Refining Modern Transplant Therapy: Current Studies in Myeloma

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When considering clinical trials, many patients are concerned about being assigned to the placebo arm of the study, missing out on the supposed benefits of new therapies. When it comes to the DETERMINATION trial, a new, phase III, multi-center study examining the timing of stem cell transplant for multiple myeloma, patients can expect to receive state-of-the-art treatment and no placebo arm. As a patient advocate living with myeloma, Jim Omel describes that patients today “…owe a deep amount of gratitude to the patients that went before us and we need to pass that onto the patients that come after us by joining trials.” A lead researcher in the study, Dr. Paul Richardson, of the Dana Farber Cancer Institute, reassures individuals considering participating that they can expect flexibility, compassion and outstanding care in partnership with a leading researcher.

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Please remember the opinions expressed on Patient Power are not necessarily the views 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:

Hello and welcome to Patient Power. I’m Andrew Schorr. We are delighted to be joined by two experts in multiple myeloma, one who came by it through a diagnosis of myeloma, Dr. Jim Omel, who’s from Nebraska, and is also a physician.

And alongside him is Dr. Paul Richardson, who many of us know from the myeloma center at the Dana-Farber Cancer Institute in Boston. Gentlemen, thank you for being with us.

Dr. Omel:

Thank you.

Dr. Richardson:

It’s our pleasure, thank you very much, Andrew.

Andrew Schorr:

Jim, let’s start with you. You’ve been so active as a patient advocate. We’re trying to answer questions now about treating myeloma appropriately in what we might consider younger patients, and there’s an important trial going on about that.

Tell us, in your perspective, what’s important about this trial and also why patients might want to consider participating.

Dr. Omel:

Well, first of all, I’d like to really thank Paul and all of the researchers that put time and effort into developing these trials. I’ve been working in cancer research advocacy for about 14 years, and I’m just astounded at the time and effort it takes to run trials like this.

I think this particular trial is really important because it’s going to answer the most ultimate question that we have for new patients. When I was diagnosed, the axiom of treatment was if you’re able to get a transplant, plan on it and do it.

The exciting thing about the trial is that it just will give us that ultimate answer that we’ve been looking for. When Dr. Richardson sits and deals with patients, new patients, they’re frightened, they don’t know what myeloma is, they don’t know what treatments they should have.

And one thing I would suggest to those patients is to take the time to educate themselves. Myeloma is a cancer that you really need to think of as a marathon, not a sprint. And sometimes the treatment decisions you make right at the first can influence later decisions, what you can do, what you can’t do later on.

So when your physician considers the first treatment, listen to him or her, think about it, and get some time to educate yourself, even a week or two. You can learn things from the IMF, the MMRF, there are many sources of myeloma education.

And one of the first things you’ll learn is the importance of clinical trials. Clinical trials have brought us these novel agents. If we didn’t have patients that agreed to those trials, we would not have bortezomib (Velcade). We wouldn’t have lenalidomide (Revlimid) and carpilzomib (Kyprolis) and thank goodness for pomalidomide (Pomalyst).

And we owe a certain amount, a deep amount of gratitude to the patients that went before us, and we need to pass that on to the patients that come after us by joining trials—a trial like this.

You’re going to get Revlimid, Velcade, dexamethasone; you’re going to get them. Whether you get them on trial or off trial, think about how important it is to add your treatment experience to all of the clinical data that we have. Your treatment can actually help further our knowledge of myeloma.

The thing I like also about this trial is the quality of the arms. Dr. Richardson will certainly explain it in depth, much better than I can, but when we think of trials so many patients are concerned about perhaps getting a placebo arm. There is no placebo with this trial.

Essentially, every patient gets the same treatment. It’s just the timing of the treatments. Does your treatment involve stem cell transplant early or perhaps later, after you’ve relapsed? And I’m afraid to tell you that you will most likely relapse.

Remember, I said that that myeloma is a long-term disease, and so almost everyone on this trial is going to get treatment from both aspects of stem cell transplantation. So again, I would plead with you to add your treatment experience to the body of knowledge that we have to make these clinical trials more effective for everyone.

Andrew Schorr:

Thank you Jim. Dr. Paul Richardson, help us understand this trial, what it is and what question it’s hoping to answer?

Dr. Richardson:

Essentially, the clinical trial seeks to ask the critical question as to where does transplant best belong in terms of timing in the era of novel therapies, and also very importantly, what is the role of maintenance, be it in the non-transplant or transplant setting in controlling disease for as long as possible.

And a critical aspect of this trial is that lenalidomide, as a maintenance strategy, is continued until progression, so to Jim’s point, treatment is continuous in both arms. The important point is not only timing but also the fact that the best therapies are made available to patients in both treatment arms.

So we really feel that this study has what we call equipoise, because it provides the very best to both groups of patients, the critical question simply is the timing. So we start with our combination of lenalidomide, bortezomib and dexamethasone. This is a proteasome inhibitor plus an IMiD.

It’s truly the Army, Navy and Air Force of therapeutics in upfront treatment of myeloma. It works synergistically. and it has response rates approaching 100 percent. When that has achieved its response after three cycles, patients then undergo stem cell collection regardless of which treatment arm they’re assigned to.

And then they go forward either with the transplant if they’re assigned to Arm B, or the transplant is kept in reserve for Arm A. For Arm A, after stem cell collection has been successfully achieved, again using a chemotherapy called cyclophosphamide; patients go on to another five cycles of RVD, followed then by lenalidomide maintenance.

Concurrently, in both arms, there’s phosphonate in the form of a drug called zoledronic acid, is administered every month. Now, for patients who go onto transplantation, as opposed to having the five cycles of RVD after stem cell mobilization, they undergo autologous stem cell transplantation.

And then, they receive two cycles of RVD after the transplant to further improve on the quality and depth of response that they may have achieved, again then, followed by maintenance.

Now, a critical point for patients to appreciate is that whichever treatment arm they’re assigned to, if, for any reason there is evidence that they would benefit from a different approach—be it transplant sooner or later—or if there’s a safety concern, those absolutely will be followed.

There is real flexibility between the two arms and very, very importantly as well, as patients are assigned to treatment arms, there is a stratification based upon their risk profile.

And this gets to a critical point that Jim made, which is that one of the central parts of the study is to best understand who benefits from what and when.

And so a critical construct in this study is that one size does not fit all and therefore, as we go forward, we need to better understand who, for example, can wait on their stem cell transplant, who in fact needs it earlier and, similarly, what genetic features of their cancer mean that therapy should be given in any particular order.

This is unique to this study and represents a sort of first in class in terms of the study going forward. What’s another piece to recognize is that this is an international effort.

The U.S. trial, which is 660 patients strong, runs in parallel to a European effort, led by our colleagues in the Intergroupe Francophone Myelome, which is the French myeloma group, the IFM, where they’re doing exactly the same thing but critically, their maintenance is not continuing until progression for various reasons that are relatively unique to the European environment.

The lenalidomide will be stopped after one year in those patients, and we will, therefore, have a critical opportunity to understand between the two studies who benefits from either longer maintenance versus less, and I think that’s a tremendously important point.

We’re very grateful to our pharma partners for providing not only free medications but also support for the study so they can be pursued in the way that we described.

And finally, another critical point, by receiving their care at expert myeloma centers, there is expert management of side effects and expert management of what we call the therapeutic index, which means that they get the best treatment possible through participation in the study.

Andrew Schorr:

Dr. Richardson, to hear you tell it, then, it sounds like patients can get great care for themselves and really make a difference in helping improve the understanding of myeloma?

Dr. Richardson:

Exactly, and just to explain the framework, Andrew, in which this trial is being performed, it’s an investigator-sponsored trial, so it’s run by investigators in the field. It’s been approved by the Food and Drug Administration.

It’s been supported by the National Cancer Institute, who is supporting critical funding for correlates and special science in the samples that are derived from the study. These are from patients’ tumors that are collected at the time of when any bone marrow testing is done.

And very, very importantly, we have the endorsement of the Alliance, which is the former CALGB, which is a fantastic national organization. And we’re particularly grateful and very, very proud, actually, to be endorsed by the clinical trials network of the ASBMT, the so-called CTN.

So this represents, for the United States, a unique effort to conduct a multi-center, randomized trial answering a critical question and also showing to our international partners in myeloma research that we can work together to help patients not just in the United States but globally as well.

Andrew Schorr:

Well, I would like to thank both of you, Jim Omel and Dr. Paul Richardson, for being with us and your devotion to patients and helping us, hopefully someday, not only have improved treatments continuing, but maybe, one day, a cure. Thank you both so much for being with us.

Dr. Richardson:

Really a pleasure, Andrew, thank you.

Dr. Omel:

Thank you, Andrew.

Andrew Schorr:

I’m Andrew Schorr. Remember, knowledge can be the best medicine of all.

Please remember the opinions expressed on Patient Power are not necessarily the views 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.

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Page last updated on April 17, 2014