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.

Receive one-on-one navigation from an LLS Clinical Trial Specialist who will personally assist you throughout the entire clinical-trial process: Click Here

Current Myeloma Research and Clinical Trials

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

Drugs and Drug Combinations. Recent advances in the treatment of myeloma have resulted in improved treatment response and overall survival rates in newly diagnosed patients and in patients with relapsed 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.

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 myeloma.

• Marizomib is a second-generation proteasome inhibitor that targets multiple activities in proteasome. It is under study for treatment of relapsed and refractory myeloma.
• Oprozomib is a new oral proteasome inhibitor that is being studied in various clinical trials for treating either newly diagnosed patients or patients with relapsed or refractory myeloma.

Patients With High-Risk Cytogenetic Abnormalities. A number of cytogenetic abnormalities are associated with poor prognosis, including (del)17p, t(4;14), t(14;16) and gain(1q21). See Table 2 on page 11 for more information. Data from recent studies has shown that three-drug combinations may improve outcomes for patients with high-risk cytogenetic abnormalities, compared to two-drug combinations. An aggressive treatment approach should be used for patients in this category. This approach may require patients to have breaks from treatment to recover, or to have shorter treatment cycles than they would if they were being treated with less aggressive regimens.

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.

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

• 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).
• 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.
• Monoclonal Antibodies. These immunotherapy agents are increasingly being used to treat myeloma, both to target the cancer cells directly and to modulate the patient’s immune system. While some monoclonal antibodies (called “naked” antibodies) work by themselves, others are coupled with a chemotherapy drug or attached to a radioactive particle (and are therefore known as “conjugated monoclonal antibodies”).
  • AMGEN-420 is a type of bispecific antibody that targets BCMA. Bispecific antibodies target the tumor and T cells, bringing them into contact, leading to cancer cell death.
  • Isatuximab, an investigational anti-CD38 monoclonal antibody, is being studied for the treatment of patients with relapsed and refractory multiple myeloma. (CD is the abbreviation for cluster of differentiation.)
  • Milatuzumab is an anti-CD74 monoclonal antibody that is being evaluated for the treatment of patients with relapsed and refractory myeloma.
  • Belantamab mafodotin is an antibody drug conjugate (ADC) that combines an antibody that targets BCMA with the toxic substance monomethyl auristatin F. The antibody binds to the antigen on the
    surface of tumor cells and then is absorbed together with the toxic substance. After the ADC is internalized, the toxic chemical is released, causing the malignant cell’s death.
  • Ulocuplumab (BMS-936564), which targets CD184 (also known as CXCR4), is under investigation in patients with relapsed and refractory myeloma.
• Chimeric Antigen Receptor (CAR) T-Cell Therapy. This is a type of immunotherapy that consists of engineering a patient’s own immune cells to recognize and then attack cancerous cells. This approach has shown very promising results in patients with blood cancers. 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. Click here to learn more about CAR T-Cell Therapy. 
  • 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.

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.

• Call: 617-582-7002
• Email: promisestudy@partners.org
• Website: promisestudy.org

Related Links

• Download or order The Leukemia & Lymphoma Society's free booklet, Myeloma.
• Clinical Trials 
• CAR T-Cell Immunotherapy