Tag Archive for: clinical trial

AML Clinical Trials | When to Consider This Treatment Option

 

How do clinical trials fit into an AML treatment plan? Dr. Eric Winer highlights the importance of clinical trials to advancing AML therapies and encourages discussing your options and trial eligibility with your care team. 

Dr. Eric S. Winer is Assistant Professor of Medicine at Harvard Medical School and Clinical Director of Adult Leukemia at Dana-Farber Cancer Institute. Learn more about Dr. Winer.

 
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Elevate | What You Should Know About Your Role in AML Treatment and Care Decisions 

Transcript: 

Katherine Banwell:

When considering treatment options, where do clinical trials fit into the plan? 

Dr. Eric Winer:

I think clinical trials are extraordinarily important. The way that I think many of us think about this is we want to continually do better, and have our patients continually have improvements. The only way we can do that is by bringing forth novel medications in order to gain that extra improvement. As mentioned, all of these small molecule inhibitors, every drug that we have out there, started off as clinical trials.   

We were able to gain benefit, and patients are able to gain benefit by taking part in these clinical trials. Not all clinical trial is successful to be fair, and different types of clinical trials have different scientific knowledge beforehand. For example, Phase I clinical trials tend to be more experimental. We don’t know as much about the drugs.  

Phase III experimental clinical trials are much more well-known. Then there are a bunch in between in terms of Phase I’s where we know the drug, but we’re studying more of a combination, but of these clinical trials, the purpose of this is to gain benefit.  

If we didn’t have a drug that we believed was going to be helpful, we wouldn’t be doing that clinical trial. So, while some people may think of these things as experimental, I think of them as rationally evaluating a way to target particular forms of leukemia to gain better responses. 

Katherine Banwell:

If a clinical trial isn’t offered, how can patients inquire about their potential options? 

Dr. Eric Winer:

The first thing to do is speak to their physician. Many physicians, if they have clinical trials, they’ll know the eligibility. They’ll know who is and isn’t eligible, and why they’re not eligible. That’s something that can be easily explained to people. The second thing is if there aren’t clinical trials available at that institution, then it’s important for the patients to talk to their clinicians, and say, is there a clinical trial available someplace else that might be good for me.  

Many of us field calls from other physicians, from other colleagues, who call us and say, “Hey, I have a patient with this particular disease. Do you have a clinical trial available?” We’re always willing to collaborate. The one nice thing about the leukemia field is it’s a relatively small field. We all know each other. We all realize that the purpose of this is to make patients better. And so, we all share information, and we all work together to try to get that accomplished. 

Katherine Banwell:

There are a couple of really good websites available too, to find out about clinical trials, correct? 

Dr. Eric Winer:

There are, and I think that by contacting different institutions that can be helpful, such as the Leukemia & Lymphoma Society is a good one. There is a national clinical trial database called clinicaltrials.gov.  

Those are all very important, but sometimes they can be a little difficult to navigate. And so, it’s always good to go back to your physician or your physician team and discuss these things to make sure that the clinical trial that someone is looking at is actually an applicable clinical trial for them.  

How Can You Learn More About Myelofibrosis Clinical Trials?

 

Dr. John Mascarenhas shares advice for patients looking to learn more about clinical trials starting with consulting a specialist. Dr. Mascarenhas also emphasizes key questions to ask, including a discussion of the benefits versus risks of participating in a potential trial. 

Dr. John Mascarenhas is Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai. Learn more about Dr. Mascarenhas.

See More from Evolve Myelofibrosis

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Myelofibrosis Clinical Trial Participation | How Does It Move Research Forward

Myelofibrosis Clinical Trial Participation | How Does It Move Research Forward?

Considering a Myelofibrosis Clinical Trial? Questions You Should Ask

Considering a Myelofibrosis Clinical Trial? Questions You Should Ask

Expert Perspective | A Concerted Effort to Advance Myelofibrosis Care

Expert Perspective | A Concerted Effort to Advance Myelofibrosis Care

Transcript:

Katherine Banwell:

What about clinical trials? How can patients learn more? 

Dr. John Mascarenhas:

Well, clinical trials is definitely a confusing area because there are many clinical trials, and some of them are relevant to some patients may be not relevant to other patients.  

So, I think two ways is, see someone who does this. So, not everyone has as their primary care provider hematologist an expert, or someone who’s dedicated to doing this. They may be in a practice where they’re in the community setting; it’s not reasonable for them to be seen at a tertiary care center. 

But if you can get there even for a consultation or an initial visit to get plugged in, I think it’s really critical to see someone who is really invested in this with research opportunities, and clinical trial availabilities. And then the other resource would be clinicaltrials.gov.  

You can go in there, you can put in your diagnosis myelofibrosis. You can even manipulate it for geography to understand what trials are in your area. 

But it is a good way of looking, just to understand what’s there. And then the MPN Research Foundation, which is very supportive of the patient community and engages them, is often a very good resource to go to, to either learn about trials or join webinars where physicians that do this will discuss, and I think that’s another excellent resource. 

Katherine Banwell:

What questions should patients ask their team about clinical trials?   

Dr. John Mascarenhas:

Well, are there clinical trials for what I have? Because again, they can be very particular to where you are in the disease process, what medications you are on, what your kidney function is, things that may really influence decision-making. So, are there clinical trials, what are the clinical trials evaluating, what should I expect from them, both potentially from a positive angle, but also from a negative. What are the known toxicities, what would be the time commitment? Trials are more involved, and they’re more involved for a reason. 

I actually personally think patients do better on clinical trials than standard of care. The reason why I think that is because they are more involved. There’s a lot more oversight and eyes on you. Not just a physician perspective, but maybe more importantly, from a research nurse and research coordinator perspective. There’s a lot of regulatory burden which translates, I think, to a lot of attention to safety and assessment for advocacy.  

So, I think understanding what that looks like at any given institution, and how it will affect the patient and their caregivers from a time perspective, and obviously toxicity. But also, what is this trial trying to achieve? Does it make sense for what I’m trying to achieve? 

Katherine Banwell:

I suppose another question might be where this trial is taking place? As a myelofibrosis patient and a caregiver, are we going to have to travel to get to this clinical trial? 

Dr. John Mascarenhas:

I think that’s a major obstacle for a lot of patients. We’re talking about a disease that typically affects people that are in there sixth, seventh, eighth decade of life. Patients don’t all live, as I’ve said, right around a cancer center or a tertiary care center, so travel, the logistics of it, the ability to have that support available. Sometimes it’s loved ones, and adult children having to take time off of work to be able to help in that process.  

It’s a lot, and I’m particularly sensitive to it, because I work in a metropolitan area, and I realize getting in and out is not easy. And a lot of times, these trials understand that, and they build into the trials stipends and support for transportation and/or lodging which helps. It’s not perfect, but it can help at least financially, and sometimes logistically, so I would definitely inquire about what those resources are.  

And sometimes foundations also help bridge the gaps for patients to help link them to trials and facilitate that. So, it’s a super important part of engaging in a trial. 

Katherine Banwell:

And who is on the health care team that might be able to answer questions like this? 

Dr. John Mascarenhas:

Well, for sure, the physician should be able to. But I think the most valuable resource often is the nurse and the nurse practitioner. There are usually research coordinators. These are often young people, but very bright young people, that are very invested in this that will sometimes show up at the clinic to talk to the patient or will be a phone resource that you could reach out to. So, it can really be, I think, three levels. The coordinator, the nurse or nurse practitioner, or physician assistant, and the physician. So, it really shouldn’t be one person, but a team of people that are available to you. 

Advice for Shared Decision-Making | Myelofibrosis Care and Treatment Goals

Myelofibrosis expert Dr. Naveen Pemmaraju advises on how patients and healthcare teams can partner together by communicating care goals and exploring treatment options.

Dr. Naveen Pemmaraju is Director of the Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Program and Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Pemmaraju.

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What Myelofibrosis Treatment Types Are Available?

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Emotional Health | Why It’s Vital for Myelofibrosis Patients to Share Concerns 

Emotional Health | Why It’s Vital for Myelofibrosis Patients to Share Concerns

Transcript:

Katherine Banwell:

When it comes to choosing therapy, Dr. Pemmaraju, it’s important to work with your healthcare team to identify what is going to work best for you. So, as a clinician, how do you define shared decision-making?  

Dr. Naveen Pemmaraju:

Very important. So, shared decision-making to me means a partnership. It means a journey that the patient and the providing team are about to embark on. It’s a very different approach than a one-way, I tell you, you do this. Instead, I see it as a bi-directional exchange of ideas.  

Each visit, each EPIC in-basket or EMR communication, each touch with the healthcare system, the pharmacist, the PA, nurse, whoever is dealing with the patient, I think that’s the key.  

So, a bi-directional exchange of ideas, what’s important to you as the patient? What’s important to the caregiver? What are the worries? What are the barriers? Designing a treatment system around that, a treatment paradigm and approach. Discussing risks, benefits, side effects, toxicities, alternatives, and then a constant dynamic reevaluation throughout. That’s what I pictured. It has to be a journey and a partnership.  

Katherine Banwell:

Well, part of making care decisions is setting goals, and I think you’ve just alluded to that. What are treatment goals for myelofibrosis, and how are they determined?  

Dr. Naveen Pemmaraju:

That’s a great question. Myelofibrosis treatment goals are changing in real-time. I would say as of this recording, 2024, the main three things that I want patients to think about and the caregivers.  

Number one is a stem-cell transplant eligible or not? It used to be based on age and comorbidities, but there are other factors. So, are we going to stem cell transplants or not? That determines a lot of the journey. Two is a clinical trial or not. So, are we doing the standard of care therapy, often one pill at a time, or clinical trial, either an IV drug, a pill, or combinations? Then three is that dynamic assessment that we talked about, which is what are the goals of care? Often our patients with myelofibrosis have decreased quality of life, enlarged organs, fatigue, cachexia, and malnutrition.  

These are the central components. A lot of times they’re due to the myelofibrosis itself. So, the treatments may improve that. A lot of times it’s the other comorbidities, other health issues. So, working with the PCP, the primary care provider, and the local team. In my case, many of my patients are referrals, as you know, the local MD team. I think these are the three components, transplant eligibility or not, clinical trial versus standard of care. 

Then once we’ve made a treatment decision, minding toxicities and quality of life.  

Evolve Thyroid Cancer Resource Guide

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What Are the Benefits of Thyroid Cancer Clinical Trial Participation?

What Are the Benefits of Thyroid Cancer Clinical Trial Participation? from Patient Empowerment Network on Vimeo.

What are the benefits of thyroid cancer clinical trial participation? Dr. Lori Wirth discusses how clinical trials provide access to promising new treatments, offering patients additional options, and the potential for significant advancements in managing their disease.

Dr. Lori Wirth is the Medical Director of the Center for Head and Neck Cancers at Massachusetts General Hospital. Learn more about Dr. Wirth.

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Thyroid Cancer Research and Treatment Highlights

Transcript:

Katherine:

Dr. Wirth, what would you say to patients who are hesitant to participate in a clinical trial? 

Dr. Wirth:

Oh, boy. So, that’s such an important question. 

Katherine:

Yeah.  

Dr. Wirth:

And I think that the natural reluctance to put oneself into an uncertain setting like a clinical trial is completely understandable.  

But a couple of things that I would say is first of all there is a lot of really deep work that goes into identifying new agents that have promise in the preclinical setting from laboratories either within the pharmaceutical industry or within academics. The amount of smarts that goes into development new drugs as well as early testing to ensure safety and that there’s a real signal of activity, that amount of work that’s done before a clinical trial is launched is really quite significant. So, when we’re bringing a new drug into clinical trials, we already know that there’s a very good likelihood that that drug is going to have good activity.  

Katherine:

Okay.  

Dr. Wirth:

The other reason for patients to think about participating in clinical trials is when patients have metastatic disease in the solid tumor setting whether it’s colorectal cancer or breast cancer, unfortunately most of our treatments don’t work so well that there’s a chance of cure. However, if we can’t cure a cancer, the next best thing is to knock it back and hold it at bay for as long as possible so that people can feel well but also live as long as possible.

However, if we don’t have a drug that can work so well that can cure cancer completely many cancers ultimately are going to escape the control, and we’re going to need new therapies for those patients. When patients participate in a clinical trial that’s just giving them a whole other treatment option. And so, to have more options available gives more chances that there’s going to be a real homerun or a real success in terms of treatment.  

So, I would much rather have my patient have three options of treatment rather than two options of treatment. And we can always turn to the drugs that we have that are already FDA approved as long as somebody’s well enough to receive cancer treatment. If there’s a promising clinical trial of a new agent that’s only available in a clinical trial, and if we use that earlier in the course of the disease, that gives us more options for down the road. 

A Patient’s Proactive Path to an Acute Myeloid Leukemia Diagnosis

A Patient’s Proactive Path to an Acute Myeloid Leukemia Diagnosis from Patient Empowerment Network on Vimeo.

Meet Paloma, a 58-year-old acute myeloid leukemia (AML) survivor. After experiencing breathlessness, sore gums, and other symptoms that were initially misdiagnosed, she trusted her instincts and sought further medical support, leading to her AML diagnosis. Discover Paloma’s journey and the vital importance of being proactive and staying [ACT]IVATED in your cancer care.

See More from [ACT]IVATED AML

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Transcript:

Being ACTIVATED in your cancer care is critical and also a continuous journey.  My name is Paloma, and I’m eager to share my journey as an acute myeloid leukemia patient in the hopes that it will help other patients and families. AML doesn’t discriminate; it can affect anyone, regardless of lifestyle or healthy eating habits.

I was 58 when I was diagnosed with AML, and my diagnosis was pretty shocking to me. I learned that you really need to trust your instincts when it comes to your health. I felt like something was off with my body, but my initial symptoms were only some breathlessness upon exertion and sore gums. But then additional symptoms started including a dry cough, some flu-like symptoms, and lumps under my armpits. I saw my primary care provider, and she prescribed antibiotics and sent me for a chest x-ray that came back without issues. 

When my breathlessness worsened along with profound fatigue, my doctor then sent me to get an ECG and additional blood tests to help determine what might be wrong. While I was waiting for my test results, my co-workers noticed that I looked thinner with my skin also being paler than usual. This was just the beginning of my AML journey. My blood tests came back with abnormal hemoglobin and blast levels, and my doctor arranged for me to be admitted to a well-regarded cancer center. I was fortunate that it was only 20 miles away but realize that not all cancer patients are this fortunate.

After seeing my AML specialist at the cancer center, he wanted to start my chemotherapy right away to fight the cancer. I was fortunate that I didn’t have issues with my intravenous line for receiving my chemotherapy. But I learned that this can be a common issue for AML patients after I joined an online AML support group. I had my first round of chemotherapy, and my daughter was able to visit me during this time. However, my care team kept monitoring my neutrophils and decided that I needed to start a second round of chemotherapy. It was now during the COVID-19 pandemic, and hospital visitors weren’t allowed.

I counted myself as fortunate that I could still continue with receiving chemotherapy. What would have happened if I’d gotten seriously ill during the early pandemic? I shudder to think that things likely would not have been easy. The hospital staff helped to keep my spirits up and also with setting up a tablet for me to do video calls with my family and friends while I was in the hospital.

Though that round of chemotherapy put me into remission for a period of time, I later came up as MRD-positive and received a targeted chemotherapy as a third round of therapy followed by a stem cell transplant. I had some graft-versus-host disease issues but got through them. I feel fortunate that there are some different treatment options for AML and would like to participate in a clinical trial to help advance treatments if I need another option on my journey. I’ve kept in touch with other patients in my AML support group during my journey from diagnosis, treatments, and recovery. I know that I couldn’t have gotten through my physical and mental challenges without them.

Though AML sounded scary at first, the future of treatment looks bright to me with emerging research and treatment options. I hope that sharing my story will make a difference for other AML patients and especially those who may come up against barriers. 

No matter who you are, being proactive is a critical step in your AML journey. Stay [ACT]IVATED by being informed, empowered, and engaged in your care.

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Empowered Care: A Patient’s Guide to Navigating Endometrial Cancer

Empowered Care: A Patient’s Guide to Navigating Endometrial Cancer from Patient Empowerment Network on Vimeo.

Meet Sharon, a 61-year-old endometrial cancer survivor who knows firsthand the importance of being an active participant in her healthcare. After a two-year struggle with misdiagnosed symptoms and a dismissive doctor, she took charge and found the right medical support, leading to a diagnosis of endometrial cancer.

Sharon’s journey, from diagnosis to treatment and recovery, highlights the vital role of self-advocacy and patient activation. Her story is a beacon for others, especially women of color facing similar challenges, emphasizing the power of being informed, asking questions, and seeking support.

Download Guide | Descargar Guía en Español

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Transcript:

Being activated is a critical part of endometrial cancer care, especially for patients like me. 

My name is Sharon, I’m 61, and my diagnosis came after a two-year struggle with unidentified symptoms. Sharing my experience is my way of reaching out, hoping it might provide guidance to others facing similar challenges.

My symptoms began with abnormal vaginal bleeding, but my periods had ended over 10 years ago. That had me worried, and my sister encouraged me to see a doctor after I told her about the bleeding. I scheduled an appointment, but my doctor dismissed my symptoms. I felt like he wasn’t really listening to me and decided to find a doctor who looks more like me and would be more likely to listen to me. I found a Black female doctor who was concerned about my symptoms, which had worsened by then. I was also feeling pelvic pain. My new doctor scheduled an endometrial biopsy, and I was diagnosed with papillary serous carcinoma shortly afterward.

With an aggressive type of cancer, my oncologist scheduled a laparoscopic hysterectomy to remove my uterus along with my ovaries, fallopian tubes, and sentinel lymph nodes. I had the surgery within a few days, which was quickly followed by radiation to help ensure any remaining cancer cells were wiped out. My recovery went smoothly, and I continue to live a full life while getting regular scans to ensure I remain cancer-free.

After my cancer experience, I want to educate other women about what I’ve learned about endometrial cancer. Black women have nearly twice the death rate from endometrial cancer compared to white women. Hispanic, Black, and Asian women are not represented in clinical trials at equal rates to white women. And Black women are also diagnosed more frequently with rare but aggressive endometrial cancer forms. Remember that you shouldn’t have to suffer with your pain, and you can advocate for yourself and ask about patient advocates to advocate on your behalf.

Here are my activation tips for patients facing an endometrial cancer diagnosis:

  1. Ask your care team questions to learn about the status of your endometrial cancer, treatment options, and what to expect during and after treatment.
  2. Join a patient support group to offer and receive emotional support.
  3. Last but not least, inquire if a clinical trial may be a potential treatment option for your endometrial cancer.

Remember, stay activated by being informed, empowered, and engaged in your cancer care.


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Miguel’s Journey: Embracing CAR T-cell Therapy as a Latinx Myeloma Survivor



Miguel’s Journey: Embracing CAR T-cell Therapy as a Latinx Myeloma Survivor from Patient Empowerment Network on Vimeo.

Myeloma survivor Miguel wasn’t experiencing any symptoms when he received his shocking diagnosis. Watch as he shares his experience as a Latinx myeloma patient dealing with testing, multiple lines of treatment, and CAR T-cell therapy – and how to stay [ACT]IVATED in your care.

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Transcript:

Being ACTIVATED in CAR T-cell therapy care is critical for patients and families. My name is Miguel and I want to share my story as a myeloma survivor and Latinx man. Even though CAR T-cell therapy has improved survival rates for myeloma patients, some disparities to treatment access still persist. 

I was 52 when I was diagnosed with smoldering myeloma, and my diagnosis came as a complete shock. I wasn’t experiencing any symptoms, and my doctor only discovered my condition after noticing that something looked abnormal in my blood work. After ordering further testing, my diagnosis was confirmed with a bone marrow biopsy. That was just the start of my long journey. My hematologist informed me that several rounds of chemotherapy would be best for my first line of treatment.

After I finished my rounds of chemo, my hematologist continued to monitor my tests closely for signs of recurrence. When my tests reached concerning levels, my doctor then recommended that I move forward with an autologous stem cell transplant – taken from my own stem cells.

Those were just my first two lines of therapy. My third line of therapy was an immunotherapy as part of combination therapy that worked for nearly two years. An allogeneic stem cell transplant – with stem cells taken from a donor – was recommended next. That second stem cell transplant kept my myeloma at bay for about two years. It was a nice break, and I was able to qualify for a CAR T-cell therapy when it came time to act on my fifth line of treatment. I had learned from my myeloma support group that patients need to have a lot of support to qualify for CAR T. 

Patients need to have a care partner to support them, and I was fortunate enough to have my sister stay with me to help me with my appointments and recovery. CAR T-cell therapy has made the future brighter for so many myeloma patients.

There have been a lot of recent advancements in CAR T-cell therapy for myeloma. I hope that sharing my story will make a difference for other myeloma patients who may have some mistrust of doctors. Remember, become empowered and stay [ACT]IVATED with these tips. 

[ACT]IVATION tips for CAR T patients: 

  • Ask your care team questions to learn about the status of your myeloma, treatment options, and what to expect during and after treatment.
  • Inquire if a clinical trial may be a potential treatment option for your myeloma.
  • Join a patient support group to offer and receive emotional support.
  • Stay updated about myeloma treatment options and research advancements. 

Being proactive is an essential step in your myeloma journey. Stay [ACT]IVATED by being informed, empowered, and engaged in your care.


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Charise Gleason: Why Is It Important for You to Empower Patients?

Charise Gleason: Why Is It Important for You to Empower Patients? from Patient Empowerment Network on Vimeo.

How can patients and families be empowered? Advanced practice professional Charise Gleason from Winship Cancer Institute discusses her perspective and communication methods that have shown benefits for her myeloma patients.

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Dr. Craig Cole: Why Is It Important for You to Empower Patients?

Transcript:

Charise Gleason:

I think it’s important to empower our patients, and we do that from day one. Patient comes to us, we’re starting to develop that relationship. And the discussions that we have early on can be very different from later, but we have to continually reinforce, ask questions, give patients the opportunity to ask us questions. I know when I talk to a patient about a clinical trial, and I’m documenting, I put that back in, patient or family member or care partner was given the opportunity to ask questions.

Our clinics go very quickly, and you have to make the time for your patients. So you have that relationship, and they know that they can bring issues to you. Sometimes we don’t get it right either, and you’ve got to own that and move on to that next step. So you continue that relationship. Patients are going through losing control with having a cancer, and like myeloma and many times patients never even heard of multiple myeloma until they come into our world.

So it is an ongoing open communication, and we don’t make decisions for them. We give them options and upfront or early relapse, you may have far more options than you do in that relapsed/refractory setting. But you’ve got to know what’s important to your patient and what their goals are. And, are they still working? Our patients vary in age. But you want to think about where your patient is, what’s important to them, and you don’t know that unless you ask the questions and have that communication. Our patients are very savvy. We go to meetings. The first thing they want to know is what did you learn? Even when they’re doing well on their current treatment, they want to know what’s next. What’s out there for me if this stops working?

 When we’re in that biochemical relapse phase where we don’t have to change treatment, we’re already having those conversations about what are those options next for you? And so I think that having that team approach, that open communication is really important for our patients and empowers them to make good decisions. As an advanced practice provider, it’s important for me to explain my role, right?

Patients will come to a practice, and sometimes they’re surprised that I don’t see the physician every time I come. So I think it’s instead of ignoring that and not telling patients, I think it’s important that we describe our roles in that care as well. That, yes, I also specialize in multiple myeloma. I collaborate with your physician. We talk about you, even if you’re not seeing your physician. And so I think that patient and family understanding the rest of the team and what we bring to the table for them is essential as well. 

What Do You Need to Know When Considering CAR T-Cell Therapy?

What Do You Need to Know When Considering CAR T-Cell Therapy? from Patient Empowerment Network on Vimeo.

How does one access myeloma CAR T-cell therapy? This animated explainer video provides an overview of the steps involved in determining whether a patient qualifies to receive CAR T-cell therapy, what the process entails, common side effects, and why having a care partner is essential.

See More From Thrive CAR T-Cell Therapy

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Understanding CAR T-Cell Therapy | How It Works and Who It’s Right For

Understanding CAR T-Cell Therapy | How It Works and Who It’s Right For 

Understanding Possible Side Effects of CAR T-Cell Therapy

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What You Need to Know About Accessing CAR T-Cell Therapy

What You Need to Know About Accessing CAR T-Cell Therapy 

Transcript:

The emergence of CAR T-cell therapy is revolutionizing treatment for some people with myeloma. But, who is it right for, and what is the process for people that qualify?  

  • The first step in accessing this treatment is to be referred by your physician to a center that specializes in CAR T-cell therapy. 
  • Then, a consultation will take place with the transplant team, and a health assessment is administered to ensure patients are healthy enough for CAR T-cell therapy. This includes testing to review the current status of your cancer and testing of your body’s major organ systems.
  • Next, the specialty center will evaluate the best type of CAR T-cell therapy for the patient, including clinical trial options.
  • After approval, financial coordinators will discuss insurance and therapy costs with the potential recipient. Logistics are also arranged at this time, which may include help with transportation and housing, if necessary.
  • Medical centers also require that patients have a care partner, such as a family member or friend, who can be with them at all times, particularly after leaving the hospital. 

So, what is the process once a patient is approved for CAR T-cell therapy? Once a patient is approved to move forward with the procedure, a date is set for collection of the patient’s T cells. T-cells are collected during a process called apheresis. During apheresis a specialized machine filters the patient’s blood to remove the T-cells for collection and the rest of the blood is returned to the patient.  

 After collection, the T cells are sent for manufacturing. During that time, the patient is given a “bridging therapy” to maintain the myeloma until the CAR T cells are infused.  

Once the CAR T cells are infused, the patient will be closely monitored by the CAR T center. This may or may not include hospitalization depending on the policies of the treatment center. Patients and their care partner should plan to stay close by the center for up to 30 days after the infusion.  

During this time, the patient is evaluated for their response to treatment and monitored for possible side effects so that they can be managed in a timely manner.  

The potential side effects of CAR T-cell therapy may include: 

  • Cytokine release syndrome, or CRS, which is an aggressive response to treatment by the immune system and may cause symptoms such as low blood pressure, high heart rate decreased oxygen saturation, fever, nausea, and body aches. 
  • Another possible side effect is neurotoxicity, which is an adverse event that may cause issues such as confusion, difficulty with communication, seizure, or tremors. 
  • And, another side effect may be low blood counts, which could impact the immune system and increase risk for infection. 

Every patient is different, so close monitoring is essential.  

So now that you know more about CAR T-cell therapy, you can work with your healthcare team to decide if this treatment option may be right for you. Be sure to speak up and ask questions. Remember, you have a voice in YOUR myeloma care. 

To learn more about myeloma and to access tools for self-advocacy, visit powerfulpatients.org/myeloma.  

Olutasidenib for Relapsed or Refractory Acute Myeloid Leukemia with IDH1 Mutation

Introduction 

Once again a new drug that was recently FDA approved for some Acute Myeloid Leukemia (AML) patients. Olutasidenib (brand name: Rezlidhia) was approved in December 2022 for AML patients who have relapsed or are refractory to treatment (R/R) with a mutation in the Isocitrate Dehydrogenase 1 (IDH1) gene. There is a different gene called IDH2, which can also be mutated in patients with AML, but different medications are used for IDH2 mutations. 

An existing drug, called ivosidenib (brand name: Tibsovo) was already approved for patients with AML and a mutation in IDH1. It was initially approved in July 2018 as a single agent for patients with R? AML with a IDH1 mutation. Later it was approved for use in combination with azacytidine for newly diagnosed AML patients with an IDH1 mutation. There was also a specific test (the Abbott RealTime IDH1 Assay) approved along with the initial approval of ivosidenib. This test was also approved to select patients to be treated with olutasicdenib.

Results of the Current Study 

The study used to support the approval was a non-randomized trial (all patients received olutasidenib). The study included patients with AML and ones with Myelodysplastic Syndrome (MDS) who had a mutation in the IDH1 gene. Patients were treated with olutasidenib as a single agent (monotherapy) as well as combined with azacytidine. The approval was based on the results of the 147 patients who received olutasidenib monotherapy (the results are described in detail here). 

In the study, about 35% of patients achieved a complete remission (CR) or complete remission with partial hematologic recovery (CRh) – the latter means that they appear to be in remission, but their blood counts are not normal. In addition, about one third of patients who were platelet and/or red blood transfusion dependent at the start of the trial eventually did not require transfusions for 8 or more consecutive weeks (transfusion independent). All patients experienced 1 or more adverse events (AEs) and almost all experience a serious (Grade III or IV) AE. These AEs are expected in patients with AML, particularly older patients, as most of the patients were. 

Comparison with Ivosidenib 

Now that there are 2 drugs approved for AML patients with an IDH1 mutation, the question becomes which drug should be used and in which circumstances. There have been no studies (at least ones that are published) that directly compare the drugs. According to the paper from Bload Advances on the responses to olutasidenib and ivosidenib as single agents was about the same, however the length of remission in the patients receiving olutasidenib (a median of 25.9 months) compared to ivosidenib (8.2 months0. The big caveat is that, since the comparison was not randomized, it is difficult to determine if there were differences in risks in each group. My guess is that there will not be a randomized comparison of these two groups.

It will be more interesting to look at these drugs in combination with azacytadine and also as a 3 drug combination of azacytidine and venetoclax. In addition, these drugs could be combined with existing intensive chemotherapy regimens (for instance, so-called 7+3 induction with cytarabine and daunorubicin). Likely the combinations would produce more remissions but might have significantly more side effects. 

In summary, the good news is that there is another drug that can help some patients with AML, the bad news is that the determination of the best therapy is yet more complicated. 

Further reading 

Olutasidenib: 

Ivosidenib: 

Advances in the Treatment of Relapsed/Refractory Acute Myeloid Leukemia (AML)

Advances in the Treatment of Relapsed/Refractory Acute Myeloid Leukemia (AML) from Patient Empowerment Network on Vimeo.

AML expert Dr. Omer Jamy discusses his approach when considering treatment for patients with relapsed or refractory AML, including transplant eligibility, molecular markers, and whether clinical trials may be an appropriate option.

Dr. Omer Jamy is a Leukemia and Bone Marrow Transplant Physician and Assistant Professor at the University of Alabama at Birmingham. Learn more about Dr. Omer Jamy.

See More From INSIST! AML

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What Is the AGILE Study? Research for AML Patients With the IDH1 Mutation

What Is the AGILE Study? Research for AML Patients With the IDH1 Mutation

What Is the Purpose of AML Genetic Testing

What is the Purpose of AML Genetic Testing?

Essential Testing | Optimizing AML Care With Personalized Medicine

Essential Testing Optimizing AML Care with Personalized Medicine

Transcript:

Katherine Banwell:

Dr. Jamy, are there any recent advances that may affect the care of patients with relapsed or refractory AML? 

Dr. Omer Jamy:

Yeah, that’s a good question. So, patients with relapse refractory AML, of course, carry a poor prognosis. That means that chemotherapy was working and has stopped working or chemotherapy didn’t work from the get-go, right?  

So, in my practice I try to divide patients into two different buckets. One is that I need to get them into remission, and they’re fit for a transplant, so I take them to transplant.  

So, then my treatment approach is a little different for those patients. As opposed to someone who’s elderly or too frail, that they may go into remission, but they may not be able to proceed to stem cell transplantation after that.  

So, what happened in the relapsed/refractory setting also depends on what the patient received in the upfront setting. Ideally, I would recommend a clinical trial enrollment for patients with relapse refractory AML if they have access to it. At the time of relapsed/refractory AML, it is very important to again profile the leukemia to see if there are any mutations that were present at diagnosis or if there are any new mutations for which there may be targeted therapy. Some of those mutations for which we have targeted therapy include FLT3-ITD for which there is a drug called gilteritnib (Xospata), which is FDA-approved in the relapsed/refractory setting. 

We spoke about IDH 1 which is ivosidenib, IDH 2 which is enasidenib (Idhifa) is also approved for patients with relapsed/refractory AML. And then more recently the FDA approved another IDH1 compound called olutasidenib (Rezlidhia) which is also for patients with relapse refractory acute myeloid leukemia with an IDH1 mutation. I think these are target therapies which have shown to get people into a second remission and beyond. And these have been approved in the last few years. And I think it is very important to basically test whether the person harbors these mutations so that we can target them accordingly.  

For patients who don’t have any mutations we would generally, outside of a clinical trial, probably use the combination of some of the approved agents that may be venetoclax (Venclexta) with azacitidine (Vidaza) or decitabine (Dacogen). Patients who may have received this venetoclax or a hypomethylating agents frontline and may still be eligible for intensive chemotherapy.  

You could offer them intensive chemotherapy in the relapsed/refractory setting, but I would say that at this point being at a center where there’s opportunities to enroll in a clinical trial would be really helpful as well. 

When Should AML Patients Consider Joining a Clinical Trial?

When Should AML Patients Consider Joining a Clinical Trial? from Patient Empowerment Network on Vimeo.

With AML research advancing quickly, clinical trials are an important consideration when making a treatment decision. AML expert and researcher Dr. Omer Jamy discusses when joining a clinical trial may be appropriate. 

Dr. Omer Jamy is a Leukemia and Bone Marrow Transplant Physician and Assistant Professor at the University of Alabama at Birmingham. Learn more about Dr. Omer Jamy.

See More from Thrive AML

Related Resources:

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Expert Perspective | Key Advice for AML Patients

Updates in AML Treatment and Research From ASCO 2023

What Are the Phases of AML Therapy


Transcript:

Katherine Banwell:

Dr. Jamy, when should AML patients consider joining a clinical trial? 

Dr. Omer Jamy:

Yeah, that’s a very interesting question. No, I have my personal thoughts on that which I share. So, I feel like clinical trials are of different flavors. They range from early phase to late phase trials. I think being at a center where there’s opportunities to enroll in clinical trials is really helpful. Now if you have a newly diagnosed patient with AML, there is good standard of care treatment. Of course, they can be improved upon.  

I would probably improve upon them in the setting of a Phase III where they get standard of care plus an additional agent versus placebo where at minimum, they’re getting standard of care, right? So, it will be very challenging unless it’s a very novel concept to enroll someone who has not seen any standard therapy on an earlier phase study. Let’s put it this way. Whereas it changes completely when they’ve relapsed meaning they’ve gone through options which are pretty standard. At that point, enrolling in the clinical trial is most likely in their best interest. I think because once leukemia relapses, we have limited options.  

I think we’ve been lucky over the past five years that we’ve had several drugs approved. But there’s still probably less than 10. And out of those, not everybody is a candidate for each of those drugs. They’re targeting specific mutations. So, the relapse refractory setting I think enrolling in a clinical trial is really helpful. Up front I just take more interest in the clinical trial design and the consent form before agreeing to participate. 

Expert Perspective | Key Advice for AML Patients

Expert Perspective | Key Advice for AML Patients from Patient Empowerment Network on Vimeo.

Facing an AML diagnosis can feel overwhelming. Dr. Omer Jamy shares tips for newly diagnosed AML patients, emphasizing the importance of a consultation with a specialist.

Dr. Omer Jamy is a Leukemia and Bone Marrow Transplant Physician and Assistant Professor at the University of Alabama at Birmingham. Learn more about Dr. Omer Jamy.

See More from Thrive AML

Related Resources:

Updates in AML Treatment and Research From ASCO 2023

What Are the Phases of AML Therapy


Transcript:

Katherine Banwell:

Dr. Jamy, for patients who have been diagnosed with AML, could you share three key pieces of advice for them. How can they be proactive in their care? 

Dr. Omer Jamy:

Sure. So, I feel like as a leukemia physician I would like to see, just to give you an example, I’d like to see all the leukemia patients in Alabama. But that’s not feasible, right? But what I would recommend to patients and caregivers is that wherever they are diagnosed, I do feel that they would benefit from a consultation with a leukemia physician at a tertiary care center or an academic center. And they would benefit due to various reasons, right? So, the first reason would be that as a leukemia physician my job is to just keep myself upgraded with leukemia care, leukemia management.  

So, one aspect of leukemia is therapeutics, right? So, drugs that are approved, easy to give. But the other aspect is understanding the biology of the disease, understanding how leukemia is going to behave. To get a better profile for AML for a patient. So, in a way saying that not all AML cases are the same. So, to be seen at a center would help the physician understand the unique cytogenetic or molecular profile of that patient’s AML which may be different from the next patient’s AML which could mean that the treatment algorithm for one person might be slightly different from the second person. So, I mean the academic and the people working at academic centers cannot survive without people working in the community, so it goes hand in hand. So, I feel like co-management of a patient with AML is extremely important. I feel like things will not get missed that way.  

I feel like the treatment plan, no matter where it is implemented, would really benefit the patient. It can be implemented closer to home as long as it’s been co-managed with someone closer to home as well as someone at the center where they have access to more information. What this would also help is get the person and the family plugged into a system where, let’s say if therapy wasn’t working, they’d have access to enroll on clinical trials down the line as well. Which unfortunately are only present at academic centers and not very widely available, especially for blood cancers. There may be trials for solid tumors easily conducted outside of academic centers, but unfortunately that’s not the case for blood cancers, specifically AML. So, the opportunity to enroll in clinical trials will also help.  

And then lastly, I feel like it’s our ability to offer bone marrow transplant to older patients has improved over the past 10 to 15 years.  

We’ve become better in identifying donors and in identifying patients, getting them ready for transplant that I feel that a person and the caregiver should inquire from their physician about the opportunity – oh, of No. 1 the need for transplant for the leukemia is because not all the AML patients may benefit from our transplant, but most of them do. And definitely anyone who relapses would benefit from a stem cell transplant.  

So, I feel like inquiring about that is very important because to get plugged in at a transplant center early on is important because you don’t want to waste time early on. You may not need the transplant, but just having the consultation and just having a preliminary donor search ongoing in the background is really helpful because when the time comes that a person needs the transplant, then you’ve already got some of that information ready, and you can proceed quickly. So, I feel like a few of those things might be helpful which I try to educate in the community as well and do outreach.  

Because I feel like it’s important to let people know that AML is an aggressive disease. Transplant is pretty intense, but we are now making it more and more tolerable for older patients. 

Updates in AML Treatment and Research From ASCO 2023

Updates in AML Treatment and Research From ASCO 2023 from Patient Empowerment Network on Vimeo.

AML expert Dr. Omer Jamy shares highlights from the recent American Society of Clinical Oncology (ASCO) annual meeting, including an update on an immunotherapy agent showing promise as well as a vaccine therapy being studied for patients in a second remission.

Dr. Omer Jamy is a Leukemia and Bone Marrow Transplant Physician and Assistant Professor at the University of Alabama at Birmingham. Learn more about Dr. Omer Jamy.

See More from Thrive AML

Related Resources:

Expert Perspective | Key Advice for AML Patients

Expert Perspective | Key Advice for AML Patients

What Are the Phases of AML Therapy


Transcript:

Dr. Omer Jamy:

My name is Omer Jamy, and I’m a leukemia and bone marrow transplant physician at the University of Alabama at Birmingham. And I’m really happy to be here today.  

Katherine Banwell:

Well, thank you. Dr. Jamy, the ASCO 2023 meeting just wrapped up recently. What were the highlights in AML research from that meeting? 

Dr. Omer Jamy:

Thank you. Yeah. There were several interesting studies in AML presented at ASCO this year. I’d like to highlight a couple of them in particular mainly because the focus on a novel mechanism of action, at least for patients with acute myeloid leukemia. And that mechanism of action being immunotherapy. So, we’re all aware that immunotherapy has had tremendous results in solid tumors.   

It’s making its way into hematological malignancies mainly lymphomas as well as B-cell ALL which is acute lymphoblastic leukemia. And we are trying to investigate it in patients with AML as well.  

And I think in that context there were a couple of abstracts which I thought were really interesting. The first one was actually presented by Dr. Anthony Stein and colleagues. Looking at a drug which is basically CD123 NK-cell engager.  

And I spent a little bit trying to explain what that is, but basically, it’s a drug which it harnesses the person’s immune system to fight the cancer basically. So, it targets an antigen which is expressed on leukemia cells called CD123. And it binds it to natural killer cells or NK cells. So, this drug is taking the host which is the patient’s natural killer cells and the leukemia cells and binding them together and then leads to the activation of the NK cells which causes killing of the leukemia cells.  

So, I think that mechanistically speaking that’s a very interesting concept to fine tune the person’s own immune system to fight the leukemia. This is obviously very early in development, so it’s a Phase I study, Phase I/Phase II. And they have presented results in 23 patients with relapsed/refractory AML.  

And just to give you some background, CD123 is expressed in the majority of patients with acute myeloid leukemia. It’s also expressed in patients with myelodysplastic syndrome as well as ALL. So, the study had all three diseases, but we’re going to focus on AML today. So, there were 23 patients with acute myeloid leukemia in the study. And because it’s a Phase I study, they have to test it at the lowest dose, and then assess for safety, and then keep on going up on the dose. So, they actually looked at six dose levels. And luckily because it’s a Phase I, the primary objective is to make sure it’s a safe drug to administer.  

And then second your objectives are basically if it’s efficacious or not. So, there were no dose limiting toxicities in the 21 evaluable patients out of the 23. So, that’s good. I think the lowest dose was 100 micrograms per kilograms per day. And the highest dose was 3,000 micrograms per kilogram per dose. The doses IV once or twice a week for the first couple of weeks, and then followed by weekly administration. In 23 patients the drug was thought to be safe. Again, no dose limiting toxicities.  

With immunotherapy you worry about side effects such as cytokine release syndrome because you are basically putting your immune system in overdrive. So, you don’t want to make sure your immune system doesn’t wreak havoc on the body itself. So, cytokine release syndrome or CRS as well as associated neurotoxicity are two common side effects of most of these novel immunotherapies.  

Which for the general audience, if they’ve heard about CAR T therapy or antibody drug conjugates or bispecifics, these are all under the same umbrella of immunotherapy. So, their side effect profile is pretty overlapping and different from what would be seen with conventional chemotherapy. So, they saw no neurotoxicity. And they saw CRS which was very manageable, right? Grade 1 or Grade 2 in a couple of patients. And as far as efficacy was concerned, out of the 23 patients they saw a response in three patients. Now that doesn’t sound very appealing, but you have to realize these are starting at a very low dose level and going up. So, when they looked at patients who were getting a dose of 1f,000 micrograms per kilograms per day, so a pretty hefty dose. Three out of eight patients, which roughly translates to 40% of the patients, achieved a remission. So, which to me for relapse refractory population is attractive. And it makes me want to investigate this molecule further.  

And that is exactly what’s going on currently with the study. And I think again CD123 is an interesting target. The other companies targeting as well either as NK-cell engagers or antibody drug conjugates with other payloads. So, this is an area of active investigation. So, that’s where – 

Katherine Banwell:

You said – 

Dr. Omer Jamy:

Yeah.  

Katherine Banwell:

Yeah, you said there was another study. Could you briefly tell us about that.  

Dr. Omer Jamy:

Exactly. So, the other study is also harnessing the person’s immune system to fight leukemia in a very different way. And this is a randomized Phase III study, ongoing. It’s international. And it’s a trials in progress meaning that it’s accruing across the country, or actually across the globe. And I wanted to highlight this in case people want to reach out to centers where this study is ongoing and want to participate in it. This is a trials in progress poster of a compound called GPS which is basically a vaccine against a protein called Wilm’s tumor 1 or WT1 which is vitally expressed on leukemia cells as well.  

Now this is a tumor vaccine actually which is a novel concept of an AML. So, vaccines as you know, are better at prevention than treatment. So, this is a maintenance drug for people in second remission or beyond who are unable to proceed to stem cell transplantation.  

So, they get the opportunity to enroll in this Phase III which is a randomized study of either GPS versus a physician’s choice which includes a wide variety of agents to choose from making it a pretty reasonable control arm and follows patients to see if the primary end point being overall survival. So, I think again for patients who achieve second remission or beyond ideally, they should proceed to stem cell transplantation. But there are several barriers to that including advanced age, comorbidities, socioeconomic barriers. So not everyone can proceed. So, for patients in that situation, there is no standardized maintenance therapy.   

And in that context I feel like an immunotherapy agent basically this vaccine which has shown very promising results in single-arm Phase I, Phase II studies is now being investigated in a Phase III study. And because it’s a trials in progress I cannot share any results with you because we don’t have any results. But I feel like people should know about this because it is open at 20 to 30 centers in the US.  

And it’s an option out there for patients who would like to participate in such a clinical trial.