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Northwestern Drug Kills Glioblastoma Tumor Cells with Priya Kumthekar, MD

An early clinical trial found that a spherical nucleic acid drug developed at Northwestern kills tumor cells in people with the fatal brain cancer glioblastoma. This is the first time a nanotherapeutic has been shown to cross the blood-brain barrier and cause cell death. Lead investigator Priya Kumthekar, MD, explains the results of the study.


Dr. Priya Kumtheker

"I think we can change the way tumors behave by delivering genetic material to them that will influence their behavior. Beyond that, I'm excited to see that we have therapeutics that can reach the brain—that could have a plethora of implications, including in the world of degenerative diseases."

Priya Kumthekar, MD

  • Associate Professor of Neurology in the Division of Neuro-oncology 
  • Associate Professor of Medicine in the Division of Hematology and Oncology
  • Member of the Robert H. Lurie Comprehensive Cancer Center at Northwestern University
  • Member of Northwestern University Clinical and Translational Sciences Institute

Episode Notes

In the past decade, no new drugs have been approved for the fatal brain cancer glioblastoma. But a team of Northwestern scientists are eager to change that with a new drug, NU-0129, designed at Northwestern.

Priya Kumthekar, MD, led the phase 0 study in eight patients with recurrent glioblastoma at the Robert H. Lurie Comprehensive Cancer Center. Patients received the drug intravenously before surgery. Kumthekar and her team then studied patients' tumors and found that a low dose of the drug caused tumor cells to undergo apoptosis, or programmed cell death.

Other topics covered:

  • Kumthekar explains how the drug, invented at Northwestern’s International Institute for Nanotechnologyuses a novel spherical nucleic acid (SNA) platform. Resembling a Koosh ball, the nanoparticle contains a gold core surrounded by SNA, spirals of genetic material. Once the particle passes into the tumor, the genetic material affects the behavior of the tumor cells.
  • "The sky is the limit," Kumthekar says of the nanotechnology. Because of its ability to cross the blood-brain barrier, the technology shows potential for the treatment of other diseases such as Alzheimer's or Parkinson's disease.
  • This is the first time in Northwestern's history that a drug that began as an initial concept in the lab was carried through pre-clinical research, FDA approval and into clinical trials, all within the university.

Additional Reading and Resources: 

  • Read the study, "A first-in-human phase 0 clinical study of RNA interference–based spherical nucleic acids in patients with recurrent glioblastoma," in Science Translational Medicine.
  • Read earlier pre-clinical research on the nanotherapeutic technology by the Northwestern team.

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Recorded on March 8, 2021.

Continuing Medical Education Credit

Physicians who listen to this podcast may claim continuing medical education credit after listening to an episode of this program.

Target Audience

Academic/Research, Multiple specialties

Learning Objectives

At the conclusion of this activity, participants will be able to:

  1. Identify the research interests and initiatives of Feinberg faculty.
  2. Discuss new updates in clinical and translational research.
Accreditation Statement

The Northwestern University Feinberg School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Credit Designation Statement

The Northwestern University Feinberg School of Medicine designates this Enduring Material for a maximum of 0.25 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Disclosure Statement

Priya Kumthekar, MD, receives a consulting fee from Orbus, Biocept and Elevate Bio. Kumthekar also conducts contracted research at Novocure, Genentech, Inc. and Orbus. Content reviewer, Irina Balyasnikova, PhD, has nothing to disclose. Course director, Robert Rosa, MD, has nothing to disclose. Planning committee member, Erin Spain, has nothing to disclose. Feinberg School of Medicine's CME Leadership and Staff have nothing to disclose: Clara J. Schroedl, MD, Medical Director of CME, Sheryl Corey, Manager of CME, Allison McCollum, Senior Program Coordinator, Katie Daley, Senior Program Coordinator, and Rhea Alexis Banks, Administrative Assistant 2.

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