Researchers at Northwestern University have introduced a novel method to significantly enhance the efficacy of the chemotherapy drug 5-fluorouracil (5-Fu) by encapsulating it in spherical nucleic acids (SNAs). This innovative formulation has been shown to be 20,000 times more effective against leukemia cells while substantially minimizing side effects.
5-Fu is a well-established cancer treatment but often faces limitations due to its poor solubility and associated side effects impacting healthy tissues. By embedding 5-Fu within SNAs—nanostructures that contain a dense shell of DNA or RNA encasing a nanoparticle—the team has improved its uptake by cancer cells, ensuring more effective delivery directly to tumor sites and reducing damage to normal cells.
In studies involving animal models with acute myeloid leukemia (AML), the SNA-enhanced 5-Fu exhibited a 12.5-fold increase in cellular absorption compared to the standard formulation. Additionally, it achieved a remarkable 20,000-fold enhancement in cancer cell destruction and a 59-fold decrease in cancer progression, all without causing detectable side effects. These encouraging results demonstrate the potential of SNAs to transform cancer treatment through more precise and powerful therapeutic options.
This research is part of the burgeoning field of structural nanomedicine, which focuses on the careful design of nanomedicines to optimize their interaction with the body. With seven SNA-based therapies already undergoing clinical testing, there is significant optimism that this approach could lead to breakthroughs in treatments for various conditions, including cancers, infections, neurodegenerative disorders, and autoimmune diseases.
The findings were published in the journal ACS Nano on October 29, 2025, under the leadership of Chad A. Mirkin, a prominent professor and director of the International Institute for Nanotechnology at Northwestern. The research team plans to further explore this treatment in additional animal studies before progressing to human clinical trials.
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