methotrexate + topotecan + cyclophosphamide

This investigational combination chemotherapy regimen is being evaluated in a Phase II clinical trial led by Nicklaus Children's Hospital (formerly Miami Children's Hospital) for the treatment of recurrent or progressive malignant brain tumors in children. The protocol involves the administration of intraventricular methotrexate via an Ommaya reservoir, paired with systemic (intravenous) topotecan and cyclophosphamide. Methotrexate is a small molecule antimetabolite that inhibits dihydrofolate reductase, thereby interfering with DNA synthesis. Topotecan acts as a topoisomerase I inhibitor, and cyclophosphamide serves as a DNA alkylating agent. This multi-modal approach aims to deliver high concentrations of chemotherapy directly into the cerebrospinal fluid to target leptomeningeal and intraventricular disease while maintaining systemic control of the primary tumor sites.

Other names

Intraventricular Methotrexate with Topotecan and Cyclophosphamidemethotrexate-Nicklaus Children's Hospital f/k/a Miami Children's Hospital-medulloblastoma-ependymoma-atypical teratoid/rhabdoid tumor-primitive neuroectodermal tumor