October 2025

ONCOLOGY

BREAKTHROUGH STRUCTURAL NANOMEDICINE WIPES OUT LEUKEMIA IN ANIMAL STUDY

Featuring: Chad A. Mirkin, PhD
Scientists re-engineered 5-fluorouracil (5-Fu) as a spherical nucleic acid (SNA), weaving it into DNA strands that form nanostructures easily absorbed by cancer cells, which allowed the therapy to destroy the cancer cells from within.

​The Topline Takeaway
In a recent study led by Chad A. Mirkin, PhD, scientists designed a new SNA-construct comprised of short snippets of DNA decorated with 5-Fu. They tested the new therapy in a small animal model of acute myeloid leukemia (AML).

​Compared to the standard chemotherapy drug, the SNA-based chemotherapy drug:
  • Entered leukemia cells 12.5 times more efficiently
  • Killed them over 20,000 times more effectively
  • Reduced cancer progression 59-fold
  • Had no detectable side effects

In the mouse experiments, the therapy eliminated leukemia cells almost completely in the blood and spleen, and it significantly extended survival.  Because the SNAs preferentially targeted myeloid cells, including AML cells, the therapy appeared to spare healthy tissues in animal models, with no detectable side effects.

Behind the Design of Better Drug Delivery
5-Fu often fails to reach cancer cells efficiently and causes a range of side effects.  According to Dr. Mirkin, the problem is how the body processes the drug. Less than 1% of 5-Fu dissolves in many biological fluids. To improve delivery, Dr. Mirkin and his team turned to SNAs.

​Invented and developed by Dr. Mirkin at Northwestern University, SNAs are globular nanostructures composed of a liposomal nanoparticle core surrounded by a dense shell of DNA or RNA. Previous studies found that cells recognize SNAs and pull them in. In the new study, his team built new SNAs with 5-Fu chemically incorporated into the DNA strands.

The structural redesign was a game changer. The myeloid cells actively recognized and internalized the SNA drug. Once inside, enzymes broke down the DNA shell to release the drug molecules, killing the cancer cell from the inside. 

Realizing the Power of Structural Nanomedicine
This work exemplifies the power of structural nanomedicine, an emerging field where scientists use precise structural, as well as compositional, control to fine-tune how nanomedicines interact with the human body. With seven SNA-based therapies in clinical trials, the new approach is leading to potent vaccines and treatments for cancers, infectious diseases, neurodegenerative diseases and autoimmune diseases.

​Dr. Mirkin’s team plans to test the new strategy in a larger cohort of small animal models, then move to a larger animal model and, eventually, in human clinical trials.
This article was originally published in the McCormick School of Engineering on October 29, 2025.
Chad A. Mirkin, PhD, is the director of the International Institute for Nanotechnology and the George B. Rathmann Professor of Chemistry, Chemical and Biological Engineering, Biomedical Engineering, Materials Science and Engineering at Northwestern University. He's also a professor of Hematology and Oncology at Northwestern Medicine and a member of Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

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