Nanotechnology Enhances Cancer Treatment: Chemotherapy Drug Strengthened

Researchers at Northwestern University have achieved a significant breakthrough in cancer treatment by redesigning the molecular structure of the chemotherapy drug 5-fluorouracil, or 5-Fu. This innovative approach has made the drug up to 20,000 times more effective, while also reducing its toxicity.

The study, published in ACS Nano, outlines the use of spherical nucleic acids, or SNAs, which embed the chemotherapy drug directly into DNA strands on tiny spherical structures, enhancing solubility and targeting efficiency.

In animal models of acute myeloid leukemia, a notoriously aggressive form of blood cancer, the SNA-based drug demonstrated a remarkable 12.5 times greater efficiency in entering leukemia cells compared to the standard formulation.

Furthermore, it was able to destroy these cancer cells up to 20,000 times more effectively and slow cancer progression by 59-fold, all without any detectable side effects. Chad A. Mirkin, who led the research, emphasized the importance of improving chemotherapy’s efficacy while minimizing its harsh side effects, a challenge that has long plagued cancer treatments.

Traditional chemotherapy drugs like 5-Fu often face issues with poor solubility, leading to low absorption and significant side effects affecting healthy cells. This new approach transforms the drug into a water-soluble form that can be more effectively delivered to cancer cells.

SNAs are recognized and absorbed by cells, especially myeloid cells, which are overexpressed in leukemia, allowing for targeted therapy. Once inside the cancer cells, the DNA shell is degraded by enzymes, releasing the chemotherapy agent directly where it is needed.

In tests, the SNA therapy nearly eradicated leukemia cells in mouse models, significantly extending their survival rates. The next steps involve further testing in larger animal models before advancing to human clinical trials, pending additional funding.

This work represents a promising advancement in structural nanomedicine, which aims to improve medical treatments by precisely controlling how drugs interact with the body. With multiple SNA-based treatments already in clinical trials, this innovative technique could lead to new therapies not just for cancers but also for infections and autoimmune disorders.

The research received support from the National Cancer Institute and the National Institute of Diabetes and Digestive and Kidney Diseases, as well as the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.