Research in multiple myeloma

We are always learning more about cancer. Researchers and healthcare professionals use what they learn from research studies to develop better practices that will help prevent, find and treat multiple myeloma. They are also looking for ways to improve the quality of life of people with multiple myeloma.

The following is a selection of research showing promise for multiple myeloma. We’ve included information from PubMed, which is the research publication database of the National Library of Medicine. Each research article in PubMed has an identity number (called a PMID) that links to a brief overview (called an abstract). We have also included links to abstracts of the research presented at meetings of the American Society of Clinical Oncology (ASCO), which are held throughout the year. You can find information about ongoing clinical trials in Canada from and Clinical trials are given an identifier called a national clinical trial (NCT) number. The NCT number links to information about the clinical trial.


Researchers are trying to find better ways to help doctors make a prognosis (how likely it is that the cancer can be successfully treated or that it will come back after treatment) for multiple myeloma. They are also trying to determine the best treatment options based on certain characteristics of the disease, such as specific biomarkers. Biomarkers are substances, such as proteins, genes or pieces of genetic material like DNA and RNA, that are found naturally in the body. They can be measured in body fluids, including blood, lymph fluid and bone marrow, or on certain types of cells, such as cancer cells. Doctors can look for and measure these biomarkers as a sign that cancer is present or that it is responding to treatment.

Prognostic and predictive biomarkers for multiple myeloma can be used to help plan treatment. Doctors can use prognostic biomarkers to identify people who have a greater risk that the disease will progress or come back after treatment (recur or relapse). They can also look for predictive biomarkers to help them identify people who are more likely to respond to treatment. Researchers are looking at the following biomarkers to see if they can help doctors diagnose, predict a prognosis for and find out which treatments will benefit a person with multiple myeloma:

Gene-based tests find differences between normal genes and genes that are changed (mutated) in cancer cells. Molecular profiling is a type of test that allows researchers to study the genetic characteristics and find unique biomarkers in cancerous tumours. Researchers hope that gene-based tests will help doctors identify the best treatments for certain cancers, including multiple myeloma. Gene-based tests will also help doctors tailor more treatments to each person’s cancer based on their unique genetic makeup (Canadian Cancer Trials, NCT 01454297).

Gene expression profiling is used to analyze many genes at the same time to see which are turned on and which are turned off. Doctors have found several abnormal gene expression patterns in people with multiple myeloma that help them predict a prognosis. A drug response prediction (DRP) score was developed based on gene expression profiling to help doctors predict which tumours would respond to which treatments (Gene, PMID 29122646; Blood Cancer Journal, PMID 27471869).


Researchers are looking for new ways to improve treatment for multiple myeloma. Advances in cancer treatment and new ways to manage the side effects from treatment have improved the outlook and quality of life for many people with cancer. The following is noteworthy research into treatment for multiple myeloma.

Chemotherapy and targeted therapy

The goal of treatment for multiple myeloma is to reduce symptoms, slow the progression (advancement) of the disease and put it into remission. Remission means that most or all signs and symptoms of the disease disappear.

Induction therapy is often the first treatment for multiple myeloma. Induction therapy is usually a combination of chemotherapy, targeted therapy and corticosteroids. Researchers are studying these new combinations of drugs for induction therapy:

  • vincristine (Oncovin) or bortezomib (Velcade) with cyclophosphamide (Procytox), doxorubicin (Adriamycin) and dexamethasone (Decadron, Dexasone) (European Journal of Haematology, PMID 28009447)
  • high-dose gemcitabine (Gemzar), busulfan (Busulfex) and melphalan (Alkeran) for autologous stem cell transplant (Lancet Haematology, PMID 28522110)
  • lenalidomide (Revlimid), bortezomib and dexamethasone for autologous stem cell transplant (New England Journal of Medicine, PMID 28379796; Lancet, PMID 28017406)
  • bendamustine (Treanda), bortezomib and dexamethasone for people who can’t have high-dose chemotherapy (British Journal of Haematology, PMID 28169430)
  • lenalidomide and dexamethasone for high-risk smouldering multiple myeloma (Lancet Oncology, PMID 27402145)
  • daratumumab (Darzalex), bortezomib, melphalan and prednisone (New England Journal of Medicine, PMID 29231133)

Consolidation therapy is given after a stem cell transplant for multiple myeloma. One study looked at using bortezomib alone for consolidation (British Journal of Haematology, PMID 28382618).

Continuous treatment until the disease progresses, rather than treatment for a fixed amount of time, was studied in a clinical trial (Leukemia, PMID 28373701; British Journal of Haematology, PMID 28106903).

New drugs or combinations of drugs that researchers are testing to treat relapsed or refractory multiple myeloma include:


Proteasome inhibitors are a type of targeted therapy that block proteasomes. Proteasomes are enzymes that cancer cells need to grow. Blocking proteasomes can help stop the growth of cancer cells. A number of proteasome inhibitors are approved to treat multiple myeloma. One study looked at whether carfilzomib (Kyprolis) or bortezomib is the best to treat relapsed or refractory multiple myeloma (Lancet Oncology, PMID 28843768). Researchers are also looking at the following proteasome inhibitors to treat multiple myeloma:


Histone deacetylase (HDAC) inhibitors are targeted therapy drugs that block the actions of HDAC. HDAC is an enzyme that can make cancer cells grow. Blocking HDAC can slow or stop the growth of cancer cells. Researchers are looking at using the following HDAC inhibitors alone and in combination with other drugs to treat multiple myeloma:

Phosphoinositide 3-kinase (PI3K) inhibitors work by switching off PI3K. When PI3K is turned on in cancer cells, they continue to grow and divide out of control. Researchers are studying drugs that inhibit PI3K to see if they may be useful in treating multiple myeloma. They found that BKM120 made multiple myeloma more sensitive to bortezomib and seemed to help overcome resistance (Leukemia and Lymphoma, PMID 27439454). Researchers are also studying CUDC-907, which inhibits both PI3K and HDAC, to treat multiple myeloma (Lancet Oncology, PMID 27049457).


Immunotherapy boosts or helps the immune system find and destroy cancer cells. Researchers are studying the following types of immunotherapy for multiple myeloma.

Immune checkpoint inhibitors work by stopping cancer cells from affecting immune system cells in our bodies. The immune system normally stops itself from attacking healthy cells in the body by having some cells make specific proteins called checkpoints. Cancer cells sometimes use these checkpoints to avoid being attacked by the immune system. Immune checkpoint inhibitors are monoclonal antibodies that work by blocking checkpoint proteins so T cells (a type of white blood cell) can attack and kill cancers cells. Researchers are studying the immune checkpoint inhibitor pembrolizumab (Keytruda) in clinical trials to find out if it improves progression-free survival when it is combined with dexamethasone and either lenalidomide or pomalidomide (, NCT 02576977; Blood, PMID 28461396).

Engineered chimeric antigen receptor (CAR) T cells are a new type of immunotherapy. T cells are part of the immune system. They help fight infection and destroy abnormal cells, including cancer cells. Doctors take T cells from a person’s blood and genetically engineer, or modify, them in the lab so they have chimeric antigen receptors (CARs) on their surface. CARs are proteins that make the T cells recognize cancer cells. Doctors can grow CAR T cells in the lab until they have billions of them. They then infuse the CAR T cells back into the person’s blood, where they will multiply, and then target and kill cancer cells. Researchers have found that engineered CAR T cells are showing promise in treating multiple myeloma (ASCO, Abstract 3010, Abstract LBA3001, Abstract 8007)

Supportive care

Living with cancer can be challenging in many different ways. Supportive care can help people cope with cancer, its treatment and possible side effects. The following is noteworthy research into supportive care for multiple myeloma.

Acupuncture with methylcobalamin may help treat chemotherapy-induced peripheral neuropathy in people with multiple myeloma (BMC Cancer, PMID 28068938).

Palonosetron, aprepitant and dexamethasone may prevent nausea and vomiting after high-dose melphalan in autologous transplantation for multiple myeloma (International Journal of Hematology, PMID 27873176).

Learn more about cancer research

Researchers continue to try to find out more about multiple myeloma. Clinical trials are research studies that test new ways to prevent, detect, treat or manage multiple myeloma. Clinical trials provide information about the safety and effectiveness of new approaches to see if they should become widely available. Most of the standard treatments for multiple myeloma were first shown to be effective through clinical trials.

Find out more about clinical trials.