Nano Drug Crosses Blood-Brain Tumor Barrier, Targets Brain-Tumor Cells and Blood Vessels

Balveen KaurAn experimental drug in early development for aggressive brain tumors can cross the blood-brain tumor barrier, kill tumor cells and block the growth of tumor blood vessels, according to a study led by OSUCCC – James researchers.

The laboratory and animal study also shows how the agent, called SapC-DOPS, targets tumor cells and blood vessels. The findings support further development of the drug as a novel treatment for brain tumors.

SapC-DOPS (saposin-C dioleoylphosphatidylserine), is a nanovesicle drug that has shown activity in glioblastoma, pancreatic cancer and other solid tumors in preclinical studies.The nanovesicles fuse with tumor cells, causing them to self-destruct by apoptosis. Glioblastoma multiforme is the most common and aggressive form of brain cancer, with a median survival of about 15 months. A major obstacle to improving treatment for the 3,470 cases of the disease that were expected in the United States last year is the blood-brain barrier, which protects the brain from toxins in the blood but also keeps drugs in the bloodstream from reaching brain tumors.

“Few drugs have the capacity to cross the tumor blood-brain barrier and specifically target tumor cells,” says principal investigator Balveen Kaur, PhD, professor and vice chair for Research of Neurological Surgery.

Kaur and her colleagues showed that SapC-DOPS does both and inhibits the growth of new tumor blood vessels, suggesting that the agent might one day be an important treatment for glioblastoma and other solid tumors, Kaur says. In addition, the agent sensitized hypoxic cells to killing by conventional treatment.

The findings, Kaur says, suggest that SapC-DOPS could have a synergistic effect when combined with chemotherapy or radiation therapy.

Published in the journal Molecular Therapy.

NIH/National Cancer Institute grants CA158372, CA136017, CA136017 and CA171733 supported this research.

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