Clinical trials and experimental therapies

Almost all cancer treatments today are a result of the research conducted in clinical trials. For this reason, clinical trials are critically important in the development of new multiple myeloma treatments and in helping doctors and researchers develop a better understanding of the biology of the disease.

For many people with multiple myeloma, participation in a clinical trial may be a good option for treatment. Be sure to talk with your doctor about which clinical trials are available and whether one of them may be right for you.

Here are a few things to know about clinical trials:

  • Broadly, there are three types of clinical trials, known as phases
    • Phase 1 determines safety and dosage, as well as how the drug is absorbed and acts in the body
    • Phase 2 evaluates effectiveness and safety
    • Phase 3 compares effectiveness and safety with standard treatment
  • In addition to the brand-new drugs currently being studied in clinical trials, drugs that have been previously approved to treat other diseases are being examined in clinical trials to determine whether they may be effective for treating multiple myeloma
  • Some patients worry that participating in a clinical trial may mean they receive only a placebo and no actual treatment. In fact, one set of patients in the trial receives standard treatment (called the control group) and the other set of patients receives the new treatment (called the experimental group)
  • Physicians and nurses who conduct clinical trials are often the most familiar with the latest research and treatments, and they are often located at larger research institutions. If you don’t live near the research institution, it may be possible for your care to be conducted in the office of your own oncologist, or your travel may be limited to a small number of visits to the larger institution
  • If you decide to participate in a clinical trial, you will receive a consent form that serves as your guide to all of the important information you need to know about the trial
  • In any clinical trial, you are allowed to quit at any point, for any reason

Interested in participating in a clinical trial? Talk to a Multiple Myeloma Research Foundation (MMRF) Patient Support Nurse to learn more, or use our clinical trials finder.

Emerging therapies

Some exciting areas of research in multiple myeloma include:

CAR-T therapy

Chimeric antigen receptor T-cell (CAR-T) therapy involves collecting immune cells called T cells from the patient’s own blood and genetically reprogramming them so they recognize and target a particular antigen on cancer cells. The reprogrammed CAR T cells are expanded by growing cells in a lab and are then infused back into the patient’s body, where they can attack and kill cancer cells.

CAR-T therapies have recently been approved to treat some other cancers, but they are still being investigated in clinical trials for multiple myeloma. Results reported for these trials so far have been promising, with many patients with relapsed or refractory disease achieving long-term remission.

Transduced T-cell receptors

Another experimental approach to engineering T cells in multiple myeloma uses transduced T-cell receptors. This involves introducing a T-cell receptor that recognizes a particular tumor antigen to the patient’s immune system.


Vaccines in development for multiple myeloma work by injecting either cancer cells or proteins expressed in cancer cells into the patient’s body. This activates the immune system against the cancer proteins in the vaccine, stimulating T cells to kill foreign invader cells—which include cancer cells within the body as well as the injected material.

PIs and IMiDs

Several studies are under way to explore new and better ways of combining certain proteasome inhibitors (PIs) and/or immunomodulatory drugs (IMiDs) with other multiple myeloma therapies.

Molecularly targeted therapies

New drugs and drug combinations that target specific proteins found on cancerous cells are currently being studied in clinical trials. These include a combination that inhibits the interaction between MDM2 and the tumor suppressor p53 for patients with relapsed multiple myeloma who have a chromosomal abnormality known as 17p deletion, a combination of a BRAF inhibitor and a MEK inhibitor for patients who have a genetic mutation known as BRAF V600E, and a pan-FGFR tyrosine kinase inhibitor for patients with a mutation of the FGFR3 gene.

Novel agents/mechanisms

Other new and emerging therapies with novel mechanisms of action currently being investigated include treatments for anemia and bone loss to potentially increase hemoglobin, hematocrit, and red blood cell counts in patients with myeloma, combinations of the proteasome inhibitor Kyprolis with a BTK or JAK1/2 inhibitor, combinations of the SINE Selinexor with a steroid or a proteasome inhibitor, and a combination of the BCL2 inhibitor venetoclax and a proteasome inhibitor.

You can learn more about experimental treatments in multiple myeloma here.