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Thiotepa + pomalidomide is an investigational combination therapy consisting of two small molecule drugs, thiotepa and pomalidomide. Thiotepa is an alkylating agent that cross-links DNA, leading to inhibition of DNA replication and cell death, primarily used in conditioning regimens for stem cell transplantation and the treatment of various malignancies such as breast cancer, ovarian cancer, bladder cancer, and as part of high-dose chemotherapy protocols[4][6]. Pomalidomide is an immunomodulatory agent (IMiD) that exerts antineoplastic effects through multiple mechanisms including direct cytotoxicity against tumor cells, inhibition of angiogenesis, modulation of the immune system (enhancing T-cell and natural killer cell activity), and suppression of pro-inflammatory cytokines[3][8]. The combination has been studied in relapsed/refractory primary central nervous system lymphoma (PCNSL) with preliminary evidence suggesting potential efficacy[1][9]. Both agents are associated with myelosuppression as a significant adverse effect when used together[7].
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