Understanding ABECMA® (idecabtagene vicleucel)
Founded in 1990, the International Myeloma Foundation (IMF) is the first and largest organization focusing specifically on myeloma. The IMF’s reach extends to more than 525,000 members in 140 countries. The IMF is dedicated to improving the quality of life of myeloma patients while working toward prevention and a cure through our four founding principles: Research, Education, Support, and Advocacy.
RESEARCH
The IMF is dedicated to finding a cure for myeloma, and we have a range of initiatives to make this happen. The International Myeloma Working Group, which emerged from the IMF’s Scientific Advisory Board established in 1995, is the most prestigious organization with 319 myeloma researchers conducting collaborative research to improve outcomes for patients while providing critically appraised consensus guidelines that are followed around the world. Our Black Swan Research Initiative® is bridging the gap from long-term remission to cure. Our annual Brian D. Novis Research Grant Program is supporting the most promising projects by junior and senior investigators. Our Nurse Leadership Board, comprised of nurses from leading myeloma treatment centers, develops recommendations for the nursing care of myeloma patients.
EDUCATION
The IMF’s webinars, seminars, and workshops provide up-to-date information presented by leading myeloma scientists and clinicians directly to patients and their families. We have a library of more than 100 publications for patients, care partners, and healthcare professionals. IMF publications are always free-of-charge, and available in English and select other languages.
SUPPORT The IMF InfoLine responds to your myeloma-related questions and concerns via phone and email, providing the most accurate information in a caring and compassionate manner. We also sustain a network of myeloma support groups, training hundreds of dedicated patients, care partners, and nurses who volunteer to lead these groups in their communities.
ADVOCACY We empower thousands of individuals who make a positive impact each year on issues critical to the myeloma community. In the U.S., we lead coalitions to represent the interests of the myeloma community at both federal and state levels. Outside the U.S., the IMF’s Global Myeloma Action Network works to help patients gain access to treatment.
Learn more about the ways the IMF is helping to improve the quality of life of myeloma patients while working toward prevention and a cure. Call us at
, or visit myeloma.org .
You are not alone
The International Myeloma Foundation (IMF) is here to help you. The IMF is committed to providing information and support for patients with multiple myeloma (which we refer to simply as “myeloma”) and their care partners, friends, and family members.
We achieve this through a broad range of resources available on our website myeloma.org, and through numerous programs and services such as seminars, webinars, workshops, and the IMF InfoLine, which consistently provides the most up-to-date and accurate information about myeloma in a caring and compassionate manner. Contact the IMF InfoLine at 1.818.487.7455 or InfoLine@myeloma.org.
What you will learn from this booklet
Myeloma is a cancer that is not known to most patients at the time of diagnosis. To play an active role in your own medical care and to make good decisions about your care with your doctor, it is important and helpful to learn about myeloma, as well as its treatment options and supportive care measures.
The IMF’s Understanding-series publications address treatments for myeloma, supportive care measures, and the tests that are used to diagnose, monitor, and assess disease status throughout its course.
This booklet discusses the drug Abecma® (also known as idecabtagene vicleucel, its generic drug name) or “ide-cel” for short. Abecma was approved by the U.S. Food and Drug Administration (FDA) in March 2021 for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM).
If you are newly diagnosed with myeloma, we suggest that you read the IMF’s publication Patient Handbook for the Newly Diagnosed, which will help you to better understand this complex disease.
To learn about myeloma in later disease settings, read the IMF’s publication Concise Review of Relapsed and Refractory Myeloma.
Words in bold+blue type are explained in the “Terms and definitions” section at the end of this booklet. A more comprehensive glossary can be found in the IMF’s publication Understanding Myeloma Vocabulary located online at glossary.myeloma.org.
If you are reading this booklet in electronic format, the light blue links will take you to the corresponding resources. All IMF publications are free-of-charge and can be downloaded or requested in printed format at publications.myeloma.org.
Immunotherapy
The immune system is comprised of a complex network of cells, tissues, organs, and the substances they make. The immune system involves multiple mechanisms that work together to protect and defend the human body from external threats (e.g., bacteria, viruses, toxins) and from internal threats such as cancer. The immune system helps to destroy infected and diseased cells and to remove cellular debris while protecting healthy cells.
Myeloma interferes with the normal function of the body’s immune system. Designed to protect us from harm, the immune system is very complex.
To learn more about the immune system in the context of myeloma, read the IMF’s publication Understanding the Immune System in Myeloma.
Immunotherapy is treatment that enhances the body’s natural defenses to protect us from harm. Abecma is a type of immunotherapy, a personalized chimeric antigen receptor (CAR) T-cell therapy that is manufactured for each individual patient using the patient’s own T cells, and is delivered as a one-time infusion.
T cells (T lymphocytes) are a type of white blood cell. T cells are an important part of our immune system by helping the body fight infection and disease.
T cells can be distinguished from other white blood cells by the presence of a T-cell receptor (TCR) on the cell surface. T cells can recognize and bind to specific antigens, thereby triggering an immune response.
T cells can recognize myeloma cells as foreign and cancerous. However, as myeloma grows, the number of T cells is significantly reduced and their function blocked, thus allowing the myeloma to continue to grow. What is needed are T cells which recognize myeloma cells as foreign and are capable of destroying them.
Indications for the use of Abecma
In March 2021, Abecma became the first-in-class B-cell maturation antigen (BCMA) -directed CAR T-cell therapy approved by the FDA for the treatment of adult patients with RRMM after 4 or more prior lines of therapy, including an immunomodulatory agent , a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Abecma is the first CAR T-cell therapy for myeloma to be approved by the FDA, and it is the first to target BCMA.
In April 2024, the FDA approved Abecma for the treatment of RRMM after 2 or more prior lines of therapy. This approval is based on data from the KarMMa-3 phase III clinical trial, which demonstrated a progression-free survival (PFS) of roughly 13 months in the Abecma arm of the study vs. 4 months in the control arm with standard regimens. This new FDA approval expands the previous indication, making this important CAR T-cell therapy available to patients earlier in their treatment journey.
How Abecma works
CAR T-cell therapy with Abecma takes the patient’s own T cells and engineers them to identify the B-cell maturation antigen (BCMA) receptor on the surface of myeloma cells, bind to this receptor, and destroy the myeloma cells.
BCMA is a protein involved in myeloma cell growth and survival. BCMA is found on the surface of nearly all cells in patients with myeloma, both on myeloma cells and on healthy cells as well. BCMA is also known as tumor necrosis factor receptor superfamily member 17 (TNFRSF17).
Collection
The patient’s own T cells are collected by a procedure called apheresis, sometimes also called leukapheresis. Apheresis is a procedure whereby whole blood is taken from the patient, then passed through a machine that separates the blood into its individual components so that one particular component can be collected. The remaining blood components are then immediately re-infused back into the bloodstream of the patient. This is similar to how a patient’s own stem cells may be collected for an autologous transplant.
Manufacturing
The harvested T cells are sent to a highly specialized manufacturing laboratory to be engineered to attack the myeloma. The laboratory inserts a gene into the patient’s T cells, genetically modifying the T-cells to recognize and attach to BCMA. This creates the Abecma BCMA T-cell product that is unique to the patient.
The patient’s genetically engineered T cells are grown in the lab until millions of cells have multiplied, enough active T cells for the therapy to be effective. This process usually takes approximately 4 weeks.
Preparation
Approximately 5 days before the CAR T cells are reinfused, the patient begins a 3-day, low-dose chemotherapy regimen in order to prepare the body to more easily receive the CAR T cells.
Infusion
Approximately 4 weeks after the initial T-cell collection, the Abecma BCMA T-cell product is administered to the patient by way of an infusion through an intravenous (IV) catheter. The Abecma dose can be administered in one or more infusion bags, with up to 30 minutes for each infusion bag.
The infused CAR T cells immediately begin to seek out the myeloma cells in the body and attack them. As a precaution, due to the specialized nature of T-cell therapy and the possibility of side effects during this process, many patients are admitted to the hospital for about a week. Abecma must be administered at a certified treatment center.
Activation
Abecma recognizes and binds to BCMA on myeloma cells, leading to the destruction of BCMA-expressing myeloma cells. The CAR T cells become activated and begin to directly destroy the cell and also to engage other parts of the immune system to help.
Monitoring
Monitoring ensures that the Abecma treatment is working and helps with identifying any potential side effects. For at least 7 days after receiving the infusion of Abecma, the patient is monitored daily at the certified healthcare facility where the treatment was administered.
For at least 4 weeks after the infusion of Abecma, the patient should remain within 2 hours travel to the certified healthcare facility where the treatment was administered. This is the approximate timeline of treatment with Abecma, but every patient is different and the total time it takes to complete the process may vary.
Effects on ability to drive and operate machinery
For at least 8 weeks following the Abecma infusion, you may be at risk for temporary memory and coordination problems, sleepiness, confusion, dizziness, and seizures. Don’t drive, operate heavy machinery, or engage in any activities that could be dangerous or hazardous if you are not mentally alert. Be sure to discuss with your healthcare team any potential issues that you may be experiencing.
Important safety information
Abecma may cause side effects that are severe or life-threatening. Immediately contact your doctor or get emergency help if you experience any of the following:
¡ difficulty breathing,
¡ fever of 100.4°F/38°C or higher,
¡ chills or shivering,
¡ confusion,
¡ dizziness or lightheadedness,
¡ shaking or twitching (tremors),
¡ fast or irregular heartbeat,
¡ severe fatigue,
¡ severe nausea, vomiting, or diarrhea.
Do not donate blood, organs, tissues, or cells for transplantation.
Treatment with Abecma may cause a false-positive human immunodeficiency virus (HIV) test result by some commercial tests.
Patients treated with Abecma may develop secondary malignancies and should be monitored life-long.
Possible side effects of Abecma
The safety profile of Abecma is generally predictable, with side effects that are mostly low-grade, with early onset and resolution.
Tell your doctor immediately if you develop any of these or other symptoms after receiving Abecma. The side effects that occurred in 20% or more patients in the clinical trial include CRS, infections, fatigue, musculoskeletal pain, hypogammaglobulinemia, diarrhea, upper respiratory tract infection, nausea, viral infections, encephalopathy (disease in which the functioning of the brain is affected), edema, fever, cough, headache, and decreased appetite. These are the most common of the possible side effects of Abecma.
Cytokine Release Syndrome (CRS)
Cytokines are proteins that circulate in the bloodstream, usually in response to infection. Cytokines can stimulate or inhibit the growth or activity in other cells.
Cytokine release syndrome (CRS) is a potentially fatal, uncontrolled immune reaction in which cytokines become highly elevated and trigger an overwhelming immune system response. A cytokine “storm” can
seriously damage body tissues and organs. The CRS toxicity is a result of the active destruction of the myeloma cells by the BCMA-targeted T cells.
Symptoms of CRS, which can be severe or fatal, may include fever, difficulty breathing, dizziness or lightheadedness, nausea, headache, fast heartbeat, low blood pressure, or fatigue. Immediately tell your doctor if you develop any of these symptoms or if you experience any other potential side effects of Abecma.
In patients treated with Abecma in the KarMMa clinical trial, CRS of any grade occurred in 85% of patients using the Lee grading system. The median time to onset of CRS was 1 day after the infusion, and the median duration of CRS was 7 days.
Other Side Effects
Abecma can lower one or more types of your blood cells, which may make you feel weak or tired, or increase your risk of severe infection or bleeding. Abecma can increase the risk of life-threatening infections that may lead to death. Tell your doctor immediately if you develop a fever, are feeling tired, or have bruising or bleeding.
Clinical trial experience with Abecma
A clinical trial is a medical research study with people who volunteer to test scientific approaches to a new treatment or a new combination therapy. Each clinical trial is designed to find better ways to prevent, detect, diagnose, or treat cancer and to answer scientific questions.
Numerous clinical trials with Abecma are ongoing, and there may be some clinical trials that are enrolling patients. Contact the IMF InfoLine or visit clinicaltrials.gov for up-to-date information.
Access to Abecma
Abecma is administered only at certified treatment centers. More information is available at Abecma.com or call 1.888.805.4555.
Abecma is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS); please visit AbecmaREMS.com or call 1.888.423.5436.
In closing
This booklet is not meant to replace the advice of your doctors and nurses who are best able to answer questions about your specific healthcare management plan. The IMF intends only to provide you with information that will guide you in discussions with your healthcare team. To help ensure effective treatment with good quality of life, you must play an active role in your own medical care.
We encourage you to visit myeloma.org for more information about myeloma and to contact the IMF InfoLine with your myeloma-related questions and concerns. The IMF InfoLine consistently provides the most up-to-date and accurate information about myeloma in a caring and compassionate manner. Contact the IMF InfoLine at 1.818.487.7455 or InfoLine@myeloma.org.
Terms and definitions
The following selected terms are used in this booklet, while a more complete compendium of myeloma-related terms can be found in the IMF’s publication Understanding Myeloma Vocabulary located online at glossary.myeloma.org.
B-cell maturation antigen (BCMA): A protein involved in myeloma cell growth and survival. BCMA is found on the surface of cells in all patients with myeloma. Also called “tumor necrosis factor receptor superfamily member 17 (TNFRSF17).”
Cancer: A term for diseases in which malignant cells divide without control. Cancer cells can invade nearby tissues and spread through the bloodstream and lymphatic system to other parts of the body.
Chimeric antigen receptor (CAR) T-cell therapy: In myeloma, this immunotherapy involves collecting the patient’s T cells, engineering them in a laboratory, and multiplying the engineered T cells for re-infusion into the patient to attack the patient’s own myeloma cells.
Frontline therapy: A general term for the initial treatment used in an effort to achieve response in a newly diagnosed myeloma patient. See “Induction therapy ” and “ Response or remission.”
Generic drug name: A brand name identifies a drug as property of the company that receives approval for it from a governmental regulatory agency, such as the U.S. Food and Drug Administration (FDA). After a drug goes “off patent,” other companies may make generic versions of the drug under a generic name that refers to the chemical makeup of a drug.
Hypogammaglobulinemia: A laboratory diagnosis made when the immune system is not producing enough immunoglobulin G (IgG) in the blood.
Immunomodulatory agent: A drug that can modify, enhance, or suppress the functioning of the immune system. An immunomodulatory agent is sometimes called an “immunomodulatory drug (IMiD®).”
Induction therapy: The initial treatment given to a patient in preparation for an autologous stem cell transplant (ASCT). See “ Frontline therapy ” and “ Line of therapy.”
Infusion: Delivering fluids or medications into the bloodstream over a period of time.
Intravenous (IV) infusion: Administered into a vein.
Line of therapy: A term used to calculate the number of therapies a patient has received. A line of therapy is 1 or more complete cycles of a regimen that can consist of a single agent, a combination of several drugs, or a planned sequential therapy of various regimens. Also see “ Induction therapy.”
Median: The mean (middle) of two central numbers in a series of numbers. For example, “median progression-free survival (mPFS)” means that half the patients had remissions that were shorter and half the patients had remissions that were longer than the mPFS.
Monoclonal antibody: An antibody manufactured in a lab rather than produced in the human body. Monoclonal antibodies are specifically designed to find and bind to cancer cells and/or immune system cells for diagnostic or treatment purposes. Monoclonal antibodies can be used alone, or they can be used to deliver drugs, toxins, or radioactive material directly to tumor cells.
Multiple myeloma: A cancer of the bone marrow plasma cells, white blood cells that make antibodies. Cancerous plasma cells are called myeloma cells.
Progression-free survival (PFS): The length of time during and after the treatment of myeloma that a patient lives with the disease but the myeloma does not get worse. In a clinical trial, PFS is one way to measure how well the treatment is working. See “ Progressive disease.”
Progressive disease: Myeloma that is becoming worse or relapsing, as documented by tests. Defined as an increase of ≥ 25% from the lowest confirmed response value in the myeloma protein level and/or new evidence of disease.
Proteasome: A joined group (“complex”) of enzymes (“proteases”) that break down the damaged or unwanted proteins in both normal cells and cancer cells into smaller components. Proteasomes also carry out the regulated breakdown of undamaged proteins in the cell, a process that is necessary for the control of many critical cellular functions. These smaller protein components are then used to create new proteins required by the cell. This is important for maintaining balance within the cell and for regulating cell growth.
Proteasome inhibitor: Any drug that interferes with the normal function of the proteasome. See “ Proteasome.”
Refractory: Disease that is no longer responsive to standard treatments. Myeloma is refractory in patients who have had progressive disease either during treatment or within 60 days following treatment. Most clinical trials for advanced disease are for patients with relapsed and/or refractory myeloma.
Relapse: The reappearance of signs and symptoms of myeloma after a period of improvement. Patients with relapsed disease have been treated, then developed signs and symptoms of myeloma at least 60 days after treatment ended. Most clinical trials for advanced myeloma are for patients with relapsed and/or refractory disease.
Side effect: An unwanted or unexpected effect caused by a drug. Also known as adverse reaction or adverse event (AE).
INTERACTIVE RESOURCES AT A GLANCE
