Mayo Clinic develops nanotherapy to deliver cancer drugs to brain tumors

U****SA—Mayo Clinic researchers have developed an experimental nanotherapy that delivers two cancer drugs to brain tumors, aiming to overcome drug resistance and improve treatment outcomes for patients with glioblastoma.

The approach, described in a study published in Communications Medicine, a Nature Portfolio journal, extended survival in preclinical models when combined with radiation.

Researchers used patient-derived tumor tissue to investigate whether the technology could improve drug delivery and make tumors more responsive to treatment.

Lipid nanoparticles target tumor cells

Glioblastoma is an aggressive brain cancer that remains difficult to treat.

Patients typically survive about 15 months after diagnosis, despite treatment with surgery, radiation and chemotherapy.

The blood-brain barrier restricts the entry of many drugs into brain tissue, while tumor cells can develop resistance to available therapies.

To address these challenges, the researchers developed a delivery system using liposomes, small lipid-based particles that carry therapeutic agents.

The formulation combines everolimus, also known as rapamycin’s derivative, or rapamycin, with vinorelbine to target cancer cells.

By delivering the drugs to the same cells, the researchers aim to strengthen their combined effects while limiting the toxicity associated with higher drug doses.

The treatment targets pathways involved in tumor growth and interferes with cancer cells’ ability to respond to DNA damage, potentially increasing their sensitivity to radiation.

“Glioblastoma remains extremely difficult to treat due to drug resistance and limited drug delivery to the brain,” said Debabrata (Dev) Mukhopadhyay, PhD, a professor of biochemistry and molecular biology at Mayo Clinic in Florida and a senior author of the study.

Mukhopadhyay said the approach seeks to address both challenges by directing treatment towards tumors and combining drugs to enhance their effects.

Further testing needed before clinical trials

Alfredo Quiñones-Hinojosa, MD, dean of research emeritus and chair emeritus of the Department of Neurosurgery at Mayo Clinic in Florida, also contributed to the study as a senior author.

He said the research could inform the development of technologies designed to deliver cancer therapies to the brain and improve outcomes for patients with glioblastoma.

The researchers are conducting additional safety and dosing studies before proceeding to clinical trials.

Depending on the findings, the treatment could eventually be developed for administration by injection or infusion and evaluated alongside standard therapies or for patients whose tumors do not respond to existing treatments.

The development highlights a continuing challenge for oncology drug developers: designing delivery systems that transport therapeutic agents across the blood-brain barrier while maintaining effective concentrations at the tumor site.

Such platforms could also influence how existing cancer drugs are combined and repurposed for brain tumors.

The study received partial support from the US National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, through award R01NS129671.

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