University of North Carolina researchers have developed a combination treatment that shows remarkable promise against glioblastoma, an aggressive brain cancer with dismal survival rates. The therapy paired a standard chemotherapy drug with a laboratory chemical called EdU to produce unprecedented results in preclinical models.
Nobel Prize winner Aziz Sancar led the research and the findings were published in the journal Proceedings of the National Academy of Sciences. His team tested EdU alongside temozolomide, known as TMZ, across multiple glioblastoma models. The concept is straightforward: combining these two compounds can destroy tumors and prevent death, Sancar explained. Together, the drugs demonstrated effects far exceeding what either could achieve alone.
Current treatment options for glioblastomas remain severely limited, with only about 7 percent of patients surviving beyond five years after diagnosis. TMZ represents the sole FDA-approved chemotherapy for this cancer, typically given with radiation therapy. Standard approaches often fail, and tumors frequently return with full aggression after treatment ends. Despite two decades of extensive research, glioblastoma treatment protocols haven’t changed much.
Several factors make glioblastoma exceptionally difficult to treat. The cancer grows rapidly within brain tissue making it difficult for surgeons to remove the tumors completely without damaging critical neural structures. Numerous genetic mutations also drive the disease, eliminating any possibility of a universal approach to glioblastoma treatment.
Sancar’s team discovered last October that a common laboratory chemical called 5-Ethynyl-2′-deoxyuridine or EdU could penetrate brain tissue and kill tumor cells while sparing healthy tissue in actual patient tumors. That finding prompted the current study examining EdU combined with TMZ across three cell lines originally derived from patients. Mouse models carrying U87 tumors showed dramatic differences while untreated subjects died within 30 days.
EdU alone extended survival to just under 45 days. TMZ alone pushed survival to roughly 53 days. Combining 200 mg/kg EdU with 5 mg/kg TMZ eliminated all detectable cancer by day 23, with every treated mouse surviving past 250 days. Similar results appeared with GBM8 cell lines, where all mice receiving combination therapy remained alive and tumor-free after 170 days.
Toxicity studies revealed only mild, reversible changes comparable to typical chemotherapy reactions. The drugs showed true synergism, where combined effects exceeded simple addition, Sancar noted. Tests on living tissue samples removed from patients at UNC Hospitals using a specialized platform called SLiCE (Screening Live Cancer Explants) also showed strong synergy in one of four samples.
Andrew Satterlee, who leads the facility, said the model demonstrates powerful potential for identifying which patients will respond most robustly to specific therapies before treatment begins.
The team now aims for human clinical trials and FDA approval. Graduate student Hümeyra Kaanoğlu notes that patient-specific sensitivities emerged clearly from the study. Personalized combinations could provide desperately needed alternatives for this devastating disease, she said, potentially granting glioblastoma patients a much higher chance of survival post diagnosis.
As companies like CNS Pharmaceuticals Inc. (NASDAQ: CNSP) register successes in their efforts to develop other effective treatments against glioblastoma and other central nervous system cancers, there is hope that these malignancies could soon be as treatable as other conditions.
NOTE TO INVESTORS: The latest news and updates relating to CNS Pharmaceuticals Inc. (NASDAQ: CNSP) are available in the company’s newsroom at https://ibn.fm/CNSP
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