Nano drug crosses blood-brain tumor barrier, targets brain tumors

Potential treatment for Glioblastoma multiforme brain cancer
July 22, 2013

Glioblastoma(credit: Huilin Shao et al./Nature Medicine)

An experimental drug called SapC-DOPS, in early development for aggressive brain tumors, can cross the blood-brain barrier.

There, it can kill tumor cells and block the growth of tumor blood vessels, according to a study led by researchers at the Ohio State University Comprehensive Cancer Center at Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James).

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

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 expected in the United States this year is the blood-brain barrier, the barrier that 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, associate professor of neurological surgery and chief of the Dardinger Laboratory of Neurosciences at the OSUCCC – James. “Our preclinical study indicates that SapC-DOPS does both and inhibits the growth of new tumor blood vessels, suggesting that this agent could one day be an important treatment for glioblastoma and other solid 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.

“Based on our findings, we speculate that SapC-DOPS could have a synergistic effect when combined with chemotherapy or radiation therapy, both of which are known to increase the levels of exposed PtdSer on cancer cells,” Kaur says.

Funding from the NIH/National Cancer Institute and a New Drug State Key Project grant helped support this research. University of Cincinnati scientists were also involved in the research.