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How Are Clinical Trials for Multiple Myeloma Structured?

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Published on March 15, 2021

Experts Explain the Structure of Multiple Myeloma Clinical Trials

What is it like to participate in a clinical trial for multiple myeloma? What is the structure, especially during COVID-19, when in-person contact is limited? Do "placebo" treatments really exist? Finally, what clinical trials are currently underway?

In this video replay from our recent "Dinner with the Docs" program on living with multiple myeloma, Dr. James Berenson, MD, Founder and Principal of the Berenson Cancer Center and Dr. Ashkan Lashkari, MD, Hematologist & Oncologist at Wellness Oncology and Hematology share everything you need to know about clinical trials for multiple myeloma in 2021. The discussion is guided by host and patient advocate Jack Aiello.

Support for this series has been provided by Karyopharm Therapeutics. Patient Power maintains complete editorial control and is solely responsible for program content.

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Transcript | How Are Clinical Trials for Multiple Myeloma Structured?

Jack Aiello: Hi, everyone. Welcome to the Dinner with the Docs for Southern California, featuring some local doctors in your area. I'm Jack Aiello. I'm hosting the event. Here with us today are Dr. James Berenson in West Hollywood from the Berenson Cancer Center and Dr. Ashkan Lashkari in West Hills from the Wellness Oncology and Hematology Group. Dr. Berenson, would you please introduce yourself?

Dr. Berenson: Dr. Jim Berenson from the Berenson Cancer Center in West Hollywood. I've been working on myeloma about 40 years. We have four groups that work on it here under one roof. We have a practice that sees 150 myeloma patients a week, and then we have a clinical trials unit that works on trials across the country with sites like Dr. Lashkari's called ONCOtherapeutics. We have a basic lab, a non-profit that does preclinical, that is before it goes into the patient, trying to develop new treatments called the Institute for Myeloma and Bone Cancer Research. And we now have a biotech group called ONCOtracker.

Jack Aiello: And Dr. Lashkari would you introduce yourself please?

Dr. Lashkari: Thank you, Jack. My name is Ashkan Lashkari and I also work in the Southern California area and our practice is called Wellness Oncology and Hematology. We're a group of three physicians. One of my areas of practice and specialty is multiple myeloma. And we have participated in various trials in the past and have participated with Dr. Berenson in the past and currently on some mutual trials.

Jack Aiello:  So now we want to run our third poll, and that is to find out if you have participated ever in a clinical trial. Pretty cool, about a third of the patients have participated in a clinical trial and I think that's really important. So, since my large screen is focused on Dr. Lashkari, I’ll ask you the question about clinical trials. Some patients are afraid to go near them. Without getting specifically in the trials, can you discuss the structure of the trial?

What Is the Structure of a Multiple Myeloma Clinical Trial?

Dr. Lashkari:  Clinical trials, as you mentioned, are designed to achieve many different things, and it really depends on where in development of, let's say it's a drug-related trial you're in. Phase 1 trials are used to assess the safety of a drug. And depending on whether the drug has some activity in one area of cancer or another, or perhaps in maybe several areas of cancer, patients will be allowed to participate depending on that. The goal of a phase 1 trial is to assess the safety of the medication and to assess what type of dose is tolerable. And there is some element of evaluating its efficacy or efficiency, but subsequently we then can step into phase 2 trials, which look at a little bit more specific profile of a patient, perhaps it's more myeloma-specific in this regard.

And we're looking at not just safety once again, which is still very important, but also some elements of efficacy. That's a big portion of phase 2 trials. Phase 3 trials, then generally we'll compare a standard treatment for myeloma and compare them with that standard, for example, plus an investigative therapy. And it's at that point that we'll be able to see without much bias hopefully, whether that drug is helpful, and if it is then attempts are made by the manufacturing company to get that medication approved for use, depending on how it was studied in that trial, in that phase 3 trial.

Jack Aiello:  Some patients hear about sugar pills or placebos. Do I, as a patient ever have to worry about a placebo?

Is it Possible to Receive a “Placebo” Drug in a Clinical Trial?

Dr. Lashkari: I think that's a very common perception, which is, unfortunately, what seems to be presented or in the minds of people, but it's never the case. When patients are on trials, they are meant to be given medication or treatment that will be effective. We would never give somebody treatment that they wouldn't potentially benefit from. And so no, that is not the case, but that is something that's very important to share, to inform them and teach them that the medication that you're on, even if you get placed on, let's say the control treatment of whatever that is, that treatment is still designed to benefit you, to help you.

Jack Aiello: Great. Dr. Berenson, we've had some recent drug approvals as a result of favorable clinical trials. One that I can think of is something called selinexor or Xpovio. There are always two names for every drug. Can you talk a little bit about that drug and how it might be used for relapsed patients?

Recent Drug Approvals for Treating Multiple Myeloma

Dr. Berenson: Yeah. I came up with an analogy from, how that drug works, selinexor, from a show my wife and I have been watching recently. And that drug is called selinexor or Xpovio. And it's a selective inhibitor of nuclear export, which is a mouthful. But basically, it's like the horses are in the corral. And when the horses get up and out of the corral, they run wild. And what this drug selinexor does is prevent the horses from getting out of the corral. And when they stay in there, the myeloma is not able to grow or be active. They actually plug up the nuclear membrane, so those proteins don't go outside of the nucleus into what we call the cytoplasm, the rest of the cell and wreak havoc and cause myeloma to grow. So, this drug is really active now in many combinations from promising early data. The initial approval of the drug was at a fairly hefty twice a week dosing, which had a lot of side effects, but we learned over the last year how to rein it in almost like the horses and at lower doses, we are doing a lot of good work on it.

Currently, we're doing a trial on it with Revlimid (lenalidomide) and early results are quite promising. And it's been recently approved in combination with Velcade (bortezomib) at a lower dose with much more benefit than Velcade alone. So, we think this drug has a very bright future and it will be combined and is being combined with a lot of other agents.

Jack Aiello:  Another couple of drugs that have been recently approved, Dr. Lashkari, are isatuximab or what also goes by Sarclisa as well as Faspro - daratumumab (Darzalex) being given sub-Q. Do you use those in your practice? And can you talk a little bit about those?

Dr. Lashkari: Absolutely, yeah. So, I think one of the other important elements of treatment is how often patients need to come into the office, with what frequency, how long do they need to sit in the chair? And these questions are more important these days as result of COVID and as result of trying to provide some physical distancing between one another. And the case in point is a drug called daratumumab, which has been an exciting advance in multiple myeloma, which has now been around for about five or six years. And it was recently approved in 2020, as a subcutaneous formulation, whereas previously, it was given intravenously and as a result of this being what we call a monoclonal antibody, so it's actually an antibody construct that is designed to target and destroy the CD38 receptor, which is found on myeloma cells that have to be given through an infusion and people could develop reactions from it.

And so, it would have to be given slowly and with pre-medications. Now, we're able to give this same medication, but with a form of technology that allows the medication to sit in the subcutaneous region under the skin of the individual and this medication now can be given in less than a 5-to-10-minute injection. Patients are monitored for a short period of time in the office, and they get up and go. And we've now transitioned all of our patients to this formulation, and it's been fantastic. I do tell people that with the first infusion or with the first injection, they do need to be monitored in the office for a few hours to ensure that there's no adverse reaction, but outside of that, the subsequent visits get shorter to the point where they're in and out of the office in less than 30 minutes.

The other medication that you're referring to is a very similar drug to daratumumab called isatuximab or Sarclisa. And this is a drug that is very similar. It targets a similar receptor, slightly different kind of drug, but a similar target. And it's been approved in conjunction with pomalidomide (Pomalyst) and dexamethasone (Decadron) for relapsed multiple myeloma. And it is very well tolerated and highly efficacious, it is one that we've had the chance to use, but of course, now there's another drug like it called daratumumab or Darzalex that's been around. So, it's a matter of finding the right individual for that. So, we have a lot of tools or lots of toys in the sandbox, so to speak. So, it's a matter of trying to figure out how to fit them together, which is one of the challenges of taking care of patients with multiple myeloma.

Jack Aiello: Again, making it so important that myeloma patients have a chance to work with a myeloma expert, at least being part of their medical team. Dr. Berenson, your horses in the barn represented a different mechanism of action from the way other drugs work. Another different mechanism of action is provided by a newly approved drug called Blenrep, or the formal name is belantamab mafodotin. Can you talk a little bit about that drug?

Dr. Berenson: Yeah. So, we were earlier talking about BCMA and its presence on the myeloma cell and not much on any other cell in the body. So, what this drug does, it uses an antibody kind of like the daratumumab, Faspro, or isatuximab. But in this case, rather than targeting a protein called CD38, it's targeting BCMA. But what they've done in addition, unlike the other, what we call naked antibodies as they have like the 747 with the shovel on top of it, they have a toxin on top of it. So, the toxin is delivered to the service of the myeloma cell, and then it punctures into the cell, the toxin and kills the myeloma. So, this antibody has been used and shown some effectiveness, but unfortunately that toxin gets loose and affects the vision of some patients. And that has been a problem. So, I have not actually used that drug until we can, if you will, reign in those wild horses. In this case, it's the toxicity. And we'll see if they can do that with modifying the dose or schedule of that drug.

Jack Aiello: Dr. Berenson, Dr. Lashkari, thank you both for offering this wonderful education to myeloma patients and their care partners.


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