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The MTZ regimen is an investigational combination therapy being evaluated by Sun Yat-sen University for the treatment of newly diagnosed primary central nervous system lymphoma (PCNSL). This regimen integrates high-dose methotrexate, a folate antimetabolite that inhibits dihydrofolate reductase to disrupt DNA synthesis; thiotepa, an alkylating agent that crosses the blood-brain barrier to cause DNA cross-linking; and zanubrutinib, a second-generation, oral, small molecule inhibitor of Bruton's tyrosine kinase (BTK). By targeting the B-cell receptor signaling pathway through BTK inhibition alongside traditional cytotoxic chemotherapy, the MTZ regimen aims to enhance therapeutic efficacy in PCNSL, a rare and aggressive form of extranodal non-Hodgkin lymphoma. The therapy is currently being studied in a Phase 2 clinical trial as a first-line treatment option.
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