Understanding Clinical Trials

They do a lot of similar things, you know. With the clinical trial, they asked for—some things are a little bit different. I’ll have to see another neurologist, because it has to be like two—my main neurologist and then a different one will have—that doesn’t like, really take care of me full time, has to also perform a neurological exam. And so, if it’s just me and her, you know, we don’t obviously do that. But other than that, yeah, I mean a lot of similarities. They ask the same, pretty much the same questions.

How do you kind of think about-- it seems like a lot of your treatment you've gotten through these clinical trials. How do you think about the difference between a clinical trial and your regular care that you get with your neurologist?

Well, the clinical trials, it was pretty straightforward, and you had to do it exactly the same every time. So, like there was one big snowstorm the one time when I was supposed to go up there, and I said, can I get it done here? They said, wish you could but you can't. You have to come up here, we have to draw it in our special tubes and then we package it up and mail it to a specific lab. That's it, it can't be done any other way. So, in that regard, it's different. Whereas your private care, if there was something happens, sometimes they'll make arrangements. Oh, yeah, you can go to the clinic just up the street from you and get it drawn and we'll get it from there. That would be the biggest difference, I think, because the labs, in particular, have to be done exactly the same. They don't want any difference.

Parallel to the clinical trial. The patient will have access to the-, his or her original neurologist. In some cases, it could be the same person that’s actually responsible for the clinical trial at the medical center. So, there are two ways that it could actually go. A patient could just participate in the clinical trial and just report back to the clinical trials officer how they’re doing.

At the same time, they can still have their treating neurologist if that’s a separate person. But that person can’t be giving them new MS drugs, for example, and any medical management that might occur. I would have to know about it, or the principal investigator of the clinical trial would have to know about it. But in our medical center, for example, I’m often the treating physician and the-, and the principal investigator.

Now, with respect to this particular trial, there is this type of lesion in the brain, white cells, that is not responsive to that DMT. So, there's activity. There's MS activity happening in your brain even when you're taking these disease modifying therapies, or at least the one that I'm taking. And I don't think there's any others that would stop it. And so, we are testing with the drug, with thinking that it could actually stop the activity, that is not stopped by other DMTs.

I'm so excited that I'm participating in my first clinical trial, actually. And it is, it's, it’s related to this brain tumor. It is a glyburide trial, and it is to assess whether or not you can use glyburide in a non-diabetic patient and effectively control swelling in lieu of steroid use. So, I'll basically get to play the part of a diabetic patient doing the finger sticks and checking my sugar to make sure that I'm not dropping out while I'm using glyburide to prevent swelling secondary to that stereotactic radiation procedure.

Dr. Chris Luzzio: To bring-, to determine whether or not a drug is efficacious and safe for human use it has to go through various phases of experimentation. First, it will go through an animal phase to determine, for example, does it have any efficacy in the-, in the animal models?

Dr. Ellen Mowry: The phases relate to, you know, first in human for early phase kinds of studies, where you’re testing out a new intervention that maybe hasn’t been tested in humans at all, or in humans, with the condition of interest. And then, the next phase, phase two, is really looking in a small group of individuals. Typically, in a randomized fashion, whether there’s any signal for or hope, basically, that yeah, this treatment might really have some potential for benefit, but this group is too small, maybe not representative enough.

Dr. Chris Luzzio: So, in the earlier phases, we determine the doses of the medication that was safe for humans. We determine whether or not it’s it has some efficacy, and is there a justification for a much larger clinical trial? And most all of my patients participate in phase three clinical trials, which are hundreds of patients. And-, by that point it’s becoming clear that the drug probably has some efficacy; we just don’t know how much and-, and that’s what the purpose of the phase three clinical trial is to determine is how much efficacy it has to justify going to market.

It was not a safety trial, which is often times what phase 1 trials are, because they already knew, in healthy individuals, it was a fairly safe drug because it had been used in a different autoimmune disease. What they didn't know was whether or not it was going to make MS worse. And so, so I signed up for that.

There, you know, wasn't really a group-assignment thing here because it was, again, it was a weird trial, and it was a – it was a test run for a bigger trial. So, it was a phase 1a kind of thing, and so there wasn't a control group.

I don't know if I would've partaken in the first stage. It was further down so- well, I don't know that I would not have partaken in the first stage, but stage– stage one is really, really starting and so you're really guinea pigging a lot of people. But by the time you're down into stage two and stage three, you know, it's different because they've isolated some things.

I don't know, though, if I would have done the first stage where they're just testing out things.

As a patient, I want to, “Try me! Try me! Pick me!” Because if you get more people like me that can participate in a trial, then maybe you can get a new drug approved quicker or a new modality approved quicker. Because that's the biggest thing for us as patients is we see all these amazing trials and drugs in the pipeline, and they're years out before we can touch them. So, for a metastatic patient, I don't know if I have seven years to wait for the FDA to clear treatment on a new drug that's still in trial. I'm glad that there's a Right to Trial legislation in some states for you to be able to do so. And we, actually, do have that here. But I still don't even know, if I were in such a position, what it would take for me to get access to those meds or those treatments so.

Just the greatest expert in cancer and genetics, what would you ask her?

Well, what's, what’s the latest docket, and what are the ready, what is in clinical trials, I guess? Because that's the thing, another thing the general public doesn't really know. For a good reason, because I don't exactly know the percentage of how many treatments or drugs go to the clinical trials and then never end up being available. But I go in, when I worked in cancer and biology, I was, I going to conferences and talks. And you always hear about that, and I felt, always, very optimistic, that if there is so much that's happening, at least a small percentage will go through. And the percentage is small, I know that, but this, it means that it’s work-, you know, all those brains of very talented people, they are all engaged, and eventually something will come out of it. And so I would be interested to know what's there, what.

So, I always look at the information, which is probably why I was interested in the different studies. And I don't believe every study that's out there. I think you need to look at the information, look at how it's presented, look at how many people were in the study. And is the information that they're reporting the same as what they were actually testing for is another tidbit. Also, when I said earlier on my husband and I just went through the different drugs at the time and picked the one with the least amount of side effects, well, then you really start to get information on the studies and the side effects. And just because a side effect is listed doesn't mean that 100% of those people had it. It could have been one person out of 20,000 people, but they were in the study. And now, it is a side effect, because that person may have had it even if it wasn't actually due to the drug itself.

Mhm. Mhm. So if the, the world's expert came through the door, that was an expert in your kind of cancer, what would you ask her or him?

I would ask them, is it worth doing all that I'm doing with these clinical trials and everything? Or should I just, or just should I just, just believe in my God and that I will be healed? Or am just wasting time with all this, with everything that I'm doing now? Is, I don't know if I'm, just waiting or something. Is it, is it worth all that I'm doing with this, you know? Is it worth it? I know it's worth it to me, but realistically. Because I believe in God. And if he were to tell me that, that I go off that. And I only have like, she told me I only one more, maybe one more. Because I'm building a resistance to chemo.

I mean, for me, seeing the difference of what my life has been like on Tysabri, and I mean even on Rebif, compared to the other people, the older people in my support group, and the sort of traditional sense of what MS means, it's incredibly clear that science is completely-- like, science gets a lot of credit for the fact that I can live a mostly normal life now. I think if I had gotten diagnosed 10 years ago, my life would be very different because the drugs available would have been very different. And so, it's really clear to me that I have a moral obligation to support any sort of science in MS because I'm benefiting from that work already.

I know that there's so much we don't know about MS. There's so much we don't know about the central nervous system that research is important, but I am probably not educated enough to know why it's important and what we're looking for. And I know any time I participate in a study or a trial or something, the only benefit that I would like to get out of it is, when you publish, please send me a copy, because I want to know what the findings are. I want to know, what did you learn? You spent all this time and you've paid your team of researchers and you put all this work into it. What did you learn? And I'm hoping one day someone says, “Well, we learned that this cures MS. We learned that this re-myelinates the spinal cord.” That's what I'm hoping for one day, hopefully. Back when I was diagnosed, I was told we'll have a cure in five to six years. And now they're still saying five to six years.

I think of it as I'm on the medication that I'm in, and my doctor's able to provide me the dietary and physical support that he can because of people that have already participated in clinical trials before me.

A lot of these trials are helpful to people outside of just MS. I'm proud to be a part of that. I appreciate the people that did it before me, and I appreciate the people that will continue to do it after me. That’s kind of my thought process on it.

And there's so much great things happening in research. You just have to be optimistic that those answers are coming. It's not on my timeline. I'm very impatient, but they're coming.