Drug intelligence / Profile preview

melphalan + capecitabine

Development stage
Unknown
Lead developer
GSK
Modality
Small Molecules
Administration
Oral, Intravenous
01

Overview

**melphalan + capecitabine** is a combination regimen of two chemotherapeutic agents, used experimentally and sometimes off-label in oncology for their synergistic cytotoxic effects against cancer cells. - **Melphalan** is an alkylating agent (class: nitrogen mustard) that crosslinks DNA and RNA, inhibiting DNA synthesis and function, leading to cell death in proliferating and non-proliferating tumor cells[1][5]. - **Capecitabine** is an oral prodrug of 5-fluorouracil (5-FU) and a pyrimidine antimetabolite; it is enzymatically converted to 5-FU within tumors, resulting in inhibition of thymidylate synthase and disruption of DNA synthesis[5]. This combination is not a standard, established regimen, but both agents are individually approved and widely used in multiple cancers (hematologic malignancies, breast, colorectal, and GI malignancies). The rationale for combining them lies in the potential for additive or synergistic efficacy, mainly in advanced or refractory cancers, but clinical evidence for their combined use is limited and mainly investigational or anecdotal.

02

Targets

DNATS (Thymidylate synthase)

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