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Clinical Trials

Taking part in a clinical trial may be the best treatment choice for some myeloma patients. Clinical trials are under way to develop treatments that increase the remission rate of myeloma or cure the disease. Today's standard treatments for cancer are based on earlier clinical trials. The Leukemia & Lymphoma Society (LLS) continues to invest funds in myeloma research.

Clinical trials can involve new drugs, new combinations of drugs or approved drugs being studied to treat patients in new ways such as new drug doses or new schedules to administer the drugs. Clinical trials are conducted worldwide under rigorous guidelines to help doctors find out whether new cancer treatments are safe and effective or better than the standard treatment.

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Current Myeloma Research and Clinical Trials

Below are some types of research and clinical trials under way:

Drugs and Drug Combinations. Recent advances in myeloma treatment have resulted in improved treatment responses and overall survival rates in both newly diagnosed and relapsed cases of myeloma. Eventually, however, nearly all patients will experience a relapse of the disease because, with time, myeloma cells become resistant to current drug therapies. This means there is a continuing role for the introduction of investigational agents that overcome drug resistance. Several new approaches, including combination therapies to counteract drug resistance, are being studied in clinical trials for myeloma treatment.

Immunotherapy. Various forms of immunotherapy are being studied in clinical studies, including:

  • Chimeric Antigen Receptor (CAR) T-Cell Therapy. This is a type of immunotherapy that consists of modifying a patient’s own immune cells to recognize and then attack cancerous cells. The patient’s T cells are genetically engineered to produce receptors on their surface called “chimeric antigen receptors (CARs).” The receptors recognize and bind to a specific target found on the cancer cells.
    • The B-cell maturation antigen (BCMA) is being targeted by CAR T cells in clinical studies for patients with relapsed or refractory myeloma.
    • Various ongoing studies are focusing on CAR T-cell therapies that target other cell antigens, such as CD19, CD38 and the signaling lymphocytic activation molecule F7 (SLAMF7), for the treatment of myeloma patients.
  • B-Cell Maturation Antigen (BCMA). This antigen, also called TNFRSF17 (part of the tumor necrosis factor superfamily of proteins), is an important cell surface protein that is involved in supporting the survival of myeloma cells. It is expressed at significantly higher levels in all myeloma cells, but not on other normal tissues (except normal plasma cells). A drug that is being studied is elranatamab (PF-06863135). This is an investigational B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody for patients with relapsed/ refractory multiple myeloma.
  • Dendritic Cell/Tumor Fusion Vaccines. Proteins on the surface of myeloma cells may be especially well-suited targets for vaccines. Dendritic cells are generally found in small amounts in the body and are responsible for immune responses against foreign substances. To create these fusion vaccines, cells are removed from the patient’s tumor and fused (mixed) with dendritic cells obtained from the blood, in order to stimulate a powerful antitumor response.

Proteasome Inhibitors (PIs). These drugs block proteasome activity, leading to cancer cell death. Several studies are examining the efficacy and safety of PIs, in combination with other agents, for the treatment of newly diagnosed and also relapsed and refractory cases of myeloma.

Stem Cell Transplantation. A number of approaches are under study, including the use of autologous and nonmyeloablative (reduced-intensity) allogeneic stem cell transplantation. For more information about all types of stem cell transplantation, see the free LLS information booklet, Blood and Marrow Stem Cell Transplantation.

Maintenance Therapy. Recent studies have shown that other drugs may be good maintenance therapy options for newly diagnosed patients who are not candidates for stem cell transplantation. There are several ongoing maintenance therapy trials evaluating the effectiveness of single and combination therapies.


For information about the drugs listed on this page, visit Drug Listings.

The Promise Study: Screening Individuals at High Risk of Myeloma. This research study identifies, screens and tracks individuals who are at high risk of developing myeloma. The goal of the PROMISE study is to increase early detection of myeloma precursor conditions in order to develop new therapies that prevent disease progression and improve survival. The study is looking for individuals from 45 to 75 years of age who are African American, and/or individuals with a first-degree relative with a plasma cell disorder such as myeloma. All participation is online or by mail. Call, send an email or visit the study’s website to learn more.

INSIGHT MM (Multiple Myeloma) Study. This study aims to evaluate the effectiveness of treatments for patients with myeloma from around the world. It is designed to collect data from more than 4,000 patients with newly diagnosed or relapsed/refractory myeloma in 15 countries for at least 5 years. The data is submitted directly by patients and include issues such as quality of life and the use of healthcare resources. Although the study is no longer enrolling new patients, new data derived from the study are published regularly. You can find more information through (identification code NCT02761187).

MyDRUG Trial. The ongoing MyDRUG (Myeloma Developing Regimens Using Genomics) trial uses genomic profiling to identify common genetic mutations and fluorescence in situ hybridization (FISH) to identify common translocations. Based on the results, patients are assigned to a specific arm of the study. The goal is to identify patients with high-risk myeloma so that they can be treated early in their disease course.

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