinical trial with a patient actually our consent forms reflect what will be the standard of care costs and what will be sponsored by the clinical trial.

And, in fact, we do facilitate meetings with a financial counselor so that if a patient has concerns about what will be covered versus what will not be covered will be discussed at length with a financial counselor.  And that actually has gone a long way in allaying some of the concerns that patients have when going on clinical trials.  So, you know, it goes hand in hand with what Mary Ellen was saying, that I think once patients hear from the oncologist that there’s another level of—from a finance person I think that really goes a long way.

And I would urge patients to actually discuss and ask the facility where they’ll be treated if there is such a person who can discuss with them, because most academic cancer centers do have this facility.

Andrew Schorr:

So many people are treated, you know, by a local oncology clinic, but often they can work in partnership with an institution like yours, Chicago, Philadelphia, others around the country.  How does that work?  How can that work where they can be in your trial but maybe some testing or some other things, or do they have to commute to your institution maybe from a distance all the time?  Let’s start with you, Dr. Aggarwal.  Can there be more partnership, or are more trials available now in the community as well?

Dr. Aggarwal:

So a lot of partnerships exist between community physicians as well as academic physicians, so I see patients for my community oncology colleagues all the time, and the goal really is to make access easier, you know, the access to clinical trials and drugs easier.  So while the administration of the drug and the monitoring of the drug may happen at the academic center, there are many tests and imaging procedures that can occur in the community.

And the goal is also to make this easier for patients.  So if a patient is 25 miles away, I try not to drag the patients here just for a clinical exam or just for a scan.  You know, so I would facilitate them getting scans closer to home with their outpatient oncologist and then ask them to perhaps bring a CD with them for review.  They can get their blood work done closer to home.

So there are lots of things and lots of procedures where we work synergistically with their community physician hand in hand to try and facilitate all of these procedures so that they don’t have to keep traveling all the time.  So we certainly do that.

Andrew Schorr:

In Chicago, too, Mary Ellen?

Mary Ellen Hand:

Certainly.  You know, there are some things that the study requires.  If an infusion needs to be done onsite, that’s what happens.  You know, we have patients who travel across the country that might have a genomic mutation.  They may be looking for second or third generations of drugs, and so those people may travel.  So they have their local oncologist, meaning near, whether that’s someone in the community or someone in the academic center.

I think that’s another thing, is that patients are concerned that their doctor, whom they’ve forged this relationship with and the nurse, they think they will be upset if they go somewhere else.  And then instead of knowing that it’s a great opportunity for us to advance the body of knowledge but it’s also—we’re always encouraging and hopeful that people can get onto a clinical trial.

And so I think it makes them feel really good that people have these connections.  I think they like to know they’ve talked, they like to know that everybody’s on the same page and this many more layers of care take care of that.

Andrew Schorr:

Pat, let’s pick up on that.  So…

Mary Ellen Hand:

Their problem, their knowledge, all of us together, so.

Andrew Schorr:

Right.  Well, Pat, let’s talk about that.  So people have a doctor, maybe the one who diagnosed them, and they have a close relationship with them, and they’re afraid of losing them.  What do you say to people?  Mary Ellen spoke about that but from your perspective.

Pat Gavin:

Well, I think it depends somewhat on the trial and where they’re going to be available.  I received all of my care through the clinical trial locally at the Lacks Cancer Center here in Grand Rapids.  It was a trial like many others that are in the national clinical trials network, and the NCI-sponsored trials are generally available at the NCORP sites, and there are a lot of those around the country.  I was fortunate to have one of the original ones here in Grand Rapids by the Cancer Research Consortium, and those trials are available in academic centers, they’re available in community cancer centers like I had.  So it depends on the trial.

Now, some of the pharma trials may be a little more isolated and localized to specific hospitals for some of their early?phase trials.  Talk to your oncologist again.

Andrew Schorr:

We are getting questions.  And so I mentioned I have chronic lymphocytic leukemia.  This came in from George, who also has CLL.  He said, I’ve had no treatment, but it’s likely that it will be needed very soon, and it seems that Medicare patients are treated somewhat unfairly when it comes to available financial assistance for oral chemo, oral drugs that are now in trials often, Mary Ellen.

And so he says, are we likely to run into problems with clinical trials if you’re on Medicare?  So, Mary Ellen, any guidance about that, Medicare patients and trials?

Mary Ellen Hand:

No.  I think that we’re an aging America, so I think that we want to be sure that patients who are on Medicare have access to clinical trials.  So I think what he’s speaking particularly to is the co?pay of some of these medications is so very high, and co?pay assistance programs are not always built to support and help them in this.  But I think that if he’s going to be eligible for a clinical trial, he should, you know, number one see if he’s eligible. And then hopefully the place that he’s at will be able to help navigate that for him so that he can be—you know, be eligible to participate in that trial.

Andrew Schorr:

Okay.  One other question, Dr. Aggarwal.  This one comes from Stacey.  Stacey also has leukemia, and we’ve had oral drugs being developed a lot now for various leukemias, this is CLL.  And so there’s a clinical trial underway combining two of these oral agents, ibrutinib (Imbruvica) and venetoclax (Venclexta), is underway at MD Anderson in Houston, and the same trial is supposed to begin at Northwestern in Chicago near where Mary Ellen is, and that’s in May.

And she says since there’s a four? to five?month period before the introduction of venetoclax following ibrutinib, what would be the chance that I could join the trial at Northwestern in May, or would this be something I would have to direct to the doctor leading the trial?  She’s wondering about since the drugs get combined but sort of one after the other, can you sort of start, and how does that work?

Dr. Aggarwal:

So I would say that each clinical trial is different in terms of how they’re designed and what eligibility criteria are outlined.  I would really encourage participation—or I would encourage her to speak with this—speak about this trial with a physician or contact the PI of the clinical trial at MD Anderson…

Andrew Schorr:

Principal investigator.

Dr. Aggarwal:

…principal investigator to try to get some clarification of that.  There are some trials that prohibit previous therapies or previous ibrutinib, for example, prior to enrolling in a combination clinical trial, and there are some trials that allow prior participation.  In some instances, they may see progression on ibrutinib prior to combination therapy that is ibrutinib and venetoclax.  So I think it’s a matter of finding what the check boxes on the trial are, and talking to the principal investigator would be the best way to go about it.

Andrew Schorr:

Okay.  Here’s a question for Pat.  So, Pat, one of the things I wonder about is there are people who are on modern therapy now like for instance some of these drugs we mentioned, people are doing well on ibrutinib or people are doing well on venetoclax, or people are doing well on some of the lung cancer drugs.  Now, none of us know how long they’ll last, so they say, well, why should I even think about trials if it’s not broken now?  What would you say to them?

Pat Gavin:

We have to as patients look at clinical trials as a form of treatment, and it should be something that should be considered right from the beginning.  I hear people saying that, well, I’m going to go with what I’ve got so far, and if that doesn’t work and nothing else is an option, then I’ll use it as a last resort.  Now, in some cases a clinical trial may be a last resort, but in other cases, like you mention, there may be things about early treatment that would disallow you from participating in a clinical trial later on, so it’s best to be talking about clinical trials as an option right from the beginning.

Andrew Schorr:

Well, you know, this is a series we’re doing, and so we’ve covered some ground today, and I just want to recap a couple of things.  We talked about the phases of trials.  We talked about financial issues, and there are counselors to help you related to that.  Pat and I told the story of each of us thinking we wouldn’t be alive if we hadn’t been in a trial.  We talked about genomics.  So we’ve covered a lot in our first one, and we have another program coming up late in June.  Dr. Aggarwal, was this a good start?

Dr. Aggarwal:

This was an excellent start.  I think we definitely look forward to more patient participation on further trials, further programs like this.

Andrew Schorr:

Okay.  And, Mary Ellen, do you think we made a good start today, and hopefully people will now consider trials?

Mary Ellen Hand:

I think that it’s always just good to have more education, so whatever venue we can give that to people, whether it’s talking one on one with their physician or nurse or whether it’s online, to give people permission that they can get more information.

I think the other important thing to know is the criteria we talked about is you can’t—if you are truly getting a second opinion, you should get it before you start something, because you don’t want to have started a treatment that you get one dose of something that blocks you from access to a clinical trial.  Or you don’t want to have your genomic testing done yet, and yet you’ve started chemotherapy.  So sometimes it’s just educating people that, you know, if they’re not very sick, there’s time to get more information.

Andrew Schorr:

Good, good point.  Pat, what’s a final comment from you?  What do we want to leave people with, hopefully have more people think about trials, get access to them and have greater participation?

Pat Gavin:

Well, every time I talk I say I’m alive today by the grace of God and the fact that I participated in a cancer clinical trial.  They make a difference.  They’re the reason why you and I are alive today.  They need our support.  If clinical research is going to advance, we have to have patients in clinical trials.

Andrew Schorr:

Right.  So if we want progress and cures for the cancers that we’re living with, we’ve got to work with folks like Mary Ellen, Dr. Aggarwal and the other folks involved in research around the world, really.

Well, I’m over here in Europe, today in Barcelona.  This is a worldwide broadcast we’re doing.  Pat Gavin, I want to thank you so much for joining us from near Grand Rapids and wish you good health, Pat.  Thank you for being with us.

Pat Gavin:

Thanks.

Andrew Schorr:

And, Mary Ellen Hand, thanks for joining us again on our programs and in Chicago and 34 years of devotion to us, Mary Ellen, you keep going.  Thank you for being with us.

Mary Ellen Hand:

You’re welcome.

Andrew Schorr:

Okay.  Dr. Charu Aggarwal from Penn Medicine, the Abramson Cancer Center in Philadelphia, thank you for being with us again on one of our programs.  And thanks for the research that you’re moving forward with and your devotion to patients with cancer.  We hope that—well, we know it makes a big difference, and we look forward to you having great discoveries in partnership with patients moving forward.

Dr. Aggarwal:

Thank you for having me.

Andrew Schorr:

Great program.  Our next program will be coming up on June 21st, and we’re going to discuss are clinical trials a gamble?  So how do you decide as a patient, you and your family?  We’ll talk about that in June.  Thank you so much for being with us.

Paying It Forward: Volunteering for Clinical Trials

Editor’s Note: This blog and video is from the Alliance for Aging Research. The Alliance for Aging Research is dedicated to accelerating the pace of scientific discoveries and their application to vastly improve the universal human experience of aging and health.

Getting medical discoveries from the research lab to patients depends on clinical trials and the people who volunteer to participate in them.   Volunteering in a trial may help society at large by bringing new treatments one step closer to patients, and could help a loved one if you have a genetic disease or condition.  Volunteering may also give you access to a cutting-edge treatment and medical team that carefully monitors your health.  But clinical trials can’t happen without volunteers, and 37% of trials don’t enroll enough patients to move forward.  Clinical trials need volunteers like you so watch this short film to find out more about why they are important, how to get involved, and what it means to participate.

How to Read and Understand a Scientific Paper

In a previous article, How to Read Beyond the Headline: 9 Essential Questions to Evaluate Medical News, I recommended you should always try to read an original study (if cited) to evaluate the information presented. In this follow-on article, you will learn how to read a scientific research paper so that you can come to an informed opinion on the latest research in your field of interest.  Understanding research literature is an important skill for patient advocates, and as with any skill, it can be learned with practice and time.

Let’s start by looking at what exactly we mean by the term “scientific paper”. Scientific papers are written reports describing original research findings. They are published in peer reviewed journals, which means they have been refereed by at least two other experts (unpaid and anonymized) in the field of study in order to determine the article’s scientific validity.

You may also come across the following types of scientific papers in the course of your research.

•       Scientific review papers are also published in peer reviewed journals, but seek to synthesize and summarize the work of a particular sub-field, rather than report on new results.

•       Conference proceedings, which may be published in a journal, are referred to as the “Proceedings of Conference X”. They will sometimes go through peer review, but not always.

•       Editorials, commentaries and letters to the editor offer a review or critique of original articles. They are not peer-reviewed.

Most scientific journals follow the IMRD format, meaning its publications will usually consist of an Abstract followed by:

•       Introduction

•       Methods

•       Results

•       Discussion

 

Let’s look at each of these sections in turn.

(a) Introduction  

The Introduction should provide you with enough information to understand the article. It should establish the scientific significance of the study and demonstrate a relevant context for the current study.  The scope and objectives of the study should be clearly stated.

When reading the Introduction, ask yourself the following questions:

·       What specific problem does this research address?

·       Why is this study important?

(b) Methods

The Methods section outlines how the work was done to answer the study’s hypothesis. It should explain new methodology in detail and types of data recorded.

As you read this section, look for answers to the following questions:

  • What procedures were followed?
  • Are the treatments clearly described?
  • How many people did the research study include? In general, the larger a study the more you can trust its results. Small studies may miss important differences because they lack statistical power. Case studies (i.e. those based on single patients or single observations) are no longer regarded as scientific rigorous.
  • Did the study include a control group? A control group allows researchers to compare outcomes in those who receive a treatment with those who don’t.

 (c) Results

The Results section presents the study’s findings.  It should follow a logical sequence to answer the study hypothesis.  Pay careful attention to any data sets shown in graphs, tables, and diagrams. Try to interpret the data first before reading the captions and details.  If you are unfamiliar with statistics, you will find a helpful glossary of terms hereClick here for an online guide to help you understand key concepts of statistics and how these concepts relate to the scientific method and research.

Consider the following questions:

  • Are the findings supported by persuasive evidence?
  • Is there an alternative way to interpret these findings?

(d) Discussion 

The Discussion places the study in the context of the broader field of research. It should explain how the research has moved the body of scientific knowledge forward and outline the next steps for further study.

Questions to ask:

•       Does the study have any limitations? Limitations are the conditions or influences that cannot be controlled by the researcher.  Any limitations that might influence the results should be mentioned in the study’s findings.

  • How are the findings new or supportive of other work in the field?
  • What are some of the specific applications of the study’s findings?

The IMRD format provides you with a useful framework to read a scientific paper. You will need to read a paper several times to understand its findings. Consider your first reading of the study as a “big picture” reading.  Scan the Abstract for a summary of the study’s principal objectives, the methods it used and the principal conclusions. A well-written abstract should allow you to identify the basic content of an article to determine its relevance to you.  In describing how she determines the relevance of a study, research RN, Katy Hanlon, focuses on “key words and phrases first. Those that relate to the author/s base proposal as well as my own interests”.  Medical writer, Nora Cutcliffe, also scans upfront “to gauge power and relevance of clinical trial data”. She looks for “study enrollment (n), country and year”. It’s important to note the publication date to determine if this article contains the latest findings or if there is more up-to-date research available. Cutcliffe also advises you should “note author affiliations and study sponsors”.  Here you are looking out for any potential bias or vested interest in a particular outcome.  Check the Acknowledgments section to see if the author(s) declare any financial interests in the research which might bias their findings. Finally, check if the article is published in a credible journal.  You will find reputable biomedical journals indexed by Pubmed and Web of Science.

Next, circle or take note of any scientific terms or keywords you don’t understand and look up their meaning before your second reading. Scan the References section – you may even want to read an article listed here first to help you better understand the current study.

With the second reading you are going to deepen your comprehension of the study. You’ll want to highlight key points, consult the references, and take notes as you read.  According to the scientific publisher, Elsevier, “reading a scientific paper should not be done in a linear way (from beginning to end); instead, it should be done strategically and with a critical mindset, questioning your understanding and the findings.”  Scientist, Dr Jennifer Raff, agrees. “When I’m choosing papers to read, I decide what’s relevant to my interests based on a combination of the title and abstract”, she writes in How to read and understand a scientific paper: a guide for non-scientists. “But when I’ve got a collection of papers assembled for deep reading, I always read the abstract last”. Raff explains she does this “because abstracts contain a succinct summary of the entire paper, and I’m concerned about inadvertently becoming biased by the authors’ interpretation of the results”.

When you have read the article through several times, try to distill it down to its scientific essence, using your own words. Write down the key points you have gleaned from your reading such as the purpose of the study, main findings and conclusions. You might find it helpful to develop a template for recording notes, or adapt the template below for use. You will then have a useful resource to find the correct reference and to cross reference when you want to consult an article in the future.

In the example below I have taken an article published in 2015, as an example. You can read the paper Twitter Social Media is an Effective Tool for Breast Cancer Patient Education and Support: Patient-Reported Outcomes by Survey on PubMed.

Template for Taking Notes on Research Articles

 

 

Further reading

How Do You Find Out About Clinical Trials?

Interview with Larry Anderson, Jr., MD, PhD, Assistant Professor, Department of Internal Medicine, Division of Hematology/Oncology University of Texas Southwestern Medical Center, Patient Advocate, Lynette, and Robert Orlowski, MD, PhD, Director of Myeloma and Professor in the Departments of Lymphoma/Myeloma and Experimental Therapeutics The University of Texas MD Anderson Cancer Center

From  the Virtual Town Meeting: Understanding the New World of Myeloma Treatment, Andrew Schorr first ask Dr. Anderson about how patients can find out about clinical trials, whether that be a governmental website, advocacy groups, or each institution’s individual website. Later he gets Lynette and Dr. Orlowski’s opinion on the matter. Check out the full video below to hear from three myeloma experts.

How Do You Find Out About Clinical Trials? from Patient Empowerment Network on Vimeo.