Published on August 9, 2021
How Can Myeloma Patients Access CAR T-Cell Therapy?
Now that CAR T-cell therapy has been approved for individuals with relapsed multiple myeloma, how accessible is it? In this Ask the Experts segment, Patient Power co-founder Andrew Schorr is joined by Nina Shah, MD, of the UCSF Helen Diller Family Comprehensive Cancer Center, to explain what this approval means for patients moving forward, how this treatment can be scaled, and when (or if) CART will be approved as a frontline therapy.
Support for this series has been provided by Janssen Oncology. Patient Power maintains complete editorial control and is solely responsible for program content.
Transcript | How Accessible is CAR T-Cell Therapy?
Andrew Schorr: Hello, and welcome to Patient Power. I'm Andrew Schorr. We're talking about, really, a new development in multiple myeloma, and we have a leading expert with us. Dr. Nina Shah from the UCSF Helen Diller Family Comprehensive Cancer Center. I hope I got it right. Dr. Shah, welcome back to Patient Power.
Dr. Shah: Thanks so much for having me. Always a pleasure to be here.
Andrew Schorr: So Dr. Shah, patients have been hearing about CAR T-cell as an approach, an approved approach, and there may be others coming for multiple myeloma. First, I want to put it in context. You have so many medicines now for multiple myeloma. Is CART going to be something that most myeloma patients either now, or maybe if it's moved earlier in treatment, will have some personal experience with?
Will CAR T-Cell Therapy Be Accessible to All Myeloma Patients?
Dr. Shah: I think that there's a very good chance that CAR T-cell, or chimeric antigen receptor T-cell therapy, is going to become more and more a part of the myeloma treatment landscape. And that will depend, first of all, on our initial experience now, but also will depend on scaling up and ramping up the production capability of this therapy. As you mentioned that we could bring it to people in earlier lines of therapy, not only the multiply relapsed and refractory myeloma patients.
Andrew Schorr: Okay. Now Let's just make sure everybody understands what we're talking about. You extract T cells, part of the immune system from a patient, and you basically make a drug with it. In the lab, it takes a few weeks and then infuse it back into the patient to go after the myeloma cells that were not killed earlier, right?
Dr. Shah: Correct.
Andrew Schorr: Right?
Dr. Shah: Yeah.
Andrew Schorr: Okay. How long does that last for? You at UCSF now are getting more and more experience with it. Somebody says, "Well, if I do this, will it be durable?"
Dr. Shah: That's a great question and the only data that we can point to is the data that we have from matured trials. And in those trials, people were very heavily pretreated. And so right now, the data that we have first from the Ide-cel or Abecma (idecabtagene vicleucel) trial, which was the KarMMa trial, shows that patients who were very heavily pretreated, like six prior lines of therapy, those patients at the top dose got about a year's worth of time without any other therapy, which doesn't seem like a lot to any one person. But if you're a myeloma patient, going a year without getting any additional therapy is something important.
Now we're seeing a little bit more mature data from Cilta-cel (ciltacabtagene autoleucel) which is another BCMA CAR T-cell product that's not yet approved, but hopefully will be, and that data is looking closer to two years that people can go without getting additional therapy. We're excited to see the final data cut on that. But in answer to your question, what the patient could expect is maybe one, maybe two years, depending. Now these are just data points. We anticipate, I'm hoping, that as we move the CAR T-cell therapy earlier, not waiting for the seventh line, that maybe we'll be able to get even more durability from this response.
Andrew Schorr: So Dr. Shah, when should a patient and a doctor start talking about CAR T as maybe a possibility for them? Where does it fit in now?
When Should Patients Consider This Type of Treatment?
Dr. Shah: I think this is a really important conversation to have. And just like any patients who are diagnosed with myeloma, they may see their doctor, they may also consult with the myeloma specialist. In that first conversation, they may have discussions about, for example, transplant. But then the first time there's any progression, for example, maybe a patient has gotten chemotherapy and then a transplant and then has gotten maintenance therapy and then the numbers start to rise, I think that's the time to start talking about it, not doing it, because that's not what the FDA approved label is, but just to start having the conversation so that it's not a surprise for things that may come down the line.
Andrew Schorr: Now you're in Northern California, one of the top medical centers in the world and a top myeloma program. People may be hearing this in a more remote area, whether in California or wherever. Where do you go for CAR T now? What's the logistics involved? What are the sort of expectations? You probably have people from central California or further. How does it work? How is it coordinated with their local physician, for example?
Dr. Shah: Right. That's a really good question because one of the challenges for all these things is access, right? You have to get to a center that's specialized. Now there are only certain centers that are specialized to do this particular therapy because you have to have the capability to collect cells and you have had experience giving these cells and understanding the training going with it. Not rocket science, but it has to be done. There are centers all over the United States and if you look on the website for the CAR T product, for example, for the Abecma product, they give you a list of centers that do do this. Once you find a center near you, you can either ask your physician to place a direct referral, or you may be able to call that center through their portal and ask to be plugged into one of their doctors.
Now, one of the great things about remote health care is that even if you live maybe a hundred, 200 miles, that first visit might not have to be in person. You could at least meet the doctor potentially via telehealth to maybe understand if you're a candidate or if the doctor thinks that perhaps you would or wouldn't qualify based on where your disease is, and get an idea before you make that entire trip.
Once you've decided, and the physician has decided that it's a good match, that's when you will start to do the things that are required at the center. During that time, very similar to a transplant, you'll be plugged in to a coordinator who will help you to get all the tests done, some of them you can get done at home, and then come and get ready to have that first step, which you mentioned, which is the cell collection. Then go back to your local doctor and maybe get a few weeks of chemo, and then come back to the specialized center and have the CAR T therapy given. That process, that second step, usually takes at least two weeks being right at the center, like right close and another two weeks maybe being local. You should budget a month for that before you can return home.
Andrew Schorr: Okay. I remember doing a video many years ago about transplant and one of the patients said it's no walk in the park. How big a deal is this? You talked about the timing of patients, but what people might expect to go through during the time they're with you and then close by?
What Should You Expect When You Begin CAR T-Cell Therapy?
Dr. Shah: Yeah, I think that that's very true. A lot of patients have gone through transplant. It's not a walk in the park. I will say from my own experience, that toxicity wise, I actually feel that CAR T-cell therapy is a little bit better tolerated than transplant because it's an immune therapy, not a traditional chemotherapy. That being said, there are toxicities that have to be dealt with and that do happen. We know that almost all patients, somewhere between 84% in one product and 97% in another product, will have something called cytokine release syndrome, or CRS, and that's basically a flu-like illness. Remember, this is an immune therapy, so you're tricking your body into thinking that it's fighting an infection when it's actually fighting a cancer. A lot of patients will have this fever, fatigue. Some people will have lower blood pressure, and this is the reason why those first 14 days, somewhere in there, it will be necessary for people to be inpatient so they can be monitored closely.
Thankfully, most of these episodes of CRS tend to be what we call low grade or manageable, not requiring going to the ICU. There is a medication we can give to help sort of calm it down. That's called tocilizumab (Actemra). But we tell all patients, they can expect probably that they'll have the cytokine release syndrome and then maybe they may have something called neuro toxicity, which we also call ICANS, that's an abbreviation. And that sort of manifests as a confusion that patients can have. They'll know it afterwards, but right then they kind of don't know that they're confused. We do these little tests, these mental status tests every day to make sure everything's intact, that they can remember things and write their name, write a sentence, et cetera.
Andrew Schorr: So Dr. Shah, you are a myeloma specialist and you've had this range of treatment that's been expanding. Now you had an approved CAR T, others in the offing. How do you feel about it as far as extending people's lives in multiple myeloma?
How Is CAR T-Cell Therapy Changing the Myeloma Treatment Landscape?
Dr. Shah: I think multiple myeloma is a very special disease because it's not curable, but it's livable. As I always say, it's a marathon of a disease. One of the things that makes the most impact for patients is frequency and toxicity of treatment. That's why I'm very hopeful, really hopeful that if we get better at giving CAR T-cell therapies, understand how to make them better, understand how to give them, and when to give them, we will start having a therapy that's a one-time treatment. That's the thing that I'm most hopeful for. Even if it lasts one to two years, that's still one to two years where people can go on vacation and not have to go to the chemo suite or not have to take a pill. I think that's something that's really a part of the quality of life, not just the quantity of life.
Andrew Schorr: Okay. Well, just to sum up then for our audience and tell me if I get it right because you're the professor. You have a new approved therapy, others in this class that are promising, and you may be using it earlier. As people have multiple lines of therapy, they maybe should have a discussion with their doctor about whether this could apply to them. Did I get it right?
Dr. Shah: That's exactly right. At this time, the therapy is approved for patients who have had at least four prior lines of therapy. That's something your physician can tell you if you've had or not. But as soon as you've heard the word "relapse, progression, refractory," that's the time you should have that discussion with your local doctor and maybe with a specialist who does CAR T-cell therapy.
Andrew Schorr: Okay. Thank you for filling us in on this. I'm sure we'll have updates as you learn more and perfect the technology and maybe other additional approaches come to bear. Dr. Nina Shah from UCSF in San Francisco, thank you so much for being with us and your dedication to myeloma patients.
Dr. Shah: Thanks so much for having me. It's a privilege.
Andrew Schorr: I'm Andrew Schorr. Remember, knowledge can be the best medicine of all.