08/03/2026 | Mayo Clinic

https://www.newswise.com/articles/nanoterapia-experimental-com-dois-f-rmacos-da-mayo-clinic-atravessa-a-barreira-hematoencef-lica-e-melhora-a-sobrevida-em-modelos-pr-cl-nicos-de-glioblastoma 

Mayo Clinic researchers have developed an experimental nanotherapy that delivers two cancer drugs directly to brain tumors, according to a study published in *Nature Communications Medicine*. The strategy extended survival in preclinical models of glioblastoma, the most aggressive form of brain cancer.

The nanotechnology-based approach encapsulates two existing cancer drugs within tiny particles designed to cross the brain's protective blood-brain barrier and reach tumor cells. In preclinical models using patient-derived tissue, combining the treatment with radiation therapy more than doubled survival compared to untreated controls.

Glioblastoma is notoriously difficult to treat. Patients typically survive about 15 months after diagnosis, even with state-of-the-art therapies such as surgery, radiation, and chemotherapy. A major challenge is that many drugs cannot effectively reach brain tumors, and those that do often lose their effectiveness as tumors develop resistance.

The new approach uses small lipid-based particles, known as liposomes, to transport and deliver a drug combination—everolimus or rapamycin and vinorelbine—directly to cancer cells, employing a novel tumor-targeting strategy. By ensuring that both drugs reach the same cells simultaneously, researchers aim to enhance the anti-tumor effect while reducing the toxic side effects associated with higher doses.

"Glioblastoma remains extremely difficult to treat due to drug resistance and the limited ability to deliver drugs to the brain," says Debabrata (Dev) Mukhopadhyay, Ph.D., a professor of biochemistry and molecular biology at Mayo Clinic in Florida. Dr. Mukhopadhyay, a nanotechnologist, is the study's senior author. "Our approach was designed to improve both aspects by targeting the treatment directly to the tumor and combining therapies to maximize their impact."

The drug combination includes agents that interfere with tumor growth pathways and impair the cancer's ability to repair DNA damage, thereby making the tumors more sensitive to radiation therapy.

"This represents a promising direction for treating glioblastoma patients and advancing new technologies and therapies, so that we may one day improve survival rates for brain cancer patients by delivering innovative cancer therapies to the brain," says Alfredo Quiñones-Hinojosa, M.D., Dean of Research Emeritus and Chair Emeritus of the Department of Neurosurgery at Mayo Clinic in Florida, who is also a senior author of the study. "Further research will be needed to determine whether these results translate into patient benefits."

The researchers are conducting additional safety and dose-finding studies required before clinical trials can begin. If successful, the approach could eventually lead to an oral or intravenous drug used alongside standard treatments or as an option for patients whose tumors do not respond to existing therapies. "Although 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 patients' quality of life," says Dr. Mukhopadhyay.

This study was partially funded by the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, under grant number R01NS129671. Please refer to the study for the full list of authors, disclosures, and funding sources.

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