Drug intelligence / Profile preview

melphalan + thalidomide + dexamethasone

Development stage
Phase 2
Lead developer
GSK
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

Melphalan + thalidomide + dexamethasone is a combination chemotherapy regimen used primarily in the treatment of multiple myeloma, particularly for patients who are not eligible for stem cell transplantation or have relapsed/refractory disease. - **Melphalan** is an alkylating agent that crosslinks DNA and inhibits DNA and RNA synthesis, leading to cell death. - **Thalidomide** is an immunomodulatory drug (IMiD) with anti-inflammatory, antiangiogenic, and direct antineoplastic effects; it modulates the immune system and inhibits tumor growth by affecting cytokine production and angiogenesis. - **Dexamethasone** is a synthetic glucocorticoid corticosteroid that induces apoptosis in lymphoid cells and enhances the efficacy of other agents through its anti-inflammatory and immunosuppressive actions. This combination has demonstrated activity in heavily pretreated multiple myeloma patients with poor prognosis[2]. It can be associated with significant hematologic toxicity (notably neutropenia and thrombocytopenia), as well as nonhematologic side effects such as fatigue, neuropathy/paresthesia (from thalidomide), nausea, hyperglycemia, mood changes (from dexamethasone), among others[2][6][7].

Other names
melphalan/thalidomide/dexamethasoneMTD regimen
02

Targets

CRBN (Cereblon)DNAGR (Glucocorticoid receptor)TNFA (Tumor necrosis factor alpha (soluble))VEGFA (Vascular endothelial growth factor A)

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