Indian-American researcher leads team on potential breakthrough in brain cancer treatment

Dr. Priya Kumthekar of Northwestern University. Photo:

An early clinical trial in individuals with the brain cancer gliobastoma, led by Dr. Priya Kumthekar of Northwestern University, Illinois, showed potential for treatment with an experimental spherical nucleic acid (SNA) drug developed by scientists at the University.

According to a March 10, 2021, report on the university website,, the SNA drug was able to penetrate the blood-brain barrier and trigger the death of tumor cells. Glioblastoma, which affects approximately 13,000 to 15,000 patients annually in the United States, is fatal, noted the report.

This is the first time a nanotherapeutic has been shown to cross the blood-brain barrier when given through intravenous infusion and alter the genetic machinery of a tumor to cause cell death, the news report said.

The Northwestern Medicine study was to be published March 10 in Science Translational Medicine.

“We showed the drug, NU-0129, even at very small doses, causes tumor cells to undergo what’s called apoptosis or programmed cell death,” Dr. Kumthekar, an associate professor of neurology at Northwestern University Feinberg School of Medicine and a Northwestern Medicine physician, is quoted saying in the report. “It’s a remarkable finding in humans that confirms what we had previously seen in our animal studies.”

The first clinical trial was conducted on just 8 individuals who had recurring glioblastoma at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

“In phase 0, investigators use a small dose of medication to make sure it isn’t harmful to humans before they start using it in higher doses in larger clinical trials,” the news report noted.

The news report pointed out that the development of the SNA drug within the Northwestern University framework from the start, through Food & Drug Administration approval, to the current Phase 0 trial, was “highly unusual” without funding from a pharmaceutical company.

“We want to move the technology forward as quickly as possible because there are patients with a disease with no current cure,” Kumthekar said.

Dr. Leon Platanias, director of the Lurie Cancer Center, said, “These exciting findings for the first time support the potential of spherical nucleic acids for drug delivery to brain tumors. They may prove to have important long-term translational implications for the treatment of these tumors.”



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