Clinical trials for the treatment of myeloma that are currently underway include:

Stem Cell Transplantation

  • Allogeneic stem cell transplantation. Also used for other types of blood cancers, this treatment is being studied as it may be particularly helpful for younger myeloma patients with high-risk disease. Allogeneic stem cell transplantation has the potential for serious side effects and death as compared to the risks of autologous transplantation. It may be considered for a patient who has a suitable donor, often a sibling; other eligibility factors include patient age and absence of any other serious health conditions. Two main advantages of this type of therapy are: using cells from a healthy donor rather than the patient eliminates the chance that myeloma cells will be re-infused into the patient, and donor cells could also work as a form of immunotherapy, providing an antimyeloma effect.


  • Vaccines.  Proteins on myeloma cells   are being used to develop vaccines that may aid the patient’s immune cells in attacking and destroying his or her own cancer cells.
  •  Antibodies. Some immunotherapy drugs work in part by flagging substances on myeloma cells in order to make the cells more visible for attack by the patient’s own immune system. This approach may allow the immune system to be more effective by helping to reduce the immune suppression caused by the myeloma.
  • Chimeric antigen receptor (CAR)-T cells. T cells collected from the patient are modified so they express receptors that are specific to the patient’s cancer cells. When these cells are given back to the patient they are able to recognize and kill the patient’s cancer cells. Trials using natural killer cells from the patient or a donor are also underway.  

A clinical trial is a medical research study that tests new ways to prevent, screen, diagnose, or treat a disease. The word "clinical" means related to examining and treating patients.

Cancer clinical trials help to determine if a treatment, drug, or procedure shows a better way of treating a particular cancer, or provides a way to treat a condition for which there wasn't a treatment before.

It takes years of thought and planning to have a clinical trial accepted by the U.S. Food and Drug Administration (FDA). The FDA must approve the trial before researchers can recruit patients. Clinical trials allow patients access to the latest research treatments before they are available to the public.

Medical or research institutions, pharmaceutical and biotechnology companies, the government, and nonprofit groups who want to improve current cancer treatment can all sponsor clinical trials. Some trials are "investigator initiated," which means a researcher or doctor created the trial, but these trials face the same strict requirements.

Phase I trials answer the questions: Is the drug or treatment safe? What is a safe dose? What are the side effects? Phase I trials are small, each only involving 20-80 participants. These trials are usually the first time a drug or treatment has been tested in humans.

Phase II trials tell researchers if the drug or treatment is effective in a larger group (100-300 participants) and help identify safety concerns.

Phase III trials determine within a large group (1,000 – 3,000 participants) if the experimental drug or treatment works better than what is currently used and if there are additional side effects. Most Phase III trials are randomized, meaning some participants will receive the experimental drug or treatment while others will receive the standard drug or treatment currently accepted by the medical community as best for their cancer type (standard of care).

Phase IV trials allow researchers to learn more about the drug or treatment and its risks, benefits, and best use.

Usually FDA approval comes after large phase III trials. However, sometimes special consideration is given to treatments that show great promise in early trials. After enough data is gathered to prove that the new treatment is effective or more effective than current treatments, the FDA will investigate the findings and approve the treatment for use. After FDA approval, doctors can prescribe treatments to their patients without enrolling patients in clinical trials.

Each clinical trial recruits and screens participants through participating treatment centers. In order to learn if a drug is effective, the participants must be similar in the type of diagnosis and other factors. Trials are designed to be very specific so researchers can know that the drug or treatment being studied is causing changes.

Participants must be eligible in order to qualify for the trial. Some possible requirements for eligibility include:

  • Cancer type
  • Stage
  • Specific tumor types or genetic markers
  • Treatment history
  • Age
  • Other medical conditions

If you qualify for a trial, your healthcare team will provide you with all the information about the trial. They will tell you, in a process called informed consent, how the trial will be conducted and the possible risks and benefits of the trial. You will then sign a document stating that you understand the details of the trial and that you are willing to participate. Even after signing the document of informed consent, you may withdraw from the trial at any time for any reason.

The way you receive treatment depends on the specific clinical trial. Clinical trial treatments can come in many forms. These include but are not limited to the following:

  • Intravenous (IV) medications given through a port or IV line
  • Shots
  • Oral medications
  • Surgery
  • Radiation

As with any cancer treatment, clinical trial treatments may come with side effects. Your healthcare team will help you manage these side effects. It is very important to report to your healthcare team any side effects that you experience. Part of what researchers are studying is how the treatment affects the whole body, not just the cancer cells.

Patients who decide to participate in clinical trials receive excellent care and attention. Throughout the trial, your healthcare team will closely monitor you for improvements and adverse side effects.

Researchers want to control as much about your care as they can to make sure it is the treatment that is helping your cancer improve, and not something else. Because of this, you may have restrictions on what you can eat and what other medicines you can take. Although you should always follow your treatment plan and not miss appointments, doing so is especially important with a clinical trial. Clinical trials must have consistency among the participants to know if the treatment really is working.

Patients who are no longer helped by existing standard of care treatments may find that a clinical trial provides hope, but many patients begin their treatment program with a clinical trial because the treatment best suited to their situation is still being tested. Some clinical trials even exclude patients who have already received another form of treatment.

When participating in a clinical trial that evaluates if a new treatment is better than the current standard treatment for your cancer type, you will always receive treatment – either the standard of care or the new treatment being studied. By being in a clinical trial, you may receive the added benefits of a new treatment before it is available to the public.

In many cases, cancer research moves at a faster pace than the FDA treatment approval process. Researchers have made important discoveries about biomarkers and mutations that affect how cancers respond to treatment. Because of the long time it takes for a treatment to be tested through the clinical trials process, many of these breakthrough treatments are still only available through participating in a clinical trial.