Multiple Myeloma: Latest Research

Approved by the Cancer.Net Editorial Board, 07/2018

ON THIS PAGE: You will read about the scientific research being done now to learn more about multiple myeloma and how to treat it. Use the menu to see other pages.

Doctors are working to learn more about myeloma, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your doctor about the diagnostic and treatment options that are best for you. Below are some of the areas researchers are looking into. With the fast pace of myeloma research, patients are encouraged to ask about available clinical trials.

  • Expanded use of stem cell transplantation. Although autologous (AUTO) stem cell transplantation is a standard treatment option for myeloma, researchers are studying the benefits of tandem (double) AUTO transplantations, ALLO transplantations, and tandem AUTO mini-allogeneic transplantations in some patients using very specific criteria. AUTO means the stem cells are from the patient. ALLO means that the stem cells came from a donor. The goal of these studies is to establish how these approaches fit best into doctors’ knowledge about current treatments. For more information about transplantation, read the Types of Treatments section.

  • New drugs. A variety of novel drugs are being studied for the treatment of relapsed myeloma and relapsed, refractory myeloma, including the following examples, with many more being studied:

    • A monoclonal antibody is a substance made in a laboratory that acts like the antibodies the body’s immune system naturally makes to fight diseases. Many of these drugs are being studied for multiple myeloma. One such drug, called isatuximab or SAR650984, is being studied. An anti-CD138 monoclonal antibody (indatuximab ravtansine) is also being studied. Elotuzumab and daratumumab were approved by the FDA in November 2015 and have both been shown to be highly effective, especially when combined with other new agents such as lenalidomide and bortezomib.

    • HDAC inhibitors are an area of active research, both as single drugs and in combination with other drug therapy. Drugs in this category include panobinostat, which is FDA approved, ACY1215, and AC241, which has shown encouraging results.

    • Lenalidomide and dexamethasone with or without elotuzumab, lenalidomide and dexamethasone with or without ixazomib (Ninlaro), and lenalidomide and dexamethasone with or without carfilzomib have all finished phase III clinical trials. These studies have shown substantial clinical benefit, leading to FDA approval.

    • The fibroblast growth factor receptor 3 (FGFR3) is expressed in approximately 15% of patients with myeloma. FGFR3 is involved in cell growth, angiogenesis, and wound healing. Drugs that inhibit FGFR3 are being developed to help control cancer in these patients.

    • Two experimental proteasome inhibitors, called oprozomib and marizomib (NPI-0052), have also been evaluated in clinical trials, with early results showing promise, particularly with marizomib combined with pomalidomide.

    • Late-phase III studies to treat myeloma are also underway with many new drugs that show promise. These include new chemotherapies such as melflufen (Evomela) and small molecule inhibitors such as venetoclax (Venclexta) and selinexor.

    Myeloma represents a new treatment paradigm (a set of assumptions and practices) in cancer because the new drugs that target the tumor cell, tumor-bone marrow interaction, and bone marrow environment can overcome normal drug resistance. Drugs are first tested using clinical trials with patients with advanced myeloma and then used to treat patients with earlier-stage myeloma.

  • Drug combinations. Most myeloma cells will eventually become resistant to standard chemotherapy, a condition called multidrug resistance. New drugs and combinations of approved drugs are being researched to provide more options for patients with myeloma. Many new drug combinations have been developed and are being studied in various settings, including:

    • Bortezomib and lenalidomide in combination with dexamethasone

    • Bortezomib, cyclophosphamide, and dexamethasone

    • Carfilzomib, lenalidomide, and dexamethasone

    • Ixazomib, lenalidomide, and dexamethasone

    • Pomalidomide, bortezomib, and dexamethasone

    • Carfilzomib, pomalidomide, and dexamethasone

    • Pomalidomide, ixazomib, and dexamethasone

    • Pomalidomide, dexamethasone, and clarithromycin (Biaxin)

  • Immunotherapy. This type of therapy is designed to boost the body’s natural defenses to fight cancer. It uses materials made either by the body or in a laboratory to improve, target, or restore immune system function. Vaccines are a type of immunotherapy being explored in the treatment of multiple myeloma.

    Checkpoint inhibitor therapies include antibodies to block PD-L1 on multiple myeloma cells and PD-1 on immune cells. PD-1 is found on the surface of T-cells, which are a type of white blood cell that directly helps body’s immune system fight disease. Because PD-1 keeps the immune system from destroying cancer cells, blocking PD-1 allows the immune system to better eliminate the disease. Currently, studies in advanced disease are taking place while clinical trials in early-stage disease are on hold due to safety concerns. New monoclonal antibodies such as immunotoxins and bi-pleuritic, also called BiTEs, target B-cell maturation protein, or BCMA.

    Chimeric antigen receptor (CAR) T-cells are another form of immune therapy in which the patient’s own immune cells are removed, modified to target their own myeloma cells, and then put back into the body to fight the myeloma.

    Research on using these therapies to treat advanced myeloma is still in its early phase. Learn more about the basics of immunotherapy.

  • Cytogenetics. Cytogenetics, which is the study of genetic changes in cells, and molecular studies may be performed on a tissue sample removed during a biopsy to find out how aggressive the cancer is. In myeloma, the genes in plasma cells are routinely studied using the FISH test to identify standard and high-risk disease. This may help guide treatment. More genetic tests are being developed, usually in ongoing research studies.

  • Minimal residual disease (MRD). As treatments have become increasingly effective in treating myeloma, new approaches to measure how well a treatment works have been developed, including MRD. This parameter may help guide treatment but is usually only used in research.

  • Palliative care. A limited number of clinical trials are underway to find better ways of reducing symptoms and side effects of current myeloma treatments to improve patients’ comfort and quality of life.

Looking for More About the Latest Research?

If you would like additional information about the latest areas of research regarding myeloma, explore these related items that will take you outside of this guide:

The next section in this guide is Coping with Treatment. It offers some guidance in how to cope with the physical, emotional, and social changes that cancer and its treatment can bring. Use the menu to choose another section to read in this guide.