Clinical Trials for Liver Cancer Patients
Guest: Dr. Melanie Thomas – Hollings Cancer Center
Host: Dr. Linda Austin – Psychiatrist, Medical University of South Carolina
Dr. Linda Austin: I’m Dr. Linda Austin. I’m interviewing Dr. Melanie Thomas, who is Associate Professor of Medicine, and an expert in the area of gastroenterology, in particular, liver disease and liver cancer. Dr. Thomas, in this podcast, let’s talk about participation in clinical trials, especially for patients with a diagnosis of cancer. In the United States, I understand, actually, unfortunately, for progress of research and finding cures for cancers, participation rates are pretty low, aren’t they?
Dr. Melanie Thomas: Yes. You know, I think most people think that almost every patient goes on a clinical trial when, in fact, across the country, it’s only four percent of patients who receive a diagnosis of cancer ever participate in a clinical trial. And yet, clinical trials are the only way we find new treatments for their disease and get them FDA approved. So, there’s lots of treatment out there but, yes, a very small percent actually participate in trials.
Dr. Linda Austin: Now, there must be reasons for that. People must have concerns or fears. What are some of the common reasons that you hear for patients being unwilling to participate?
Dr. Melanie Thomas: Sure. So, probably, the biggest thing is patients will say, or people will say, well, I don’t want to be experimented on; I don’t want to be a guinea pig. And, yes, these are investigational studies, but the whole clinical trial process, it’s very structured. It’s very organized. It’s all done under the oversight of the university and the Institutional Review Board, and patient safety, and patient privacy, are number one. You know, they’re number one, two, and three. That’s important for patients to realize. There are lots of other groups involved in making sure that patients’ rights are attended to. So, safety is always number one. I mean, we’re trying to find effective therapies, but we have to pay the most attention to make sure that they’re safe.
Dr. Linda Austin: And, I would underline something about that. You know, this is an era when you hear a lot of stories about medical errors, or doctors and nurses being overworked, that sort of thing, or patients feeling like they’re just not getting enough time, or not enough attention, and one good reason to participate in a clinical trial is, if there’s ever a setting where it’s least likely for there to be errors, it’s in a clinical trial. Every i is dotted, every t is crossed.
Dr. Melanie Thomas: Right. Actually, that’s a great point. They get the royal treatment. Every study has requirements; a weekly phone call. There’s a lot of contact with the patient; more clinic visits than normal. In some ways, a trial is more work for a patient, but it’s work where we’re being vigilant; watching over them.
The staff that takes care of patients, coordinators, nurses, certainly the investigators, that’s our passion, to do clinical trials. We really take extra special care of any patient who participates in a clinical trial because we know they’re doing more work, and they’re looking beyond themselves. That, actually, is kind of a remarkable thing I’ve seen in the patients who participate in trials. It’s independent of whatever stage of the disease they’re in, whether this is the first treatment they’re undergoing, or whether it’s the fifteenth. There seems to be a common theme that they know they’re helping; they’re going to contribute to helping other people, and that makes us feel good as well.
Dr. Linda Austin: Another concern that I’ve heard patients talk about is the fear that they will get placebo, that they will not get treatment at all. What is the reality of that?
Dr. Melanie Thomas: Trials are done in a kind of systematic way. Phase I trials, everyone’s getting the drug. These are for new investigational agents, not approved yet by the FDA. They’ve been used in individuals before, healthy volunteers, and the doses are very low. Principally, we’re paying attention to safety. We’re not really guaranteeing any kind of benefit, but looking for safety. Those are usually trials for patients who’ve already exhausted all their other treatment options. So, that’s a Phase I.
Phase IIs are, now you know the dose of the particular drug, or the combination. All the patients in a Phase II trial would be getting the investigational drugs; you’re looking for the effect. There are usually between 40 and 60 patients in a Phase II study, so there’s no comparison. In a Phase II trial, now you’re saying, we want to see if this is going to work a little bit better than what we currently use for that particular disease. We call that a signal, a biologic signal; something that gets us excited.
Where placebo comes into play is the next step, which is a Phase III. With those, there’s always a comparator. So, one group of patients will get, usually, the current standard treatment, and the other group of patients will get, generally, that standard plus something else, plus the new drug, or the new combinations, and those are usually blinded. Neither the doctor nor patient knows which group they’re in. The reason for blinding is to take out any kind of bias, either way, so we treat every patient the same. Placebo is a sugar pill; you hear of it being a sugar pill, so it’s an inactive drug.
Placebo trials are actually very uncommon now in this country, where patients would be getting placebo alone. Most trials, they would be getting some sort of standard therapy, so, let’s say two drugs, and they might get placebo as the third drug. But they are still getting active treatment. And then the other group of patients would be getting, again, standard care and, now, the new investigational drug. So, the misconception that patients will often have when they hear placebo is that they might get that sugar pill, and no other treatment, and that’s very rare because most diseases have some sort of standard therapy.
Dr. Linda Austin: What kind of experience do most patients have who have participated in a clinical trial? What are some of the comments you hear?
Dr. Melanie Thomas: You know, I can only comment on my own experience. I think, probably, a lot of it is how much attention you give to them. Patients who participate in trials are special. I think every patient I’ve ever had feels that it was a very positive experience. If they have bad side effects, we catch them early because we are paying such close attention to them. Every patient I’ve ever had, even if they didn’t get that positive result we were seeking, and they may have had to come off the study earlier than we had hoped for, I’ve never heard one say that they regretted it, or that they thought they let another option go because they participated in the trial. A lot of that comes back to: did you discuss it thoroughly with them in the beginning? We call that consent. You know, they have to give informed consent, and, for trials, that’s a very important process. They really understand what they’re getting and what they’re, perhaps, giving up. If you do that well upfront, I think all the patients are going to feel good about their participation in the trial.
Dr. Linda Austin: I’m reminded, as you say, of a quote by Kierkegaard: you live your life forward, and you understand it backwards. Somebody who has cancer, or a loved one with cancer, has to live their life forward and make decisions. But then, afterwards, they have time to think about the decisions that they’ve made. And if you participate in a clinical trial, regardless of how it turns out, you can say, well, gee, we did everything we could: we tried to make the most of that experience, and we tried to give back and contribute, and make some kind of sense, and even some spiritual sense, out of giving back to others with the same illness by joining with the doctors and healthcare team to try to understand this illness better.
Dr. Melanie Thomas: Yes. That’s, again, that theme across patients who go on clinical trials. Patients, when they’ve been given a diagnosis of cancer, obviously, it’s very scary. Many things come along with that. But, take a deep breath, take some time, a few days, a couple of weeks, to really look around for your options. You know, now, with the internet, there’s so much information there. You shouldn’t make a quick decision, an immediate decision, unless you’re in a situation that’s an emergency, which is not very common, actually, in cancer. But, really, take some time. Use your resources of family, and so forth, and dig around to see what your options are, either where you live or some place else, ask. So, really, do your due diligence upfront.
Dr. Linda Austin: Now, let’s talk about that a little bit more, though. The internet is a wonderful thing, but there’s a lot of bad information too. I mean, there’s fantastic information, and there’s misinformation. What are some trusted sources on the internet, and what should you stay away from?
Dr. Melanie Thomas: I think the best website for clinical trials is: www.clinicaltrials.gov.
Dr. Linda Austin: g, o, v?
Dr. Melanie Thomas: That’s right: g, o, v. Obviously, I use it for cancer trials, but it is thee national repository for clinical trial information, and it’s very easy to use. You know, the little search box, you can put in liver, heart disease, whatever it is, and it will take you to trials. You can sort all over the country. It’s very user-friendly. The information on that website is fairly up to date.
One of the features of clinical trials is that their status can change from one day to the next. So, if yesterday there were three openings left in a clinical trial, today, there might not be. The website information is only step one. Then, they really need to be calling those places to see: is this trial still open? How do I get in? And so forth. So, the website is really just step one. And I know that part can be daunting for patients. They see 35 trials in their particular type of lung cancer, let’s say, and then they don’t know how to go the next step. The best thing I’ve found, really, for that is, hopefully, they’re with an oncologist, someone who will spend the time helping them go through that process.
I guess I would say that the next best resource, after the clinicaltrials.gov, would be the disease-specific websites. For example, one that comes to mind is one for pancreatic cancer; there are a couple for pancreatic cancer. There’s a group called PanCAN. They have a very good website that’s geared to patients. They actually have a service, sort of a clinical trial concierge service, if you will, that came about for the reasons I mentioned. People can find information, but how do they go the next step? Pancreas cancer has good ones.
Melanoma has good ones. There’s one called National Melanoma Alliance. People can actually pick up the phone and call for assistance with the myriad trials. Breast, I’m sure, there’s a lot of information. I think, in breast cancer, there’s so much information that it can be difficult for patients. Another one is Leukemia & Lymphoma Society, for that group of diseases. If I had someone who I couldn’t help, that’s what I would recommend. Use clinicaltrials.gov first, see what’s around, see where, and then tap into the disease-specific things.
Dr. Linda Austin: Dr. Thomas, thank you so much.
Dr. Melanie Thomas: You’re welcome.