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New Drug Delivery Trial Using Spherical Nucleic Acids and Nanoparticles Provides Hope for Glioblastoma Patients

Susanna Sisson's picture
Spherical Nucleic Acid Gold Nanoparticle

A new classification of drugs, spherical nucleic acids attached to nanoparticles are making headlines and may provide hope for glioblastoma multiforme (GBM) patients as Phase 0 clinical trials begin. The drug is the brain child of researchers at Northwestern University who are collaborating with other scientists at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and Northwestern Medicine to study the response in humans.


“Glioblastoma affects about 30,000 people in the US every year and it is incurable with an expected lifespan of 16-18 months,” says lead researcher Dr. Priya Kumthekar, M.D. GBM is rare and particularly difficult to treat. Currently the only patient options are surgery, chemotherapy and radiation which all have potential catastrophic effects.

This novel drug, NU-1029, targets the gene BCL2L12 which is involved in apoptosis or cell death and changes the genetic makeup of the tumor cells to stop them from dividing. Unlike normal cells, tumor cells do not undergo apoptosis, which allows them to grow uncontrollably. What is unique is about NU-1029 is the 3D spherical structure of densely attached short strands of RNA to a gold carrier nanoparticle. This unique shape not only crosses the blood brain barrier in mice but is non-toxic as opposed to the linear arrangement.

“The spherical shape of the drug allows it to cross the blood brain barrier carried by gold nanoparticles,” says Alexander Stegh Assistant Professor of Neurology Feinberg School of Medicine Northwestern, “and targets the gene within glioblastoma tumor to “turn down” genes that are at an elevated level.”

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Chad A. Mirken, Director of the International Institute for Nanotechnology and the George B. Rathmann Professor of Chemistry at the Weinberg College of Arts and his group invented spherical nucleic acids. Alexander Stegh was responsible for identifying the gene to target.

In a 2013 animal study co-authored by Mirken and Stegh, it was discovered that spherical nucleic acids once they reach the brain have a selective preference for tumor cells over normal cells perhaps due to leaky vessels damaged by the tumors. They discovered using this drug delivery system reduced tumor progression and drastically increased survival rates.

“If you combine nanotechnology with gene technology,” says Mirken, “you can selectively knock down genes that are at elevated levels in tumor cells you can make those cells more prone to cell damage or apoptosis or apply chemotherapeutics. The ability to get these drugs across the blood-brain barrier is really important in terms of treating not only diseases like glioblastoma but we can begin to think about treating all sorts of other neurodegenerative diseases including Parkinson’s, Alzheimer’s disease, maybe even Huntington’s disease.”

What is really unusual about this project is the unique collaboration between departments within Northwestern and that the drug is not being funded by a pharmaceutical company. “Without this unique collaboration,” says Jay Walsh, Northwestern University Vice President for Research, “and without that unique collaboration we would not have this technology moving so quickly from the laboratory into clinical use. We want to get the drug to patients as quickly as possible…to move the drug forward because there are patients with a disease with no current cure."