Clinical Trials and Emerging Therapies for Multiple Myeloma

Some people with multiple myeloma may find clinical studies and emerging therapies to be the best option for treatment.

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Clinical Studies

Clinical studies are essential to the development of new multiple myeloma treatments and contribute significantly to developing a greater understanding of the disease. Almost all cancer treatments available today resulted from research conducted in clinical studies.

If you enroll in a clinical study, you will have the opportunity to be among the first to receive the newest drugs and therapies in development. However, it is important to understand that new treatments may be equivalent to, more effective than, or not as effective as existing treatments. They may also have unexpected side effects. That’s why new treatments must be studied in clinical studies first.

Because clinical study participation is a good option for many patients, it is important to understand what they are, how they work, and how to find a study that is right for you. Before a drug is considered for testing in people, it needs to show evidence of activity against myeloma in laboratory and animal studies—these are called preclinical studies.

Different Types of Clinical Studies

Types of clinical studies and what is invovled in each phase such as treatment dose, number of patients, and specific questions answered based on the results.

*The FDA approves treatments that are safe, effective, and shown to be better than the standard treatments available. When no standard treatment is available, the FDA may approve drugs based on study results of phase 2 studies. Conducted to receive FDA approval of new drugs, in most cases.

In addition to the new drugs currently being evaluated in clinical studies, drugs that have been previously approved to treat other diseases are being examined in clinical studies to determine whether they may be effective for treating multiple myeloma.

Some patients worry that participating in a randomized clinical study means that they receive only a placebo and no actual treatment. In fact, all patients in a clinical study receive treatment; one set of patients receives standard treatment (called the standard-of-care group) and the other set of patients receives the new treatment (called the new-treatment group). Physicians and nurses who conduct clinical studies are always the most familiar with the latest research and treatments, and the studies often take place at larger research institutions.

Interested in participating in a clinical study?

Be sure to talk with your doctor about which clinical studies are available and whether one of them may be right for you. 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.

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Emerging Therapies

All drugs and therapies that have shown activity in multiple myeloma cells during laboratory studies must be studied in patients by way of clinical studies before they are approved by the FDA for use in myeloma patients.

Emerging therapies for multiple myeloma are in phase 1, 2, and/or 3 clinical studies. They can be drugs in the same class as existing FDA-approved drugs, or they can be in a new class.

Emerging multiple myeloma therapies in development*

Drug Class Phase 3 Phase 1,2
Novel Agents Precision Medicine Venetoclax† Abemaciclib† Dabrafenib Erdafitinib† Trametinib
Cobimetinib† Enasidenib† Idasanutlin Vemurafenib
Novel Mechanism of Action Venetoclax† AMG-176 Duvelisib Nelfinavir TAK-981
AMG-232 Eltanexor Nirogacestat Tasquinimod
APG-2575 EZM0414 NMS-03597812 Telaglenastat
Azacitidine Ibrutinib ONC201 Vactosertib
BGB-11417 ION251 ORIC-533 Tinostamustine
BMF-219 INCB000928 Pirtobrutinib Pirtobrutinib
CFT7455 KTX-1001 PRT1419 PRT1419
COM902 Leflunomide PT-112 PT-112
CYT-0851 LOXO-338 Ricolinostat Ricolinostat
Disulfiram LP-118 Ruxolitinib Ruxolitinib
Immunotherapies Immuno-modulatory Agents Iberdomide Avadomide Modakafusp alfa
Naked Antibodies AB308 Feladilimab Mezagitamab TTI-621
ALT-803 GEN3014 NKTR-255 Tiragolumab
AO-176 GSK3174998 Relatlimab
BMS-986207 Lirilumab SAR442085
EOS884448 Magrolimab SEA-BCMA
Antibody-Drug Conjugates Belantamab mafodotin CC-99712 HDP-101 STI-6129
FOR46 MT-0169 STRO-001
Bispecific Antibodies and T-Cell Engagers Talquetamab ABBV-383 CC-95266 REGN5458 WVT078
AMG 701 Elranatamab REGN5459
Cevostamab HPN217 SAR442257
CC-92328 ISB 1342 SLAMF7 BATS
CC-93269 ISB 1442 VP301
CAR T-cell Therapies ALLO-605 CS1-CART JCARH125 PHE885
ALLO-715 CT103A LMY-920 STI-1492
bb21217 CYAD-211 MCARH125 UCARTCS1A
CART-ddBCMA Descartes-08 PBCAR269A Zevor-cel
CART-TnMUC1 Descartes-11 P-BCMA-101
NK cell and T-cell Therapies AGENT-797 FT576 MUC-1 RAPA-201
CYNK-001 Kappa 
CD28 T cells NEXI-002 TEG002
FT538 LAVA-051 NK-92 TriPRIL
Checkpoint Inhibitors Abatacept Durvalumab Pembrolizumab
Cemiplimab Ipilimumab TTI-622
Dostarlimab Nivolumab Zimberelimab

*Drugs listed are based on a search of with the following search parameters: multiple myeloma, United States, recruiting, not yet recruiting, or active not recruiting (last updated January 2024).

Being studied in the MyDRUG study.

Additionally, research into myeloma therapy is increasingly focusing on personalized medicine—the treatment of patients according to their unique, individual characteristics, such as immune profile and myeloma genetic (DNA) makeup.

Precision medicine uses molecularly targeted therapies—that is, drugs that target genetic errors (DNA mutations) in myeloma cells—and better enables the patient and the healthcare team to choose the myeloma drug(s) that will be most effective.

The MMRF MyDRUG study is evaluating drugs designed to target the mutations identified in myeloma patients. All of the drugs being investigated in MyDRUG have already been FDA-approved to treat other types of cancer, but their effectiveness in patients with multiple myeloma is not yet proven. The results from MyDRUG will hopefully allow doctors to select precision medicine treatments that are most likely to help patients achieve better treatment results (such as longer remissions).

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