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.

Fact or Fiction? Lung Cancer Symptoms, Side Effects & Treatment

Fact or Fiction? Lung Cancer Symptoms, Side Effects & Treatment from Patient Empowerment Network on Vimeo.

When it comes to lung cancer information you find online, how do you decipher fact from fiction? Dr. Martin Edelman, a renowned lung cancer expert and researcher, shares his insight and expertise on symptoms, side effects and treatments for lung cancer.

Dr. Martin J. Edelman is Chair of the Department of Hematology/Oncology and Deputy Director for Clinical Research at Fox Chase Cancer Center. More about this expert here.


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Transcript:

Patricia:                      

Welcome to Fact or Fiction: Lung Cancer Symptoms, Side Effects, and Treatment.

Today, we’ll debunk common misconceptions about lung cancer symptoms, side effects, and treatment. I’m Patricia Murphy, your host for today. Joining us is Dr. Martin Edelman. Dr. Edelman, why don’t you introduce yourself.

Dr. Edelman:              

So, I’m a medical oncologist. I’m the Chair of the Department of Hematology/Oncology and Deputy Director for Clinical Research at the Fox Chase Cancer Center in Philadelphia, Pennsylvania.

Patricia:

And before we get started, we should say this program is not a substitute for medical advice. Please refer to your healthcare team with any questions.

Let’s start with an overview of lung cancer’s research. Can you tell us a little bit about the field right now?

Dr. Edelman:              

So, I think the field has been remarkable over the last few years. There’s been more progress, more drugs, more things that have happened in the last five years than probably the prior 50. It’s been an amazing time both for developments in microbiology as well as in immunotherapy of the disease, which is exciting for all concerned.

For patient’s, of course – really a promise of longer, better lives, even cures where we previously did not see any in advanced disease. For the scientists – an amazing amount of new information. And for clinicians and clinical investigators – just almost too many questions for us to answer.

Patricia:                      

It sounds like the field is really advancing quickly. What do you attribute that to?

Dr. Edelman:              

Well, you know, I think there are a number of things. Everybody always talks about breakthroughs, but breakthroughs really happen after decades of other work. And what’s happening now is really a result of many, many years of different types of work. There were our colleagues in immunology who built this area of cancer immunology for many years – I have to say with much skepticism from many, myself included.

The advances in molecular biology – our abilities to do things with tumors to determine genetics at a rate and a pace and a cost that was previously unimaginable. All of these things have developed in the last few years but really are a result of the decades of work before that. If you look at immunotherapy – probably one of our biggest areas of progress – the roots of that are a century old. So, nothing’s really new. It’s just now we have the technology and the ability to really use it. And then I would also say that we’ve created the infrastructure that lets us test this – the people who have done the studies, the endpoints for the studies, the expertise in doing clinical trials – that also was there for decades, and we frequently were kind of ridiculed at times.

Oh, you’re just testing this drug against that drug, but the reality is is it was those incremental advances. It was the ability to know the endpoints, to refine the populations, to develop the infrastructure that allowed for all of this to happen.

Patricia:

How is genetic testing changing the landscape?

Dr. Edelman:

So, genetic testing – and in this case the testing of the tumor, not the germline, not the individual – has been very, very crucial. If you go back about 20 years ago, there was a family of drugs called epidermal growth factor receptor inhibitors or EGFR inhibitors.

And the basic science at the time made it look like these would be best combined with chemotherapy in squamous cell carcinoma. And as it turned out, combined with chemotherapy they weren’t very useful. But as single agents, there were these occasional very dramatic results.

So, that came at a time when we were able to evaluate tumor DNA, sequence it with some degree of ease at a reasonable cost. So, there was a discovery of specific mutations, which were targeted by these drugs. So, it was sort of interesting in that it was the clinical observation that led to the discoveries in biology, not really the other way around.

But then that in turn resulted in looking for other mutations, which were found, and then the development of other drugs – in some cases, the repurposing of other drugs for those. And now we have about a half a dozen very validated targets, each one of which in a small slice of the population – between say 1 percent and 5 percent – 10 percent of the lung cancer population – but these – if the patient has within their cancer that particular mutation, these are drugs that are 80 percent-plus effective and frequently can be administered with relatively little toxicity.

And usually they’ll give them benefit for one-plus years or more. So, that’s been an example of progress there.

Patricia:

How does lung cancer generally present in people? What might someone notice?

Dr. Edelman:

So, when I teach my residents how do people show up, which is, of course, very different for me – they usually show up with the diagnosis in hand. But for somebody who’s a primary care physician, what are you going to see? Well, you could see symptoms at the site of the origin of the disease – in the lungs. So, the pneumonia that doesn’t go away, the cough that doesn’t go away, the chest pain. So, that’s one way that it can present. It can also present, unfortunately, all too frequently as advanced or metastatic disease where the tumor has spread to other organs in the body, such as bone or brain. So, you may have a pain or a fracture, seizure, headache. Those are all possibilities.

And then sometimes the tumor can secrete various factors. We see this particularly in small cell lung cancer where there are certain metabolic syndromes that can develop or neurologic syndromes as a result of hormones or antibodies that the tumor can secrete. These are called paraneoplastic syndromes.

And then tumors sometimes show up and increasingly so now that screening has been validated, and screening in lung cancer is every good if not superior to screening in breast cancer. There’s a common myth that it doesn’t work. But in fact, this has been now demonstrated in multiple randomized trials done in the United States, in Europe that clearly demonstrate improved outcomes for patients who are at risk who undergo screening exams with low-dose CT.

So, frequently, we see those patients and then again sometimes just incidental discoveries when somebody’s getting a scan for another reason. So, those are all the ways that it can present.

Patricia:

So, it sounds like we’re very good at getting people to doctors like yourself who can specialize in their disease once it’s diagnosed.

How are you approaching treatment decisions with your patients?

Dr. Edelman:              

Well, the treatment decisions that we make – that I make are those that are in ways similar to other medical oncologists. It really depends because some of the patients may first go to a surgeon or whatever. However they come into the system, there are a few key factors in this. First is – make your decision based upon, Number 1, which kind of lung cancer. So, there are two major varieties. You have small cell and non-small cell, and they are treated – they are biologically distinct, and they are treated in distinct ways.

And then the next major consideration is the stage of the tumor, which is our way of expressing how advanced that is and deciding on both the therapy as well as conveying a prognosis and evaluating a patient for a clinical trial. And that’s based upon the size and location of the tumor; presence, absence, and location of lymph nodes; and the presence or absence and, these days, the number of metastatic areas of disease.

And then, lastly, and again depending a little bit upon the stage and interacting with all the others is what condition is the patient in? Anybody can get lung cancer, but still the median is in older individuals. Many of these patients have compromised cardiac and pulmonary status as well as other diseases of aging, hypertension, cardiac disease, etcetera. Those people – one obviously has to tailor one’s treatments to fit those comorbidities. So, that’s sort of how the basic assessment – obviously, some patients show up with metastatic disease. We know that, but we go through a whole process for this.

The staging system that we use is complicated, and it keeps changing. We’re, gosh, up to version eight of this? I started with version three. I’m not quite sure I’ve fully mastered the current one, and the ninth edition is coming soon. And why does it keep changing? Because our knowledge of the disease keeps changing. The database keeps expanding.

We’re able to be more refined. Molecular variables have not yet fully entered into our considerations. Unquestionably, they will. But basically, one could consider lung cancer – despite the four major stages and multiple substages – that you really have three buckets that people will fit into. They have localized disease, which we will predominantly address with a localized therapy – surgery, radiation. And many of those patients, however, particularly those who might have a lymph node that’s positive, will benefit from chemotherapy to prevent recurrence.

We have patients with locally advanced disease. Primarily, those are patients who have lymph nodes located in the middle of the chest as opposed to more localized disease where if there’s a lymph node present it’s more in the lobe of the lung. Those patients with lymph nodes in the middle of the chest or larger tumors are approached with frequently a combination of chemotherapy, radiation, sometimes surgery.

And then we have patients with advanced disease who will be predominantly treated with drug therapies, which nowadays, depending upon the molecular background of the tumor, could be a targeted treatment if they have a specific mutation.

Something we see most frequently, though certainly not exclusively, in patients with scant or no smoking history, they may be approached with immunotherapy or chemotherapy combined with immunotherapy.

And there are many considerations that go into those decisions. And even in advanced stage, there are certainly roles for surgery and radiation depending upon whether there are structural abnormalities, occasionally whether there are relatively few areas or several areas of metastatic disease. And in the localized and locally advanced disease, our goal is cure in those, though we certainly are not there for every patient yet.

And in advanced disease, it’s extension of life, which is now quite considerable compared to untreated disease. And I think in certain situations, particularly those who only have a single area of metastatic disease, curative treatment is a realistic possibility. And even those with more disseminated disease, we’re now beginning to see a substantial fraction of patients who are still alive at five years or more. So, we’re beginning very cautiously to think that perhaps some of those patients may even be cured of their disease, though I’m not quite ready to say that.

Patricia:                      

Well, it sounds, though, like there is a lot of reason to have hope if you are diagnosed with lung cancer, especially if it’s diagnosed early. Of course, that would not stop a patient from worrying.

So, I hope what we can do next is talk a little bit about some of the things we’ve heard patients say, and you can fact-check us on that.

Dr. Edelman:              

Sure.

Patricia:

This sounds like a real worrier. There are no new treatments in lung cancer.

Dr. Edelman:

Well, there’s nothing but new treatments in lung cancer. So, I’ve been involved in oncology, I think – let’s see. My fellowship was in the late 80s. That ended about 1990, so we’re about – what is it not quite 30 years later? Virtually every drug that I use was in development during my professional career. Just within the last few years, all the immunotherapeutic agents were developed. Within the last say 48 months, they were licensed. The targeted drugs are all new within the last 15 years or so. So, we’re pretty much nothing but new drugs in lung cancer.

And not just drugs, but also surgical techniques have proceeded from open thoracotomies in almost all patients to video-assisted thoracoscopic surgery, which is less morbid and gets the patient out of the hospital faster.

Radiation progressed from relatively low intensity radiation that was done where you drew it on x-ray. I can still remember that when I was a resident to now four-dimensional assessments and the use of intensity-modulated radiotherapy.

Perhaps a role – maybe, maybe not – for proton therapy in this situation; the use of stereotactic body radiotherapy for treatment of localized disease in patients who are medically unfit – I think we’re nothing but new therapies.

Our supportive care is massively better than it was 30 years ago. Nausea and vomiting, severe problems – it’s largely a thing of the past. We have extremely effective antinausea agents. I may disappoint some people by telling you that marijuana is not one of them. But the fact is is that many of those drugs were developed because the drugs 20 years ago, 30 years ago, were quite nausea producing. And it was heavily lung cancer folks across the country – my colleagues, Dr. Brower, Dr. Gandara, Dr. Einhorn, others – who are very involved in lung cancer, genitourinary malignancies, gynecologic malignancies, but we’re using what’s termed highly emetogenic chemotherapy. We developed many of the antinausea drugs. We were extremely concerned about this.

So, our drugs are better. They’re more active. They’re less toxic. We have better supportive care. We have better integration with other modalities, such as radiation and surgery.

There are still many, many questions with treatment. Many areas we can improve. Many things we don’t know, but it’s nothing but new therapies.

Patricia:                      

Your history as a physician and noticing all this change will likely help you advise patients who worry that their lung cancer diagnosis is a death sentence, which is something else that we hear from patients.

Dr. Edelman:              

So, life is a death sentence. It’s a little bit flippant, but I think that there are many, many bad diseases out there. And certainly, there is no good lung cancer. And I don’t want anybody to leave this and think – oh, everything’s rosy. It’s not. Though I do a lot of administration these days, I’m still in clinic. I see a fair number of patients, and the news is not always good. Not everybody responds. Not everybody benefits. And that’s why we still need to do the trials and advance what we’re doing both in terms of increasing the efficacy and decreasing side effects.

Having said that, we have many, many patients who are living excellent productive lives, able to make life events – anniversaries, birthdays, etcetera – who would not have otherwise been alive to do that. And as I said, there is an increasing fraction of cured patients where the disease is no longer at all an issue. But it’s one of those things – we don’t know until we try. And there is no shortage of bad things that can happen to people. Lung cancer is one of them. I think what we do have is increasing options for people that truly meaningfully improve their lives.

Patricia:                      

Sure. Here’s one I hadn’t heard until just now. Surgery causes lung cancer to spread.

Dr. Edelman:              

Yeah, that’s common in certain states. When I was in Maryland that was a biggie.

So, there’s a myth that the air gets to the tumor, and then it spreads. But that’s certainly not true. It certainly is possible that in a bad surgical procedure that disease can be spread, but I think historically what that was was in the days before we had as accurate of radiographic studies. So, it’s kinda interesting. I always say, “I’m not that old, and I began medical school before there were CT scans.” So, the way you would diagnose something was with a chest x-ray. That was your best chest imaging. And the brain you’d image with something called a pneumoencephalogram, which is – you don’t know what that is. Most people don’t, and they should be thankful for that. But we had no real way of knowing these things. So, what would happen is there would be a surgical exploration. They would say, “Well, it looks very localized.” But then you’d go in, and there was lots of disease all over the place.

And for the most part, that doesn’t happen anymore. Now we have CT/PET scans. We have MRIs. Patients before they go to surgery usually have had – our pulmonary physicians will usually have sampled the nodes in the middle of the chest, the mediastinum. So, it isn’t that there aren’t surprises, but there are far fewer. And certainly, a properly done operation should not spread lung cancer. I would emphasize the properly done operation. It is my strong belief that nobody should have surgery for lung cancer from other than a board certified thoracic surgeon who spends their time thinking about lung cancer, preferably in an institution with a fair volume of this.

We know – it should be no surprise to people, practice makes perfect. People who really focus in an area – people at the NCI-Designated Cancer Centers, comprehensive cancer centers – who do a lot of this have greater expertise.

Patricia:                      

How about this one? Treatment is not effective in older patients.

Dr. Edelman:              

Treatment is highly effective in older patients. It’s interesting. So, we had long arguments about, when I started in this field, whether treatment ever worked, and there were a number of studies that showed that chemotherapy – that one platinum was better – what’s called a platinum-based agent – was better than no therapy.

And then that two drugs were better than one drug. And people would say, “Oh, well, that doesn’t work in the elderly. And they should only get one drug.” And that’s because, I guess, their burning bush on the lawn told them this. And the fact is is that then got evaluated in a controlled trial, a very nicely done study by my European colleagues. But what was crucial was that they used somewhat lower doses of chemotherapy, a little bit different schedule of chemotherapy, and it was clearly superior to a single agent. And those were even days before immunotherapeutics and these targeted agents. So, many patients will benefit. You just have to be aware of certain basic principles in geriatric medicine as well as basic principles of lung cancer care.

So, first off, if the patient is elderly but their tumor is characterized by a driver mutation, they get one of the so-called targeted agents. And these are these days very non-toxic, easy to take, and highly effective.

Patients – many are going to be eligible for immunotherapy either as a single agent or combined with chemotherapy. Chemotherapy drugs could certainly be cut in their doses and still preserve much activity and be done safely.

I had a woman with small cell lung cancer. This is now about a year and a half ago or so. And she’s in her 80s. And she came to me because she was told – oh, just sorta get your affairs in order. And her disease was what we term an extensive small cell. The staging system’s a little bit different, but she didn’t have a really vast bulk of disease. And we treated her with standard chemotherapy drugs but at somewhat lower doses and some careful TLC and some other supportive things like growth factors.

She got all of her treatment on an outpatient basis, had an excellent response. We used radiation later to consolidate her treatment, and I see her back every couple of months. I wouldn’t say that she’s necessarily cured of her disease, but she does yoga every day. She lives a full life. She sees her grandchildren. And she’s, I think – I wanna say 83-84 years old. I think she’s quite grateful for that. It’s not the numerical age.

The flipside is if somebody’s 50 years old and they’re extremely ill when they come in, then one has to be very cautious about what one does. We used to say that those patients who come in who are severely impaired should simply get supportive care and hospice services.

And actually, how would I put it? Our lives have gotten a little bit more difficult lately because as things have gotten better for patients – because I can’t necessarily say that as much because some patients may be very susceptible to the effects of – their disease may be very susceptible to the effects of immunotherapy. I had one patient who was a younger gentleman who was on a gurney. He was in his 50s, lost an enormous amount of weight , he was on oxygen. We immediately gave him fluids. My fellow – I had an excellent fellow at the time – came to me and said, “Should we admit him and send him to hospice? Or just send him to hospice?” And I looked, and he had a biomarker that indicated that he might have an excellent response to immunotherapy, so we gave him solely immunotherapy and saw him back a few days later. He was still pretty touch and go. We gave him some fluids. A week after that – still, we were kinda touch and go, but he was still with us.

And then a week after that my medical assistant, comes in, and she says, “You know, he looks a little bit better today.” And he was in a wheelchair that day. And then a few weeks after that, he had a walker, and a few weeks after that a cane and about a year after that was asking me about whether or not he could go on a cruise. Again, I still see this gentleman – a couple weeks ago. It’s now almost two years later. And the question now that we have is – should we stop his treatment? And he is restored to complete full health, has had almost no side effects of treatment.

So again, this is not every patient. Some people will be treated and get every side effect and no benefit, but I think I’ve become a lot more reluctant to say that any patient should not at least be offered the opportunity for treatment knowing what the potential side effects are. And there still are considerable and sometimes severe side effects from therapy.

Patricia:                      

Yeah. And again, your experience and your long perspective on this disease can help you advise your patients thoughtfully. Here’s the last one that I have on my list here. Clinical trials are experimental and risky.

Dr. Edelman:              

Yeah. Well, so is the rest of life. So, there generally – is there risk? Yes. Essentially, every patient is always a trial because we for the most part don’t – even in the disease states where we have very active treatment – so, let’s say – for example, we were talking about the EGFR mutation. So, we have excellent drugs. We have a drug now, osimertinib – outstanding drug, easy to take, low risk of side effects.

The earlier generations – there was a lot of rash, diarrhea. That’s been pretty much done away with. But on average, patients benefit from this drug for about a year and a half.

So, that’s not great if you’re 40 or 50 years old. You want to do better. So, what are our current studies? Well, we’re looking – we’re re-addressing a question that we thought had been answered, but really it wasn’t – about, well, what’s the value of chemotherapy plus this drug? What about the value of other drugs?

So, we can’t promise anybody anything, but our current treatments are still not good enough. There are certain diseases, let’s say Hodgkin’s disease, where you know you’re gonna cure almost all the patients up front or testicular cancer, etcetera, where – again, but thanks to trials, clinical trials, we now are at that stage. We’re not there yet in lung cancer, and the reality is is every patient should really be on a study. I think it’s – and we have this problem now in that our studies have also become far more complicated to enter people in because there are many more variables one has to look at it. What’s the molecular background of the tumor? How many prior therapies?

The condition of the patient, their organ function, etcetera – and the regulatory burden has become much, much greater. But clinical patients are in clinical trials. Let’s look at the question. Are they risky? Well, everything is risky, but we do a lot to manage that risk. Patients who are in studies are observed more closely. We have to. It’s the law. There’s frequently additional personnel assigned. They’re usually getting standard of care plus a new treatment or a new treatment followed by the standard of care or some variation of that.

They’re observed, like I said, much more carefully than we would otherwise. And so, I think actually patients on trials generally will do better, and we actually have evidence. Multiple individuals have looked at this – everything from first-in-man trials or early dose escalation studies, controlled studies – that show that patients, even those on the control arm, generally do better than similar types of patients who are not treated on studies because we just are more careful.

And the physician who participates in trials is generally someone who has a greater knowledge of the disease.

Patricia:                      

Sure. What do you notice from your patients? What do they tell you that you think needs to be debunked?

Dr. Edelman:              

Well, very similar to some of the questions that you’ve asked. I mean we address these issues all the time about – is there hope with this? How bad is it gonna be, etcetera? Sometimes people think that inevitable diagnosis is gonna have pain and misery, etcetera, or a lot of admissions. I spend a lot of time particularly in their first visit addressing many of the questions that they may have.

And again, there’s always this problematic balance with the disease, particularly in the advanced setting in particular, where one has to balance out what is, I think, an increasingly positive picture with the reality that still the vast majority of patients will ultimately die of their disease, but the question is – how long can we put that off? How can we improve quality and quantity of life, even if one is going to ultimately die of the disease?

I think those are the things – there’s this weird dichotomy that people come to believe in that either you get treated and you’re gonna always have symptoms or your life will be pleasant and wonderful, and you’ll have this quiet wonderful peaceful demise if you’re not treated. And it’s really not true. The disease can be extremely uncomfortable, painful, distressing, etcetera. And treatment puts that off. Treatment prevents symptoms. Treatment improves quality of life.

And it takes a little bit of time because that’s how people are very socialized with this. Not every drug causes hair loss. Not every drug results in nausea. There’s too much misinformation out there.

Patricia:

Sure. Sure. Treatment can arrest the disease or slow down the progression of the disease, but it also has side effects.

Let’s talk a little bit about some of the concerns that patients have about the side effects. Let’s see: Side effects are unavoidable.

Dr. Edelman:

Well, that’s not true. As I said, what were the side effects? If you go back a couple decades and you ask patients what were they concerned about, many of them were concerned about nausea and vomiting. And that is largely a thing of the past. Many patients will still have some queasiness with treatment, but even our most nausea-producing drugs – we really do have outstanding drugs for the prevention of that. You have to use them. You have to take them.

It’s very important to give them appropriately. There are very excellent guidelines that are out there. Sometimes, patients are still undertreated, no question about that. Not every drug has industry strong backing. There’s one drug – for example, olanzapine, (Zyprexa) was actually developed as an antipsychotic, and I always tell the patients, “No, I don’t think you’re crazy.”

But it’s at a lower dose, and we have excellent, excellent evidence that that drug given for a few evenings after chemotherapy is extraordinarily effective along with the other drugs in preventing nausea and vomiting. So, that’s one thing.

Hair loss is still somewhat inevitable with certain drugs – the taxanes. But many of our regimens don’t cause hair loss.

Or as I tell folks – only you and your hairdresser will know for sure because its hair on the pillow, but the average person won’t pick you out of a crowd. Those are big concerns still. There still are potentially life-threatening effects from chemotherapy, and we spend a lot of time educating people about that. But those are not inevitable, and it’s actually a minority of patients in lung cancer.

One should not confuse – there are different malignancies. Still, the treatments for say leukemia, though even that’s changing, can be extraordinarily toxic or the bone marrow transplant patients. Many, not just lung cancer, but in the other diseases as well – many of the things that people attribute to the drugs are more due to the disease. So, I always say, “The greatest failure and side effects to the drugs are they don’t work well enough because the side effects of the disease can be considerable.” So, that’s the bigger issue. The immunotherapeutic drugs have a rather interesting set of side effects.

They are clearly initially or frequently better tolerated than the older cytotoxics, which still have an extremely valuable place in the treatment and cure of lung cancer. The immunotherapeutics have clearly been quite beneficial, but their side effects can be subtle and far less predictable and can be very severe. Virtually, any organ in the body can be affected by this. We like to say, “If it ends in ‘itis,’ you can get it from immunotherapeutics.”

So, there are lots of side effects, no question. But they can be managed. They can be prevented. They can be treated. Sometimes, we have to abandon a drug. So, people who get severe – what we call immunotherapy-related adverse events – may not be able to continue on their drugs. But even that is not necessarily always the case.

Patricia:

This next one really gets to the heart of the doctor-patient relationship.

I shouldn’t share my side effects with my healthcare team because I don’t want them to stop my treatment routine.

Dr. Edelman:

Well, you can’t prevent the side effects if you don’t know about them. And I always would tell patients, I said, “You know, if you’re having a problem, please don’t call me at 4:00 on Friday afternoon. I’m gonna end up sending you to the emergency room, which I may anyway.” But a lot of times, we can solve certain things over the phone. There are a lot of side effects that can be treated and particularly if one is aware early on. So, yeah, you should share the side effects because how’s somebody gonna know how to deal with them?

Now, the problem we run into sometimes is in a population that’s on average 60s and 70s, could be younger. There’re lots of things that can be just part of ordinary life. Everybody gets headaches, back pain, etcetera, etcetera.

We have to treat those sometimes and evaluate them much more aggressively because of the possibility of them being related to disease or drug, but it helps to sort it out. You can’t be too blasé about it because sometimes things need to be looked at very urgently, particularly with immunotherapeutic drugs. Some of the side effects that can be severe can sometimes be very subtle in their onset.

Patricia:

What else to patients talk to you about? What kinds of things do they come in and talk about that may need to be debunked or that you need to correct?

Dr. Edelman:

Well, it’s not contagious. It’s not hereditary, things like that. Many people – I’ll ask always about asbestos. And they’ll say, “Well, I worked in this old building that had asbestos in it.” Well, that doesn’t count, particularly one of the rarer – we’re not really talking about it today, but mesothelioma, which is associated with asbestos.

You know you gotta actually really be exposed, which means that somebody has to have torn into that. The latency is 30-40 years, so it’s the pipefitters; but actually, the most common cancer associated with asbestos is non-small cell lung cancer. It’s not mesothelioma. There are lots of those sorts of things. But in general, many of the questions you’ve raised are quite common questions.

Patricia:

As a patient, how can I differentiate between symptoms of lung cancer versus the effect of treatment? What should I be thinking about as a patient?

Dr. Edelman:              

It’s not always easy. And again, that’s why you gotta discuss it, and it’s not always easy for me to determine that because there are always several possibilities. It could be a side effect of treatment, it could be a side effect of disease, or it could be a side effect of people’s comorbidities. And these frequently interact. So, a patient – anemia is a common problem where you have low red blood cells.

Well, we know that you get anemia from disease. That causes a degree of what’s called anemia of chronic disease.

Our drugs frequently can result in anemia, and then anemia can bring out other symptoms. Patients who have lung and heart dysfunction to start with are gonna have more problems. They may get angina. So, there’re a lot of these things that interplay. And it’s not always straightforward.

Patricia:

And Dr. Google can really get involved here.

Dr. Edelman:              

Yeah. That’s always a problem, yeah.

Patricia:

Yeah. Which brings us to our next section – myths about lung cancer in general. How about this one? All lung cancer is the same.

Dr. Edelman:              

Well, I think by now one should be clear that not only is it not the same but even what we used to term – as I said, my life as a clinical investigator used to be a lot easier because we had non-small cell lung cancer. We had a particular stage.

And now we have EGFR mutated. We have non-small cell lung cancer that occurs in people without a driver mutation. And then, well, do they have something called PD-L1 expression? Which if it’s high, predicts for benefit from immunotherapy alone; and if not, then chemotherapy and immunotherapy is kind of the way to go in patients who are reasonable for that. We have patients who may have an EGFR mutation and then, which kind of EGFR mutation? Patients without mutations, ROS, RET, cMet. It goes on and on and on and on.

And all of these are different in small-cell lung cancer and then stage of disease. And even within the stages, there are all sorts of subtleties in terms of the optimal treatment. So, it really is a team decision for many of these patients how to treat them. And like I said, there are an increasing number of options.

And the answers are not always clear or perfect.

Patricia:

Right. How about this one? Lung cancer only affects the lungs.

Dr. Edelman:

Well, obviously, lung cancer can spread and kinda goes wherever it wants. There is essentially no organ in the body – I’ve had patients who were referred to me as “lung cancer” – rather who initially showed up with a breast mass and were seen by breast cancer physicians. They would biopsy it, and it was clearly lung cancer that had metastasized to the breast. Lung cancer can go to the eye then go to the brain, the skin, the adrenal glands, the liver. It’s a disease that unfortunately likes to travel and metastasize in the body very early in its natural history. In other words, when you say early in late lung cancer that’s not necessarily a time. It’s really low stage and high stage. You can see a lung cancer that can be a rather small tumor in the lung that may have already spread elsewhere in the body. 

Patricia:                      

Right. Right. How about this one? Supplements will help with symptoms and side effects.

Dr. Edelman:

Not likely, and more likely the other way around. So, as I said, we have some very good ways of preventing things like nausea and vomiting. There’s a lot of advice that is quite reasonable in terms of dealing with side effects – staying well hydrated. Hydration means salt-containing fluids – chicken soup, of course, being just about perfect or Pedialyte. Things like that are very good. But exercise is extremely good.

The problem with supplements is nobody really knows what’s in those. Many things can interact with various drugs. The term nutraceutical to me is nonsense. They’re unregulated drugs. And what do I mean by that? Many substances and many foods metabolize through the liver or influence enzymes in the liver. Many of our drugs are processed through the liver.

Drugs can influence – so, a drug that might inhibit the metabolism of a chemotherapy or a targeted drug will increase the body’s exposure to that. That can increase the side effects. Or alternatively, it can accelerate the processing of the drug, which will decrease the efficacy. I’ve seen this on many occasions.

One should think that much of the population is on anticholesterol drugs, cholesterol-lowering drugs called statins. Well, if you – I’m sure anyone who does it – you look at the bottle or you got the advice from the physician that says, “Don’t have it with grapefruit juice.” So, let’s think about that. If grapefruit juice can substantially increase the side effects from a very commonly utilized drug like a statin, just think about what an unknown thing that you bought – and remember, everything you buy at these stores – that so-called supplement – you have no idea what’s in it. There’re no standards for these.

The FDA is not really checking on those. I believe a few years ago the New York State Attorney General looked at this and found out a lot of these supplements were sawdust or weren’t what they say they were. So, I’m very – I would strongly discourage the use of anything outside of what’s actually a prescribed medication. If one wants to use an alternative therapy, like yoga, massage, image therapy, and again exercise, things that we know really work with people – absolutely, do that. But I would discourage these herbal medication supplements, etcetera. Or if you insist upon it, definitely tell your physician because then when they’re dealing with the side effects, it helps them to figure out what it was.

Patricia:

Yeah. Discussing what you’re taking or what you would hope to take with your physician and your care team is probably paramount.

Let’s talk a little bit about health literacy. What would you suggest patients use for online resources? What are good resources?

Dr. Edelman:              

So, there are some excellent resources. The International Association for the Study of Lung Cancer has resources for patients. The National Coalition of Comprehensive Cancer Center Network (NCCN) has resources. American Society of Clinical Oncology has resources. So, those or American Cancer Society. So, there are some really reliable sources out there. And there’s a great deal that’s very unreliable – people’s Facebook pages. I’ve seen this.

Patricia:                      

It’s a big place.

Dr. Edelman:              

Everybody always – and I think it’s important for people to understand. There will be people who will get something and have a fantastic response. I’ve used anecdotes.

The anecdotes I’ve used are to illustrate the potential hope of benefit. They’re not exceptions to the rule anymore. They’re the good case scenarios. I could have just as many anecdotes of people who didn’t benefit and stuff. And I think it is important going into this – and that’s why we are reassessing patients constantly and getting repeat scans because we don’t necessarily know always – even if something’s 90 percent effective, it means 10 percent of the time it’s not.

And each patient – we’re getting better at individualizing and personalizing therapy, but we’re not perfect yet. And we probably never will be. So, there will always be anecdotes. I think what’s – as a friend of mine puts it – the plural of anecdotes is not data. When I say, “Well, chemoimmunotherapy works.” It’s not because I have anecdotes of that, though anecdotes illustrate the magnitude of benefit.

I have data that shows that the chemoimmunotherapy regimen was compared to chemotherapy and was clearly and unequivocally superior. When I give a statistic that 60 percent of patients, 65 percent, can benefit from those types of regimens. That’s based upon prospective randomized control trials.

Patricia:                      

Dr. Edelman, as a researcher in the field, tell us why you’re hopeful about lung cancer research.

Dr. Edelman:              

Well, I think that we have gone from trials with very small incremental improvements and frequently a very slow degree of progress where if we had a positive study every two or three years, we were thrilled – to the point where we’ve had an avalanche of positive studies. I don’t think my younger colleagues know what a negative trial looks like anymore. Even our negative trials are pretty impressive. We’ve had studies where an immunotherapy agent was compared with chemotherapy. And it was designed to show that the drug would be better.

And it was just as good, and that was a negative study. That’s the correct interpretation, but still I would point out that that’s quite remarkable because these other drugs had taken us 25-30 years to develop. And now we have another drug with a very different mechanism of action that’s as good potentially. That’s impressive. I think we’ve just had an amazing degree of progress in the last few years. We have far more drugs. We understand far more about the disease – the technology at every point from diagnosis to assessment of response to the ability to evaluate better what we’re not doing well. So, our studies now frequently have biopsies before, during, and after treatment in a way of trying to figure out why is stuff working or not working.

Back in 2006 or so, I proposed a study. We ended up doing it, but it took two or three years because we were requiring a biopsy result – actually, not even a new biopsy but just an archived specimen from the original biopsy to determine eligibility, and there was strong pushback that we would never be able to do that. And now, we routinely are getting biopsies and re-biopsying, and that’s over a brief period of time.

So, we’re getting to get better understanding of the disease, and why stuff works and doesn’t work. And I think that that’s why our progress will accelerate. And I would again emphasize progress only happens – real progress – only through clinical trials. We’ve cured a lot of mice for many decades. A mouse is not a person. You actually have to do the studies patient by patient, and I think we are making substantial progress. We almost have too many things to test right now.

Patricia:                      

That’s a good problem to have. Dr. Edelman, thanks so much for taking the time today.

Dr. Edelman:              

You’re welcome. My pleasure.

Patricia:

And thank you to all of our partners. To learn more about lung cancer and to access tools to help you become a proactive patient, visit powerfulpatients.org. That’s powerful patients with an S .org. I’m Patricia Murphy, your host. Thanks. 

The Empowered Lung Cancer Thriver and Expert Chat

The Empowered Lung Cancer Thriver and Expert Chat from Patient Empowerment Network on Vimeo.


Transcript:

Laura Levaas:

Hello, and welcome to this Patient Empowerment Network program, the empowered cancer survivor and expert chats. I’m your host, Laura Levaas, the lung cancer community manager for Patient Power, and a two-year survivor and thriver of lung cancer. This program is produced by Patient Power. We thank Celgene Corporation, Novartis, and Pfizer for their financial contributions to this program. They don’t have editorial control, but we do really appreciate them helping us make this program happen.

So, our guest today is Dr. Ross Camidge, the Director of Thoracic Oncology at the University of Colorado here in Denver. He’s also one of the top doctors in the U.S. for the very type of lung cancer that I have. It’s a rare mutation called ALK positive. And hopefully he can talk about that a little bit more later.

Dr. Ross Camidge:

We can talk about that until the cows come home.

Laura Levaas:

That’s good. Well, I’m excited to be interviewing somebody who is in the same town as me. So, you’re right down the road.

Dr. Ross Camidge:

Yeah, and we’re doing it virtually. Isn’t that crazy?

Laura Levaas:

It is crazy. So, we’re both in Denver, but we’re both online. So, I hope you’re having a good day. And thank you for joining us. So, can you estimate how many lung cancer patients you’ve worked with during your career?

Dr. Ross Camidge:

More than 1,000, I would have thought. So, I tend to see about 30 people a week, of whom about two or three of them are new each week. And then you can do the math. And then I’ve been here…it’ll be 15 years in October. So, someone really clever with a calculator can do that calculation, but it’s several thousand.

Laura Levaas:

That’s a lot.

Dr. Ross Camidge:

Yep.

Laura Levaas:

Is there a case that stands out to you in your career? Maybe somebody who beat the odds of their prognosis, or somebody that had a very interesting or unusual case?

Dr. Ross Camidge:

Well, you know, it’s funny. I mean, there are lots of people who I’ve looked after who’ve inspired me in different ways. But the ones that I keep thinking about the young patients who were diagnosed before we knew about all these molecular sub-types of lung cancer.

And I remember one young guy. He was 21 years old. He was really into skateboarding and art. And his parents were busy getting a divorce at the time. And it was a total disaster to have a diagnosis of lung cancer, and he’s stuck in the middle. And his disease was incredibly aggressive, and he didn’t survive very long. And somewhere in me, it’s like, well, he must have had something. He must have had ALK; he must have had ROS1.

And these things weren’t even described at the time. And part of life is about timing. So, nobody wants to have lung cancer. But it’s a much better time to have lung cancer now than it was last year, and certainly last decade.

Laura Levaas:

Right. So, there is hope for people who are diagnosed now?

Dr. Ross Camidge:

Well, I mean, I think that the best example of that is, people who now have Stage 4 lung cancer, the questions they have to ask are, “Shall I go for promotion in my job? Shall I go on this fun vacation? Am I gonna marry this person?” The same things that we all struggle with before a diagnosis of lung cancer. Because there used to come a time when you got a diagnosis of lung cancer, and the same conversation at least that the doctor was concerned was, “You’re about to drop down dead.” We phrased it differently, but you get the drift.

And now, those are completely separated by an unspecified amount of time, in the same way that we’re born and we die at some point in the future, and we don’t quite know when that’s gonna be. And so, we don’t have the two things – “Hi! Mrs. Jones! You’ve got a bouncing boy and they’re about to drop down dead.” Now, they’re separated by life. And we are gradually increasing the distance between those two events.

Laura Levaas:

I think that’s amazing. And this is a good segue, actually, for me to tell a little bit about my story. I don’t wanna get too far into the weeds. But my story, I think it was unique because I had a threemonth prognosis, basically, by the time they got a hold of me. I’d been misdiagnosed for about a year, which is pretty common, I think, with –

Dr. Ross Camidge:

Yeah.

Laura Levaas:

– lung cancer. You know, allergy symptoms, some migraine symptoms. And mine was actually caught, oddly enough, during a breast cancer screening. Because my mother is a breast cancer survivor, and she was diagnosed very young. So, my doctors have always been really proactive about that. But my original prognosis was three months. And that’s before they knew that I was ALK positive. So –

Dr. Ross Camidge:

So, who told you that you had three months?

Laura Levaas:

It was –

Dr. Ross Camidge:

That’s what drives me crazy, some well-meaning person in the emergency room.

Laura Levaas:

Yes. And I think it’s because when they discovered what I had, I had 50 brain mets and 50 spine mets, and my brain was swelling. And they were telling my family, “We’ve gotta get her into whole-brain radiation right away.”

We found out about two weeks later that I was ALK positive. So, they stopped the radiation, and I went right into taking Alectinib, which is a newer drug. And it was approved by the FDA I think about three months after I started taking it as first line for ALK.

Dr. Ross Camidge:

It’s all about timing.

Laura Levaas:

And then it stopped – yeah. Yeah. So, it’s kind of – I feel a bit like a champion. Because they said, “Well, you have three months.” And that can be a real bummer. And it’s a real shock to friends and family and my boyfriend at the time, who’s no longer. But here I am, 26 months later. And I feel great. And nobody ever thinks that I’m sick. They’re always shocked to find out that I have lung cancer. So –

Dr. Ross Camidge:

I think you’ve done great. And you’re still doing great.

Laura Levaas:

Thank you. And let me explain to our audience how I met you. One of the things that helped me have a positive outlook on being diagnosed with lung cancer is, No. 1, because I have this mutation, there was a targeted therapy available to me. And so, within six months, all of the cancer ground to a halt.

And I was basically able to resume most of my normal activities. I could drive again. I could go out to eat. I could do some normal things. But a friend of mine told me that there was a Facebook group for my specific type of cancer. And it was so valuable, and it helped me sort of like find my people. I refer to them affectionately as mutants because we’re all mutants together. But we share information. And they told me about your second opinion program, which I hope is okay to talk about on –

Dr. Ross Camidge:

Sure.

Laura Levaas:

– this program. But that’s how I found out about you. And you’re now my oncologist. And I’m in a Phase 2 clinical trial for a drug that’s new to me. And I’m very excited about that.

Dr. Ross Camidge:

You haven’t started it yet, have you?

Laura Levaas:

I have. I started it last week.

Dr. Ross Camidge:

Oh, you started last week, didn’t you?

Laura Levaas:

I did. I did. The first couple days, I felt weird. But now, I feel great. So, for those –

Dr. Ross Camidge:

Yeah, that’s fantastic.

Laura Levaas:

– that are watching, just know I do think having a positive attitude will help you through those really tough times when you’re feeling low. Reach out to your sub-group. Reach out to the people who have what you have. Because they’ve been walking that path, and they can help you.

Dr. Ross Camidge:

I mean, I think that one of the things is – I mean, it’s the same like when doctors talk to doctors. You can do the shorthand. You don’t have to explain what you’ve got and what it means. You don’t have to explain to me that you weren’t a smoker. You can just sort of jump in and say, look, this is the stuff that’s happening with me. And they understand.

Laura Levaas:

Absolutely. Absolutely. So, I am going to ask you a couple of quick questions. And then we got a lot of audience questions for you. So, I hope you’re ready.

Dr. Ross Camidge:

Yep. Bring it on.

Laura Levaas:

Lots of really good questions. So, before we transition into those, I wanted to ask whether you have noticed a mindset shift? You mentioned right at the beginning that this is the best time to be diagnosed with lung cancer because there are options. But are you noticing a mind shift in your patients?

Dr. Ross Camidge:

Yeah, I mean, I think there is. I mean, I think lung cancer has gone from being – or let me rephrase that. Certain sub-groups of lung cancer has gone from being this kind of embarrassing thing, that you were sort of hidden in a closet, and nobody knew a lung cancer survivor because they didn’t exist – to now, I can show a room full of people and you can’t pick out who’s the lung cancer patient and who’s their significant other in the picture because everybody looks the same. And that, to me, is huge success.

So, I mean, one of the things we did last year – and I may have shown you the picture that we have up in the clinic – is we actually had a survivors’ celebration.

Laura Levaas:

Awesome.

Dr. Ross Camidge:

And to get your invite, you had to be at least five years out from your diagnosis. And we invited 400 people. Now, to be honest, we messed up the timing, and we sent the invites out about two weeks late. But we still had about 100 people turn up –

Laura Levaas:

That’s great.

Dr. Ross Camidge:

– which was pretty awesome. And we took a big picture. And it’s framed and sitting up in the clinic, for the simple reason that when you’re first diagnosed, you know these people exist, but you don’t believe they’re real. And I wanted to be able to come outside and say, “See that guy there? Well, he’s 10 years out. And look, he still looks fine, and he’s leading a normal life.”

So, I don’t mean everybody’s gonna do that. But it’s gone from being this fantasy – I might win the lottery – to, well, I might graduate from high school. I mean, it becomes a much more realizable dream.

Laura Levaas:

Right. Well, what questions do you think patients should be asking when they’re first diagnosed? They go to the doctor. They’re like, “You have lung cancer.” What should a patient ask?

Dr. Ross Camidge:

Well, some of the basics are, what’s the stage of the cancer? How far has it spread around the body? So, usually, at least in the USA, people are getting a PET scan and an MRI of their brain.That’s the kind of standard bread and butter. I mean, 10 year ago, probably the most common thing I would encounter in the second opinion is somebody who wouldn’t have scanned the brain. They were waiting until someone had symptoms before they scanned it, which was like, well, you’ve lost a few neurons by then.

Now, probably the big thing is, have they done molecular testing? And I think the education has been, that’s not a uniform box. If you find something, that’s great. But if somebody says, “Well, you don’t have a mutation,” the next question is, “Well, what have you looked for?” Because if you haven’t looked for A, B, and C, you don’t know that that’s not there. So, the things that we test for have become more expansive.

And then the last one – and it’s hard not to say this without sounding like a complete jerk, but I’m going to do it anyway – is that the disease has become super complex and super specialized. And you don’t have to have all of your treatment with a thoracic specialist, but you should have a relatively early appointment with a thoracic specialist to just check that you’re on the right path.

Laura Levaas:

Good. That’s –

Dr. Ross Camidge:

Those are the three things.

Laura Levaas:

Okay. Those are really, really good things to ask. I wanted to ask also how long you’ve been involved in lung cancer clinical trials in the development of new medicines?

Dr. Ross Camidge:

Well, I’ve been here, as I said, nearly 15 years. I trained before that amongst other places in Edinburg, in Scotland, which is where I did most of my training. And that’s where I first encountered lung cancer patients. And it was actually probably the very first – so, you were taken round to different centers in your training. And I landed in lung cancer. And I really liked the patients. And I kind of felt that they were … they were very undemanding. Often, many of them had smoked, and they were kind of feeling a little embarrassed. And so, they made you want to step towards them because they were kind of stepping away from you. And I also felt that it was kind of poised for a breakthrough. So, that was kind of how I got involved.

And then since I’ve been here, when I first arrived in Colorado, it was pretty well known for lung cancer. But it had not a huge clinical program. I think when I arrived, they put nine patients a year on clinical trials. And within a few years, we were putting more than 100 on. So, I really helped to build that. And then with my colleagues here, we’ve been able to build the program.

Laura Levaas:

What’s the best advice you can give someone who is newly diagnosed with cancer?

Dr. Ross Camidge:

Well, the first thing is, for those of you who’ve seen The Hitchhiker’s Guide to the Galaxy, the first thing is, don’t panic.

Laura Levaas:

That’s good advice. That’s good advice.

Dr. Ross Camidge:

The thing is, what you do is, you get diagnosed. And there’s a period of time where the room – you just can’t hear anything, and you feel distant from it. And what you’ve gotta do is, you – absolutely, you can wallow in self-pity for a period of time. And then you have to get up and move on. And that’s when you say, okay, this is a problem like anything else in life. And I will figure out the best of all possible solutions.

Laura Levaas:

Absolutely. Conversely, Terry wanted to know, what is the biggest mistake patients make in decisionmaking about treatment?

Dr. Ross Camidge:

Well, listening to people who say you only have three months to live.

Laura Levaas:

Yeah. That’s not good.

Dr. Ross Camidge:

Yeah. I don’t know what – I think perhaps believing that everything you see about cancer on the TV – which is everyone who’s bald and throwing up – must automatically apply to you. Or that that person down the street who died from a brain tumor automatically applies to you. I mean, so, cancer isn’t cancer. There are different diseases. And until you can find out, like you said, your peer group, you don’t know what the truth will be for you. And then you’re still gonna make your own rules up anyway.

Laura Levaas:

That’s true. That’s true. And I was thinking the other day, my needs when I was first diagnosed are very different than what they are now a few years later. Because in the beginning, I didn’t have coping skills. And I just didn’t know what to do. But you do develop them over time. And I remember a woman telling me, “Oh, you’ll figure it out.” And that made me really mad. But I see the wisdom –

Dr. Ross Camidge:

Yeah.

Laura Levaas:

Yeah. I see the wisdom in that now because you do figure it out over time.

Dr. Ross Camidge:

But how did you figure it out? How did you develop those coping skills? … Am I allowed to ask you questions?

Laura Levaas:

Oh, absolutely! Yeah, I think it was helpful, oddly enough, that I wasn’t allowed to drive and that I was in such a bad state. Because it allowed me to sort of withdraw from society for a while, withdraw from my work, withdraw from relationship drama. Because I ultimately ended up breaking up with my partner because he wasn’t capable of handling what I was going through, and he wasn’t supportive. So, all of the things that were familiar to me, like my job, my apartment, I retreated from all of that. And at the time, it sucked. But now, I’m like, that allowed me to have a perspective that was removed from everything. And I just –

Dr. Ross Camidge:

How old was your son at the time when you were diagnosed?

Laura Levaas:

Four.

Dr. Ross Camidge:

So, I mean, there’s an element of where you can withdraw from society, but you’ve got a 4-year-old.

Laura Levaas:

That’s right.

Dr. Ross Camidge:

So, how do you deal with that?

Laura Levaas:

Yeah. Well, I ended up moving in with my sister. Because at that time, I couldn’t drive, and I couldn’t take care of myself. So, I did rely really heavily on her. And their daughter is the same age as my son. So, they were going to school together. I relied very heavily on them, and I’m so thankful for that because that allowed me to just rest and heal. Because in the beginning – not to get too far in the weeds – but I couldn’t watch TV. I couldn’t be on my phone. I couldn’t be on the computer. Just no attention span whatsoever because of whole brain, I think. So, retreating from everything actually was good for me. And I’m also kind of a loner. So, I liked it, being alone too, oddly enough.

Good question.

I have another question from Christine C. She says, how long do you think it will take until lung cancer will be a chronically managed disease?

Dr. Ross Camidge:

Well, I think for some people, it already is. So, I now have 10-year Stage 4 survivors who are still alive and still thriving, to use your word. So, for those people, it’s a reality. And I don’t know – as I said, people will make their own rules – I don’t know how long they will go. I mean, I honestly do not know how long I can control their disease. You just have to stay alive and in the game and hope that breakthroughs will happen.

Now, then the challenge is, okay, “Well, what about me? I don’t have ALK. I don’t have – whatever.” And you go, okay, well, so, everyone – we have to try and replicate the success of the ALK positive population with all of the other sub-types of lung cancer or the ones that don’t even have a label yet. And so, there’s plenty of work to do.

Laura Levaas:

Definitely. Leslie wants to know, what do you see in the near future for treatment of lung cancer? And she lists a couple of things like a fourth generation TKI, immunotherapy – a couple of things that I don’t even know what they are, SHP2, Protex, anything else?

Dr. Ross Camidge:

Yeah. I don’t know what Protex is, but I know what SHP2 is. So, first of all, so, the concept of the fourth generation TKI, I mean, I assume that’s because we have a third generation TKI and therefore, the next one must be called the fourth generation. So, I don’t know that the generations of TKI is going to be the immediate solution.

If I had to say what I think the future is gonna hold, there’s a couple of things. So, one is I think we can – and we’ll use ALK as an example. But really, ALK is this model system that everybody else with lung cancer might like to replicate. So, we’re really good at developing drugs that are great at suppressing one particular pathway that is driving some people’s cancer.

But the cancer still grows eventually. Usually now, with some of the drugs – like the one you’re on and the third-generation drug – is that they’re not growing because they’re turning back on the same pathway. What they’re doing is, they’re growing through some other pathway coming up. So, finding these other pathways, these so-called second drivers, is going to lead to rational combinations of drugs. That’s one way.

The other thing which is kind of the elephant in the room is, well we have these drugs. You have these fantastic responses on the scans. But if you stop the drug, the cancer starts to grow. And if you go back on the drug a week later, it’ll shrink down. So, you clearly haven’t killed all of the cells which are even sensitive to that drug. So, until we can address why we can’t get 100 percent cell kill – that’s a technical term – we’re never gonna deal with the elephant in the room, which is, why can’t we actually cure people?

And that’s a very different situation from, why does the cancer grow three years later? The question is, why, when you walk through the door and you have a great response on the scan, if you had a magic microscope, why is there still one in 1,000 cells left? And that to me is actually the horizon we need to look for.

Laura Levaas:

Okay. Okay. That’s a great answer. A few more questions. Will R. wants to know about a lung cancer vaccine.

Dr. Ross Camidge:

Well, so, you could view that in a couple ways. So, if you think about how we use vaccines, we use them when we don’t have a disease to prevent us from getting that disease. We don’t really use a vaccine when we’ve already got the disease. So, if you’ve got chicken pox, I don’t vaccinate you for chicken pox. I treat the chicken pox. And so, lots of people are trying to develop vaccines, but they’re giving them in the wrong way. They’re giving them to somebody with an established lung cancer, and then they’re surprised that it doesn’t work. But that’s not what vaccines do.

The question is, could we find a way of saying, well, these are the people who are at highest risk for lung cancer, and give them something before they have lung cancer to reduce their risk? And the answer is, maybe. But if you can imagine, that’s a really difficult study to do. It would take years and years and years.

I’ve just come back from something called the World Conference on Lung Cancer, which was in Barcelona – tough life – but the biggest breakthrough there wasn’t about treatment. It was about a study that was actually done in Scotland about screening people. So, we’re pretty familiar with, if you smoke this much, you meet a certain criteria, and you go get a CT scan. But that’s no good if you’re not a smoker. You don’t meet those criteria.

So, they still have to look at a blood test. And they can show that that particular blood test, it wasn’t definitive. It wasn’t, you’re gonna get cancer or not. But it bumped up your risk if you are positive on the blood test to then make that screening even more effective.

Laura Levaas:

That’s awesome.

Dr. Ross Camidge:

And they had some evidence – loose evidence – that it might even work in never smokers. And I think that’s what will come in the future too. And then what if you identify this high-risk group? I’m getting all excited now – all that higher-risk group? Maybe then say, okay, well, why are they at higher risk? Is that the group we give a vaccine to?

Laura Levaas:

Right. And then how would you identify a non-smoker, high-risk group? Can you?

Dr. Ross Camidge:

Yeah, well, so, it’s a work in progress. So, one of the things that they’re starting to do is find some of the mutations which are driving people’s cancer in the blood. Okay? So, the problem is that the sensitivity of the test isn’t very good. So, you can find it when somebody has lots of cancer in their body. But to get the screening, you want to find it when there’s one little ditzel in your lung. So, you have to really turn up the sensitivity.

And I think that’s where the field is kinda going. So, they would know that if they found ALK in your blood, if they made a super sensitive test, that that would be wrong. Shouldn’t be there. And therefore, they would say, you should go get a CT scan. And so, the sensible thing would be, develop a cocktail of tests for every one of the things that drive lung cancer and say, if we find it, that’s bad news. Go get a CT scan.

Laura Levaas:

I like that. A cocktail of tests. Good. Well, hopefully, that will be soon. Two more questions. This is a really great question, actually, from Gail O. Is there a resource for local oncologists to reach out to for information and collaboration about lung cancer? Because as I’m sure you know, some of these smaller centers, maybe those physicians aren’t seeing lung cancer patients. So, they – I don’t wanna say they don’t know what to do, but maybe a patient is not getting the appropriate treatment protocol.

Dr. Ross Camidge:

I mean, that’s a really good question. So, it depends on where you are in the world. So, there are guidelines that NCCN, National Comprehensive Cancer Network – which is a common guideline used in the USA – is updated every few months. And that’s a common thing that a private practitioner could look at. And yet, it’s astonishing how many people sort of still don’t follow that. That’s a guideline. And the trouble with guidelines is, they don’t describe every possible scenario. In terms of how do you –? This may come as a huge surprise to you, but doctors have egos.

Laura Levaas:

No!

Dr. Ross Camidge:

No! So, how do you convince a person who may be a very good general oncologist that they don’t know everything? And that’s really hard. So, it’s not that we don’t necessarily have the resource. But we have to have people feel comfortable, if you like, asking for help. And I think that may be the biggest challenge.

I mean, I’ll give you an example. So, here we are in Colorado. There are probably several hundred medical oncologists in the state, of whom a handful ever send us patients for clinical trials. And you go, well, they must all see lung cancer. Lung cancer’s common. So, why do only some of them send people for clinical trials? Either they’re sending them somewhere else – and that’s okay – or they’re just not asking for help. And that is a huge tragedy if that’s happening.

Laura Levaas:

Yeah. So, is there a resource for local oncologists, like –?

Dr. Ross Camidge:

Do you want me to actually answer the question?

Laura Levaas:

If it’s possible. It’s a big question.

Dr. Ross Camidge:

No. I mean, not in a – I mean, there are lots of separate resources. So, all oncologists are subject to CME, continuing medical education. There are videos they can watch. There are updates of all these conferences. But they have to want to do it. Nobody is getting down and forcing them to do it.

Laura Levaas:

Right. And I think that’s where an empowered patient comes in. An empowered patient will seek out the care that they’re looking for.

Dr. Ross Camidge:

Yeah. I mean, I do lots of second opinions. And for many of my patients, they’re around the world and around the country. And sometimes, their oncologist I form a very close relationship with because we both feel like we’re looking after the same person. And you almost feel like you’re kind of a co-parent. And that’s great because they don’t feel threatened by me, and I don’t feel threatened by them, and we can work together. “Well, this has happened. This is what the scan shows. What do you think? And I’ll do this.” And others don’t. But that’s how it can work well.

Laura Levaas:

Okay. Last question. This person’s name is Parentin B. I’ve never heard that name before. It’s very interesting. Are there recommendations about what patients can do themselves, like supplements, diet, exercise, etc., that could be helpful? And I know when I was first diagnosed, that was one of my first questions. Because my physician said, “Well, eat healthy.” And I was like, “Well, what does that mean?”

Dr. Ross Camidge:

What does that mean? Yeah.

Laura Levaas:

So, I think there’s a glut of, should we do Keto? Should we do Paleo? Should we go vegan? Vegetarian?

Dr. Ross Camidge:

I think one of the things is, what this is actually telling us is that when we’re diagnosed, we want to be part of the solution ourselves. We don’t want to be passive and have people do things to us. And I think the physicians who go, “Well, no. Nah,” I mean, they’re missing out on that need to take some aspect of control of our lives.

And so, some of it, you can channel that energy into becoming empowered and educating yourself about it. Not to the point that you’re obsessed about it, but I mean so that you’re, again – occasionally, I get patients who come in, and you go, “So, what treatment are you on?” And they go, “I don’t know.” And you go, “Well, you’re hardly taking control if you wanna change your diet, yet you can’t be bothered to learn the name of your chemotherapy. That’s not empowerment.”

I think diet is something we can all control in our lives. It can also make you – a diagnosis of cancer makes you vulnerable to anyone who wants to sell you any kind of quack theory. I think most people, at least our cancer dietitians here, would say, you bump up the fresh fruit and vegetables. You don’t have to become a juicer. But fresh fruit and vegetables generally make you feel better. They keep your bowels moving more, which sometimes, some of the treatments can interfere with that. You don’t have to feel guilty if you have a candy bar. But if you minimize the amount of highly processed food you have and the amount of sweets, that’s fine. It’s like anything else. You can have cheat dates. Don’t feel bad about it.

But all of that is kind of subjective. There’s people who are gonna tell you, you have to have cottage cheese and flax seed oil or the Gerson diet and have coffee enemas. I prefer my coffee this way, but –

Laura Levaas:

Me too.

Dr. Ross Camidge:

And there are always testimonials about these things, but there’s very little hard evidence that it actually makes a difference. The one exception is exercise. Actually, there’s quite a lot of data that being a healthy weight – so, not overweight, and just being active. It doesn’t mean you have to sign up for a triathlon, but just going for a walk every day or doing something actually makes people feel better, makes them cope with the treatment better. And there’s even some data that actually survival is improved. So, that’s definitely something that people can do.

Laura Levaas:

Well, those are all really good things. And I appreciate these questions. Many of them came from the ALK positive Facebook group that really helped me cope through some of my tough times. And there are some really smart folks in there, way smarter than me. Probably not as smart as you. But they –

Dr. Ross Camidge:

No! Way smarter than me! They’re all like nuclear physicists and things.

Laura Levaas:

I’m really amazed at the amount of specialized information that I’ve been able to find in these support groups. So, kind of winding up. Thank you, Dr. Camidge, for joining us today for – it’s a new program, actually, from the Patient Empowerment Network, but it’s produced by Patient Power. And again, we want to thank Celgene Corporation, Novartis, and Pfizer for their support, even though they don’t have editorial control. We’re kinda driving the bus. And we’re really grateful that you could join us today and answer all of these pressing questions.

Dr. Ross Camidge:

My pleasure.

Laura Levaas:

Thanks. We’ll catch you next time. And everybody, thanks for watching. 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.

Lung Cancer New Therapies

This podcast was originally published by City Of Hope Radio here.

 

Ravi Salgia, MD – Speaker Bio
  • Topic Info:


    There is extensive collaboration between lung cancer clinicians and researchers to develop and evaluate new therapies designed to improve survival and quality of life outcomes

    Listen in as Ravi Salgia, M.D., Ph.D. explains all of the new therapies available if you or a loved one has been diagnosed with lung cancer.

Radiotherapy for Lung Cancer

This podcast was originally published by City of Radio Hope here.

Helen Chen, MD – Speaker Bio
  • Topic Info: Stereotactic radiotherapy is used to treat tumors in the lung without having to make an opening in the skin. The treatment machine directs beams of high-dose radiation directly to the area in the lung that needs to be treated. The radiation beams are silent and invisible.

    Radiotherapy can play a pivotal role in the treatment of lung cancer in all stages and can result in long-term curative outcomes for patients.

    Listen in as Helen Chen, MD explains how Stereotactic radiotherapy is used to help treat lung cancer.

Lung Cancer Latest in Research and Therapy

This podcast was originally published by Cornell Weill Cancer Cast on April 4, 2019, here.

Brendon Stiles, MD – Speaker Bio
  • Hot topics in lung cancer, including screening, immunotherapy, vaping, and more.Guest: Brendon Stiles, MD, thoracic surgeon at Weill Cornell Medicine and NewYork-Presbyterian Hospital.

    Host: John Leonard, MD, world-renowned hematologist and medical oncologist at Weill Cornell Medicine and NewYork-Presbyterian Hospital.

Clinical Trial Toolkit

A Conversation With Becky Pleat

Specialty Pharmacy and the Patient Journey with Specialty Medication

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

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

Medication Maintenance Tips for Caregivers

Managing medications can be difficult to do, especially if you’re a senior caregiver. Helping someone else remember to take medications on time and work to find the right balance for them can seem like a daunting task. Thankfully, we’ve got a list of tips and tricks to help make things flow more smoothly.

Make Sure Providers Are Aware Of Vitamins And Supplements

Medical providers should be aware of any vitamins and supplements a person is taking. Regardless of how natural they are, they can interfere with medications and other treatments. For example, someone on blood thinners should not be taking a supplement with vitamin K. Most blood thinners work by inhibiting the production of this vitamin in the body. Taking a vitamin K supplement can negate the work of blood thinners.

Instructions

Make sure to go over medication instructions with the senior you’re caring for. If they are able to, they should know the names of each medication along with dosages and what times to take them. It doesn’t hurt to type up instructions about medications so that all information is in one place and easy to access. Consider adding in what side effects they should seek help for. That can serve as a list for caregivers and seniors to check on in case of adverse events.

Alarms

Set alarms to remind seniors to take their medications. There are many options to choose from. Smartphones allow you to set up reminders with different sounds each time which can help people differentiate between medication doses and other alerts. Electronic personal assistants like Alexa or Google Home can easily be used for reminders as well. If the senior you’re caring for struggles with newer technology, consider a few alarm clocks around the home.

Keep A List

Keeping a list of medications can help seniors and caregivers alike remember what medications are due at what time. Lists that have both a visual of what the medications look like and allow people to check off a medication dose can be useful tools. If you’re going with this kind of list, make sure that you have multiple copies. Placing one next to a pill organizer and another on the fridge can help remind people to take medication before they’ve even missed a dose.

Smartphone apps can also be helpful in tracking this information.

Follow Up

It’s important not to just set alarms or reminders, but check in to ensure that someone has taken their medication. It can be easy to turn off an alarm and still forget to take medication as scheduled. Following up with the senior in your life can remind them that they didn’t take their most recent dose.

Store Medications Properly

Most medications do best when stored between 68 and 77 degrees Fahrenheit. Additionally, many of them need to avoid humidity, direct sunlight and more. Medications should not be stored in vehicles, on windowsills or other sunny and warm spots or even in the bathroom. Consider storing them in a cool, dry space in the kitchen or living space.

When medications aren’t stored properly, it can affect their potency and make them potentially dangerous. If you’re concerned that your senior’s medications have been affected, here’s what you need to watch out for:

  • Odd smells
  • Discolored pills, tablets and injections
  • Cracked or crumbled pills
  • Pills and tablets that are stuck together
  • Creams and ointments that show separation
  • Cloudy injections

If you see these signs, contact your senior’s pharmacist as soon as possible.

Sort Medications Into Pill Organizers

Set aside time each week to go through the medication your senior takes and place them into pill organizers. These can make it easier to remember to take medications as prescribed or even transport them while traveling. Some organizers can remind people to take their medications and even alert others that a dose has been missed.

Make Sure All Caregivers Know About Medications

A sure way to have seniors miss their medication doses is to have senior caregivers who aren’t on the same page. Without everyone being in the know, it becomes increasingly difficult to set reminders and follow up with seniors about medication doses.

Plan Ahead For Refill Needs

Refills may come up on days where a senior is alone. When that’s the case, they may forget or be unable to pick up their refilled medications. Refills may even be due when someone is planning to be out of town. Make sure to plan ahead adequately for refills and work with a person’s pharmacist.

Consider Compounding Medications If Needed

Compounding is a process where medication is tailored to a person’s specific needs. This can help remove any dyes a patient is allergic to or turn a pill into liquid for those who struggle with swallowing pills.

Get Tips from A Medical Provider

When methods to help your senior aren’t working as well as you had hoped, take some time to check in with their medical providers. Nurses have amassed a wealth of information on improving their patients’ quality of life. They are likely to have some ideas on how to make managing medications more effective.

Always Communicate With Family Members

Whatever steps you take to maintain a senior’s medication schedule, make sure that you’re communicating any difficulties with the senior’s loved ones. Family should also always be aware of any medication changes. When so many seniors rely on a variety of paid and family caregivers, it’s incredibly important for everyone to be in the loop on the storage, administration and organization of all medications, vitamins and supplements.

How Do We Increase Precision Medicine’s Reach in Lung Cancer?

Living Well With Lung Cancer

In this webinar in partnership with H. Lee Moffitt Cancer Center & Research Institute, Dr. Jhanelle Gray, a medical oncologist, Dr. Stephen Rosenberg, a radiation oncologist, and Dr. Theresa Boyle a molecular pathologist will discuss the latest understanding of lung cancer research; currently approved therapies and promising clinical trials.

Downloadable Guide


Transcript:

Andrew Schorr:   

And greetings from here, San Diego-Carlsbad, California. I’m Andrew Schorr from Patient Power. Welcome to this Patient Empowerment Network program. I am so excited. It is really a wonderful opportunity for anybody dealing with lung cancer. Whether you’re the patient or you’re a close friend or a family member, to get the latest information.

So, let’s meet that team. I wanna start with a medical oncologist and that is Dr. Jhanelle Gray. And she is director of thoracic clinical research in the Department of Thoracic Oncology at the Moffitt Cancer Center. Dr. Gray, thanks so much for being with us.

Dr. Gray:                

Thank you very much. It’s a pleasure to be here.

Andrew Schorr:   

Okay. We’re gonna understand the role of the medical oncologist. But it’s a bigger team than that. So, I wanna also have us meet Dr. Stephen Rosenberg who is a radiation oncologist and works in concert with medical oncology. He’s normally down in Tampa, but today – where are you in Wisconsin, Dr. Rosenberg?

Dr. Rosenberg:    

I’m currently in Madison, Wisconsin giving a talk later today and tomorrow. 

Andrew Schorr:   

Okay. Home of the University of Wisconsin. Thanks for joining us.

And then there’s another member of the medical team none of us usually get to meet. And that is the pathologist who’s looking at our biopsy. Whether it’s taken from the lung or now increasingly liquid biopsies, our blood, and we’re gonna talk about that. And that is a pathologist and that is Theresa Ann Boyle who joins us. Dr. Boyle, thank you for being with us.

Dr. Boyle:               

Thank you for inviting me. It’s nice to be dragged out from behind the scenes.

Andrew Schorr:   

All right. Well, you won’t be beside the scenes anymore.

And of course, all of this – we have this whole group, but it doesn’t mean anything unless there’s a patient that they can help and maybe a family with them. So, let’s meet a patient from Tampa who’s been living with lung cancer for a number of years, Ed Cutler. Ed, thank you so much for joining us on this program and we’re gonna share your story. Hi, Ed.

Edward Cutler:     

Hi. It’s a pleasure to be here and an honor to be honest with you.’

Andrew Schorr:   

Well, Ed, so, you’ve been living with lung cancer how many years now? 

Edward Cutler:     

Just over five years.

Andrew Schorr:   

Okay. But let’s face it, it wasn’t that long ago if somebody was told they had lung cancer they were not long for this world with more advanced lung cancer. So, modern medicine has made a big difference for you, hasn’t it?

Edward Cutler:     

It certainly has. When I received my diagnosis, I was given the quote average life expectancy statistics and they didn’t look very good.

Andrew Schorr:   

Right.

Edward Cutler:     

So, I went the whole way.

Andrew Schorr:   

And we should mention that for the last couple years you’ve been in a clinical trial and it’s an immunotherapy. And we’re gonna talk about immunotherapy along the way. We’re gonna talk about target therapies, immunotherapies. The doctors are gonna help us understand this whole idea of precision medicine. Which means, “How do you get what’s right for you?”

And you’ve had some changes along the way, right Ed? I mean there you are in Tampa continuing your work as a tax consultant, I know. And been married more than 50 years to Donna, which is great. Children and grandchildren. But you’ve had kind of a journey that’s had changes along the way, right?

Edward Cutler:     

It has been a journey. Yeah. Yeah. Initially I started out with standard of care chemotherapy. And that basically took over 16 months. Basically, the first two or three months I was on the full medication of three drugs. And then they dropped off one and then I was on maintenance. But at the end of the 16th month they discovered that there was a new tumor. And I was told I was now chemo-resistant and that was the end of chemotherapy for me.

So, my etiologist and I sat down, and we started searching. And at that point, I don’t think there were any other approved medications. Everything else I think was still in trials at the time. Now I know that there are maybe two, three, a half a dozen medications that are out of trials and FDA-approved. But at that time, I was limited to clinical trials. And Dr. Tan, who is my oncologist, gave me the options looking at two or three different trials. And my goal was to live as long as possible with good quality of life. And that’s what I was looking for in each of the trial descriptions.

And we ended up selecting one and I took all of the various testing to qualify for that trial and I was ultimately accepted. It was a two-drug combination of infusion. And unfortunately, I only lasted seven – roughly seven months in that trial because of side effects that almost killed me. 

Andrew Schorr:   

But now there’s another trial?

Edward Cutler:     

And fortunately, it took another few months, but we located another trial that was being performed only at Moffitt. I said, “Well, that’s convenient.” So, I said, “Yeah.” Everything looked good on that. Sure, there were potential side effects, but I was willing to take my chances with it. And here I am nearly three years into that trial and I’ve been stable most of that three-year period. There was a little bit of tumor size reduction initially and basically stable the rest of the time. It’ll be three years come the end of January.

Andrew Schorr:   

That’s such great news.

So, Dr. Gray, you have lots of trials at a major center like Moffitt, maybe you could just tell us in this world of lung cancer, patients who participate in trials have not only paved the way for everybody, but it’s given them great hope, hasn’t it?

Dr. Gray:                

Absolutely. So, we have a lot of trials at Moffitt. We try to organize ourselves within a way of doing a personalized medicine approach. And basically, that means that any patient that comes to Moffitt we wanna give them the best treatment possible. And many times, that is a clinical trial. With clinical trials a lot of times you have access to more novel agents and things that you can’t necessarily get through your regular route through FDA approval. And also, a lot of that work is spurred by research developed here at Moffitt and partnering with the basic science researchers as well as us at a lot of the clinical – on the clinical side and making sure that we move these drugs forward and into the clinic for patients.

And Edward, I just wanna say I’m very happy about your story. And thank you for taking the time today to be with us and to share your journey with us. And give us your perspectives to this too.

Andrew Schorr:   

Yes. It’s very inspiring to all of us. And Ed, we know you’re a golfer. And so, you’ve been… 

Edward Cutler:     

Well, I was.

Andrew Schorr:   

Well, please God you can do that and travel with Donna. And the main thing is you are with us.

Dr. Gray:                

Yes.

Andrew Schorr:   

And I know that means so much to you and your family.

We’re gonna talk about the team approach as we go forward. So, Dr. Rosenberg, radiation oncology comes into play here because often when somebody’s diagnosed radiation can help shrink the tumors, right? And also alleviate some of the pain and other issues that people may have, right?

Dr. Rosenberg:    

Yeah. No. I think you hit on a lot of big major points there. And it really is a team approach. Particularly at Moffitt when we approach lung cancer trying to think about how we can do best for the patients, whether it’s a clinical trial or not. Radiation plays a big role for patients who have a locally advanced disease. Some of the standard of care is combining chemotherapy with radiation for patients with some advanced disease. But for patients who have had cancer spread to other parts of the body as well, radiation’s really good to help alleviate pain and even approve breathing and the other things that are happening.

And with newer agents we often find that radiation may even be potentiating the immune effects of some of the new immunotherapy drugs that are out there. And so, we’re really excited about some of the trials and studies we’re doing with Dr. Gray and her team. And the team as a whole at Moffitt combining irradiation with some of their new – with targeted drugs and immunotherapy drugs right now.

Andrew Schorr:   

Wait. Let me see if I get that. And Dr. Gray, feel free to comment too. Are you saying that radiation can sort of boost the effect of some of these medicines?

Dr. Rosenberg:    

Yeah. There is a lot of anecdotal evidence out there. And some basic science that’s right now emerging about how the immune system and radiation really are so interconnected. And how that helps us actually attack cancer by actually basically releasing the immune system to recognize the cancer in the body. And so, by combining that with immunotherapy drugs we’ve really found our ability to potentiate some of the effects of these immunotherapies.

Most of this is basic science. There are some anecdotal case reports out there with some of the newer drugs that have just come out in the last year or two FDA-approved have been after chemoradiation and we think they really work together well. And some of the newer trials at Moffitt are gonna be trying to combine these things up front. And I know Dr. Gray has been helping to lead that effort with Dr. Perez and others within our kinda joint departments here.

Dr. Gray:                

Yes. Absolutely. And so that work that Dr. Rosenberg was just talking about was actually developed at Moffitt. So, there’s a trial out there that’s now published in the New England Journal of Medicine. It led to the FDA approval of a drug called Durvalumab, which is actually named because the company, AstraZeneca, wanted to add durable responses and add value to patients. So, Durvalumab is where the name came from, interestingly enough.

And the study, what we wrote, was to look at those patients getting chemotherapy plus radiation therapy completing what’s considered standard of care therapy for those patients with that particular type of non-small cell lung cancer. And following this with an immunotherapy agent, the durvalumab (Imfinzi), for one year. And it significantly improved the outcomes for patients. Patients are living much longer when we utilize this method.

And now this has become the standard here in the United States. It’s working its way through the approval mechanisms over in Europe and through other companies. And I think this has really revolutionized how we approach and treat patients. And we are looking at – now we know it’s safe to give them sequentially. And so, can we safely and effectively – meaning can we actually improve outcomes for patients by moving these therapies upfront?

And so, it would be giving a lot of therapies together. So, it would be chemotherapy plus immunotherapy plus radiation therapy to patients. But at the end of the day, the goal here is to improve our outcomes in a – and still maintain quality of life for patients. So, it’s always challenging pushing the bar and reaching these goals for our patients.

[Crosstalk]

Andrew Schorr:   

Right. Right.

So, Ed, just a little bit more about your story and then we’re gonna bring Dr. Boyle into this too as we talk about personalized medicine. So, when you were originally diagnosed, you were, I think, consulting with a doctor about a concern about an aortic aneurism. It had nothing to do with cancer.

Edward Cutler:     

Yeah.

Andrew Schorr:   

You went to one doctor and then maybe a GI specialist. That’s where they found a mass. And then you went to Moffitt and saw a lung cancer specialist. Did I get that right?

Edward Cutler:     

That’s right. Yeah. The triple-A test, the Abdominal Aortic Aneurism test, was negative. But down at the bottom of the report in the footnote was that they saw a mass in my liver and follow-up was recommended. So, we went from there. I went to my primary care and he referred me to a GI doc and they pretty much agreed that there was some kind of cancer, but they didn’t know what exactly. They recommended that I get a PET scan, which Medicare would not permit. They said you have to have a diagnosis before you can get a PET scan.

So, the alternative was to have the biopsy. I said, “Okay. But let me get a second opinion first.” And that’s when I came out to Moffitt and they confirmed everything. I had my biopsy here at Moffitt. And there was a tumor in my liver, but the biopsy traced it back to my lung. Therefore, I have lung cancer.

Andrew Schorr:   

Right. And I wanna explain that. Okay. So, let’s start with Dr. Boyle on that. So, Dr. Boyle, you look at the biology of tissue samples or sometimes blood.

Dr. Boyle:               

Correct.

Andrew Schorr:   

And so, somebody says, “Oh, I have liver cancer.” But that just came up where he said well, he didn’t really have liver cancer, he had cancer that originated in the lung and the biology of it was it needed lung cancer treatment, right? And that’s part of what you figure out, right?

Dr. Boyle:               

Right. Right. Right. And actually, within the pathologist group there’s different fields of pathology. There’s the anatomic pathology where they’re looking at the diagnosis, “Is it lung cancer origin or is it kidney cancer origin?” I’m in the field of molecular pathology. So, I’m looking at the genetic changes inside the tumor, or in the blood too, and I’m trying to understand what those changes might mean in terms of what would be the best therapy for the patient. I’m also in the lung cancer research field. So, trying to better understand all of these immune checkpoints and how can we look at them. Why do some patients respond, and others don’t respond?

So, pathology is a large field. So, I’m working very closely with the thoracic department. In fact, I belong to them. 50% of my job is with the lung cancer department and 50% with the pathology department. And we found that that was helpful because the field is expanding so dramatically. Even within every year there’s great advances. And for anyone to keep up with everything is too difficult these days. And so, we really do all work as a team here at Moffitt. And so, as Jhanelle and I talk back and forth – a lot of emails with.

Dr. Gray:                

Right. Yes.

Dr. Boyle:               

So, I even consulted them on some of these questions you have. So, it’s great.

[Crosstalk]

Andrew Schorr:   

All right. Well, we’re gonna delve into this personalized medicine world. And the doctors will help us understand. We’ll understand how it applies to patients like Ed or others who may be watching. So, let’s put up the personalized medicine slide. Kat is our director and she’ll put it up.

I have a little dog barking here. I’m sorry.

Dr. Gray:                

No, you’re fine.

Andrew Schorr:   

Very cute.

Dr. Boyle:               

I like dogs.

Andrew Schorr:   

Kat, if you could put up this slide? There we go. Okay. So, help us understand – let’s start with you, Dr. Boyle.

Dr. Boyle:               

Yeah.

Andrew Schorr:   

This whole wheel around the right, what is this alphabet soup there? What is it?

Dr. Boyle:               

Right. Right. Right. So, this is showing the variety of different genes that can have genetic changes in the tumor. And it’s focused on the genetic changes that have potential clinical action or proven clinical action. EGFR is probably a more familiar one because that one came out first with better responds to EGFR inhibitor therapy than chemotherapy and others have come along. Like with ALK, ALK inhibitor therapy works well. With MEK, XM14 has become important, MET amplification.

Andrew Schorr:   

Okay. These are genes that have gone awry that are driving someone’s cancer, right?

Dr. Boyle:               

Correct. Right. Right. Right. And this wheel is trying to pick up on the driver mutations. There’s even more genes not on this wheel here that are passengers. Other mutations that might have some specs, but they might not necessarily be causing the tumor or driving the tumor but might be worth considering in terms of the therapy. In immunotherapy, tumor mutations burden has been something we look at. And they’re looking at many, many gene changes to see if there are more mutations than usual. And when that occurs, there might be a better likelihood of response to immunotherapy. So, we’re learning more and more everyday about all of these genes and more.

Andrew Schorr:   

Okay. We’re gonna define this. And Dr. Gray, you can help us. These kinda big bubbles to the right.

Dr. Boyle:               

Yes.

Andrew Schorr:   

So, first of all, a myth: All lung cancer tumors are the same. This right here says, “No,” right?

Dr. Gray:                

No. Absolutely. Yes. The fact is that each patient’s tumor has a unique biology. And the wheel on the left I think really helps to define this. That at the end of the day when we get a patient we’re concerned about, we get a biopsy, get a piece of tissue, send it over to pathology to Dr. Boyle’s team. She’s not only looking under the microscope to help us with, “What’s the diagnosis? What’s the origin of the tumor?” But we also wanna look at, “What is driving your tumor?”

And so, how I’ve explained it to patients is in two ways. You have a computer that has all these different parts, but at the end of the day what drives the computer is really that hard drive. And if you open up the hard drive there’s this little piece of hardware that’s actually making everything run. And that’s what we’re doing with the tumors is going into the cell, looking at the DNA level and seeing what is turning on your specific tumor.

Another way of thinking about it is as a hub for an airline, for example. So, a lot of us know Delta has a very big hub in Atlanta. They have a lot of flights that go through there. But if you were to shutdown Atlanta you would significantly impact the feasibility of Delta being able to function.

And that’s what we’re doing by looking at these driver mutations. We wanna find what’s turning on your tumor and then match that patient to the correct medicine. So, if you have the EGFR mutation, I wanna give you an EGFR inhibitor. If you have an ALK rearrangement, I wanna give you an ALK inhibitor. If you have a MEK mutation, I wanna give you a drug that targets MEK. What I don’t wanna do is if you have an EGFR mutation give you an ALK inhibitor. I’m doing you a disservice.

And so, it is very important – I think you brought up a very good point at the beginning of this that the team approach for lung cancer is imperative so that we can all work together to get the right patient the right treatment at the right time. 

Andrew Schorr:   

We’re gonna look at a graphic for that in just a second. I wanna just go over a couple of other things you mentioned. So, somebody might have a lung biopsy. Get some tissue. That goes to Dr. Boyle and her colleagues.

Dr. Gray:                

Yes.

Andrew Schorr:   

Wherever in the world you get treatment. And they’re taking a look at it to see where in this wheel – what comes up for them?

Dr. Gray:                

Correct.

Andrew Schorr:   

And then also there’s a – in that purple bubble there it says, “Tumor testing can happen at any point.” And so, we talked about driver genes.

Dr. Gray:                

Yes.

Andrew Schorr:   

And then Dr. Boyle mentioned passenger genes.

Dr. Gray:                

Yes.

Andrew Schorr:   

Well, they can change over time, right? 

Dr. Gray:                

Correct.

Andrew Schorr:   

There’s an argument for having testing again at some later time, right?

Dr. Gray:                

Absolutely. And so, for example, if you have an EGFR mutation and I give you an EGFR inhibitor, you then have a chance that your tumor can mutate against that specific drug that I’m giving you. And you can acquire a different mutation. And so, how do I know what’s going on? I need to get more tissue or – I don’t know when we’re planning on talking about it, but this is a good segue into liquid biopsies.

So, a liquid biopsy is getting a blood sample from patients and looking – specifically looking again at this wheel, looking at those mutations to see if we can identify them.

Andrew Schorr:   

Okay. 

Dr. Gray:                

And so, it is very, very important to keep monitoring patients, getting their blood, getting their tissue over time so we can make educated decisions. Again, just to relate this to something I think we’re all very familiar with is infections. If you keep giving the infection the same antibiotic, what happens? It develops resistance. And these drugs are no different and cancer’s no different. It’s just we have to stay ahead of the game and try to keep trying to outsmart the tumor.

Andrew Schorr:   

Right. Right.

[Crosstalk]

Dr. Gray:                

So, absolutely monitoring.

Andrew Schorr:   

Many researchers and specialists talk about cancer being really a wilily enemy.

Dr. Gray:                

Yeah. Enemy. Yes.

Andrew Schorr:   

And so, you can knock it back. But there’s sometimes the survival of the fittest of some cells that have some other property, like another gene?

[Crosstalk

Dr. Gray:                

Yes.

Andrew Schorr:   

So, Dr. Boyle, just help us to understand this idea of liquid biopsy. Because I know over the last few years sometimes there’s been a concern – I don’t know who does it. Whether a surgeon does it. Who does it? To get a lone biopsy and as much tissue as they can. But you’re saying, “Well, I need more to make other decisions.” Where does liquid biopsy come in now, basically a blood test, to help inform targeted, well-informed lung cancer care?

Dr. Boyle:               

Right. Right. Yes. Pathologists always want more tissue, but now we have an alternative. And sometimes an alternative gets the results faster back to the oncologist and the patient. And that’s the blood testing. And it has less risk than taking a sample from the lung. Now, the interpretation of the results from the liquid or the blood specimen is a little different than the interpretation from a tissue specimen. And when you get a positive result from the small amounts of cell-free DNA circulating in the blood, you can really count on it. And the oncologist can treat the patient with a targeted therapy based on that.

There are times when the results are all negative and you don’t know if the results are negative because there just wasn’t enough cell-free DNA in the blood or because the tumor is truly negative for all the mutations being checked. And so that’s where it really is important to follow-up with tissue testing. So, it’s been really a great advance in the field to be able to test with a specimen that’s much more easily available and can be tested right away.

Andrew Schorr:   

I’ve had the opportunity to tour foundation medicine back in Boston area and also be in some labs at other hospitals. And I’m amazed that these super sophisticated analyzers now to try to see what’s going on, whether the blood or the tissue

Okay. So, we’re gonna come back to where does that go out? I wonder if Kat, our director, could put up the personalized medicine slide? One more time, Kat. No, not that one. We’re gonna come back to that. That one.

So, there’s that whole area, Dr. Gray, on the left where it says, “Other.” And I know that Dr. Boyle and her colleagues around the world are trying to say, “Well, are there driver genes that we just haven’t identified yet?” And you keep having these analyzers looking at more and more, a bigger – 100, 200, and 300 whatever.

Dr. Gray:                

Yes.

Andrew Schorr:   

But some people, and I think this was Ed’s case. There wasn’t a driver gene that was identified.

Dr. Gray:                

Right.

Andrew Schorr:   

Well, what do you do there? 

Dr. Gray:                

So, one of the things that we’re testing for in addition to these driver mutations is also looking if patients may be a better candidate for immunotherapy and looking at a marker called PD-L1. It’s programmed death-ligand 1 which can be found on the tumor tissue cells. So, most patients will undergo simultaneous testing for both the immunotherapy marker as well as one of these driver mutation markers. So, if we’re unable to find a driver mutation, but we’re able to find that the tumor’s positive for PD-L1, that then triggers to us, as a medical oncologist, that, “Hey. We need to kind of shift and let’s focus on getting the patient immunotherapy.”

And particular now with the approvals and the trials that we’ve participated in here at Moffitt, as well as at other centers, that really if you have lung cancer, you don’t have a driver mutation. Outside of having a specific rational why you shouldn’t get immunotherapy, really you should be starting on immunotherapy.

I just wanna make – within that setting I just wanna make something clear. There are also some patients where you can find a driver mutation and you can find that they’re positive for the marker for the immunotherapy. And how do you choose between the two? For right now, most of the data points toward that you should focus on treating the driver mutation. That that does take precedence over the PD-L1, the marker for immunotherapy, okay?

And I know there’s a lot of commercials out there and a lot of excitement about immunotherapies for very good reason, but I would reserve the immunotherapy in those subsets of patients who have both markers to maybe a later line of therapy. But that also gives you a backup plan, right? It’s to your point you’re always trying to project and sequence things for the patients as much as we can. So, it is helpful to do both markers upfront and then act accordingly.

Andrew Schorr:   

Okay. I wanna make a point. I think that we’re done with this slide now. I wanna make a point that some people – it’s the minority, but it’s still a significant group – don’t have non-small cell lung cancer. 

Dr. Gray:                

Correct. 

Andrew Schorr:   

They have small cell lung cancer. And this has been tougher. But my understanding, and Dr. Gray, maybe you can inform us, that in some of the latest meetings you’ve been learning that immunotherapy along with chemotherapy can make a difference for people with small cell lung cancer, is that correct? 

Dr. Gray:                

Yes. Yes. That is absolutely correct. So, at our recent world congress on lung cancer meeting – now, this is our global international meeting for lung cancer where all the lung cancer experts get together now on a yearly basis. A lot of that has to do with that so much is changing that we now need to meet yearly. We used to meet biyearly.

One of the key presentations there that was in the presidential symposium seat or that’s the big session there was looking at combining chemotherapy with immunotherapy versus giving chemotherapy alone. And when they looked at this in patients with newly diagnosed, what we consider extensive stage or most people refer – may refer to it as stage four small cell lung cancer, that those patients derived a bigger benefit if we did the combination therapy. So, we’re talking about going from two IV infusions now to three IV infusions. So, you do add an hour, but there’s significant benefits that we all feel is also clinically beneficial for these patients.

If you happen to have small cell lung cancer and you are on chemotherapy there is also data that following your chemotherapy that utilizing immunotherapy in the subsequent line can also be helpful. So, these data are showing us consistently that immunotherapy is certainly effective in small cell lung cancer. And thank you for raising that point and that distinction. 

Andrew Schorr:   

Okay. So, Dr. Rosenberg, I’m gonna bounce this off of you as a cancer specialist. Not particularly about radiation but let me see if I get it right because we’re talking about immunotherapy.

When I developed cancer – and I’ve actually developed two blood cancers. So, I’m living with it. But whatever the cancer is, our bodies let us down, right? Our immune system has let us down and we’re starting to create aberrant cells, right? And they can develop masses like Ed had and they can spread. There’s a certain biology that the other doctors were talking about that they try to target. But with immunotherapy, that’s trying to leverage our immune system to do what it didn’t do right the first time, is that right? Is that the way you see immunotherapy? 

Dr. Rosenberg:    

Yes. Very much so. And one of the things that can define cancer is its ability to evade the immune system. In terms of our normal body, the way our immune system is set up is set up to go around our body and take care of any precancerous cells and try to destroy them. And unfortunately, what cancer does it has molecular mechanisms that try and basically get around these things. And so, the immunotherapy drugs that are out there help release the break in terms of the immune system to go back and attack these cancer cells.

And in terms of the interaction with radiation, because I am a radiation oncologist and I tend to bring it back there, is that what radiation can help do in that setting is actually destroy some of the cancer cells and kind of release what we call antigens into the blood stream or the nearby tissues to hopefully help the immune system better recognize the cancer. And when you take the break off the immune system and then allow the immune system to hopefully better recognize the cancer cells, these things all work together moving forward.

And so, I think there’s – only as we move forward, we see more interactions with radiation and the immune system and even these targeted therapies. I know that we went over – Dr. Boyle and Dr. Gray talked about how – these new targeted therapies that are out there. But even from a radiation point of view, we’re now really pursuing radiogenomics where we’re actually using some of these genetic signatures actually to determine how cancers respond to radiation treatment. And we’re actually sculpting our radiation to actually target certain areas of tumors to higher doses or lower doses based on some of these molecular mechanisms.

And it’s a really exciting time. And Moffitt particularly is really pioneering both of these areas to kinda push the boundaries in science right now.

Andrew Schorr:   

Well, good for you.

And I think, Ed, in listening, with this new age of cancer care it really argues wherever possible for people to get a second opinion at a major center like Moffitt where this brain power and leading edge of research can be brought to bear, wouldn’t you agree?

Edward Cutler:     

Oh, absolutely. No question about it.

I do have a question for Dr. Boyle. And that’s going back to the wheel of the mutations. Go back to 2013 when I was diagnosed, were all of those mutations tested when I had my biopsy or has the state of the art evolved such that now they do test for all of them? Or are there still some that they don’t test for until a certain event occurs?

Dr. Boyle:               

Right. So, there is still some disparity about where you go for your care and how many genes are tested. Certainly, Foundation One has a very large panel of genes. And I believe that was available in 2013. At Moffitt in this past year we validated a 170 gene panel that tested both the DNA and the RNA at the same time so that we can detect fusions like ROS1 and ALK at the same time as we’re detecting EGFR, KRAS, BRAF. All things on the wheel.

And so, I mean that only became available this year. In the past it was more piecemeal. And so, there were certain things that could be tested early on and possibly more things tested later. More and more we’re doing more comprehensive testing early on at diagnosis so that we know more at the beginning.

Andrew Schorr:   

I think there’s a point to underscore here is the field is evolving. The other day, Dr. Gray, we did a program that included someone you know from Harvard, Dr. Siegrist. And her advice, and I think you’d echo it, is wherever our audience gets care you wanna get a broad panel testing. And as we’re hearing from Dr. Boyle, the panel’s increasing, but the downside – and I know patients like this, for instance, with one of those more rare driver genes, ROS1, where they were tested sequentially and given, as you said, the wrong therapy for a while until they finally got around to doing the right tests and knew what was going on and did what’s right for them.

Dr. Gray:                

Yeah.

Andrew Schorr:   

So, I think for our audience, wherever you get care, and certainly it happens at Moffitt, you want a broad panel and as Ed was getting at is that panel is expanding.

Dr. Gray:                

Yes.

Andrew Schorr:   

What’s going on for you, and I think what you all say, and personalized medicine is, the right treatment for the right patient at the right time.

Dr. Gray:                

At the right time. Yeah.

Andrew Schorr:   

Okay?

Dr. Gray:                

Exactly.

Andrew Schorr:   

Okay. Let’s go to the role. We have another slide that describes the role. Really the team medicine approach, if you will. And let’s take a look at that.

So, okay. So, Ed was referred to Moffitt. Came there for a second opinion. And so, let’s see how it goes. So, Ed was the first place you went was to an oncologist. Was that first stop for you?

Edward Cutler:     

That’s correct.

Andrew Schorr:   

Oh, okay.

Edward Cutler:     

I went to a GI oncologist.

Andrew Schorr:   

A GI oncologist? But then it was discovered that it was really coming from your chest. And there was testing done. So, Dr. Rosenberg, let’s let you lead it. So, here I see radiation oncologist, medical oncologist, way down at the bottom we have molecular pathologist, like we have Dr. Boyle. How does all this work together?

Dr. Rosenberg:    

I think you’re gonna hear this theme over and over again, but it’s really a team with all of us. Especially say we meet to go over patients in a weekly tumor board which includes both our medical oncologists, radiation oncologists, surgeons, our pathology colleagues, our radiologists. It’s really all of us together. And so, as we gather information from a new patient, we’re trying to really determine what their stage is. Usually first of all based on imaging and then trying to establish a diagnosis through the tissues that we’ve gotten.

And once we kinda have that information we can meet as a team and kinda come up with a comprehensive treatment plan. And that includes gathering not only the tissue information, but the molecular information that Dr. Boyle really helps us put together there. And then after we have all that information gathered then we can kinda go down these different paths depending on what stage the patient has, what their molecular drivers are, and what sort of clinical trials and opportunities we have available for them that fits them in a very personalized way which really goes back to that personalized medicine that you were talking about there.

Andrew Schorr:   

Dr. Gray, any comments from you about this wheel? 

Dr. Gray:                

No, I completely agree with what Dr. Rosenberg summarized. I think we’re at – we’re a big referral center at Moffitt Cancer Center. And many times patients may come in with a biopsy. And I wanted to just touch base with what was mentioned before about getting enough tissue. When you do do biopsies to work up for the lung cancer that is very important. And I think this wheel here and this summary here helps to highlight that. That we really want to get down to the point where we’re really collaborating with the molecular pathologist looking at your biopsy within the lab. And perhaps getting that circulating tumor DNA analysis in a blood also to make this decision.

And I fully concur that this is a team approach. We really need the pathologist to let us know what’s going on. We really need to sit down as a team and make sure that we all come up with the right decision for the patient. And that’s certainly one of the benefits of going to a place like – you mentioned Harvard and coming to the Moffitt Cancer Center certainly also.

Andrew Schorr:   

Right. So, Dr. Boyle, so there are people listening who maybe have had a biopsy somewhere else. Maybe at the community level. And here, you’ve got this big lab and you have other groups that you work with with huge analyzers and pathologists and all that that you work with. Somebody says, “Well, if I come to Moffitt” – oh, any other major cancer center, sometimes the request is made to have another biopsy or other tests. Why is that important today? Because do you have sometimes where maybe the initial analysis wasn’t as correct as it could be?

Dr. Boyle:               

Right. It’s kinda like the bane of our existence. People like to say the tissue is an issue.

Andrew Schorr:   

Nice. 

Dr. Boyle:               

And it goes along with the “if you don’t have an adequate specimen you can only get so much information out of it.” The blood testing has really helped alleviate some of that pain, but when a procedure’s going to get a small bit of tissue from the lung, it can be less than 100 cells. And we’re trying to do the best we can to learn about 100 tumor cells. So, that’s why the biopsy is so important. And I was thinking maybe we can go around the wheel. We are missing the surgeon in here, but I love this appearance and how you can go around and around and around.

Andrew Schorr:   

Right. 

Dr. Boyle:               

But the patient usually first comes in and sees their oncologist and then a biopsy can be taken, and it goes to the anatomic pathologist and they determine is it adenocarcinoma or squamous cell cancer, small cell or some other primary cancer.

And then the specimen get to genetic testing. They go to the lab and that’s where we come in. We’re looking, “What’s the tumor cellularity? Is it enough for us to even test? Can we test with the targeted small panel if it’s not enough for big next generation sequencing panel?” And we do the sequencing on our big fancy machines, but we get the results. And it really requires pretty intensive interpretation to understands the results and make sure that we’re reporting out accurate results.

Andrew Schorr:   

Yeah. I wanted you to speak to that. There’s an art to – there’s an art to medicine.

Dr. Boyle:               

Well, yes. 

Dr. Gray:                

Yes.

Andrew Schorr:   

Of course. But there’s an art to pathology. And so, you wanna give accurate recommendations of what are we dealing with to the medical team, the rest of the medical team, and that has an art to it, right? And you’re a subspecialist in that area.

Dr. Boyle:               

Okay. Right. And we don’t want to overwhelm oncologists with too much information either. So, we’re very receptive to feedback about what’s most important for actually taking care of your patients that you’re seeing. And the resistance mutations have become very important. We used to only check for one part of the ALK gene and we got feedback from the oncologist that that wasn’t good enough. They need to look at all of the ALK gene for resistance mutations. So, back and forth.

And then we also have the help of the personalized medicine group here at Moffitt. And that’s wonderful. They have [inaudible] [00:47:19]. So, they know about drug side effects. They know how to work with insurance companies. They know how to answer questions about what’s the functional effects of genetic changes. So, when we send out a report, it goes straight to the oncologist, but it also goes to the personalized medicine group for a more in-depth look. And maybe some help identifying clinical trials that the patient might be newly eligible for based on the genetic findings.

And the radiation oncologists have become more involved too. As Stephen was talking about how the genetics can play a role in the care in terms of the radiation. There’s more and more clinical trials that are getting involved in together to better understand what’s the best therapy.

Andrew Schorr:   

So, Dr. Rosenberg, what I’m getting from this is a patient might see Dr. Gray or see you or maybe a surgeon with earlier stage lung cancer as well, but that there’s this whole group – Dr. Boyle, but she rattled off a few other groups as well that are all behind the scenes. And you guys are talking about me, the patient, right?

Dr. Boyle:               

Oh, yeah.

Dr. Rosenberg:    

Yeah. Absolutely. I think that we are really in communication with each other on a pretty regular basis as a team. And I think that’s what really leads to this personalized care for the best outcome for the patient is really being very communicative about – between Dr. Gray and between Dr. Boyle, between the surgeons that we work with and just everybody really working together to try and make the best decision we can for each patient.

And gathering all the right information upfront. I think that’s really the key is making sure we have all the right information we need, whether it’s molecular or imaging, before we go down a certain path, so we don’t go down the wrong path for any particular patient. And yeah, I think as we kind of put that information together we can help really personalize each person’s care that way. And from a radiation point of view we’re using both the imaging information that we’re getting and the molecular information to help make radiation decisions.

And at Moffitt, we’re really trying to push those boundaries from imaging as well. And we talk about personalized care from these molecular changes, but Moffitt, this room will be opening up our MRI-guided radiation treatment unit which is the first in the country. There’s only a handful of places that are doing MRI-based radiation treatment. And that’s really another form of personalized care by seeing somebody’s anatomy up close and in a very particular way and designing the radiation based on individual anatomy. And so, with that better imaging we’re able to do that. So, there’s a lot of ways to personalize care for patients moving forward.

Andrew Schorr:   

So, Dr. Gray, I wanna talk about the spread of cancer. So, Ed talked about how he had this on his liver. But you figured out – you all figured out it came from his lung?

Dr. Gray:                

His lung. Yes.

Andrew Schorr:   

Lung cancer can spread. Cancer can spread generally. But here we have people with metastatic cancer who are living longer with some of these approaches. 

Dr. Gray:                

Yes.

Andrew Schorr:   

Radiation, immunotherapy.

Dr. Gray:                

Therapy.

Andrew Schorr:   

Targeted therapy. 

Dr. Gray:                

Different therapies.

Andrew Schorr:   

So, when you tell somebody, “Mr. Jones, yes, you’re right. Imaging, we see your cancer spread.” That’s not the end of the story.

Dr. Gray:                

Absolutely not. No. So, what we also look at are these genetic findings, the pathology findings, the markers for the immunotherapy. But at the end of the day what your goal is is to extend life and to add quality of life to patients. And so, as we look at this information, we want to make sure that we’re making the right decisions. And this is the goal ultimately of personalized medicine.

Yes, your cancer may have spread, but we can give you treatments that are gonna knock down the cancer, get it to shrink down, and to a point that sometimes may become undetectable on the scans. We do think that there’s cells still circulating there, but we could still continue to follow you and keep track of the cancer and make sure that you’re – we’re helping you to manage this properly.

Andrew Schorr:   

Okay. So, I wanna remind our audience, send in your questions. This is an ask the expert question. We have an expert patient who’s lived it. And I know Ed, you spend time talking to other patients and family members. We have Dr. Boyle and we got her out of her lab there.

Dr. Boyle:               

Yeah.

Andrew Schorr:   

You might not see her in the exam room, but she plays a key role. We have Dr. Rosenberg who’s even travelling and joining us. And Dr. Gray. So, if you have a question send it to lung@patientpower.info.

And I wanna tackle a question. Here’s one we got from Gretta, “What percentage of blood biopsies are accurate?” She said, “I had one done and it showed I no longer had the BRAF mutation, but subsequent tissue biopsy showed I did still have it. So, how reliable are the liquid biopsies?” And I think you, Dr. Boyle, mentioned that a little bit.

Dr. Boyle:                                 

Right.

Andrew Schorr:   

So, this is a new area of pathology. In this area, how much can you rely on it?

Dr. Boyle:               

Right. So, the positive results we are finding when you’re testing with a reputable company they are – can be very reliable. Just as reliable as the tissue testing for the positive results. And there are some advantages even. Because in the body you might have a tumor on the lung that’s a little bit different from the tumor that spread to the liver, whereas the blood is a big mixing bowl. And if you have some DNA sloughing off the two different tumors, that DNA is mixing in the blood. So, you’re getting a more comprehensive look at the mutations in the blood.

One big, big disadvantage that’s represented in this question is when you get a negative result. When you get a negative result like she got for her BRAF in the blood, it’s really a non-informative result as opposed to a negative result that you can hang your hat on. Because you don’t really know how much cell-free DNA is actually sloughing off the tumor and circulating in the blood. And so, if it’s not in the blood, you might get a negative result when really, it’s still BRAF positive in the tumor as she found when she had this tissue tested again.

Dr. Gray:                

Positive.

Andrew Schorr:   

But this is evolving, right? I mean we couldn’t even talk about liquid biopsies not too long ago. So, the sophistication and the sort of getting down almost Nano – the Nano-level, you’re working on it, right?

Dr. Boyle:               

And that’s what Jhanelle and I have talked about this so much with algorithms for how to really understand the results and use the results.

Dr. Gray:                

Yes.

Dr. Boyle:               

And this is something that – at Moffitt we know well about the negative results being more like non-informative results with the blood. And we find them very helpful when we interpret them appropriately. 

Andrew Schorr:   

Okay. Well, there we go to the art and the sophistication of the tools that continue to develop. So, a lot of computing power folks that go into this. And then the wisdom of folks like Dr. Boyle.

All right. Here’s a question we got from Greg. I think this is for you, Dr. Gray. “What is happening in research for those of us who do not have target therapy gene mutations and also have a low tumor burden. Is chemotherapy the only option?” 

Dr. Gray:                

No. So, if you look at the studies that have occurred so far, looked across the patients that have a low tumor burden or have a negative PD-L1, or have no actionable or driver mutations, we still know that chemotherapy plus immunotherapy’s the way to go. We’re also doing a lot of research in that setting is that once those or if those stop working for you, what are gonna be the next steps? And that I think is certainly an area of need. One of the things that we’re looking at is combination immunotherapy strategies. That perhaps giving you one immunotherapy therapeutic agent was not enough and that you perhaps need two.

I think that chemotherapy is still very important. And doing combination strategies down the line with some of these novel agents. When we look at some of the – a lot of the trials, even the ones where you give chemotherapy with immunotherapy, if you look at the data, most of the benefit upfront, for now at least, appears from the actual chemotherapy. So, chemotherapy is very good at reducing disease bulk. But the immunotherapy can then come in and activate – help to activate your immune system. And the ultimate goal of immunotherapy is to create immune memory, okay?

Almost along the lines of a vaccine. You get your flu vaccine once a year because it mutates. You get your hepatitis vaccine. You’re not getting it every year. You only get it for a sequence. And the purpose there is that your immune system should be able to sustain on your own. And we wanna do that for patients upfront. That would be the ideal. But we also recognize that we just – this is very new, and we don’t know enough.

So, I think expanding more in combination immunotherapy strategies, looking at novel agents, looking at where chemotherapy’s target also and probably repurposing those drugs a little bit so that we can actually hit the target even better than regular systemic chemotherapy and reducing toxicities. There is a plethora of research going on within all avenues.

So, I think the key thing there is that if you have something and it’s not working for you, come to a center of excellence like Moffitt Cancer Center and sit down and talk to us and we can let you know. And exactly to your point. We talk to physicians all across the globe. Work very closely with Dr. Siegrist at Harvard. We share patients. Share data constantly. Even if we may not have something for you here at Moffitt, there may be somewhere else in the United States that we can send you also.

So, I would not – I don’t think – the key thing there is that giving up should not be the first option by any means.

Andrew Schorr:   

Amen. I just wanna drop back for a second and make sure everybody understands this whole world Dr. Rosenberg talked about a little bit. So, the immune system let us down. And Dr. Boyle a while ago used this term checkpoint.

Dr. Gray:                

Yes.

Andrew Schorr:   

So, Dr. Graham let you be the professor here. See if I get a good grade. The cancer cell has this kind of protective world around it where medicines traditionally maybe don’t kill it. 

Dr. Gray:                

Yes. Correct. Correct. 

Andrew Schorr:   

Right? Okay. So, what the immunotherapies are doing, maybe more than one, is to knock down that barrier.

Dr. Gray:                

Yes.

Andrew Schorr:   

So, that whether it’s with radiation like Dr. Rosenberg talked about with these immunotherapies where your immune system can do its job in killing the cancer cells, the abnormal cells.

Dr. Gray:                

Yes.

Andrew Schorr:   

And you also eluded to something else where the immunotherapy can continue to do this surveillance wherever the cancer may be. Whether it’s spread to Ed’s liver, whether it’s gone to somebody’s bone.

Dr. Gray:                

Yes.

Andrew Schorr:   

Wherever it is it says, “Oh, now I see you.”

Dr. Gray:                

Yes. 

Andrew Schorr:   

“And guess what? Bad news. I’m gonna kill you.” Right? 

Dr. Gray:                

Kill you. Correct. 100% right. So, one of the ways to think about this is that the immunotherapies, if you see the commercials out there, for example, for Opdivo or Keytruda, they actually do not kill the cancer cells. So, this is very different than chemotherapy. Traditional chemotherapy we’re very used to goes in, actually kills the cancer cells. Exactly. The immunotherapies are unmasking the tumor to the immune system allowing the immune system to now recognize the cancer cell as foreign and then attacking the cancer cells. And exactly, your immune system should then sustain on its own. And that is the ultimate goal of immunotherapy.

Andrew Schorr:   

And Ed, that’s what you’ve been living with, right? You said earlier you’re stable. So, you’re taking immunotherapy and it’s kinda knocking it back, right?

Edward Cutler:     

Yes, it is. To what Dr. Gray said with respect to, I guess, first line of treatment with the combination of chemo and immunotherapy, what is the standard now with the combination? Is it Alimta, Avastin and then Keytruda or Opdivo?

Dr. Gray:                

Yeah. So, right now as of 2018 what is approved by the FDA – I’m just going back a little bit. Remember there’s two main types of lung cancer. There’s small cell lung cancer, there’s non-small cell. There’s two main types within non-small cell of a non-squamous type of lung cancer and then a squamous cell type lung cancer. And to Ed’s point, he’s completely right, your chemotherapy that we choose for you depends on your type of lung cancer. And I think that’s what you’re alluding to in the question.

Edward Cutler:     

Yeah. Yeah. 

Dr. Gray:                

So, there are data that has shown for non-squamous, non-small cell lung cancer if you combine Carboplatin plus Pemetrexed plus Pembrolizumab together, that is the FDA-approved regimen for first-line – for that type of non-small cell lung cancer.

Now, if you have non-small cell lung cancer and the subtype is squamous cell, the drugs that are approved right now are Carboplatin, Paclitaxel, plus Pembrolizumab. Or you can substitute that Paclitaxel for something called Nab-Paclitaxel or Abraxane. Paclitaxel can cause some infusion reactions in patients. And so, the Nab-Paclitaxel is formulated to minimize that infusion reaction. So, there’s some flexibility there, but they’re still in the same class there.

So, and then if you have the small cell lung cancer actually the regimen that is approved there is a platinum – so cisplatin (Platinol) or carboplatin (Paraplatin) plus Etoposide plus etesolismab. So, it’s really even within that spectrum these are all ways of personalizing medicine for patients. And really having that level of information from the pathology and the biopsy side so that we can make the best decision for the patients. 

Andrew Schorr:   

Okay.

Dr. Gray:                

And then when that data comes in is having that expertise about which one is gonna be the right for which patient.

Andrew Schorr:   

Right. But there’s one other aspect I wanna put on. So, for patients, whether you go to Moffitt or another major center, rather than some of those names she mentioned, there may be a clinical trial where it has a number.

Dr. Gray:                

Yes.

Andrew Schorr:   

And it says, “We’re gonna give you X, Y, Z, 1, 2, 3, 4. That’s what we recommend,” which hasn’t been approved, but they believe may offer a better option for you.

Dr. Gray:                

Yes.

Andrew Schorr:   

Did I get it right?

Dr. Gray:                

Yes. Correct. And so, we call them license plates, right? License plate numbers. And so, when the drug first comes out of drug development, it kind of gets this license plate number and Nivolumab, Pembrolizumab, all of them came out with these license plate numbers as we call them. And then you just – as they move forward, and they show promise that they then develop a more formalized name – nomenclature for the naming. But to your point, it is very important to also look at clinical trials within that setting. That’s how we make these strides. That’s how we make these improvements.

We participated in a very first trial with Nivolumab here at the cancer center and I still have a patient that is alive six years out. His daughter was five when I met him. She’s 11 now. They sent him to hospice on the outside and I said, “You know what? We’re gonna try this medication.” And he has not received the immunotherapy in four years. And this is a perfect example where his immune system was able to work, get the tumor down, and now it’s sustained on its own off therapy. And if he didn’t come to Moffitt, he would’ve just been sent to hospice.

Andrew Schorr:   

Okay.

Dr. Gray:                

So, this is where exploring – making sure that you explore all of your options is very, very important. What I always recommend, you know what? Can you get treated locally? 100%. But at least go in for that consultation. Make sure that there’s not something newer, more novel, something that we think may be a little bit better. Clinical trials is always a way to explore things. At the end of the day the standard of care FDA-approved therapies are always there, and we can always give them to you whenever. You may have this option for this trial. And I think my patient got one of the last slots on the trial nationally. And I think… 

Andrew Schorr:   

…wow. What a story. I just wanna recap a couple things for our audience, okay? So, you got it now about personalized medicine and getting what’s right for you, whether it’s one of these targeted therapies in this growing list of genes that Dr. Boyle has talked about. And drug companies and government working to development things that match up with that. Immunotherapy, maybe more than one. Some that have a commercial name and some that have a license plate like Dr. Gray just described.

And then this whole idea of radiation oncology where Dr. Rosenberg and his colleagues are finding out how does all this work together? How can radiation actually trigger something in a cancer cell that then also helps it say to the cancer drug, “Hi, I’m here.” Boom. They get hit by a cruise missile, right? So, that’s what they’re working on.

All right. We’ve got a bunch of questions. Remember, lung@patientpower.info. This one came in from Leo. And Leo says, “Any new research or treatment regarding patients with the TP53 mutation?” So, first of all, Dr. Boyle, what is TP53? 

Dr. Boyle:               

Oh, goodness. I don’t off the top of my head know how that spells out, but it is a tumor suppressor protein. And it’s a gene which we find frequently mutated in all cancer types. And it often causes a worse prognosis. And there are many researchers trying to see if there are better drugs to target therapies. And lung cancer, the clinical trials are pretty early. The highest you get is phase one and two. So, there has not been a lot of success yet. In leukemia I think there’s more phase two trials.

We have an excellent researcher here, Elsa Flores, who is looking at animal models in vivo studies to try to understand more. Now, one thing sort of interesting about TP53 is that if you have lung cancer with TP53 and a KRAS mutation, that mutation is gonna be more likely to respond to immunotherapy.

Dr. Gray:                

KRAS mutation. 

Dr. Boyle:               

There’s a really nice paper out by John Heymack about this if there’s also an STK11 mutation. So, then it’s a lower likelihood of response to immunotherapy.

Dr. Boyle:               

So, with more and more research we’re lending some of the nuances of these and we’re hopeful that there’s going to be more that can be done with the TP53 mutations in the future.

Andrew Schorr:   

Okay. So, Dr. Gray. So, you were nodding your head while she explained that? 

Dr. Gray:                

Yeah. Yeah.

Andrew Schorr:   

So, in other words, you’re looking at not just one gene being the bad guy, but this constellation in a given patient.

Dr. Gray:                

Right.

Andrew Schorr:   

And does that tell you something that you could do in a more refined way for them?

Dr. Gray:                

Right. I think this is we’re coming into a center where we have this level of expertise and we’re sharing data across the different centers. But exactly what Dr. Boyle noted is that when we look at these genomic reports, right, you’re getting a lot of information coming out back at the medical oncologist. And knowing how to fully understand and interpret that data so you can make the best decisions for the patient is very helpful.

So, if we see a KRAS mutation, the P53, without an STK11 mutation, certainly that will move immunotherapy up on for the armamentarium for the patient. Now, this is a little bit in experimental mode, but we’ve seen similar data here at Moffitt. And it’s really starting to pick up traction across different cancer centers and lung cancer experts.

Around the specific question of the P53 mutation, we do have a compound that we’re looking at here in collaboration with AstraZeneca. It came from a trial that I had written and am working on that came from work derived here from Moffitt Cancer Center. It’s called AZD1775. But it basically what it is is it’s looking at inhibiting the cell cycle. I’m gonna take us back to biology a little bit. And cells, how they replicate, basically they have to go through mitosis, right? You have to replicate your DNA and then split off and divide.

And so, what the P53 does is it’s almost – as Dr. Boyle mentioned, it’s a tumor suppressor. What does that mean? It actually puts a stopgap, an intentional stopgap when cells go to replicate. And it makes the cell stop and check and say, “Do I have any mutations? Should I move forward or not?” What cancer cells do is they’ve lost – they mutate the P53. So, they don’t get that stop in place. They just keep replicating. Even though technically these are abnormal cells, they’re damaged cells and they shouldn’t replicate themselves.

So, what that drug does is it intentionally incorporates – if you have that P53 mutation your cells are not stopping when they’re replicating abnormally. This AZD1775 helps to add that stop so the cells can check themselves and say, “Hey, you know what? We’re really not replicating ourselves properly. We should actually go towards cell death and not cell survival and replication.” So, there are definitely trials that are looking at the P53, to Dr. Boyle’s point, including one that was derived here. And as Ed mentioned, something that you can only find here at Moffitt. And we hope to have that data out maybe later this year or early next year.

Andrew Schorr:   

Okay. Stay tuned.

Here’s a question we got from Jim. “How does immunotherapy work in EGFR mutations after targeted therapies no longer work?” Do you wanna comment?

Dr. Gray:                

Yeah. Yeah. So, that’s a great question. So, one of the key things as I mentioned before is if you find a mutation such as the EGFR mutation you go down the realm of a targeted therapy. So, say to treat patients with a targeted therapy’s very, very important.

I wanna take this opportunity to say that you should not combine an EGFR inhibitor with an anti-PD1 with an immunotherapy. It significantly raises patient’s toxicity. So, if a physician ever – if that ever comes up, at least for right now, the answer, you should decline that, and no one should be offering that to you. Exactly.

So, I agree that the best way to incorporate them right now is through a sequential approach. So, you start with the EGFR inhibitor. And there’s four of them actually FDA-approved right now. So, you may get sequenced from one EGFR inhibitor to the next. What people are looking at right now is should we go straight to immunotherapy, should we go straight to chemotherapy, or should we go straight to a combination strategy of chemotherapy and immunotherapy?

I think based on the data for right now, most of us as long as we think that it’s safe will go to a combination of chemotherapy plus the immunotherapy based on the data. This is gonna be looked at more in detail to finally answer this question. It also depends on the wishes of the patients too. So, if you think that you cannot – if a patient cannot tolerate, for example, immunotherapy combination with chemotherapy, we may start with one or the other and then move on. But definitely I agree that the sequencing is gonna be the best way to do that.

Andrew Schorr:   

Okay. Let’s talk about the toxicity for a minute. So, Ed, you had – earlier you had some treatment that was pretty tough to take, right?

Edward Cutler:     

Yes. Yes, it was. The only major side effect that impacted me was colitis. But it was major. It was really, really major.

Andrew Schorr:   

And you had to change? You changed?

Edward Cutler:     

And when I read the protocol, yes, that was one of the potential side effects.

Dr. Gray:                

Right.

Edward Cutler:     

But that’s all it was was a potential side effect. I took my chance with it. I’d never had colitis before and then it hit me. And I’m still kind of dealing with that to some extent. Nowhere near what I dealt with three years ago.

Andrew Schorr:   

Okay. And your treatment was changed?

Edward Cutler:     

And my treatment was changed. Yes.

Andrew Schorr:   

Okay.

Edward Cutler:     

And that was a combination – a two drug combination trial.

Andrew Schorr:   

Yeah.

Edward Cutler:     

A phase one trial.

Andrew Schorr:   

Here’s a question we got in from Wendy. So, it’s a little technical, but she says, “I’m currently keeping my stage four non-small cell adenocarcinoma at bay with monthly maintenance infusions of Pemetrexed.” Did I get that?

Dr. Gray:                

Yes. Absolutely.

Andrew Schorr:   

“I was diagnosed in August of 2015. Nothing visible on PET scans, but the chemo’s been prescribed to keep the cancer reappearing.” And her concern is the long-term damage, she wonders, of getting chemo infusions over a long time. She says, “What could be the downside of chemo over a long term?” Dr. Gray?

Dr. Gray:                

Yeah. So, one of the things that we – well, congratulations. I’m glad that you’re doing so well. That’s really inspiring to hear. And I think that speaks to the fact that there are patients and cancers out there that respond to chemotherapy and I think that we should still keep that in mind.

The long-term side effects that we generally worry about with chemotherapy are how they affect your blood counts. And by blood counts, I’m talking about your bone marrow. So, your red blood cell counts. So, your hemoglobin and your hematocrit. Your white blood cell counts. Your leukocytes. Your neutrophils. Things that help you fight bacterial infections, viruses. And then your platelet counts. These really help with your clotting. So, if you cut yourself.

Pemetrexed, in particular one of the things that we’ve noted when you keep receiving this treatment in particular over time is that the anemia seems – can sometimes be a rate limiting step. So, I’d definitely keep an eye on your hemoglobin and hematocrit.

But I’ve had patients on these maintenance therapy agents for many years. A lot of times what I will do to lessen the burden for the patients. Normally the drug is infused as an IV infusion over ten minutes every three weeks. I will go to once every four weeks so that you’re only coming in once a month for a treatment to add more to quality of life. And then I’ll start increasing the frequency of the scans to less frequent. So, maybe quarterly you’ll get a scan instead of every six weeks. So, hopefully all these scans can help lessen the burden of the infusion and also help to improve quality of life at the end of the day. But I would certainly be careful of watching the blood counts within the study.

Oh, I think you’re on – are you on mute?

Andrew Schorr:   

Yeah. I’ve got it. Sorry.

Dr. Gray:                

Yeah? Sorry. 

Andrew Schorr:   

Dr. Rosenberg, related to toxicity you referred earlier about MRI-guided radiation. What are you doing in the radiation oncology field to get at the cancer, but not effect either healthy tissue – and also lower the side effects that can go with radiation. People that fatigue and other things that go along with it. And all of you have been talking about higher quality of life where you might be living with lung cancer.

Dr. Rosenberg:    

Yes. Yeah. It’s a great question. And I think how we’ve approached this in radiation oncology is actually by shortening our treatment courses. And as our technology has improved it will also give us very small volumes of irradiation with high doses to destroy cancer cells, but also sparing normal tissues. And as patients are living longer with lung cancer, we kinda have to say sometimes they’re responding well to chemotherapy or immunotherapy or targeted therapy, but one area is starting to grow, we use this targeted therapy called stereotactic body radiotherapy, SBRT. So, [inaudible] go after these important small areas that might be not responding appropriately or may even be resistant.

But these are targeted areas that we’re irradiating that are very small in volume. That’s really helped us limit toxicity, but to normal tissues going forward. And with the new MRI-guided treatment program, which is where my focus is gonna be, is that by having the MRI help us guide our treatment in real time, we can make our volumes even smaller. And by shrinking our volumes and targeting tumors more appropriately we can hopefully spare normal tissues and actually decrease side effects long-term for patients.

And so, again working with our medical oncology colleagues is that if there’s an area of resistance that pops up, an area that we can very precisely target, we’re still sparing a lot of the normal tissues in your body.

Andrew Schorr:   

Okay. Precision radiation oncology?

Dr. Gray:                

Yeah. Yes.

Andrew Schorr:   

Okay. 

Dr. Gray:                

And we do that also. And if I may add that if there’s somebody who’s on a treatment benefiting and they just have one area that’s kinda this rogue tumor that breaks through and becomes resistant, that definitely looping in the radiation oncologist, working with Dr. Rosenberg and his team, and targeting that specific area can be very effective for patients.

Andrew Schorr:   

Okay.

Dr. Gray:                

Before you switch therapy.

Andrew Schorr:   

Here’s another aspect of immunotherapy. So, we talked about these PD1, PD-L1 drugs, checkpoint inhibitors. So, another area that’s particularly happening in the leukemia’s that I know well is what’s known as CAR t-cell therapy, chimeric antigen receptor t-cell therapy. Where if I get it right, correct me if I’m wrong, you can sort of engineer t-cells to become sort of a targeted therapy.

Dr. Gray:                

Yes.

Andrew Schorr:   

All right. So, what about this in lung cancer, Dr. Gray?

Dr. Gray:                

Yeah. So, it’s a great question. So, one of the areas – this has really taken off in the hematologic malignancies are these CAR-T therapies. The hematologic malignancies are very well-defined by specific markers on the cells that are uniformly found across different types. So, lymphomas, leukemias. In the solid tumor realm, it’s been a little bit more of a challenge with finding where to specifically target. And also, to target the cells without adding significant toxicity to the patients.

So, we do have what’s called an ICE-T therapy here. It’s the immune and cellular therapy. It has medical oncologists on that team, both hematologists and hematologists. And they’re working together to help bring what we’ve learned from the hematology world over to the solid tumor realm. So, it’s new. I don’t think it’s yet ready for FDA-approval, but absolutely a very exciting, exciting field. Again, the purpose of these is to create these long-lasting responses with a personalized medicine approach.

Andrew Schorr:   

Yeah. I wanna thank Gordon for that question. I think we hear about – you mentioned TV commercials, or we see an article in the paper.

Dr. Gray:                

Yeah. Great question.

Andrew Schorr:   

And we say, “Oh, how does that apply to me?” Or, “Should we get on a plane and go somewhere because they’re trying this out?” It’s really tough. So, Dr. Boyle, you see this changing field.

Dr. Boyle:               

Okay.

Andrew Schorr:   

What would you say knowing what you know in going on and identifying new genes, if you had a family member – and I hope you haven’t, but if you had a family member diagnosed with one of these conditions, what advice would you give them? Because you’re on the inside. Or maybe you have friends or neighbors that call you up, “Oh, my God. We got this diagnosis. What should we do?” What’s sort of an operating system for patients and families today? What would you say?

Dr. Boyle:               

Right. Well, one thing I want to ask always, what Jhanelle was talking about earlier with the clinical trials, if you are getting the optimal standard of care plus whatever new innovative potential therapy might be available with those trials. And there actually is a better outcome with the participation in the clinical trials. They’re very carefully designed.

So, I would want a family member or a patient, it is the same, to get as much information as possible. And like Jhanelle said, it’s fine to get your care locally, but to get a second opinion at the most advanced center available to you for a second opinion. Get more information. See what’s available. Consider clinical trials. Sometimes just following the basics. Like if you have an EGFR mutation to get an EGFR inhibitor therapy and not be wanting say immunotherapy just because it’s the newest thing. That’s what I’d be thinking about. And getting the rapid care can be important too.

One thing I wanted to add onto what Jhanelle said earlier about the T-CAR conversation is that we also have a trial here with the tumor infiltrating lymphocyte. It’s not by definition a T-CAR trial, but it’s in lung cancer. And they’re basically taking lymphocytes out of tissue and growing them up in cell culture and reinfusing them into lung cancer patients in the hopes that the reinfused cells will attack the tumor. And I just think this is amazing progress that this being tried in lung cancer too.

Andrew Schorr:   

Here’s a point I wanted to make. So, I hope what all our viewers get – and I think we have some pretty savvy questions that have come in. The field is changing. And so, Dr. Gray, I’m sure when you went through your training there wasn’t always a lot to talk about with patients, right? 

Dr. Gray:                

Oh. No. That’s very true.

Andrew Schorr:   

And now we have people like Ed who are living longer, living pretty well. There are side effects we’ve talked about. Ed was talking about trying to limit that. So, the quality of life goes along with living well and living longer. But there’s a lot of progress being made, and you and your family have to be plugged into that. And yet, Dr. Boyle just referred to that. Don’t get excited about something just that you see on TV because it may not be right for your specific situation. And Dr. Gray was warning about that too. If you have EGFR, that’s not the time for immunotherapy, right?

Dr. Gray:                

Correct.

Andrew Schorr:   

Even though you saw the ad of people in town square. New hope for lung cancer. 

Dr. Boyle:               

On the highway. Yeah.

Dr. Gray:                

Yes.

Andrew Schorr:   

Yeah. So, you really have to – you really have to think about that. So, testing, broader panel, second opinion, team approach? 

Dr. Gray:                

Yes.

Andrew Schorr:   

We’ve got a wonderful team here.

Here’s a question that we got in. We just have time for a few more questions. But Helen asked this question, “Is there any research or anecdotal information on how much the drug Alimta adds to the efficacy of another immunotherapy Keytruda. Does it continue to be effective indefinitely or does it only work for a while?” Dr. Gray?

Dr. Gray:                

Yeah. So, there’s a recent study called the Keynote-189 study that combined Pemetrexed, Carboplatin, plus Pembrolizumab. So, Pembrolizumab is the immunotherapy. The Pemetrexed is the chemotherapeutic agent. And they treated those patients with the Pembrolizumab up to about two years.

The study was published, and it was found to be positive in the sense that it improved patient’s overall survival. So, how long were you gonna live? And also, what we consider your progression for your survival. How long did it take your cancer to actually progress to the point that we would need to switch your therapy? So, it was longer in that group than just giving the chemotherapy group.

You ask a very good question. These are the questions that we also, within the lung cancer field, are asking. How long do we really need to give these therapies for? Especially when you’re giving them in combination with immunotherapy when the goals of immunotherapy are to create long-term memory.

We have studies looking at giving immunotherapy for one year. We have studies continuing the immunotherapy indefinitely. And we have studies looking at giving the immunotherapy for two years. I think outside of the stage three – in the stage three setting, the clear data is that you give it for one year. Outside of that, I still think that it’s still a tossup. My suspicion is that you should at least probably go beyond one year if you can and see if you can’t get to the two-year mark. That’s where most of the data is at a minimum.

I actually have a patient now coming up on her two-year mark in February of this year on Pembrolizumab and I’ve started having those discussions with her and it’s an open discussion that, “This is what the data shows. What do you feel comfortable with?” And so, I think there needs to be a shared decision-making process within this realm also. And what the patient feels comfortable with and what the data helps to support. So, I think keep having those conversations, especially if you’re getting it combined with the immunotherapy. And hopefully there will be more to come definitively.

Just for a historical perspective, if you look back many decades ago when chemotherapy first came out, we used to give chemotherapy for a year. Then they did the trials where they actually looked at it at a year versus six months. The outcomes were the same with giving it over six months. And then they went from about six months versus about three months of therapy. And now went back a little bit, but added the maintenance in. And so, there’s definitely these trials will come, it’s just going to take time. The world of immunotherapy is very novel within the realm of lung cancer. And so, we have lots of growth to do.

But fantastic question. That’s probably one of the things that we sit and debate at our meetings very frequently.

Andrew Schorr:   

So, as we come near the end of our program, I wanted to get some final comments from our panelists. I’m gonna end with Ed because Ed, I want you to talk to other patients and family members.

But what I get from this is the field is pretty rapidly changing. Whether it’s in radiation and how that applies to other therapies. Whether it’s combination therapies, sequential therapies, duration of therapies we were just talking about with Dr. Boyle. It’s about identifying new genes or combinations of genes and trying to figure that out. So, what do you wanna say to a patient audience?

I’m gonna start with you, Dr. Rosenberg. So again, we have people all around the world and they or a family member has been given what’s a pretty terrifying diagnosis.

 Dr. Rosenberg:    

And it’s a scary time as they’re facing this. And actually, what I’m talking about here in Madison is actually putting together your medical and emotional teams as you’re basically facing this new diagnosis. And I think that’s the big thing is putting together a team and being someplace where you have the support and you feel comfortable. And also seeking out multiple experts to try to come up with the best plan you can moving forward.

And I think as Dr. Boyle, Dr. Gray, as the panels all alluded to, seeking that second opinion just to at least know what all your options are and are available to you is really important. Building your treatment team which includes so many different experts both that you’re gonna meet in person and behind the scenes. And I think that’s the real key aspect to this.

Andrew Schorr:   

Well, thank you for what you do. And this cool area you were talking about having radiation trigger a response in the cell that can make it more responsive to new medicines is really great. Good luck with all of that. Thank you.

Dr. Boyle, so you are a CSI detective. You are. You have like a magnifying glass.

Dr. Boyle:               

I love my job.

Andrew Schorr:   

Yeah. You have much more powerful tools than that. But you’re a sleuth. So, are you confident that this field is – will continue to expand to really unlock these secrets so you can say to these other team members, “Hey, I think this is what we’re dealing with and here’s a key pressure point to go beat that cancer.”

Dr. Boyle:               

Yes. Yes. I’m very optimistic. When we validated this 170 gene panel we did not even know if we would be reimbursed, but we did it anyway because we have so much optimism that it will have value and show value. And I really feel like understanding the cancer better. And it’s a key to better therapy. And my thinking is that patients should hold onto their hope throughout their whole experience and stand their ground. 

Andrew Schorr:   

Yes.

Dr. Boyle:               

Know what they want and don’t want and ask questions to their oncologist if they have questions. Because there’s a whole new world here and we’re all trying to figure it out together as a team. But we really appreciate the input from the patients as well. I think that’s helpful to helping all patients and future patients as well.

Andrew Schorr:   

Well, I wanna thank you for what you do behind the scenes as far as we patients and family members see you. But with your colleagues around the world continue to make these discoveries so that the therapies can be targeted or more broad. But whatever they are, know what we’re dealing with, so we get what’s right for you. Dr. Boyle, thank you for being with us too.

Dr. Gray. So, you have these partners here and we have patients and family members who you’re partners to. And as I alluded to earlier, in your own career you’ve seen a lot of change.

Dr. Gray:                

Yes.

Andrew Schorr:   

Is this a message of hope? And are you comfortable that more of us – even now we’re talking about small cell lung cancer where there’s progress being made that can extend life.

Dr. Gray:                

I’m very hopeful. I think that we have completely revolutionized how we treat patients – treat lung cancer and treat the patients battling lung cancer. We’re with you there right along helping you with that fight. And to your point, when I first started doing this I literally spoke to patients about chemotherapy. That’s what I had to offer. And it was just trying to make that selection process about which chemotherapy I thought was going to be right for you. And helping you with sequencing. “Okay, we’re gonna start with this and then we’re gonna plan for this and we’re gonna plan for that.”

And the game has completely changed, I think, with the genomic profiling. It is extremely important. We really have to go to these broad-based panels up front. And for right now, I just wanna emphasize tissue is the gold standard, but I really think that circulating tumor DNA is something that we can – certainly we’ve made a lot of significant progress and then can identify these mutations.

As you identify these mutations, checking them longitudinally over time to see how they evolve is gonna be very important. And that will help us continue to personalize treatment at what point do you pivot from a targeted therapy to a clinical trial to an immunotherapy to a chemotherapy. And all of these things come from sitting down, looking at the scans, looking at the patient, looking at these molecular reports, getting everybody on the same page and then making – again I think having a shared decision model. Setting, “What are your goals? What are your hopes?” And then making sure that we match that as best as we can.

Andrew Schorr:   

Wow. Get tested, folks.

Dr. Gray:                

Yes.

Andrew Schorr:   

Have your family member get tested and then raise the question with your team, “Do we needed to be tested again?” 

Dr. Gray:                

Yes. Yeah. 

Andrew Schorr:   

All right.

Dr. Gray:                

Absolutely.

Andrew Schorr:   

And then I think Dr. Gray, I just wanna underscore a point she made about your goals. 

Dr. Gray:                

Yes.

Andrew Schorr:   

So, Ed thinks about that. And Ed, I’m gonna give the final comments to you about speaking up for yourself.

Dr. Gray:                

Yes. 

Andrew Schorr:   

How do you get the care that’s right for you and how do you wanna live your life? I mean you and Donna wanna do some more traveling, right, Ed? I hope you can.

Edward Cutler:     

We’ve been very fortunate. No question about it. When I first got my diagnosis, I was devastated. I thought my world was gonna end in a year. But I started talking to my doctors, started talking to other patients in similar situations and I found that, yeah, there was hope. I put together a bucket list. And I found that my bucket list wasn’t gonna be limited to a year. So, I expanded my bucket list. And now it goes on at least ten years out now and I’m very hopeful of that.

As I said, I’ve spoken with a lot of patients here within Moffitt, around the state, around the country, and some internationally through support groups. And we help each other. We cry on each other’s shoulder and then we tell each other our problems. And somebody has an experience that they went through and it might be helpful to another patient. Now, I think it’s very important to open up your heart and open up your ears and your mind and listen to other people. Don’t just look at what you read on the internet. Who knows what the truth is what you read on the internet. A lot of it is – I don’t know. It’s not necessarily factual.

But I think if you talk to you doctors, to your team, and your team doesn’t include just your doctors. It’s your nurses, it’s the nurse’s aide, it’s the social worker, it’s the nutritionist.

Dr. Gray:                

No. 

Edward Cutler:     

All of these people can be very helpful for just about everybody who has an advanced diagnosis.

Andrew Schorr:   

Well said, Ed. We have this medical team here that represents some of those others that Dr. Boyle mentioned, the personalized medicine people, the pharmacist, you mentioned social workers. Before we have to go, I wanted to give you the chance if you want to say thank you to these folks or what they represent on behalf of patients. What would you wanna say?

Edward Cutler:     

I am just so thankful to every person that I’ve dealt with here at Moffitt. They have made the process if not the simplest thing in the world to deal with, pretty darn simple anyhow. My doctors have explained things to me that I didn’t even know how to ask the questions in some cases. And it’s just been wonderful. I’ve become an advocate for Moffitt. I talk to people in the community. I go to Tallahassee with a group of people and talk to our legislatures about funding for Moffitt. I’m participating in the Miles for Moffitt next month as a volunteer. Not as a runner, but as a volunteer, to help raise money for Moffitt to find if not the cure at least the way to extend someone’s life with good quality.

Andrew Schorr:   

Wow.

Edward Cutler:     

And I thank you all for that.

Andrew Schorr:   

We wish you all the best. I wanna say to our audience that could be anywhere in the world, I think the lesson of what Ed is saying is go to a center where they’re knowledgeable to at least get a second opinion, Dr. Boyle had mentioned that earlier, and connect with a team like this. And then when they help you and you’re given higher quality of life hopefully and longer life, then go to bat for other people. Whether it’s with your center, like Ed has, or in a state, speak out because you can help a lot of other people. I try to do that too.

Edward Cutler:     

One other thing, Andrew. I have another thank you. Well, it’s actually two other thank yous. First to my wife for being a great support. And second, to the man who had the vision to make Moffitt reality. And that is H. Lee Moffitt who at one point in time was the speaker of the house of the Florida legislature. It was his vision. Unfortunately, he had cancer and that generated his vision. But he’s still going strong and he’s still working very hard for this institution.

Andrew Schorr:   

Well, I wanna mention then lastly that we can all make a difference. The doctors making difference, patients, family members in that collaboration and in helping others. I wanna thank everybody for being with us today. I’m Andrew Schorr. Remember, knowledge can be the best medicine of all.


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

What Is the Value of Diversity in Clinical Trials?

Clinical Trial Mythbusters

Clinical Trial MythBusters: What Is the Value of Diversity in Clinical Trials? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

In this MythBusters program, Senior Vice President and Chief Medical Officer (CMO) of ASCO, Dr. Robert Schilsky, and 20+ year CML survivor, Mel Mann along with Cecelia Mann, will unpack some of the issues that have led to the lack of diversity in clinical trials and initiatives in place that are changing all of this.


Transcript:

Andrew Schorr:
And greetings from near San Diego, Carlsbad, California. I’m Andrew Schorr from Patient Power. Welcome to this Patient Empowerment Network program, the next in our series Clinical Trial Mythbusters, and this program is so important, discussing what is the value of diversity in clinical trials. And, believe me, you’ll hear it is so critical. We have to do better, and we’re going to discuss that over the next hour.

I want to thank the companies that have provided educational support through grants to the Patient Empowerment Network. They have no editorial control, but their support is welcome. And that is AbbVie Incorporated, Astellas, Celgene and Novartis. All right.

We’ve got a lot to discuss today, and we welcome your questions along the way. I want to first introduce someone who, like me, has greatly benefited from a clinical trial and believes that they are alive today because of their participation. And so joining us from Atlanta is Mel Mann along with his wife and care partner Cecelia Mann. Mel, welcome to the Patient Empowerment Network program.

Mel Mann:
Thank you very much.

Andrew Schorr:
And we’re going to hear more of Mel’s story in just a minute. I want to introduce a very prominent medical expert who joins us. He is the senior vice president and chief medical officer at really the largest cancer organization, the American Society of Clinical Oncology, and that is Dr. Richard Schilsky. Dr. Schilsky, welcome to our program.

Dr. Schilsky:
Thank you, Andrew. Happy to join you.

Andrew Schorr:
Okay. And are you in the Washington, DC, Virginia area?

Dr. Schilsky:
That’s where our organization is based, in Alexandria, Virginia, yes.

Andrew Schorr:
Thank you for being with us. I should mention that Dr. Schilsky has had a lot of experience related to trials. He was the head of hematology/oncology at the University of Chicago, which of course Chicago is a very diverse city, and the University of Chicago does a lot of research. And he also helped run a big research group that doctors from around the world are part of, and he did that for many years. So we’re going hear more from Dr. Schilsky in just a minute. But, first, Mel. So Mel, in the late ’90s you were dying, right, of chronic myeloid leukemia, correct?

Mel Mann:
Yes, I was.

Andrew Schorr:
Losing weight and being told that there wasn’t much to do, right?

Mel Mann:
Correct, yes.

Andrew Schorr:
Maybe a transplant. But you were in Atlanta and you went from doctor to doctor, but somehow you got to MD Anderson, a major research center in Houston. What did they offer you there?

Mel Mann:
When I first went out to MD Anderson they said they were going to put me on a clinical trial after clinical trial. And the first thing they did was increase my dose of interferon, and that was the medication, the standard therapy at the time, and then they tried different combinations of drugs, and eventually I started on different clinical trials.

Andrew Schorr:
Okay. And, Cecelia, you were there in Atlanta and he was scooting over to Houston, it’s not exactly around the corner. Why were you supportive of that?

Cecelia Mann:
I was supportive of that because that was the last chance that he had to a cure and for survival. So from the very beginning, whatever type of treatment he needed when he was flying around, whether he was going looking for bone marrow transplants, doing bone marrow drives, and therefore I was supportive of. We had a five‑year‑old daughter at the time, and so anything that Mel needed I was there to support him.

Andrew Schorr:
So, Mel, this is a happy story because here we are in 2018 as we do this program and you are with us when many people with CML at the time were not with us that long. Hopefully, a transplant could be curative, but a lot of people passed away. You were lucky enough to come back as they were rolling through different trials and there was a new one that opened up for a drug called Gleevec, a pill.

Mel Mann:
Yes.

Andrew Schorr:
What happened?

Mel Mann:
Okay. So in the summer of ’98 the Phase 1 Gleevec study opened up, and I went out to MD Anderson, and I was patient number two, and I started taking it at a low dose, and it was effective for me. And eventually they increased the dose and it started changing my leukemia around to eventually I reached what they call a major molecular response. And that was 20 years ago. This summer I went over 20 years.

Andrew Schorr:
Wow. Well, Dr. Schilsky, is that an example of a patient getting, if you will, tomorrow’s medicine today, what we hope for?

Dr. Schilsky:
Absolutely. And, first of all, it’s such a wonderful story, Mel. It’s great to hear you tell it, and it’s exactly why we do research, exactly why we do clinical trials, to try to discover the new therapies that people need that will give them the kind of long‑term survival and quality of life that you’ve been experiencing. It’s just‑‑it’s wonderful.

Andrew Schorr:
So, Dr. Schilsky, let’s get into the problem. So, generally, there are many clinical trials that take longer than one would hope to fill, and the FDA and I know scientists have been seeing well, gee, how do we know what we’re testing applies to people maybe with different ethnic, gender backgrounds, a variety of situations, and often we can’t find people who fit those categories to be in. What is that‑‑how poorly have we been doing in the past with diversity in trials, and what does that mean for developing new medicines?

Dr. Schilsky:
Well, we don’t do well in almost any dimension. We don’t get enough underrepresented minorities in clinical trials. We don’t get enough older people in clinical trials. You have to remember that 60 percent of cancers occur in people 65 years and older, and yet only about 10 percent of people participating in clinical trials are 65 and older. So we are having to treat the majority of older people, and I would say the majority of minority people, with data derived from participants in clinical trials who are not like them.

We need to change that for a whole host of reasons. It’s historically been very challenging, and the problems really sort of boil down into three big areas that I think we can discuss a little bit further.

First is awareness. Many people are not aware that clinical trials are even an option for them. Many people think that a clinical trial is a last resort, and I want to dispel that myth right out of the box. Clinical trials can be a very good option for patients right from the time of their cancer diagnosis even if it’s their very first treatment. So clinical trials may be a last resort, but they don’t have to be, and there are many clinical trials that are appropriate for people right following the initial diagnosis of their cancer.

So there’s the awareness issue, and sometimes, frankly, not even the doctors are aware of what clinical trial options are for their patients. And the one thing we know for sure is that the most influential person as to whether or not a patient goes into a clinical trial is their doctor. If the doctor does not recommend it, if the doctor is not aware of it, it’s not going to happen.

But then you get into the more technical issues. There are things, there are rules for clinical trials because they are research studies. They are experiments. There are very well defined rules, most of which are in place to protect the people who are participating in the study. Some of these rules are called eligibility criteria, and they specify the characteristics of people who can enroll in the study. Well, historically, they tend to be very rigid and very limiting, and you’ll often hear people talk about how the only people who can get into clinical trials are Olympic athletes. That may be the case, but it’s not Olympic athletes that we’re treating in the clinic every day, so we need to make our clinical trials more representative so that they’re more applicable to the typical person that a doctor sees in their office.

And then there are the logistical or operational issues of the clinical trial. The clinical trial can be very burdensome. Mel just described how he had to travel from his home in Atlanta to Houston to participate in a clinical trial. Not everybody can afford to do that. Not everybody can take time away from work, time away from home. And the clinical trial requires not only that you travel sometimes but that you travel on a specific schedule because of the requirements of the trial.

So all of these are issues that are‑‑can limit participation in trials, and many of them are magnified in minority populations or in populations that don’t have the economic resources to be able to meet the requirements of the trial.

Andrew Schorr:
Right. Let’s talk about that for a minute. So, Cecelia, you go out in the community and speak to people, and you probably meet some people who maybe are diagnosed with a blood cancer, like you’re active with the Leukemia and Lymphoma Society so you may speak to them, and they say even if you brought up about a trial, they say, hey, Cecelia, I’m working two jobs. Or my husband or spouse is working two jobs, and we’ve got two kids, three kids. How could we ever participate? We just can’t get away or we don’t have the family support or whatever. Those are real issues, right?

Cecelia Mann:
True. True, those are real issues, and I try to direct them towards resources that Mel and I found out about along the way. The Leukemia and Lymphoma Society, they have resources to help with travel, and American Cancer Society has resources that help with the hotel and lodging. And there are a number of other different types of funds that can be assessed to make that a possibility.

But you’re right, Andrew. I had one young lady at a health fair and a second cancer had popped up, and she was coming there to get information, and she was saying that they were saying it wasn’t too much they could do, and I brought up MD Anderson. And she immediately said, I can’t go out there, I have to go to work, and she turned around rough. And so when they listen to me then always glad to tell them about it and let them know there is an option and that clinical trials work, and I point to Mel, my husband, as a success story also.

Andrew Schorr:
So, Dr. Schilsky, you talked about physician awareness. It’s also about more physicians participating in trials at I think what you call the community level. So, in other words, MD Anderson and where you work, at the University of Chicago, those are big city centers and where they are in Atlanta there’s Emory and some other mainly centers, but what about out in the hinterland, if you will? Can somebody who lives there diagnosed with a cancer, how do they have access to a trial that their doctor knows about and maybe that’s more close to home, if you will?

Dr. Schilsky:
Right. So of course we know that anybody with cancer prefers to be treated in their community, and most are. So one of the goals is to be sure that oncologists practicing in all sorts of community settings have access to clinical trials. Now, one of the ways that happens is that for more than 50 years now the National Cancer Institute has actually been operating and funding a community‑based clinical trials network. It used to be called the CCOP program. That’s an acronym that we don’t have to go into. They’ve recently changed the name. It’s now called NCORP program, but‑‑that stands for, I think, the National Community Oncology Research Program.

But the point is that the program, which is in most but not all of the states in the United States, funds community oncologists to participate in NCI‑sponsored clinical trials, and there are at least 65 or 70 such clinical facilities around the country right now. So in those medical practices patients can find those clinical trials in their communities without having to travel.

There are also other community‑based networks that are active participants in cancer clinical trials. So I think at the end of the day the critical thing for patients, and this is sometimes easily forgotten because you’re so, you know, your thinking and your time and your emotion are all taken up in dealing with the cancer diagnosis. It’s really important, though, to ask the doctor, do they have access to clinical trials. Do they have a clinical trial that might be appropriate for you? And if not you might want to consider where else you could go, hopefully still relatively nearby to get access to a clinical trial.

Andrew Schorr:
Mel, so for you, you went around to some doctors who were not aware of anything new to do for you, right? And that’s still true in so many areas of cancer. Now, what do you say to patients about speaking up because Dr. Schilsky just referred to it, people are terrified. They really just want the doctor to have the answer. What do you tell people so that they maybe advocate for themselves?

Mel Mann:
Okay. So if you’re looking for a clinical trial and I’m out at, like you say, a health fair, we have a‑‑the Leukemia and Lymphoma Society has something called the clinical trial support center, and they have nurses who work early in the morning till late at night. And you call them up and you tell them about your illness, and they check the availability for what clinical trials are out there, and then they narrow it down to what you actually qualify for, and then they take into consideration your finances and other issues.

And then you’re left with a number of possible clinical trials that you have, and you can take that back to the doctor and you can discuss that with him. So that’s one of the things I talk with them about.

Andrew Schorr:
I could mention, now this is really more broadly across cancer, too. So there are breast cancer groups, there are lung cancer groups, and I would just make a pitch to find out, is there a local chapter or national number for you, for the cancer you or a loved one been diagnosed with, and that’s the question.

Mel Mann:
Yes.

Andrew Schorr:
Say, look, A, I don’t want to feel I’m alone, and, B, how do I get‑‑how do I get connected with what could be lifesaving or life‑extending treatments for me and that I could discuss with my doctor. And understanding‑‑and then, boy, if there are obstacles like financial issues, logistical issues, travel issue, is there support for that.

Mel Mann:
Yes.

Andrew Schorr:
So let’s go back to the inclusion/exclusion or eligibility issue you spoke about, Dr. Schilsky, because, you know, somebody who has cancer may also have heart problems or diabetes or some other issue. Maybe they previously had another cancer, and so for the companies developing new drugs they may be happy with narrow inclusion criteria because they don’t want to have anything get in the way, some previous thing you’ve had, to affect their ability to have a new drug go on the market.

So what kind of work is going on between government and the drug manufacturers so that the criteria, not so tight, but you can still get legitimate scientific answers?

Dr. Schilsky:
Right. So, as you alluded to, Andrew, there are good reasons that there are eligibility criteria. One of them is to protect the patients in the study from circumstances that would increase their risk of participating in the study. Another is because the companies or whoever is sponsoring the study wants to be able to isolate the specific effect of what they’re studying without having a lot of confounding factors that could muddy the water and makes it difficult to interpret the results. But that said, the bad thing about all that is that the results of the study might not be applicable to the majority of people who could benefit from the treatment because they weren’t included in the study to begin with.

So one of the things that my organizations has been working on very hard over the last couple of years now, and we’ve been doing this collaboratively with people from the Food and Drug Administration and the National Cancer Institute and a lot of clinical experts and a patient advocacy group, Friends of Cancer Research, is to try to expand or broaden or simplify some of these eligibility criteria that tend to keep people off of trials and in particular tend to keep minority populations off of clinical trials.

So, for example, it’s not unusual for someone who has a new diagnosis of cancer to have previously had some kind of cancer earlier in their lives. So we might see a patient who has lung cancer who 15 years ago had a diagnosis of prostate cancer. Well, for that lung cancer patient to go on a trial that has the typical inclusion and exclusion criteria that doesn’t allow for this previous malignancy, they would be excluded even if they had been cured of that prostate cancer 15 years ago.

We also see, and you mention what we call in the medical profession comorbidity. So if someone’s got cancer and they also have heart disease, they also have diabetes, high blood pressure, anything that affects the functioning of your normal organs, can also exclude people from participating in trials, and there are certain limits that we feel can be expanded and still allow the treatment to be given safely.

So just about a year ago now we came out with a set of recommendations for how eligibility criteria can be modified to make clinical trials more inclusive. And now just recently, I’m really pleased to say, the National Cancer Institute expanded their sort of template protocol document that many investigators follow to incorporate our recommendations, so now their standard protocol includes these broader inclusion and exclusion criteria. And the FDA now is working on what they call guidance documents to advise commercial companies that are running clinical trials to do just the same thing. So we are very optimistic now that we’ve got this ball rolling. We’re going to be removing these obstacles, and we’re going to be able to have more inclusive and diverse population of patients who participate in cancer clinical trials.

Andrew Schorr:
Great leadership. I hope it works great, and we’ll be happy to support you. So, Mel and Cecelia, let’s talk about the money part of it a little bit. So you were making trips to Houston, Mel, Cecelia was home with a five‑year‑old, and so admittedly there may be hardships, financial hardships, being away from family if you have to go to a trial somewhere else, checking back. What do you say to people when they say, well, I’m just going to go with the traditional stuff. It’s close to home. In other words, if there can be programs that can help them it still takes courage, if you will. So what would you say to people about investing in their life, if you will?

Mel Mann:
Well, you know what the standard, what the current treatment is and the outcome of that, so if you want to have a different outcome then you have to try something new which is probably going to be a new drug. So you have to weigh that with the cost and the travel. Some people may not have the support, the caregiver support to go a long distance, so you have to take that into consideration. As far as the Leukemia and Lymphoma Society they do have certain funds where they can help with travel pay, co‑pay, insurance premiums, that could help alleviate some of it. So there’s a lot of stuff that’s involved, and it’s an individual decision.

Andrew Schorr:
So we’ll just make a comment, though. So, many people have a church or synagogue, friends, neighbors even if they’re living alone, but yet people are sometimes hesitant to ask for help. And I would say speak up. People do want to help you.

Dr. Schilsky, let’s talk about another reality of trials. There’s a history certainly and some fear still in the black community of whether they were tested on, without their knowledge even, going back years and the general thought, you’ve heard it through your career, I’m sure, people say, well, I don’t want to be a guinea pig for a couple reasons. One is we don’t know if it’s going to work. And second of all if there are different arms of a trial I don’t know if I’m going‑‑I’m going to go to all this trouble and expense, I don’t know if I’m going to get the good stuff. So maybe you could speak about that a little bit. First of all, the fears of being experimented on, and then also about whether you will get what could be a breakthrough.

Dr. Schilsky:
Yeah. Well, for sure, you know, there is this sort of sordid history of inappropriate experimentation on people, and clinical trials are a form of experimentation. They are a form of research. There’s no doubt about that. But clinical trials these days are highly regulated, overseen by independent groups that include patients and clinical experts that come together in committees called IRBs, Institutional Review Boards, and they evaluate on both the risks and the benefits to patients who participate in clinical trials.

They make sure that the trial has an appropriate consent process associated with it, that it’s explained in plain language to patients, so I think these days a lot of those concerns no longer exist. And I hope that people can get beyond the history that led to some of those concerns. The‑‑sorry, I lost a train of thought on the rest of your question.

Andrew Schorr:
The issue about are you going to get the good stuff.

Dr. Schilsky:
Oh, yes. So a couple points there. One point I want to make clearly is that in most cases cancer clinical trials do not include a placebo or an inactive treatment. That’s not always the case, but it’s true most of the time. So patients are always going to get at least the standard of care treatment, and of course the standard of care is what is at that time known to be the best available treatment.

The whole point of doing the research is to determine if the new thing is better, and of course we always hope it will be. It’s not always better, but sometimes it is, as in Mel’s experience. And I think this has to be clearly laid out to patients. They have to clearly understand why the research is being done. In many trials nowadays even if the patient is assigned to get the standard of care treatment there still may be an option to get the new treatment at a later point. So if the standard of care doesn’t work many times there’s still the opportunity to get the new treatment following the standard of care treatment.

So the trial really boils down to not standard versus new but new versus standard followed by new. So eventually everybody may have a chance to get the new treatment. That’s not always the case, but I think the key‑‑my key take‑home, in a sense, is that we’re doing the research because we think and we hope the new treatment is better, but we have to do the research to prove that. And everybody in a clinical trial I think can be assured that they’re going to get, at the very least, the best available standard treatment.

Andrew Schorr:
Mel, when you signed the papers to be in a trial, and you probably shared them with Cecelia, especially back in the late ’90s and I participated in one trial in 2000 and another in 2011, there’s a lot of paperwork, things in bold face written by lawyers. I didn’t always understand it. What propelled you beyond that? Was it just that, oh, my god, if I don’t get something I’m going to die? Or how did you two deal with the paperwork and feel comfortable signing on the dotted line?

Mel Mann:
Well, I saw a lot of hope in the paperwork. For example, one trial I was on was peginterferon, and I had been taking interferon every day, injecting myself, and I had to keep it refrigerated and when I travelled it made it difficult. So with peg I can take one shot a week, so that would make the cancer journey easier. It may not make me live longer, but it will improve my quality of life, so I saw my quality of life improving with that clinical trial. And I looked at the paperwork, and I went through it, and I felt comfortable with it.

Andrew Schorr:
And how about you, Cecelia? I mean, your husband says, well, I’m going to be in a trial and I’ve got to sign all these papers. Did you say at any point, wait a minute, that’s scary?

Cecelia Mann:
Well, no, I didn’t. I didn’t because with Mel, he had three years to find a marrow match, and he was at the end of year two and no match in sight. And so when he had the opportunity to go out to MD Anderson and be on a clinical trial or several, I was okay with that. I was okay with that. And I looked at it as actually being a blessing. And it turned out to be, and we’re grateful.

But I would say to anyone else who is contemplating a trial and that person and their caregiver, their spouse, to just educate yourself, and get as much information as you can, ask as many questions as you can, but please don’t just throw it away out of hand. It’s definitely worth considering.

Andrew Schorr:
Dr. Schilsky, so we have more than 50 million people with a Hispanic background in the United States, and even if many people are speaking English they may speak Spanish at home. And then when you are diagnosed with a cancer there’s a whole new language of stuff that comes into play that even if you’re fluent in English it may not be either what you easily understand or even aligns‑‑what’s being asked of you aligns with your cultural background. Okay? So how, beyond, let’s say, the African‑American community, when you look at the Hispanic community, how do we encourage participation there and get over some of these cultural or language nuances, if you will?

Dr. Schilsky:
Yeah. So it’s much the same thing in the sense that the same information has to be conveyed but it may have different meaning and different interpretations in different ethnic and cultural groups. Most clinical trials now will have a consent form that is fully translated into Spanish. But, of course, there are many different languages on the globe. When I was practicing at the University of Chicago for many years on the south side of Chicago, we had Polish‑speaking people, we had Russian‑speaking people, we had people‑‑Chinese‑speaking people.

So the requirements actually are that there must be a consent form, at least some reversion of which is translated into the first language of the patient. So if you’re a native Spanish speaker, a native Chinese speaker, you have to have, be able to see a consent form written in that language, and generally speaking you have to have your native language interpreter present in the room to help you go through the consent form and respond to your questions. And that person has to be someone who is independent from the research team so they can give you the straight answer and not be influenced by any member of the research team. So I think all of that certainly helps.

But, again, what helps a lot more is to have members of the care team who look like the patient. So we have problems with diversity in our profession as well. We have very few African‑American oncologists. We have more Spanish‑speaking oncologists, but again we have few Asian oncologists. So we need to do a better job of improving the diversity of our profession, improving the diversity of the care teams. We need nursing staff and research staff and other people who work with our patients who represent them and gain their trust, who look like them, who talk their language. And I think that will go a long way toward making people feel more comfortable about participating in clinical trials.

Andrew Schorr:
I was at a conference last week and I heard some of the patient experience, people from different drug companies talking about how they were trying to simplify their forms because I know in 2000 when I entered a Phase 2 trial there were all kinds of black boxes, you could die, you could this, everything in the kitchen sink was in it. I’m still here, and I think because of the trial, and most of the side effects I didn’t have or they were definitely handled extremely well.

So right now, where are we, Dr. Schilsky, with participation? And why is it important? In other words, in this age of personalized medicine why do we need more black people in certain trials? Like, I know in multiple myeloma, one of the areas we cover, there’s a higher incidence in the black population, right, but yet few black people are in the trials for myeloma drugs. Or maybe there are differences with Asian populations or other populations. So is it that you can’t really get a clear scientific answer on the differences? Is that it?

Dr. Schilsky:
That’s part of it. First of all, we want anybody who could potentially benefit from being in a trial to be able to be in the trial for their own personal benefit. Secondly, we need to learn about the performance of the drug or the intervention in all the diverse populations in which it might be used. And one of the things we have learned is that not all populations respond the same way. Some treatments are more toxic in certain racial or ethnic groups. Some are more effective in some racial or ethnic groups.

And, you know, since you brought up this whole new world of precision medicine, I’ll give you the example of the lung cancer drugs that are used to treat the specific mutations in a gene called EGFR. So that’s a gene which has mutated in about 15 percent of Caucasian patients with lung cancer, but it’s mutated much more commonly in Asian patients. And in fact one of the clues that there was even a gene mutation that was important in determining whether these drugs worked or not was because it was observed that the drugs worked better in the Asian patients in the clinical trials even before the genetic abnormality had been discovered. And the clue was what’s different about the Asian patients than the other patients in the trial.

So the diversity is critical to our learning and critical to our application of the therapy in all the diverse populations that we serve.

Andrew Schorr:
If you’re in our viewing community and you have a question, send your questions into questions@patientpower.info, questions@patientpower.info. We’ll continue our discussion of course, but we invite you to join in.

So, Mel, when you get to talk to people, what do you say? Somebody is sick, diagnosed with a cancer, what do you say? Dr. Schilsky was talking about not seeing clinical trials as a last resort, and you weren’t seeing it that way (?) Inaudible, but today what would you say to people when you talk to them about it?

Mel Mann:
Well, I will say explore your possibilities because there are all different opportunities at each phase. You may not go into Phase 1 but you could do a Phase 2, 3, 4‑‑or Phase 3, and you don’t know what’s going to happen in each of those phases. So you just have to see what’s out there. And I’m exhibit A, so they look at me and they say, well, I can work, and then not as suspicious, you know. We have Tuskegee, and that was 1972, and it was that dark period of cancer history so that kind of rolls around in their mind, but you can’t let that jeopardize the opportunity such as Gleevec that I took advantage of. So we know that Gleevec worked, and there are other drugs that have improved the quality of life and the lifespan of cancer patients. So definitely research those drugs.

Andrew Schorr:
Did you lose heart when you were first in one trial and the medicine wasn’t working for very long? Some would say, well, all right, I tried a trial, forget about it, you know. But you then pursued other trials. What propelled you to do that?

Mel Mann:
Well, I was still in the game, so I saw that these trials took‑‑well, first of all, I could not find a bone marrow donor, but a bone marrow transplant was pretty drastic in itself so I was looking at these other opportunities as maybe not even having to take part in‑‑have a bone marrow transplant. So that was another incentive. So‑‑and I knew that if I didn’t find one‑‑there was a very small chance, there was only about 5 percent of Americas who are on the marrow registry, so basically I was helping to build a list, maybe not for myself but for people in the future who needed a transplant.

Andrew Schorr:
Dr. Schilsky, let’s talk about the pace of research. So, first of all, if we don’t get enough participation in trials how does that slow drug development?

Dr. Schilsky:
Well, it slows it down enormously because we have to have a certain number of people in each trial to be able to get a reliable answer. And these days it’s becoming even more challenging because as we’re developing drugs that only target a specific genetic abnormality in the tumor which sometimes is very rare so we may be looking for a genetic abnormality that only occurs in 2 or 3 percent of all people with a certain kind of cancer. First you have to find the people who have that genetic abnormalities, then you have to be able to enroll them in a clinical trial. They have to be willing. They have to meet the enrollment criteria. So it can take a long time, and even a global effort to find enough people to fill out a clinical trial.

And most clinical trials in order to produce a reliable result are going to require a minimum of 50 to 100 patients. Some require many hundreds of patients or even many thousands of patients depending on the question being asked. So you can see if people are not participating it’s going to take long time to get those answers.

Andrew Schorr:
Now, Mel, you got Gleevec in a trial at least three years before it was approved, and it was approved fast because it was such a breakthrough, right? So you literally got tomorrow’s medicine today, and it saved your life in the process, right?

Mel Mann:
Yes, because I was past the three years. I was about three years and eight months in my diagnosis, so you add another three years onto that and I would not have been here.

Andrew Schorr:
Right. In my case I was in a trial related to chronic lymphocytic leukemia, three‑drug combination, and I received that in a Phase 2 trial 10 years before that was approved. So it was a long time.

So I have a question for you about personalized medicine, Dr. Schilsky. So for instance in chronic lymphocytic leukemia I’m not‑‑I’m Caucasian but I’m Ashkenazic Jewish, okay? So where we’re going with personalized medicine, are we beginning to find subsets among Caucasians, among African‑Americans, among Asians, where there are even more narrow slices to help us understand targeting of medicines and what’s effective for whom? Is that where we’re headed?

Dr. Schilsky:
Absolutely. And, as I said earlier, we’re seeing that all the time. So almost every common cancer now is being broken down into a whole basketful of rare cancers under the broad umbrella of whatever the cancer type. So lung cancer, there’s probably six or eight different kinds of lung cancer now that each have a specific genetic abnormality, that each requires a specific treatment. And many of those treatments now are FDA approved, but the first thing you have to know is does the cancer have the genetic abnormality and then what is the appropriate treatment to use. We’re seeing that in breast cancer, in melanoma, in many other kinds of cancer types.

There’s another‑‑there’s a related issue here, though, of course, which is that not everybody metabolizes drugs the same way, and so another reason to have diverse populations in a clinical trial is to learn about side effect profile of the drug, learn about the right dose of the drug to use. And we know full well, for example, that African‑Americans metabolize some drugs differently from white people, and so, depending upon how the drug is working in the body, an African‑American person might require a higher dose or a lower dose of the same drug that a white person would require in order to get the same therapeutic effect.

So it just speaks to the point again where if you don’t have diverse populations in the trials you can’t learn this stuff so that doctors then have the information they need to be able to prescribe the drug in the best way for their particular patient.

Andrew Schorr:
Okay. Here’s some questions we’ve been getting in. Kaitlin wrote in, Mel, she wants to know, do you still participate in follow‑up activities related to the trial you were in? So tell us about your participation and sort of follow‑up.

Mel Mann:
My follow‑up is I go out to MD Anderson twice a year, and it’s just a one‑day, one‑hour doctor visit where they take the blood work and they check and see if everything’s stable. And then when I’m back home, twice a year I have my blood work checked back at home, and that’s the extent of the follow‑up. I still have to take medication, one pill a day.

Andrew Schorr:
Right. And is that still covered by the trial?

Mel Mann:
Well, it’s covered by the trial, but my insurance also covers it. I did Gleevec for life because of the trial.

Andrew Schorr:
Okay. Dr. Schilsky, let me just ask you, is that a benefit typically of trials? Like with these oral cancer medicines which you know can be so expensive, if you’re in a trial for one do you get it for life or an extended time or how does that work?

Dr. Schilsky:
Depends a little bit on the trial and the sponsor for the trial, but the one thing for sure is when you’re on a trial whatever the investigational drug is, whatever is being researched, that’s provided for free. And any testing that would be considered to be for research purposes is provided by free‑‑for free. So that’s a benefit of participating in the trial.

Typically the drugs continue to be provided for free for as long as the patient continues to benefit. Now, sometimes if the drug ultimately gets FDA approved then it may be necessary at some time in the future for a patient to switch over from the research drug to the commercial drug, but of course at that time the drug is FDA approved and if the person has insurance it will generally be covered by their insurance.

Andrew Schorr:
Okay. We got a question in though for you, Dr. Schilsky, from Darrell. We were talking about genomic testing to understand what version of a disease we have either because of our ethnic background or some other thing that’s going on with us. As you know, insurance companies for a while have been balking at some of these more sophisticated tests, yet we and our doctor need that for us to get what may be right on target for us. So maybe you could talk about work that ASCO’s doing at all related to that. We want the testing done, but we also want to get it paid for so we can get that right, precise care.

Dr. Schilsky:
Yeah. It’s a complicated question because the testing is done at different points in the person’s illness. And so typically a test on a tumor specimen that’s necessary to determine a standard of care treatment, and many of these tests are referred to as companion diagnostic tests. Those tests typically are covered by insurance because the treatments themselves are also covered by insurance and the only way to know if you can get the treatment is to have the test done.

Now, where it gets a little bit uncertain is when you get into this sort of large‑scale genomic testing where a patient’s tumor might be tested for many hundreds of genes not really knowing what you’re looking for and not really knowing what you’re going to do when you find it. And that is where you’re beginning to bump up a little bit against, research and that’s where the insurance companies, sometimes some are reluctant to pay for that kind of testing.

Now, at least one of those large genomic profiling tests earlier this year was approved by both the FDA and Medicare and now will be reimbursed. So that’s the good news, and I think that’s the direction that most insurance companies are heading in.

One of the things that my organization is doing to try to understand how best to use these tests and how best to use targeted cancer drugs is we’re doing our own clinical trial that’s available in 20 states around the country, so not the entire country yet, but has already enrolled more than 1200 people on the study over the last two and a half years. And we’re doing this study to understand how this kind of genomic testing is done, what kind of treatment is recommended based on the results of the genomic test and whether or not that treatment actually works.

Andrew Schorr:
Cecelia, you mentioned earlier about the lady walked into the health fair and when you started talking to her about trials she said I got to go to work, and she walked out. And Dr. Schilsky was talking about eligibility requirements, but there are other issues where the study may be asking you to come back to some place or have multiple tests with some frequency so it’s not just leaving work one time. It may be leaving work 20 times. Have you had people voice that concern to you, that it’s just‑‑what’s being requested is just too much?

Cecelia Mann:
Yes. I think the lack of convenience for people who don’t have the funds or don’t search out the funds would definitely hinder them being on a clinical trial or being open to hear about the clinical trial. So, yeah, convenience and awareness. We try to spread awareness that, yes, after we talk about the disease, the myeloma and the symptoms then we go into the resources. And, you know, I make sure I tell them about calling the information line and talking to the masters level oncologist professional there and finding out about the latest trials, the latest treatments in addition to what they’re doing currently with their doctors or their family members or friend that is diagnosed with one of the blood cancers.

Andrew Schorr:
Dr. Schilsky, she’s getting at helping people sort out what trials are available. So medical science is a moving forward, and I’m sure you as an oncologist say, hallelujah, there are more trials than ever, but it’s often not only daunting to understand any one of them but to go through a bunch of them to understand what might be right for you. So how can ASCO help that? Is it just simply educating your doctor, or what can we do for families affected by cancer so they can get at what could be a match for them?

Dr. Schilsky:
Yeah, it’s a great question. So there are a lot of resources available, as Cecelia has mentioned. We can help patients understand and even begin to sort of, you know, wade through and winnow down the potential clinical trial options for them. One things that we’re working on and very interested in doing is sort of flipping the current paradigm by which trials are done. So right now, typically speaking, the patient has to travel to whatever site has the trial available. If they’re fortunate enough that that’s their own doctor’s office, that’s great, but, as in Mel’s case he had to travel to MD Anderson to get the trial.

The technology these days is at a point where we think we can flip that. Instead of making the patient go to the trial we’re going to work toward making the trial come to the patient. So if your doctor is aware that a trial exists somewhere in the world it should be possible for the doctor to basically just go to a website, find the research study, find the consent form, find the other documents that are necessary and present that to the patient. And if the patient qualifies just sign them up instead of making the patient travel hundreds of miles away to the one place that has the trial available.

Some trials have already gone down this road, and they’ve actually been recruiting very successfully, but it’s still not the usual way in which things are done, and we think we need to try to flip things around a little bit to make it easier for the trial to come to the patients. Let the trial travel. Let’s not make the patient travel.

Andrew Schorr:
That sounds great.

Mel Mann:
Can I add something to that, Andrew?

Andrew Schorr:
Sure.

Mel Mann:
Okay. As a veteran, I was part of the VA system, and I had to go out to MD Anderson, but this summer they started something called Navigate, the VA had started something called Navigate, which partners with the NCI. And it’s at 12 sites around the country, and it’s to bring the clinical trials to the VA. It’s right there. So if it’s an NCI clinical trial then the veteran can get on that clinical trial. And there’s a lot of African‑American veterans who can take advantage of that.

Andrew Schorr:
For sure. That’s terrific. So we’re going in the right direction. One other thing I think that needs to change is we talked about the scientists, whether they’re federal government scientists or drug company scientists, and they want to get answers to a whole bunch of scientific questions. So they may say, as you write the protocol I think it is, well, you have to get so many CT scans and you have to get so many blood tests and stuff like that. And it can become onerous, Dr. Schilsky.

What’s happening in trial design so that, A, we talked about eligibility, you can get into the trial, but the things you’re asking of me may have logistical hurdles as well that you’re kind the lightening up on it to get to the key scientific question without all these other bells and whistles that make it tough on me.

Dr. Schilsky:
Yes. I like to think of it as the need to know and the nice to know, right? There are certain things you need to know in the trial to be sure that the treatment is working, the patient is safe and not having any severe side effects and things of that sort. A lot of that stuff is the same stuff that doctors order every day on their patients as part of routine clinical care, and so much of what needs to be collected in clinical trials really aligns pretty well with standard of care.

Now, that said, because clinical trials are research and because there’s always new frontiers to explore, sometimes testing in a clinical trial extends beyond what the standard of care is. Sometimes patients are asked to give extra specimens of their blood, of their normal tissues, of their tumor tissues. Extra biopsies might be required, things of that sort. Patients need to understand why they’re being asked to do that, what those specimens are going to be used for, how is it going to advance research.

And, frankly, they’re very important to expanding the scope of the research. So, for example, oftentimes those specimens are used. If the treatment doesn’t work in a patient having those specimens can help the scientists understand why the treatment didn’t work, and that opens up a whole new horizon to explore to potentially make the treatment better in other patients.

Andrew Schorr:
Mel, do you recognize that by being in a trial and the work that you and Cecelia have been doing that you’ve probably helped thousands of patients by first being in a trial and then you and Cecelia talking about it?

Mel Mann:
Yeah. Yeah. I guess that’s kind of hard sometimes. You don’t see yourself in that role, but as I look back on it, yes.

Andrew Schorr:
Cecelia, you’ve probably talked to a lot of people. Have you seen a change where‑‑you’ve been doing this for a number of years where earlier on people said no, no, no. Are people more receptive? Do you see a change going on? Let’s say in the African‑American community, do you think people are a little more receptive?

Cecelia Mann:
Yes, I think so. I think they are more receptive, and this has a lot to do with education and awareness, and that’s what we are out there doing when we are out there in the community. And the more they hear about it and the more they read about it and the more they can see examples like Melvin, and we know one or two other people that we’ve met that were also on a clinical trial. One is in our church, and he had a type of leukemia, and we didn’t know why he was sick. But he is doing very well.

And so the more we can get those examples out there in the community of successful clinical trial patients, it really helps and goes a long way toward helping people of color relax and come aboard. And I just say, please, do your research, educate yourself and ask questions and please stay open and don’t dismiss clinical trials out of hand.

Dr. Schilsky:
And, Andrew, if I could just add to that. I just want to make the point that it’s people like Mel who are creating the future. Everything we know about how to treat cancer we learned from the people who participated in the clinical trials. We’ve been doing clinical trials in cancer for at least 70 years, and all of the standard of care treatments that we have today came from the participation of people in clinical trials. And that’s how we make progress. That’s how we’ll continue to make progress.

So it’s the clinical trial participants who, sure, they’re in it for themselves. We understand that. They’re looking for a new treatment, a better outcome, but they are the heroes of oncology because they are paving the way, trying the course and ultimately making a better future for every cancer patient who follows them.

Andrew Schorr:
Amen. Let me just recap a couple of things, and correct me if I get anything wrong, either of you. So, first of all, Dr. Schilsky, I know there are more trials now than ever before, and they’re now looking at these rare subtypes as well, and so if we participate we may get the benefit of tomorrow’s benefit today. Cecelia was talking about assistance programs, people to help you sort it out, that you are noticing how there are difference among us about the ways that drugs are effective or not, and that’s so important to learn.

If we partner with you, Dr. Schilsky, and the many thousands of oncologists and researchers that you represent, can we get to the goal line faster? In other words, are you hopeful that if we really consider trials and participate in trials and stay in trials and the different groups that we can get closer to cancer cures?

Dr. Schilsky:
Absolutely. I mean, we have more and better cancer treatments today than we’ve ever had before. We have all sorts of new and hopeful treatments on the horizon. We have to prove that they are safe and effective treatments to get them out there into routine clinical practice, and that’s where the clinical trials come in. So the more people who participate in trials the more quickly those trials can be completed and give us an answer, then the more quickly those drugs will make their way into standard clinical practice where everybody can benefit from them.

Andrew Schorr:
Okay. So a couple of to‑dos for our audience if you’re a patient or a loved one or however you hear this. Ask your doctor about whether trials apply to you or your loved one who is diagnosed with cancer even if it’s on day one. You don’t have to be at death’s door. You’ve been diagnosed or a diagnosis is suspected, what tests can we do, how do we know what we’re dealing with, and when we look at the treatment options is a trial a possibility if that makes sense, right?

Dr. Schilsky:
Absolutely.

Andrew Schorr:
Okay. Step two, are there resources to help me overcome any obstacles I may have to participation, first understanding the trial, understanding it in my first language, sorting out is it right for me, getting to where it is and then staying in the trial because many people, unfortunately, don’t stay in the trial and so how do be help the trial get to the goal line?

And then lastly, Dr. Schilsky, it sounds like you’re doing a lot at the community level to have more doctors have an easier time of the bureaucracy that we’ve had with trials before and the understanding of this flood of trials that’s happening, right?

Dr. Schilsky:
Absolutely. And, you know, to be perfectly honest, the clinical trial community has‑‑we ourselves have created some of the bureaucracy, some of the excess regulation, some of the barriers to participation. It’s up to us to strip those away and solve the problem and make clinical trials more broadly available. We are working very hard now to make that happen.

Andrew Schorr:
Okay. So whatever community you’re in. I’m in the Ashkenazic Jewish community. Mel and Cecelia are in the African‑American community. We have people watching who are in the Hispanic community, the Asian community. If you have benefited from a trial, talk it up, right? Cecelia, people should talk it up, correct?

Cecelia Mann:
Exactly, yes. Please, talk it up.

Mel Mann:
Yes.

Andrew Schorr:
Mel, thank you. I wish you really continued good health. How many years has it been since you were diagnosed?

Mel Mann:
Well, in about two months it will be 24 years.

Andrew Schorr:
Twenty‑four years, and for me it’s 22 years. And had there not been trials either that we were in or somebody else was in we wouldn’t be here.

Mel Mann:
Yes.

Andrew Schorr:
So, thank you. And also, Cecelia, thank you for being a community activist when it comes to trials and being supportive of Mel as he’s been in a trial because some other people would say, you can’t go there, you can’t do this, and you’ve been supportive every step of the way. Thank you for that.

Cecelia Mann:
Oh, you’re so welcome. It was a pleasure to do it.

Andrew Schorr:
Mel and Cecelia Mann from Atlanta. And Dr. Richard Schilsky, you’ve devoted your life to this, Dr. Schilsky, and I just want to say on behalf of the cancer patient community thank you and thank you for the leadership that ASCO is trying to do, both with changing research requirements, working with government, working with industry, and you thank you personally for your devotion to us. I really appreciate you being with us.

Dr. Schilsky:
It was my great pleasure. And, again, congratulations to Mel and Cecelia.

Andrew Schorr:
Okay. Thank you all. So this is what we do with our Clinical Trials Mythbusters program. Please tell others about it. The replay is available very shortly and all kinds of little highlights that we’ve done today. But what’s important is wherever you are is remember we can’t develop new medicines unless all of us work together to participate to get the scientific answers that apply to us, whatever our unique situation is, and then we can work with government to approve new medicines, get them on the market and so many people can benefit in the US and worldwide.

Thank you so much for being with us on this Patient Empowerment Network program. I’m gratified to be part of it. Thanks too to our financial supporters AbbVie, Astellas, Celgene and Novartis and their dedication to drug development and supporting and sponsoring clinical trials. I’m Andrew Schorr near San Diego. Remember, knowledge can be the best medicine of all.


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

How Can Lung Cancer Patients Stay Involved in Research to Innovate New Treatments?

Living Well With Lung Cancer

Downloadable Program Guide

Noted lung cancer experts, Dr. Lecia Sequist, Marisa Wittebort, a lung cancer advocate with a very rare mutation, ROS1, and lung cancer advocate, Janet Freeman Daily joined this program to provide an expert perspective on the impact of patient involvement in research and how both lung cancer patients and care partners can contribute to bringing new medicines to the market.


Transcript:

Andrew Schorr:
And greetings from Carlsbad, California, near San Diego. I’m Andrew Schorr from Patient Power. Welcome to this Patient Empowerment Network program. I’m so excited. It’s where we can learn how can lung cancer patients stay involved in research and innovate new treatments to benefit the lung cancer community.
Let’s meet our guests. First of all, we wanted to have Marisa Wittebort, who is a ROS1 lung cancer patient, but unfortunately Marisa is having a medical procedure and so she couldn’t be with us. But joining us from New York City is her sister, Jess, who’s been with her every step of the way. Jess, thank you so much for joining us. And, first of all, how is your sister doing?

Jessica Wittebort:
Yes, she’s doing good. Thanks so much, Andrew for having me join today. Marisa’s good. She has another pesky effusion that needs more attention today, so I’m joining you, but thank you very much.

Andrew Schorr:
Okay. Well, all our best to Marisa.

Jessica Wittebort:
Yeah, I appreciate that.

Andrew Schorr:
You know, the role of a care partner such as yourself, a sister, a spouse, and other family members is so critical. Okay.
Let’s also meet someone else who has been living with lung cancer personally and that is our old friend–she’s not old, though–Janet Freeman-Daily who joins us from Seattle. Janet also happens to have the ROS1 mutation like Marisa, and she is so active in going to medical conferences all around the world. Janet, thanks for being with us.

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.

Janet Freeman-Daily:
Thanks for inviting me, Andrew.

Andrew Schorr:
Okay. And, Janet, you–how many conferences have you spoken to that are medical conferences, but you’re a patient who gets up and says, here’s our perspective? How many?

Janet Freeman-Daily:
I think it’s five or six at this point.

Andrew Schorr:
I bet. And we’re going to get–we’re going to talk more about the importance of that. So you’re one side of the coin, as is Marisa, and then we have a leading cancer researcher joining us from Mass General in Boston devoted to people with lung cancer. That’s Lecia Sequist. Dr. Sequist, thanks so much for being with us.

Dr. Sequist:
Thank you for having me. This is really a treat.

Andrew Schorr:
Okay. So you’ve been at medical conferences where you’ve heard people like Janet speak. Does that inspire you when you are actually at what would otherwise be just thousands of cancer specialists, but the patient perspective is put right front and center?

Dr. Sequist:
It’s very inspiring, as I’m sure we’ll talk about. It was especially palpable this year at the World Lung Cancer Conference in Toronto just about five or six weeks ago. Janet was there. There were so many lung cancer advocates there, and this is a conference that’s focused only on lung cancer, and it was really exciting.
But I would say Janet and I have been running into each other at the hallways of medical conferences for many years, and it is always really interesting to get the patient perspective about a big result that was just presented maybe an hour earlier. And I love running into people at meetings and talking to them about it. It really helps inform our research.

Andrew Schorr:
That’s what I was going to ask–go ahead, Janet.

Janet Freeman-Daily:
It’s also very nice to run into a doctor after a presentation and say, what did they just say?

Andrew Schorr:
Right. Right. So do you, Dr. Sequist besides inspiring you, and then there are people in labs who don’t even–you see patients, but there are other people who are only in labs, do you feel that this communication with people who are living it can actually help get information, promote collaboration and accelerate us towards what we hope will be cures?

Dr. Sequist:
Oh, absolutely. It’s a really vital two-way communication road. I think having patient advocates learn more about the research process, both the pros and cons about went research process, and see what all is involved and what hurdles we have to deal with all the time as researchers can be really helpful. We need their help advocating to get rid of some hurdles and the obstacles in our way.
And there is nothing more informative than finding out what really is important to patients, especially when you’re developing a new treatment, hearing from them about what they value, what they–you know, someone who is not living with it may think that a certain side effect is a big deal, yet someone who is taking the medicine will say, you know, actually that’s–I can deal with that if it’s going to help me live longer. And finding out where that balance lies is really important and not something you can just guess if you’re not in the shoes of a patient.

Andrew Schorr:
So, Marisa, you’ve been every step of the way with your–rather, excuse me, Jessica.

Jessica Wittebort:
I’m channeling her, it’s fine. I’m channeling her.

Andrew Schorr:
All right. You’ve been with Marisa every step of the way, and unfortunately she was diagnosed in 2015 at what, age–

Jessica Wittebort:
She just turned 30, yeah.

Andrew Schorr:
She just turned 30. You’re her big sister. From the family perspective what do you hope, with closer collaboration with researchers, practitioners like Dr. Sequist, what do you hope?

Jessica Wittebort:
Well, gosh, I think we’re really just hoping to expedite research, and we want to be part of that journey. You know, I think when Marisa goes in to see her oncologist and he gives her a high five because she’s doing well, you know at a granular level that that relationship and that everybody is pushing for the same thing.
I think a little bit that gets lost in translation sometimes when you can get swallowed by the information that comes out of a conference if you’re not carefully, right, so learning how to translate that information into something tangible and consumable and being able to respond back to your healthcare professionals I think is just that bridge that’s essential to moving things forward.

Andrew Schorr:
And you’ve been to some conferences. I saw you at the Biden Cancer Summit, which had a lot of patients and patient advocates there, but I think you’ve been to–where did you go? To Austria or someplace?

Jessica Wittebort:
Yeah, I went to World Lung in Austria, to meet Janet, frankly. No, I mean, to see some incredible work in progress and some incredible work, and it’s a tremendous amount of content. I probably understood, you know, 5 percent of it, but at least it got me there starting to understand what the language was, starting to understand what the potential impact of clinical trials are, starting to feel just a tremendous amount of hope that lives through science, and to see my colleagues. You know, Janet is pretty much family, so I think these conferences, it’s incredible when patients not only part–you know, really participating, I think that’s a big deal.

Janet Freeman-Daily:
It was also really great for the–there were several ROS1ders there, people who had ROS1 cancer dealing with it at the end of conference, and we got to go up en masse and talk to the researchers about what they were doing, which was educational for us, and I think most of them felt fairly enthused about it too.

Andrew Schorr:
Janet, you’ve spoken at some of these congresses. What do you want to say to that clinical and research audience? What are you trying to bring forward to them as somebody living now, what, four or five years with stage IV lung cancer?

Janet Freeman-Daily:
I was diagnosed seven and a half years ago.

Andrew Schorr:
Seven and a half years ago. So, thanks god, treatment, and you’ve been in a trial for a long time, has just been remarkable for you, life-extending. What’s the message you bring when you speak?

Janet Freeman-Daily:
Well, it depends on the setting that I’m in and what I’ve been asked to speak about. It’s been different topics. Once I’ve talked about value in cancer care and the cost of cancer drugs. Once I’ve talked about the research that the patients with ROS1 were doing. I’ve also talked about the importance of goals of care discussions with the doctors to talk about what our treatment options are and what our chances are of them being effective so we could make our own choices about treatment rather than having the doctor decide what we’re going to do.
There’s a lot of different topics out there that patients can share their background and perspectives on. I think one of my more favorite things is running into Dr. (?) Jean Kooey who created the drug that I’m on and that Marisa started with and that Marisa then took next. She’s the lead chemist on those designs, and we ran into her at the poster session at ASCO and she got to meet the patients that her drug (?) Inaudible, which was a really big deal for her. And we’re all kind of awe struck, fan girl, oh, my god.

Andrew Schorr:
So, Dr. Sequist, does that make a difference? Because there are maybe many thousands of people working around the world on lung cancer now, some people only in labs, and never meet a patient like with a more rare mutation like ROS1. Does that make a difference when that connection can happen?

Dr. Sequist:
Oh, absolutely. I do think it’s really important for people who are working on the basic science aspects of cancer and in a laboratory, a little bit removed from the patients, to meet patients and survivors and see what their work is leading to. At Mass General we routinely have tours of our lab so that the people that work in the lab, not just the lead scientists but even the techs who are there for 10, 12 hours a day working hard for them to see how their work can really make a difference. And I know lots of other centers will do that as well.

Andrew Schorr:
So we’re getting into this age of personalized medicine, and I was in Boston a week or so ago and whether it’s out of MIT or your partners group in Boston, there’s all this computing power coming into play to try to understand what is our personal situation with a cancer and how do you develop or do you have medicines or trials that line up with that. And that’s been a real work of yours, right, is to try to look at the subsets of lung cancer. How are we doing in that? We talk about ROS1 and you have KRAS and ALK and EGFR and all these different types and then some types that haven’t been identified yet, right?

Dr. Sequist:
That’s right. I think if you take the long view and look at 10 or 15 years ago where the field of lung cancer was, it is a totally different landscape today. We have come so far in being able to personalize not only the clinical trials that are available for patients but then subsequently the approved treatments. And there’s been a lot of exciting advances in lung cancer that are a little bit less personalized lately, specifically immune therapy. That works with a bit of a broader brush, but the success in the personalized targeted therapy is unparalleled in other tumors types at the moment, and so I think everyone that works in lung cancer is really proud of how much the field has moved forward.

Andrew Schorr:
But you’re doing detective work, so some of these genes weren’t originally identified, and you have probably a lot more to go, so what’s going on now where for people where a gene wasn’t identified maybe you’ll have that? You’ll find out what the factors are or if somebody switches from one driver gene to another?

Dr. Sequist:
Yeah, there’s a lot of important things that go into that. One is being able to test each patient, and there are now several ways that you can test for the key mutations. The gold standard is still testing tumor biopsy, but liquid biopsies are also coming really into the forefront ready for prime time. Janet and I actually collaborated–well, Janet led the collaboration on an article that we wrote together about liquid biopsies and how it’s–and demystifying some of these things for patient audiences.
But looking at the tumor is important, and then actually important is getting patients to the right trials. You’re not going to be able to prove that something works if you can only find one patient with that mutation. You really have to reach all over the country and sometimes all over the world to find patients specifically for a situation. And that’s one area where patient advocacy groups have been extremely helpful helping bring patients together with the trials that fit their situation.

Andrew Schorr:
So tell me–go ahead. I was just going to–Janet, what’s the message then to people watching so that they can get the care or the testing or help involved to push research further? What do you want to say to people?

Janet Freeman-Daily:
Well, I think one of the valuable things that Lecia brought out is that we are developing or identifying new mutations all the time. When I was first diagnosed nobody knew about ROS1. It hadn’t even been published yet. And when I found out about it and I brought the article to my local doctors in the community setting they didn’t know how to test for it. And yet when I got tested and they found that I had ROS1 I have been on a drug now that I’m coming up to my six-year anniversary for my clinical trial, and I’m still no evidence of disease.
So what I would tell people is it’s really important to keep track of the research and to stay on top of the new developments. And so the patient communities are really good at that because you might find a new option that didn’t exist when you were first diagnosed.

Andrew Schorr:
And so that’s something that you, Jess, and your sister do all the time, right? And so you know you have this ROS1 version of lung cancer for your sister, you don’t know if something will change or other factors will come in, so you keep your ear to the ground very much and connect with the community.

Jessica Wittebort:
Absolutely. So tactically what do we do? We have our Google alerts always set to any medicines that we’ve heard about, any clinical trials that we’ve heard about, any researchers that are working in the space. For us, we have a ROS1 community online which is–we have a public one, and we also have a private one on Facebook where we’re able to just very openly bounce ideas around and talk about things we don’t understand and get those concepts in our heads.
And oftentimes those relationships lead to actually meeting off line. So most cities that Marisa or I visit for whatever reason, whether it’s going to see a doctor or going to an event, we get to meet somebody offline as well. So finding–keeping your ear to the ground, yes. We have great luxury of really–Marisa has a great team, so they will always drive that for her. But I think it’s also something that she is always very keen to share the information that she’s getting so that other people are privileged to have that information as well.

Andrew Schorr:
Go ahead.

Janet Freeman-Daily:
And a few key researchers like Dr. Sequist, Dr. Camidge, Dr. Shaw, at a few key universities are the experts in some of these driver oncogenes, and they’ve been very generous in their time in allowing us to e-mail them questions and say, gee, this question came up in the group, and we don’t have any experience with that. Could you give us an idea of what to do? So the researchers are key to this.

Andrew Schorr:
They are. And, Dr. Sequist, thank you for your devotion. I have a question for you, and that is most people though don’t get treatment at University of Colorado or Mass General or Dana-Farber or City of Hope or MD Anderson, and we could list a bunch of the leading institutions. Most people are told they have lung cancer, they’re at a community oncology practice, they’re terrified, and you’re leading change. You’re on the leading edge, all of you, in lung cancer, but that sometimes hasn’t quite–I don’t want to say trickle down, but you’re on the podium at World Lung or ASCO and you’re talking to a thousand doctors sitting there and we’re hoping that it gets to them, and a patient walks into their clinic, though and maybe some of this isn’t brought to bear.
What can the patient or the family member do so that this knowledge that’s emerging in lung cancer can be brought to bear at the community level? What’s the patient or the family member’s role today?

Dr. Sequist:
I think medicine is changing, and we are no longer in an era where any one doctor can know everything about medicine. I mean, we haven’t been in that era for a long time. And it’s very difficult to be a community oncology, a general oncologist today. There are so many new treatments and new genes and new strategies coming out for every type of cancer in rapid succession, so keeping up with all of lung cancer advancements plus all the other tumor types is quite a challenge.

That’s why I think that now more than ever as cancer gets so complicated it does work really well for patients to be able to connect with other patients and lung cancer specialists online, through activities like this, through many other educational activities that are available and advocacy groups because–just because a community oncologist has never heard of ROS1 I don’t think makes them a bad community oncologist, but hopefully the message is getting out to the community to partner with super sub-sub specialized academic centers if a mutation like this is found in a patient.
Andrew Schorr:
Okay. So, Janet, what do you tell people, what do you want to tell our viewers who were probably treated at least initially at a community center and they have no clue whether they have some subtype, rare or not, of be lung cancer and what to do about it? Janet, (?) Inaudible.

Janet Freeman-Daily:
If a person has lung cancer and it’s non-small cell lung cancer you should have gotten genomic testing at some point, and if you didn’t you need to ask your doctor about that. If your doctor is not familiar with it, and some of the general practitioners and community oncologists may not be as comfortable with it as other lung cancer specialists, then get a second opinion, preferably at a major academic cancer center.
If you want to learn more about this there are a large number of online patient groups where you can ask questions and get educated about this, or you can go to websites of some of the lung cancer advocacy organizations like LUNGevity, Lung Cancer Foundation of America. They have a good deal of information where you can start learning about things to get yourself educated on the topic. It’s–I still hear patients who are stage IV lung cancer, and their doctor sent them home on hospice without ever doing genomic testing. It’s really important that you make sure you get the tests that are in the standard of care.

Andrew Schorr:
So, Dr. Sequist, just back to you. This genomic testing is to see, is there an oncogene or cancer gene that’s driving your cancer that either an approved or maybe a clinical trial experimental medicine may target, right? Okay?

Dr. Sequist:
That’s correct. And, as Janet was saying, it’s vitally important for every patient that’s diagnosed to get tested at a minimum for the genes that correspond to FDA-approved medications, but there are several second-tier mutations that I believe everyone should be tested for because there are clinical trials that even if it’s not available at the community site where they first sought care hopefully it’s available someplace that’s not too terribly far from where they live.

Andrew Schorr:
Okay. So I’m sure that Janet follows this and Jess of course, can the genes change? So, in other words, in lung cancer if Mrs. Jones is seen to have a KRAS mutation, just to pull one out, early on, does that always remain what’s driving her lung cancer, or might it change and there might be a need to test again?

Dr. Sequist:
I think we’re all experts in this, so we can everybody chime in as well. If the cancer truly has a driver oncogene what that means is that every single cancer cell in the tumor carries that genetic mark. Probably the very first cancer cell that came up in the body had it, and then every daughter cell that was created afterwards carries this mark. As patients–so typically these are EGFR, ALK, ROS, MET, RET. These are the ones that we have targets for, BRAF, targeted drugs.
Now, once a patient is on a targeted drug you can think of it like evolution, like survival of the fittest. So a drug is exerting pressure on the cancer, many cells are dying, but sometimes a cell will have a certain characteristic that allows it to live through the drug treatment, and then from there a resistant tumor can grow. And so second mutations or second pathways can become activated after patients have been treated with certain drugs. And the more drugs that people have been exposed to over time the more different subpopulations that might have varying signatures come up.
But you never lose that original mutation. It’s something that is always carried forward. It’s just what else piles on top of it across the different arms. I describe it as different arms of the family or cousins. Like this tumor is a cousin of that tumor because they do have some different characteristics but still that same core characteristic.

Andrew Schorr:
And you were saying about retesting?

Janet Freeman-Daily:
So some drugs we know that if they stop working there’s another drug that you can go to, but as we develop more and more drugs and EGFR, with which Dr. Sequist is very familiar, has more drugs than the rest of us. When patients take certain of those drugs second or third line they actually might develop a different mutation and will have to get retested to find out how to treat that. We’re right on the forefront of learning about how the genomics of cancer works, and we learn new things all the time.

Andrew Schorr:
So, Jess, you and your sister have sought out eminent specialists at major centers, but, as you said, not everybody goes there. What advice do you have to patients and family members, especially family members because sometimes the patient is so terrified just being led through care and the family member has to pick up the mantle? What would you say so that the loved one gets the best care?

Jessica Wittebort:
For us the most profound change has been to find a specialist at an academic institution. I think if you don’t–if you’re not able to do that, it is really important to find your patient group and start asking, what are they doing. What information can you get your head around? And keep your head above water because I really do believe there’s so much hope and there’s so much energy right now and momentum in this space that it’s important to just keep finding, keep looking for the information. And if you’re not getting the answers that you need or are too complicated figure out a way to not feel shy about asking again.

Andrew Schorr:
Amen. So you mentioned earlier, Janet, about getting tested, right?

Janet Freeman-Daily:
Yes.

Andrew Schorr:
So what if the test doesn’t identify anybody? Should they be forlorn? I’m going to ask Dr. Sequist, too. If one of these genes that we rattled off doesn’t show up or driver gene should they say, oh, my god I’m out of luck?

Janet Freeman-Daily:
No, not necessarily. Targeted therapies are easy to take in that you can take a pill once or twice a day, but they’re not the only new therapy that’s come out, and most of the patients who do not have a targeted treatment can take immunotherapy. That’s the new standard of care, and it works really well. I’ll let Dr. Sequist talk to that.

Andrew Schorr:
Let’s understand that, Dr. Sequist. So if somebody doesn’t have any of those genes but both of you have mentioned immunotherapy, how does that work and how does that help?

Dr. Sequist:
So one quick point before we get to immune therapy is that it’s really important if you are told that you don’t have any specific mutations that you make sure that the correct panel was done. Sometimes there are small panels that may miss important genes simply because they’re not part of the panel. So the test may be negative for everything that was assayed, but it may not rule out some of these rare mutations. Like Janet was saying, her mutation wasn’t even known about at that time she had the first testing done so she had to have repeat testing. And this is a very common story. So that’s what I wanted to say about testing.
But immune therapy is–really been a game changer in cancer in general including lung cancer, but this is the idea of trying to get someone’s own immune system so attack the cancer. Our bodies are supposed to do this. Our immune system is supposed to be on surveillance for cancer cells, treat them as foreign and destroy them, but obviously if a tumor grows to a point where you’re getting a diagnosis of cancer something has gone wrong in that process. Usually it is that that tumor is camouflaging itself in some way from the immune surveillance, and some of the new treatments that have been approved over the last couple of years in multiple types of cancer essentially rip off that camouflage, allow the immune system to see that the cancer is there as a foreign invader and start to attack it. In lung cancer this works best on the, as Janet was mentioning, the type of cancers that don’t have a driver mutation, the types of cancers that are more often associated with a history of smoking or exposure to some other carcinogens, and immune therapy has really changed the survival and the treatment options for a large population of lung cancer patients.

Janet Freeman-Daily:
And I just want to reiterate that it’s very important that you get genomic testing before you start immunotherapy because the data we have now indicates that immunotherapy usually does not work for those of us who have driving mutations.

Dr. Sequist:
And it may increase the toxicity of some of the targeted drugs, so not only may it not work but it might harm your chances of having a nice, long response like Janet and Marisa are having.

Andrew Schorr:
Hmm. This is complicated stuff. We talked about how difficult it is for the community oncologist who sees sort of all comers to keep up with this. Let’s just review some of the things that have come up recently at medical meetings that you’ve been at.
So first of all, Janet, from your perspective as a patient, you go to the World Lung meeting, you go to some of the other meetings, what do you think are the big deals for patients? Is it more genes being identified? Is it having immunotherapy work for more people? What are the big take-home messages we should review for people here?

Janet Freeman-Daily:
Well, you touched on two of them. One, there are more genes identified. I’m not sure I’ve got quite the right percentage, but at the moment I believe it’s about 70 percent of patients with non-small cell lung cancer have a driving mutation for which there’s an approved drug or a clinical trial. Is that right, Dr. Sequist? About?

Dr. Sequist:
I don’t know the exact number, but it’s got to be close to there.

Janet Freeman-Daily:
And then there’s immunotherapy, which not only works for some people who didn’t have treatment choices but in some cases continues to work after they stop taking the drug for a good period of time.
But I think one of the other big notes is it appears that immunotherapy may be working for small-cell lung cancer, which has not had a new treatment option in decades, so that is huge.
However, in addition to treatments I would say the next big thing, and it’s not too surprising I’m going to say this because this is what I talked at World Lung, but the fact that we have new patient groups forming around these driving oncogenes, we have enough patients who have been taking these targeted therapies enough, long enough and feeling good enough that they’re becoming active as advocates.
And they want to learn more about their disease, so we now have a group for ROS1 called the ROS1ders, for EGFR, EGFR resisters, for ALK, called ALK Positive, or RET, called the RET Renegades, and a separate group for a subset called Exon 20 group for insertions or Exon 20 of HER2 and EGFR.
And these patients groups are providing guidance to help patients find clinical trials, to help them understand their treatment, to deal with their side effects, to find experts, and we’re also funding research. So there are new research studies being funded by these patients, and the ROS1ders have actually created a study where we are making cancer models of our own rare cancer because researchers didn’t have anything to study, and now they have more cells. In fact, we’ve got, I think, four new cell lines in the past year and more in development.
And we also have three patients who have donated to creating mouse models of ROS1, and they hopefully will be useful for us. And they’ve already had two different publications on the subject. And without it some of the ROS1 research couldn’t be done, so we’re very excited about that.

Andrew Schorr:
Wow, just congratulations to all of you who are involved in this, and I know you’ve got a big smile on your face, Dr. Sequist. We used to have such a very short turn for most people with advanced lung cancer, and now, thank god, with research you’ve done and your peers around the world and in collaboration with patients we have people living much longer, like Marisa, who unfortunately couldn’t be with us today, but Janet and some others who are probably watching.
So that then gives you the opportunity to try to understand them and a lot of aspects of their care and their biology more than you ever could because people are living, right? So that chance for dialogue is really critical to understand how are we not just, yay, we have the medicines helping people live longer but what’s going on, right?

Dr. Sequist:
Yeah. I think that’s right, and it gives us an opportunity to think more critically about how we can do things differently, whereas 10, 15 years ago we were just trying it to find a way to help people live beyond a year. That was the glass ceiling that we were trying to break. And now that we’ve come so far in lung cancer we can really start looking at some of these important questions about sequencing medications, combining medications. What does that do to quality of life? What are other things that affect patients being on clinical trials for years and years, having to go through the scans and the tests? Trying to make clinical trial more accessible to people because of eligibility criteria that are obsolete.
So these are some of the lessons I’ve learned from working with patients in various forums, and it’s really very satisfying for me for sure.

Andrew Schorr:
I know a lot of your work is in EGFR, and if I have it right maybe the incidence of, if that’s the right term, of EGFR, let’s say in the Asian community is higher. Is that right? And so I know the percentage of people in clinical trials is low, like 3 percent. We need more participation of people from different groups so that you can understand how these different mutations are active more or less in different groups, right, and how certain medicines come into play? That’s one of the collaborations we from all groups need to do with you, right?

Dr. Sequist:
Well, I think another–that’s absolutely right, and another really important role that patient advocates can play is to educate their peers about what clinical research involves. Many people in this country are just scared about clinical research. They don’t want to be considered as a lab rat, and they think that’s something maybe for at the very end of the line when you’ve exhausted all other options when in fact some of the most promising clinical trials these days are for the very first treatment that you may take as soon as you’re diagnosed. And having people be aware that clinical trials are not just a way to experiment on a patient but to really offer the patient cutting-edge treatment that they couldn’t get outside of a trial and work together to bring new treatments to approval, that message is critical to get out to the public.

Andrew Schorr:
Right. And can accelerate medicines getting to the goal line quicker, right? I mean, Janet, I know you–a lot of what, for the community living with lung cancer, like you don’t know how long your ROS1 medicine will work.

Janet Freeman-Daily:
That’s right. It won’t last forever. I will eventually have to try something else, and the drug that I take will probably be in a clinical trial. I think it’s important to know that especially for those of us with driving oncogenes but also for people with cancers that don’t have a good effective treatment option, clinical trials may be your best treatment option. Clinical trials provide hope. There’s no guarantee that they will work, but when you don’t have any other option that looks effective or that lasts a long time clinical trials can be very useful.

Andrew Schorr:
So, Jess, a lot of times a physician will say to a patient, well, I might have a clinical trial for you and the patient comes home to review a whole stack of (?) legalist documents to try and simple–and the family member says, oh, no. What would you say to family members too about this idea of clinical trials and supporting your loved one in maybe getting tomorrow’s medicine today?

Jessica Wittebort:
I think it’s really important again to find a group of people that are on a clinical trial so you can see how real it is, how okay it is, you know, sort beat down those major misunderstandings, you know. Fears that you’re going to be given a placebo and then you’re left to go or whatever the case is. I think we’re still getting in a place where (?) ct.gov or Cancer Commons are able to really very clearly articulate it. The research is there, the information is there, but I do find it still a bit daunting for people who probably are just freshly diagnosed to understand what it means, so I think–

Andrew Schorr:
Right. As Janet said, there are people who can help you with the lung cancer groups she’s rattled by, online groups. There are all sorts of people who can help you, so I want you to–I hope our viewers will take advantage of that.
So, Dr. Sequist, people–Jess just mentioned about people have this fear of getting a placebo. If you’re in a trial, people want to get the good stuff even though you’re not sure what the good stuff is or how good the good stuff could be, but are they taken care of no matter what?

Dr. Sequist:
Patients are absolutely taken care of no matter what. There are many different kinds of clinical trials. Some of them have one arm where everyone on the trial gets the same treatment. Some of them may have multiple arms, and there could be a randomization where a computer basically rolls the dice and tells you and your doctor which arm you’re going to be placed in and you don’t have a choice. But patients are informed about the design of the trial and the various treatments before they sign up. We’re still–scientifically, before something can become standard of care, we still need to compare it to the old standard of care. Luckily, in lung cancer there really aren’t too many spaces left where standard of care would be placebo, so most patients getting lung cancer clinical trials are treated with a standard chemotherapy or a standard targeted therapy or a standard immune therapy, and then the experimental arm might be a variation on that or something totally different.
But it’s really important, and if you do participate in a clinical trial the person who is talking to you about the participation and getting your consent will inform you of all those things. What are the options? What could you be treated with? What is the purpose of the trial? How will it help you as a participant? These are all really important things to understand before you jump in.

Andrew Schorr:
Here’s a question–oh, sorry. Please.

Jessica Wittebort:
I was just going to say that Marisa just signed a stack of papers in Boston this week for participating in the blood biopsy trial, and that’s maybe the fourth pile of paperwork I’ve seen her sign. And it was an incredible process of just her being able to ask any questions, the nurse practitioner sitting down with her answering, answering everything and anything and understanding what it meant. And, you know, it’s–I just think we probably need to figure out how to eliminate some of the fear and the mystery around that process.

Andrew Schorr:
We did a program the other day and the replay will be posted soon with Dr. Richard Schilsky who is the chief medical officer of ASCO, the big cancer organization, and they’re really working hard with industry and government to simplify the forms. And, for instance, for people where English is not their first language to make sure that things are explained to you in your language, whether you read or if there’s a translator there so that you fully understand.
Here’s a question we got in from Ed, Dr. Sequist. He says, I’ve been an active participant in a Phase 1 trial for nearly three years. What is the average length of time it takes for a clinical trial to get to FDA approval?

Dr. Sequist:
That can really vary. I don’t think there is a standard answer, but a lot of people ask me, okay, doc, I’m going on to this Phase 1 trial at what paint will I be graduated up to Phase 2 or Phase 3? And, you know, patients usually don’t switch from a Phase 1 trial to a Phase 2 or 3. The drug development may continue and–continue on its pathway towards FDA development, but patients usually stay in the same trial that they started on.
The record time in oncology for first patient dosed–interval between first patient dosed in a Phase 1 trial to FDA approval was probably for crizotinib, which is an ALK, ROS and MET inhibitor, where the time was, what, about three years, Janet?

Janet Freeman-Daily:
Inaudible.

Dr. Sequist:
But most drugs take a little longer than that. But when I was training the–what I was taught was that it usually takes 10 years for a drug to get from Phase 1 to approval. Thankfully, that is not the case anymore. Most drugs are getting there in three, four, five years.

Andrew Schorr:
Well, I think, as Dr. Schilsky said the other day, they’re really trying to work with the FDA, the NCI, industry to try to do it, but part of it–now, for instance, the government is looking for patient-reported outcomes. How do things affect the patient in their life? So again doesn’t that come into play, too, Janet, that we need to be–we need to be not just part of the trial but we need to be giving information to help with as decisions are made about whether a new drug is a big deal, right?

Janet Freeman-Daily:
Yeah. Patient-reported outcomes are just starting to be incorporated into clinical trials, and it will be great to have them more involved and for patients to be able to provide inputs that are important to them about how they feel on the drug and how it affects them so that we will have more information about side effects when a drug gets approved. But it’s still fairly early.
But I want to go back to one thing that Dr. Sequist said, that the FDA is trying to put programs in place that will help get drugs approved faster. So the clinical trial that I’m on has been going for seven years and will keep going even though the drug is already approved because the drug was approved under what they call accelerated approval based on a Phase 1, 2 trial. Usually the FDA used to require that you had to have a big Phase 3 trial with hundreds of people where you compare the drug against the current standard of care and get a positive result before you could get the drug approved.
But now they’re making drugs for small populations like ROS1 patients. We’re 1 percent of the non-small cell lung cancer population, and you’ll never get enough of us together in one place to do a Phase 3 trial. So the FDA has something in place that allows you to approve drugs based on the Phase 2 trial. Everybody in this Phase 2 trial knows they are taking crizotinib. There is no placebo. So there are–the clinical trials are evolving.

Andrew Schorr:
So, Dr. Sequist, let’s back up for a second. So we’ve had–we have these meetings that you all go to, World Lung meeting, which was in Toronto I think a few months ago. And you have the ASCO meeting and others you probably go to around the world. What do you think is a big deal now? And I know I’ve seen you on the podium at some of these meetings. What do you think is a big deal for patients if you take away from some of the key studies that have been–you’re releasing data on?

Dr. Sequist:
It’s been a huge year for lung cancer. I mean, the standard of care has changed in lung cancer in almost every little corner that you look in. A year ago or certainly two years ago most patients who were diagnosed would get chemotherapy as the first pass treatment. If you happened to have one of the driver mutations then you would try and get one of those treatments first.
Now the standard of care has completely changed. Most patients get immune therapy with or without chemotherapy. There are new approved drugs for ALK and for EGFR in the frontline setting. There’s a new standard of care for stage III lung cancer which we haven’t had in 30 years. There’s a new standard of care for small-cell lung cancer which we haven’t had in 30 years. There’s more evidence from this past year about screening for lung cancer with low-dose CT scans and how this is really effective at diagnosing people earlier and saving lives, potentially especially so in women, we learned at World Lung. So every corner of lung cancer that you can shine a light into there’s been advancements over the last one to two years. It’s really quite amazing.

Janet Freeman-Daily:
We’ve also had one liquid biopsy approved where they can use a blood test to determine whether you’re eligible to take a certain kind of drug. That just happened last year I think.

Andrew Schorr:
So, Jess, you listen to this as a family member. What hope do you take away from that for your sister? Jess, could you hear me okay?

Jessica Wittebort:
Yes, sorry. You’re breaking up a little bit, Andrew.

Andrew Schorr:
I said you hear what Janet and Dr. Sequist were just saying. What hope can you take away from this because you worry about your sister of the week?

Jessica Wittebort:
Every single day I worry about her. And she has to worry about me as well. I often wonder who the real carer is. But, frankly, it’s, you know, she was given a brutal diagnosis three years ago, and she’s kicking. You know what I mean? She’s kicking. She’s doing great. She’s doing yoga teacher training. You know, she has good days and bad days, and I just think there’s an incredible amount of hope.
So get your head in the game, get some information. Get yourself a plan, and you move forward. And if you don’t find the doctor, and it happens all the time, can’t find the doctor you can trust or you can get the right answers from, then you keep looking.

Andrew Schorr:
So here’s some questions that we’ve got in. And, again, if our viewers have a question just send it to questions@patientpower.info.

Kevin writes in for you, Dr. Sequist, for many cancer patients there’s a learning curve. What are your thoughts on how a patient might know when they’re ready to learn and what are the first-stop resources that might give them education they’re ready for? And, Janet, I’m sure you’re going to weigh in. How about the ready to learn? Because otherwise at the beginning you’re drinking–you’re terrified, and you’re drinking from a fire hose?

Dr. Sequist:
Yeah, that’s a great question and I don’t think it’s one-size-fits-all. I mean, patients, it’s like all of us. They come with much different preferences about how they like to learn, about what they want to know, about whether they want to be the primary person learning things or they’re going to designate a family member to help them with this information.
Some people like to learn on the internet. That can be tricky because there’s a lot of bad information on the internet in addition to a lot of good information on the internet. Some people aren’t that into the internet, and they need to learn in-person or through meeting people or phone calls. Luckily, the lung cancer community has so many support systems and education systems that are out there.

Janet mentioned a few, LUNGevity and the American Cancer Society has some information on their website, but a lot of academic medical centers also have information on their websites about lung cancer and resources to connect you to learning more when you’re ready.

Janet Freeman-Daily:
So just to add to that, because there are a lot of wonderful, very educational resources on the internet the Lung Cancer Social Media group put together a reference page for vetted online resources. So if you go to lcsmchat.org under resources and look for what’s there you can find a list that includes links under various categories like for those who are newly diagnosed or looking at lung cancer screening or whatever. And on that list we’ve tried to pull a sample from all of the various pages we know of, all the various organizations that have good lung cancer information. So you can start there.

Andrew Schorr:
Dr. Sequist, I wanted to call out small-cell lung cancer, which I know is the minority of lung cancer. And Janet referred to immunotherapy there, and you talk about overall about hope. Where are we with small-cell now?

Dr. Sequist:
Well, there was a very exciting presentation in Toronto at the World Lung meeting and it got published in the premier journal, The New England Journal of Medicine, that same day that set a new standard for small-cell lung cancer, something that–it was actually really moving. The whole audience burst into applause and cheered essentially when this result came up because for most of us in the audience we had never witnessed an advance in small-cell lung cancer in the course of our career. So this advance is taking the standard chemotherapy for small-cell and adding immunotherapy to it, and patients had an improved survival when that happened.

Andrew Schorr:
Okay. So where do we go from here? Janet, you’re living with it. You wonder how long your medicine is going to work. You have one rare subtype. Other subtypes are being identified and then other people

where it hasn’t been identified yet. What do you want to say to people as far as just keeping on keeping on, if you will, and the importance of a dialogue with a doctor, a researcher in partnership?

Janet Freeman-Daily:
I think the only thing I would make sure everyone does, no matter whether you want to know all the details, whether you want to be involved in research is that it’s essential that you tell the doctor what is important to you. They can do all the rest of it if you need them to, but they can’t know whether it’s more important to you to try every last treatment no matter how lousy you feel, or whether you would rather make sure that if you can’t get out and walk in the woods then life isn’t worth living. They won’t know if you don’t tell them, so it’s important for you as a patient to start thinking about what matters to you in terms of your treatment.
Likely, you’ll be on more than one treatment at some point if you have metastatic lung cancer, and you need to know whether the side effects are acceptable to you. So even if you don’t want to do the research at least be able to tell the doctor what matters to you. I hope Dr. Sequist that you get some patients who do that.

Andrew Schorr:
So, Jess, so some people have trouble speaking up for themselves. I don’t think you’re sister is that way, but you go with her to a lot of treatments and visits. What would you say to family members to support their loved one, and if their loved one isn’t, isn’t feeling strong enough to speak up that the family member has permission to do that and that it makes a difference.

Jessica Wittebort:
Yeah, I think Marisa has her boyfriend, my dad, (?) Inaudible happy to hem and holler about the questions we have and the questions that she raised since the last time we saw the oncologist. But more recently she referred to us as the peanut gallery. I think she’s, you know, at the beginning of this diagnosis I was the one that reached out to the ROS1 group, and now she has a pleural effusion and she’s trying to figure out all the places that that pleural fluid should go to support research.
So I think that the journey will change. I hate that word, journey. I think the path changes as you go. You know that old when you come to a fork in the road, you can take the path or whatever it is, and I think you just have to figure out how to be flexible and flex with that journey. There was–one of the really nice pieces at the Biden Cancer Initiative, I’m terrible with names, the athlete was talking about, you know, everybody talks about diagnosis and the shoot for the cure, but it’s that middle, it’s that middle part that is so tenuous and you have to get really comfortable with the uncomfortable middle part.
So I think, gosh, it could be a strain and stress on your loved ones, and I think the communication is just one must of the exercise as you go, and if you can figure out how to lean into that as a carer, as a patient, as a loved one, then you’re probably ahead of the curve.

Andrew Schorr:
Thank you for that, and we wish your sister all the best, Marisa. My last question is for Janet and then Dr. Sequist. So it used to be the doctor was in the white coat, and the doctor said we’re going to do this, and you were scared, and you went down the hall to have a scan or this or a biopsy, whatever, you just did it. You’re just sort of literally the walking wounded, and you and your family were terrified. And whether you understood or not you sort of nodded your head, and that’s what would happen.

Dr. Sequist, do you welcome the change? Do you welcome the change that we’re sort of all in this

together? And I don’t mean just physicians but I mean researchers too, that this feeling that the patients, the family members, that together, we can solve things. Alone, it’s slower or more difficult?

Dr. Sequist:
Oh, yeah. It’s a very welcome change. I’ve gotten a lot of information and education as well as satisfaction from participating in the lung cancer social media group that Janet mentioned. It’s really great to be able to connect with people on Twitter who are researching lung cancer around the world or who are patients living with lung cancer around the world. And it’s a way to get lightning-fast updates about conferences, and everybody working together towards a common goal is a good feeling to be in that pack.

And I would say to patients out there if you’re in a relationship with a provider where it feels more like what you were describing, Andrew, like that you’re just being told what to do and you’re not being listened to or you don’t have the ability to speak up or have your loved one speak up for you, you need to seek out a different oncologist. Because it’s too important.
It’s too important of a disease to be dealing with someone you don’t have a great relationship with. And I would define a great cancer patient/oncologist relationship is one where both people can feel free for express what’s on their mind and to listen to each other and just feel heard and feel part of the decision-making.

Andrew Schorr:
I just think has a tragedy if, as you say, the landscape is changing so much–we have a long way to go, but it is changing so much in welcome. What a shame if you or your loved one passes away because there wasn’t a certain test done or a wide enough panel testing and there was something either approved or in trials that could make a difference to extend life. What a tragedy.
So Janet, I’m going to leave the last sort of empowerment message to you, what you want to say to people so that that doesn’t happen.

Janet Freeman-Daily:
I think there’s been a lot of good comments in the entire presentation along those lines. I think there’s a lot of evidence to show that engaged patients with serious diseases live longer. That patients who become more educated about their disease when it’s on the cutting edge as lung cancer is right now, they have a much better chance of making sure that they’re getting the best care.
But I also want to point out one interesting thing that’s evolving as we get these more empowered patient groups. We actually had a doctor, a researcher approach us because he had heard that ROS1 patients supposedly didn’t have as many brain mets as outpatients did, and that didn’t seem right to him. So we actually worked with him and did a survey on our own patient group and were able to tell him, yeah, it’s a lot more common than people are giving it credit for, which stimulated a whole new path of research that’s changing the way that people think about the disease. And if we had not had that open communication between the patients and the researchers, if we hadn’t had the empowered patient groups that survey wouldn’t have happened. So I think this change in paradigm being patients learning about their disease and getting involved in patient groups is making a huge difference.

Andrew Schorr:
Well, Janet Freeman-Daily thank you for being with us once again. I hope we get to do this for years and years, Janet, and one day we can say cured. Wouldn’t that be great? And I’m so delighted to see you and for joining us.

And Jess Wittebort, thanks so much for being with us too. All the best to your sister Marisa with the procedures she has, and, as you say, she’s kicking it, and I hope that keeps happening.

And Dr. Lecia Sequist from Mass General, thank you for your devotion to patients and helping lead the way in research so that we can really everybody can get the personalized care they need.
I’m Andrew Schorr from Patient Power. Remember, knowledge can be the best medicine of all.


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

Clinical Trial MythBusters: Actionable Advice for Knocking Down Obstacles to Trial Participation

Actionable Advice for Knocking Down Obstacles to Trial Participation

Downloadable Program Guide

Clinical trials offer tomorrow’s medicine today, but more often than not, only a small fraction of patients ultimately enroll in a trial due to barriers posed by financial logistics, distrust and travel, to name a few. In this MythBusters program, we will examine the barriers to enrollment, evaluate patient needs and discuss resources to help guide people through the clinical trial process with the help from two experts, Dana Dornsife of Lazarex Foundation and Myeloma Survivor Reina Weiner.


Transcript:

Andrew: Hello from Carlsbad, California, near San Diego. I’m Andrew Schorr from Patient Power. Welcome to today’s Patient Empowerment Network program, clinical trials myth busters and actionable advice, resources for knocking down obstacles to trial participation. I wanna thank the companies that have provided financial support for this program. They have no editorial control, but we definitely thank them for their support. Those supporters are AbbVie Incorporated, Astellas, Celgene Corporation, and Novartis.

Okay. We have a lot to talk about. First of all, I’ll just say I’ve been in two clinical trials; one Phase 2 many years ago at MD Anderson for the leukemia I have, chronic lymphocytic leukemia. And that gave me tomorrow’s medicine today. It worked, but I had travel far to do it and there were costs involved.

And then I was in a second Phase 3 trial close to home, and that was good too, and discovered another cancer that I have, myelofibrosis, through the monitoring in the trial. So, I’m a believer, but there are obstacles, and let’s talk about some of these. And we’re gonna give you some very specific resources to overcome these obstacles, so that hopefully, if a clinical trial is right for you, you can participate, you can feel good about it, and you can move medical science along to help everyone who is dealing with that condition.

So, what are some of the issues? Financial, of course; logistical issues, of course; distrust, are they really gonna take care of you or are they gonna protect your safety? Is it really right? And are you being given the straight scoop? What about travel costs? I went from Seattle to Houston, Texas a few times. Costly, okay? Stay in a hotel. It’s costly. Get a babysitter, leave work; costly.

The guinea pig syndrome; you’ve heard about it so many times. Are they gonna experiment on you, and are they really protecting you, and are you a number, or you are a person with cancer, or your loved one? And then is your medical team that you’re talking to about your treatment, are they informed about clinical trials? Or are they pooh-poohing clinical trials because they don’t wanna do the paperwork, or it’s happening down the road and not at their clinic. Lots of issues; we’ll talk about that.

Okay, I got some great helpers. So, first let’s go to Asheville, North Carolina, and you are used to live in Charlotte. Reina Weiner joins us. Reina, welcome to our program today. There we go. Say that again, Reina, you were muted.

Reina: Thank you.

Andrew: Okay. Now we should tell you that last June, well, June of 2017, Reina had a autologous transplant for multiple myeloma. And along the way, leading up to that, over many years she was in four trials. So, first of all, Reina, let’s start with what’s most important. Post-transplant, how are you feeling today?

Reina: I’m feeling very well. Thank you, Andrew.

Andrew: Okay, and what’s coming up at the beginning of September?

Reina: What is coming up at the – oh, a big party is coming up. Our children are throwing us our 50th wedding anniversary party, so that’s been cool.

Andrew: Yeah. Well, congratulations. And you’ve been dealing with what became multiple myeloma since 1999. We’re gonna come back and track that in a minute, as far as the steps along the way, the concerns you had or not at different times about being in four clinical trials.

And now let’s go up near San Francisco in the East Bay of San Francisco Bay, Danville, California. Dana Dornsife. And Dana is the Chairman of the Lazarex Cancer Foundation. Dana, thank you so much for being with us.

Dana: Thank you, Andrew.

Andrew: Okay. Now ladies and gentlemen, I want you to know Dana and her husband and her family, overall, they’re incredibly philanthropic across a number of issues that are faced globally, and also in the US. But one of them is helping people with the financial issues that prevent them from being a clinical trial. So, Dana, this is a personal story for you, so maybe you could just tell us why did you start the foundation? It was a family issue.

Dana: It was a family issue, and that family issue really revealed to me a gap that exists in cancer care for advanced-stage patients who want to remain in their battle with cancer through clinical trial participation. My youngest sister’s husband, Mike, was diagnosed with pancreatic cancer in his early 40s. He was given one half of one percent chance to live, and at the time 35,000 people a year were diagnosed, and 35,000 people a year were dying from pancreatic cancer.

So, we decided as a family that if Mike wanted different results that we would need to do something different. And Mike and Erin went ahead and pursued standard of care, and I was tasked with identifying clinical trial opportunities for Mike. And, of course, that sounds very linear, but in fact, for a layperson it was a very difficult task to undertake. I did identify some trial opportunities for Mike. He did participate in a trial and responded well for a period of time, he had good quality of life.

And during that period of time he was meeting people who were asking him, “Hey, what are you doing? I wanna do what you’re doing.” And he would say to them, “Oh, just call my sister-in-law, Dana. She’ll help you.” And that’s literally how this organization began. Through those phone calls that I was receiving from other pancreatic cancer patients, I began to understand that Mike was able to take advantage of medical breakthroughs in clinical trials because he had a family who could afford to support him through the process. And all of these other families that I was talking to, they just didn’t have the financial wherewithal.

So, we started Lazarex in order to fill that gap and help people identify clinical trial opportunities, and then provide financial assistance to them to help cover the out-of-pocket expenses that create huge barriers for patients who are already experiencing financial toxicity due to their disease.

Andrew: Well, thank you for what you do. And we’re gonna talk a lot along the way about resources. There’s a downloadable guide that you’ll be provided with, along with a link to the replay of this program. And that’s gonna have specific resources that you can access, whether it’s financial issues, other issues you may be facing. So, look for that.

Today we’re really focused on actionable resources. So, let’s go to Reina for second. So, Reina, you had been in the pharmaceutical industry.

Reina: Yes, I had.

Andrew: So, you knew about drug development, and you understood about clinical trials. So, I’m willing to bet you were pretty proactive. People who weren’t in the field, they don’t know from clinical trials, and maybe they’d been worried about it. They’ve worried would they be experimented on, would they be a number and not name, would they get quality care. But you were probably, I have a feeling, pretty proactive. And you write about that. I know you have a book as well. So, is that Step 1 for people to speak up for themselves?

Reina: It is Step 1; absolutely, Step 1. And what I found is, first of all, people don’t know about trials. And if you go to a small community practice where they’re very busy, they don’t have the time, they don’t have the staff to really educate patients about trials, the best, best step for patients to take is to ask, “Is there a clinical trial that might be appropriate for me?” That’s huge.

Even when I went to a very well respected hospital and there was a researcher who was following me as I had smoldering myeloma and the numbers kept going up and up and up. I said is there – because I was living close to the NCI – is there a trial that would be appropriate to me at the NCI. And he said just a minute, turned around, went to his computer, found the trial and that’s how I got in.

Andrew: But it wasn’t at where you were receiving care or being monitored at that time. It was somewhere else.

Reina: It was not. It was at somewhere else.

Andrew: Okay. Dana, is one of the obstacles, not just financial, or maybe it’s even the business of cancer where if an oncology practice that you’re going to that’s maybe close to home is not doing the trial, maybe it’s not even in their financial interests to tell you. I mean, is there an awareness issue, do you feel?

Dana: There’s a huge awareness issue there, Andrew. And it all starts with knowledge is power, right, so I completely agreement with Reina’s comment about one of the first questions you need to ask is, is there a clinical trial out there for me because many doctors who are in community environments don’t offer that information. It’s not what they do every day. They’re there to administer standard of care. Only 6 percent of doctors actually engage in conversation with their patients about clinical trials, and that’s usually the 6 percent who are associated with research universities, right?

So, knowledge is power. If the patient doesn’t know about a clinical trial, they’re never going to participate. But once you find out and once you’ve identified an opportunity, the second biggest hurdle is that out-of-pocket expenses associated because most patients have been dealing with their disease for a longer period of time, and they’re basically broken in every way: physically, emotionally, spiritually, and, sadly, financially.

So, patients start to make decisions about the outcome of their care based on the size of their checkbook, and not focused on what’s best for them. And so, Lazarex eliminates that financial barrier as well to help patients say, “Yes, I can participate,” and we can get them where they need to be when they need to be there.

And that’s just the tip of the iceberg, Andrew, because there are many other barriers that exist; socioeconomic barriers, language, culture, historical barriers. And we are tackling all of those barriers one at a time. But really, the biggest two barriers are knowledge and financial.

Andrew: Right. And you mentioned about the historical barriers. Some people know about the Tuskegee experiments with African-American people, so in the African-American community, there still is a distrust among some people. Yet if you think about it from the FDA’s point of view where a company that’s developing a drug, or the NIH, they say okay, how does it work on broader populations or different ethnic groups or different ages or genders, et cetera?

They want to understand that data, and so not just having a number of people participating in the trial, but having it reach people who are in different situations, if you will. And so –

Reina: And if I may – ooh, I’m sorry.

Andrew: Reina, please, go ahead.

Reina: Well, if I may say that because people don’t know about it and the trials, the best trials, are trials with a variety of patients, but they do try to accrue populations who are certain ages, certain genders, ethnic groups, whatever they can get. And only 3 to 5 percent of patients participate, cancer patients, participate in clinical trials, and so much is lost if people don’t participate.

Andrew: Here in San Diego it’s sort of a pocket of a lot of medical research. There’s a lot up in your area, Dana, in the Bay Area, San Francisco Bay Area. I mean, it’s in North Carolina in the research triangle where that’s home state for Reina. And not to disclude others, and then certainly up around Boston. There are like companies all over the place and many of them are in earlier drug developments.

So, when you talk about immuno-oncology now, can we harness our immune system with the help of some medicine to fight the cancer, and I know some people who’ve received it; lung cancer patients who are living, et cetera, melanoma patients who are living for an extended time. These companies can’t move forward unless there’re people who are in the trials. So, the FDA says where’s your data? And they’re saying well, we’re trying, but we haven’t been able to complete this trial. Right, Dana? So, we can’t move towards cures unless we all come together.

Dana: That’s exactly right. So, let me just throw a few statistics out at you that I found astounding when I learned of them. So, we have a 48 percent failure rate of clinical trials, and it’s not because the drug didn’t work. We will never know, quite frankly, if the drug would have worked or not. And we will never know because there weren’t enough patients enrolled in the trials to find out.

So, 11 percent of trials never enroll a single patient, if you can believe that. So, here we are with an almost 50 percent failure rate, and yet we have 600,000 patients a year in this country who are dying from cancer. So, there’s this incredible disconnect between the thousands of patients who would participate in clinical trials if they could, and the thousands of clinical trials that need patients to participate in order to succeed. And without successfully completing those trials, those drugs are never going to get market to help the cancer patients that they are intended to serve and help.

That’s why Lazarex Cancer Foundation exists, and that’s why removing the barriers to clinical trials is so important. Our process does not lend itself well to that. And I just want to take a step back, Andrew, to address the minority participation in clinical trials. We all understand because of epigenetics and, yeah, advances in medical science that we need to have the full spectrum of our population participating in clinical trials. But that doesn’t happen. When you look at the 5 percent of patients who actually participate and you break it down ethnically and racially, less than 5 percent are from minority communities combined.

So, in theory, though we say we understand the importance of that, we’re actually not in practice doing what needs to be done. And so a lot of our work is also focused on reaching out to those socioeconomically challenged and racial and ethnic minority communities to raise awareness and help people like you’re doing on this program dispel the myths around clinical trials, so that they’re more inclined to ask better questions.

Andrew: Right. So, so important, and I applaud for that work. We’re gonna talk about the financial process in a second. Reina, so you were involved in a National Institutes of Health or National Cancer Institute trial.

Reina: Yes.

Andrew: A couple of them, I think, and one at Memorial Sloan Kettering in New York’s premier resources. So, we talked about your tip was you gotta speak up and ask about trials, where they’re at that center, wherever you are, by XYZ oncology in a suburban area, whatever it is or not. So, what’s Step 2? So, for instance, now I understand there are people – and Dana, I’d like your comment on it too.

At some clinics now where there are clinical trial – there are nurse navigators, but often sometimes there are clinical trial navigators too, but often you gotta ask about that too, right, Reina? I mean, it’s speaking up and looking for the resources that are available to you there or wherever you choose to go, right?

Dana: Yes, and there are organizations like Dana’s who help people do clinical trial searches because that’s a bit overwhelming when you are already frightened, you already have the financial issues coming up. And like you mentioned, logistical issues. So, there is Dana’s organization; therefore, myeloma, the SparkCures. There’s the MMRF. There’s the International Myeloma Foundation. There is something called Cis Crypt. And so, they will help you find a trial.

And there are lots of regional trials groups, so you may not need to go to the big, big research center. They might be able to do it locally for you. But I always want to bring up the fact that there’s so much misinformation about trials and what it entails. There’s a tremendous amount of fear. And when I went on the first trial, as I wrote about a little blog recently, everybody said to my husband – well, not everybody, but an awful lot of people said why would you let your wife going in a clinical trial? She’s definitely gonna be a guinea pig.

And I can tell you very, very, very clearly that you get so much care. There’s so much documentation. And the patient’s health is never sacrificed for the research ever. And so, and you sign a consent form, so you’re very clear about what is going to happen. And yes, there’s more there’s more bloodwork. Yes, there are more biopsies. And it’s part of research. And when you sign up, you sign up. And I had more than I’d like to even talk about, but I feel very grateful and very humbled for the care that I received.

And I can tell you, too, that I talked to other people on the trial. And yes, they hope to gain better control of their cancer. But, in addition, they really hope to help the next group of patients who are coming up, so that these new treatments actually happen.

Andrew: I feel the same way. I was in a trial at MD Anderson in 2000, and the three-drug combination I got was not approved till ten years later, but they learned a lot. And you were on a three-drug combination, which I think still has not been approved for first line, but it’s is widely used, I think.

Dana: Right.

Andrew: So, in multiple myeloma. I wanted to mention some other resources, the Leukemia & Lymphoma Society also has a resource center. You can call them. So, there’re these different groups that help you identify a trial, and doctors who specialize, so let’s say pancreatic cancer, you mentioned earlier Dana. I got a call from a friend in Miami, “How do I find a pancreatic cancer specialist?” And I connected them with PanCAN, Pancreatic Cancer Action Network in Los Angeles, who knows who are the doctors who have the most experience with that.

Now, Dana, so then the next thing comes up is alright, I’ve identified the trial, but it’s not where I am. So, now we talk about logistics and finance. So, let’s say somebody calls your foundation. Tell us how it works. So, I don’t have the resources. Maybe they live in Northern California and the trial is in Southern California or in Salt Lake City. What happens next?

Dana: So, Andrew, in some cases it’s not even that distance. In some cases it’s getting from Sacramento to San Francisco, which is literally a one hour, one-and-a-half hours without traffic, in your car. And sometimes it’s a tank of gas, a bridge toll, and parking. I mean, we’re not talking about thousands of dollars in some cases, but it’s still the difference between life and death.

When someone calls Lazarex Cancer Foundation, they can contact us directly. We have a financial application that we use to determine the degree of eligibility for patients to get their out-of-pocket expenses reimbursed. Or they can be referred to us by their social worker at the institution where they are receiving, or thinking about participating in a clinical trial.

We take a look at the household income of the patient, and I believe our guidelines are very generous. We go up to seven times the federal poverty guidelines for patients. And we arrived at that number through trial and error. Our goal is to help as many patients as possible participate in clinical trials, and turn away as few as possible. And then we reimburse on a sliding scale from 100 percent to 75 percent to 50 percent depending upon your household income.

And it’s a pretty easy process to go through in order to be enrolled and receive the reimbursement. And then we reimburse our patients monthly, on a monthly basis. And in some cases, we’ve been working with patients, we follow them, like Reina, through two, three, four clinical trials. And we’ve been supporting them in trials for years. And without doing what we do, they may not be here with us today.

Andrew: Well, I’m sure you’ve saved some lives and lengthened some lives. Reina, so you were in the pharmaceutical industry and in oncology, I believe, before all of this started happening to you. And you’ve continued teaching nurses and devoting yourself to education and your book and your blogs. Thank you for all that. Maybe that’s what life’s about.

But knowing on the inside there are pharmaceutical programs, in some cases, I think, particularly for rare cancers where they may provide assistance. They can’t pay you to be in the trial, but there are at times assistance and travel logistics, particularly for rare cancers where maybe the trial is not, not one hour away. Am I right, Reina? Are you familiar?

Reina: Oh, there are. And sometimes when I was working, there would be a patient who had a cancer that really was not aligned with a particular treatment that would be effective for them. And so, the doctor wanted to try an off-label use of a product, and so then they would come to me and asked me if I could get the pharmaceutical company to provide the drug for free.

And sometimes it takes a little doing, but I was concerned about the patient and hoping to get them a better quality of life, if not an extended period of life. And so, yeah, the company would do that. Not every day, not all the time, but if the company had evidence that this was a patient who would benefit from the off-label use of a product then they would help them out.

Andrew: Okay. So, Dana, related to other organizations providing assistance, and I recently interviewed someone from the Family Reach Foundation where they help with rent or things, groceries, things like that. So, somebody says, “Oh my God, I’m afraid of a trial, I can’t go there,” or if they hear about it and they say, “Hmm, well, maybe I could, but I’d have to leave work, or maybe my spouse would have to leave work, we’d have to find somebody to pick up the kids from school, oh my God.” There are organizations that can help with some of these family processes, aren’t there?

Dana: Absolutely. And I think we’ve provided the Patient Empowerment Network with a list of those. 21st Century C.A.R.E. is an organization that provides patients with immediate financial assistance for expenses related to active cancer treatments. Cancer Care provides assistance for cancer-related costs. There’s a Cancer Care Co-Payment Assistance Foundation. We get that question a lot.

We’ll help with the out-of-pocket travel expenses, and in fact, some of the medical and diagnostic expenses that aren’t covered by insurance. When you’re participating in a trial, sometimes you have to get more stems than insurance will cover or whatever. But co-pays are a big deal for people to be able to afford those, and so, that is another organization that can help. Patient Advocate Foundation, which is an underinsured resource directory.

So, there are a lot of you nonprofits out there who exist to support patients through the fifth process. It’s just a matter of helping patients really understand and put together all of those resources in a way that they can access them.

Andrew: Okay. So, Reina, you’ve been through it four times, and you’re a pretty savvy person. Not all of us know as much, so help us now. So, one of the questions in a trial is, and in cancer, am I gonna get what I describe as the good stuff, knowing that the good stuff that’s being tried may not be good. I mean, it may not work out. There are trials that go bust. Not just for not getting people, but they got people, but it wasn’t as effective as they hoped it would be.

But let’s say we’ve done our homework and we go to a certain clinic, but it’s some sorta controlled trial. We don’t know whether we’ll be in the arm. So, was a concern for you? Were you gonna get the good stuff, and why do it?

Reina: Well, no, really, Andrew, because I know that like if it’s a Phase 3 trial, so you’re comparing standard of care versus the newest and hopefully the latest and greatest. If it turns out that one arm of the trial really shows a significant improvement, patients are always switched to the more effective arm of the trial. They don’t leave you on this arm of the trial thinking well, what the heck, we’ll just leave you there and see how the research pans out. So, they are always switched over to the most effective.

So, I wasn’t really concerned about that. And in the Phase 2 trial, it’s just seeing if the product was effective. And so, that was obviously not a concern for me. So, it worked out, and I do think, though, like what Dana does is absolutely wonderful at totally, totally, totally past wonderful.

But I always try to let people know who have friends and family who are facing some chronic significant illness that don’t just call and say let me know, let me know if I can help you because that’s so ambiguous. And most people will not call because they have pride or they think they can do it all by themselves.

So, I always try to suggest to people that if you’re calling somebody who you think might need some help, be specific. Call and say, “Can I walk the dog? I’m going to the grocery store in an hour. Is there something I can pick up for you? Can I mow the grass?” Anything that will help, but make sure that you are specific in your offering.

Andrew: I want to talk about a related issue. You use the word pride. Some people, maybe in some cases it’s even shame. They developed a certain cancer. Where these are maybe middle-class people who’ve had some resources. They’ve been paying their mortgage. They’ve been paying their expenses, making do. But now they get hit with a cancer diagnosis, which is catastrophic, and there is help available, Dana, but they’re too proud to ask for it when this could happen to anybody. And maybe you’ve even countered that along the way or know there’re people out there. What would you say to people, to not be shamed and to speak up?

Dana: Yeah. Well, sadly, one in three women will be diagnosed with cancer, and one in two men. And so, this is not an uncommon scenario, right? The likelihood of knowing someone who will receive a cancer diagnosis is very likely. So, I think that patients have to understand that pride doesn’t help you in your process with battling this disease. You have to take advantage of every opportunity that’s out there in order to come out on the positive side of this experience. And if you don’t take advantage of every opportunity, you may not.

And so, it’s one of those things that we just have to deal with right from the beginning, and just say okay, again, knowledge is power. I’m going to surround myself or engage with the people that are around me who want to help me. And you have to put that team together because you will need your team with this disease.

Andrew: Okay, so great advice. Reina, part of your team maybe could be the first doctor you saw who gave you the diagnosis, but they might not be the one where a trial was offered. So, first step is you talked about speaking up, but it takes a lot of courage to say to the doctor in the white coat with all the letters after their name, you know, thank you so much, Doctor, and I’ve either found out about a trial, or your turned and typed it in somewhere else. I hope you don’t mind, but I am going to go over there. Maybe you can advise me along the way.

But that takes courage because people are terrified, and they may be bold in principal in that situation with the person in the white coat. What advice would you give?

Reina: Ooh, well, that’s a big one for a lot of people. And, really, you know what, I imagined that it would be people who are older, who come from a generation where the doctor has the final word. But what I found out when I was writing my third book is that there were younger people who also feel very uncomfortable speaking up, asking a doctor, and so forth. But really, what to really put in your little mind and in your heart is this your life.

This is not just kind of a trip to the mall. This is really important for you to either improve the quality of your life or extend the quality of life, so take a deep breath, be very polite, and I think most doctors who are professional and open-minded will hear what you have to say if you present it in a way that they can hear. And if they really don’t hear you then it might be time to have a look around to see who will.

And, really, the bottom line is you need to trust yourself. And if you feel that this is really right, that there is a clinical trial that you would be eligible for and you can participate in with Dana’s help, with the financial, with the logistics, and so forth. Like I said, you just take a deep breath. And most doctors, like I said, really want the best for you.

Andrew: Okay, let’s talk about something that comes up. One of the things for people is the criteria of different trials. Dana, I don’t know if this is in your area, too, related to financial, but people let’s say okay, I wanna be in a trial, but the criteria are so narrow that I really wanna be in the trial, but they say I can’t.

Dana: Yeah, so that is a sad reality in many cases. And I refer to this as Clinical Trial Nirvana Syndrome where as a drug sponsor for trial, you want to attract the healthiest patients you can to participate in your trial, so that you have the greatest chance of success. But, unfortunately, in many instances, in most instances, a cancer diagnosis is accompanied by other comorbidities like heart disease or diabetes or other maladies that would preclude a patient from being able to participate in a trial.

So, that is an area that we are looking into and trying to – we have several proposals out there with various aspects of our government to try and really take a closer look at that, to try and make the trial makeup in relation to patient participants better mirror the realities of our situation because the likelihood of someone, if the drug gets approved, taking that drug and having a comorbidity is pretty likely.

And yet we won’t know what will happen there, right. So, we have to drill down on these issues and it’s a great, great issue to bring up. So, we’ve got a lot of work to do ahead of us.

Andrew: Right. And another thing that comes up too, and Kevin sent in a question. Kevin, thank you for this, matching what’s available in clinical trials to where you are in your journey with an illness. So, on Day 1 you’re diagnosed. I know Esther and I, we were crying and almost on the floor. And I thought I would be dead the next day. And it really took a while to overcome the terror of the diagnosis. And so, we were not even – well, the doctor wasn’t talking about trials; we wouldn’t have been hearing it anyway.

And some of us, thank God, with some trials, with some cancers now, are blessed with living longer and we start to learn. And then we want to know, in our situation, what applies to us. So, I know there are a lot of efforts being made to match trial offerings to where you are and what you might need to know now, what might need to be offering.

And some of you have heard this term, artificial intelligence, where we in the Internet business are all trying to fine tune what we’re suggesting or putting in front of you based on who you are and where you are, recognizing privacy and all those kinda things to make it more manageable.

We still have a long way to go. I mean, we have clinicaltrials.gov, but it’s not tied to where I am, who I am, where I am in my journey. It’s just what’s being done in a certain illness, right, Reina?

Reina: Yes.

Andrew: So, we have to refine our tools.

Reina: Absolutely.

Andrew: We have to refine our tools. Well, we’ve been getting in a number of questions. So, here’s one. So, David; so, he says as the excessive use of CT scans in clinical practice moves away from being the norm, have they lessened their use in clinical trials? In other words, this is about testing, and maybe it’s about the requirements.

Dana, I don’t know if you have feelings about it, but the scientists who are doing these trials, they wanna know everything. They would like to test us. So, the CT scan, and I mean I’m gonna have one next week, but it has radiation, right?

Dana: Right.

Andrew: So, let’s do a bunch of CT scans. No, let’s do a bunch of bone marrow biopsies. No. So, I’m saying I’m sorry. Not just do I have to pay something for these tests, or is there a co-pay or whatever, but also am I gonna be radiated? Am I gonna be poked? So, what about those issues? Is there dialogue going on, not just to help us financially, but also make it less onerous, I guess?

Dana: Yes, in fact there is dialogue going on about that, and it’s good, heartfelt dialogue. And it’s coming from a myriad of stakeholders, right, not just from patient advocacy organizations, but also from within industry insurers. And the whole goal is to okay, let’s stop looking at patients as a chart or a number on a piece of paper, and let’s understand that these are living, breathing human beings who are voluntarily participating in this clinical trial process for the benefit of not only themselves, but future patients to come and our industry.

And let’s start treating patients as humans who are participating, and let’s see what we can do to lessen the number of visits or minimize the number of scans and blood work, et cetera. So, there is active dialogue around that, and I think there’s a much higher degree of sensitivity on behalf of the teams who are actually putting the protocols together now.

Andrew: All right, I think so. And I know in some cases they’re doing what’s called trial simulations with a panel of patients and saying okay, we’re trying to answer these scientific questions and see if this drug that’s in development can do better for patients and would require so many office visits. Or so many, you come to the site, but so many could be done, maybe with your local doctor if that’s closer to home. So many blood tests, so many CT scans, so many biopsies. Imagine lung cancer patients with another lung biopsy. Not fun, and often not available.

So, there are all these kind of questions. And I think that’s going on although it needs to happen more. Now Dana, do you talk to the pharmaceutical industry? We had a question from Vi Life wanting to know related to trial awareness. Beyond the financial, do you work with pharmaceutical companies at all, as you are now, today? I mean, what we’re doing here is just to raise awareness about trials or other programs that you may do.

Dana: So, we are engaging with pharma right now. We were very fortunate to work with the FDA earlier this year in securing language around reimbursement of patients’ out-of-pocket expenses associated with clinical trials. There was some very nebulous language out there that was really preventing pharma from being able to support programs like ours.

And what we’re doing now is, in addition to we’re bridging this gap for patients that exist every day by reimbursing patients, but that is not a sustainable business model. It’s noble, but we have to have our tin cup out every day. And the number of patients we can help is directly related to the amount of money that we have in our account, right?

So, in addition to that program, our Lazarex Care Program, what we are also doing is trying to fix this problem and do it in a sustainable way. And in order to do that, we actually have to shift the burden from the patient back into industry, right, and help industry understand why they should include these out-of-pocket expenses as part of the clinical trial protocol every time, right, so they can enroll trials on time, on budget, save R&D dollars, preserve patent years, right?

I mean, there are a lot of reasons why pharma would want to participate in a program like that, in addition to the fact that it’s the right thing to do, right? And then we get more drugs to market faster, and we provide a platform of equitable access for everyone. So, we are engaging pharma in discussions right now about funding this program, we call Lazarex our IMPACT Program, that’s being rolled out at comprehensive cancer centers across the country. And it stands for Improving Patient Access to Cancer Clinical Trials.

It has been received very well and I’m happy to say that Amgen actually stepped up and funded this, so we are rolling it out here in California, and we are hoping that we’ll have similar opportunities in a couple of other areas in the United States. So, they are interested, and they want to improve clinical trial enrollment retention, and especially minority participation.

Andrew: Right. Boy, that you. Again, I keep saying thank you for what you’re doing, but you’re a real leader in the field. I’m going back next month to the Biden Cancer Initiative Summit continued by Vice President, Biden, former Vice President Biden, and his wife who continue to do leadership in this. And there’ll be a lot of senior people there and I’m hoping we can talk. And I know this issue of how can we advance cancer care through research in partnership with patients is a big one. So, Dana, thank you for helping lead the way in getting this going. And thanks to Amgen just as an example.

Reina, so, we talked about the cultural differences of people being in trials. We talked about the pride people may have in asking for assistance, the fear people have maybe participating in trials. You still have a – not now. I mean, you’re doing so well and you’ve been through trials and it’s worked out well. But there must have been some bumps along the way. Were there any misgivings at different times? And if so, how did you overcome it?

Reina: Oh, yeah. Well, there were definitely misgivings, I am sure. The first trial was when I asked the doctor if there’s something going on at the NCI. And there was no misgivings about that because that was a very observational trial. The second trial was much more progressive and I felt kind of a little uncertain about it, and so I asked the researcher at this well-known institution if I should participate because the trial, I should back up a little bit, that was for either smoldering myeloma patients or active disease patients.

At the time I was smoldering, and most physicians didn’t believe that that was a good idea to treat smoldering and wait until it became active. So, I asked this one researcher and he said absolutely, not, do not participate in the trial. And then I called someone else also from a very respected institution where I had been, and he said well, if you join that trial you’ll be crossing the Rubicon, which I didn’t even know what the Rubicon was at the time. I had to go look it up.

But, basically, once you start treatment, you kind of go on that journey and there’s no way to step off. But then I thought about it, I thought about it, thought about it, and finally I decided to trust myself because I had been to the NCI. I felt very safe there. And I decided to move ahead with it.

So, yes, I had plenty of misgivings about that. The other trials, not really because that trial changed my life and it gave me a very reasonable complete response. And the other ones, like I said, they just kinda fell in with the collecting a good amount of stem cells for a transplant and so forth.

Andrew: I wanna talk about family issues. So, the decision to be in a trial affects the family, whether it’s somebody’s driving you to the doctor, somebody’s taking off work, their worry, how they feel about trials, their own view of it, family logistics, costs, et cetera. We’ve talked about that. So, you wrote this blog about people questioning your husband, I guess, was your wife gonna be in a trial? So, how did you overcome that, whether if not with your husband, just with your community that you weren’t like crazy?

Reina: Well, they already know I’m crazy, so that’s a total aside. But, really, trying to educate people about the misinformation about trials; say, look, I will never be a guinea pig because that’s not what trials are about. And it’s very well controlled and there’s a lot of data that follows you. The care that I got was excellent.

And I try to dispel, like I said, a lot of the myths; that you signed consent form, which clearly explains what the trial is about, what your commitment to it is, and you can also drop out for any reason. There was hope that you don’t because they would like to have some results that then will lead into future treatments for patients. But you can drop out, so, really, taking that opportunity to educate people about what a clinical trial is like and that there are no guinea pigs.

Andrew: I wanna just – oh, yes, please, Dana.

Dana: Yeah, if I could just offer something in that regard. For people who haven’t gone down this path, the journey with cancer, having a cancer diagnosis is not like other chronic diseases, right, like diabetes, for instance, that you can typically control with insulin or whatever, right? For a cancer patient who has failed standard of care, who’s gone through maybe second- or third-line treatment options, but still has progressive disease, that patient will die if they don’t do something, right?

And so, clinical trials offer tomorrow cures today in some instances, right, and we don’t always have positive results in clinical trials. But for a patient who’s at that crossroad where their doctor has delivered those words, “You need to get your affairs in order,” right, it’s not a matter of am I crazy if I participate in a clinical trial. What it is a matter of is do I wanna live? And if so, what clinical trial can I participate in? It’s a very different decision tree.

Andrew: Right. And I certainly say that all the time. I got a call, as I mentioned, from a friend in Miami. The mother has a very serious cancer. And I said part of the initial discussion, even the initial discussion, Dana, can also be are there clinical trials that we should consider along with standard therapy? So, certainly, if you’ve failed or they’ve failed you, the treatments no longer work, what is the 360 degree view? And if you don’t do it here, so they do it down the road, or do they do it across the country? And what are the issues for you participating?

So, a lot of thinking, but it’s gotta be part of the discussion. So, so sadly now, what are we seeing; 3 percent, 4 percent, 5 percent of adults participating in cancer clinical trials in the US. Not good at all. And are we hurting ourselves with the chance of future therapies that can be more effective, or even cures because some of these companies sometimes are venture-backed. They don’t have money forever, you know, and they’re trying to get to the goal line to go the FDA.

Look, here is another question we got in. Tamara, our producer, just sent in. She says well, what happens when you join a clinical trial and it doesn’t have a beneficial impact? So, Reina, they didn’t know that the trials would necessarily work out for you. So, what happens then? Do you go on another trial? What do you do?

Reina: Well, if it doesn’t and you don’t seem to be responding to the therapy on the trial, or you find it intolerable yourself, then they will always return you to your oncologist who you had been seeing previously. But, on the other hand, they may offer you another trial that’s available that you would be eligible for as well. So, I mean, I really try to stress to people that the researchers are looking out for you. They want the best income, in addition to accruing the data that they hope.

And I can tell you that when I was on a trial at the National Cancer Institute, when I had questions, especially about the trial with smoldering versus active disease for myeloma, they would spend a couple of hours for me, explained with me, can I say that, right? Yes, spent a couple hours with me explaining all of the aspects of the trial, so yeah.

Andrew: I wanna point up an example that some people have heard of a woman I’ve become friendly with in the myeloma community, Reina. Cherie Rineker. So, Cherie’s down in Houston, and she was dying of myeloma. And she’d been in trials and treatments. She was at MD Anderson. Bob Orlowski is one of the top doctors in the world, and her doctor. And she was in different trials and then things were not working.

And she was put in touch with another researcher doing this CAR T investigation for multiple myeloma, which is pretty new, pretty new. And they’re learning a lot. It’s not a slam dunk, but so far it’s worked for her. It saved her life. She went to Nashville, Tennessee from Houston where she lives, and maybe I’m not sure the financial issues, Dana, about going. But that’s where she’d been in successive trials. And some were not working or no longer were working. There was another approach.

I wanna ask about another concept I’ve heard called siteless trials. And I don’t know, Dana, you’re nodding your head. Maybe you are familiar with this. One is a siteless trial because we talked about these trials going on at these academic medical centers, but not much elsewhere.

Dana: So, I have tell you, I don’t have a lot of experience with siteless trials, but there is a lot of dialogue taking place around rather than having the patient go to the trial, bringing the trial to the patient, and I think that’s the impetus behind a siteless clinical trial.

I think cancer has some unique challenges, especially blood-based cancers in clinical trials, and the oversight of patients participating in those trials that make siteless trials a bit of a challenge. But I think the place to start is in other diseases, or perhaps where you have a cancer diagnosis that’s not a rare form of cancer, whatever that requires, a high degree of oversight.

But the whole goal in doing this is to understand how we can get more people into these trials and make it less obtrusive on their life, right, so that more patients would be inclined to participate, increase our enrollment retention, our minority participation, and, ultimately, reduce the burden on the patient to participate.

Andrew: Esther and I’ve given a lot of talks at different conferences, and we said you have to see patients who might be considering or are in a trial as investors. So, they’re gonna invest with their body, their time, sacrifices, and other things in their lives for the hope of being cured if they could, or doing better.

And there needs to be the communication, financial support, logistical support in really treating people with a lot of respect as a person. Reina, do you agree with that, that we have to get to that concept where we’re taken care of? And you felt that way, but we need to do it for more people and have more people feel confident that it’ll work out that way.

Reina: Oh, certainly. Certainly, I do. And the education is really essential. And after I was in the first trial, I talked to everybody who would listen to me. And even if they didn’t, I would talk anyway just to try to say this is a place where you can go where you will receive what is hopefully the newest and the best treatment that’s available. That you will be cared for as well as you can possibly be, and that everything is documented. You know all the options that you have staying on the trial, giving consent, making sure you have all the information that you need to feel comfortable.

And Dana’s organization, hopefully, helping people out financially and logistically. There are ways to get into trials that at times are very successful. For me personally, I don’t know that I would be alive now if I had been on that trial, and that’s really my claim to fame, what can I say?

Andrew: And, Reina, I would say the same thing. Had I not been in a Phase 2 trial for chronic lymphocytic leukemia in 2000, I wouldn’t be around to have had retreatment last year, which has work quite well; 17-year remission. And I wouldn’t have been able to do this, and really have a purpose in life. So, I’m very grateful for being in the trial.

Dana, I can’t tell you – we were talking about gratitude, for you; came up in your family. You saw the gap for, not so much your family, but so many other families. The issues, financial issues, and you’ve been very philanthropic and, obviously, trying to have leadership in getting at some of these – we have a very imperfect system right now, so we have a long way to go. But for our viewers, if you’re living with cancer now, if your loved one is living with cancer, there are resources, people like Lazarex, people have been through it, like Reina.

We’re gonna give you this downloadable guide. And you’re gonna connect with these resources. Don’t… Put your pride away. Dana said it so well. There’s a very high likelihood we’re gonna be affected by cancer in our families, and there is help to navigate what’s kinda complicated right now, but is doable and can offer you the chance of doing better. Dana, did I say it right?

Dana: You did. You did. You did a great job, Andrew. Thanks.

Andrew: Okay. Well, thank you. And thanks to the Lazarex Cancer Foundation and, really, all you’re doing. And let’s hope that we can improve this process, increase participation, and have so many of these companies and the government that are trying to get scientific answers. We participate as respected patient investors. And we do better well. Reina, any final words from you with your 50th wedding anniversary coming up?

Reina: I’m very grateful. I’m very grateful to be here. I’m grateful for all the clinical trials, all the physicians who have taken care of me and who listened to all my concerns and fears. And I am super-duper grateful to my husband who has supported me, helped me, been there, been my caregiver, and washed the food for me when I had the transplant, and really, all the people who have been on the journey with me. So, if you are considering a clinical trial, if there is one that you might be eligible for, give it some thought. It’s a really important choice for you to make.

Andrew: Reina, thank you so much, all the best. Happy anniversary, early. Dana, best to you. Dana Dornsife, joining us from the Lazarex Cancer Foundation in the San Francisco Bay area. Dana, good health to your family, and thank you for all you do. Thanks for being with us, Dana.

Dana: Thank you.

Andrew: And, Reina, all the best, and thank you for those great words of wisdom. And we’ll meet in person sometime and I’ll give you a big hug, okay?

Reina: I hope so. I hope so. You take care of yourself, Andrew. Thank you so much.

Andrew: Thank you for joining us for this Patient Empowerment Network program Clinical Trials Mythbusters. We hope to do more. I wanna thank the companies that have helped provide funding for it; Abbvie Incorporated, Astellas, Celgene, and Novartis, for their support.

Thank you for joining us. I’m Andrew Schorr from Patient Power down near San Diego. Remember, knowledge can be the best medicine of all.

Reinventing the Clinical Trial: Start at Ground Level

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

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

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

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

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

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

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

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

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

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

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

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

We’ve got to start somewhere, right?

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.