Safety results of completed Phase I clinical trials and pre-clinical trials of CX-02 indicated a striking glioblastoma stem cell-killing effect and the ability of disulfiram + copper to reverse temozolomide resistance. Based on the results of these studies, Cantex is enrolling patients in a Phase II clinical human trial of CX-02 in recurrent glioblastoma at major U.S. cancer centers with top-line results from this clinical trial expected in the second half of 2017. CX-02 has been granted Orphan Drug Designation for glioblastoma by the U.S. Food and Drug Administration.
Glioblastoma, a highly malignant brain tumor, is one of the most lethal cancers. With current therapy, the median survival is approximately 15 months. Among the impediments to successful treatment is a stem-like population of cells, termed brain tumor-initiating cells (BTICs), that are resistant to conventional therapies. It has been discovered that disulfiram + copper, has a very high level of cell-killing effects on patient-derived-BTICs. In addition, patient-derived BTICs, including those resistant to the current glioblastoma front-line chemotherapy, temozolomide, were highly sensitive to disulfiram + copper, and low dose disulfiram + copper significantly enhanced temozolomide cytotoxicity and was capable of reversing temozolomide resistance. Importantly, disulfiram + copper in combination with temozolomide significantly prolonged survival in animals with BTIC implanted directly into the brain. These observations indicate that disulfiram + copper is a potential treatment of glioblastoma.