National Cancer Institute 5P50CA221747-08

A Phase 1 Adaptive Dose Escalation Study of Mycophenolate Mofetil in Combination with Temozolomide for Patients with Newly Diagnosed Glioblastoma

A Phase 1 clinical trial studying the safety and efficacy of mycophenolate mofetil combined with temozolomide for newly diagnosed glioblastoma patients, aiming to improve treatment outcomes by overcoming chemoresistance.

Description

This project proposes conducting a Phase 1 clinical trial of mycophenolate mofetil (MMF), an FDA-approved drug from the organ-transplant setting, in combination with standard chemo and radiotherapy for newly diagnosed patients with glioblastoma (GBM). The study aims to evaluate the safety, toxicity, and maximum tolerated dose of MMF combined with standard therapy, and to explore secondary endpoints such as progression-free and overall survival. The research hypothesizes that MMF inhibits IMPDH2 activity, enhancing the efficacy of temozolomide (TMZ) and extending survival in patient-derived xenograft models.

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