Mayo Clinic researchers announced on Apr. 6 the development of an experimental nanotherapy designed to deliver two cancer drugs directly to brain tumors, according to a study published in Communications Medicine. The new approach was shown in preclinical models to extend survival for glioblastoma, which is considered the most aggressive form of brain cancer.
The significance of this research lies in its potential to address the major challenge of treating glioblastoma, a disease where patients typically survive about 15 months after diagnosis despite current treatments such as surgery, radiation, and chemotherapy. Many existing drugs cannot effectively reach tumors in the brain due to the protective blood-brain barrier, and those that do often lose effectiveness as tumors become resistant.
Researchers used tiny lipid-based particles called liposomes to package two existing cancer drugs—everolimus or rapamycin and vinorelbine—and engineered them so they can cross into the brain and target tumor cells directly. In studies using patient-derived tissue models, combining this treatment with radiation more than doubled survival compared with untreated controls.
"Glioblastoma remains extremely difficult to treat due to drug resistance and limited drug delivery to the brain," said Debabrata (Dev) Mukhopadhyay, Ph.D., professor of biochemistry and molecular biology at Mayo Clinic in Florida and senior author of the study. "Our approach is designed to improve both by targeting the tumor directly and combining therapies in a way that enhances their impact." The therapy aims not only for improved tumor-killing effects but also for reduced toxic side effects by ensuring both drugs reach cancer cells simultaneously.
The drug combination works by interfering with pathways involved in tumor growth while disrupting cancer cells' ability to repair DNA damage—making them more sensitive to radiation therapy. Additional safety and dosing studies are underway before clinical trials can begin.
"While this work is still in development, it represents an important step toward developing more precise cancer treatments that are both more effective and less toxic, potentially improving quality of life for patients," Mukhopadhyay said.
