The Basics of Clinical Trials for Patients with Multiple Myeloma

: Colleen Moretti: Can you give us a clinical trials 101 lesson? Why are they important for patients with multiple myeloma?

Dr. Joseph Mikhael: Absolutely. I mean, this is such a topic that can be confusing to people and mystifying to people. Let me try and simplify it. Clinical trials are an opportunity for us to test and evaluate new drugs and new processes to see if we can help the lives of myeloma patients in their quality of life and in their quantity of life. That's really the goal of all that we do and research to ultimately find a cure for this disease.

Specific clinical trials that the probably the simplest way I can describe it is we have three main categories — what we sometimes call phase one, phase two and phase three trial. A phase one trial is one of the earliest of clinical trials we typically test drugs in the lab. But phase one means we're bringing it to human subjects. And here, we're trying to learn how safe the drug is. Usually, we use small numbers of patients, typically patients that have already seen a lot of other myeloma treatments, and we're testing to see what is really the right dose of this drug. Knowing often that can be obviously very helpful to patients, but we really have to focus on safety. Phase two is when we found what that right dose is in a phase one trial, we now bring it to a larger proportion of patients to really see how helpful this drug is, or the strategy is. We use it in a larger number of patients. We typically give it in a way that we can evaluate how effective is it, how many people is it helping. And then that leads ultimately to a phase three trial, which is a comparative trial between the new strategy or the new drug that we have comparing it to or in addition to the standard of care at the time. So often if we already use drug A, and we know it works well and now we're developing drug B, in the clinical trial, we'll have drug A as one arm and then in the new arm, we'll add drug A and B together. So, both groups of patients get at least the standard of care, so we're not giving patients anything less than that. But in that second group, they may benefit from having the additional drug.

Moretti: What is important for patients with multiple myeloma to know before joining a clinical trial?

Mikhael: One of the most important things about a clinical trial is that patients really understand that they are not just being told what to do, that they have a choice in the matter. We conduct clinical trials under very strict and ethical criteria so that we only provide the best we can to our patients, but that discussion becomes really important for patients understand. Historically people have been mistreated in clinical trials and it's a dark mark on our history of research. But now we've really come to a place where we want to do it in the right way for the benefit of the patient. I think it's important for a patient to know many, many things or lots of things we can discuss, but to really understand what is the purpose of this trial? How could it be helpful to me, how could it be harmful to you, what are the potential side effects that I need to know and understand and how will I be treated throughout this? Well, how many visits is going to take what is going to be involved and then what their potential costs may be — more often than not the costs have anything to do with the trial are covered. But we want to make sure that it's very clear upfront. And a lot of this reflects a good relationship with the healthcare team taking care of them so we can have an open honest discussion.

Moretti: What are some current challenges in this area?

Mikhael: There are always a lot of challenges with clinical trials. One of the good challenges we have in myeloma is that we have so many of them, because we are developing so many new drugs and that's actually a good challenge to have as we evaluate all sorts of different ways to attack this disease. But, as I indicated earlier, there is a history of mistreatment of individuals in clinical trials, and so there is often a lack of trust in the healthcare system and in research and that's why it is so important for us to really openly and transparently discuss what these clinical trials are about. Also, furthermore, we know that in multiple myeloma in particular, there are certain populations that have really not had access to clinical trials as they should have. Myeloma is twice as common in the African American community. In fact, 20% of all individuals in this country with myeloma are of African American descent — and this is personal for me because I am of African American descent. And yet typical representation of clinical trials is around 5% or 6% or 7%. And so that disconnect is a challenge that we have to discuss and deal with openly and find very important ways not just to bring up the numbers but to build the confidence and trust in multiple communities so that people can potentially see the benefit of a trial and opt in or opt out based on their prevalence.

Moretti: What kind of resources can the International Myeloma Foundation (IMF) offer to help make patients more aware and educated on clinical trials?

Mikhael: Well, the IMF has built many resources around clinical trials because this is such a hot button issue and because there are so many trials going on. So indeed at our website myeloma.org or just picking up the phone and calling the info line, our patients can learn about what are the basics of clinical trials, a deeper discussion than I've quickly explained here, what trials may be available near them, or what very specific questions we should ask the healthcare team and what benefits and risks there may be from those individual trials. I think one of the most important things we can do with our patients when it comes to the aspect of clinical trials is to truly empower them to realize that trials can actually have a tremendous impact. We've shown for many years that patients on many of these clinical trials have lived much longer than they would have not on the trials, but we want to set realistic and appropriate expectations and really do it in partnership with the health care team carefully.