Clinical trials for multiple myeloma treatment

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Today there are 104 clinical trials for multiple myeloma:

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What is the treatment for multiple myeloma?

Multiple myeloma treatment is complex and varied as, to date, there is no definitive cure for this haematological cancer. However, there are treatment options that can address the disease and improve patients’ quality of life. These therapeutic approaches are individually tailored, taking into account the stage of the disease, age and general health of each patient. The main objective of the multiple myeloma treatment is to improve patient survival and quality of life. This involves reducing the symptoms associated with the disease, slowing disease progression and seeking prolonged remissions.  


As multiple myeloma is an incurable disease, it is essential that affected individuals take a holistic approach to their care. In addition to medical treatment, a balanced and healthy diet plays an important role in the patient’s well-being. It is also recommended to seek psychological support to cope with the emotional impact of the disease. Joining a patient advocacy group can provide additional support throughout the cancer process. 


There are two main types of multiple myeloma: smouldering and active. The smouldering multiple myeloma doesn’t present symptoms. Usually diagnosed at an early stage, this type  of multiple myeloma has a minimal extension and low malignancy. The best approach is an active surveillance, delaying the initiation of treatment until symptoms appear. When myeloma symptoms appear, patients with active myeloma need immediate treatment and are divided into two groups:


  • Patients eligible for stem cell transplantation.

  • Patients who are physically ineligible for a stem cell transplant.  


Multiple myeloma is a disease that requires personalised treatments, making it crucial to classify it according to age, general health, comorbidities and other personal factors. The therapeutic approach to multiple myeloma is divided into different phases, with the last phase being closely linked to clinical trials. In some cases, the initial treatment may not be effective and additional therapeutic options may be required, among which clinical trials play a prominent role due to the innovative specialisation of their treatments.

Phase 1: induction therapy

Induction therapy is the first line oftreatment for multiple myeloma that patients receive. Its main goal is the growth control exhibited by cancer cells, to improve the patient’s response to therapy and to allow stem cells to be harvested for transplantation in the second phase. This therapy combines drugs that are administered throughout the body via the bloodstream, known as systemic drug therapy. In this first phase, two or three treatments are usually administered together: 


  • Corticosteroid therapy: This treatment uses high doses of steroids to control inflammation by destroying malignant lymphocytes, thereby improving the body’s immune response. Corticosteroids commonly administered include dexamethasone or prednisone. 
  • Immunotherapy: This treatment helps the immune system fight cancer by blocking the pathways myeloma uses to evade the body’s immune response. This allows the immune system to identify and destroy cancer cells. Immunotherapies used to treat this disease include: 
    • Immunomodulatory agents: the first such agent used for multiple myeloma was thalidomide, which has a high risk of causing neuropathy, among other adverse effects. Currently, lenalidomine is preferred because it is a more potent version with fewer side effects than thalidomide
    • Proteasome inhibitors: these are drugs that work by blocking a cellular structure called the proteasome, which breaks down damaged proteins. In multiple myeloma, these malignant cells overproduce abnormal proteins, which can lead to their uncontrolled proliferation. Proteasome inhibitors cause a build-up of proteins inside the cancer cells, leading to their destruction. Proteasome inhibitors to treat multiple myeloma include bortezomib, ixazomib and carfilzomib.
    • Monoclonal antibodies: Antibodies are proteins produced by the immune system to defend against infection. By creating synthetic versions, also known as monoclonal antibodies, they can be designed to specifically target certain proteins, such as proteins found on the surface of myeloma cells. In this way, these synthetic antibodies can selectively attack cancer cells and aid in the treatment of multiple myeloma. Commonly used monoclonal antibodies include daratumumab, isatuximab and elotuzumab.
    • Nuclear export inhibitor: The nucleus of a cell houses DNA, which is essential for the production of proteins that are necessary for the proper functioning of a cell. The XPO1 protein helps transport other proteins from the nucleus to other parts of the cell. The drug selinexor is a nuclear export inhibitor that blocks the XPO1 protein. In doing so, the myeloma cell cannot transport proteins from its nucleus and, as a result, dies.

    • Biospecific T-cell activators (BiTE): teclistamab is a type of immunotherapy calledBiTEthat attaches to T-cells and the BCMA protein on myeloma cells. It works together with the immune system to fight cancer cells. It is used to treat multiple myeloma that has not responded to the previous 4 other treatments.

  • Chemotherapy: Although no longer administered as the main treatment, it is still used in some cases. Chemotherapy destroys malignant cells and inhibits their growth, although it can also affect healthy cells and cause side effects. To improve its effectiveness and minimise adverse effects, it is often combined with new drugs. Chemotherapy drugs administered include cyclophosphamide, etoposide, doxorubicin, melphalan, bendamustine and others. The medical team will rule out the use of some drugs that may cause bone marrow damage, such as melphalan, if the patient is a candidate for autologous transplantation.  


These therapies are often combined with each other to determine the most effective approach to treat multiple myeloma and to reduce adverse effects. In addition, as long as the patient is a candidate, they facilitate the process of autologous stem cell transplantation. 

Phase 2: consolidation therapy

This second phase of treatment aims to destroy any malignant cells that have remained in the body.


Patients, usually under the age of 65, who have achieved complete or total remission of the disease and are in good enough health to receive high doses of chemotherapy, are given the option of undergoing a transplant. Transplant options may include: 


  • Autogenous stem cell transplantation: This procedure, which is performed in one or two consecutive interventions, is carried out if the induction treatment has allowed for the collection of healthy stem cells from the patient himself. After administering high doses of chemotherapy in this second phase, the healthy stem cells are returned to the bloodstream through an intravenous infusion. The main goal is to protect the bone marrow from the side effects of chemotherapy while restoring the production of normal blood cells. In this way, the aim is to maintain bone marrow function after aggressive treatment, allowing the body to continue producing the blood cells necessary for its proper functioning. 


  • Allogeneic stem cell transplantation: this has the same purpose as autologous transplantation, except that the stem cells the patient receives come from a donor. Patients who receive this type of transplant (allogeneic transplant) are at increased risk of infection or graft-versus-host disease (GVHD). 


For patients who are not physically fit for transplantation but have stable tumour markers, treatment is usually stopped. Another option is to continue maintenance therapies in the next phase, but at lower doses. 

Phase 3: maintenance therapy

Maintenance therapy is a strategy that helps to prolong disease remission over a longer period of time. In this phase, drugs are administered at lower doses or less frequently, while maintaining the efficacy of induction or transplant phase therapies. Medical research is currently underway to determine the best treatment to prevent recurrence of multiple myeloma. Although previously used therapies are employed, there are some drugs, based on the results of clinical trials, that tend to be the most commonly used in this maintenance phase:  


  • Lenalidomine, due to its low neurotoxicity.  

  • Both bortezomib and ixazomib are recommended for patients with cytogenetic abnormalities, i.e. changes in chromosomes t(4;14), t(14;16), del (17p) and t(14;20). 

The clinical trial: the opportunity to receive the latest multiple myeloma treatment

Multiple myeloma clinical trials are of vital importance to increase knowledge about the disease, which could lead to the development of more precise drugs and the delivery of new treatments that are better suited to the specific needs of each individual patients.


Research into multiple myeloma treatment has several goals, including: developing curative treatments, achieving prolonged remissions (as seen in the maintenance phase) and improving quality of life during the cancer process.   


Participating in a clinical trial can provide significant benefits, such as access to new therapies and increased follow-up by healthcare staff. This participation in research can have a positive impact on the progression of multiple myeloma and on the patient’s overall well-being. 

Treatments for relapsed or refractory multiple myeloma

Almost all patients with multiple myeloma will relapse or the disease will become resistant to therapies (refractory), and there are several treatment options available to address these situations. These options include standard treatments that are commonly used as well as investigational treatments that may only be available through clinical trials.    


If previous treatments have had a positive response for the patient, they may be re-administered.


Conventional treatments for recurrent or refractory myeloma may include different combinations of the drugs used in the induction phase. These approaches aim to control cancer growth and improve the patient’s quality of life. 


In addition to previous treatments, there are clinical trials evaluating new therapies and innovative approaches to treating multiple myeloma, the aims of which are: 


  • Treating cancer with:   

    • New drugs: CAR cells versus BCMA.  

    • New treatment combinations: new monoclonal antibodies together with existing ones (daratumumab) or immunomodulatory agents (lenalidomide).  

    • New routes of drug administration: oral, intravenous, cutaneous, etc.  

  • Controlling cancer symptoms and reducing side effects.  

  • Prevent recurrence of multiple myeloma.  

  • Mitigate long-term side effects. 

  • Maintaining remission for a longer period of time. 


The treatment of multiple myeloma is complex and must be adapted to each patient’s individual needs, so it should always be directed and supervised by a medical team specialized in hematology and oncology, which can evaluate and adjust the treatment plan according to the patient’s evolution.


American Cancer Society, 2023: Mieloma múltiple, Inmunoterapia

Instituto Nacional del Cáncer, 2023: Inmunoterapia para tratar el cáncer, Tratamiento de las neoplasias de células plasmáticas (incluso mieloma múltiple)

Comunidad Española de Pacientes con Mieloma Múltiple, 2023: ¿Qué es el mieloma?

Clínic Barcelona, 2023: ¿Qué es el mieloma múltiple?

Asociación Española de Afectados por Linfoma, Mieloma y Leucemia (AEAL), 2023: Mieloma Múltiple España

Leukemia & Lymphoma Society, 2023: Mieloma

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