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Melphalan is a small molecule, bifunctional alkylating agent of the nitrogen mustard class used primarily as chemotherapy. It exerts its antineoplastic effects by binding to the N7 position of guanine in DNA, causing interstrand and intrastrand cross-links that inhibit DNA and RNA synthesis, leading to cell death in both dividing and non-dividing tumor cells[1][2][7][8]. Melphalan is indicated for the treatment of multiple myeloma (including as high-dose conditioning before hematopoietic stem cell transplant), ovarian carcinoma, and uveal melanoma with unresectable hepatic metastases[1][2]. It may also be used off-label for other cancers such as breast cancer and amyloidosis[4]. Melphalan can foster an immunogenic microenvironment by inducing immunogenic cell death and modulating immune responses, which has implications for adoptive T-cell therapy[6].
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