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(Narrator 1)
Major funding for Second Opinion is provided by the Blue Cross and Blue Shield Association. An Association of independent locally owned and community based Blue Cross/Blue Shield Plans, committed to better knowledge for healthier lives.
(clock ticking)
(heart beats)
(music)
(Dr. Peter Salgo)
Welcome to Second Opinion where each week our health care team solves a real medical mystery. When we close this file half an hour from now, you'll not only know the outcome of this week's case, you'll be better able to take charge of your own health care. I'm your host Dr. Peter Salgo and our story today concerns Roger.
Now, you've already met our special guests who are joining our casts of regulars, Primary Care Physician Dr. Lou Papa and Doctor/patient communications expert Kathy Cole Kelley. No one on the team knows this case. So we're going to get right to work, and I'm going tell you about Roger. Roger is 62 years old, and he's been married for, uh, about, um, 30 years. He has 2 grown children. He's a lawyer. He's been very active in his community for many, many years, and, uh, about, uh 20 years ago, Roger awoke with a tremor in his left arm. So he goes to see his primary care doctor, and because it was a tremor, his primary care doctor simply said, "Go to the neurologist".
(Dr. Lou Papa)
Oh, really?
(Peter)
Fortunately, we have one of those on our panel, and he was soon diagnosed with Parkinson's Disease.
Monique, what is Parkinson's Disease?
(Monique)
Well, Parkinson's Disease, it's a neurologic condition, um, in which certain cells in the brain, dopaminergic nerve cell, degenerate. They die over time.
(Peter)
What are dopaminergic nerve cells?
(Monique)
They're nerve cells in the brain that produce the neurotransmitter chemical called dopamine, and it just so happens, in this area of the brain, helps control movement. So with Parkinson's, your predominant symptoms are movement symptoms.
(Peter)
So if you lose the dopamine, you lose the chemical that signals the brain to make motion possible, is that basically the deal here?
(Monique)
Correct, correct.
(Peter)
20 years ago, what did we have to offer the people with Parkinson's disease?
(Monique)
20 years ago, we really didn't have a lot to offer. There was a newly emerging medication, Levodopa, which still is one of our main gold standards for Parkinson's.
(Peter)
Roger was told that there was drug therapy for Parkinson's Disease, but that he might not want to start it, and he didn't start it for about 2 years. Why not?
(Dr. Robert Holloway)
There was some concern that Levodopa may actually worsen the symptoms over time, and,
(Dr. Peter Salgo)
What do you mean --- but I thought it was gonna treat the symptoms?
(Robert)
Well it does.
(Peter)
Why would it worsen?
(Robert)
Well, there could be, uh, for example, some effects that Levodopa has that could hasten... There's theories out there that actually could theoretically could hasten the progression of cell death, and therefore, worsen the symptoms.
(Dr. Monique Giroux)
So that's hard for patients to understand, we have a good medicine, it works well, but is it the one for me, and the patient might be still thinking about this diagnosis and can barely hear anything else, and then ---
(John)
You got that right.
(Monique)
And then ---
(John)
That's all I heard, you've got a disease that's not curable.
(Monique)
Right.
(John)
Okay. I'm thinking, I'm gonna be dead in a few years, which is totally not the case. Parkinson's is not fatal, but all I've heard is incurable. Okay.
(Peter)
But --- but you then had to live, after you got over that initial shock --- how long did it take you, by the way, to get over the initial shock of hearing the dia ---
(John)
Oh, uh, it --- it took me about a minute. I went through the seven stages in about a minute, and I said, "This is the hand I've been dealt in life. I'm gonna play it. I don't have any other choice."
(Peter)
What's it like living with this Parkinson's Disease day by day, and how has it progressed for you over time?
(John)
Uh, day by day, I've got my on periods and off periods. On periods is when the medication is --- is working. Off periods is when it's not working, and I try and get as much done during my on periods as I can because when I'm off, my --- my symptom is mainly a tremor on the right side, which limits me in doing things, and I have trouble walking at times, and it's progressed to --- for me that when I'm, at times, in my off periods, I have to walk with a cane now. Now, yes, it's a progressive disease, but it's also --- we've also called it a designer disease because it progresses differently in --- in --- in everybody, and, like me, for the first 5 years, it was pretty good. It was, I was pretty much under control. I did have my --- my off periods, but after that, it's progressively, uh, grown worse, I'd say, that my off periods are longer, and they're more intense. Like I say, at times, I even have to walk with a cane, and there's depression connected with it. I mean I didn't feel any depression until, maybe 5 years after I was diagnosed, and it just hit me one day at work. I'm sitting working and thinking, "I'm tired of this nonsense of doing --- I'm tired of this disease", and I felt real down, and then my medication started to take effect, and I said to myself, "You idiot, this is a symptom of your disease. You know people that are taking medication for depression. You --- friends of yours in the Parkinson's community --- then I decided, "Okay. Yeah, I'm gonna be depressed at times. So I just have to fight against it, unless I want to get --- want medication, which a lot of people need --- need to --- to control it.
(Peter)
Can we all vote for you as patient of the year?
[laughs]
(Peter)
I mean, you ask a question, you answer it and go on. You don't even make an appointment to see somebody. How has your wife reacted to all of this?
(John)
She took it badly for the first year, but then after a year she said, "Okay. We gotta do something about this. Let's get active in the community, see what we could do to work to --- to get funding for research to --- to get a cure for this disease. We got involved in the local support group.
(Dr. Peter Salgo)
Well, Roger's not unhappy either. I'm sure he is from time to time, but in regard to his --- his Parkinson's medication, it's working. What's going on though, is he needs more and more, and over the, oh 5, 6, 7 years that he's on this medication, it's controlling his symptoms effectively, but the dose of his medication is going up. What is it like for them? We heard this eloquent description of what his life is like, what is like for your patients that you hear from them, living with Parkinson's, emotionally, physically, every way?
(Monique)
I think it's true that patients do go through stages in the beginning that there is the anger and the fear, and the biggest thing I hear over and over again is, "What's going to happen? How's it going to be for me down the road?" But I think it's --- it's the fear of the unknown that is the biggest thing, and then the other thing that John touched upon, which is very difficult, is that on and off fluctuation. People with Parkinson's, they can take their medicine, do very well and move very well, but when that wears off and they turn off and they can no longer move and have those difficulties, that's --- that's very difficult.
(Peter)
You'll be happy to hear, Lou, that Roger's lynchpin in his healthcare team is his primary care physician.
(Lou)
I'm very happy to hear that, and it makes perfect sense. I mean, when a patient's diagnosed with a --- a progressive illness or terminal illness, you're going to be seeing your primary care doc a lot, and especially if you're getting physical therapy involved, you're getting a neurologist involved, other specialists, you really need that care coordination.
(Robert)
Wait a minute. We're talking about primary care doctors and neurologists, you know, Parkinson's patients requires often many more kinds of providers, social work, psychiatrists, physical therapists and a variety of others. So really the care is often complex, needs to be coordinated, and we actually do use primary care physicians as that care coordinator as well.
(Peter)
Well, I'll tell you about Roger just a little bit more. It's now 7, 8, more like 10 years, now that I look at the chart, since he first was diagnosed. He waited 2 years, then was put on his medication, and although they're going up on the dose of the medication, it's having less and less effect, and now, Roger's really getting worried, and he is posing the following question, "What do I do," and I guess he's posing it here, "When, not if, but when I run out of time on this medication?"
(Monique)
Well, I think there's a couple options, and the first thing, have you really tried the appropriate therapies, have you really tried the medicines and --- at --- at the --- the right medicines, the right dosage? Let's say that's been done, there are other options. There are new investigative medicines, clinical trials.
(Peter)
I'll tell you what Roger was offered --- or at least was mentioned to him in this context. Three different things are mentioned here in the chart, one was ablative or destructive surgery, one was deep brain stimulation and one was transplantation surgery or some sort of repopulation surgery, putting cells into his brain. All 3 of these things were offered to him as potentials, and this is a guy with Parkinson's Disease, who's been taking a couple of pills a day, and suddenly, they're talking about taking a drill to his skull. I mean that's a big leap here, isn't it?
(John)
Yeah.
(Peter)
What --- what's going on, was there nothing else to offer or was that the best that people had to offer or is that reasonable therapy?
(Dr. Monique Giroux)
It was appropriate offer surgery, and I think it was appropriate to offer the best that we had available and the current research --- the investigative therapy that looks like it was going to be as good.
(Peter)
You know, how desperate do you have to be to agree to have brain surgery, uh, because you have Parkinson's disease? Would you have considered it if your medicines had stopped working?
(John)
I --- I don't know. I --- that's --- that's --- it's a hard decision.
(Lou)
You have to go into it with your eyes wide open, knowing what --- what precedence there for, what kind of information is that for, is it just kind of one doc that's doing this or is there some consensus in the community that there --- that this is a viable option, and making sure the patient's going in with his or her eyes wide open.
(Dr. Peter Salgo)
Well, let me focus on that ---
(Kathy Cole-Kelly)
That's the --- the challenge.
(Lou)
Right.
(Kathy Cole-Kelly)
The patient going in with their eyes wide open because understanding the complexity of these. We talk about informed consent, and just simple informed consent for any kind of procedure is difficult for lots of patients to read. Now, we're talking about some very difficult choices that require a very deep understanding.
(Peter)
Is somebody facing the terror of chronic disease, which is untreatable, ever in the mental state to be able to give you an informed consent?
(Dr. Glenn McGee)
I think, yes.
(Peter)
Okay.
(Dr. Glenn McGee)
And I think we're beginning to recognize that's true for all kinds of activities, from driving to voting. Uh, the more we think about that, the better understanding we have of their ability to communicate so ---
(Peter)
Let's pause just a moment and sort of recap what we've discussed already. Treatment options for Parkinson's Disease, as well as other chronic diseases, change over time, therapies and research, some standard therapy, maybe unproven. You need to be informed. You need to talk to your doctor and satisfy yourself that what's being offered to you makes sense for you, and this is for therapy now. We're not even talking about research.
I'll tell you a little bit more about Roger. Roger actually agreed to have a thalamotomy. What is a thalamotomy? It's surgery.
(Monique)
A thalamotomy is surgery.
(Peter) Thalamotomy? (laugh)
(Monique)
Right, right. It is surgery, and that's one of the ablative surgery --- ablative surgeries that you talked about earlier, where the surgeon will go into the thalamus, the nucleolus in the brain that's part of the circuitry that controls movement, and make a lesion --- make a cut to try to normalize activity of the circuit. In case of Parkinson's Disease, it's very effective for the tremor.
(Dr. Peter Salgo)
All right. Roger had the surgery. It says here that he had a good result. He was placed on a cocktail of other Parkinson's meds and did well for a while, but then his Parkinson's began to get worse again, and standard therapy is beginning to fail Roger here in the chart. He's worried he's not going to be able to function at home, not being able to --- to function without help, not going to be able to go to work, and what is his life going to be like.
(Monique)
You know, at this point, I --- I suspect since he's --- he's had surgery and with medications, again, he's beginning to have a lot of dysfunction, a lot of more off time, less on time, greater difficulty moving and doing all of his activities of daily living throughout the day.
(Peter)
At this point, his doctor says 2 words, and these 2 words are clinical trial. What's a clinical trial, and what does that have to do with Roger?
(Dr. Robert Holloway)
Well, clinical trial is a form of research, where actually it's an investigation to assess whether or not an intervention actually works.
(Peter)
This includes what, new drugs, old drugs used in new ways ---
(Robert)
It can be ---
(Peter)
--- surgery.
(Robert)
--- actually any kind of medical intervention from drugs, procedures, healthcare programs and surgeries.
(Peter)
When you're dealing with a progressive disease, such as Roger has --- now, Roger's not just panicked, I'm assuming at this point he's feeling helpless, I mean, he --- he took one big risk, had neurosurgery, and it seemed to work for a while, but in the end it didn't pan out. So now, ethically, where are we? Are we dealing with a person who's vulnerable, and will try anything, so he's --- he's just out there, an easy target for people who want to do research?
(Glenn)
Well, there's --- that's certainly true. Roger, as we've described him, is increasingly having trouble making decisions. He's beginning to see problems in terms of his mobility that trig --- you know, it might trigger the family's fears and so on. So when Roger comes to the clinic and says, "You know what" --- and Roger's family, and says, "We're desperate now," it's a real problem for, I think, all of us in society, but especially for doctors doing clinical trials. To make a distinction between helping Roger and doing good science, most research fails. The vast majority of the things you can offer Roger will never make their way to market.
(Peter)
But let me ask you this, which is --- I think you alluded to this, Roger's looking for treatment. Roger's looking for improvement ---
(Glenn)
That's right.
(Dr. Peter Salgo)
A trial is not. A trial is doing something else. It's doing science. What's the distinction?
(Dr. Glenn McGee)
The goal is to improve diagnosis, prevention, and treatment. The pathway is, first, you look to see whether or not a proposed drug or device will hurt someone.
(Peter)
But you design trials?
(Robert)
Yes.
(Peter)
When you design a trial, is the object to make everybody in the trial better?
(Dr. Robert Holloway)
No, I mean, the trial is based on the best sound, scientific evidence to date, that there's some that we believe, based on medical scientific principles, will help.
(Peter)
I want to be very clear. How are you going to measure if patients are getting better? Some patients in your trial will get the new therapy, some won't, and they may not know what side of that group --- what side of that arm in they're in?
(Robert)
That's correct. So you actually often blind them to what they're getting, and then --- meaning that they don't really know what they're getting actually. So it can be, for example, in a placebo controlled trial, where they get a pill that actually contains the intervention and another one that does not, and then you systematically follow them over time with measures, scales that --- that measure this.
(Peter)
But is that what Roger's hearing, and --- and if you were the primary care doctor, is he gonna ask you about that?
(Lou)
He --- he probably would ask me, but I'm very clear when patients enter into this. I say, "You need to go into this thinking that you're going into this to improve the science for others. So it may or may not help you, it could potentially harm you, and you need to go into it, if you're gonna do this, your hope is that, yes, it could help you, but you need to go into it knowing that the --- the majority that --- for the science of it, is that you're doing it for the public good so someone else could benefit from your disease."
(Peter)
John, if someone had told you, "Look, put yourself in Roger's place, if your therapy's not working, but there is a clinical trial we've heard about. You're doctor's involved with it and knows the people doing it. You can take part in this clinical trial, but be aware that you may not get the test drug, you might be in the placebo side or you might have what's called sham surgery. They're going to be doing an operation, half the people will get the real operation, half the people will simply go to sleep, have something done and wakeup and have no surgery." Does that appeal to you at all?
(John)
It doesn't appeal to me personally. I admire people that participate in clinical trials because they're doing this for the benefit of everybody, but it doesn't --- this isn't something that would appeal to me personally, uh, uh, because I've heard the stories, too, about how even if these trials are effective, the drug or treatment might never become put into practice and, uh, so a person goes through this, getting the new thing, feels good, and that's it, you can't get it ---
(Dr. Lou Papa)
And that's the danger right?
(John)
--- it's not FDA approved.
(Lou)
And I've seen that happen with patients.
(Robert)
But that's --- that's the reason why those sham surgeries or placebos are important ---
(Robert)
because there is this placebo effect ---
(Lou)
From the scientific point. But from the patient's point of view --- But that's --- exactly right. That's the problem.
(Dr. Robert Holloway)
And that is the issue.
(Dr. Peter Salgo)
Yeah, there's --- there's a real patient perspective here, right, of "What's in it for me?"
(Dr. Monique Giroux)
Absolutely.
(Lou)
Right.
(Peter)
And I recall in early days of AIDS, when zillions of drugs were out there, some of them made sense, most of them did not. The cry from the AIDS community was, "Give it to us. We're the ones with the disease. We'll take it. We don't care." Who's right here? What's ethical?
(Glenn)
These trials really are pointed at making people better, but the person administering the trial can't think that way. That can't be thinking about improving the patient's health. That's tough. So, you know, in --- in the days of HIV, patients literally stormed the FDA and demanded and got special exceptions. I did sound a lot like the Parkinson's community today, who are saying, "We want," and then name your therapy, stem cell research, fetal cell transplants, et cetera, and they don't appear to work, for example, at the moment, but there are companies that are going to do them any way, and families who are desperate --- now, you ask is that ethical? Uh, I think there's a really open question here about whether or not people should be able to choose the kind of care they want.
(Peter)
It's their life.
(Glenn)
It's their life.
(Peter)
All right. Let me --- as long as we're on this thing, I --- I --- I know that people got stuck when I said sham surgery. I just want to go back to this to be sure we answer this. There is sham surgery, which is ethical, at least in --- in the view of the scientific community, where they have an operation ---and half the people had something done in the operation, the half had an incision, and then it's closed up.
(Robert)
But you have to also understand the alternative of not doing sham, what's the alternative and impact that that may have if you don't do sham surgery or have a placebo controlled trial? Then you may falsely conclude that many therapies that are being studied are actually effective when, in fact, they're ineffective.
(Peter)
Clearly, somebody's got to regulate this, right? There's got to be some oversight, how is that accomplished?
(Robert)
There's a whole host of regulation that regulates research in the national level. Certainly, uh, if --- from the National Institutes of Health, there's actually tight regulations in terms of actually what institutions need to do who perform research. The FDA has its own sets of regulation, that they regulate in terms of actually drug registration in the new drugs that are being developed. So there's a huge regulatory environment that actually is available and --- and needed.
(Peter)
So let's pause for just --- just a moment here and sum up where we are because we've covered a lot ground. I think it's important to realize that clinical trials are essential to developing any new treatments and possible cures. Without clinical trials, there is no progress.
However, I think it's also important to remember that participation in a clinical trial is a selfless act that may help many other people in the future. It is not a guarantee of therapy. It is research, and by its very nature, research is a leap into the unknown. You might be worse, you might be better, and you might get no therapy at all.
So I'll tell you what Roger's doing. Roger got himself into a research protocol for a new drug therapy, and for a while, it says here, his symptoms improved, or least got no worse. However, he then began not to respond to whatever that experimental drug was, and he had just heard, about an article, which he researched on the, web. He read this article, and he took it to his doctor, and he said, "Look at this, 500 pages later, stem cells are the wave of the future. I want them," and his doctor then says the words that Roger didn't want to hear, "You're not a candidate." Here's a guy who wants to be in the trial, understands it's science, says, "Bring it on," and someone says, "You're not candidate," what's that all about?
(Robert)
Well, these are the complexities of actually trying to design a trial. To design trials to try to deliver unambiguous, uh, as unambiguous as possible, truths or sort of viable knowledge, you need to actually have some restrictions to allow for that to happen.
(Dr. Peter Salgo)
But, you know, again, we come up against a buzzword here, and that's stem cell research.
(John)
Yeah, yeah.
(Peter)
Roger was really excited about it.
(John)
It's important to us in the Parkinson's community. It's hope. You mentioned the word hope. That's what we have. I don't --- I don't expect this, uh, the regulations are lifted that anything will come about that'll help me, but it'll help my wife who was diagnosed with ALS, but at least let the scientists do their work. Let them have the opportunity.
(Peter)
What is it about stem cells that's so exciting --- or so potentially so exciting?
(Dr. Robert Holloway)
Well, I think one of the mystiques of stem cells is their ability to differentiate into other cells.
(Peter)
We all begin life as one cell.
(Robert)
Correct.
(Peter)
Or 2 half cells, but let's call it one cell. From that cell, everything else develops.
(Robert)
Right.
(Peter)
That one cell is a stem cell.
(Robert)
That's exactly right.
(Dr. Peter Salgo)
So the theory is what? The theory is if you can find some of those stem cells, they can make new dopamine?
(Dr. Robert Holloway)
And you can regulate that differentiation, you can control it, you can actually create lost dopaminergic or neurons that --- that are lost in Parkinson's Disease.
(Peter)
What are the diseases people talk about with stem cells that might be impacted by stem cell research?
(Lou)
I mean, that's just about every disease you can talk about.
(Monique)
It's almost every --- every single organ system. If you could replenish, replace damaged --- damaged tissue, damaged organs, just think of the power, the potential of that at this point.
(Peter)
So what's the ethical debate around here?
(Dr. Glenn McGee)
The ethical debate, really, ironically, isn't what you'd guess. It's not, are embryos being destroyed so much. It's really about that hope that you were talking about. So when Roger isn't selected for the clinical trial that was described, he's disappointed because he hoped that those stem cells would help him ---
(John)
Yeah.
(Dr. Glenn McGee)
--- and in phase 1 trial of stem cell therapy, every fetal cell transplant, so far, has failed miserably. The real ethical worry is the hope that patients have that's tied up in that politics that this is going to do something now, if we can just put more money into, when, in fact, the best case scenario, 15 years.
(Monique)
Somehow you have to strike some balance. You're absolutely right. We can't just stagnate and say we're going to focus on treatments --- bona fide treatments, we know there's outcome. Somehow we have to advance the science, and we also have to think about something geared towards Parkinson's, will also help many different people, many disorders, the concepts of what's learned. So this is not just Parkinson's we're talking about either.
(Peter)
I'm going to tell you that Roger, being rejected as --- as a non-candidate for this research study, had a deep brain stimulator operation done, and he's doing a little bit better.
Let me take a moment here and sum up a lot of what we've discussed because it's been a --- far ranging discussion over here. There ethical considerations in all clinical trials. Participation can have personal and societal benefits, but it is important that research be done ethically. It's important to understand that it's research. I got to emphasize this again, this is not therapy, and you can get harmed by research, even the best research done in the best possible hands.
Well, I can tell you a little bit about Roger. After his surgery, after his therapy, Roger is doing better. He's getting around with a walker, but he is still self-sufficient. He is ambulatory, and his spirits are good. John, how --- how are you doing?
(John)
I was able to take advantage of an early retirement offer. I retired, 3 years ago, and I've been enjoying retirement, and, you know, enjoying my on periods and struggling through my off periods and, as best as I can.
(Peter)
I want to thank you for being here today. I mean, it's --- it's a sacrifice to come, and to share all of this with us. It's not easy, but thank you so much or being here.
(John)
My pleasure. Thanks for asking me.
(Peter)
We've covered a lot of ground today. Let me just sum up the key things to remember. Treatment options for Parkinson's Disease, as well as for other diseases, change over time, therapies or research, some standard therapy may be unproven. You need to be informed about the state of the art where your disease is and what your choices are.
Well designed clinical trials are essential to developing new treatments and possible cures. Participation in a clinical trial is a selfless act that may help many other people in the future. It is research, it is not therapy, and it is not guaranteed to help you.
There are ethical considerations in all clinical trials. Participation can have personal, societal benefits, but it's important to understand that research needs to be done ethically at all times, and all research has some potential downside to you and to the people around you.
Our final message is this, taking charge of your health means being informed, having honest communication with your doctor. I'm Dr. Peter Salgo, and I'll see you next time for another Second Opinion.
(Narrator 2)
Search for health information and learn more about doctor, patient communication on the Second Opinion web site. The address is pbs.org.
[clock ticking]
[music]
(Narrator 1)
Major funding for Second Opinion is provided by the Blue Cross and Blue Shield Association, and association of independent, locally owned and community based Blue Cross/Blue Shield Plans, committed to better knowledge for healthier lives.
(music)
(Narrator 3)
We are PBS.
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