MULTIPLE MYELOMA KNOWLEDGE CENTER

Additional Experimental Treatments

Unless otherwise indicated, these treatments are being evaluated in relapsed and/or relapsed-refractory myeloma.

Name Company Description
Phase III 
Aplidin® (plitidepsin) PharmaMar, SA Marine-derived antitumor agent being evaluated in combination with dexamethasone (dex) in a trial being conducted in the US, Europe, and Australia, which has completed enrollment
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Denosumab (AMG 162) Amgen Human monoclonal antibody that targets the receptor activator of nuclear factor kappa B ligand (RANKL) and inhibits bone destruction; approved as Xgeva® for use in bone disease in solid tumors and as Prolia® for osteoporosis in women after menopause
Keytruda® (pembrolizumab, MK-3475) Merck Immno-oncology monoclonal antibody (anti-programmed death-1 [PD-1]) approved for use in melanoma and lung cancer; being evaluated in Phase III trials in combination with Revlimid-dex in newly diagnosed myeloma and in combination with Pomalyst-low-dose dex in relapsed/refractory myeloma, as well as with other agents in early-stage trials
Phase II
Imbruvica® (ibrutinib, PCI-32765) Pharmacyclics/Janssen Biotech, Inc. Anticancer agent (Bruton’s tyrosine kinase inhibitor) that targets B cells and is FDA-approved for use in Waldenström’s macroglobulinemia, mantle cell lymphoma, and chronic lymphocytic leukemia; being evaluated in a fully-enrolled Phase II trial and in combination with Kyprolis +/- dex and Pomalyst-dex in Phase I/II trials
Istodax® (romidepsin) Celgene Histone deacetylase inhibitor approved for use in lymphoma; being evaluated in combination with Pomalyst-dex and with Revlimid
LCL161 (SMAC mimetic LCL161) Mayo Clinic/National Cancer Institute (NCI) Oral apoptosis inhibitor being evaluated alone or with cyclophosphamide
Marizomib (NPI-0052) Triphase Research and Development 1 Corporation Novel proteasome inhibitor being evaluated in combination with Pomalyst-dex in a Phase I study LEARN MORE
Melflufen Oncopeptides AB A novel, more potent form of melphalan that is preferentially targeted to tumor cells
Mekinist® (trametinib) University of Arkansas Oral agent (kinase inhibitor) approved for use in melanoma
Oncolytic measles virus (MV-NIS) Mayo Clinic, University of Arkansas Genetically engineered measles virus being administered with or without cyclophosphamide; early activity demonstrated
PVX-410 OncoPep, Inc. Therapeutic cancer vaccine targeted against four proteins found on the surface of myeloma cells; being tested following stem cell transplant (trial set to begin) and alone and in combination with Revlimid in asymptomatic (smoldering) myeloma in a fully-enrolled study
Ricolinostat (ACY-1215) Acetylon Pharmaceuticals, Inc. Oral histone deacetylase (HDAC) inhibitor being evaluated in combination with various agents, including Velcade-dex (fully enrolled), Revlimid-dex (enrolling by invitation), and Pomalyst-dex
Selinexor (KPT-330) Karyopharm Therapeutics, Inc. Oral agent that blocks an essential protein for myeloma cell growth (XPO1); also known as a selective inhibitor of nuclear export (SINE) compound; being evaluated as part of combination therapy in several Phase I and II trials LEARN MORE
Sonidegib (erismodegib, LDE225) Mayo Clinic Oral agent (smoothened [SMO] inhibitor) being evaluated in combination with Revlimid following stem cell transplant
Sotatercept (ACE-011) Acceleron/Celgene Agent that stimulates bone formation; being evaluated for its effect on myeloma bone disease in patients with complete or very good partial responses who are not currently receiving antimyeloma therapy, as well as its antimyeloma activity with Revlimid-dex in a Phase I study
Treanda®, (bendamustine hydrochloride; known as Ribomustin® in Germany) Cephalon, a subsidiary of Teva Pharmaceuticals Alkylating agent FDA-approved for use in treating leukemia and lymphoma; approved in Germany for use in myeloma; being evaluated as part of various combination therapies for use in newly diagnosed and relapsed and/or refractory disease
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Vidaza® (azacitidine) Celgene Corporation Cytotoxic drug FDA-approved for the treatment of myelodysplastic syndromes (MDS) being evaluated in combination with Revlimid-dex in relapsed or refractory myeloma
Zolinza® (vorinostat, SAHA [suberoylanilide hydroxamic acid]) Merck Histone deacetylase (HDAC) inhibitor, FDA-approved for cutaneous T-cell lymphoma; although Merck is not continuing development of Zolinza in myeloma, it is being evaluated as part of combination therapy in a Phase I/II clinical trial and in a Phase III trial in the UK, and could potentially be used off-label. LEARN MORE
Phase I
ABBV-838 AbbVie Antibody-drug conjugate directed against the CS1 marker on myeloma cells
ABT-199 AbbVie/Genentech Oral agent (Bcl-2 inhibitor) that has shown activity against myeloma cells with the t(11;14) translocation ; being evaluated in combination with Velcade-dex in an MMRC-facilitated study
ACP-196 Acerta Pharma BV Novel agent (bruton tyrosine kinase inhibitor) being evaluated alone or in combination with dex
ACY-241 Acetylon Pharmaceuticals Oral histone deacetylase (HDAC) inhibitor being evaluated alone and in combination with Pomalyst-dex
Afuresertib (GSK2110183) GlaxoSmithKline Clinical activity noted with this oral anticancer agent (AKT inhibitor) in combination with Velcade-dex in an MMRC-facilitated trial; being evaluated in combination with Kyprolis
ALT-803 Altor Bioscience Corporation Novel anti-tumor immunotherapeutic (IL-15 superagonist complex)
AMG 224 Amgen Antibody-drug conjugate
Anti-BCMA CAR T cells National Cancer Institute (NCI) Engineered T cells directed against B-cell maturation antigen (BCMA)
Arava® (leflunomide, Sanofi City of Hope Medical Center/National Cancer Institute (NCI) Oral agent approved for use in treating rheumatoid arthritis that has shown anticancer activity in animals
AR-42 Ohio State University/Celgene Histone deacetylase (HDAC) inhibitor being evaluated in combination with Pomalyst-dex; trial set to begin
AT-101 (R-(-)-gossypol acetic acid) Mayo Clinic/ National Cancer Institute (NCI) Agent that stimulates apoptosis of cancer cells; being evaluated in combination with Revlimid-dex (trial set to begin)
Atezolizumab Hoffman-La Roche Immuno-oncology monoclonal antibody (anti-PD-L1) being evaluated alone or in combination with Revlimid
ATRA (all-transretinoic acid [tretinoin])/Celebrex® (celecoxib)/itraconazole University of Iowa Three-drug combination being evaluated as maintenance therapy given after an autologous stem cell transplant in patients with relapsed myeloma
bb2121 (CAR T cells) bluebird bio Engineered T cells directed against B-cell maturation antigen (BMCA)
Cord blood natural killer (NK) cells M.D. Anderson Cancer Center/Celgene Immune cells obtained from cord blood; being administered in conjunction with autologous stem cell transplant
CPI-0610 Constellation Pharmaceuticals Novel oral agent (BET protein bromodomain inhibitor)
CT7, MAGE-A3, and WT1 mRNA-electroporated Langerhans cells (LCs) Memorial Sloan-Kettering Cancer Center/Rockefeller University Immunotherapy (vaccine made with altered dendritic cells) being evaluated when administered following autologous stem cell transplantation
CUDC-907 Curis, Inc. Novel agent (phosphoinositide 3-kinase [PI3K] and histone deacetylase [HDAC] inhibitor) that inhibits cancer cell survival and growth
Dinaciclib (SCH 727965, MK-7965) Merck/Ligand Pharmaceuticals Cyclin-dependent kinase (CDK) inhibitor; being evaluated in combination with Velcade-dex in a Phase I trial; anti-myeloma activity observed in Phase II testing
DS-3032b Daiichi Sankyo/M.D. Anderson Cancer Center Oral agent (MDM2 inhibitor); trial set to begin
Durvalumab (MEDI4736) Medimmune/AstraZeneca Immuno-oncology monoclonal antibody (anti-PD-L1) being evaluated alone or in combination with Pomalyst +/- dex; trials in combination with Revlimid +/- dex and with tremelimumab (immuno-oncology anti-CTLA-4 monoclonal antibody) and autologous stem cell transplant are set to begin
Filanesib (ARRY-520) Array BioPharma Agent that inhibits kinesin spindle protein (KSP) that has shown encouraging activity in early trials; being evaluated in combination with Kyprolis; combination trial with Pomalyst-dex set to begin
GO-203-2C Genus Oncology/Dana Farber Cancer Institute/National Institutes of Health (NIH) Therapy targeted against MUC1, a protein that is increased on myeloma cells; being evaluated in combination with Velcade; trial set to begin
GSK2857916 GlaxoSmithKline Antibody-drug conjugate directed against B-cell maturation antigen (BCMA), which is highly epxressed on myeloma cells
I-131-CLR1404 Cellectar Biosciences Radiolabeled therapeutic being evaluated with or without dexamethasone in patients with relapsed or refractory myeloma who have previously received, or are intolerant of, an IMiD and a proteasome inhibitor
Ibrance™ (palbociclib) Pfizer Novel agent (CDK inhibitor) approved for use in breast cancer; being evaluated in combination with Revlimid-dex
Idasanutlin (RO5503781) Mayo Clinic/National Cancer Institute (NCI) Oral agent (MDM2 antagonist) that causes apoptosis of tumor cells; being evaluated in combination with Ninlaro and dexamethasone
Isatuximab (SAR650984) Sanofi Antibody directed against the CD38 marker on certain cancer cells; being evaluated as a single agent, and in combination with Revlimid-dex, Pomalyst-dex, and Kyprolis; encouraging activity seen in Phase I/II testing
Lirilumab (BMS-986015) Bristol-Myers Squibb Monoclonal antibody (anti-KIR) that activates natural killer cells; being evaluated in combination with Empliciti
Kineret® (anakinra) Mayo Clinic/National Cancer Institute (NCI) IL-1 receptor antagonist approved for use in treating rheumatoid arthritis being evaluated in combination with Relimid-dex in early stage myeloma; trial set to begin
KPT-8602 Karyopharm Therapeutics, Inc. Oral agent that blocks an essential protein for myeloma cell growth (XPO1); also known as a second-generation selective inhibitor of nuclear export (SINE) compound
LGH447 Novartis Oral anti-cancer agent (PIM kinase inhibitor); single-agent activity noted
MEDI-551 MedImmune/Astra Zeneca Monoclonal antibody (anti-CD19) being evaluated in combination with Revlimid-dex in a pilot study in patients with previously untreated myeloma
NY-ESO T cell receptor (TCR) Adaptimmune Engineered patient-derived T cells expressing a receptor that targets a cancer antigen (NY-ESO-1/LAGE-1 cancer testis antigen); results of an earlier trial demonstrate clinical activity and durable persistence
Opdivo® (nivolumab, BMS-936558, MDX-1106) Bristol-Myers Squibb Immuno-oncology monoclonal antibody (anti-programmed death-1 [PD-1]) being tested in combination with other immuno-oncology agents in myeloma and lymphoma
SL-401 Stemline Therapeutics Therapy directed against the interleukin-3 receptor (IL-3R) present on certain blood cancer cells; being evaluated in combination with Pomalyst-dex
SVN53-67/M57-KLH Peptide Vaccine Roswell Park Cancer Institute/National Cancer Institute (NCI) Vaccine being tested in patients with newly diagnosed myeloma who are receiving Revlimid maintenance therapy
Th1/Tc1 Immunotherapy National Cancer Institute (NCI) Immunotherapy (modified helper T cells) administered following autologous stem cell transplantation in patients diagnosed with high-risk myeloma
Tremelimumab MedImmune/Astra Zeneca Immuno-oncology monoclonal antibody (anti-CTLA-4) being evaluated in combination with durvalumab and autologous stem cell transplant; trial set to begin
Tumor-associated antigen (TAA)-specific cytotoxic T-lymphocytes (CTLs) for multiple myeloma (TACTAM) Baylor College of Medicine Immune cells directed against 5 tumor-associated antigens (NY-ESO-1, MAGEA4, PRAME, survivin, and SSX)
Ulocuplumab (BMS-936564) Bristol-Myers Squibb/Dana-Farber Cancer Institute Monoclonal antibody directed against CXCR4, a marker expressed on myeloma and other cancer cells; early patient access is available through a single named patient program
Urelumab (BMS-663513) Bristol-Myers Squibb Monoclonal antibody (anti-CD137 receptor) that stimulates immune responses against tumor cells; being evaluated in combination with Empliciti
VLX1570 Vivolux AB Novel proteasome inhibitor being evaluated in combination with low-dose dex
Wild-type reovirus University of Southern California/Oncolytics Biotech/National Cancer Institute (NCI) Virus being evaluated in combination with Velcade-dex
WT1-specific donor-derived T cells Memorial Sloan Kettering Cancer Center Donor white blood cells immunized against a protein expressed on myeloma cells (Wilms’ tumor protein, WT1) given after allogeneic stem cell transplant