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

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Topics include: Caregivers , Clinical Trials , Patient Stories and Understanding

Before considering a trial, many patients wonder: “Is a clinical trial right for me?” “How would it affect my family?” “Do the risks I might be taking outweigh the benefits?” These are all questions our panel of experts discuss, disect and debunk during this program. Dr. Michael Weitz shares his story of being diagnosed with lung cancer and being  given a prognosis of three to six months to live. Now, 11 years and three clinical trials later, Dr. Weitz says, “I owe my life to targeted therapies and personalized medicine. There is no advancement in research without clinical trials.” Watch now.

The Clinical Trial MythBusters series is a Patient Empowerment Network program produced by Patient Power. We thank Astellas, Amgen and AbbVie for their support.

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Transcript

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. 
 

Andrew Schorr:

Welcome to this Clinical Trials MythBusters program, the second in our series.  I'm Andrew Schorr broadcasting from Carlsbad, California.  We're going to cover the country today in this next 30 minutes or so with people from around the country who we'll call myth busters to really help you understand can you or your loved one get tomorrow's medicine today?  I got that years ago for chronic lymphocytic leukemia in a Phase II trial, just kind of an intermediate trial, at MD Anderson Cancer Center in Houston, and I think I'm alive today. 

But in a minute you're going to meet a lung cancer patient who feels much the same way.  You'll also meet a leading lung cancer specialist but someone who is very involved in research across oncology from the University of Pittsburgh and the UPMC Hillman Cancer Center there.  And you'll also meet someone from the clinic that I go to, Moores Cancer Center at UC San Diego, who is a clinical trial nurse specialist and so people with lots of experience. 

So we welcome your questions.  You can send them to questions@patientpower.info.  I want to thank the Patient Empowerment Network for supporting this program.  Their program, we're producing it for them, and they are a wonderful organization, powerfulpatients.org.  Take a look at that for so many wonderful support services.  And also I want to thank our sponsors who provided financial support, and that is Astellas, Amgen and AbbVie.  Okay. 

Let's meet somebody I was just talking about.  And let's go just up the road from me in California, to Woodland Hills, California, near Los Angeles, Michael Weitz.  Michael has been living almost 11 years now with lung cancer.  Michael, thank you so much for being with us. 

Dr. Weitz:

It's my pleasure.  Thank you. 

Andrew Schorr:

So, Michael, I've heard you quoted as saying that you believe you're alive today almost 11 years now after a lung cancer diagnosis because of clinical trials. 

Dr. Weitz:

No question about it.  I would not be here today without the amazing new discoveries that have emerged during this last decade. 

Andrew Schorr:

Now, you've been on three clinical trials and other therapies, some approved therapies that were available along the way as well, and some have been infused and some have been oral.  You've had surgery and radiation, but these trials keep allowing you to live your life, and I know you have three boys, and you've been married to Janice about 28 years.  It's given you your life back. 

Dr. Weitz:

Without question.  It's our quest for normalcy, and it's a quest for not necessarily curing the disease but successfully managing the disease. 

Andrew Schorr:

Right. 

Dr. Weitz:

Which I think we can now successfully do. 

Andrew Schorr:

Right.  Well, the dream is that if we can't cure cancer, we can make it chronic. And while you've had ups and downs along the way, but the through line is here you are sitting with us, that the hope is we can make cancer chronic, right, if we can't cure it? 

Dr. Weitz:

Without question.  Chronic manageable disease, just like we did with HIV.  When I was a young doctor HIV was a death sentence.  Now I'm an emergency physician I don't even—I can't even remember the last time I saw an HIV patient come into the emergency department and be diagnosed, where we were doing that like 30 years ago.  So now they have a chronic manageable disease and with drugs, so they're not curing the disease, they're not getting rid of the virus, but through medicines they are able to successfully manage the disease.  And that's my hope, because I don't think that I will ever be cured of lung cancer.  My great hope, and I think hope for others, is that we can turn this into a chronic manageable disease. 

Andrew Schorr:

Okay.  One myth I'm going to pose to you, and then we're going to meet our physician and our trial coordinator.  So there are patients who fear, it's broadly around, the fear I will be an experimental guinea pig if I were in a trial.  Did you ever have that fear? 

Dr. Weitz:

I didn't, because I didn't have any choice.  My back was up against the wall, I was running out of options, and so I just had to go with where the science took me, and so I had no hesitations whatsoever. 

Andrew Schorr:

Okay.  Well, let's meet a physician who is actively involved in research.  We're going to go across the country from Michael Weitz to Dr. Lisa Villaruz.  She is at the University of Pittsburgh Medical Center better known if you're from that part of the country as UPMC.  She's at the Hillman Cancer Center.  She's a lung cancer specialist, very involved in trials.  We're talking broadly of course about whatever the cancer is you may be dealing with.  Dr. Villaruz, thanks for joining us. 

Dr. Villaruz:

It's a pleasure, an absolute pleasure. 

Andrew Schorr:

Okay.  So let's just go to that myth we were talking about a second ago about guinea pigs, and also people worry are they not going to get the good stuff, if you will, or are they going to get a sugar pill if it's an oral therapy.  Where are we now with cancer trials now where people can feel confident that they're getting the best medicine has to offer, and if there are other people who—you know, they're randomized, I guess, sometimes is the thinking?  Patient A may even unknown to you may go in one arm of a trial, somebody else may go in another, the concern is well, if I'm going through a trial, will I get good care?  What do you tell people? 

Dr. Villaruz:

Yeah.  So I think the first part of that question and something that I've experienced with a lot of my patients is the feeling that you're being a guinea pig, so this feeling that sure, there's an altruistic component of being part of a clinical trial, you're benefitting the people in the future, but is this really going to help me as a patient?

And one of the things that I like to tell patients is absolutely.  You know, the primary focus of offering a patient a clinical trial at that moment is to ultimately benefit them, and while there's a possibility of benefitting patients in the future, really we're doing that for that particular patient. 

There can be some anxiety associated with that.  You know, when patients seek out clinical trials oftentimes they want the experimental drug.  They want what could potentially be at the forefront of therapy, pushing the envelope so to speak. And what we sometimes encounter in doing larger clinical trials that are what we call randomized, so these are clinical trials where a proportion of patients will be getting the experimental therapy. And then a proportion of patients will be getting what's considered the standard of care, there's an anxiety associated with that.  There's a 50/50 chance I may not even get it, why should I even bother enrolling on the clinical trial?

And one of the things I like to tell patients is that you would absolutely be getting at least the standard of care if not the quote/unquote good stuff, and the reason that we're doing the clinical trial is that there's a hope that the good stuff truly is good. But there's no guarantee, and that's why the clinical trial is being done.  So we have to sort of enter the clinical trial with the hope that the experimental therapy will work but knowing that there's a possibility that standard of care may be just as good and that if there is randomized component to the clinical trial, that the patient sort of has that ability to be comforted that they're getting at least what's considered the standard of care and something potentially better. 

Andrew Schorr:

Right.  Right.  Well, I want to bring into the discussion our third myth buster, and that's near me in San Diego at the Moores Cancer Center at University of California San Diego Health, and that is Sheila Hoff.  And Sheila is a research nurse and works actually with my doctor in chronic lymphocytic leukemia, so I like to give Sheila big hugs.  Sheila, here's a virtual hug.  Thank you for being with us. 

Sheila Hoff:

Thank you, Andrew, for having me. 

Andrew Schorr:

So, Sheila, one of the things that people I think feel good about when you're in a trial—and I bet Michael did, I know I did, I've been in two trials—is the monitoring that goes on to protect safety.  Because if you add an experimental thing not only, you know, you have a dream that it's the good stuff that we're talking but also that if there are questions to be answered or side effects that could be observed or complications, that you all are on top of it.  When somebody's in a trial they're monitored very carefully, aren't they? 

Sheila Hoff:

Yes, definitely.  One of the end points of all studies is safety, making sure that we keep our patients safe.  And I definitely explain to them that it's a team effort.  When you participate in a clinical trial you always have your physician available but you—there is usually—or sometimes an extra nurse like myself in addition to their clinical nurse and then a study coordinator who is on top of it and always checking to see how they're doing.  So I try and encourage them that it's a benefit of participation no matter which arm they may get. 

Andrew Schorr:

I might mention that I was in a second trial, not for a cancer drug but I got a DVT, a deep vein thrombosis in my leg, and it was working with the nurse and then the doctor for that trial that they observed in my blood as they were monitoring it for a blood thinner that I had maybe something else going on that turned out to be a second cancer that I'd been treated successfully for.  So I like the attention. 

Now, one other question for you, Sheila, is some people, whether it's in Pittsburgh or San Diego, people come from a distance, and you have requirements or tests they may have to have or visits, but increasingly now there's some often logistical help with that, isn't there?  Some of the programs have some stipend or something else to help them, is that right? 

Sheila Hoff:

Yes.  Many of the sponsored trials, the ones that are funded by the pharmaceutical companies, do have travel reimbursement.  And we had talked a little bit before about different types of trials, and there is some flexibility with a trial that is run by only our institution, PI-initiated trials, physician-initiated trials, and those tend to have more flexibility as far as scans.  There are many of those where we can have a patient get their scans and blood work done closer to home and then just come to us for the therapy. 

Andrew Schorr:

Okay.  Well, yeah.  That's what we did.  When I was in a trial in Houston, my doctor in Seattle where I lived then there was a lot of faxing and blood test results going back and forth, and that was very helpful. 

So, Michael, so you ended up being in three trials.  But tell me how about how you and Janice worked together maybe with your doctors to find the trials that were right for you. 

Dr. Weitz:

Yeah.  I think that fortunately I had excellent advice, excellent doctors, and what I try to tell everyone who has a life-threatening illness for sure is that you need not only a second opinion but even in some cases a second, third or fourth opinion, and so you get a lay of the landscape and that's how you educate yourself.  So we were very open, very involved in terms of contacting different people throughout the country.  As a physician I'm somewhat, you know, can be connected to something that not everyone can necessarily get, but just being involved is what opened up the door to discovering this. 

And my wife was, Janice, was spectacular.  I mean you couldn't ask anything more.  She was at every single appointment, every single—when lung cancer patients and any cancer patient, when you go to the doctor it's very difficult to remember what the doctor said.  So it's so important to have an advocate that can chronicle your journey, because especially the longer you live the more complicated your story becomes. And you don't know when the last scan was, the last drug test, the last blood level, all those sorts of things, so it's really instrumental.  But it changed her life irrevocably. 

Andrew Schorr:

Dr. Villaruz, so I always encourage, and I know you do, family members to come, and there's a lot to absorb.  But let's say they're sitting across from you and somebody says, well, what about this trial we heard about or what about trials?  And you tell the patient and the family member, well, you know, based on some criteria, that trial isn't right for you, or that trial is just closed. And they have a long face, and you're forlorn.  Talk about the pace of research now and how if one door closes, that doesn't mean that another door won't open.  Dr. Villaruz. 

Dr. Villaruz:

Absolutely.  So you said it.  So one door doesn't necessarily—one door closing doesn't necessarily mean that another door is not going to be open, and I think lung cancer and cancer in general is such a complex field and it's very—it's moving incredibly quickly. 

So as—in the stages of drugs development, as drugs are being developed sometimes the larger what we call Phase III, late-phase confirmatory trials might be closing. And just as those are closing novel therapeutic agents are sort of coming up through the pipelines of these various developmental projects and entering the early phases of clinical trials.  And so just because a larger Phase III clinical trial was closing it doesn't mean that there isn't necessarily going to be an earlier phase clinical trial that might not be an opportunity. 

And then specifically with regards to I think what you're asking in terms of the eligibility for clinical trials, so the eligibility for clinical trials can vary based on the clinical trial, based on the criteria. And I would say that primarily it's to ensure the patient's safety, because a lot of these are still novel, but that's why it's important to make sure that you've done your research.  Ask your doctor questions.  Potentially go on the Internet.  Go to the NCI clinicaltrials.gov website and see what's available to you. 

It can be a very complex landscape to navigate. And that's why I would say that the care partner really is very important, both as that second set of ears in the doctor's office itself but also as a person who's doing a lot of the leg work in the background as your support system and a resource to make sure that you're asking the right questions, to make sure that you sort of have as big a grasp as possible of the landscape. 

Andrew Schorr:

I wanted to mention there are tools that are available and organizations to help you.  So on our patientpower.info website there are if you look at, find clinical trials certainly in the lung cancer area and CLL area, a number of different areas, multiple myeloma. You can put in some information about yourself, and it has this sort of algorithm, if you will, or computer program that helps identify, pulled from that clinicaltrials.gov and help see where you may match up and then even connect you with someone like Sheila at UCSD who is the contact for a trial where you can get her on the phone or maybe some of these programs. Send her an email and say, Mr. Schorr may match up with trial X, Y or Z at your center and wants to ask questions or whatever. 

Sheila, let me ask you, somebody—one myth I think that is around, and I'd love for you to debunk it, is whether or not a trial could go forward with a therapy that's known to be ineffective. 

Sheila Hoff:

Oh, no, that's definitely not the case.  Not only am I a nurse, but I also have been involved with the submission of studies, and every study done in the United States goes through a regulatory process where there's an institutional review board that looks at—their primary goal is the safety of a patient.  So we would never have a trial where we knew that one arm was not efficacious, that they wouldn't help the patient at all.  They often will question trials that have arms that are maybe with weaker drugs and then wouldn't pass them, wouldn't allow the institution to participate in that study. 

Andrew Schorr:

Okay.  And let me ask you, Liza, so this Institutional Review Board with the acronym IRB, you may be an investigator. But let's say at UPMC where you are, there's an IRB or an IRB operating nationally that's watching all this, right? 

Dr. Villaruz:

Absolutely.  So the Institutional Review Board is pretty critical to this process, but also all clinical trials have a data and safety monitoring process which is involved in the trial as well.  To echo Sheila's point, safety is the primary concern.  So if there's ever any concern that there's an adverse safety signal, then that's the type of thing where the trial might be halted, and a bigger picture might be taken of the clinical trial to see whether it's worthwhile to continue. 

And then the converse of that is that, you know, the best case scenario is that you start seeing some pretty extraordinary responses, or you start seeing some extraordinary effectiveness of the trial treatment, and that might be a case where a data and safety monitoring committee might look at the data and actually decide to stop the trial in order to make the trial have are intervention available to all patients including the patients that may have been randomized to the standard of care arm. 

Andrew Schorr:

Right.  And we've seen examples where the FDA where results have been so extraordinary with breakthrough therapies, that drugs have been approved after a Phase II trial rather than moving on to a Phase III.  So I think the whole idea is—I know it is for me, and I know it's happened for you, Michael, is does it give us the chance to get tomorrow's medicine today and also participate in accelerating the process of getting the questions answered, so it can help many other people.  So I feel good about having done it.  I'm sure you do, not just for your own life, Michael, but for helping others. 

Dr. Weitz:

Absolutely.  I think what is happening in cancer is remarkable.  We're on the precipice of amazing new discoveries.  Every year I go to a targeted therapies meeting in Santa Monica, and the drugs that are coming out are incredible. And not only do you have the first?generation drug, now you're getting into the second and third generation drug, and each drug is getting better and better to help you manage this disease. 

Andrew Schorr:

Right.  Now, so let me ask you a question, how you may view this, Michael, is do you view—because you've had some trials you've been in.  As I said, you've been in three, so that presumes that whatever was in the trial for a while kind of you developed a resistance, or it wasn't as effective as it had been. So do you view for now for you being in clinical trials as a bridge to kind of what's next, kind of knocking your cancer back, managing it so that as science moves ahead you're around to take advantage of it? 

Dr. Weitz:

Absolutely.  You said exactly what I tell people is that all these therapies are a bridge to get to the next bridge, and so that's why I'm so encouraged and so hopeful for our cancer community that these bridges are getting more and more frequent and longer and longer. 

Andrew Schorr:

Sent in a question, she said, “Are clinical trials only for patients starting on a new treatment?  How about those already in an FDA-approved therapy plan looking for an improvement in their treatment?”

Dr. Villaruz:

So that's a wonderful question, and what I would say is that we have clinical trials available for patients at every stage of their treatment.  So a proportion of clinical trials will be evaluating patients that are just starting their journey where other clinical trials are looking at or aimed at patients that may have already progressed on their first line of therapy, their second line of therapy, their third line of therapy. 

And I think speaking to Michael's point is that the idea is to turn this into a chronic disease, whatever cancer you have.  You know, so if you weren't eligible or you progressed on your first-line therapy, that's no reason to lose hope, because there could potentially be a clinical trial option for you at that point in your treatment course. 

Andrew Schorr:

So, Sheila, maybe you know the answer to this one.  Susan wrote in with a question, “If a patient is not doing well at a certain point in a clinical trial, can they be removed, or can they stop the trial at that point but then later be brought back to it?”  So in other words, because I sometimes think people have the fear, oh, my God, having this side effect, I don't want to tell them because I don't want to be dropped from the trial.  So how does that work?  

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Page last updated on April 27, 2018