When should a NDMM consider a clinical trial as a treatment option?

When should a newly diagnosed patient consider a clinical trial as a treatment option?

I think clinical trials are hugely important as you know, because that's what leads to all the breakthroughs and discoveries and advancement of science. So now, you know, the newly diagnosed myeloma trials are always going to build upon what's already great, right? So we do know these frontline regimens really work well.

We've gone from two drugs to three drugs to now four drugs with, you know, really, really high response rates, high rates of minimal residual disease negativity, and in many, many circumstances, years and years and years of long term remission. Right. So how do we move beyond that? I think we have to start thinking about, you know, different strategies based on the response to therapy.

Right? So if you've had, for example, MRD negative disease and you have, you know, no high risk genetic abnormalities in your bone marrow biopsy, and if you achieve that kind of a response, can we take you off therapy or can we significantly de-escalate therapy. So when we say clinical trial there's so many of them. Right. So you have to think about what question they're trying to answer.

So some of them may be like that where they're trying to actually tailor your therapy based on the risk of disease and response, which I think is always going to give more information. And I encourage patients to participate in trials like that. There's also trials of newly diagnosed myeloma looking at immunotherapies now. Right. That that's kind of been the big breakthrough recently.

And how will we know if those are better than current therapies without studying them in some of these trials. So there's head to head trials with stem cell transplant and Car-T cell therapy, for example. We're looking at these new bispecific antibodies and using them in the newly diagnosed space. So you know I think there's always going to be some unknowns and risk with any clinical trial.

But what I usually tell patients is you're always getting the best. And this is just trying to see if you can beat that. Right. So in that circumstance, you know, I do encourage patients to consider clinical trials. Even with newly diagnosed myeloma, Are first in human trials ever conducted in people with newly diagnosed multiple myeloma. Or are they typically limited to relapsed refractory patients?

So you have to remember, all these studies go through regulatory agencies for approval before we can do anything. Right. So you have to prove that they've been study, they're safe and they're going to have good scientific rationale to be studied alongside or against some of the highly effective therapies of multiple myeloma. So because the field has evolved so much, we have so many new therapies, that bar of getting some of these newly diagnosed trials approved, even or open has gone higher and higher because we have to make sure that they're effective and safe for these patients.

So it's extremely unlikely that first in human trial is going to be studied in newly diagnosed patients going forward. That's really reserved for patients that have had all the other approved therapies and maybe even some experimental therapies before they get to those phase one trials, you know, to determine safety and then ultimately efficacy for some of those compounds.

Similarly, a lot of patients worry about, am I going to get quote unquote, placebo, or am I going to not get the best therapy that's ethically cannot be done because you have to offer patients, you know, what's already effective, as I said before, and then add on top of that. So the role of clinical trials for the newly newly diagnosed patient is a little bit, it's a very interesting question, because in that scenario, what the because for the vast majority, for many of our myeloma patients, we actually have good therapy that we know that will, you know, effectively control their myeloma, or actually shrink it down to the point where they just have barely detectable disease or they go into a complete remission. But now we know that if we just stop at that point, whatever microscopic amounts of myeloma they have will come back. So the issue is that, well, all right, you got pretty good therapy for a newly diagnosed myeloma. You can put them into complete remission.

Many of them. But then arises this question, especially in the newly diagnosed, patients, is can you cure them? And I think that there are actually a lot of clinical trials that are not saying, okay, let's not use the drugs that we know are effective, already in, newly diagnosed patients. But can we combine those drugs with other drugs to actually show that we can actually cure people?

So I think in the newly diagnosed space for those patients, the question is not do you have something that works better? And we do test those things, and ask those questions. Does something that works in a relapsed refractory patient, will it work better in a newly diagnosed patient and add to our armamentarium of, what we treat newly diagnosed patients with?

But I think a real question in the newly diagnosed patients is can we cure them? So can we actually do this set of therapies? And they won't have myeloma in five, ten, 15 years? I think all newly diagnosed patients should consider a clinical trial if it's appropriate, if it's available and it's appropriate. Our center, I think a lot of centers are very selective in what trials they have open.

We try to open trials that are appropriate. We try to fit patients who are appropriate. It is I would say it's as important that a patient picks the right trial or the doctor picks the right trial. Then just going on any trial, there are trials that aren't going to answer any questions. For example, some phase three trials where the comparator arm is not effective.

So there's no equipoise there, there's no... You want to put people on a randomized trial where they have equal chance of good response in both. You don't want to put them on a trial where one is clearly going to be the winner, and the other one is clearly going to be the loser. You don't want them to be randomized to the lose your arm.

So you have to be careful. And but I think all patients should be considered for clinical trials. And that's why the bigger, better centers have multiple trials available. Ever wanted to join a clinical trial but didn't know where to start? When you connect your medical records to your HealthTree account, you're shown the clinical trials that are tailored to you, and you can connect with a patient navigator to get you started, visit HealthTree.org today.