Mycophenolate Mofetil Combined With Standard of Care Chemoradiation in Newly Diagnosed Glioblastoma

Brain Cancer
Yoshie Umemura, MD
University of Michigan


Glioblastoma (GBM) is the most common and aggressive brain tumor in adults. Despite initial aggressive treatment with surgery followed by radiation therapy and chemotherapy, most patients relapse within one year, develop treatment resistance, and less than 10% live longer than five after diagnosis. Thus, overcoming treatment resistance in GBM will improve the effectiveness of treatment and prolong survival. Furthermore, the majority of the recurrences occur within the initial radiation treatment area. Preclinical studies have demonstrated that GTP, a building block of DNA, protects GBM tumor cells from radiation therapy. Conversely, depleting GTP slows the tumor from repairing radiation therapy-induced tumor DNA damage. Mycophenolate mofetil (MMF) is an FDA-approved, widely available, and inexpensive drugs that inhibits GTP synthesis. Other research suggests that MMF may also improve the effectiveness of temozolomide, a chemotherapy used for brain tumors. This phase I study will determine the maximum tolerated dose of MMF when combined with radiation therapy and temozolomide in patients with newly diagnosed GBM.

Trial Registration: Identifier: NCT04477200